Disease of genetic or genomic mechanism

This early-phase trial tests whether a bone marrow transplant from a partially matched relative can safely cure chronic granulomatous disease (CGD), a condition that causes severe infections and inflammation. Four people aged 4 to 65 with CGD and no perfect donor will receive che…

This early-stage trial is testing whether a gene therapy called NGGT001, injected under the retina, is safe and tolerable for people with Bietti's crystalline dystrophy, a rare inherited condition that causes progressive vision loss. The study enrolls about 11 adults with a confi…

This trial tests an experimental gene therapy called EA0010 in children aged 1 to 17 with hearing loss caused by mutations in the OTOF gene, who already have cochlear implants. The therapy is injected into one ear and aims to deliver a working copy of the OTOF gene to inner ear c…

This trial tests an experimental gene therapy called NGGT001 for people with Bietti Crystalline Dystrophy, a rare inherited condition that causes progressive vision loss. The therapy is delivered as a single injection under the retina, aiming to replace a faulty gene with a worki…

This trial tests an experimental gene therapy called MWAV201 in adults with Wilson disease, a genetic condition that causes copper to build up in the body. The treatment uses a harmless virus to deliver a corrected gene, potentially restoring the body's ability to remove copper. …

This trial tests a personalized cell therapy called GCAR1 in a single patient with metastatic alveolar soft part sarcoma (ASPS) that has relapsed and cannot be surgically removed. The therapy uses the patient's own immune cells, engineered to recognize and attack cancer cells dis…

This trial tests a gene therapy called ATSN-101, given as a one-time injection under the retina, for people with Leber congenital amaurosis caused by GUCY2D gene mutations. The goal is to see if the treatment is safe and can improve vision. Participants receive the therapy in one…

This trial tests an experimental gene therapy called OCU400 in people with retinitis pigmentosa or Leber congenital amaurosis, which are inherited conditions that cause progressive vision loss and can lead to blindness. The therapy is given as a single injection into the eye, wit…

This trial tests a new, lower-intensity combination of chemotherapy and radiation before a donor stem cell transplant for people with high-risk blood cancers or aplastic anemia. The goal is to see if this approach is safe enough to allow the donor cells to take hold and cure the …

This trial tests whether anti-CD19 CAR-T cell therapy can put severe lupus into remission. The treatment involves taking a patient's own immune cells, engineering them to target and destroy faulty B cells, and infusing them back. The study enrolls 12 adults with refractory system…

This study tests whether the HPV vaccine Gardasil 9 can trigger an immune response in people with idiopathic CD4 T cell lymphocytopenia (ICL), a rare condition that weakens the immune system. About 54 adults with ICL and some healthy volunteers will receive three doses of the vac…

This early-stage trial is testing a new drug called VOY-101 for people with advanced dry age-related macular degeneration (AMD) and geographic atrophy, a condition that causes blind spots. The drug is injected into the eye and aims to slow damage to the retina and preserve vision…

This trial tests whether a newer surgical technique, which combines sleeve gastrectomy with a rerouting of the digestive tract, can control acid reflux as well as the standard gastric bypass in adults with obesity. The new approach aims to keep the entire digestive tract visible …

This study looks at whether the way a toric intraocular lens is implanted during cataract surgery affects how well it stays in place. Toric lenses correct astigmatism, but if they rotate after surgery, vision can blur. The trial compares two techniques: one using a gel-like subst…

This study tests whether a lower dose of the drug cyclophosphamide, given after a stem cell transplant from a partially matched donor, can reduce serious infections in the first 100 days while still preventing graft-versus-host disease (a common complication where donor cells att…

This phase 2 trial tests the safety and effectiveness of avelumab, an immunotherapy drug, in people with thymoma or thymic carcinoma that has progressed after platinum-based chemotherapy. About 56 participants will receive avelumab intravenously every two weeks. The study aims to…

This phase 1 trial is testing whether amivantamab, an antibody drug given by IV, is safe and tolerable for people with high-grade malignant brain tumors like glioblastoma. The study enrolls about 12 patients with recurrent glioblastoma or certain molecular subtypes. Participants …

This study tests an oral drug called TAK-279 (Zasocitinib) in 207 adults with moderate-to-severe ulcerative colitis. Participants take either the drug or a placebo for 12 weeks, then everyone gets the drug for up to a year. The goal is to see if TAK-279 reduces bowel inflammation…

This trial compares two types of intraocular lenses implanted during cataract surgery: the Clareon PanOptix and the Tecnis Odyssey. About 266 adults with cataracts in both eyes will receive one lens type in each eye, and researchers will measure how well they see at various dista…

This study compares the real-world effectiveness of mirikizumab against standard biologic therapies for adults with ulcerative colitis or Crohn's disease. Researchers will track whether mirikizumab reduces the need for extra medical care, like emergency visits or treatment change…

This study tests a high-dose stem cell transplant for people with chronic granulomatous disease (CGD), a condition that weakens the immune system. Participants receive donor stem cells along with drugs to prevent rejection and graft-versus-host disease. The goal is to improve tra…

This study compares two types of corneal transplant surgery (UT-DSAEK and DMEK) in 160 people with Fuchs dystrophy, a condition that clouds the cornea. It also tests whether a drug called rho-kinase inhibitor can reduce loss of important eye cells after surgery. The goal is to fi…

This study tests a new medicine called ORKA-001 for adults with moderate-to-severe plaque psoriasis, a skin condition causing red, scaly patches. About 160 participants will receive either the drug or a placebo to see if it can completely clear the skin by week 16. The goal is to…

This early-stage study tests a single intravenous dose of a gene therapy called LY3884961 in 15 adults with Gaucher disease type 1. The goal is to see if it is safe and can reduce or replace the need for ongoing enzyme replacement or substrate reduction therapy. Participants must…

This phase 2 trial tests whether the drug afimetoran can safely control active systemic lupus erythematosus (SLE) better than a placebo. About 268 participants with lupus will receive either afimetoran or a placebo for 48 weeks, with an extension period allowing placebo users to …

This study tests a weekly injection called TransCon CNP in children and teens with achondroplasia, the most common form of dwarfism. The goal is to see if it is safe and helps improve growth over several years. Participants who completed a previous TransCon CNP trial can join thi…

This study tests a drug called vudalimab in people with advanced anaplastic thyroid cancer or aggressive hurthle cell thyroid cancer that has spread. The drug helps the immune system attack cancer cells and may slow tumor growth. About 54 participants will receive the treatment t…

This study tests a gene therapy for people with X-linked chronic granulomatous disease (X-CGD), a genetic condition that weakens the immune system and leads to severe infections. Researchers take the patient's own stem cells, add a working copy of the faulty gene, and return the …

This phase 3 trial tests sefaxersen, a drug that targets a part of the immune system, in 428 people with IgA nephropathy at high risk of kidney decline. Participants receive either sefaxersen or a placebo as a shot under the skin. The main goal is to see if the drug reduces prote…

This trial tests whether adding an antibiotic to a blood-derived gel (platelet-rich fibrin) placed in the socket after wisdom tooth removal improves recovery. It compares the antibiotic-enriched gel to a standard gel in the same person, measuring swelling, pain, and infection ris…

This study tests a new ultraviolet femtosecond laser for creating corneal flaps during LASIK surgery to correct refractive errors. About 128 participants in Australia will receive LASIK using either the new UV laser or an existing infrared laser. The main goal is to check how saf…

This Phase 3 trial tests a new injectable drug called IBI306 in 198 Chinese adults with non-familial high cholesterol and mixed high lipids. Participants receive either IBI306 or a placebo every 2 or 4 weeks for 12 weeks. The main goal is to see how much the drug lowers LDL (bad)…

This study tests a new treatment called Orca-T for people with advanced blood cancers like leukemia. It uses a special mix of stem cells and immune cells from a donor to help the body accept the transplant while reducing serious side effects like graft-versus-host disease. The go…

This study looks at the long-term safety of the drug brivaracetam in children and teenagers with childhood absence epilepsy or juvenile absence epilepsy. About 120 participants who have already taken the drug in earlier studies will continue treatment and be monitored for side ef…

This study tests the Sentio bone-anchored hearing system in 50 children aged 3 to 11 with hearing loss or single-sided deafness. The device is already approved for older kids and adults. Researchers want to see if it safely improves hearing compared to no aid. The trial is happen…

This study tests a new medicine called bimekizumab against an existing one (ustekinumab) for children and teens aged 6 to 17 with moderate to severe plaque psoriasis. The goal is to see if bimekizumab works better at clearing skin symptoms. About 168 participants will receive inj…

This phase 2 study tests a combination of two nucleoside drugs, doxecitine and doxribtimine, in 47 people with thymidine kinase 2 (TK2) deficiency, a rare genetic disorder that weakens muscles. Participants already receiving nucleoside therapy continue treatment to see if the dru…

This study tests whether stem cells from donated umbilical cords can help boys with Duchenne muscular dystrophy (DMD). The cells are given through a vein and are meant to reduce inflammation and improve muscle function. The trial includes boys aged 5 to 10 who can still walk and …

This study tests a treatment made from healthy donor stool (MTP-101-C) for cancer patients whose skin or gut side effects from immunotherapy don't get better with steroids. About 30 participants will take the oral capsules to see if the therapy is safe and can calm these side eff…

This early-stage trial is testing a new drug called ABBV-291 in adults with Non-Hodgkin's lymphoma that has come back or stopped responding to treatment. The study will first find the safest dose, then check if the drug can shrink tumors. About 165 participants will take part wor…

This clinical trial is testing whether risankizumab, an experimental drug, can safely and effectively maintain remission in people with moderate to severe Crohn's disease who have already responded to initial treatment. Participants will receive either risankizumab or a placebo f…

This study tests a new drug called zifibancimig for people with wet age-related macular degeneration (AMD), a leading cause of vision loss. The drug is given either as an eye injection or through a small implant placed in the eye that releases the drug over time. The trial involv…

This study tests if adding another drug (belzutifan or quavonlimab) to a standard two-drug treatment can help people with advanced clear cell kidney cancer live longer or stop their cancer from growing. About 1,650 adults who have not had prior treatment for advanced disease will…

This study tests a drug called GLM101 for people with PMM2-CDG, a rare inherited disease that affects movement and coordination. About 50 children and adults will receive weekly infusions of either GLM101 or a placebo for 24 weeks, followed by an open-label phase where everyone g…

This study checks the long-term safety of tofersen (Qalsody) in 12 Chinese adults with a specific genetic form of ALS (SOD1-ALS). Participants receive 13 doses of the drug injected into the spine over about a year. The main goal is to track any side effects and how the drug moves…

This phase II trial is testing whether a combination of talimogene laherparepvec (a modified herpes virus that infects and kills cancer cells) and nivolumab (an immunotherapy drug) can shrink tumors in people with rare skin cancers or lymphomas that haven't responded to standard …

This study tests a drug called selumetinib in adults with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be removed by surgery. The goal is to see if the drug can shrink these tumors or slow their growth. Participants take the drug twice daily and are monitored …

This study tests a new growth hormone medicine called somapacitan, given once a week, in 47 children who are very short due to being born small for gestational age, or having Turner syndrome, Noonan syndrome, or idiopathic short stature. The main goal is to see if it is safe and …

