Disease of genetic or genomic mechanism

This study tests a new gene therapy called DB-OTO for children with hearing loss caused by changes in the otoferlin gene. The treatment is injected into the inner ear to help restore hearing. Up to 30 children and infants will take part to check safety and how well it works.

This early-phase trial tests a personalized treatment for people with blood cancers like leukemia. Doctors take a patient's own T cells, modify them in the lab to recognize unique cancer mutations, and infuse them back after a short course of chemotherapy. The goal is to see if t…

This Phase 3 trial tests elafibranor, a daily pill, against a placebo in 276 adults with primary biliary cholangitis (PBC) and cirrhosis. The goal is to see if elafibranor can prevent disease worsening, such as liver failure or death, over up to 3.5 years. Researchers will also m…

This phase 3 trial is testing whether adding a targeted therapy called Sacituzumab tirumotecan (Sac-TMT) to standard chemotherapy and immunotherapy (pembrolizumab) can better eliminate cancer before surgery in people with high-risk, early-stage triple-negative or hormone-receptor…

This early study tests baricitinib, a drug already approved for other immune conditions, in 20 people with Job syndrome who also have lupus-like disease or eczema. Participants take the pill daily for 6 months. The main goal is to see if it is safe and tolerable, while also check…

This study tests brain surgery as a treatment for people whose epilepsy doesn't improve with medication. About 300 children and adults aged 8 and older will have surgery to remove or treat the part of the brain causing seizures. The goal is to stop or greatly reduce seizures, tho…

This study watches how well IL-23 inhibitors work for adults with active Crohn's disease in everyday clinics. About 665 people will get treatment chosen by their doctor and be checked at 12 and 52 weeks. The main goal is to see how many reach remission (symptom-free) by week 12, …

This study tests a stem cell transplant to control peripheral T-cell lymphoma in people whose cancer has not responded to standard treatments. Participants receive a donor's stem cells after chemotherapy or antibody therapy to prepare their body. The goal is to see if this approa…

This Phase 3 trial tests an oral drug called gildeuretinol acetate (ALK 001) in 230 people aged 8 to 45 with Stargardt disease, a genetic condition that causes progressive vision loss. Participants take the drug or a placebo daily for 24 months. The goal is to see if the drug can…

This early-phase study tests whether Mozobil, a drug already used to boost stem cells for transplants, can safely raise white blood cell counts in people with WHIMS. WHIMS is a rare genetic condition that traps infection-fighting cells in the bone marrow, leading to frequent infe…

This phase 2 trial tests whether the drug nivolumab can slow or stop the growth of Epstein-Barr virus (EBV)-positive lymphoproliferative disorders and non-Hodgkin lymphomas. About 40 people aged 12 and older whose cancers have not responded to standard therapy will receive nivolu…

This phase 3 study tests a drug called afimkibart in 600 people with moderate-to-severe Crohn's disease. Participants receive either the drug or a placebo to see if it can reduce symptoms and gut inflammation. The trial is double-blind, meaning neither patients nor doctors know w…

This study tests an oral medication called LUM-201 for children with growth hormone deficiency who have never been treated before. The goal is to see if taking a pill every day for 12 months can improve growth rate compared to a placebo. The study involves 150 prepubertal childre…

This study tests a new drug called LMP744 in people whose glioblastoma has come back after standard treatment. The drug is designed to kill cancer cells by blocking a key growth signal and interfering with DNA repair. Participants will receive the drug through a vein for 5 days e…

This phase 3 trial is testing whether adding immunotherapy drugs (brentuximab vedotin and nivolumab) to standard chemotherapy (with or without radiation) improves survival for people aged 5 to 60 with newly diagnosed stage I or II classical Hodgkin lymphoma. About 1,875 participa…

This study tests whether clemizole HCl (EPX-100) can safely reduce seizures when added to current treatments for Lennox-Gastaut syndrome, a severe form of epilepsy. About 260 children and adults aged 2 to 55 will receive either the drug or a placebo. The main goal is to measure c…

This study tests a drug called enpatoran for people with lupus who have active skin rashes or lesions. About 202 participants will take either enpatoran or a placebo twice daily for 24 weeks, alongside their usual treatments. The goal is to see if the drug can significantly impro…

This Phase 1 trial tests surovatamig, a bispecific T-cell engager, in 48 adults with rheumatoid arthritis or systemic lupus erythematosus. The study gives the drug as injections in increasing doses to check safety, side effects, and how the body processes it. It is too early to k…

This study tests whether programming a cochlear implant based on each person's unique inner ear structure improves hearing compared to the standard one-size-fits-all approach. Adults with severe to profound hearing loss who already have a MED-EL cochlear implant will receive both…

This clinical trial tests a partial stem cell transplant for people with severe sickle cell disease or beta-thalassemia who need regular blood transfusions. The transplant uses a lower-intensity conditioning regimen with alemtuzumab, low-dose radiation, and abatacept to help dono…

This early-stage study tests a new drug called SR604 in healthy people and those with hemophilia A, B, or factor VII deficiency. The main goal is to check safety and how the drug moves through the body. About 31 participants will receive single or multiple doses to find the right…

This study follows about 300 people with Duchenne muscular dystrophy who are taking the drug givinostat as part of their normal care. Researchers will track safety issues like low platelet counts and bleeding, and see how patients' muscle function changes over up to 5 years. The …

This early-stage trial is testing a new gene therapy called nula-cel for people with severe sickle cell disease. The therapy modifies a patient's own blood stem cells to produce normal hemoglobin instead of sickle hemoglobin. About 15 participants aged 12 to 40 will receive the t…

This study tests whether adding a personalized mRNA vaccine (V940) to standard immunotherapy (pembrolizumab) can stop advanced melanoma from growing or spreading. About 160 people with stage III or IV melanoma that cannot be removed by surgery will receive either the vaccine plus…

This early-stage trial is testing whether a skin gel made from curcumin (a compound in turmeric) can be safely absorbed by adults with stable sickle cell disease. Researchers will apply the gel to the forearms twice a week for six weeks and monitor for side effects. The goal is t…

This study tests a new medicine called ANG003 for people with cystic fibrosis who have trouble digesting food (exocrine pancreatic insufficiency). The medicine is a type of enzyme replacement therapy, similar to the current standard treatment Creon. About 113 participants will re…

This phase 2 trial is testing a combination of blinatumomab (an immune-boosting drug) with dasatinib or imatinib (targeted pills) and standard chemotherapy for people with a specific type of leukemia called Philadelphia chromosome-positive or Philadelphia chromosome-like B-ALL. T…

This phase 3 trial tests whether adding the experimental drug sacituzumab tirumotecan (sac-TMT) to standard immunotherapy (pembrolizumab) and a targeted therapy (bevacizumab) helps people with metastatic cervical cancer live longer. About 1,000 participants whose cancer has sprea…

This early-stage trial tests a single intravenous dose of AFTX-201, a gene therapy designed to deliver a working copy of the BAG3 gene to heart cells. It enrolls 22 adults aged 18–55 with dilated cardiomyopathy and a BAG3 mutation. The study primarily checks safety and tolerabili…

This study looks at whether placing a small drain near the surgical site after one- or two-level lumbar spine surgery reduces complications like infection or the need for another operation. About 975 adults having open back surgery for spinal stenosis or spondylolisthesis are ran…

This study tests a drug called glofitamab, which helps the immune system find and kill cancer cells, in children and young adults (ages 6 months to under 30) whose B-cell lymphoma has come back or not responded to treatment. Some participants receive glofitamab alone, while other…

This early-stage study tests a new drug called casdatifan (AB521) alone or combined with other cancer drugs in people with advanced clear cell kidney cancer or other solid tumors. The main goals are to check safety and find the right dose. About 362 participants will take part.

