Disease of genetic or genomic mechanism

This trial tests an experimental therapy called PM577a, which uses prime editing to correct a common genetic mutation that causes Wilson disease. The treatment is given as a single intravenous infusion and aims to restore the liver's ability to remove excess copper. The study inc…

This early-stage trial is investigating whether a new type of CAR T-cell therapy, which targets a protein called BAFFR on cancer cells, can safely and effectively treat adults with B-cell acute lymphoblastic leukemia or B-cell lymphoblastic lymphoma that has returned or not respo…

This trial tests a single injection of GEB-101, a gene-editing treatment, directly into the cornea of people with TGFBI-related corneal dystrophy, a hereditary condition that causes progressive vision loss. The study aims to see if the treatment is safe and whether it can slow or…

This early-phase trial tests a single injection of ASP2020, stem cells turned into retinal cells, placed under the retina in people with Stargardt disease or similar inherited macular dystrophies. The main goal is to check safety and tolerability, while also looking for signs tha…

This phase 2 trial tests whether a single injection of EO2002, made from magnetic human corneal endothelial cells, can safely improve vision in people with corneal edema caused by endothelial dysfunction. Participants receive either a low or high dose of cells or a placebo inject…

This phase 2 trial is testing whether a single infusion of a patient's own genetically modified immune cells, called C-CAR168, can safely and effectively treat lupus nephritis that has not responded to standard therapies. The cells are designed to target and eliminate specific im…

This phase 3 trial tests whether an experimental drug called KP-100IT, injected into the spinal fluid, can help people with severe acute spinal cord injuries regain function. Participants, aged 18 to 89 with a complete loss of motor and sensory function (AIS grade A), receive fiv…

This phase 2 trial tests a one-time gene therapy called HGI-001 in people with transfusion-dependent β-thalassemia, a severe blood disorder that requires regular blood transfusions. The therapy takes the patient's own blood stem cells, modifies them outside the body to produce he…

This study tests whether bone marrow from deceased donors can be used to treat people with blood cancers like leukemia and lymphoma. Participants receive a transplant of cryopreserved marrow from a deceased donor or standard cells from a living donor. The goal is to see if the de…

This pilot trial tests a gene-editing approach for sickle cell disease. Participants receive their own blood stem cells that have been edited to increase fetal hemoglobin, a healthy type of hemoglobin that prevents sickling. The goal is to cure the disease with a one-time treatme…

This trial is testing whether an experimental mRNA vaccine can safely reduce moderate-to-severe acne in adolescents aged 12 to 17. Participants receive either the vaccine at different dose levels or a placebo, and researchers monitor safety and changes in facial acne lesions. The…

This pilot trial tests a procedure called FETO for fetuses with severe congenital diaphragmatic hernia (CDH), a condition where the diaphragm has a hole and the lungs don't develop fully. In FETO, a balloon is temporarily placed in the fetal windpipe to encourage lung growth, the…

This trial tests whether combining two non-invasive brain stimulation techniques—transcranial direct current stimulation (tDCS) and transcranial ultrasound stimulation (TUS)—can improve balance and posture in people with Parkinson's disease. Participants are randomly assigned to …

This trial tests whether the LapBox Manual Containment System, a double-wall bag used during laparoscopic surgery, can prevent leaks when removing uterine fibroids. About 30 women having laparoscopic hysterectomy or myomectomy will use the device, and surgeons will check for any …

This study tests whether adjusting chemotherapy doses for each patient can improve outcomes after a stem cell transplant for blood cancers caused by a RUNX1 gene mutation. Up to 98 people aged 4 to 70 with these cancers will receive a transplant from a half-matched donor. The goa…

This study tests a radioactive drug called 177Lu-DOTA-EB-TATE in 18 adults with a rare thyroid cancer (Hurthle cell) that has spread and not responded to standard radioactive iodine treatment. The drug is designed to seek out and attach to cancer cells. Participants will receive …

This study tests whether red blood cells processed to be low in oxygen (hypoxic) last longer in the body than standard cells for people with sickle cell anemia who need regular transfusions. About 48 participants aged 7 and older will receive both types of blood in a crossover de…

This Phase 3 trial is testing whether a microneedle pad delivering botulinum toxin A can improve symptoms of Raynaud's phenomenon and digital ulcers in people with systemic sclerosis. The study compares the microneedle treatment to a placebo, measuring changes in finger temperatu…

This clinical trial is testing an experimental oral medication called AC-101 in adults with moderately to severely active ulcerative colitis (UC), a chronic inflammatory bowel disease. The study aims to see if AC-101 can help achieve clinical remission—meaning fewer symptoms and …

This early trial is testing whether a new investigational drug called HX15001, given as an injection, can be safely combined with an existing pill (etrasimod) to treat moderate-to-severe ulcerative colitis. The study will include about 32 adults with the condition and will focus …

This trial tests whether rusfertide, a synthetic version of the hormone hepcidin, can help people with polycythemia vera—a condition where the bone marrow makes too many red blood cells—by lowering iron availability and keeping red blood cell counts in check. The study enrolls Ch…

This phase 3 trial is testing whether a radioactive drug called iopofosine I 131 can keep Waldenstrom macroglobulinemia (a rare blood cancer) from getting worse for longer than the standard combination of rituximab, cyclophosphamide, and dexamethasone (R-CD). The study enrolls ab…

This trial tests whether an oral medication called zasocitinib (TAK-279) can reduce the redness, thickening, scaling, and painful cracks of palmoplantar psoriasis, a type of psoriasis that affects the palms and soles. Adults with moderate-to-severe non-pustular palmoplantar psori…

This phase 2 trial tests whether adding the drug epcoritamab to standard chemotherapy (DA-EPOCH-R) helps people with high-risk Burkitt lymphoma. About 43 adults will receive the combination. The study will check how many complete at least three cycles of treatment and track overa…

This phase 2 trial is testing whether combining two immunotherapy drugs—vilastobart and retifanlimab—can shrink tumors in people with metastatic pancreatic cancer that has a BRCA1, BRCA2, or PALB2 gene mutation. Participants receive both drugs by IV infusion over several months. …

This phase 2 trial is investigating whether adding a ketogenic diet and/or high-dose intravenous vitamin C to standard chemoradiotherapy and immunotherapy can improve outcomes for patients with locally advanced rectal cancer that is mismatch repair proficient (pMMR/MSS). The stud…

This trial is testing an experimental eye gel called BETA-TT8 for people with wet age-related macular degeneration (AMD), a condition that causes rapid vision loss. The gel is applied to the eye up to three times daily for 12 weeks, and standard injections remain available if nee…

This early-stage trial tests an oral capsule containing a genetically modified probiotic yeast designed to neutralize toxins produced by C. diff bacteria. The study first evaluates safety in healthy adults, then in people at risk for recurrent C. diff infection. Participants take…

This trial tests a non-surgical treatment for advanced Parkinson's disease that uses focused ultrasound to create a tiny lesion in a brain pathway linked to motor symptoms. It compares treating one side of the brain versus both sides, against standard medication. The goal is to s…

This Phase 3 trial tests whether vatiquinone can slow the progression of Friedreich's ataxia, a rare genetic disorder that damages the nervous system and impairs movement. About 120 adults with moderate symptoms will take the drug for 24 months. Researchers will measure changes i…

This phase 2 trial is testing whether an experimental oral drug called HS-20118 can improve moderate to severe plaque psoriasis, a condition that causes thick, scaly patches on the skin. About 120 adults with the condition will receive either HS-20118 or a placebo once a week for…

This study tests whether an investigational drug called tilrekimig, given as injections alongside medicated creams or ointments, can improve moderate-to-severe eczema (atopic dermatitis) in people aged 12 and older. Participants will be randomly assigned to receive either tilreki…

This trial investigates whether combining two behavioral therapies can help people with non-fluent aphasia—a condition where stroke damages the brain's language centers, making speech difficult. The approach uses Melodic Intonation Therapy (MIT), which uses singing-like intonatio…

This trial tests whether icosapent ethyl, a purified form of fish oil, can stabilize high-risk coronary plaques in people with heart disease. Participants have plaques that narrow a major heart artery by 30–70% and show at least one high-risk feature on a CT scan. Half will take …

This phase 2 trial tests the safety of belzutifan, a daily oral tablet, in adolescents with rare solid tumors that cannot be surgically removed or have spread. The study includes three tumor types: pheochromocytoma/paraganglioma (PPGL), wild-type gastrointestinal stromal tumors (…

This trial tests whether aspirin and a non-invasive physical therapy called ischemic conditioning can slow the progression of moyamoya disease, a rare brain vessel disorder that can cause strokes and cognitive decline. The study enrolls adults with moyamoya disease or syndrome wh…

This trial tests whether a calcium-rich food chew, made from milk-derived minerals, is easier for women to take daily than a standard calcium carbonate pill. Women aged 40 to 90 at increased risk for osteoporosis will be randomly assigned to one of the two forms for four weeks. T…

This pilot trial tests whether tirzepatide, a weekly injection used for diabetes and weight loss, can treat active central centrifugal cicatricial alopecia (CCCA), a permanent scarring hair loss condition. Twenty adults with active CCCA will inject tirzepatide for 12 months and u…

This study tests a 6-month program where patients work with a pharmacist and their primary care doctor to make shared decisions about medications and lifestyle. The goal is to see if this team-based approach improves heart and kidney health scores and medication adherence. The st…

This Phase 1 trial is testing an experimental oral drug called QLS1510, which is being developed for inflammatory bowel disease (IBD). The study enrolls healthy adults aged 18 to 45 to evaluate the drug's safety, tolerability, how it moves through the body, and the effect of food…

This clinical trial is testing an experimental medication called TJ0113 in people with early-onset Parkinson's disease. The study aims to see if TJ0113 can improve movement symptoms compared to a placebo. About 300 participants will take either TJ0113 or a placebo for 26 weeks, f…

This Phase 2 trial tests a combination of two drugs—blinatumomab (given as a shot under the skin) and olverembatinib (a pill)—in 60 adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL). The goal is to see if this combo can control the disease safely. Pa…

This study tests a new way to treat a fast-growing blood cancer called BCR-ABL positive B-cell acute lymphoblastic leukemia. About 80 adults will receive two drugs: ponatinib pills and blinatumomab injections under the skin. The goal is to see if this combination can control the …

This study tests a combination of the immunotherapy drug pembrolizumab and three chemotherapy drugs (GVD) in 38 adults whose Hodgkin lymphoma returned or did not respond to one prior treatment. A special blood test (ctDNA) is used to see how well the treatment is working and to g…

This trial tests whether a low-sodium diet can improve blood pressure control in people who had a hypertensive disorder of pregnancy. Participants receive either a provided diet with less than 2000 mg of sodium per day or a standard diet with no salt restrictions for the first we…

This study will test whether living in a self-contained, AI-managed community (called a Microcity) can improve mental health and reduce addiction in adults with conditions like autism, ADHD, substance use disorders, and behavioral addictions. Up to 1 million participants will liv…

This trial explores whether transplanting gut bacteria from healthy donors can help restore the immune system in people who have weak immunity after a stem cell transplant. The study first observes immune recovery in patients to identify those with severe deficiency, then gives a…

This study looks at whether switching to a higher-dose eye injection called aflibercept 8 mg can allow people with neovascular age-related macular degeneration (nAMD) or diabetic macular oedema (DME) to go longer between treatments. It follows adults in Portugal who have already …

This study follows people with bone loss caused by long-term steroid use to compare two treatment approaches. One group received romosozumab for the first year and then switched to denosumab, while the other group took denosumab throughout. After six years, researchers will measu…