This study offers open/aftercare treatment to adults with obsessive-compulsive disorder (OCD) who have completed an earlier study at Stanford's Rodriguez Lab. Participants receive evidence-based psychotherapy and/or pharmacotherapy from trained professionals. The goal is to see i…

This study tests whether giving the drug anakinra early can prevent severe cytokine release syndrome (CRS) in children and young adults with B-cell acute lymphoblastic leukemia (B-ALL) who are receiving CAR-T cell therapy. Participants receive anakinra at the first sign of fever,…

This study tests a drug called litifilimab for people with lupus-related skin disease that didn't get better with standard antimalarial pills. About 450 adults with active skin lesions will receive either the drug or a placebo every 4 weeks for 24 weeks, then everyone gets the dr…

This study compares a targeted drug called selumetinib to standard chemotherapy (carboplatin/vincristine) in children aged 2 to 21 with neurofibromatosis type 1 (NF1) and low-grade glioma, a type of brain tumor. The goal is to see if selumetinib works as well or better at control…

This early-phase trial tested whether combining two anti-cancer drugs, pomalidomide (taken as a pill) and liposomal doxorubicin (given through an IV), is safe and tolerable for people with advanced or hard-to-treat Kaposi sarcoma. The study enrolled 62 adults, including those wit…

This trial investigates whether delivering a mucus-thinning medication (NAC) with positive airway pressure improves its ability to clear mucus plugs in the lungs of people with asthma or COPD. Participants will receive NAC plus albuterol through either a standard nebulizer or a d…

This study tests a medicine called concizumab for people with hemophilia A or B who do not have inhibitors. The goal is to see if a daily shot under the skin can prevent bleeding episodes safely. About 156 participants, aged 12 and older, will either start concizumab right away o…

This trial compares the drug ruxolitinib against the best available standard treatments for people with high-risk polycythemia vera (PV) or essential thrombocythemia (ET), two rare blood cancers that cause overproduction of blood cells and can lead to clots, bleeding, and severe …

This phase 2 trial tested vismodegib, a pill that targets a specific growth pathway, in 45 people with advanced chondrosarcoma (a rare bone cancer). The goal was to see if the drug could shrink tumors or stop them from growing. Participants had to have measurable disease and no m…

This phase 3 study tests two doses of crizanlizumab against a placebo in 255 adolescents and adults with sickle cell disease who have frequent pain crises. The goal is to see if the drug can reduce the number of severe pain episodes that require a hospital or clinic visit. Partic…

This study is testing a new type of hip resurfacing device made entirely of ceramic, designed for people with hip osteoarthritis, necrosis, or dysplasia. The goal is to see if it is as safe and effective as existing metal-on-metal devices, but without releasing metal ions into th…

This early-phase trial is testing a radioactive drug called 131I-TLX101, given alongside standard care, for people newly diagnosed with glioblastoma, an aggressive brain cancer. The study involves 12 participants and aims to find the safest dose and check for side effects. It is …

This study tests satralizumab, a drug that calms inflammation, in 30 children aged 8 to 17 with Duchenne muscular dystrophy. The goal is to see if it improves bone density and muscle function. Participants receive injections for several months while continuing standard steroid th…

This study tests deep brain stimulation (DBS) surgery for people with severe focal hand dystonia, including musician's dystonia, that hasn't improved with botulinum toxin. Five participants will have electrodes placed in brain areas that control movement, connected to a stimulato…

This phase 1 trial is testing an experimental pill called flonoltinib maleate in people with certain bone marrow tumors, including myelofibrosis, polycythemia vera, and essential thrombocythemia. The drug targets two proteins involved in abnormal blood cell growth. The study aims…

This study follows 284 people with blocked leg arteries (peripheral arterial disease) who are treated with a paclitaxel-coated balloon. The balloon is inflated inside the artery to open it and release a drug that helps prevent it from narrowing again. Researchers are tracking saf…

This trial explores whether adding vitamin D and iron supplements to usual OCD treatment can improve the ability to control impulses. It involves 60 people with OCD who will receive the supplements for 12 weeks. Researchers will use brain scans and behavioral tasks to see if thes…

This study tests a vaccine called AADvac1 in 146 people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement and thinking. The vaccine aims to target a protein linked to the disease and slow its progression. Participants will receive injections o…

This study follows about 200 people with Prader-Willi syndrome who are taking or starting VYKAT XR. Researchers will track side effects and how the drug affects their health over time. The goal is to gather more safety information, not to test if the drug cures the condition.

This study looks at the long-term safety of a drug called litifilimab for people with active lupus. About 864 adults who finished earlier studies will continue taking the drug for up to 3 years, along with their usual treatments. The goal is to see how safe it is and how well it …

This study tests whether a structured care program (called a transition bundle) helps people with COPD recover better after a serious flare-up. The program includes guided steps and regular check-ins. About 1,368 patients across 36 hospitals will take part. The goal is to see if …

This Phase 2 trial is testing dupilumab (Dupixent) in 17 people with aspirin-exacerbated respiratory disease (AERD), a condition that includes asthma, nasal polyps, and severe reactions to aspirin. The study aims to see if the drug can reduce markers of inflammation and improve s…

This study tests a daily injection called concizumab in children under 12 with hemophilia A or B, with or without inhibitors. The goal is to see if it safely prevents bleeding episodes. Participants receive daily shots under the skin for at least 2 years, and the study tracks how…

This study tests a granule (sprinkle) form of the drug selumetinib in children aged 1 to under 7 years who have neurofibromatosis type 1 (NF1) with painful, inoperable tumors. The goal is to find the right dose, check safety, and see if it shrinks tumors. About 36 children will t…

This long-term study tests the safety and effectiveness of bitopertin (DISC-1459) in people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), rare conditions that cause severe pain from sunlight exposure. About 230 participants who previously took bitoper…

This Phase 3 trial is testing STSP-0601, an injectable drug, to control bleeding episodes in people with hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The study will enroll 40 participants aged 12 to 70 and measure how well the drug s…

This study tests a new medicine called ALN-6400 for people with hereditary hemorrhagic telangiectasia (HHT), a condition that causes abnormal blood vessels and frequent nosebleeds. First, healthy volunteers will receive a single dose to check safety. Then, HHT patients will recei…

This phase 2 trial is testing whether a combination of two drugs, talazoparib and temozolomide, can shrink tumors in people with rare cancers that have spread. The study includes 14 participants with advanced rare solid tumors, such as pheochromocytoma or paraganglioma, who have …

This study looks at whether a daily dose of milk polar lipids (a type of fat found in milk) can improve cholesterol levels, reduce inflammation, and support gut health in adults with high cholesterol and abdominal obesity. Researchers will measure changes in blood fats and other …

This study tests the safety of a drug called ASP3021 (avacincaptad pegol) for people in Japan with geographic atrophy, an advanced form of age-related macular degeneration that causes vision loss. Twenty participants will receive monthly injections into the affected eye for 12 mo…

This trial tests whether a daily nutritional supplement, designed with artificial intelligence to target gut bacteria, can increase testosterone production in men aged 30-65 with functional secondary hypogonadism—a condition where the body doesn't make enough testosterone despite…

This Phase 2 trial tests whether the drug LM11A-31 can slow the progression of Progressive Supranuclear Palsy (PSP), a rare brain disorder that affects movement and balance. About 147 people with early-stage PSP will receive either the drug or a placebo twice daily for several mo…

This trial tests whether giving zinc supplements before and after colonic surgery in children can help protect the gut lining and reduce inflammation. Children aged 1 month to 18 years undergoing colonic surgery with anastomosis will be randomly assigned to receive either routine…

This Phase 3 trial tests whether bitopertin, a daily pill, can help people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) spend more time in sunlight without pain. About 183 participants aged 12 and older will take bitopertin or a placebo for 6 months. …

This trial investigates whether nicotinamide riboside (NR), a form of vitamin B3, can improve blood vessel health in the brain, memory, and physical function in people with Parkinson's disease. Participants are randomly assigned to take either NR or a placebo daily for 12 weeks. …

This study tests an experimental drug called LP352 to see if it can safely reduce seizures in children and adults with developmental and epileptic encephalopathies (DEE), a group of severe epilepsy syndromes. About 320 participants will receive either LP352 or a placebo, and rese…

This study offers continued access to the drug mitapivat for adults with pyruvate kinase deficiency who completed an earlier Agios-sponsored trial and cannot get the drug commercially. Only 6 participants are enrolled, and the main goal is to monitor side effects. The study does …

This early-phase study tests a new drug called INCB160058 in adults with myeloproliferative neoplasms, a group of rare blood cancers. The main goal is to check the drug's safety and how the body processes it. About 186 participants will receive the drug to find the right dose and…

This study follows about 500 people who already had cataract surgery with a special adjustable lens (RxSight LAL) or a standard lens. Researchers want to see how well the lens works over time, focusing on vision sharpness and correction of astigmatism. Participants just need to c…

This study tests a new medicine called zodasiran in people aged 12 and older who have a rare genetic condition that causes extremely high cholesterol (homozygous familial hypercholesterolemia). The goal is to see if the drug can safely lower bad cholesterol levels over 12 months.…

This phase 3 study is testing the long-term safety and effectiveness of LP352 (Bexicaserin) in 324 children and adults with developmental and epileptic encephalopathy (DEE), including Dravet and Lennox-Gastaut syndromes. Participants who completed earlier studies will receive LP3…

This study tests a drug called blinatumomab, given as a shot under the skin, combined with low-intensity chemotherapy for people aged 60 and older (or younger adults who are not healthy enough for strong chemo) with B-cell acute lymphoblastic leukemia. The goal is to see if this …

This early trial is testing whether the drug olaparib can shrink tumors in people with newly diagnosed BRCA-mutant ovarian, fallopian tube, or peritoneal cancer before they have surgery. Fifteen participants will receive olaparib for two cycles, then undergo standard chemotherapy…

This Phase 2 trial tests solrikitug, a biologic drug given as a shot, in 171 adults with moderate-to-severe COPD and high eosinophil levels. The study compares two doses of solrikitug against a placebo to see if it safely reduces inflammation and improves lung function over time.…

This study tests an experimental eye drop called ZOC2017217 in 40 people with age-related cataracts. Participants will use the drops twice a day for 24 weeks to see if it improves vision and lens clarity compared to a placebo. The goal is to find a non-surgical option for catarac…

This trial tests whether losartan, a common blood pressure medication, can reduce scarring in the heart's main pumping chamber and improve exercise capacity in adults who had surgery to repair coarctation of the aorta. Participants will take losartan, amlodipine, or a placebo dai…

This study tests an experimental drug called VX-407 in 24 adults with autosomal dominant polycystic kidney disease (ADPKD) who have specific changes in the PKD1 gene. The goal is to see if the drug can slow kidney growth and is safe. Participants take VX-407 tablets by mouth and …

This study tests a combination of three drugs—ipilimumab, nivolumab, and brentuximab vedotin—in people with Hodgkin lymphoma that has returned or not responded to treatment. The drugs work by helping the immune system attack cancer cells and by delivering a toxin directly to them…

This study tests a new treatment using a patient's own immune cells that are genetically modified to better recognize and attack certain solid tumors. It is for people with advanced cancers like synovial sarcoma that have not responded to other treatments. The goal is to see if t…

This phase II trial is testing whether adding the drug asciminib to standard treatment can improve outcomes for people newly diagnosed with Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL). The study will enroll 55 participants and involves several phases o…