This study tests an experimental drug called AZD0292 in 435 people aged 12 and older who have bronchiectasis and a long-term lung infection with Pseudomonas aeruginosa. The goal is to see if the drug can reduce the number of moderate-to-severe lung flare-ups compared to a placebo…

This study tests an experimental pill called enpatoran for people with lupus who have active skin rashes. About 202 participants will receive either the drug or a placebo for 24 weeks, while continuing their usual treatments. The goal is to see if enpatoran can significantly redu…

This early-phase study tests a new drug called NX-5948 in adults with advanced B-cell blood cancers (like certain leukemias and lymphomas) that have not responded to other treatments. The drug works by breaking down a protein (BTK) that helps these cancer cells grow. The main goa…

This study tests alpelisib, a drug that targets a specific gene mutation (PIK3CA), in people with lymphatic malformations—abnormal growths of lymph vessels. The trial includes children and adults and compares alpelisib to a placebo to see if it can shrink the growths and ease sym…

This study tests whether MK-8690, a drug given as a shot, can help people with moderate-to-severe ulcerative colitis achieve remission. About 100 adults who haven't responded well to other treatments will receive either MK-8690 or a placebo. The main goal is to see if more people…

This study tests two experimental drugs, sapanisertib and serabelisib, combined with standard therapy for people with HR+/HER2- advanced or metastatic breast cancer. The goal is to find safe doses and see if the combination can shrink tumors or slow the disease. About 32 adults w…

This study tests the drug abemaciclib, typically used for cancer, in people with neurofibromatosis type 1 (NF1) who have atypical neurofibromas that cannot be surgically removed. The goal is to find a safe dose and see if it can shrink or stabilize these tumors. Participants take…

This early study is testing whether giving a transplant of healthy gut bacteria (called IMT) through a feeding tube is safe and doable in 40 adults who have had part of their colon removed due to diverticulitis or sigmoid colon cancer. Participants get the transplant a couple of …

This study looks at whether Emicizumab, a newer drug, prevents bleeding during sports better than standard Factor VIII in children with moderate to severe hemophilia A. About 72 kids aged 6 to 19 who already play sports and take one of these drugs will be followed. The goal is to…

This study tests an experimental drug called BAY 3401016 in 60 adults aged 18 to 45 with Alport syndrome, a rare genetic condition that damages kidneys, hearing, and eyes. The drug works by blocking a protein thought to cause kidney injury, with the goal of slowing the loss of ki…

This early-phase trial tests an experimental antibody called DIAG723 in 93 adults with hereditary hemorrhagic telangiectasia (HHT), a condition that causes abnormal blood vessels and frequent nosebleeds. The study will check if the drug is safe and how the body processes it, with…

This study tests if the drug vorinostat is safe and can help people with moderate-to-severe Crohn's disease by reducing gut inflammation. Participants who respond well may then receive a maintenance treatment with ustekinumab for up to two years. The goal is to control symptoms a…

This study follows 30 people with Alagille syndrome who are taking odevixibat (Bylvay) in their daily lives. The goal is to see if the drug helps them avoid serious surgeries like liver transplant or bile duct repair over the long term. Researchers will also track weight, height,…

This study looks at a second surgery for people who had gastric bypass but still suffer from severe acid reflux that medication can't control. The procedure wraps part of the stomach to stop reflux. Researchers will follow 17 adults for up to 2 years to see if the surgery works a…

This early-stage trial tests imatinib, a drug already used for certain cancers, in adults with a harmful RUNX1 gene mutation. The mutation causes easy bleeding and a high risk of blood cancers. The study aims to find the best dose and see if imatinib can improve platelet function…

This registry will follow 20 people in China with progressive familial intrahepatic cholestasis (PFIC) who are taking odevixibat (Bylvay) as part of their normal care. The goal is to see how safe the drug is over the long term and how well it controls symptoms like severe itching…

This study looks at whether giving the osteoporosis drug romosozumab in two separate 6-month blocks, with breaks for another drug called zoledronate, can build more bone than the standard 12-month course. It involves 270 postmenopausal women with osteoporosis and a recent fractur…

This study tests whether a combination of three drugs (cyclophosphamide, bortezomib, and maraviroc) can safely prevent graft-versus-host disease after a half-matched bone marrow transplant in people living with HIV who also have a blood cancer. The trial involves 265 participants…

This study will follow 100 people in Saudi Arabia with moderate to severe Crohn's disease or ulcerative colitis who are starting guselkumab. Researchers will measure how long patients continue the treatment in real-world clinics, along with early symptom improvements. No new drug…

This study tests a daily pill called enlicitide decanoate in 153 children and teens with heterozygous familial hypercholesterolemia (HeFH), a genetic condition causing very high cholesterol. The goal is to see if the drug safely lowers LDL cholesterol more than a placebo. Researc…

This study tests a new drug called GSK3862995B in 446 adults with bronchiectasis, a long-term lung condition. The goal is to see if the drug can reduce the number of serious lung infections (exacerbations) compared to a placebo. Participants will receive either one of two doses o…

This study tests a new drug called RO7790121 for people with moderate to severe ulcerative colitis, a condition that causes inflammation in the colon. About 350 adults who haven't responded well to other treatments will receive either the drug or a placebo. The main goal is to se…

This study tests a new automated method to fine-tune adaptive deep brain stimulation (aDBS) for people with Parkinson's disease. aDBS uses brain signals to adjust stimulation in real time, but setting it up is complex. The trial will compare automated aDBS to standard continuous …

This study tests a new device called Rapidlink, used during open surgery to repair or replace blood vessels in the chest. It involves 150 adults already scheduled for aortic surgery. The goal is to see if the device reduces major complications like stroke or death. Participants w…

This study tests an experimental pill called silmitasertib combined with standard chemotherapy in children and young adults (under 30) whose solid tumors (like neuroblastoma, Ewing sarcoma, or osteosarcoma) have come back or not responded to treatment. The goal is to find a safe …

This study tests a combination of chemotherapy drugs (DA-EPOCH) plus an enzyme called asparaginase (Rylaze) in adults newly diagnosed with a fast-growing blood cancer (Philadelphia chromosome-negative ALL). The goal is to see if this mix can kill more cancer cells and achieve dee…

This study follows about 424 people with primary biliary cholangitis (PBC) who are taking elafibranor as part of their normal care. Researchers will track how well the drug controls liver damage and symptoms like itching over 5 years. The goal is to see if elafibranor works safel…

This phase III trial is testing whether adding inotuzumab ozogamicin—a drug that targets cancer cells—to standard chemotherapy can improve outcomes for young adults (ages 18-39) newly diagnosed with B-cell acute lymphoblastic leukemia. About 310 participants will be enrolled. The…