This study tests whether a traditional Chinese herb called Polygonatum sibiricum, or Huangjing, can help reduce visceral fat—the deep belly fat around organs that raises risks for diabetes, fatty liver, and heart disease. Adults with visceral obesity will receive either the herb …

This trial is testing an investigational drug called brenipatide in adults with moderate-to-severe COPD. Participants will receive either brenipatide or a placebo for about a year, alongside standard care. The goal is to see if brenipatide improves lung function and reduces the n…

This pilot study tests whether adding extra doses of the antibiotic azithromycin to standard care can improve outcomes for pregnant people whose water breaks very early (between 22 and 28 weeks). Thirty participants will be randomly assigned to receive either the standard antibio…

This phase 2 trial is testing whether an investigational drug called SL-325 can help adults with moderate to severe Crohn's disease. The study will compare SL-325 against a placebo to see if it reduces gut inflammation and improves symptoms. Participants will receive the treatmen…

This early-stage trial is testing an experimental oral medication called HRS-7085 in adults with moderate to severe ulcerative colitis, a chronic inflammatory bowel disease. The main goal is to see if the drug is safe and tolerable, and to understand how it moves through the body…

This phase 1b trial tests an experimental drug called ST316 in adults with familial adenomatous polyposis (FAP) who have already had their colon removed but still develop new polyps. The study aims to see if ST316 is safe and can reduce the number and size of polyps that grow bac…

This trial investigates whether guanfacine or cromolyn sodium—two drugs already approved for other conditions—can improve physical functioning and reduce symptoms like fatigue, brain fog, and gastrointestinal issues in adults with postural orthostatic tachycardia syndrome (POTS).…

This early study tests a new drug called HN2302, which aims to create CAR-T cells inside the body to target and control autoimmune diseases like lupus and scleroderma. The trial will enroll 12 adults to check safety and see if the treatment works. It is a first step, so results w…

This trial tests whether an experimental drug called ESG206 can safely reduce disease activity in adults with systemic lupus erythematosus (SLE). Participants will receive either ESG206 or a placebo, and researchers will measure how many achieve a meaningful improvement in lupus …

This early-stage trial tests a new cell therapy called Rincell-1 for adults with certain types of hearing loss who are getting a cochlear implant. The study will compare the safety and early effectiveness of receiving the cell therapy plus the implant versus the implant alone. Ab…

This study tests the long-term safety and tolerability of acoramidis, an oral medication that stabilizes the transthyretin protein, in people newly diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition that can lead to heart failure. Participants who complete…

This pilot study explores whether a gentle manual therapy applied to the hip bone, in addition to standard treatment with a Pavlik harness, can improve hip joint coverage in infants with developmental dysplasia of the hip (DDH). Thirty infants aged 1 to 5 months with moderate to …

This trial is testing a new device called MRace to repair a leaky mitral valve in people with severe functional mitral regurgitation, a condition where the valve doesn't close properly, causing blood to flow backward. The device is placed through a vein in the leg and guided to t…

This study tests a new device called MRace, designed to repair severe mitral regurgitation—a condition where the heart's mitral valve doesn't close properly, causing blood to leak backward. The device is implanted through a vein in the leg, avoiding open-heart surgery, and is aim…

This phase II trial is testing an investigational drug called SHR-1139 in adults with plaque psoriasis who have not responded well to at least one biologic treatment. Participants will receive either SHR-1139 or a placebo via subcutaneous injection over a 52-week treatment period…

This study tests whether combining two drugs, inotuzumab and blinatumomab, is safe and effective for adults newly diagnosed with B-cell acute lymphoblastic leukemia (B-ALL). The trial includes 26 people aged 18 and older who are either 55+ or cannot tolerate standard chemotherapy…

This study tests whether eating a daily serving of pecans for 12 weeks can improve cholesterol levels and other markers of heart-disease risk. Adults aged 25–75 who are overweight or have borderline-high cholesterol will either add pecans to their diet or stick with their usual e…

This trial tests whether adding the immunotherapy drugs cemiplimab (with or without fianlimab) to standard chemotherapy before chemoradiation can improve outcomes for people with advanced nasopharyngeal cancer. About 60 adults with non-metastatic, EBV-positive nasopharyngeal carc…

This trial tests whether applying a platelet-rich fibrin (PRF) membrane during hypospadias repair surgery improves healing and reduces complications like fistulas. PRF is made from the patient's own blood and is thought to support tissue repair. The study will include 80 boys wit…

This study tests a new device called MRace, designed to repair a leaky mitral valve in the heart without open surgery. The device is placed through a vein in the leg and guided to the heart to help the valve close properly. The trial will include up to 15 people with severe mitra…

This trial explores whether a single dose of psilocybin, combined with motivational support therapy, can help people with HIV who have moderate-to-severe methamphetamine use disorder reduce or stop their meth use. Participants will be randomly assigned to receive either a higher …

This trial evaluates Paragon CRT contact lenses with a 5mm optic zone diameter, worn overnight to temporarily reshape the cornea and correct myopia and astigmatism. Participants aged 6 to 35 will wear the lenses daily for a short period, and their comfort and visual performance w…

This trial tests whether a daily routine of tummy time and careful positioning can help correct cranial asymmetry—a flattened head shape—in premature newborns. About 300 babies born before 37 weeks who are in the neonatal ICU or intermediate care unit will be split into two group…

This trial tests whether an experimental cell therapy called agenT-797 is safe for people receiving a stem cell transplant for blood cancers like leukemia or myelodysplastic syndrome. Participants get an infusion of agenT-797 about a week after their transplant. The main goal is …

This early-stage trial is testing an experimental drug called SHR-7590 to see if it is safe and tolerable when given by injection under the skin or into a vein. It involves healthy volunteers and people with moderate-to-severe ulcerative colitis, a condition that causes inflammat…

This trial tests whether tirzepatide (Zepbound) can reduce daily bowel frequency in adults with an ileal pouch-anal anastomosis (IPAA) who have chronic high bowel frequency despite standard anti-diarrheal therapy. Participants will be assigned to receive tirzepatide or standard c…

This early-stage trial is testing whether a single infusion of universal STAR-T cells—a type of engineered immune cell that targets both CD19 and BCMA—can safely and effectively treat two autoimmune kidney diseases: IgA nephropathy and primary membranous nephropathy. The study wi…

This pilot trial is asking whether emergency abdominal surgery for newborns with a serious gut condition called necrotising enterocolitis should be done in the operating theatre or right on the neonatal unit. It will compare outcomes like stability during surgery, wound infection…

This study looks at how well the drug asciminib works and how safe it is for young adults with chronic myeloid leukemia (CML) in the Gulf region. Researchers will collect information from medical records of about 80 people aged 18 and older who have taken asciminib. The main goal…

This trial tests whether different intermittent fasting schedules—5:2, alternate-day, and time-restricted eating—can improve blood sugar, cholesterol, inflammation, and body composition in adults with prediabetes. Participants will be randomly assigned to one of three fasting pla…

This early-stage trial tests an experimental oral drug called SNV1521, a PARP inhibitor, combined with the RAS-targeting medicine daraxonrasib in adults with pancreatic cancer that cannot be surgically removed or has spread. The main goals are to find safe and tolerable doses of …

This study follows people with moderate to severe pulmonary regurgitation or stenosis who receive the PULSTA transcatheter pulmonary valve, a less invasive option for replacing a faulty heart valve. Researchers will track how well the valve works over time, measuring improvements…

This trial tests a new device called VividFlo, a small implant placed under the eye's surface to help drain fluid and lower pressure in people with glaucoma. The study involves 10 adults with glaucoma in one eye, who will receive the implant during surgery. Researchers will track…

This study tests NeoMayor, a smartphone app that gives personalized advice on exercise, diet, sleep, and mood to improve heart health. Researchers will enroll 240 adults aged 55 to 75 in Chile who are at risk for heart problems. Participants use the app for 4 months, and some als…

This phase 2 trial is testing whether an oral drug called flonoltinib maleate can help adults with myelofibrosis, a rare bone marrow disorder that often causes an enlarged spleen and severe symptoms. The study compares three different daily doses (50 mg, 75 mg, and 100 mg) to see…

This phase III trial is testing whether regular antibody infusions (immunoglobulin replacement therapy) can prevent infections better than standard antibiotic prophylaxis in people who have received CAR-T cell therapy for B-cell acute lymphoblastic leukemia or B-cell lymphoma. CA…

This trial tests whether adding a chemical injection to microwave ablation improves treatment for uterine fibroids, common non-cancerous tumors that can cause heavy bleeding and pain. Microwave ablation uses heat to destroy fibroid tissue, but blood flow can cool the area and red…

This phase 2 trial tests an experimental drug called KER-065 in adult and pediatric males with Duchenne Muscular Dystrophy (DMD), a genetic condition that causes progressive muscle weakness. The study includes both those who can walk and those who cannot, and all participants mus…

This study asks whether classifying people with carpal tunnel syndrome into different pain 'phenotypes' can predict how well they respond to standard rehabilitation. Researchers will enroll 50 adults diagnosed with carpal tunnel syndrome who fit the neuropathic pain profile. Part…

This trial is testing whether a probiotic supplement called Colipral, which contains a specific strain of E. coli bacteria, can improve gut health in people with ulcerative colitis who are in remission but still have digestive issues. Participants will take either the supplement …

This study tests whether adding a type of bodywork called Structural Integration to standard pulmonary rehabilitation can help people with COPD expand their chest more easily. Participants will be randomly assigned to receive either the full 10-Series of Structural Integration pl…

This trial tests whether deep brain stimulation (DBS) targeting the cerebellum is safe and feasible for people with spinocerebellar ataxia types 1 and 3, a group of inherited disorders that cause progressive problems with movement and coordination. The study will enroll 12 adults…

This trial is testing whether anifrolumab, an experimental antibody, can help Chinese adults with moderate-to-severe systemic lupus erythematosus (SLE) achieve remission. Participants receive anifrolumab infusions over 52 weeks alongside standard care, with a focus on safely redu…

This study is testing whether guselkumab, a biologic drug, can help people with moderate-to-severe ulcerative colitis achieve remission. Participants receive IV infusions followed by maintenance injections. The trial tracks clinical, endoscopic, and histological healing over 48 w…

This trial compares a new gene therapy to standard care in people aged 12 to 35 with severe sickle cell disease. The gene therapy uses a patient's own blood stem cells, modified to produce a therapeutic form of hemoglobin and reduce the sickling hemoglobin. The study measures how…

This early-stage trial is evaluating an investigational drug called QLS2404, given as an injection, to see if it is safe and tolerable. The study first tests single doses in healthy volunteers, then moves to multiple doses in people with systemic lupus erythematosus or primary Sj…

This trial tests whether a topical gel called GM-XANTHO can reduce the severity of psoriasis, a chronic skin condition that causes red, scaly patches. About 65 adults with plaque psoriasis will apply the gel or a placebo daily for 28 days. Researchers will measure changes in skin…

This phase 3 trial is testing whether giving men with metastatic prostate cancer half the standard dose of certain hormone therapies (called ARPIs) can be just as effective as the full dose, while causing less fatigue and fewer treatment stoppages. About 1,500 men with prostate c…

This trial is testing an experimental oral drug called PTT-621 in adults aged 18-70 with moderate-to-severe ulcerative colitis who haven't responded well to standard treatments. Participants will take PTT-621 tablets for 12 weeks. The main goal is to see if the drug can improve t…