This study tests a special drilling technique called osseodensification to widen narrow jawbone so dental implants can be placed without extra bone grafting. Thirty adults with healed tooth sockets and narrow ridges will have their bone width measured before and after the procedu…

This early-stage study tests the safety of acamprosate, a drug used for alcohol dependence, in 30 adults with ALS who have a specific mutation in the C9orf72 gene. Participants take the pill three times daily for 24 weeks and undergo regular checkups, breathing tests, and spinal …

This study looks at how well and how safely the drug mirikizumab works over the long term for people with Crohn's disease. About 996 adults who were in earlier mirikizumab studies will continue treatment. The goal is to see if the drug can keep the gut healed and symptoms under c…

This Phase 3 study tests whether a gel containing patidegib can lower the number of new basal cell carcinomas (a type of skin cancer) on the face of adults with Gorlin syndrome. Participants apply the gel or a placebo to their face twice daily for a year. The study will compare h…

This phase 2 study tests two drugs, sunitinib and temsirolimus, in 137 people with advanced rare tumors that have no cure. The goal is to see if these drugs can shrink tumors or slow their growth. Participants take one of the two drugs, and researchers measure how long the cancer…

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the…

This phase II trial tests whether a new drug called Briquilimab can make stem cell transplants safer for people with GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of infections and blood cancers. The study involves 13 participants a…

This pilot study tests a new surgical procedure for people with anterior shoulder instability, where the shoulder repeatedly dislocates. The procedure uses a semitendinosus tendon graft to create a sling around the subscapular tendon and form a new labrum, aiming to hold the shou…

This phase 2 trial is testing several new drug combinations for people with aggressive B-cell lymphoma that has come back or not responded to standard therapy. About 129 participants will receive one of several experimental combinations, including drugs like ibrutinib and selinex…

This study looks at the long-term safety and effectiveness of the drug combination Trikafta (elexacaftor/tezacaftor/ivacaftor) in people with cystic fibrosis who have certain non-F508del gene mutations. About 297 participants who completed a prior study will continue taking the m…

This trial is testing an investigational drug called DYNE-251 in boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The goal is to see if the drug can safely help their muscles produce dystrophin, a protein that is missing in DMD. Participants recei…

This study tests a single dose of SGT-001 gene therapy in 12 boys (children and teens) with Duchenne muscular dystrophy. The main goal is to check safety and how well the body tolerates the treatment. Participants will be followed for about 5 years to monitor side effects and any…

This trial investigates whether a long-acting liquid stimulant medication (Quillivant XR) can safely and effectively reduce ADHD symptoms in children with Down syndrome, who are at higher risk for ADHD but often go untreated. The study will compare the medication against a placeb…

This study tests a new medicine called INZ-701 in babies up to 1 year old who have a rare genetic condition (ENPP1 deficiency) that causes severe hardening of the arteries and bone problems. The goal is to see if the drug can raise a key substance in the blood, improve survival, …

This phase 2 trial tests an experimental drug called tominersen in 301 people with early-stage Huntington's disease. The drug aims to lower levels of the harmful mutant protein that causes the disease. Researchers will compare tominersen against a placebo to see if it can slow do…

This study tests a new way to treat adults with a specific type of leukemia (Ph+ ALL) that has come back or not responded to treatment. The approach combines low-intensity chemotherapy with two targeted drugs, ponatinib and blinatumomab, to kill cancer cells and boost the immune …

This study tests whether metformin, a widely used diabetes drug, can safely slow vision loss in people with ABCA4 retinopathy (Stargardt disease). Fifty-five participants aged 12 and older will take metformin by mouth for 24 months, with regular eye exams to track changes. The go…

This program offers continued free access to the experimental drug BIA 28-6156 for people with Parkinson's disease who have a specific change in the GBA1 gene and have already completed the ACTIVATE study. Participants take one 60mg pill daily. The goal is to provide ongoing trea…

This study tests the long-term safety and effectiveness of an experimental drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). Participants who completed a previous VX-670 study can join. The drug is given through a vein, and researchers will monitor side effects a…

This early-stage trial is testing an experimental biologic called MK-1484, given alone or with the immunotherapy drug pembrolizumab (Keytruda), in adults with advanced or metastatic solid tumors. The main goal is to assess the safety and tolerability of the treatment and to find …

This early study tests if a laser can safely destroy small thyroid cancers (papillary thyroid microcarcinoma) without removing the thyroid. Ten adults with small tumors will receive laser treatment, then have their thyroid removed 6 months later to check if the cancer is fully go…

This study tests whether polyphenols from muscadine wine, served as a non-alcoholic beverage, can improve cognitive performance, memory, mood, and anxiety in healthy adults over 50. Participants will drink either the polyphenol-rich beverage or a placebo and complete a series of …

This study tests a new 3D-printed implant for adults with hip dysplasia, a condition where the hip socket doesn't fully cover the thigh bone, causing pain and gait problems. Ten participants aged 18-45 will receive the implant to see if it is safe and helps improve hip function a…

This study tests the long-term safety and effectiveness of XEN1101 (azetukalner) in 880 adults with focal or tonic-clonic seizures. Participants who completed earlier studies will take XEN1101 capsules for up to 6 years. Researchers will track side effects and changes in monthly …

This study tests the drug zanubrutinib (Brukinsa) in 18 Chinese adults who have Waldenström macroglobulinemia but have not yet received any treatment. The goal is to see how well the drug controls the disease and to check for side effects. Participants take zanubrutinib by mouth,…

This study tests a new drug called INZ-701 in up to 16 infants (up to 1 year old) with rare genetic conditions (ENPP1 or ABCC6 deficiency) that cause dangerous calcium buildup in arteries and other problems. The main goal is to check the drug's safety and how the body handles it,…

This study tests whether drinking 15 ml of apple cider vinegar twice a day for two weeks can improve blood sugar stability and cholesterol in adults with type 2 diabetes. Thirty-eight participants will compare the vinegar to plain water using a continuous glucose monitor. It's a …

This study tests a gene therapy called delandistrogene moxeparvovec in 13 children under age 4 with Duchenne muscular dystrophy. The goal is to see if it is safe and can help produce a key muscle protein. The children will be followed for about 5 years.

This early-phase trial tests a single intravenous dose of LY-M001 gene therapy in 9 children (ages 6 to 17) with type 1 Gaucher disease. The goal is to see if it is safe and can improve key symptoms like liver size and blood markers. Researchers will monitor participants for side…

This phase II trial tests inotuzumab ozogamicin, a drug that targets and kills leukemia cells carrying a specific marker (CD22). It enrolls 40 adults with B-cell acute lymphoblastic leukemia who are in remission but still have minimal residual disease (MRD) — tiny amounts of canc…

This study tests whether frozen capsules containing processed stool from healthy donors can help people with mild-to-moderate active ulcerative colitis. Participants will take either the real capsules or a placebo for 12 weeks. The goal is to see if the treatment can bring the di…

This study is checking the long-term safety of a drug called dapirolizumab pegol for people with systemic lupus erythematosus (lupus). It involves 760 participants who have already completed earlier studies of the drug. Researchers will monitor side effects over time to see if th…

This early-stage trial is testing an experimental drug called MK-0472, alone or combined with the immunotherapy Keytruda or another experimental drug MK-1084, in people with advanced or metastatic solid tumors. The main goals are to check safety, find the right dose, and see how …

This study tests a combination of two drugs—venetoclax and vincristine—in people with T-cell or B-cell acute lymphoblastic leukemia that has returned or not responded to prior treatment. The goal is to find the safest dose and see if the combo can lead to remission. About 40 adul…

This phase 3 trial is testing the safety and tolerability of fenfluramine (Fintepla) in 25 infants aged 1 to 2 years with Dravet syndrome, a severe form of epilepsy. The drug is given as an oral solution twice daily alongside other seizure medications. Researchers will monitor he…

This early-stage trial is testing whether a single same-day treatment using a patient's own bone marrow cells is safe and tolerable for people with Parkinson's disease or related conditions. The procedure involves drawing bone marrow from the hip, giving one portion through an IV…

This study is testing the long-term safety of a high-dose antioxidant called N-Acetylcysteine (NAC) in 30 adults with retinitis pigmentosa, an inherited eye disease that causes gradual vision loss and blindness. Participants take 1800 mg of NAC twice daily for two years, with reg…

This phase II study tests a vaccine called SurVaxM, designed to train the immune system to attack glioblastoma cells, when given alongside standard chemotherapy (temozolomide). The trial includes 66 patients with newly diagnosed, survivin-positive glioblastoma or gliosarcoma. The…

This study tests two different doses of TEV-48574 (duvakitug) given as a shot every 4 weeks to adults with moderate to severe ulcerative colitis or Crohn's disease. Participants who completed a previous 14-week study can join. The goal is to see if the drug can keep the disease u…

This study tests a drug called selumetinib in children and young adults (ages 3-18) with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be removed by surgery. The goal is to see if the drug can shrink or slow the growth of these tumors. Participants take the dru…

This early-phase study tests a new drug called MK-2870, either alone or with the immunotherapy pembrolizumab, in Japanese patients with advanced solid tumors or a type of lung cancer. The main goal is to check the safety and side effects of these treatments. About 30 people will …

This trial tests whether a technique called PICSI, which selects sperm by their ability to bind to hyaluronic acid, can improve pregnancy rates and reduce miscarriage compared to standard ICSI. It involves couples who had poor fertilization in their first IVF cycle and are now tr…

This early-phase study tests a single IV dose of NGGT002 gene therapy in 15 adults with phenylketonuria (PKU), a genetic disorder that causes harmful buildup of phenylalanine. The main goal is to check safety over one year, with long-term follow-up for five years. Researchers wil…

This study tests a mobile app called WiseApp along with a smart pill dispenser to help Spanish-speaking people with HIV take their medication regularly. About 248 participants from New York City and the Dominican Republic will use the app for 12 months. The goal is to see if the …

This study tests a specific timing of chemotherapy drugs (busulfan and fludarabine) before a donor stem cell transplant, followed by cyclophosphamide after the transplant, to treat high-risk blood cancers like leukemia and lymphoma. The goal is to reduce the chance of the transpl…

This phase 2 trial studies whether lower doses of the chemotherapy drug busulfan can help infants with severe combined immunodeficiency (SCID) achieve good immune function after a stem cell transplant, while reducing short- and long-term risks. The trial includes 56 babies receiv…

This study tested a simplified care plan for people with acute promyelocytic leukemia (APL), a rare blood cancer. The goal was to see if following easy-to-use guidelines and getting support from APL experts could lower the number of deaths soon after starting treatment. About 200…

This Phase 2 trial tests whether the experimental drug SAR441344 can safely control disease activity in people with active systemic lupus erythematosus (SLE). About 109 adults aged 18–70 will receive either the drug or a placebo for 24 weeks. The main goal is to see if more peopl…

This trial tests whether sodium butyrate, a short-chain fatty acid, can reduce protein in the urine and slow kidney function decline in people with IgA nephropathy, a kidney disease. Participants receive either sodium butyrate or a placebo for 12 weeks, then switch after a washou…

This study is testing a minimally invasive weight loss procedure called endoscopic sleeve gastroplasty (ESG) in 12 people who have both obesity and ulcerative colitis and are planning or have had colon removal surgery. The procedure uses a suturing device to reduce stomach size b…