This registry will follow 10 people with progressive familial intrahepatic cholestasis (PFIC) in South Korea who are taking odevixibat (Bylvay). The goal is to see how safe and effective the drug is over the long term in real-world use. Researchers will track side effects and how…

This phase 2 trial tests whether the experimental drug E6742 can reduce lupus disease activity better than a placebo. About 256 adults with lupus will take either E6742 or a placebo pill for 24 weeks. The main goal is to see if more people on E6742 achieve a meaningful reduction …

This early-stage trial is testing whether a pill made from freeze-dried healthy donor stool (MTP-101P) is safe and can restore good bacteria in people having part of their colon removed. Forty adults aged 18-75 with colon polyps, early-stage cancer, or diverticulitis will take th…

This phase 3 trial tests whether adding the experimental drug MK-2870 to the standard immunotherapy pembrolizumab helps people with advanced non-small cell lung cancer live longer compared to pembrolizumab alone. The study enrolls about 614 adults whose tumors have high PD-L1 lev…

This study investigates whether a homemade bottle-based breathing device can strengthen the muscles used for exhaling and improve balance in people with Parkinson's disease. Parkinson's often weakens respiratory muscles, leading to breathing and coughing problems. The trial will …

This trial tests whether using ultrasound during surgery to choose where to cut the bowel leads to better outcomes for people with Crohn's disease. About 44 adults with Crohn's disease affecting the last part of the small intestine will be randomly assigned to have their surgery …

This study is checking the long-term safety of an implant called OTX-TKI (axitinib) for people with wet age-related macular degeneration (AMD). The implant is placed in the eye and slowly releases medicine to control the disease. About 850 people who already completed two years o…

This phase 3 trial tests duvakitug, an injected drug, in about 980 adults with moderate to severe Crohn's disease who haven't improved with other treatments. The study is double-blind and placebo-controlled, meaning some participants get the drug and some get a dummy shot. The go…

This Phase 3 study tests a drug called zorevunersen in 170 children aged 2 to 17 with Dravet syndrome, a severe form of epilepsy. The drug is given as an injection into the spine and compared to a sham procedure. The goal is to see if it reduces seizures and improves daily living…

This phase 2 trial tests a powerful mix of short-course radiation, chemotherapy (mFOLFOX6), an immunotherapy (PD-1 antibody), and a targeted drug (cetuximab or bevacizumab, depending on genetic markers) in people with high-risk rectal cancer that is mismatch repair proficient (pM…

This study tests a new type of CAR T-cell therapy that targets two proteins, CD19 and CD22, on cancer cells. It is for children and young adults aged 3 to 39 with B-cell cancers like leukemia or lymphoma that have not been cured by standard treatments. Participants receive their …

This study tests whether a two-month hormone pretreatment (GnRH agonist plus letrozole) before standard frozen embryo transfer can improve live birth rates in women with adenomyosis. About 222 participants will be randomly assigned to either the new regimen or standard care. The …

This study follows about 665 people with myelodysplastic syndromes, beta-thalassemia, or myelofibrosis who have already taken luspatercept in earlier trials. Researchers want to see how safe the drug is over many years by tracking side effects, disease progression, and survival. …

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 study tests whether a supplement called nicotinamide riboside (NR) can reduce inflammation and improve immune cell function in women with mild-to-moderate lupus. About 78 participants will receive either NR or a placebo for 12 weeks. Researchers will measure changes in immun…

This clinical trial tests an oral drug called SKY-0515 in 400 adults with Huntington's disease. The goal is to see if it can lower harmful proteins linked to the disease and improve symptoms like movement and thinking. Participants take the drug or a placebo daily for 18 months.

This study is testing the long-term safety and effectiveness of a drug called ORKA-001 for people with moderate-to-severe plaque psoriasis. It is an open-label extension, meaning everyone gets the drug, and it includes 240 adults who already completed a previous trial with ORKA-0…

This study tests an experimental pill called ISM5411 (Garutadustat) in 80 adults with active ulcerative colitis. Half receive the drug, half a placebo, and neither patients nor doctors know who gets what. The goal is to check if the drug is safe and helps control the disease.

This early-stage study tests a single dose of ABBV-519 in 30 adults with moderate lupus or rheumatoid arthritis. Researchers will check how the drug moves through the body, how it affects immune cells, and whether it is safe. Participants will be followed for about a year to moni…

This study checks the long-term safety and lasting effects of a gene therapy called IPS101A in 6 people with severe Parkinson's disease who already received it in an earlier trial. Researchers will monitor side effects and measure changes in movement symptoms over time. The goal …

This study looks at the long-term safety and effectiveness of a medicine called marstacimab for people with severe hemophilia A or B. Participants receive regular injections to help prevent bleeding episodes. The goal is to see if this treatment is safe and reduces bleeds over ti…

This study tests two oral drugs, eflornithine (DFMO) and AMXT 1501, in children and young adults up to age 26 with certain cancers that have come back or not responded to treatment. The cancers include neuroblastoma, brain tumors, and sarcomas. The goal is to find a safe dose and…

This early-stage trial tests whether implanting specially grown dopamine-producing cells into the brain is safe and can help people with Parkinson's disease. Six participants receive their own lab-grown cells placed into a brain region that controls movement. The study measures c…

This Phase 3 trial tests whether adding immunotherapy and a targeted drug to short-course radiotherapy and chemotherapy improves outcomes for people with high-risk rectal cancer. About 204 participants will receive either the standard treatment or the intensified combo. The study…

This study tests a new type of stem cell transplant for children with severe blood disorders like sickle cell disease or aplastic anemia. The transplant uses stem cells from a parent or other half-matched family donor, which are specially processed to remove certain immune cells.…

This study tests an experimental drug, fosigotifator, in adults and children with Vanishing White Matter disease, a rare genetic brain disorder. The main goals are to check the drug's safety and how the body processes it. About 50 participants will take the drug for up to 201 wee…

This study investigates the safety and symptom-relieving effects of the drug Tegoprazan (K-CAB) in adults with gastroesophageal reflux disease (GERD). Researchers will track how well the drug reduces heartburn and acid reflux after 4 weeks of use. The study includes over 5,000 pa…

This study tests a new type of stem cell transplant for children with primary immune deficiencies or inherited metabolic disorders. The transplant uses stem cells from a half-matched family donor, which are specially processed to remove certain immune cells. The goal is to see if…

This early-stage trial tests an experimental drug called PL54 in adults aged 18 to 55 with phenylketonuria (PKU), a genetic disorder that prevents the body from breaking down the amino acid phenylalanine. PL54 is an enzyme designed to help lower high blood phenylalanine levels, w…

This study tests a drug called felzartamab in people with IgA nephropathy, a kidney disease where abnormal antibodies build up and damage the kidneys. The goal is to see if felzartamab can reduce protein in the urine, a sign of kidney damage. About 454 adults will receive either …

The DETERMINE trial is testing whether drugs already approved for some cancers can help people with rare cancers or common cancers that have rare genetic mutations. It will enroll 825 adults, teenagers, and children whose cancers have specific DNA changes. The goal is to find new…

This study tests a single injection into the eye of a new drug (SAR446597) for people with geographic atrophy, a progressive form of age-related macular degeneration that causes vision loss. About 104 participants aged 60 and older will be followed for 2 years, with an optional 3…