This Phase 3 trial will test whether a combination of three cholesterol-lowering drugs—bempedoic acid, ezetimibe, and a high-intensity statin—can reduce and stabilize plaque buildup in the coronary arteries. The study enrolls 103 adults with coronary atherosclerosis who have neve…

This early-stage trial evaluates an investigational oral drug called ONO-6414 in healthy adults and in people with active ulcerative colitis. The main goals are to assess the drug's safety, tolerability, and how the body processes it. The study includes single and multiple doses,…

This early-phase trial is testing an investigational drug called ACG102, given by intravenous infusion, in people with refractory active systemic lupus erythematosus (SLE) — a form of lupus that has not responded well to standard treatments. The main goals are to check the drug's…

This study tests whether mepolizumab, an injectable medication, can improve lung ventilation and reduce mucus plugs in adults with COPD who have elevated eosinophils. Participants receive mepolizumab every 4 weeks for 48 weeks and undergo MRI and CT scans along with breathing tes…

This trial evaluates a gene therapy skin graft called prademagene zamikeracel (pz-cel) for treating wounds in people with recessive dystrophic epidermolysis bullosa (RDEB), a severe genetic condition that causes fragile skin and chronic wounds. The study includes three parts: one…

This study tests the long-term safety and effectiveness of an experimental oral drug called S-606001 in adults with late-onset Pompe disease, a genetic condition that causes progressive muscle weakness. Participants who completed a prior S-606001 study can join, and they will rec…

This study tests a new type of artificial lens for cataract surgery that may adjust focus like a natural lens. About 100 adults needing cataract surgery will get this lens in one eye and a standard lens in the other. Researchers will check for complications and measure vision imp…

This trial tests whether active remote management—where a respiratory therapist regularly reviews ventilator data and adjusts settings—can improve quality of life and reduce flare-ups in COPD patients with chronic hypercapnia using home breathing support. Participants are randoml…

This phase 2 trial is testing whether an investigational drug called arumakimig can improve symptoms of VEXAS syndrome, a rare genetic autoinflammatory condition. The study will compare arumakimig against a placebo in about 120 adults with VEXAS, with the goal of seeing if the dr…

This study is testing whether an experimental drug called FXS6837 can safely reduce protein in the urine and help preserve kidney function in people with IgA nephropathy, a disease that can lead to kidney failure. About 60 patients who previously completed a shorter study of FXS6…

This trial tests the drug mercaptopurine in people with hereditary leiomyomatosis and renal cell carcinoma (HLRCC), a rare genetic condition that raises the risk of skin, uterine, and kidney tumors. The study aims to find the safest dose and see if the drug can shrink kidney canc…

This trial tests whether injecting a diluted solution of terlipressin, a drug that narrows blood vessels, under the outer layer of a uterine fibroid before removal can reduce blood loss during myomectomy surgery. Women scheduled for myomectomy will be randomly assigned to receive…

This early-stage trial is testing an experimental drug called SNC116 in people with refractory systemic lupus erythematosus (SLE), a form of lupus that hasn't improved with standard treatments. The study aims to see if a single intravenous infusion of SNC116 is safe and tolerable…

This trial tests whether antiplatelet medications, specifically clopidogrel and aspirin, can reduce inflammation in the blood vessels of people with psoriasis or psoriatic arthritis. Participants take each drug or a placebo for four weeks, and researchers measure changes in infla…

This trial tests a non-invasive brain stimulation technique called transcranial temporal interference stimulation (tTIS) in people with spinocerebellar ataxia type 3, a genetic condition that affects coordination and balance. The stimulation targets a deep brain region involved i…

This observational study tracks how long patients with moderate-to-severe Crohn's disease or ulcerative colitis continue taking guselkumab, a biologic treatment, in everyday clinical practice. Researchers will measure time until treatment discontinuation and early responses like …

This early-stage trial is testing whether a drug called aletekitug is safe and tolerable for people with moderate to severe ulcerative colitis, a chronic inflammatory bowel disease. The drug works by blocking a protein called IL-18, which may play a role in the inflammation that …

This study looks at whether adding a special liquid diet (partial enteral nutrition) to standard medication helps people with active Crohn's disease achieve remission without steroids. About 80 participants will choose to either add the diet or not. The main goal is to see if mor…

This pilot trial tests whether tirzepatide, a once-weekly injection used for diabetes and weight loss, can improve lung function and muscle health in overweight adults with COPD. About 30 participants will receive either tirzepatide or standard care for 12 months, with tests meas…

This study tests whether the drug ganaxolone can reduce seizures in children aged 6 months to 2 years with CDKL5 deficiency disorder, a rare genetic condition causing hard-to-control seizures. Twenty children will receive either ganaxolone or a placebo alongside their usual seizu…

This trial tests whether dual-focus contact lenses, which have special rings to slow eye growth, can reduce the progression of myopia (nearsightedness) in young adults aged 18 to 30. Participants will wear either these lenses or standard single-vision lenses for 24 months. The st…

This trial tests whether taking coenzyme Q10 (CoQ10) for 12 weeks can reduce fat buildup in the liver of people with mitochondrial diabetes, a rare form of diabetes caused by mitochondrial dysfunction. Participants with the m.3243A>G mutation will take 300 mg of CoQ10 daily, and …

This phase 2 trial tests a new drug called brenetafusp (IMC-F106C) in people with rare cancers—synovial sarcoma and myxoid liposarcoma—that have spread or cannot be surgically removed. The drug is a type of immunotherapy designed to guide the body's immune cells to attack cancer …

This study will test whether a diet rich in protein and fiber can reduce harmful gas-producing bacteria in the gut of people with ulcerative colitis. Forty adults with mild to moderate disease will eat specially prepared frozen meals for two separate week-long periods. Researcher…

This trial tests whether treating high blood pressure in pregnancy to a stricter target (below 140/90) is better than the usual approach (below 160/110) for women with gestational hypertension or preeclampsia without severe features. About 4,120 pregnant women will be randomly as…

This early trial is testing an experimental biologic drug called aletekitug in adults with moderate to severe ulcerative colitis. The main goal is to see if the drug is safe and tolerable when given repeatedly. Researchers will monitor side effects and changes in lab tests, vital…

This Phase 3 trial compares a new once-daily formulation of trientine tetrahydrochloride (TETA 4HCl) to the standard drug D-penicillamine for people newly diagnosed with Wilson's disease. About 38 adults and children (ages 8 and up, weighing at least 55 lbs) will be randomly assi…

This trial studies children aged 8 to 16 who have used repeated low-level red light (RLRL) therapy for one year to control nearsightedness (myopia). Participants are randomly assigned to either continue the therapy, stop it completely, or gradually reduce it. The goal is to see h…

This trial investigates whether a sensitive blood test (Signatera) can detect when it is safe to pause immunotherapy in people with advanced solid tumors like lung cancer, melanoma, and colorectal cancer. Participants are randomly assigned to either continuous standard immunother…

This phase 3 trial tests whether an experimental oral drug called engasertib can reduce the number of nosebleeds in adults with moderate to severe hereditary hemorrhagic telangiectasia (HHT), a genetic condition that causes abnormal blood vessels and frequent bleeding. About 240 …

This phase 2 trial is testing whether the combination of avutometinib and defactinib can stop or shrink malignant peripheral nerve sheath tumors (MPNST) that have returned or cannot be removed with surgery. The study enrolls people over 12 years old with relapsed, refractory, met…

This early-stage trial is testing an experimental oral medication called Alpha-0261 in about 10 adults with moderate to severe ulcerative colitis. The main goal is to check the drug's safety and tolerability, while also looking at whether it can help reduce symptoms and lead to c…

This study tests whether a low-intensity laser applied to the diseased area just before an anti-VEGF injection helps the drug work better in people with wet age-related macular degeneration (AMD). Wet AMD causes abnormal blood vessels to leak fluid under the retina, and current t…

This Phase 3 trial is testing whether an experimental drug called adimanebart can help people aged 12 and older with certain congenital myasthenic syndromes (CMS) feel better and do daily activities more easily. Participants are randomly assigned to receive either adimanebart or …

This Phase 3 trial tests whether combining two drugs, tofacitinib and imatinib, can safely clear the painful pustules of palmoplantar pustulosis (PPP), a rare chronic skin disease on the palms and soles. About 135 adults with moderate-to-severe PPP will receive either the combo o…

This phase 2 trial is testing whether the drug ivonescimab can help control advanced or metastatic adrenal cancers, specifically adrenocortical carcinoma (ACC) and pheochromocytoma/paraganglioma (PPGL), that have not responded to prior treatment. About 20 adults with these rare c…

This early-phase trial tests whether low doses of erlotinib, a drug originally used for cancer, can improve symptoms of rare genetic skin conditions called keratinopathies. About 44 adults with confirmed diagnoses will take increasing doses over 24 weeks. The goal is to see if it…

This trial tests whether converting a prior sleeve gastrectomy to a procedure called intestinal transit bipartition can improve stomach emptying, reduce acid reflux, and help with weight control. Forty adults who had sleeve gastrectomy and now have weight regain or reflux will un…

This early-stage trial is testing an experimental oral drug called HL40626S in healthy adults aged 18 to 55. The goal is to check the drug's safety, how the body processes it, and the best dose range. If it passes these initial safety checks, the drug could eventually be studied …

This trial investigates how the drug zoledronic acid is processed in the body of people with chronic kidney disease who are on dialysis and also have osteoporosis. It aims to find the best dose—either 2.5 mg or 5 mg—given as a single intravenous injection during a dialysis sessio…

This trial tests whether two self-assessment methods in a smartphone app can generate hearing aid settings that match those from a standard hearing test. Adults with perceived mild-to-moderate hearing loss will use the app to self-test their hearing and also undergo conventional …

This trial is testing whether a single injection of stem cells from umbilical cord tissue, given into the spinal fluid, is safe and tolerable for people who have had a traumatic spinal cord injury within the past ten years. The study will monitor participants for side effects and…

This pilot study tests whether adding dipyridamole, an approved drug that improves blood flow, to standard care is feasible and safe for pregnant women with early-onset preeclampsia. The trial will enroll 15 hospitalized women between 26 and 34 weeks of pregnancy with high levels…

This phase 2 trial is testing an oral drug called PLX-200 (gemfibrozil) in children aged 2 to 15 with certain lysosomal storage disorders (LSDs), including CLN2, CLN3, Sandhoff disease, and Krabbe disease. The study aims to see if the drug is safe, tolerable, and may slow the pro…

This phase III trial is testing whether switching liver transplant recipients to an everolimus-based, calcineurin inhibitor-free regimen one year after surgery can better preserve kidney function compared to staying on a low-dose calcineurin inhibitor. The study enrolls 150 adult…

This trial tests an investigational medicine called ATR 1072 in adults with PRKAG2 syndrome, a genetic condition that causes abnormal heart muscle thickening and rhythm problems. The drug is designed to reduce the activity of the faulty PRKAG2 gene. Researchers are evaluating its…

This trial tests whether 3D-printed metal retainers (made from cobalt-chromium or titanium) work better than the standard twisted-wire retainer at keeping teeth from shifting after braces. Thirty-six adults who have just finished orthodontic treatment will receive one of the thre…

This study looks at whether diazoxide, a drug that reduces insulin release, can help children with type 1 glycogenosis maintain steadier blood sugar levels. Type 1 glycogenosis is a rare inherited condition where the body cannot properly release glucose from stored glycogen, lead…

This trial investigates whether a 12-week home-based circuit training program can reduce intraocular pressure and improve retinal health in people with glaucoma. Participants are randomly assigned to either the exercise program plus standard care or standard care alone. The study…