This study tests a device called iFuse TORQ, which is surgically implanted to fuse the sacroiliac joint—a common source of lower back and buttock pain. The trial includes 110 adults who have had pain for at least 6 months and haven't found relief from non-surgical treatments. Res…

This Phase 3 trial tests a drug called GTX-102 (apazunersen) in 129 children with Angelman syndrome, a genetic condition causing severe developmental delays. The study compares the drug to a sham procedure to see if it improves cognitive function and other symptoms over about 11 …

This early study tests a new drug called ABBV-101 in people with several types of non-Hodgkin lymphoma that have come back or not responded to other treatments. The drug is a pill that targets a protein (BTK) involved in cancer growth. The main goals are to find a safe dose and s…

This study tests whether adding an experimental drug called NanO2 to standard radiation and chemotherapy can help people with newly diagnosed glioblastoma, an aggressive brain cancer. NanO2 is designed to deliver more oxygen to tumors, which may make radiation work better. About …

This trial is testing whether mebendazole, a drug commonly used to treat intestinal parasites, can help children with brain tumors called gliomas. The study combines mebendazole with standard chemotherapy drugs to see if it is safe and effective. It includes children and young ad…

This trial tests whether a non-invasive technique called temporally-interfering electric field stimulation (TIEFS) can reduce abnormal brain activity linked to epilepsy. Researchers will measure seizure-related signals in patients with medial temporal lobe epilepsy who are alread…

This study looks at whether using a CPAP machine during pregnancy can reduce the chance of developing high blood pressure disorders like preeclampsia. About 1,500 pregnant women with mild to moderate sleep apnea will be randomly assigned to use CPAP or not. The goal is to see if …

This study follows people who previously received an experimental gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. The goal is to see how safe and effective the treatment remains over up to five years after the injection.…

This early-stage study tests a new drug called AZD1705 in adults with high cholesterol (dyslipidemia). The main goal is to check if the drug is safe and how the body processes it. About 98 participants will receive either the drug or a placebo. This research helps determine if th…

This early-stage trial tests a new treatment called P-CD19CD20-ALLO1, a type of CAR-T cell therapy, for people with severe lupus that hasn't improved with standard treatments. The study will enroll 162 participants and aims to check if the therapy is safe and tolerable. It also l…

This study tests a simplified, accelerated HIV care model called ACCELERATE. It combines rapid telehealth appointments, a pre-approved HIV medication, and a free 30-day starter supply to help people with HIV who have fallen out of care get back on track. The goal is to see if thi…

This early-stage study tests a new cell therapy for adults with a hard-to-treat type of leukemia (ALL). Doctors take a patient's own white blood cells, add a special gene to help them find and kill cancer cells, and give them back after chemotherapy. The main goals are to check s…

This study explores whether regular aerobic exercise, specifically walking about 300 minutes per week, can change certain biomarkers in the blood that are linked to cancer risk. Participants are adults who have completed all cancer therapy and have either clonal hematopoiesis (a …

This early-stage trial is testing an experimental oral drug, TYRA-200, in people with advanced bile duct cancer (intrahepatic cholangiocarcinoma) and other solid tumors that have specific changes in the FGFR2 gene. The study aims to find a safe and effective dose and to see wheth…

This study tests an investigational drug called relutrigine in 160 children with a severe form of epilepsy called developmental and epileptic encephalopathy (DEE). The goal is to see if the drug can safely reduce monthly seizure frequency compared to a placebo. After the initial …

This phase 3 trial tests EYP-1901, a new eye injection that may last longer than current treatments for wet age-related macular degeneration (AMD). About 400 people with wet AMD will receive either EYP-1901 or standard aflibercept injections over 2 years. The main goal is to see …

This study tests a new medication, linaprazan glurate, to see if it heals esophagus damage from acid reflux better than a standard drug. About 500 adults with erosive esophagitis will take either the new drug or lansoprazole daily for up to 8 weeks. Doctors will use cameras to ch…

This phase 3 trial is testing whether adding the immunotherapy drug atezolizumab to standard chemotherapy can help prevent cancer from coming back in people with stage III colon cancer that has a specific DNA repair defect (dMMR). About 712 participants will receive either chemo …

This study follows 155 people in Japan with hereditary angioedema (HAE) who are taking TAKHZYRO (lanadelumab) injections every two weeks. Researchers will monitor side effects and check if the drug helps control swelling attacks over 12 months. The goal is to see how well TAKHZYR…

This study compares two approved drugs, risankizumab and vedolizumab, in about 530 adults with moderate to severe ulcerative colitis who have not tried targeted therapies before. Participants will receive one of the two drugs for about a year. The main goal is to see which drug b…

This study tests a new drug called AZD7798 in 120 people with moderate to severe Crohn's disease. The goal is to see if it can reduce symptoms and improve the appearance of the gut lining. Participants are randomly assigned to receive either the drug or a placebo, and neither the…

This study is testing an experimental drug called ACE-86225106 in adults with advanced solid tumors that are difficult to treat or have stopped responding to standard therapies. The drug works by blocking a protein that helps cancer cells repair themselves, potentially slowing or…

This phase 3 trial tests EYP-1901, a new eye injection that may last longer than current treatments for wet age-related macular degeneration (AMD). About 400 participants will receive either EYP-1901 or the standard drug aflibercept. The goal is to see if EYP-1901 can maintain or…

This study compares two standard psoriasis creams—a steroid and a newer drug called tapinarof—to see which one causes less skin color change after treatment. Forty adults with darker skin types will use one cream daily on their legs for up to 12 weeks. Researchers will track pigm…

This study tests a new drug called elafibranor in people with primary biliary cholangitis (PBC), a rare liver disease that can lead to scarring and liver failure. Participants either did not get enough help from or could not take the standard medicine. The goal is to see if elafi…

This Phase III trial is testing whether adding the immunotherapy drug blinatumomab to standard chemotherapy helps adults with a newly diagnosed type of acute lymphoblastic leukemia (ALL) live longer and stay cancer-free longer. About 488 participants are randomly assigned to rece…

This study tests a daily tablet called BI 1291583 (Verducatib) in 268 adults with bronchiectasis, a long-term lung condition. Participants who were in earlier trials continue or switch to the drug for up to one year. The main goal is to check safety and see if the drug reduces lu…

This phase 3 trial compares two immunotherapy drugs—nivolumab (Opdivo) and brentuximab vedotin (Adcetris)—when added to standard chemotherapy for people aged 12 and older with newly diagnosed stage III or IV classic Hodgkin lymphoma. The goal is to see which combination better de…

This study tested a combination of the immunotherapy drug blinatumomab with either standard chemotherapy or dasatinib and prednisone in adults aged 65 and older with acute lymphoblastic leukemia (ALL). The goal was to see if the combination could improve survival and control the …

This study looks at a rare disease called KSHV-associated multicentric Castleman's disease (MCD), caused by a virus. Researchers are testing several drug combinations, including chemotherapy and antivirals, to see if they can control the disease. The study also tracks how the dis…

This early-stage trial is testing a new treatment called CC-97540, which uses a patient's own immune cells modified to target and destroy faulty immune cells. It is for people with severe forms of lupus, myositis, scleroderma, or rheumatoid arthritis that have not improved with s…

This study is testing three long-acting antibodies, alone or in combination, in 645 people with ulcerative colitis. The goal is to see if they are safe and can improve bowel inflammation over 48 weeks. Participants are joining from a previous study and may have already seen some …

This early-phase study tested a new gene editing treatment called HG302 in 4 boys with Duchenne muscular dystrophy (DMD), a severe muscle-wasting disease. The therapy uses CRISPR technology to fix the genetic error and help muscles produce dystrophin, a protein essential for musc…

This phase 2 trial tests the drug selpercatinib in children and young adults (ages 1 to 21) with advanced cancers that have a specific genetic change called a RET alteration. The goal is to see if the drug can shrink tumors or stop them from growing. Only one participant has been…

This study tests the long-term safety of an experimental drug called UCB0107 (bepranemab) in people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement and balance. Participants who completed a prior study receive regular infusions of the drug. …

This study tests whether guselkumab, given as a shot under the skin, can help people with moderate to severe Crohn's disease achieve remission and heal their gut lining. About 350 adults who have not responded well to other treatments will receive either guselkumab or a placebo. …

This study tests a daily pill called elafibranor in about 18 Japanese adults with primary biliary cholangitis (PBC), a rare liver disease that can lead to scarring and liver failure. Participants had not responded well to standard treatment. The main goal is to see if the drug im…

This study tests a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. About 714 adults aged 50-89 who currently need regular eye injections will receive either the gene therapy or standard treatment. The goal is to…

This study tests whether adding ASTX727 to the standard drug dasatinib can better control chronic myeloid leukemia (CML) in newly diagnosed patients. About 70 adults with a specific genetic marker (Philadelphia chromosome) will receive both pills. The goal is to see if the combin…

This early-stage study tests a new drug called RG6496 in people who carry the gene for Huntington's disease. The main goal is to check if the drug is safe and how the body handles it. About 40 participants will receive a single dose injected into the spine, with some getting a pl…

This trial is testing an experimental drug called PRAX-222 in children aged 2 to 18 who have a rare, severe form of epilepsy caused by changes in the SCN2A gene. The goal is to see if the drug is safe and can reduce how often seizures happen. Participants will receive either PRAX…

This large study tests whether guselkumab, an injected drug, can control symptoms and heal the gut lining in people with moderate to severe Crohn's disease. About 1,400 participants will receive guselkumab, an existing treatment (ustekinumab), or a placebo. The goal is to see if …

This study looks at the long-term safety and effectiveness of spesolimab in people with generalized pustular psoriasis (GPP), a rare and severe skin condition. All 131 participants had already taken spesolimab in a previous study and will continue treatment for up to 5 years. Res…

This trial tests whether a culturally adapted program of case manager support can improve survival, reduce hospital readmissions, and boost quality of life for adults recovering from accidental burn injuries in Pakistan. Participants receive up to eight sessions with a case manag…

This study is testing the long-term safety of a once-daily drug called vormatrigine in 700 adults with focal or generalized tonic-clonic seizures. Participants must have already taken vormatrigine in a previous trial or program. The main goal is to check for side effects and chan…

This study is testing whether the drug spesolimab can safely and effectively treat repeated flares of generalized pustular psoriasis (GPP), a serious skin condition. Adults with GPP who have had a flare will receive a single intravenous dose of spesolimab, with a possible second …

This phase 2 trial tests a one-time treatment called CC-97540, made from a patient's own immune cells engineered to target and calm the overactive immune system in lupus. The study enrolls 89 adults with active lupus (including kidney inflammation) who have not responded well to …

This study tests an oral medication called tofacitinib in children aged 2 to 17 with moderate to severe ulcerative colitis, a chronic bowel disease causing inflammation and bleeding. The goal is to see if the drug can bring the disease into remission (no symptoms and healed gut l…

This trial investigates whether ropeginterferon alfa-2b can help people with polycythemia vera, a condition where the body makes too many red blood cells. The study will compare the drug to standard care, which includes phlebotomy (removing blood) and aspirin. About 70 adults wit…

This study tests an experimental drug called GSK4771261 for safety and how the body processes it. Part A involves healthy volunteers, and Part B includes people with autosomal dominant polycystic kidney disease (ADPKD), a genetic condition that causes kidney cysts and can lead to…