This study is testing an experimental drug called DNL628 in people with early-stage Alzheimer's disease. The main goal is to see if the drug is safe and how the body processes it. About 68 participants will receive either DNL628 or a placebo. This is an early-phase trial, so it f…

This Phase 2 trial tests an oral drug called NEU-411 in 150 adults aged 40-80 with early Parkinson's disease who have a specific genetic marker (LRRK2-driven). The study is double-blind and placebo-controlled, meaning neither patients nor doctors know who gets the real drug. Rese…

This program provides early access to the experimental drug venglustat for children and adults with Type 3 Gaucher disease who have brain-related symptoms. Participants must be at least 12 years old, weigh at least 15 kg, and be stable on standard enzyme replacement therapy. The …

This phase II trial is testing whether a combination of venetoclax and rituximab works better than current standard treatments (ibrutinib plus rituximab or zanubrutinib alone) for people with previously untreated Waldenstrom's macroglobulinemia, a rare type of blood cancer. The s…

This study tests a new medicine, HMB-002, in about 108 adults with von Willebrand disease (types 1 and 2). The goal is to check safety, how the drug moves in the body, and whether it helps control bleeding. It is the first time this drug is being tested in humans, starting with s…

This phase 3 trial tests whether adding the drug sapablursen to standard treatment helps people with polycythemia vera, a blood disorder causing too many red blood cells. About 250 participants who currently need regular blood removal (phlebotomy) will receive either sapablursen …

This expanded access program provides bitopertin (DISC-1459) to patients aged 12 and older with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) who have no satisfactory treatment options in the US. The goal is to offer access and gather safety information. Pa…

This Phase 3 trial tests whether DYNE-101 can improve muscle function and daily life in 150 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo by IV every few weeks for 48 weeks. The study measures how quickly people can stand from a ch…

This early-stage trial tests a new gene therapy called SKY-GJB2 for children aged 9 months to 7 years with hearing loss caused by changes in the GJB2 gene. The therapy is given as a single injection into the inner ear using a special delivery device. The study will enroll 10 chil…

This study tests whether a stem cell transplant from a healthy donor can help people with severe primary immunodeficiencies. Participants aged 4 to 75 receive chemotherapy before the transplant to prepare their body. The goal is to see if the procedure is safe and can improve imm…

This study tests a drug called tivozanib in people with bile duct cancer that cannot be removed by surgery and has already been treated with chemotherapy. The goal is to find the safest dose and see if the drug can shrink tumors or slow the cancer. Participants take tivozanib by …

This study follows 200 adults with lupus who are starting anifrolumab for the first time, to see how well it works in everyday clinics. Researchers will measure disease activity, skin symptoms, fatigue, and quality of life over time. The goal is to provide real-world evidence to …

This study tests a new drug called ITU512 in healthy people and those with sickle cell disease. The goal is to see if it is safe and can increase fetal hemoglobin, which may reduce sickle cell symptoms. The trial is in early phases (1 and 2) and involves about 161 participants. I…

This study tests a one-time gene therapy called RGX-202 in boys with Duchenne muscular dystrophy (DMD). The therapy delivers a mini version of the missing dystrophin protein to muscle cells. Researchers will check safety and whether it improves muscle function, like standing and …

This study tests a new medicine called tiespectus (EYE201) for wet age-related macular degeneration, a leading cause of vision loss. About 960 people will receive either tiespectus or the standard treatment aflibercept via eye injections. The goal is to see if tiespectus can impr…

This early-phase study tests the drug TURALIO (pexidartinib) in people aged 3 to 35 whose cancer has not responded to standard treatments. The goal is to find a safe dose and see if it can shrink tumors or control leukemia. Participants take a daily capsule for up to 2 years, wit…

This study tests Mim8, a drug that helps blood clot by mimicking a missing protein, in people with hemophilia A (with or without inhibitors). Over 450 participants will receive injections for up to 5.5 years to see if it safely prevents bleeding episodes. The goal is to provide a…

This study tests whether HBS-301 (pitolisant) can reduce excessive daytime sleepiness and cataplexy (sudden muscle weakness) in adults with narcolepsy. Participants receive either the drug or a placebo for several weeks, followed by an open-label phase where everyone gets the dru…

This phase 2 trial tests a drug called DISC-0974 in 21 adults with inflammatory bowel disease (IBD) who also have anemia caused by inflammation. The study aims to see if the drug safely raises hemoglobin levels compared to a placebo. Participants receive injections of either DISC…

This study tests whether wearing a continuous glucose monitor (CGM) for 3 months helps adults with prediabetes better control their blood sugar and stick to lifestyle changes compared to standard finger-prick testing. About 80 participants will be randomly assigned to use a CGM o…

This pilot trial tests whether feeding directly into the small intestine (transpyloric) is better than feeding into the stomach (gastric) for very preterm infants with bronchopulmonary dysplasia, a chronic lung condition. The study will measure how often babies have low oxygen le…

This study uses botulinum toxin (Botox) injections to treat movement disorders such as dystonia, tremor, and spasticity. Up to 2,000 participants aged 2 and older will receive tailored injections, with doctors trained in proper techniques. The goal is to manage symptoms and build…

This early-stage trial tests a one-time gene therapy called RP-A701 in 8 adults with dilated cardiomyopathy caused by a faulty BAG3 gene. The therapy uses a harmless virus to deliver a working copy of the gene, aiming to improve heart function. The main goal is to check safety, b…

This early-stage trial tests a single dose of TSHA-105, a gene therapy injected into the spinal fluid, in 8 people aged 2 to 20 with SLC13A5 citrate transporter disorder. The goal is to see if it is safe and whether it can improve motor and thinking skills. Because the trial is v…

This study will give 800 pregnant women biweekly food kits with fruits, vegetables, and other healthy foods to see if it improves their diet and pregnancy outcomes. Participants will be followed from early pregnancy through birth. The goal is to learn whether providing healthy fo…

This trial investigates whether adding low-intensity pulsed ultrasound to standard twin-block braces can improve jaw joint growth in teenagers with a receding lower jaw (skeletal class II malocclusion). The study involves 24 participants who will receive the combined treatment. T…

This study looks at whether taking a mix of probiotics (good bacteria) can change the gut microbiome of children and teens aged 5-18 with inflammatory bowel disease (IBD). About 40 participants will be randomly assigned to take probiotics or a placebo, then switch after a period.…

Cystinosis is a rare inherited disease that causes kidney failure and poor growth due to cystine buildup in cells. This study follows 330 patients taking cysteamine, a drug that lowers cystine levels, to track long-term effects, detect new complications, and support genetic resea…

This study tests a new drug called tiespectus (MK-8748) against the current standard treatment, aflibercept, for people with wet age-related macular degeneration (AMD). About 960 participants will receive injections into the eye and be followed for a year to see if tiespectus can…

This study tests an experimental drug called EPX-100 (clemizole) in 150 children and adults with Dravet syndrome, a severe form of epilepsy. The goal is to see if adding EPX-100 to current treatments can safely reduce the number of motor seizures. Participants will be randomly as…

This early-phase trial is testing a new drug called MTS109, made with mRNA technology, for people with moderate to severe autoimmune diseases like lupus and scleroderma. Fifteen participants will receive several injections over a month to see if it is safe and tolerable. The goal…