This pilot study tests whether a Mediterranean diet and regular exercise can alter the gut microbiome in people with Parkinson's disease. Researchers will track changes in gut bacteria and look for links to symptom severity and quality of life. The study involves 80 participants …

This study looks back at medical records of children with spinal muscular atrophy (SMA) type 1 who received a one-time gene therapy called onasemnogene abeparvovec (Zolgensma). Researchers want to see if treated infants can sit independently for 30 seconds or more within a year. …

This trial investigates whether eating purple-fleshed potatoes daily for four weeks can improve blood pressure and other heart health markers in men with prehypertension. Participants will consume either purple or white potato puree in a crossover design, with measurements taken …

This trial tests whether adding high-intensity laser therapy (HILT) to a physiotherapy programme can improve bone health in postmenopausal women with osteopenia or osteoporosis. Forty women will receive either real or sham laser sessions on their lower back, three times a week fo…

This trial explores whether starting transdermal estradiol (a skin gel or patch) soon after menopause can protect the heart and brain in women who have hot flashes and at least one cardiovascular risk factor. One hundred women will be randomly assigned to receive the hormone ther…

This trial tests whether implanting dopamine-producing cells into the brain can improve motor function in people with Parkinson's disease. The cells, made from stem cells, are surgically placed into the putamen. The study focuses on safety and whether the treatment can reduce sym…

This trial tests an inhaled investigational drug called ALX1 in adults with bronchiectasis, a chronic lung condition that causes coughing and mucus buildup. Participants receive either ALX1 or a placebo for 14 days. The study aims to find safe and effective doses for future resea…

This trial tests whether a special vibrating vest, worn around the chest, can improve lung function in children aged 6 to 12 with non-cystic fibrosis bronchiectasis — a condition where the airways are permanently widened and clogged with mucus. The vest shakes the chest at high f…

This phase II trial tests an experimental drug called QLS7305 in people with several kidney diseases driven by overactivation of the complement system, a part of the immune system. The drug is a small interfering RNA (siRNA) designed to reduce production of complement protein C3,…

This phase 1 trial tests an experimental cell therapy called LMY-922 (BAFF CAR-T cells) in people aged 16 to 75 with refractory autoimmune diseases, including rheumatoid arthritis, lupus, dermatomyositis, and systemic sclerosis. The therapy uses donor immune cells engineered to t…

This early-stage trial tests whether an experimental drug called TLN-499, combined with the approved cancer drug venetoclax, is safe and tolerable for people with certain advanced solid tumors that have stopped responding to standard treatments. The study includes adults with sma…

This trial tests whether an experimental drug called JP-1366 can prevent the return of erosive esophagitis in people whose condition has already healed. About 384 adults aged 19 to 75 will take either JP-1366 or the standard drug lansoprazole daily for 24 weeks. The goal is to se…

This trial investigates whether ECC4703, a potential new treatment for non-alcoholic fatty liver disease (NAFLD), changes how the body processes two other drugs: sulfasalazine and pitavastatin. Healthy adults aged 18 to 65 will take these drugs alone and then with ECC4703 to meas…

This trial tests whether a daily application of roflumilast 0.3% cream can improve both chronic plaque psoriasis and moderate-to-severe nail psoriasis. People aged 12 and older with stable disease for at least 6 months apply the cream to affected nails and plaques daily for 24 we…

This phase 1 trial is testing an experimental drug called ATG-201 in adults with systemic lupus erythematosus, systemic sclerosis, or Sjögren's disease. ATG-201 is designed to bind to specific proteins on immune cells, potentially quieting the overactive immune response that driv…

This pilot study tests whether an antioxidant cocktail called Rett-T can improve motor function in children and adolescents with Rett syndrome, a rare genetic disorder that affects movement and coordination. Participants, aged 2 to 21 and at least partially able to walk, will rec…

This phase 1 trial tests two experimental oral drugs, UI059 and UIC202201, in healthy adult volunteers to see how safe they are and how well they control stomach acid. Each participant takes one drug daily for a week, then switches to the other after a break. The goal is to compa…

This trial tests whether a gradual dosing schedule of cannabidiol oral solution (CBD-OS) can help reduce seizures in adults with Lennox-Gastaut syndrome, a severe form of epilepsy. Participants will take CBD oil alongside their usual seizure medications. The study measures how ma…

This trial is investigating whether a higher 8 mg dose of aflibercept, given every three months, can effectively control wet age-related macular degeneration (nAMD) in people who have not been treated before. The study will measure changes in eye fluid biomarkers, vision, and saf…

This trial tests whether a procedure called pulsed field ablation can safely treat ventricular arrhythmias—abnormal heart rhythms that can be life-threatening. About 30 people with frequent premature heartbeats or ventricular tachycardia will receive the treatment using a special…

This trial tests whether adding the bispecific antibody glofitamab to standard chemoimmunotherapy can improve outcomes for people with Burkitt lymphoma or high-grade B-cell lymphoma with MYC and BCL2 rearrangements. These are fast-growing cancers that can be hard to treat. The st…

This trial tests a new suture system designed to close a patent foramen ovale (PFO), a small hole in the heart that can increase stroke risk. Researchers will compare the new device to an existing occluder in about 244 adults aged 18 to 65 with moderate-to-large PFOs. The goal is…

This phase 3 trial is testing an experimental daily capsule called TQH3906 in adults with moderate to severe plaque psoriasis. The drug works by blocking a key immune pathway involved in skin inflammation. Researchers are comparing TQH3906 against a placebo and an approved treatm…

This trial tests whether inebilizumab, an antibody that wipes out certain immune cells called B cells, can improve physical function in adults with ME/CFS that started after an infection. Participants receive three infusions over six months, and the study measures changes in mobi…

This trial tests an experimental oral drug, TTI-0102, in people aged 5 to 55 with Leigh syndrome spectrum, a rare genetic disorder that damages the brain and nerves. The study aims to find the right dose and check safety over 12 weeks of twice-daily treatment. Participants will h…

This phase 2 trial is testing whether combining an experimental drug called S-531011 with a precise radiation technique known as RadScopal can help control metastatic colorectal cancer that has not responded to prior treatments. The study enrolls about 30 adults with advanced dis…

This study tests an experimental drug called dibotatug (DR-01) in adults with bone marrow failure syndromes, including severe aplastic anemia. The goal is to see if the drug is safe and can improve blood cell counts. Participants receive dibotatug by IV infusion, and researchers …

This trial tests whether an experimental drug called JZP926 can safely reduce the number of days people aged 10 and older experience myoclonic seizures—brief, shock-like jerks—due to juvenile myoclonic epilepsy. Participants are randomly assigned to receive either JZP926 or a pla…

This trial investigates whether continuous positive airway pressure (CPAP) can help adults with sickle cell disease recover faster from acute chest syndrome, a serious lung complication. Participants who need extra oxygen are randomly assigned to receive either standard oxygen al…

This trial tests whether an experimental drug called bomedemstat can help people with polycythemia vera (a condition where the body makes too many red blood cells) who did not get better with or could not tolerate the common treatment hydroxyurea. Participants will receive either…

This phase II trial tests whether obinutuzumab, a B-cell targeting antibody, can alone achieve complete kidney response in people with pure class V lupus nephritis—a form of lupus kidney disease with no approved treatments. Participants receive up to four infusions over seven mon…

This trial tests whether a package of support from a community health worker can help adults hospitalized with advanced HIV in Lusaka, Zambia, stay in care and survive longer. Participants are randomly assigned to receive the ARC intervention or standard care and are followed for…

This trial tests whether adding a posterior cervical fusion (using the CORUS PCSS 3D device) to the standard anterior cervical discectomy and fusion (ACDF) improves bone fusion success for people with two-level degenerative disc disease in the neck. About 225 adults with neck or …

This trial tests whether a single injection of carbetocin, a longer-acting relative of oxytocin, can reduce blood loss in women undergoing open abdominal surgery to remove uterine fibroids. About 900 participants will be randomly assigned to receive carbetocin or a placebo, and d…

This trial tests whether different scaffolds—a blood clot or various platelet-rich fibrin preparations—can improve healing in teeth that need a second root canal treatment. Sixty participants will receive one of four scaffolds during the procedure and be followed for a year. The …

This phase 2 trial tests whether the experimental drug SHR-1139 can improve skin clearance in adults with moderate to severe plaque psoriasis. About 60 participants will receive either SHR-1139 or a placebo over 52 weeks. The main goal is to see how many achieve complete skin cle…

This early trial tests a gene therapy called ZR001 in people with moderately advanced Parkinson's disease. ZR001 is injected into a brain region involved in movement, and a very low dose of an existing drug is then used to activate the therapy. The goal is to see if this approach…

This trial asks whether adults with ulcerative colitis who are in deep remission can safely stop taking their JAK inhibitor medication (tofacitinib, upadacitinib, or filgotinib) instead of continuing daily maintenance therapy. Half of the 224 participants will stop treatment and …

This trial tests a 12-week home-based program called SPRINT for infants aged 7-15 months who were born preterm or have a family history of conditions like ADHD, autism, or developmental delay. Parents use an app to guide daily play activities aimed at improving parenting skills a…

This trial tests several drugs in people with NF2-related schwannomatosis, a condition that causes tumors on nerves and in the brain. The study uses a flexible 'platform' design to evaluate treatments for four tumor types: vestibular schwannomas, non-vestibular schwannomas, menin…

This phase 2 trial tests an oral drug called zanzalintinib in people with metastatic pheochromocytoma or paraganglioma that cannot be surgically removed and is growing. The study measures how many participants see their tumors shrink or stop growing. All participants take the dru…

This trial compares two types of artificial lens implants used during cataract surgery: enhanced monofocal lenses and extended depth-of-field lenses. The goal is to see which one provides better vision at far, intermediate, and near distances, and whether one causes fewer visual …

This study tests whether low-level laser therapy, when added to standard physiotherapy, can improve nerve healing in people with mild-to-moderate carpal tunnel syndrome. Participants receive either real or sham laser sessions three times a week for four weeks. The researchers mea…

This study looks at how well a device can close a common heart hole called a ventricular septal defect (VSD) in children aged 6 months to 18 years. Instead of open-heart surgery, doctors use a small tube to place a device that plugs the hole. The goal is to see if this method is …

This phase II trial tests whether a personalized dendritic cell vaccine targeting the Survivin protein can improve survival in people with newly diagnosed glioblastoma, an aggressive brain cancer. Participants receive the vaccine alongside standard chemotherapy after surgery. The…

This study looks at whether taking macaúba oil supplements while doing outdoor exercise can improve how the body burns fat and handles mental stress. Researchers will enroll 60 physically active adults aged 35 to 59 who are overweight but not obese. Participants will take either …

This trial tests whether adding orlistat, a weight-loss drug that blocks fat absorption, to standard progestin therapy can improve treatment response in young women (45 or younger) with early-stage endometrial cancer or atypical endometrial hyperplasia who have low progesterone r…

This study compares two treatments for palmoplantar psoriasis, a form of psoriasis affecting the palms and soles. One group receives a topical steroid cream alone, while the other receives the same cream plus a fractional CO2 laser treatment every four weeks. The goal is to see i…

This study tests an experimental drug called nizubaglustat (AZ-3102) in people aged 4 to 30 with Gaucher disease type 3, a rare genetic condition that can cause anemia, low platelets, an enlarged spleen, and neurological problems. Participants take the drug daily as a dissolvable…

This phase 3 trial tests whether an experimental drug called NPI-001 can slow the loss of light-sensing cells in the eyes of adults with Usher syndrome, a genetic condition that causes both hearing and vision loss. About 80 participants take either NPI-001 or a placebo twice dail…