This phase 2 trial tests an experimental drug called AOC 1044 in people with Duchenne muscular dystrophy whose genetic mutation makes them candidates for exon 44 skipping therapy. The drug is given by intravenous infusion. Researchers are evaluating long-term safety and whether i…

This trial tests whether a custom-made, absorbable polycaprolactone (PCL) scaffold, combined with the patient's own fat, can safely improve the appearance of pectus excavatum (a sunken chest) in people who cannot have standard corrective surgery. The scaffold is designed to suppo…

This trial tests whether implanting a fresh, donated corneal lenticule — a thin layer of corneal tissue — can safely correct residual farsightedness in people who already have an artificial lens implant after cataract surgery. The procedure uses a femtosecond laser to create a po…

This case report tests whether implanting a fresh corneal lenticule from a myopic donor, along with autologous serum drops, can safely thicken the cornea and improve vision in a person with advanced keratoconus. The procedure uses a femtosecond laser to place the graft. The goal …

This trial investigates whether implanting a fresh corneal lenticule — a thin layer of donor cornea taken from myopic patients — can improve transparency and vision in people with macular corneal dystrophy, a condition that clouds the cornea. Using a femtosecond laser, surgeons r…

This trial tests a one-time gene therapy injection for people with Charcot-Marie-Tooth disease type 1A (CMT1A), a genetic nerve disorder that causes progressive muscle weakness. The therapy delivers a gene that produces a protein to support nerve health. Three adults aged 18 to 3…

This trial is testing the long-term safety of fenofibrate, a drug usually used for high cholesterol, in people with primary biliary cholangitis (PBC), a rare liver disease. The study involves 15 adults who have already completed a previous fenofibrate trial for PBC. Researchers a…

This trial tests whether a corneal lenticule — a thin disc of tissue taken from a donor eye during a type of laser surgery — can be implanted into the cornea of people with progressive keratoconus, a condition where the cornea thins and bulges outward. The goal is to increase cor…

This trial tests whether a laser procedure called Relex-Smile can correct leftover nearsightedness or farsightedness in people who had cataract surgery six months earlier. About 130 participants with blurry vision after receiving a trifocal lens implant will undergo the laser tre…

This phase 3 trial is investigating the long-term safety and effectiveness of an experimental clotting factor called BIVV001 in people with severe hemophilia A. The study aims to see if regular infusions of this therapy can prevent bleeding episodes, protect joint health, and imp…

This trial tests whether a monthly injection of lerodalcibep can safely lower LDL cholesterol in children aged 6 to 17 with heterozygous familial hypercholesterolemia, an inherited condition that causes dangerously high cholesterol. Participants receive either the drug or a place…

This study tracks the safety and effectiveness of Spinraza (nusinersen) in people with spinal muscular atrophy (SMA) who receive the drug as part of routine care in Korea. Researchers will monitor for side effects and measure changes in motor skills over time. The goal is to see …

This UCLA study is testing whether the Autoimmune Protocol (AIP) diet — which avoids dairy, grains, and sugar — can reduce protein levels in the urine of people with IgA nephropathy, a kidney disease that can lead to kidney failure. Thirty adults aged 18 to 65 with active disease…

This trial explores whether stem cells taken from a patient's own bone marrow during surgery can help treat fistulas—abnormal tunnels under the skin—caused by Crohn's disease. The stem cells are prepared quickly in the operating room and injected into the fistula tract. Researche…

This study follows people who previously received a one-time gene therapy injection for a rare inherited eye disease caused by RPE65 mutations. Researchers will test vision and light sensitivity over up to 15 years to see how well the treatment holds up. The goal is to understand…

This study tests whether a topical cream containing ruxolitinib can safely restore skin color in people who develop white patches (vitiligo-like depigmentation) as a side effect of cancer immunotherapy. The cream is applied twice daily for 52 weeks. Researchers will measure how m…

This study looks at how well a surgical technique called AMIC holds up ten years after repairing cartilage damage in the ankle. Researchers are checking whether the pain relief and function patients gained in the first few years remain stable or fade over time. About 40 people wh…

This trial tests whether applying tranexamic acid (TXA) directly to the face during facelift surgery can reduce bleeding and bruising better than a placebo. TXA is a medicine that helps blood clots stay intact, and it is already used intravenously for heavy bleeding. Here, it is …

This phase II trial is testing whether combining two drugs—pembrolizumab, an immunotherapy that helps the immune system attack cancer cells, and entinostat, a drug that may make cancer cells more visible to the immune system—can shrink lymphomas that have come back or stopped res…

This trial investigates whether a single or repeat infusion of zoledronate, or daily oral alendronate, can maintain the bone density improvements achieved with denosumab in postmenopausal women with osteoporosis. Participants who have been on denosumab for at least three years wi…

This early-stage trial is testing an experimental drug called belzutifan in people with advanced solid tumors that have not responded to standard treatments. Belzutifan works by blocking a protein (HIF-2α) that tumors use to survive in low-oxygen conditions. The main goal is to f…

This trial investigates whether a 12- to 36-week program of aerobic and strength exercises can improve body composition, muscle function, and bone density in adolescents and adults with thalassemia. Participants will follow a self-directed routine of 30 minutes of exercise, 5 day…

This trial investigates the long-term safety of ecopipam, a drug that blocks dopamine D1 and D5 receptors, in children, adolescents, and adults with Tourette syndrome. Participants who completed a prior study may enroll and receive ecopipam for up to 24 weeks or longer. The main …

This trial tests three approaches to slow the progression of nearsightedness (myopia) in children aged 5 to 12: daily atropine eye drops, MiSight contact lenses, and NaturalVue multifocal contact lenses. Over two years, researchers will measure changes in eye length and prescript…

This phase II trial tests whether simvastatin, a widely used cholesterol medication, can reduce the size of uterine fibroids and ease related symptoms. Researchers will give 60 women either simvastatin or a placebo daily for 12 weeks, measuring fibroid volume by ultrasound and tr…

This study investigates whether intensively lowering systolic blood pressure with FDA-approved medications can reduce the buildup of amyloid and tau proteins in the brain—hallmarks of Alzheimer's disease. The trial enrolls adults aged 60-85 with high blood pressure who are cognit…

This study tests an experimental drug, BBP-418, in people with limb girdle muscular dystrophy type 2I (LGMD2I), a rare genetic condition that causes progressive muscle weakness. The drug aims to fix a molecular defect by helping a faulty enzyme work better. The trial includes bot…

This study tests an experimental drug, NPI-001 (AT-001), in people with hereditary cystatin C amyloid angiopathy (HCCAA), a rare genetic condition that causes repeated brain bleeding and stroke. The trial includes about 15 participants aged 12 and older who carry the specific L68…

This study investigates whether tirzepatide, a drug used for diabetes and weight loss, can reverse biological aging in people with HIV who are virally suppressed and have obesity. Participants receive tirzepatide for 25 weeks after a diet program. Researchers measure epigenetic a…

This trial compares two surgical techniques for treating cervical degenerative disc disease: using a stand-alone cage versus a cage-screw fixation system. Both aim to relieve pain and improve function by removing the damaged disc and fusing the vertebrae. The study measures pain …

This study tests whether giving patients hyperbaric oxygen (breathing pure oxygen in a pressurized chamber) after an umbilical cord blood stem cell transplant helps the donated stem cells grow faster. The trial enrolls people with blood cancers like leukemia or lymphoma who lack …

This study follows people with recessive dystrophic epidermolysis bullosa (RDEB), a severe blistering skin disease, who previously received an experimental treatment called EB-101. EB-101 uses gene-corrected skin cells to help wounds heal. Researchers will track wound closure, pa…

This study tests whether an experimental drug called D-2570 can help people with moderately to severely active ulcerative colitis achieve remission. About 120 adults will receive either D-2570 or a placebo daily for 12 weeks. The main goal is to see if more people on D-2570 reach…

This study tests whether transplanting a person's own fecal bacteria into the bowel before ileostomy reversal can improve bowel and anal function after rectal cancer surgery. Participants receive four weekly enemas made from their own stool. The goal is to reduce symptoms like le…

This study tests a new drug called ACC017 in adults with HIV that has become resistant to standard treatments. The trial aims to see if ACC017 can lower the amount of virus in the blood to undetectable levels. Participants receive either ACC017 or a placebo for two weeks, followe…

This study follows 300 people who receive a hip replacement using either the Vivacit-E or Longevity liner, both made of a special plastic designed to last longer. The goal is to see how well these liners hold up over time, measuring how many are still in place without needing rep…

This study tests a personalized antisense oligonucleotide drug designed for a single person with ALS caused by a specific TARDBP gene mutation. The drug aims to reduce harmful TDP-43 protein and slow disease progression. The participant's clinical function, survival, and biomarke…

This study follows over 3,300 people with HIV in Spain who switched from daily oral medication to a long-acting injectable combination of cabotegravir and rilpivirine. Researchers track how well the injections control the virus, how long patients stay on treatment, and any side e…

This phase 1 study tests whether adding a highly targeted, high-dose radiation boost (stereotactic body radiotherapy, or SBRT) to standard radiation and chemotherapy can safely treat head and neck cancer that has come back or spread. The trial includes two groups: patients whose …

This study tests a personalized medicine called an antisense oligonucleotide, designed specifically for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic brain disorder. The treatment aims to reduce seizures and improve quality of life by targeting the …

This study tests a program called PIXI that coaches parents of infants diagnosed with rare neurogenetic disorders (such as Fragile X, Angelman, or Down syndrome) during the first year of life. The program combines education about the disorder, guided parent-child interaction acti…

This study explores whether a very high-powered microscope can help diagnose acid reflux disease (GERD) in children by examining tiny gaps between cells in the esophagus. Researchers will compare biopsy samples from children with GERD, those with a related condition called eosino…

This study investigates whether cone-beam computed tomography (CBCT), a 3D imaging technique, can improve the diagnosis and surgical treatment of otosclerosis, a condition where abnormal bone growth in the middle ear causes hearing loss. Researchers will enroll 100 adults suspect…

This study investigates whether a combination of skin biopsies, speech analysis, and ultra-high field 7T MRI can detect early signs of Parkinson's disease and dementia with Lewy bodies. It focuses on people with a specific type of REM sleep behavior disorder triggered by serotone…

This trial is testing a new imaging tracer called FLT, which is used with a PET scan to measure how fast tumors are growing. The study includes people with various cancers or those awaiting a biopsy for a suspicious mass. The goal is to see if FLT PET imaging can help find and di…

This study enrolls about 4,000 adults aged 60 and older with heart failure who are at high risk for ATTR amyloidosis, a rare disease that stiffens the heart. Doctors receive special training on how to spot and diagnose the condition using standard tests. The goal is to see if thi…

This study looks at whether a special imaging technique called intraoperative optical coherence tomography (iOCT) can help doctors better diagnose and treat lens diseases like cataracts and lens dislocation. Researchers will review data from 1,200 patients who had lens surgery to…

This study compares a self-administered oral glucose tolerance test (OGTT) at home using a continuous glucose monitor (CGM) with the standard clinic-based OGTT. 75 adults with prediabetes will undergo both tests to see if the home version accurately diagnoses blood sugar problems…

This trial tests whether an AI tool called DeepBMD can identify people at high risk for osteoporosis using CT scans they've already had for other reasons. Researchers will compare the AI's findings with the standard bone density test (DXA) to see if it's accurate. If it works, th…