This study tests a special stem cell transplant for children, teens, and young adults (ages 6 months to 25 years) with solid tumors that have come back or not responded to treatment. The transplant uses cells from a donor, with certain immune cells removed to reduce side effects,…

This early-stage trial tests whether adding the drug pomalidomide to standard chemotherapy (with or without rituximab) is safe and effective for people with HIV who have aggressive B-cell non-Hodgkin lymphoma. About 25 adults with high-risk features will receive up to 6 cycles of…

This phase 3 study tests an intravenous C1 esterase inhibitor (C1-INH) in people with hereditary angioedema (HAE) to treat acute attacks and prevent attacks before medical procedures. About 124 participants, aged 2 and older, will receive either the drug or a placebo. The main go…

This study tests whether increasing the dose of guselkumab can help people with Crohn's disease who don't get better with standard doses. About 210 adults with active Crohn's will receive guselkumab, and after 12 weeks, those who haven't responded will get a higher dose more ofte…

This study is testing an oral medication called asciminib in children aged 1 to 18 with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML-CP) who have already tried other treatments. The goal is to find the right dose that works as well as it does in adults and t…

This study tests a daily oral drug called TYRA-300 in 92 children aged 3–10 with achondroplasia (a common form of dwarfism). The goal is to see if it safely increases their growth rate. Researchers will try different doses to find the best one. It's a Phase 2 trial, so it's still…

This study tests a drug called mosunetuzumab in 30 people with lupus, a disease where the immune system attacks the body. The goal is to see if the drug can put lupus into remission so that participants can stop most lupus medications for at least 6 months. However, some people m…

This study tests a new gene therapy, PS-002, for adults with IgA nephropathy, a kidney disease that can lead to kidney failure. The therapy aims to control the disease in people who are at high risk of getting worse despite current treatments. Participants receive a single dose a…

This study tests whether a special probiotic supplement can change fat-related molecules in the blood of people with ALS and frontotemporal dementia (FTD). Researchers will compare those taking the probiotic to those taking a placebo over 24 weeks. The goal is to see if the probi…

This study follows 250 people with hemophilia A who are taking efanesoctocog alfa to prevent bleeding. Researchers want to see how well it protects joints from bleeds over time in everyday life. Participants will be observed for several years to track bleeding rates and treatment…

This study follows 300 adults with Parkinson's disease, essential tremor, or dystonia who receive deep brain stimulation (DBS). DBS uses a device implanted in the chest to send electrical pulses to brain areas controlling movement. Researchers will track symptom severity, medicat…

This Phase 2 trial tests whether the oral drug RAP-103 can improve skin clearance and quality of life in people with moderate to severe psoriasis. About 90 adults aged 18 to 70 will take either a daily or twice-daily dose of RAP-103 or a placebo for 8 weeks. The study measures ho…

This early-stage trial tests a gene therapy called RC001 in children aged 2 to 18 with Dravet syndrome, a severe form of epilepsy caused by a genetic mutation. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety and how the body proc…

This phase 3 trial tests whether a new drug (MK-2870), alone or with another drug (pembrolizumab), works better than standard treatments for people with a common type of advanced breast cancer (HR+/HER2-). About 1,200 participants whose cancer has worsened after hormone therapy w…

This study tracks how well the drug Koselugo (selumetinib) works and what side effects it causes in people with Neurofibromatosis Type 1 and painful nerve tumors. About 200 patients in South Korea will be followed during their normal doctor visits. The goal is to confirm the drug…

This study tests a single dose of GB221, a gene therapy that delivers a working SMN1 gene, in infants aged 2 weeks to under 12 months with spinal muscular atrophy (SMA) Type 1. The goal is to see if it is safe and helps improve motor function. The trial includes both symptomatic …

This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease and improve muscle function. Participants must be at least 2 years …

This study is testing a device called ExAblate 4000 that uses focused ultrasound waves to treat non-cancerous brain tumors in children and young adults. The goal is to see if the treatment is safe and can shrink tumors without open surgery. About 20 participants will be enrolled,…

This Phase 2 study tests whether a single dose of AAV2-GDNF gene therapy, delivered directly into the brain, can improve motor function in adults with moderate Parkinson's disease. About 127 participants will receive either the gene therapy or a sham surgery. The main goal is to …

This study tests a medicine called anifrolumab for people with lupus who have not taken strong immune-suppressing drugs before. The goal is to see if the drug can help patients reach remission and reduce or stop their steroid use. About 245 adults will receive weekly injections f…

This study tests whether injecting a person's own bone marrow stem cells into or near the eye can help treat various retinal and optic nerve diseases, including age-related macular degeneration, retinitis pigmentosa, and glaucoma. Participants receive stem cell injections via dif…

This early study tests whether a smart brain implant can help people with spinocerebellar ataxia type 6, a condition that causes trouble with balance and coordination. Five adults will get the implant, which records brain signals and adjusts stimulation automatically. The goal is…

This study tests an experimental drug called IMC-002 (also known as IMM0306) in people with active systemic lupus erythematosus (SLE), a chronic autoimmune disease. The trial compares the drug against a placebo, both given alongside standard care, to see if it can reduce disease …

This study tests a new radioactive drug, [212Pb]VMT-alpha-NET, in people with advanced gastrointestinal neuroendocrine tumors or pheochromocytoma/paragangliomas that have already been treated with radiation therapy. The drug is designed to attach to a protein on the surface of th…

This study tests whether adding the drug lenacapavir to standard HIV treatment can reduce the hidden HIV reservoirs that remain in the body. About 50 adults with well-controlled HIV will either continue their usual therapy or receive lenacapavir injections plus pills. Researchers…

This early-stage trial tests a new medicine called EYC-0305, given as an eye injection every 24 weeks, in 30 people aged 50 and older with wet age-related macular degeneration (AMD). The main goal is to check if the treatment is safe and tolerable. Researchers will also measure h…

This study tests a single intravenous dose of a gene therapy called GNR-097 in boys aged 4 to 9 with Duchenne muscular dystrophy (DMD). The therapy uses a harmless virus to deliver a shortened version of the dystrophin gene, which is missing or faulty in DMD. The trial aims to se…

This study tests a gene therapy called ETX101 for children with Dravet syndrome, a severe form of epilepsy caused by a genetic change. The treatment uses a harmless virus to deliver a working copy of the gene to help control seizures. The trial includes infants and children up to…

This study is testing the long-term safety and effectiveness of a daily pill called atumelnant for people with classic congenital adrenal hyperplasia (CAH). The drug works by blocking a hormone receptor to help control excess androgen production. Researchers will monitor 150 part…

This study tests whether feeding preterm infants with chronic lung disease through a tube placed past the stomach (into the small intestine) helps their breathing more than feeding through a tube in the stomach. About 50 high-risk preterm babies born before 32 weeks will receive …

This early-stage study tests a new treatment called CHT101 for people with severe lupus that hasn't improved with standard treatments. CHT101 uses specially engineered immune cells (CAR-T cells) to target and destroy certain immune cells that drive lupus. The study will enroll 15…

This study tests a gene therapy for people aged 2 to 50 with X-linked severe combined immunodeficiency (SCID-X1), a condition that leaves them without a working immune system. Participants receive their own blood stem cells that are genetically corrected in a lab, then infused ba…

This phase 3 study tests whether the experimental drug WD-890 can safely improve skin clearance in adults with moderate to severe plaque psoriasis. Participants are randomly assigned to receive either WD-890 or a placebo tablet daily for up to 52 weeks. The main goals are to see …

This study tests whether a new drug, sacituzumab tirumotecan (Sac-TMT), given alone or with pembrolizumab, helps people with advanced triple-negative breast cancer live longer or keep their cancer from growing. About 1,000 adults whose cancer has spread and who have not had prior…

This early-stage trial is testing a new drug called DNL952 in 32 adults with late-onset Pompe disease. The main goal is to check if the drug is safe and how the body processes it. Participants will receive the drug through an IV infusion at different doses.