This study tests a combination of four drugs—sotoclax, carfilzomib, lenalidomide, and dexamethasone (called SonKRd)—in people newly diagnosed with multiple myeloma that has a specific genetic change called t(11;14). Participants must be healthy enough for a stem cell transplant. …

This pilot study tests a personalized lifestyle program called PRISM for adults with metabolic syndrome and insulin resistance. Participants follow a time-restricted eating schedule (eating all meals within an 8-hour window) and are later assigned extra lifestyle changes like phy…

This study tests whether a digital bottle cap called FORTISKAP™ can detect missed doses of proton pump inhibitors (PPIs) in people with GERD more accurately than standard methods like pharmacy fill records or patient questionnaires. Fifty adults with GERD who take a daily PPI wil…

This phase II trial tests an injected drug called SR604 in people with congenital Factor VII deficiency, a rare bleeding disorder. The goal is to see if SR604 can reduce the number of bleeding episodes and improve safety. The study includes 12 participants aged 12 to 65 who have …

This study tests whether a low-dose sugarcane-derived supplement called policosanol, combined with a structured exercise program, can improve physical fitness and blood lipid levels in adults with elevated cholesterol who do not yet need medication. Over 12 weeks, participants ar…

This study tracks how well the FLOWRUNNER Aspiration System removes symptomatic blood clots from veins in the arms or legs during routine care. About 218 adults with deep-vein clots will be followed to see if the device safely restores blood flow and reduces clot-related disabili…

This trial investigates whether a 7-day course of antibiotics works as well as the standard 14-day course for adults hospitalized with a sudden worsening of COPD. The study involves over 500 participants and measures clinical success at 30 days, including symptom improvement and …

This trial tests a gene therapy called PST-611 in people aged 65 and older with geographic atrophy, an advanced form of age-related macular degeneration that causes blind spots. PST-611 is a DNA plasmid injected into the eye muscle that instructs cells to produce human transferri…

This study tests whether a modified version of the Crohn's Disease Exclusion Diet (CDED) can ease symptoms of non-constipation irritable bowel syndrome (IBS) better than the standard low FODMAP diet. About 150 adults with moderate-to-severe IBS will follow one of the two diets fo…

This trial tests whether a liquid called Theravex can improve healing and stability when a dental implant is placed right after a tooth is pulled. Theravex is applied directly into the gap between the implant and the bone socket to stimulate bone growth. Researchers compare it to…

This trial tests the drug selumetinib in people with NF2-related schwannomatosis, a genetic condition that causes non-cancerous tumors on nerves, often leading to hearing loss. The study includes both adults and children whose tumors cannot be surgically removed or are growing. R…

This study tests an 8-week digital exercise program for people with Parkinson's disease. The program combines exercise with cognitive challenges and educational sessions, all delivered online. Researchers want to see if it is feasible and whether it can increase physical activity…

This study investigates whether combining high-intensity interval training (HIIT) with intermittent fasting (14-hour fast, 10-hour eating window) can reduce insulin resistance in obese women with polycystic ovary syndrome (PCOS). Participants will follow a supervised exercise pro…

This trial tests whether combining two drugs—luvometinib (a targeted therapy) and serplulimab (an immunotherapy)—can shrink tumors and improve hearing in people with NF2-related schwannomatosis. Participants have progressive vestibular schwannomas or meningiomas that are not suit…

This trial tests whether adding a 3D holographic visualization tool, called EchoPixel Holographic Therapy Guidance, helps doctors perform two common heart procedures more accurately. The procedures are left atrial appendage occlusion (for atrial fibrillation) and mitral valve rep…

This study is for people with a slow-growing type of lymphoma called MALT lymphoma that has not spread. It compares a very low dose of radiation (4 Gy) combined with the targeted drug orelabrutinib against the standard higher dose of radiation (24 Gy). The goal is to see if the g…

This study looks at how the oral medication etrasimod works for Chinese adults with active ulcerative colitis (UC) in real-world settings. About 500 participants will take etrasimod as part of their routine care and be followed for at least 52 weeks. Researchers will track change…

This study is looking at whether a blood test called Galleri can help detect ovarian cancer early in people with BRCA1 or BRCA2 gene changes, who are at higher risk. Participants will give a blood sample and answer questionnaires about their experience. The study will also check …

This study asks whether levels of uromodulin, a protein in the blood, can indicate the severity of kidney damage in people with lupus nephritis. Researchers will compare uromodulin levels with kidney biopsy results from 90 participants, including those with different classes of l…

This study investigates whether levels of a protein called RNASE1 in the blood can serve as a biomarker for lupus nephritis, a serious kidney complication of systemic lupus erythematosus (SLE). Researchers will compare RNASE1 levels in 128 adults with SLE, some with lupus nephrit…

This study tests whether combining several advanced genetic analysis techniques can find the cause of intellectual disability or neurodevelopmental disorders in people who have already had standard genetic testing with no answer. Participants will have their existing genetic data…

This study investigates whether measuring two proteins, CD38 and CD56, on white blood cells called neutrophils can help diagnose chronic myeloid leukemia (CML). Researchers will compare the levels of these markers in 200 people with CML to see if a high percentage is a reliable s…

This trial tests whether a new ultra-rapid genetic test, using third-generation sequencing, can diagnose familial lymphohistiocytosis (FHL) in children within 5 days instead of the usual 6-8 weeks. FHL is a rare, life-threatening genetic condition where the immune system attacks …

This study tests whether combining two stool tests—fecal immunochemical test (FIT) and fecal calprotectin (FC)—can accurately detect precancerous or cancerous changes in people with long-standing ulcerative colitis. Participants will provide stool samples and then undergo a speci…

This study tests whether annual liquid biopsies (from blood, urine, stool, and vaginal swabs) can safely replace colonoscopies every 18 months for people with Lynch syndrome. 2,000 participants will be split into two groups: one gets standard care (colonoscopy every 18 months), t…

This study tests whether a handheld, ultra-widefield eye scanner (OCT) can accurately identify preterm infants with severe retinopathy of prematurity (ROP), a condition that can harm vision. The scanner takes detailed images of the retina without touching the eye, potentially red…

This study tests a community-based screening strategy for tuberculosis (TB) in migrant communities in New York City. A mobile van offers digital chest X-rays with AI interpretation, along with TB symptom checks, blood tests for latent TB, and HIV testing. The goal is to see wheth…

This study tests whether a combination of blood markers, ultrasound, and genetic information can accurately diagnose metabolic dysfunction-associated steatotic liver disease (MASLD) in children aged 8 to 18 with obesity. Each child provides a blood sample, undergoes a liver ultra…

This pilot study tests a new imaging system that uses OCT and vibrometry to let doctors see the middle ear bones and measure how they move in response to sound. The study enrolls 500 adults with conductive or sensorineural hearing loss. The goal is to compare these detailed motio…

This trial tests whether a finger-prick HbA1c test can detect type 2 diabetes better than the standard random blood glucose test in at-risk adults living in rural Dali, China. Researchers will compare detection rates and costs among 720 permanent rural residents with no known dia…

This study tests whether combining two screening questionnaires with a musculoskeletal ultrasound can detect spondyloarthritis—a type of inflammatory arthritis—in people who already have inflammatory bowel disease (IBD) but have not been diagnosed with arthritis. Two hundred adul…

Lupus nephritis is a serious kidney complication of lupus that can lead to kidney failure. This study investigates whether levels of neutrophil extracellular traps (NETs)—web-like structures released by immune cells—in the blood can predict how well patients respond to therapy. R…

This study tests whether combining point-of-care screening with community-based strategies can help identify infants with sickle cell disease early and connect them to care. Researchers will screen up to 6,750 newborns in Mozambique and track their health outcomes. The goal is to…

This study tests whether placing a pH monitoring probe closer to the stomach (1 cm above the squamocolumnar junction) instead of the standard 6 cm position improves detection of acid reflux in women and people with lower body weight. Participants receive two probes simultaneously…

This study is developing a computer-assisted tool that uses a camera to objectively measure movement problems caused by antipsychotic medications. Researchers will enroll 180 people—half with these movement disorders and half healthy—to see how well the tool's results match stand…

This trial tests whether special glasses with defocus lenses, aided by artificial intelligence, can prevent myopia in children aged 3 to 6 who are at high risk. The glasses are worn for at least 10 hours a day, and a smartwatch reminds kids to spend time outdoors. The study measu…

This trial tests whether two natural supplements—quercetin (from foods like apples and onions) and coenzyme Q10 (a substance your body makes)—can reduce inflammation and improve metabolic health in older adults. Over 12 weeks, participants aged 50 to 75 who are sedentary will tak…

This trial asks whether taking 50 mL of extra virgin olive oil every day can reduce the chance of heart attacks, strokes, and related deaths in people living with HIV. About 1,300 adults aged 50–75 with well-controlled HIV and at least one heart risk factor will be randomly assig…

This trial tests whether giving students accurate information about their peers' actual behavior can reduce heavy drinking and risky sexual activity. About 1,350 adolescents in three Ugandan schools will complete surveys and receive personalized feedback comparing their own estim…

This trial tests whether cleaning the chewing surfaces of children's teeth with a fine erythritol powder spray (Guided Biofilm Therapy) before applying a protective sealant helps the sealant stay in place longer than the standard rotary-brush polishing. Each child aged 6–10 will …

This trial tests whether combining a daily 18-minute virtual reality training session with low-dose atropine eye drops can slow the worsening of nearsightedness in children aged 6 to 12. Participants will also wear special glasses that provide defocus to the peripheral vision. Th…

This study explores whether community health workers can use a new skin test, with or without AI help, to test close contacts of TB patients at home instead of at a clinic. The goal is to make it easier for people, especially children and families in Tajikistan and Tanzania, to g…

This pilot study tests whether a simple program of brief, frequent movement throughout the day can help reduce cardiometabolic risk in rural women. Participants wear a Fitbit that reminds them to move each hour and complete health assessments before and after the 12-week program.…

This study compares three commonly used instruments for scleral depression, a routine eye exam technique that presses on the eye to view the retina. The goal is to see which tool causes the least pain and is easiest for doctors to use. Adults with symptoms of posterior vitreous d…

This trial tests whether giving patients a prescription for haloperidol to use as needed at home can help control symptoms of cannabinoid hyperemesis syndrome — a condition marked by severe nausea, vomiting, and abdominal pain from heavy cannabis use. The goal is to see if having…

This trial tests an online platform called Ready Set Recover, designed to help people prepare for and recover from spine surgery. The platform offers education, mindfulness, gratitude exercises, and gentle movement to support mental well-being and patient engagement. Researchers …

This study compares two types of virtual reality (VR) during common ear, nose, and throat (ENT) procedures. One group will watch calming scenery, while the other plays an interactive puzzle game. Researchers want to see which better reduces pain and anxiety. The study will includ…

This study explores whether a type of visual training called perceptual learning can improve uncorrected vision in children aged 6 to 10 with low myopia (mild nearsightedness). Thirty children will be randomly assigned to either a training group that uses a digital Vision Trainin…

This trial tests whether giving a combination of dexmedetomidine and esketamine during and after laparoscopic ureteral reconstruction can reduce depressive symptoms and anxiety in patients. The study enrolls 658 adults with ureteral stricture who are scheduled for this surgery. P…

This trial tests whether using a virtual reality headset during regional anesthesia can lower patients' anxiety and pain. Adults aged 18 to 64 having elective surgery with regional anesthesia will either receive VR distraction or standard care. Researchers will measure anxiety an…