This study looks at whether a protein called ApoC3 in the blood can help identify insulin resistance in women with polycystic ovary syndrome (PCOS), a common cause of infertility and other health issues. Researchers will measure ApoC3 levels in 200 women with PCOS and compare the…

This study investigates how well different imaging methods—ultrasound, CT, and MRI—can identify the cause of neck swellings in children. Researchers will compare imaging results with the final diagnosis from biopsy, lab tests, or follow-up. The goal is to see if imaging can relia…

This study tests whether a special heart scan using a radioactive drug called 123I-MIBG can help identify Lewy body disease, a type of dementia that also affects movement and sleep. Researchers will compare scan results with clinical diagnoses in 50 people who have conditions lik…

This trial is developing an artificial intelligence system that reads electrocardiograms (ECGs) to detect congenital heart disease in children. Researchers are collecting ECG data from thousands of children, including those with common and rare heart defects, to train a deep-lear…

This trial tests whether magnetic resonance elastography (MRE), a special MRI that measures tissue stiffness, can distinguish normal pressure hydrocephalus from brain atrophy. Current scans often cannot tell the two apart, leading to missed or delayed diagnosis. Researchers will …

This study tests whether a radioactive tracer called 124I-evuzamitide can detect amyloid deposits in the heart and elsewhere in people aged 60 and older who have lumbar spinal stenosis or carpal tunnel syndrome. Participants will receive a single dose of the tracer and undergo a …

This trial tests whether a 7-day course of the acid-blocking drug vonoprazan can guide treatment for adults with frequent heartburn or regurgitation as effectively as standard tests like endoscopy and 24-hour pH monitoring. Participants are randomly assigned to either the drug-te…

This trial asks whether hydroxyurea, a daily oral medication, can reduce the risk of a first stroke in children with sickle cell anemia. Researchers will use transcranial Doppler (TCD) ultrasound to measure blood flow in the brain, a key indicator of stroke risk, in children aged…

This early-stage study tests an experimental vaccine designed to prevent cancer in adults with BRCA1 or BRCA2 gene mutations. The vaccine is given alone or with another drug, followed by a mild electric pulse to help it enter cells. Researchers want to see if it is safe and wheth…

This study will test a telehealth group prenatal care program called Centering HER in 72 low-income, predominantly Black pregnant women in Kansas who are at moderate or high risk for preeclampsia. Participants will receive routine care plus seven doula-led virtual group sessions,…

This trial tests whether providing free corrective glasses to young motorcycle drivers with myopia (nearsightedness) can reduce the number of crashes and near-misses. Participants are university students in Ho Chi Minh City who drive regularly and have uncorrected or under-correc…

This study looks at how to keep a youth-friendly HIV self-testing program called 4YBY running in Nigeria. It will involve over 1,200 young people aged 14-24 who are at risk for HIV. Researchers will compare standard program delivery with an enhanced version that includes extra su…

This trial is testing whether taking daily vitamin D3 and fish oil supplements can reduce the risk of lung problems like COPD flare-ups, asthma attacks, and pneumonia in older adults. It involves over 25,000 men and women aged 50 and older who do not have a history of cancer or h…

This trial tests whether an educational program on sustainable diet and breastfeeding, given before delivery, can increase the number of mothers who exclusively breastfeed for up to six months. The study includes 429 pregnant women and also measures the environmental impact of di…

This study tests whether a dedicated care coordinator can help people with psoriasis or psoriatic arthritis reduce their risk of heart disease. About 520 adults aged 40-75 who do not already take cholesterol medication will receive support to manage risk factors like high cholest…

This study tests an investigational drug called OHB-607 to see if it can prevent bronchopulmonary dysplasia (BPD), a serious chronic lung disease, in extremely premature infants. About 338 babies will be randomly assigned to receive either the drug or standard care. The goal is t…

This study tests if teaching children and parents about avoiding certain chemicals (like BPA) can actually lower the levels found in kids' urine. About 700 children aged 6 to 8 in Belgium and Spain will take part in a school program over two and a half years. The goal is to see i…

This study tests whether a 6-month program combining Bollywood-style dance classes and a fitness tracker can increase physical activity and improve metabolic health in South Asian Indian immigrant women aged 40-65 who are not very active. The goal is to see if this culturally tai…

This trial tests whether spinal cord stimulation can reduce severe, treatment-resistant pain in people with erythromelalgia, a rare condition causing red, hot, and painful extremities. Participants receive either burst stimulation or sham (inactive) stimulation to see if the ther…

This study tests whether a simple ear acupressure technique, called auricular point sticking therapy, can reduce anxiety and improve sleep in women undergoing IVF. 178 women aged 20-45 with anxiety or insomnia will either receive standard IVF care or standard care plus ear acupre…

This study tests a personalized workplace intervention for 124 Dutch workers with Parkinson's disease, cerebellar ataxia, hereditary spastic paraparesis, or slowly progressive neuromuscular/mitochondrial disorders. A trained facilitator helps employees and their managers identify…

This trial tests whether spending time in nature can reduce anxiety in children aged 9-12 who have experienced adverse childhood events. Participants are assigned to either group nature outings, family outings, or a waitlist. The goal is to see if these nature-based activities im…

This study tests whether using a hand extension rubber during IV insertion can reduce pain, anxiety, and improve satisfaction. Researchers will enroll 114 adults who need an IV and randomly assign them to use the rubber or not. The goal is to see if this simple, low-cost distract…

This trial tests a counseling program called Mpata Yathu ('Our Space') for adolescent girls and young women in Zambia who have experienced gender-based violence and may be living with or at risk for HIV. The program uses problem-solving therapy delivered by trained lay counselors…

This pilot study will test whether virtual Reiki therapy can reduce pain and improve well-being in people with lupus. Twenty adults with lupus will receive 20-minute Reiki sessions once a week for 8 weeks, delivered remotely by a certified practitioner. Researchers will measure c…

This study tests whether atorvastatin, a drug typically used for cholesterol, can reduce bile acid production and improve symptoms in people with bile acid diarrhoea (BAD). Twenty adults with moderate-to-severe BAD will receive atorvastatin and a placebo in random order, each for…

This study asks whether a procedure called percutaneous ablation can reduce symptoms and improve daily life for adults with desmoid tumors. Participants will complete surveys before and after the procedure to track symptom severity, pain, recovery, and how the tumor responds on i…

This trial explores whether an affordable robot can help people with HIV who have had a stroke improve their arm and leg function. Participants will use the robot for rehabilitation exercises, and researchers will measure changes in movement, balance, and quality of life. The goa…

This study tested a school-based program called Proud & Empowered, designed to help LGBTQ students cope with minority stress and improve mental health. About 228 high school students took part. The program was led by school counselors and social workers. The study was terminated …

This trial tests whether listening to music for 30 minutes before and after surgery can reduce pain, anxiety, and the need for painkillers in women undergoing gynecologic surgery. Participants will choose from several music genres and report their pain, anxiety, and satisfaction …

This study tests whether a specific ultrasound-guided nerve block can reduce pain after elective lower back surgery better than the usual method of injecting pain medicine into the wound. Adults aged 18-65 undergoing surgery for conditions like a herniated disc or spinal stenosis…

This trial tests whether nurse-guided breathing exercises can reduce shortness of breath and anxiety in people with advanced heart failure who come to the emergency department. Participants are randomly assigned to receive either standard care plus guided diaphragmatic and pursed…

This trial tests whether using virtual reality (VR) relaxation before and after gynecologic surgery can reduce anxiety and pain. About 220 women undergoing day or inpatient gynecologic procedures will either use a VR headset with calming scenes or receive standard care. Researche…

This study tests whether mild electrical stimulation of the ear, combined with physical and speech therapy, can improve symptoms like voice problems, swallowing, and balance in people with Parkinson's disease. 46 participants will receive either active or sham stimulation during …

This study tests new versions of Clareon Vivity Pro intraocular lenses (IOLs) to see if they improve vision better than current lenses. About 231 adults with cataracts in both eyes will receive one new lens and one current lens to compare results. The main goal is to measure how …

This study tests whether two simple, drug-free techniques can ease the pain, fear, and crying that 9-month-old babies often experience during routine vaccinations. One method uses a small bee-shaped device called Buzzy, which vibrates and cools the skin near the injection site. T…

This study explores whether art therapy can help people with progressive supranuclear palsy (PSP) feel better and improve their daily life. Ten participants will take part in art sessions, and researchers will measure changes in quality of life, mood, and caregiver stress. The go…

This study is testing whether a daily practice of Tao Calligraphy meditation and drinking so-called 'energized water' can improve symptoms of unipolar depression in adults. Fifty participants will be split into two groups to compare the effects over 12 weeks. The goal is to see i…

This study tests whether a special hat with built-in goggles can make phototherapy more comfortable for full-term newborns with jaundice, compared to standard eye patches. Researchers will measure the babies' comfort levels, bilirubin changes, treatment duration, and parental sat…

This study tests whether a non-invasive magnetic brain stimulation technique, called rTMS, can help ease depression in people with Parkinson's disease. Fifty adults with mild to moderate depression will be randomly assigned to receive either real rTMS or a sham (fake) treatment f…

This pilot trial tests whether a home-based exercise program, paired with a self-guided education booklet, can help adults aged 60 and older who have benign paroxysmal positional vertigo (BPPV) with or without vestibular hypofunction. Participants are randomly assigned to either …

This study tests whether deutetrabenazine can improve speech clarity and walking ability in people with Huntington disease. About 30 adults with early to moderate symptoms will take the drug and be evaluated using speech tests and motion capture technology. The goal is to see if …

This study tests whether a smartphone app called AIM+ can help juvenile justice officers and other professionals who work with youth in the legal system manage chronic stress, anxiety, and burnout. Over 30 days, participants use the app for short guided meditations and videos. Th…

This study looks at whether using the Calm meditation app for 10 minutes a day can help blood cancer patients who have trouble sleeping. Researchers will track sleep, mood, and inflammation in 276 participants over 20 weeks. The goal is to find a simple, drug-free way to improve …

This study tests whether a TENS (transcutaneous electrical nerve stimulation) device can reduce the pain of biologic injections in people with psoriasis. About 20 adults will receive their injection in one thigh without TENS and in the other thigh with TENS pads placed nearby. Th…

This study tests whether a device called CES (cranial electrotherapy stimulation) can reduce anxiety symptoms in 200 active-duty military members and their families. Participants receive either real CES or a sham (placebo) device to see if the gentle electrical pulses truly help.…

This trial tests whether a small device that stimulates the vagus nerve through the ear can improve quality of life for people with long COVID who also meet the criteria for ME/CFS (chronic fatigue syndrome). Participants will use the device daily for six weeks, and researchers w…

This trial is testing whether watching educational videos before minimally invasive gynecologic surgery can lower patient anxiety. Participants are randomly assigned to either watch the videos or receive standard counseling alone. The study measures anxiety and preparedness throu…

This trial compares two forms of cognitive-behavioral therapy (CBT) for children and teens with anxiety or obsessive-compulsive disorder (OCD). One approach uses internet-delivered modules with therapist guidance; the other trains parents to lead exposure exercises with their chi…

This study tests a coach-guided digital therapeutic called Toivoa-004, designed to help adults with Long COVID manage symptoms of anxiety and depression. The program delivers cognitive behavioral therapy through a smartphone app and is built to be accessible for people with disab…