This Phase 2 trial is testing three long-acting antibodies, alone or in combination, for people with moderate-to-severe ulcerative colitis. The study will enroll 645 adults to see if these treatments can reduce inflammation and lead to remission. Participants receive the antibodi…

This study tests a gene therapy called ABBV-RGX-314 for people with wet age-related macular degeneration (AMD), a leading cause of vision loss. The goal is to see if a single treatment can reduce the need for frequent eye injections while keeping vision stable. About 561 adults a…

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 Phase 3 trial tests an oral drug called deucrictibant in adults with acquired angioedema due to C1 inhibitor deficiency, a rare condition causing sudden, painful swelling attacks. The study has three parts: one to see if a daily extended-release tablet can prevent attacks, a…

This study tests whether a daily probiotic called AKM Lab-01 can safely lower cholesterol levels in people with high cholesterol. About 60 adults aged 40-60 will take either the probiotic or a placebo for a period, and researchers will compare changes in their blood cholesterol a…

This study tests a fetal surgery called FETO for severe congenital diaphragmatic hernia (CDH), a condition where a hole in the diaphragm allows organs to crowd the lungs. Researchers will place a tiny balloon in the fetal windpipe to encourage lung growth, then remove it before b…

This early-phase study tests the drug lorlatinib in children and young adults (ages 1 to 21) with newly diagnosed high-grade glioma, a fast-growing brain tumor, that has a specific genetic change (ALK or ROS1 fusion). The goal is to see if the drug can shrink or control the tumor…

This study looks at how well setmelanotide works in real-world settings for people with Bardet-Biedl syndrome, a rare genetic condition that causes severe obesity and constant hunger. Researchers will track changes in body mass index, cholesterol, liver fat, and quality of life i…

This phase 2 trial is testing whether adding the immunotherapy drug pembrolizumab to standard GVD chemotherapy can improve outcomes for people with Hodgkin lymphoma that has returned or not responded to initial treatment. About 257 participants will receive up to 4 cycles of the …

This study tests a new drug, asciminib, as the first treatment for adults newly diagnosed with chronic myeloid leukemia (CML). If the drug alone does not achieve a deep response within 2 to 3 years, a low dose of another targeted therapy is added. The goal is to see if this appro…

This phase 3 trial is testing a drug called tulisokibart in about 1,200 people with moderate-to-severe Crohn's disease who haven't had success with other treatments. The drug is given by IV or injection and aims to reduce symptoms and heal the gut lining. The study compares tulis…

This early-stage trial tests a single IV dose of VERVE-102, a gene-editing drug designed to permanently lower LDL (bad) cholesterol by turning off a specific gene in the liver. It involves 85 adults with inherited high cholesterol or early heart disease who still need lower chole…

This study is testing the drug T-DXd (Enhertu) in 100 adults with advanced HER2-positive solid tumors who have already tried other treatments and have no good options left. The goal is to see how well the drug works in real-world settings, including how many patients respond and …

This study tests a drug called radiprodil in people with GRIN-related neurodevelopmental disorder, a rare genetic condition that often causes seizures and developmental delays. The trial includes two groups: one with frequent seizures and one with fewer or no seizures. Participan…

This study follows 700 adults with moderate-to-severe plaque psoriasis who are already prescribed risankizumab by their doctor. Researchers will track changes in quality of life, including skin symptoms and daily impacts, over up to 2.5 years. The goal is to see how well the drug…

This early-phase study tests a combination of three drugs (avutometinib, defactinib, and everolimus) in people with recurrent endometrial cancer that has specific genetic changes in the RAS pathway. The main goal is to find a safe dose and understand side effects. About 31 partic…

This early-stage study tests a new medicine called PYC-001, given as an injection into the eye, for people with a rare genetic eye disease (autosomal dominant optic atrophy) caused by a change in the OPA1 gene. The main goal is to check the safety of different doses and schedules…

This study tests a procedure called FETO, done during pregnancy, for babies with a severe congenital diaphragmatic hernia (CDH). The goal is to see if FETO helps more babies survive after birth and have fewer long-term health issues compared to standard care. The study will enrol…

This study tests a new targeted therapy pill called MK-1084, alone or with other drugs, for people with advanced non-small cell lung cancer that has a specific genetic change called KRAS G12C. The goal is to see if the treatment is safe and can shrink tumors. About 130 participan…

This Phase 2 study tests atacicept, a drug that may reduce immune system attacks on the kidneys. About 250 adults with various autoimmune kidney diseases (like IgA nephropathy or membranous nephropathy) will receive weekly injections. Researchers will check safety and measure cha…

This study is testing whether a cochlear implant is safe and effective for infants and toddlers who are deaf in one ear. Sixty children will receive the implant and be followed until age 5. Researchers will compare their hearing abilities to children with normal hearing and to th…

This study looks at whether placing a permanent crown on a dental implant right away (immediate loading) works as well as waiting 4 weeks (early loading) for lower first molars. About 100 adults who need a tooth removed and replaced will get an implant 12-16 weeks after extractio…

This phase 2 trial tests an experimental drug called vamifeport in 84 adults with HFE-related hereditary hemochromatosis, a genetic condition causing iron buildup. The drug aims to reduce liver iron levels, potentially offering an alternative to regular blood removal. Participant…

This early-phase trial tests a new treatment called KN5501, which uses specially engineered immune cells (CAR NK cells) to target and destroy harmful B cells in people with relapsed or refractory immune nephropathy, a kidney disease caused by the immune system attacking the kidne…

This early-stage trial tests whether anti-estrogen drugs (fulvestrant or anastrozole) can shrink inguinal hernias in men aged 50 and older. Thirty men with a symptomatic hernia will receive one of the drugs for 6 months. The main goal is to check safety and see if the hernia gets…

This Phase 2 trial tests FWY003, an experimental drug, in 272 people with geographic atrophy (a form of advanced age-related macular degeneration). The goal is to see if different doses can slow the growth of damaged areas in the eye and preserve vision. Participants receive eith…

This study tests whether adding an experimental mRNA vaccine (V940) to the immunotherapy drug pembrolizumab (Keytruda) can keep lung cancer from returning after surgery. It includes 680 people with stage II to IIIB non-small cell lung cancer whose tumors did not fully respond to …

This phase 2 trial tests a drug called XL092 in 33 people with advanced thyroid cancer that has stopped responding to radioiodine therapy. XL092 works by blocking a protein that helps cancer cells grow. The study aims to see if the drug can slow or stop cancer progression.