This pilot study tests whether N-acetylcysteine (NAC), an antioxidant supplement, can reduce repetitive and self-injurious behaviors in people with Cornelia de Lange syndrome (CdLS). Ten participants aged 13–35 will receive both NAC and a placebo in random order over 18 weeks. Th…

This trial tests whether a structured 12-week physiotherapy exercise program can improve balance, posture, and reduce fall risk in people with osteoporosis. Participants, aged 50 and older, will be randomly assigned to either the exercise program or a control group. The program f…

This pilot study tests a new preservative-free eye drop called NOX-01 in people with mild to moderate dry eye who are having cataract surgery. The drop is designed to lubricate and protect the eye surface, potentially reducing the dry eye symptoms that often occur after surgery. …

This trial tests whether an AI-based system can provide preoperative counseling for elective cesarean delivery as effectively as a physician. Women scheduled for a C-section will be randomly assigned to receive either standard doctor-led counseling or AI-delivered counseling with…

This trial tests whether a structured reading rehabilitation module can improve reading speed and reduce errors in people with glaucoma, a condition that often impairs vision. Participants will practice the module for 20-30 minutes daily, at least 3 days a week, for 6 months. The…

Many cancer patients feel anxious before radiotherapy, partly due to unfamiliar equipment and procedures. This trial tests whether adding a simulation-based education session — a virtual preview of the treatment environment — before standard education can reduce that anxiety and …

This pilot study tests a therapy protocol based on cognitive-occupational principles (CO-OP) for children and adolescents aged 10-21 with mild to moderate intellectual disability. The intervention involves weekly 45-minute sessions over 12-15 weeks, delivered by occupational ther…

This pilot trial is testing whether a topical gel called Filsuvez, made from birch triterpenes, is safe and helpful for people aged 6 months and older with moderate-to-severe epidermolysis bullosa simplex (EBS), a condition causing fragile skin and blisters. Participants will app…

This trial tests whether a 12-week Latin dance program—featuring Rumba, Cha-cha, and Jive—can improve psychological health in primary school children aged 6 to 12. Participants are randomly assigned to either the dance program or regular physical education classes. Researchers wi…

This trial tests whether vonoprazan, taken as needed, can quickly relieve heartburn episodes in adults with non-erosive gastroesophageal reflux disease (GERD). Participants will take either vonoprazan or a placebo when they experience heartburn, and researchers will track how wel…

This study tests a virtual reality app called Amani designed to help adults with sickle cell disease manage pain, emotional distress, and social isolation. Researchers will enroll 40 adults to see if the app is easy to use and helpful for improving coping skills and peer support.…

This trial tests whether adding kinesiotaping to standard exercise and night splinting can improve symptoms of carpal tunnel syndrome, a condition causing hand numbness, tingling, and pain. Adults with mild-to-moderate carpal tunnel syndrome will be randomly assigned to one of tw…

This study tests whether a gentle, non-invasive brain stimulation technique called transcranial alternating current stimulation (tACS) can improve sleep and reduce anxiety in adults over 60 who have chronic insomnia. Sixty participants will receive tACS sessions, and researchers …

This large citizen-science trial is testing whether two prebiotic products can reduce feelings of anxiety and improve related outcomes like sleep in adults. Around 1,000 participants will be randomly assigned to take either an active prebiotic or a placebo for 12 weeks, without k…

This study tests an inhaled powder called LIQ861 (treprostinil) in adults with scleroderma who have frequent, painful Raynaud's attacks—episodes where fingers or toes turn color and become numb or tingly. About 75 participants will be randomly assigned to receive either a dose th…

This trial tests whether chewing sugar-free gum for 20 minutes, alongside standard intravenous medication, can reduce dizziness and nausea in adults with a common inner-ear condition called posterior canal BPPV. Participants are randomly assigned to either chew gum or receive sta…

This study tests whether using brain images to program deep brain stimulation (DBS) devices works better than the usual method for reducing hand tremors in people with essential tremor. Twenty adults who already have DBS electrodes in their brain will try both programming approac…

This trial tests whether a type of talk therapy called Acceptance and Commitment Therapy (ACT) can help cancer survivors and their spouses cope with the fear that cancer might return. It compares ACT to a more traditional therapy, Cognitive Behavioural Therapy (CBT), in a two-arm…

This trial tests whether ischemic conditioning — brief cycles of blood-flow restriction using pressure cuffs on the arms — can improve motor function and cognitive performance in people with Parkinson's disease. Over 12 weeks, 34 participants will receive 24 sessions of the thera…

This study tests whether regular breathing exercises can improve symptoms of Postural Tachycardia Syndrome (POTS), a condition that causes dizziness and rapid heartbeat when standing. Twelve adults with POTS will practice breathing exercises daily for four weeks while researchers…

This study tests whether injections of platelet-rich plasma (PRP) can reduce pain and improve jaw function in people with chronic jaw muscle pain (myogenous TMD). Fifty women aged 20–50 with at least 3 months of pain will receive either PRP or a saltwater shot, then be followed f…

This study tests whether virtual reality (VR) can reduce pain, fear, and distress in children aged 6 to 12 during burn dressing changes. Half of the participants will receive standard care plus VR distraction, while the other half receives standard care alone. Pain, fear, and dis…

This pilot study explores whether a personalized telerehabilitation program can help adults who notice cognitive slips in daily life. Participants include older adults and adults with Ehlers-Danlos Syndrome who report subjective cognitive complaints. Over six weeks, they receive …

This trial tests whether a culturally tailored mobile health program, called Self-Compassion, Health, and Empowerment (SHE), can improve mental health in Chinese immigrant women who have experienced intimate partner violence. Participants receive six weekly sessions covering safe…

This study compares transanal irrigation (using a device to rinse the bowel) against standard care (diet, fluids, laxatives) for treating severe constipation in people with Parkinson's disease. Researchers will also check if the treatment affects gut bacteria and motor symptoms. …

This trial tests whether a four-week improv comedy program can reduce anxiety and improve mental well-being in Chinese young adults in Hong Kong who have mild-to-moderate anxiety but no diagnosed mental disorder. Participants attend weekly three-hour improv workshops and complete…

This trial tests whether a structured art therapy program, grounded in positive psychology, can improve self-compassion and hope in children aged 8 to 12 who are staying in a hospital ward. Half of the 80 participants will receive the art therapy sessions alongside standard nursi…

This 28-day study is testing whether a lotion containing ubiquinol, pomegranate peel extract, ceramides, and salicylic acid can improve acne lesions and skin comfort in adults with mild-to-moderate acne. Forty participants aged 18 to 40 will use the lotion daily alongside their u…

This trial tests whether mirror therapy — a low-cost technique where a child watches the reflection of their good arm moving, creating the illusion that their affected arm is moving — can improve arm and hand function in children with hemiparetic cerebral palsy. Twenty-six childr…

This trial tests whether flushing the inside of a tooth with cold saline (about 2.5°C) after root canal retreatment can lower pain and inflammation. One hundred adults needing retreatment on a lower premolar will be randomly assigned to receive either cold or room-temperature sal…

This trial tests whether two simple distractions — watching a betta fish or playing with a hand puppet — can lower pain, fear, and anxiety in children aged 5 to 7 during a blood draw. One group will use the fish, another the puppet, and a third will receive standard care. Researc…

This study adapts a psychosocial support program called Caregiver Speaks for parents of children with Rett syndrome. Delivered through a private Facebook group over six weeks, the program aims to reduce caregiver stress and improve well-being. Researchers will test whether the ap…

Pseudo-angina is chest pain that feels like a heart attack but comes from muscle knots, not the heart. This trial tests whether two hands-on treatments—Graston Technique (using a stainless-steel tool to massage muscles) and local ischemic compression (sustained finger pressure on…

This trial tests whether a guided digital program delivered through a mobile app can reduce anxiety and depression in young adults (ages 18–40) who have fertility-related distress after a cancer diagnosis. Participants use the app to complete four modules covering coping skills, …

This pilot study tests whether a nurse-led digital cognitive behavioral therapy for insomnia (dCBT-I), delivered through a chatbot on WhatsApp, can help university students with mild to moderate anxiety or depression. Participants will complete a two-week daily sleep diary via th…

This trial tests whether watching a calming nature video through a virtual reality headset can reduce fear of childbirth and anxiety in high-risk pregnant women who are on bed rest in the hospital. Participants in the intervention group will view a VR nature scene with relaxing s…

This trial tests whether a simple mandala coloring activity can reduce perceived stress and anxiety in women hospitalized with high-risk pregnancies. Sixty participants will be randomly assigned to color mandalas daily for three days or to receive standard care. Stress and anxiet…

This trial tests whether pain reprocessing therapy (PRT) — a brief psychological treatment that combines education, cognitive restructuring, and body-focused techniques — can reduce chronic pain. Adults with persistent pain lasting over three months are randomly assigned to full …

This trial investigates whether sensory-motor hand rehabilitation can improve cognitive functions like memory, attention, and reaction time in people with Parkinson's disease. Thirty-two participants with mild cognitive impairment will be randomly assigned to either hand rehabili…

This trial compares two types of laser eye surgery—SMILE and ray tracing-guided LASIK—in people with nearsightedness and astigmatism. Each participant will receive one technique in one eye and the other in the other eye, allowing a direct comparison. The goal is to see which meth…

This study tests whether video-based education can reduce anxiety, correct myths, and boost motivation in first-time mothers. Pregnant women between 32 and 36 weeks will receive three educational video modules via WhatsApp alongside routine care. The goal is to see if this techno…

This trial investigates whether taking melatonin before bed can improve sleep quality, reduce fatigue, and lower inflammation in adults with active lupus. Sixty-four participants will either receive standard care plus melatonin or standard care alone for 12 weeks. Researchers wil…

This early study tests whether a single session of focused ultrasound—using sound waves to gently stimulate a specific brain region—can safely reduce emotional symptoms like agitation, irritability, and anxiety in people with mild dementia. Fifteen participants aged 45 to 80 with…

This trial tests whether a two-week tongue-strengthening exercise, done with a non-invasive device, can improve speech fluency in people with tongue-tie or slurred speech. The exercise relies on muscle resistance training, not medication or electronics. Researchers will measure c…

This trial tests whether two simple breathing exercises—alternate nostril breathing and bellows breath—can help people with COPD clear mucus from their lungs, improve lung function, and feel better day-to-day. About 54 participants will practice one of the techniques for 15–20 mi…

This study tests whether a 16-week personalized home exercise program can improve muscle health and reduce fatigue in adults with stable lupus. Participants do three weekly sessions combining high-intensity interval training, strength exercises, and walking. Researchers measure c…

This study tests whether a four-week online mindfulness-based education program can help women with Polycystic Ovary Syndrome (PCOS) manage menstrual symptoms, lower stress, and improve quality of life. About 78 women with PCOS will be randomly assigned to either the program or r…

This trial tests a smartphone app called E-breathing that detects when a teen is feeling distressed and prompts them to practice slow-paced breathing. The study includes 100 adolescents aged 11 to 17 in Hong Kong who have at least mild symptoms of depression, anxiety, or stress. …

This study compares two airway clearance methods—autogenic drainage (a controlled breathing technique) and a flutter device (a handheld tool that vibrates to loosen mucus)—in people with moderate COPD. Over six weeks, researchers will measure changes in shortness of breath, lung …

This study tests whether a low dose of aripiprazole, a drug used for psychiatric conditions, can reduce fatigue and improve thinking in adults with Long COVID or other post-infection syndromes. About 138 participants will receive either the drug or a placebo for 8 weeks, then swi…