This trial tests whether Relex-Smile laser surgery can safely and effectively correct residual nearsightedness in people who still have blurry distance vision six months after cataract surgery. Participants will undergo the laser procedure and have their vision and refraction mea…

This study tests whether a robotic exoskeleton called Ekso can help people with Parkinson's disease improve their walking, balance, and quality of life. Over eight weeks, participants will use the device in two weekly sessions, with the robot gradually reducing assistance as they…

This trial tests whether using a silicone aligner seater (a chewy) more often can improve how well clear aligners move teeth as planned. Adults aged 18 to 45 with mild to moderate crowding or spacing will be randomly assigned to use the chewy only when first inserting each aligne…

This pilot study explores whether robot-assisted walking devices can be used in schools to help children who cannot walk independently. The devices attach to a walker and power leg movements, allowing children to stand and walk upright. The study aims to identify the best ways to…

This trial tests whether combining resonant breathing (slow, paced breathing) with Qigong exercises can improve lung function, reduce shortness of breath, and boost quality of life in adults aged 40–65 with bronchiectasis, a chronic lung condition. Participants are randomly assig…

This trial tests whether a non-invasive brain stimulation technique called transcranial magnetic stimulation (TMS) can reduce anxiety in people with Parkinson's disease. Anxiety affects up to 40% of people with Parkinson's, and standard medications often fall short or cause side …

This early-stage trial is testing whether a non-invasive procedure called focused ultrasound can safely reduce both seizures and anxiety in people with treatment-resistant epilepsy. The ultrasound targets a small region in the brain thought to be involved in both conditions. Rese…

This study investigates whether a 16-week individually tailored exercise program, guided by a physiotherapist, can help adults with post-COVID POTS (postural orthostatic tachycardia syndrome) spend more time upright and feel better. Participants are randomly assigned to either th…

This study tests whether virtual reality-assisted vestibular rehabilitation can reduce dizziness and improve balance and walking more than conventional vestibular rehabilitation in people with benign paroxysmal positional vertigo (BPPV). Participants, aged 18 to 40 with residual …

This study explores whether a type of electrical stimulation, called afferent-specific electrical stimulation (asES), can improve how the brain senses and controls hand movements. Researchers will test it in healthy adults and people with essential tremor, a condition that causes…

This study looks back at the medical records of 500 children and adults with cancer who received immunotherapy at the National Cancer Institute. The goal is to understand the side effects, like inflammation and immune reactions, that can happen with these treatments. By learning …

This study looks at whether taking extra random biopsies during a colonoscopy helps find precancerous growths in people with inflammatory bowel disease (IBD), including Crohn's disease and ulcerative colitis. About 1,600 participants will be randomly assigned to one of two biopsy…

This study collects and analyzes blood, DNA, and tissue samples from 315 adults with pulmonary fibrosis, their relatives, and healthy volunteers. The goal is to learn more about the disease's causes, not to test a new treatment. Researchers hope the findings will guide future the…

This study collects small skin samples from people with primary immunodeficiency or immune regulation disorders, as well as from healthy volunteers. Researchers will use these cells to create cell lines for laboratory research. The goal is to better understand immune diseases and…

This study follows 68 people aged 12 and older who have ABCA4 gene mutations, which can cause vision loss from diseases like Stargardt disease. Over 10 years, researchers will collect blood and skin samples and perform detailed eye exams to understand how the disease progresses. …

This study follows 46 people with gyrate atrophy, a rare genetic condition that causes vision loss, over 4 years. Researchers measure ornithine levels in the blood and track changes in the retina using eye scans and photos. The goal is to learn how the disease progresses under st…

This observational study aims to measure how many people in Portugal have Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS), two rare and severe forms of epilepsy. Researchers will review existing medical records from about three public hospitals to estimate the percentage of…

This study looks at people and families who have a high risk of cancer to learn what genes and environmental factors might increase that risk. Over 5,000 participants are followed over time with questionnaires, medical records, and optional genetic testing. No treatment is given,…

This study follows 300 adults with Crohn's disease who are starting standard treatments like vedolizumab or IL-23 blockers. Researchers will track how quickly patients feel improvements in pain, fatigue, and daily functioning using simple questionnaires. The goal is to better und…

This study looks at whether short educational videos can help people with inherited cancer gene mutations learn more about their condition. About 43 adults who carry mutations in genes like BRCA1 or BRCA2 will watch the videos and then take a quiz. The goal is to see if this simp…

This study investigates whether children with Wilson's disease, who appear to have normal heart function, may actually have subtle heart problems. Using advanced echocardiography, ECG, and a blood test for a heart stress marker, researchers will compare these children to healthy …

This study follows 1,500 volunteers with normal memory, mild cognitive impairment, or Alzheimer's over several years. Researchers use brain scans, memory tests, and blood samples to track how the disease progresses. The goal is to find better ways to diagnose and treat Alzheimer'…

This study follows 25 people with spinocerebellar ataxia type 7 (SCA7) for up to 5 years to learn how the disease changes vision, coordination, and thinking. Participants will have yearly eye exams, brain scans, and neurological tests. No treatment is given; the goal is to gather…

This long-term study looks at people and families with a higher chance of developing blood or lymph node cancers like leukemia or lymphoma. Researchers collect medical histories, genetic samples, and sometimes perform exams to find inherited genes or environmental triggers. The g…

This observational study will examine how the drug mitapivat changes red blood cells in 6 people with sickle cell disease. Researchers will measure oxygen binding and cell survival markers from blood samples and muscle oxygen tests. The goal is to understand the drug's effects, n…

This study is collecting blood, bone marrow, and tissue samples from 5,000 people with blood cancers like leukemia and lymphoma. Researchers will analyze the samples to find genetic abnormalities that cause cancer cells to grow. The goal is to understand why these cancers develop…

This study collects blood and other samples from people with sickle cell disease and healthy volunteers to help researchers understand how inflammation affects blood vessels. Up to 2,000 adults aged 18 and older will take part. The samples will be used in lab tests to support fut…

This study is screening 115 underserved children and adults who are at risk for type 1 diabetes or celiac disease. Researchers will use blood tests to find early signs of these conditions, aiming to improve how we detect them. The goal is to catch these diseases sooner, especiall…

This study follows adults with pathogenic variants in the anoctamin 5 gene, which causes a form of limb-girdle muscular dystrophy. Over three years, researchers will use whole-body MRI to measure changes in muscle fat content, and questionnaires to track fatigue and quality of li…

This observational study is looking at a protein called neurofilament light chain (NfL) in the blood of people who carry a gene variant for hereditary ATTR amyloidosis, both those without symptoms and those with nerve damage. Researchers want to see if NfL levels can help detect …

This study compares over 1,500 people living with HIV to a similar group from the general population to see if conditions like heart disease, COPD, and liver disease are more common or develop faster in those with HIV. Using detailed scans and tests, researchers will measure the …

This study aims to describe the pattern of muscle involvement in people with limb girdle muscular dystrophy caused by mutations in the anoctamin 5 gene. Researchers will collect and analyze MRI scans from 200 participants to see which muscles are affected and how much fat replace…

This study looks at how often CD4 cell counts are measured when adults in Zambia begin or restart antiretroviral therapy (ART) after a long break. The goal is to see how many people have advanced HIV disease and whether low testing rates prevent proper care. Researchers will asse…

This observational study will use cardiac MRI to examine how stimulant and polysubstance use affects heart scarring and inflammation in 400 people with and without HIV. Participants will have one study visit for a detailed heart scan. The goal is to better understand the combined…

This pilot study tests whether daily, at-home stimulation of a nerve in the ear can change brain activity in people with inflammatory bowel disease (IBD) who are in remission. Fifteen adults with Crohn's disease or ulcerative colitis will use a small device for 28 days and underg…

This study tests whether seeing a picture of a supportive friend or family member helps people with social anxiety unlearn fear more effectively. Researchers will compare fear responses in 100 participants (50 with social anxiety, 50 healthy) after pairing threatening cues with e…

This study tests a video-based mobile app designed to reduce mental health stigma and medical mistrust among Black adults with moderate to severe depression or anxiety. Participants watch personal stories from others with similar experiences. The goal is to see if this approach i…

This study looks at whether seeing a picture of a supportive person can help people with social anxiety feel less afraid. Researchers will compare 50 healthy adults and 50 adults with social anxiety disorder. Participants will learn to associate a neutral image with a mild electr…

This study tests whether a standard nutrition screening tool, called PYMS, can accurately identify children with Crohn's disease or ulcerative colitis who are at risk of malnutrition. Researchers will use the tool on 2,000 children aged 4–18 with IBD in outpatient and inpatient c…

This study investigates whether people with neurodegenerative diseases—like Alzheimer's, Parkinson's, or ALS—show changes in behavior, social circumstances, or health before and after their diagnosis. Researchers will compare the records of over 61,000 patients with those of matc…

This observational study follows 2,000 Korean COPD patients over time to learn how the disease develops, especially in its early stages. By tracking symptoms, lung function, and flare-ups, researchers hope to identify patterns that could lead to earlier detection and better preve…

This observational study explores whether levels of a protein called soluble CD163 in blood or urine can reflect how active IgA nephropathy is and how it might progress. Researchers will compare these levels among people with IgA nephropathy, those with a similar kidney condition…

This study observes 44 people with type 3 von Willebrand disease, a rare bleeding disorder, who are already on standard preventive treatment. Researchers will track how often bleeding episodes occur and how the condition affects quality of life over at least 24 weeks. No new drug…

This study follows 1,000 patients in China who are already receiving faricimab for diabetic macular edema, retinal vein occlusion, or wet age-related macular degeneration. Researchers will collect data from routine clinic visits over one year to see how well the drug works and ho…

This study looks at balance and movement problems in people with stroke, Parkinson's disease, or multiple sclerosis. Researchers will use special devices to measure how well participants can keep their balance and walk. The goal is to learn more about these issues so that rehabil…

This study aims to better understand fatigue by comparing people who have felt tired for more than a month with those who are not fatigued. Researchers will use questionnaires, physical tests, and optional brain scans to identify different types of fatigue. The goal is to gather …

This study follows 11 males with Hemophilia B who already receive regular factor IX therapy to prevent bleeding. Researchers will collect information on bleeding events, treatment use, and quality of life over at least 6 months. No experimental treatment is given; the goal is to …

This study looks at whether the bite correction from a Twin Block appliance—a removable brace used to fix overbites—stays put over time. Researchers will compare dental records from before, right after, and years after treatment in 72 people who previously wore the appliance. The…

This 5-year study follows 250 people with von Hippel-Lindau disease who have brain or spinal cord tumors. Researchers use regular MRI scans and blood tests to see how fast tumors grow and what factors like hormones might affect growth. The goal is to better understand which tumor…

This observational study follows about 198 patients in Spain who are already taking inclisiran (Leqvio) for high cholesterol linked to heart disease or a genetic condition. Researchers will check if patients reach their LDL cholesterol goals after 6 and 12 months. The goal is to …

This study collects blood, bone marrow, urine, and tissue samples from up to 6,000 people with solid tumors, blood cancers, or non-cancerous blood disorders, as well as from their healthy family members who are stem cell donors. The samples are stored and used in research to bett…

This study looked at how sounds played during sleep affect brain waves and memory in children with Rolandic epilepsy. Researchers used brain scans (MRI, MEG/EEG) to understand which brain circuits are involved. The goal was to find new treatment ideas for this type of childhood e…