This phase 3 trial compares a new drug, BMS-986365, against standard treatments (docetaxel or other hormone therapies) in 960 men with metastatic castration-resistant prostate cancer that has stopped responding to hormone therapy. The main goal is to see if the new drug delays ca…

This study tests two ways to treat a slow heart rate caused by overactive nerves, without using a permanent pacemaker. One method uses freezing (cryoablation) and the other uses burning (radiofrequency) to calm the nerves. Sixty adults aged 18 to 60 with symptoms like fainting or…

This phase 3 trial tests a new drug called FRSW107, a lab-made clotting factor VIII fused with a protein that helps it last longer in the body. The study enrolls males aged 12 to 65 with severe hemophilia A who have had at least 150 prior exposure days to factor VIII products. Pa…

This trial tests a gene therapy for Fanconi anemia, a rare genetic disorder that causes bone marrow failure and increases cancer risk. Participants receive their own stem cells that have been genetically corrected with a lentiviral vector to fix the faulty FANCA gene. The study e…

This study compares Tai Chi and Qi-Gong, two gentle movement therapies, to see which better improves balance and motor function in people with stage I Parkinson's disease. Fifty participants aged 50 to 65 will receive either Tai Chi or Qi-Gong alongside standard physical therapy …

This trial investigates whether specially engineered T cells can safely target and fight lung cancer. Participants have advanced or relapsed lung cancer and receive a single infusion of their own T cells that have been trained to recognize cancer-specific markers. The study aims …

This study tests a decision aid—a tool that provides information and goal-setting exercises—to help women who had high blood pressure during pregnancy take steps to protect their heart health after childbirth. The goal is to improve engagement in preventive care like blood pressu…

This trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare and life-threatening genetic disorder that damages the nervous system. The treatment uses a lentivirus to deliver a working copy of the ARSA gene directly into the spinal fluid and bloodstream. Up to 1…

This trial investigates whether specially engineered immune cells (CAR-T cells) can safely treat advanced or recurrent cervical cancer. The cells are designed to recognize and attack cancer cells that carry certain markers like GD2 or Mesothelin. The study involves 20 adults with…

This study investigates whether a wrist-worn sensor (KinesiaU) can accurately measure how long people with Parkinson's disease experience 'good on-time' — periods when medication works well and symptoms are controlled without troublesome movements. Researchers are testing IPX-203…

This phase 2 trial tests whether combining pembrolizumab (Keytruda) with bendamustine (Treanda) is safe and effective for people with Hodgkin lymphoma that has come back or is no longer responding to standard treatments. About 40 adults will receive the drug combination, and rese…

This trial tests a 4th-generation safety-engineered CAR T cell therapy for people with relapsed or late-stage sarcomas, including osteosarcoma and Ewing sarcoma. Participants receive a single infusion of these immune cells designed to target and attack sarcoma cells. The study ai…

This trial tests whether a new robotic system that uses only one small incision (single port) can safely perform common abdominal surgeries in children. The study includes children aged 1 to 17 who need procedures like pyeloplasty or reflux treatment. The goal is to see if the si…

This clinical trial tests a new type of immunotherapy called 4SCAR-T, which uses a patient's own immune cells engineered to recognize and attack three different markers (GD2, PSMA, and CD276) on neuroblastoma cancer cells. The study enrolls children and adults (ages 1 to 65) with…

This study tests an experimental drug called Tulmimetostat (DZR123) in people with advanced solid tumors or lymphomas that have not improved with standard treatments. The drug is taken as a daily pill, either alone or combined with another drug (enzalutamide) for certain prostate…

This trial compares two blood products—prothrombin complex concentrate (PCC) and fresh frozen plasma (FFP)—for controlling bleeding in people undergoing heart valve surgery. Doctors use a special test to guide which product to give and measure how quickly bleeding stops. The goal…

This trial compares two types of full-arch dental implant frameworks—one made from a single piece of zirconia and the other from titanium with a zirconia top—in people who have lost all teeth in one or both jaws. The goal is to see which material leads to fewer mechanical problem…

This trial investigates whether using a lab test (IL-6) from amniotic fluid to guide antibiotic and steroid treatment can safely prolong pregnancy in people whose water breaks too early (preterm premature rupture of membranes, or pPROM). Participants are randomly assigned to eith…

This study tests a new approach for people newly diagnosed with a specific, aggressive leukemia (Ph+ ALL). It combines a single infusion of specially engineered immune cells (CAR-T cells) with a daily targeted pill (olverembatinib). The goal is to see if this powerful one-two pun…

This trial tests a procedure called FETO, where a small balloon is placed in the windpipe of a fetus with severe left congenital diaphragmatic hernia (CDH). The balloon blocks the airway temporarily, which may help the lungs grow larger before birth. The study includes pregnant w…

This early-stage trial tests BDHK-2009, an experimental tablet for inflammatory bowel diseases like ulcerative colitis and Crohn's disease. The study involves 68 healthy adults aged 18 to 55 to see if the drug is safe and how the body handles it. Participants receive either a sin…

This study investigates whether continuous glucose monitor (CGM) data can pinpoint the time of day when a person with prediabetes experiences the largest blood sugar spikes after eating. Participants wear a CGM and activity tracker for 10 days to identify their personal 'vulnerab…

This study evaluates PathFinder 1.0, a software that analyzes OCT images of the retina to detect macular abnormalities such as epiretinal membrane, dry age-related macular degeneration, and other retinal conditions. About 400 adults with and without macular issues will undergo st…

This study explores whether MRI scans can safely and accurately detect cancerous or precancerous mouth lesions in people with Fanconi Anemia, a rare genetic condition that raises cancer risk. Researchers will compare MRI results to standard biopsies in 80 adult participants. If s…

This study will test a handheld ultrasonic spirometer against a conventional hospital spirometer in 100 adults with chronic respiratory diseases like COPD or asthma. Each participant will use both devices to see if the portable one gives accurate and reliable results. If successf…

This study aims to find better ways to diagnose Parkinson's disease in its earliest stages. Researchers will use wearable devices to track movement and analyze blood samples for specific markers from nerve cells. By combining these digital and biological clues, they hope to creat…

This study tests a new radioactive tracer called 18F-mFBG to see if it can detect nerve damage in the heart caused by Lewy body diseases like Parkinson's and Lewy body dementia. About 20 adults will get a PET scan after an injection of the tracer. The goal is to see if the scan c…

This early-stage study tests a new radioactive tracer called SST001 that lights up on PET scans. Researchers want to see if it is safe and can help tell the difference between multiple system atrophy (MSA) and Parkinson's disease. The study will include 30 healthy volunteers and …

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 early study tests a single oral dose of HB-2121 in 20 adults with suspected celiac disease. The dye is given four hours before a standard endoscopy to see how it interacts with the small intestine and whether it is safe. Researchers will track side effects and measure the dy…

This study explores whether measuring heart rate variability—the natural ups and downs in heartbeat timing—can help detect complications like leaks or abscesses after planned colorectal surgery. Researchers will monitor patients for a week after surgery and compare heart rate pat…

This early-phase trial tests a vaccine (CMV-MVA Triplex) given to both stem cell donors and recipients to boost immunity against cytomegalovirus (CMV) and prevent active infection after a half-matched stem cell transplant. About 46 people with various blood cancers will take part…

This study tests whether a nightly drop of low-dose atropine in both eyes can delay the start of nearsightedness in children aged 6–11 who are not yet nearsighted. Over 600 kids will be randomly assigned to get either the atropine drops or placebo drops for two years. The main go…