This study tests whether an AI-assisted expressive writing program can reduce anxiety, emotional distress, and improve quality of life in adults receiving chemotherapy for any type of cancer. Participants are randomly assigned to either use the AI writing tool alongside their usu…

Many Veterans with heart failure or COPD struggle with breathlessness, fatigue, depression, and anxiety that routine care often misses. This study tests whether a virtual team—a nurse and a social worker—can improve quality of life by helping Veterans manage their most bothersome…

This study tests whether dimdazenil, a sleep medication, can safely improve sleep in people with Parkinson's disease who also have insomnia. Researchers will measure sleep changes using overnight brain-wave recordings. The trial involves 50 adults with mild-to-moderate Parkinson'…

This study tests whether applying mild electrical pulses (TENS) to the shins can reduce pain and anxiety in people receiving Botox injections into the bladder for overactive bladder. Participants are randomly assigned to receive either active TENS or a sham device during the inje…

This study tests whether pulsed radiofrequency (PRF) — a procedure that uses electrical pulses to calm nerves — can relieve sacroiliac joint pain better than standard steroid injections. Adults aged 18 to 65 with chronic pain lasting at least 6 months and a pain score above 5 out…

This study tests a 13-week remote stress recovery program for people who had a heart attack before age 55 and still feel distressed. The program includes online sessions with a therapist, a workbook, and videos. The goal is to see if a larger trial is possible by checking how man…

This study tests a new AI-assisted computer mouse designed to reduce hand movements for people with carpal tunnel syndrome or hand/wrist injuries. Participants complete short computer tasks using both a regular mouse and the AI-mouse while researchers measure muscle activity, pai…

This phase 2 trial tests whether nemolizumab, an injectable biologic, can reduce severe itching in women with vulvar lichen sclerosus, a chronic inflammatory skin condition. Participants receive weight-based doses of the drug and track their itch scores daily. The study focuses o…

This study tests whether an AI-driven mobile app, paired with a Fitbit, can deliver personalized exercise plans that older adults find feasible and acceptable. Over 8 weeks, participants aged 65 and older will receive daily and weekly exercise prescriptions tailored to their acti…

This study looks at brain activity in teenagers aged 10 to 17 with ADHD, OCD, or no diagnosis. Researchers will use EEG, MRI, and MEG to measure brain signals while participants do computer tasks. The goal is to understand how the brain controls thinking and behavior, which may h…

This study asks whether swapping a beef burger for a plant-based pea burger changes how the body handles fats and sugar after a meal. Ten adults with metabolic syndrome (and a BMI of 25 or higher) will eat both types of burgers on separate occasions in a crossover design. Researc…

This study will follow 100 adults with Crohn's disease, ulcerative colitis, or other IBD, plus their healthy relatives, for up to 3 years. Researchers will collect blood, stool, tissue samples, and imaging to track how immune cells and genetics change over time. The goal is to fi…

This study evaluates whether an investigational eye imaging device, UNITY DX, can measure the thickness of the cornea and its epithelial layer as accurately and consistently as a commercially available device. About 110 participants with normal or abnormal corneas will undergo mu…

This study uses ultrasound to examine the joints of adults with mild hemophilia, a group that often goes understudied. Researchers will scan six joints (ankles, knees, elbows) to look for early signs of damage that may not cause symptoms yet. The goal is to see how common joint p…

This study screens up to 1,000 people with personal or family histories of certain cancers to see if they qualify for ongoing genetics research at the National Cancer Institute. Participants fill out a 15-20 minute online survey about their health and family history. No treatment…

This study follows 80 people with spinal muscular atrophy (SMA) in Japan who receive Zolgensma (onasemnogene abeparvovec) as an intrathecal injection. The goal is to track safety issues, such as liver problems or blood clotting abnormalities, and to measure changes in motor funct…

This study looks at how eating a high-fat or high-carb breakfast changes fat levels in the blood over 8 hours. Researchers will compare results among people with conditions like diabetes, fatty liver, or kidney disease, as well as healthy volunteers. Participants will have two ov…

This study investigates how eating three high-fat meals in a row changes vitamin E levels in the blood. Researchers will compare people with normal blood fats to those with high blood fats (hyperlipidemia). The goal is to understand whether fats in the blood can trap vitamin E, p…

This early-stage trial is evaluating the safety, tolerability, and how the body absorbs PAN-90806, an experimental eye drop being developed for wet age-related macular degeneration (AMD), a leading cause of vision loss. Healthy adults aged 18 to 50 will receive either PAN-90806 o…

This study looks at whether people with cystic fibrosis (CF) who take newer modulator drugs are developing heart disease. Researchers will use ultrasound to measure blood vessel and heart health, then repeat the tests a year later to see how things change. They will also check if…

This observational study will take small liver samples from 8 adults with hemophilia A or B who had gene therapy at least 6 months ago. Researchers want to see how the gene therapy affected liver cells, including how many cells still carry the therapy and whether there is any liv…

This study follows 70 people with PRKN- or PINK1-linked Parkinson's disease, as well as those with typical Parkinson's and healthy volunteers, over 5 years. Researchers will track movement, thinking, and mood symptoms, and collect blood, urine, and optional spinal fluid or muscle…

This study is a registry that will collect health information from 1,000 people who carry the gene for hereditary amyloidosis, including those with and without symptoms. Researchers will track who develops the disease and how it progresses, including the need for heart transplant…

This study looks at whether brain activity patterns, measured by EEG, can help predict long-term thinking problems in people with sepsis. Researchers will monitor brain waves for 72 hours in 78 ICU patients with different types of inflammation. They will then track cognitive func…

This study explores whether electrical signals from the eye and brain can serve as a noninvasive marker of dopamine activity. About 50 adults—half with Parkinson's disease and half without—will have their eye and brain responses measured using sensors on the skin. People with Par…

This study investigates whether levels of a protein called neurofilament light chain (NFL) in the blood can predict neurological recovery in people who remain comatose after a cardiac arrest. Researchers will measure NFL at several time points over the first week in the ICU and c…

This study investigates how the brain processes social decisions like trust and cooperation. Participants play a modified trust game, either in an MRI scanner or online, where their choices affect monetary gains or losses. The goal is to link brain activity and behavior to person…

This study explores whether a digital tool can help monitor adults with chronic myeloid leukemia who have stopped treatment for up to two years. Over 12 months, researchers will track how often patients use the tool, aiming for a usage rate of at least 60%. The goal is to see if …

This study measures specific substances (metabolites) in the breath and blood of people with inherited metabolic disorders, those on a special ketogenic diet for epilepsy, and healthy volunteers. The goal is to see if a simple breath test can reliably track these metabolites, pot…

This study will compare custom 3D printed splints made at the hospital with those made by outside companies for jaw surgery. The splints help surgeons align the teeth and jaws correctly during the operation. Researchers want to see if the in-house splints are faster to get, cheap…

This study looks at whether the ratio of the second to fourth finger (2D:4D) is linked to anxiety and work stress in anesthesiology residents. Researchers will measure finger lengths and use a questionnaire to assess worry and anxiety in about 116 residents aged 20-35. The goal i…

This study follows about 146 mother-infant pairs to see if tongue-tie (ankyloglossia) affects how well breastfeeding goes. Newborns are checked for tongue-tie within their first 72 hours using the TABBY tool, and breastfeeding effectiveness is measured with the LATCH scale. The r…

This study investigates how often muscle relaxants used during general anesthesia remain in the body after surgery, a condition called residual neuromuscular blockade. It will follow 160 adults aged 18-90 who are scheduled for post-anesthesia care unit admission after elective su…

This study uses brain scans to see how epilepsy changes a brain chemical called adenosine in areas that control breathing and wakefulness. Researchers will compare 50 people with drug-resistant epilepsy to healthy volunteers. The goal is to better understand why some epilepsy pat…

This study tests a new way to deliver two common Parkinson's medications, carbidopa and levodopa, using a small pouch placed between the cheek and gum. The pouch releases the drugs slowly, allowing absorption through both the cheek lining and the stomach. Researchers are measurin…

This observational study compares corneal thickness six weeks after routine cataract surgery in people with Fuchs endothelial corneal dystrophy (FECD) versus those without the condition. Researchers aim to see whether FECD eyes swell more or recover differently after surgery. Par…

This study follows adults having cataract surgery who choose either a trifocal or monofocal intraocular lens implant. It asks whether one type leads to more difficulty or visual disturbances, like glare or halos, when driving at night. Participants must be licensed drivers with r…

This study looks at whether people with acid reflux or a hiatal hernia can safely skip a test called esophageal manometry before their surgery. The test, which involves a thin tube through the nose to measure throat muscles, can be uncomfortable and delay surgery. Researchers wil…

This study investigates Marsili syndrome, a rare genetic condition that reduces sensitivity to pain and injury. Researchers will apply heat, pressure, itch-inducing substances, and knee injections to a small group of people with the condition, measuring their pain and itch respon…

This study looks back at the medical records of 200 people who received a SONATA 2 cochlear implant. The goal is to understand how well the implant works over time in real-world settings, including speech recognition in quiet and noisy environments. Researchers will also analyze …

This study uses a non-invasive brain stimulation technique called TMS to temporarily change activity in a brain region linked to anxiety. Researchers will use fMRI scans to see how this affects brain connections and anxiety-related startle responses. 140 adults with anxiety will …

This study looks at how blood test results change after delivery in people with severe preeclampsia. Participants are randomly assigned to receive magnesium for either 12 or 24 hours after birth. The goal is to see if a shorter treatment is safe and effective, and to understand h…

This study is testing a program called Mothers and Babies for Down Syndrome (MBDS) to help prevent depression in new and expectant parents of infants with Down syndrome. About 72 parents will take part in a small pilot study to see if the program helps with feelings of grief and …

This study looks at how anticipated stigma and illness identity affect quality of life in men with hypogonadism (low testosterone). It compares these men to those with other chronic endocrine conditions like diabetes, hypothyroidism, or hypertension. The goal is to see if these p…

This study looks back at medical records of people with congenital thrombotic thrombocytopenic purpura (cTTP), a rare inherited blood disorder that causes dangerous blood clots. Participants received a manufactured version of the missing ADAMTS13 enzyme through an early access pr…

This study looks at how beta-cells (which make insulin) respond to growth hormone in people with prediabetes. Researchers will measure insulin release in 10 adults with impaired fasting glucose or impaired glucose tolerance. The goal is to better understand what raises the risk o…

This exploratory study looks at whether the community of bacteria in the gut and certain related chemical byproducts differ between people with Parkinson's disease and healthy individuals. Researchers will collect one stool sample from 15 people with Parkinson's and 15 matched he…

This study investigates how brain signals relate to freezing of gait and balance problems in people with Parkinson's disease. Participants, who already have a deep brain stimulation (DBS) device, will walk in a virtual reality home-like environment while their brain activity is r…

This study aims to develop and test an AI system that uses smartphone photos with a color card to measure redness in psoriasis lesions. Fifty adults with psoriasis will take weekly photos at home and have clinic photos taken. The goal is to see if this simple, low-cost method mat…

This study tests whether a non-invasive brain stimulation technique called theta burst stimulation (TBS) can change how people remember the order of emotional movie clips. Researchers will compare active TBS to a sham (fake) version in 120 healthy adults. The goal is to understan…

This trial tests whether a scenario-based simulation training session can reduce anxiety, boost self-confidence, and improve clinical skills in nursing students when counseling pregnant women about nutrition. Half of the participants will take part in a 20-minute simulated counse…

This study investigates how often older adults in China experience anxiety and depression before undergoing major non-cardiac surgery. Researchers will use standard questionnaires to screen 8,000 patients aged 65 and older across multiple hospitals. The goal is to better understa…