This study evaluates a sensor-based version of the Timed Up and Go test (QTUG) for measuring balance and predicting fall risk in older adults with dizziness (vestibulopathy). Researchers will compare QTUG results with other balance tests, track whether a six-month vestibular reha…

This study aims to find the genes that cause inherited hearing loss by analyzing DNA from 24,000 people in large Pakistani families. Researchers will compare affected and unaffected family members to identify new deafness genes. The goal is to improve genetic testing and counseli…

This study tracks 585 patients with familial or regular high cholesterol who are already prescribed Leqvio. Researchers will monitor side effects and cholesterol levels over time to see how safe and effective the drug is in everyday use. No new treatment is given—it's purely obse…

This study follows people with Phelan-McDermid syndrome, a rare genetic condition linked to autism and intellectual disability, to map how the syndrome unfolds over two years. Researchers will measure changes in thinking, behavior, language, and motor skills, and use brain imagin…

This study looks at whether specific brain wave patterns on an EEG can predict how well the drug fenfluramine works for people with Lennox-Gastaut Syndrome (LGS), a severe form of epilepsy. Researchers will follow 20 children and adults aged 2 to 35 who are already taking fenflur…

This study aims to improve how doctors communicate with patients facing serious illnesses like cancer, heart failure, COPD, or dementia. Researchers will give doctors detailed feedback on their conversations about goals of care. The goal is to help patients receive care that matc…

This study looks at the genetic makeup of rare blood diseases like Castleman disease and Langerhans cell histiocytosis. Researchers will analyze blood or tissue samples from 135 participants to find genetic changes that could be targeted by existing or experimental therapies. The…

This study follows individuals with autism who carry a PTEN gene mutation, along with comparison groups, to track changes in thinking, language, and behavior over time. Researchers will also collect blood samples and medical data to build a large database for future studies. The …

This study investigates whether advanced brain imaging and electrical recordings can predict the presence and severity of autism spectrum disorder (ASD) and intellectual disability (ID) in people with tuberous sclerosis complex (TSC). Researchers will follow participants over tim…

This study follows 419 people who do not have pancreatic cancer but are at high risk due to family history or genetic mutations. Researchers collect blood samples and health information over time to find markers that could predict or detect pancreatic cancer early. The goal is to…

This study explores whether combining three eye-imaging techniques can reveal how structural damage in the retina relates to vision loss in people with geographic atrophy, a form of age-related macular degeneration. Researchers will use OCT scans, ultra-high-resolution AOSLO imag…

This study is looking at how magnetic resonance imaging (MRI) and spectroscopy can be used to detect and track changes in the muscles of people with facioscapulohumeral muscular dystrophy (FSHD). The goal is to identify reliable biomarkers—measurable indicators of disease activit…

This study is testing whether functional MRI brain scans can predict and track how well children and teens with obsessive-compulsive disorder (OCD) respond to a type of therapy called Exposure and Response Prevention (ERP). Thirty participants aged 10 to 17 will receive 14 weekly…

This study aims to build a research group of 100 people with neurological diseases like stroke, Parkinson's, epilepsy, or dementia. Researchers will use overnight sleep tests and questionnaires to measure sleep patterns, breathing, and heart rate during sleep. The goal is to bett…

This study looks at how cannabis ads that claim to help with anxiety or depression affect what people buy. About 2,000 light-to-moderate cannabis users with mild to severe anxiety or depression will shop in a realistic online store. Some see ads with mental health claims, others …

This study is a nationwide registry in China that will follow adults with a genetic form of spinal muscular atrophy (SMA). The goal is to describe how the disease naturally progresses over time and how disease-modifying treatments are used in real-world settings. By collecting da…

This study uses health records from millions of people to see if GLP-1 receptor agonists (drugs for type 2 diabetes and weight loss) are linked to a higher risk of medullary thyroid carcinoma, a rare cancer. Researchers will compare how often this cancer occurs in people taking G…

This study investigates how the brain makes decisions when visual information is uncertain, focusing on the roles of the basal ganglia and cerebellum. Researchers will compare healthy adults with patients who have Parkinson's disease, dystonia, or cerebellar damage, and will test…

This trial is testing an investigational imaging device that uses shortwave infrared light to help surgeons see important structures like nerves, ureters, and bile ducts during surgery. The study involves adults aged 18 and older who are undergoing certain surgical procedures, su…

This study looks at children diagnosed with a ventricular septal defect (a hole in the heart) to understand if and when the hole might close without treatment. Researchers will follow children from birth to age 14 and use echocardiography to track the defect. The goal is to build…

This study is testing whether a blood test (liquid biopsy) can detect a rare but serious nerve tumor called MPNST earlier than current methods in adults with Neurofibromatosis Type 1 (NF1). Researchers will compare the timing of tumor detection by the blood test versus standard i…

This study follows people who previously received NTLA-2001, an experimental gene-editing therapy, for a type of amyloidosis that can affect nerves or the heart. The goal is to monitor long-term safety and see if the therapy's effects on disease markers persist over time. Partici…

This study will test how changes in oxygen levels affect the heart's electrical signals in 70 people, some with oxygen dependence and some healthy. Participants will receive increasing amounts of oxygen at night while their heart activity is monitored. The goal is to better under…

This study looks at a rare liver disease called PFIC, which causes severe itching and liver damage. Researchers compare patients who took the drug odevixibat to those who did not, using data from past studies and a patient registry. The goal is to see if odevixibat helps avoid li…

This study is investigating whether testing for a panel of 24 genes, in addition to the well-known BRCA1 and BRCA2 genes, can better estimate the risk of breast and ovarian cancer. Researchers will analyze genetic samples from people with a family history of these cancers, as wel…

This study tests whether everyday rehabilitation therapy sessions can be recorded and measured more objectively using wrist-worn sensors, a video camera, and an audio recorder. Adults with neurological conditions like stroke, Parkinson's disease, or spinal cord injury will wear t…

This study is a patient registry that will follow people of any age with bile acid synthesis disorders who are treated with Cholbam (cholic acid). The goal is to collect information over 10 years on how the drug performs in routine clinical care, including its safety, effectivene…

This study aims to create a large, long-term research cohort of women with polycystic ovary syndrome (PCOS). By systematically collecting follow-up data, researchers hope to understand how the condition evolves across different life stages and identify key factors that influence …

This study looks at brain activity in people already receiving deep brain stimulation (DBS) for severe obsessive-compulsive disorder (OCD). Researchers will collect recordings from the implanted device to see how brain signals relate to OCD symptoms. The goal is to better underst…

This registry follows patients who receive donor cartilage or meniscus transplants for joint defects in the knee, hip, ankle, or shoulder. Researchers are collecting long-term data on pain, function, and whether the grafts need revision or fail over time. The goal is to understan…

This study examines the medical records of 270 adults with bronchiectasis (a lung condition causing persistent cough and breathlessness) who were treated with respiratory physiotherapy. Researchers aim to identify common patterns in lung function, symptoms, and overall health to …

This study follows people newly diagnosed with Crohn's disease for up to five years to see how the condition damages the bowel over time. Researchers will use imaging scans and other tests to track disease progression and understand how different treatments affect long-term outco…

This study investigates why some people with plaque psoriasis experience a relapse after treatment with biologic drugs, while others do not. Researchers will compare patients who relapsed with those who did not, looking at factors like age, lifestyle, lab results, and treatment h…

This study follows people treated for anal cancer with radiotherapy to see how their sexual quality of life changes over time. Participants complete questionnaires during treatment, three months later, and two years after diagnosis. The goal is to understand the long-term impact …

This study investigates whether a combination of brain MRI scans, sleep recordings, and cognitive tests can track early brain changes in people with idiopathic REM sleep behavior disorder (iRBD), a condition that often precedes Parkinson's disease. Researchers will follow 50 peop…

This study aims to create a disease-specific quality-of-life questionnaire for children with bronchiectasis, a chronic lung condition. Researchers will interview children, teenagers, and parents to understand the key impacts of the disease. The final questionnaire will be tailore…

This study looks at whether diabetic kidney disease and blood-sugar control before surgery can help predict a common complication called pseudophakic macular edema (PME) — swelling of the central retina — after cataract surgery in people with type 2 diabetes. Researchers will rev…

This study is testing whether a machine learning model can predict the extent of ulcerative colitis (UC) in the colon using only symptoms, physical signs, and lab results — before a colonoscopy is done. Researchers will analyze data from 1,500 people with UC to build and validate…

This study is testing whether a simple stool test can predict if the gut lining is healing in people with moderate-to-severe ulcerative colitis who are starting biologic therapy for the first time. Researchers will measure levels of a protein called calprotectin in stool samples …

This study follows children aged 6 to 21 who have a CNKSR2 gene mutation, which can cause epilepsy, language difficulties, and intellectual disability. Researchers will use brain scans, EEGs, and developmental tests to track how the condition changes over time. The goal is to cre…

This study explores whether whole-genome sequencing can uncover genetic causes in adults with unexplained medical conditions. One thousand participants will provide a blood or cheek-swab sample and allow researchers to review their medical records. The goal is to see how often se…

This study aims to find genetic causes of conditions that affect the development of the gonads and genitals, such as hypospadias and ambiguous genitalia. Researchers will use whole exome sequencing to analyze DNA from 300 children and their families. The study also measures how r…

This study explores whether the immune system plays a role in starting and driving Parkinson's disease. Researchers will collect blood, spinal fluid, stool, and saliva from people with REM sleep behavior disorder (a strong early sign of Parkinson's), people already diagnosed with…

This study looks at how Friedreich's ataxia, a genetic nerve disease, affects thinking skills like memory and problem-solving. Researchers will compare cognitive test results between people with the condition and healthy volunteers, and see if these results relate to the number o…

This study looks at whether taking muco-active agents (mucus-thinning drugs like N-acetylcysteine and ambroxol) after a lung cancer diagnosis is linked to better outcomes in people who also have COPD. Researchers will analyze data from a large Korean database, comparing those who…

This study tests whether a 12-lead wearable Holter monitor can be worn during MRI, CT, X-ray, and echocardiography without affecting image quality or the device's ability to record heart signals. Researchers will enroll 500 adults who need these imaging exams and will check for e…

This observational study follows 200 adults with gastroesophageal reflux disease (GERD) who are scheduled for antireflux surgery in Poland. Researchers collect data on surgical techniques, symptom relief, quality of life, and complications over 12 months. The goal is to identify …

This pilot study looks at whether spinal cord stimulation can improve nerve health in people with small fiber neuropathy, a condition that causes chronic pain. Researchers will measure changes in nerve fiber density from skin biopsies taken before and after 12 months of routine c…

This study investigates how peripheral electrical stimulation (PES) to the forearm muscles can reduce tremors in people with Parkinson's disease or essential tremor, as well as in healthy volunteers. Researchers use brain and muscle recordings to understand the underlying nerve s…

This observational study looks at how common overweight and obesity are in adults with phenylketonuria (PKU) and what factors might contribute. Researchers will collect clinical data, lab results, and questionnaire responses on diet, exercise, and mental health from 100 adults at…

This study looks at whether a rare tooth condition called taurodontism, where teeth have enlarged pulp chambers, is linked to gum disease. Researchers will review dental records and X-rays from healthy adults to compare gum health between those with and without taurodontism. The …