This study follows 200 children and teens aged 7-17 who are at higher risk for depression, anxiety, or obsessive-compulsive disorder (OCD) due to family history or early symptoms. Over two years, researchers will use interviews, questionnaires, brain scans (EEG and MRI), and biol…

This study explores whether short, AI-generated videos from a virtual doctor can help people with psoriasis stick to their topical medication routine. Fifteen adults will be split into three groups: weekly AI videos, weekly human doctor videos, or standard care (one follow-up vis…

This study looks at people with primary biliary cholangitis (PBC), a chronic liver disease that can lead to cirrhosis and liver failure. Researchers will review medical records from hundreds of patients to identify risk factors for faster disease progression. The goal is to build…

This observational study will track 87 people who are already scheduled to receive treatment with the Recana Thrombectomy Catheter System for blocked veins in their legs. The goal is to see how safe the device is and how well it works at clearing the blockages during the procedur…

This early-stage study looks at how a common tuberculosis drug, rifampin, affects levels of a new HIV prevention medicine called MK-8527. Fourteen adults with latent TB (no active symptoms) will take MK-8527 with and without rifampin. The goal is to see if the two drugs can be us…

This study looks at how a small opening in the heart, called a patent foramen ovale (PFO), affects blood flow and oxygen levels during exercise and simulated high altitude. About a third of people have this opening, which may allow oxygen-poor blood to mix with oxygen-rich blood,…

This study is looking for biological markers in the body that might predict how well people with treatment-resistant psychiatric disorders—like schizophrenia, depression, bipolar disorder, OCD, and catatonia—respond to new treatments. Researchers will collect blood, stool, cerebr…

This study looks at the long-term urinary, sexual, and bowel health of people aged 7 to 25 who had surgery for Hirschsprung disease as young children. Hirschsprung disease is a rare birth defect where part of the large intestine lacks nerve cells, causing blockages. Surgery remov…

This study investigates how common malnutrition and sarcopenia (muscle loss) are in children and adolescents with primary ciliary dyskinesia (PCD), a rare genetic condition affecting the lungs. Researchers will measure grip strength, body composition, and nutritional risk in 15 p…

This study investigates why children with inflammatory bowel disease (IBD) have an overactive immune response in the gut, and whether blood stem cells can be engineered to restore tolerance. Researchers will analyze blood and intestinal tissue samples from 80 children with IBD, w…

This study compares how well adults with hemophilia (ages 18–35) perform a 30-second sit-to-stand test versus healthy peers of the same age. The goal is to see if this simple test can detect differences in leg strength and endurance linked to joint damage from bleeding. Participa…

This study asks whether children and adolescents with primary ciliary dyskinesia (PCD) — a rare condition affecting the lungs — have poorer nutrition and muscle health than healthy children their age. Researchers will measure hand grip strength, jump power, and other physical and…

This study looks at how the absence of a protein called Dp140 in people with Becker muscular dystrophy might affect the brain, thinking, and muscle function. Researchers will use brain and muscle imaging, along with cognitive and psychological tests, to explore these connections.…

This study explores whether polycystic ovary syndrome (PCOS) independently contributes to fatty liver disease in adolescent girls, beyond the effects of obesity. Researchers will compare three groups of girls aged 10–18: those with PCOS and obesity, those with obesity alone, and …

This study looks at whether a hormone called preptin, which is released alongside insulin, is linked to insulin resistance in women with polycystic ovary syndrome (PCOS). Researchers will measure preptin levels in 60 women diagnosed with PCOS and compare them to markers of insuli…

This study follows people with Multiple Acyl-CoA Dehydrogenase Deficiency (MADD) over time to learn how the condition progresses and affects daily life. Researchers will collect health data and patient feedback to identify patterns and potential markers of the disease. The goal i…

This observational study tests whether wearable sensors and digital health tools can reliably measure falls, movement, speech, and thinking skills in people with progressive supranuclear palsy, Parkinson's disease, or multiple system atrophy. Participants wear a pendant sensor an…

This quality improvement initiative tests whether a standardized electronic medical record template for progress notes, combined with a pharmacist-led medication titration service, can improve the management of polycythemia vera (PV). The study enrolls hematologists and oncologis…

This study is testing a new, investigational eye scanner that uses light waves to measure the eye before cataract surgery. Researchers want to see if its measurements match those of a standard device, and whether an artificial intelligence model can better predict the correct len…

This observational study at Sohag University Hospital will look at children from birth to 18 years old who have congenital anomalies of the kidney and urinary tract (CAKUT). Researchers will collect data from medical records and routine follow-ups to describe the types of anomali…

This study follows 100 adults with myotonic dystrophy (types 1 or 2) for two years to understand how muscle stiffness, daily function, and heart health change over time. Researchers will look back at up to 18 months of past medical records and then track participants with clinic …

This study is testing whether a new digital tool—using AI-powered video games and a single camera—can accurately measure movement problems in people with Friedreich ataxia, a rare inherited condition that affects balance and coordination. Participants play short movement-based co…

This observational study is investigating whether vitamin D levels are linked to cholesterol and other blood fats in obese adults with type 2 diabetes. Researchers will measure vitamin D status and lipid profiles in 150 participants from a diabetes clinic in Cairo. No treatments …

This study tests whether a specialized MRI technique, called hyperpolarized 13C-MRI, can detect early metabolic changes in gastrointestinal stromal tumors (GIST) during treatment with imatinib. Standard scans like CT and MRI only measure tumor size, which can take months to chang…

This study is testing whether a specialized eye scanner called the MONA IV can capture clear, detailed images of the retina's tiny cells and blood vessels. Researchers will use it in people with conditions like glaucoma or age-related macular degeneration, as well as in healthy v…

This study looks at whether deep brain stimulation (DBS) — a standard surgical treatment for Parkinson's disease — affects a person's thinking and movement in ways that could increase their risk of falling. Researchers will assess 20 patients before and three months after DBS sur…

This study looks at whether the stiffness of the cornea influences how much astigmatism (blurring from an irregularly shaped cornea) develops after cataract surgery in people with pseudoexfoliation syndrome, a condition that weakens eye tissues. Researchers will measure corneal b…

This study tests a personalized, digitally supported prehabilitation program for adults waiting for elective cardiac surgery. Participants receive education, psychosocial support, nutritional advice, and home-based exercise guidance during the routine 6-10 week preoperative perio…

This study follows 70 adults aged 50 and older who are having routine cataract surgery. Half have pseudoexfoliation syndrome, a condition that can weaken eye tissues, and half do not. Researchers measure corneal stiffness before surgery and at several points afterward to see whet…

This study investigates how a drug called Cobenfy KarXT, which affects the brain's cholinergic (acetylcholine) system, alters brain activity during memory and thinking tasks. About 30 people with essential tremor or epilepsy who are already scheduled for deep brain stimulation (D…

This study uses questionnaires and focus groups to understand the challenges children with spinal muscular atrophy (SMA) and their families face when moving between schools or starting a new educational stage. Researchers aim to identify barriers to inclusion and support, and to …

This study investigates whether the triglyceride-glucose (TyG) index, a simple blood-based measure of insulin resistance, can predict early signs of atherosclerosis in people with lupus nephritis. Researchers will measure carotid artery thickness and plaque in 156 adults with lup…

This lab study tests whether a cell-free mixture of molecules and particles released by human amniotic mesenchymal stromal cells can reduce inflammation, prevent scarring, and promote healing in gut tissue from people with inflammatory bowel disease (Crohn's disease or ulcerative…

People with inflammatory bowel disease (Crohn's disease or ulcerative colitis) have a higher risk of developing colorectal cancer. This study follows 800 adults with IBD who need a screening colonoscopy to look for precancerous growths called dysplasia. Researchers aim to underst…

This study investigates how the brain uses visual attention to help maintain balance. Healthy adults aged 18 to 35 will perform a visual attention task while their brain activity is recorded with EEG and their balance is measured. The goal is to understand the neural connections …

This study uses advanced MRI to examine whether a deformity in the upper spinal canal is linked to syringomyelia (fluid-filled cavities in the spinal cord) in people with Chiari malformation type I. Researchers will compare MRI scans from 50 adults with Chiari malformation, with …

This study explores whether at-home saliva samples can reliably measure key steroid hormones in people with congenital adrenal hyperplasia (CAH), a condition requiring lifelong hormone therapy. Currently, monitoring involves morning blood tests that miss daily hormone fluctuation…

This study tests whether an artificial intelligence model can estimate a person's risk of developing Parkinson's disease. Participants aged 50 and older who have certain early warning signs—like REM sleep behavior disorder, drops in blood pressure upon standing, or reduced sense …

This trial tests whether an artificial intelligence model called OCT-PRO can predict how well a person will see after cataract surgery more accurately than standard doctor predictions. The AI analyzes eye scans and other clinical data to forecast visual recovery. About 534 adults…

This study creates a biospecimen and data repository at Mayo Clinic to help evaluate patients with rare or undiagnosed genetic conditions. Participants, who have already had standard genetic testing without a diagnosis, will provide samples and medical data. A multidisciplinary t…

This study tracks how Sanfilippo syndrome type C, a rare genetic disorder that affects the brain, progresses over time. Caregivers of children and young adults aged 1 to 25 will record videos of daily activities and answer questionnaires using a smartphone app every six months fo…

This study investigates how often atrial fibrillation (AF), a type of irregular heartbeat, occurs in people with certain blood cancers who are starting a targeted cancer drug called a Bruton's tyrosine kinase inhibitor (BTKI). Researchers will follow 500 participants—400 starting…

This study compares two methods for aligning toric intraocular lenses during cataract surgery: a digital image-guided navigation system (Verion) and the traditional manual corneal marking technique. Researchers measure how closely the manual mark matches the digital reference in …

This study looks for VEXAS syndrome, a recently discovered genetic condition, in adults over 50 who are hospitalized with unexplained fever, inflammation, or blood abnormalities. Researchers will screen participants using clinical exams and genetic testing of the UBA1 gene. The g…

This study looks at saliva from people with osteoporosis to measure certain immune markers and an enzyme called MMP-8. Researchers also check participants' dental cavities. The goal is to better understand how osteoporosis might affect oral health. No treatments are given—only sa…

This study investigates whether people with central or peripheral vision loss develop heightened senses of taste and smell. Researchers will measure brain responses to sweet and vanilla flavors in 50 visually impaired adults and compare them to sighted controls. The goal is to un…

This study follows babies and young children (up to age 5) who have a genetic diagnosis of ataxia telangiectasia (A-T), a rare disease that affects movement and immunity. Researchers use brain and lung scans, blood tests, and movement analysis to spot early signs of disease progr…

This study follows 5,000 people with common inflammatory skin conditions—like eczema, psoriasis, and hidradenitis suppurativa—over up to 17 years to see how their disease severity changes. Researchers will collect medical history, treatments, and symptom scores to identify factor…

This study follows infants who had a positive newborn screening test for severe combined immunodeficiency (SCID), a group of genetic disorders that leave babies vulnerable to life-threatening infections. Researchers will track these children to identify the underlying causes of t…

This study looks at children with Von Hippel-Lindau (VHL) disease, a rare genetic condition that causes tumors in the brain and spinal cord. Researchers want to understand how these tumors grow over time and what factors predict when a child will need surgery. By reviewing medica…

This study looks at heart function and rhythm in children and young adults up to age 25 who have had surgery to repair Tetralogy of Fallot, a congenital heart defect. Researchers will use echocardiography, a 12-lead ECG, and Holter monitoring to detect changes such as arrhythmias…