This study tests a laser treatment called OSC/SS to prevent retinal detachment in people with Stickler Syndrome, a genetic condition that raises the risk of vision loss. About 500 children and adults will receive the procedure in one or both eyes and be followed for 5 years. Rese…

This study tests if the drug milrinone can prevent a serious heart complication called post-ligation cardiac syndrome (PLCS) in very preterm infants after surgery to close a heart vessel. About 316 babies born before 28 weeks will receive either milrinone or standard care. The go…

This study is testing whether a home-based screening kit can identify hidden heart risks better than standard doctor visits. 45,000 adults in Sweden aged 50-75 will either receive a home blood pressure monitor, a finger-prick blood test, and a health questionnaire, or continue wi…

This study tests whether ursodeoxycholic acid (UDCA), a drug used for bile duct problems, can stop statins from raising blood sugar. 128 adults with high cholesterol but normal blood sugar will take either UDCA or a placebo alongside atorvastatin for 6 months. Researchers will tr…

This phase 3 trial tests MK-8527, a once-monthly oral pill, to prevent HIV-1 infection in women. About 4580 HIV-negative women will take either MK-8527 monthly or a daily PrEP pill (Truvada) to see which works better and is safer. The goal is to offer a more convenient prevention…

This study tests whether Aquamin, a natural mineral supplement from seaweed, can prevent pouchitis—a painful inflammation of the J-pouch—in people with ulcerative colitis who have had their colon removed. About 52 participants will take Aquamin or a placebo for up to 8 months. Th…

This phase 3 trial tests whether acoramidis can prevent or delay transthyretin amyloidosis (ATTR) in 587 adults who carry a faulty gene but have no symptoms yet. ATTR causes sticky plaques to build up in the heart and nerves, leading to heart failure and nerve damage. Acoramidis …

This large phase 3 trial is testing whether a new oral pill called MK-8527, taken just once a month, can prevent HIV infection as well as the standard daily PrEP pill. About 4,390 people at risk of HIV will be randomly assigned to receive either the monthly pill or daily Truvada …

This study compares two different doses of vitamin D3 supplements in pregnant women who have low vitamin D levels. The goal is to see if a higher dose (50,000 IU every two weeks) is better than a lower dose (5,000 IU weekly) at preventing preeclampsia, a serious pregnancy complic…

This trial tests whether a community-based screening program using advanced eye imaging (OCT, fundus photography, and pressure checks) can prevent vision loss from age-related macular degeneration, diabetic retinopathy, and glaucoma better than simple vision tests alone. About 60…

This study tests a smartphone app designed to help teenagers aged 14-18 who self-harm and are not currently in therapy. The app teaches coping skills and connects them with support through a friendly chatbot. The goal is to see if the app is easy to use and helps reduce self-inju…

This study from Yale University will test whether 20 minutes of moderate cycling can reduce obsessive thoughts, compulsive urges, and improve thinking flexibility in adults with OCD. Fifty participants will be randomly assigned to either moderate or low-intensity cycling. Researc…

This study tests whether adding music-like sounds (sonification) to standard walking rehabilitation can improve gait, balance, and quality of life. 120 adults with Parkinson's disease, stroke, or multiple sclerosis will be randomly assigned to standard rehab or rehab with sonific…

This study tests a group therapy program called the Unified Protocol for adults with emotional disorders such as anxiety, depression, and related conditions. The program teaches skills like emotional awareness, flexible thinking, and facing fears. Researchers want to see if it im…

This study tests whether a four-session mindfulness class (Primary Care Brief Mindfulness Training) can help veterans reduce symptoms of anxiety, depression, and PTSD compared to a problem-solving class. The classes are designed for primary care settings and focus on meditation, …

This study is testing whether a specially designed amino acid supplement can improve nutrition, reduce oxidative stress, and boost physical and mental health in people with Parkinson's disease. Thirty adults aged 60-80 with Parkinson's will take either the supplement or a placebo…

This study tests whether a mobile app that provides psychological tools can improve quality of life for people with eczema, psoriasis, or chronic hives. Participants use the app over 32 weeks to learn coping strategies and wellness tips. The goal is to see if this digital approac…

This pilot study tests whether a gentle, non-invasive electrical current applied to the brain can improve how people with tunnel vision (from retinitis pigmentosa, rod-cone dystrophy, or advanced glaucoma) navigate obstacles. Twenty adults will receive both real and sham stimulat…

This study tests a personalized program combining exercise and nutrition advice to help reduce fatigue in people with indolent (slow-growing) lymphomas, such as chronic lymphocytic leukemia and follicular lymphoma. Participants receive an individualized exercise plan and dietary …

This study tests whether two types of facial sunscreen (untinted and tinted) can improve dark spots caused by melasma, sun spots, or post-inflammatory hyperpigmentation. Adult participants with mild to moderate dark spots apply the sunscreen at least twice daily for 56 days. Rese…

This study tests whether online group education and lifestyle coaching can improve quality of life and health markers in women with PCOS and obesity. Forty non-pregnant women aged 18-49 will be assigned to either the virtual program or usual care. Researchers will measure changes…

This study tests whether a mobile app with short weekly exercises can help first-time parents manage emotions and reduce depression during pregnancy. About 572 expectant mothers and fathers in Singapore will use the app or receive standard care. Participants will also wear a Fitb…

This study tests whether a non-invasive brain stimulation technique called HD-tDCS can improve visual and thinking abilities in people with posterior cortical atrophy (PCA), a rare condition often linked to Alzheimer's. Fifty participants will receive either real or sham stimulat…

This trial tests whether adding dexmedetomidine or magnesium sulphate to lidocaine for a peribulbar block (anesthesia around the eye) helps keep heart rate and blood pressure steady during dacryocystorhinostomy (tear duct surgery) under general anesthesia. Sixty adults undergoing…

This pilot study tests whether a single session of breathing air with slightly higher carbon dioxide and lower oxygen can improve speech, cough, and movement in people with Parkinson's disease. Participants are randomly assigned to one of two breathing patterns, each lasting abou…

This study explores whether a mobile app called FlowMD can help adults with mild anxiety. Participants use the app for 1-5 minutes of guided breathwork each day for four weeks. Surveys measure changes in anxiety levels and how easy the app is to use. The goal is to see if this ap…

This study tests an investigational drug called gemibotulinumtoxinA (a type of botulinum toxin) injected into arm muscles to reduce tremor in adults with essential tremor. About 94 participants will receive either the drug or a placebo injection. Researchers will measure changes …

This study tests whether wearing a headset that delivers a flickering 60Hz white light for 30 minutes a day can help ease depression symptoms. Adults with major depressive disorder will use the device at home for three weeks. Researchers will measure how well people stick with th…

This study tests whether osteopathic manipulative treatment (OMT) — a hands-on therapy using gentle techniques on the head, neck, and ribs — can improve sleep quality in people with Parkinson's disease. Participants are split into two groups: one receives OMT, the other a light t…

This study tests a new online therapy platform called Teleo, designed specifically for children with anxiety or mood disorders. Researchers want to see if kids are more engaged during therapy sessions on Teleo compared to standard video calls. About 156 children will take part, a…

This study tests whether the drug sonlicromanol can reduce fatigue and improve physical abilities like balance and leg strength in adults with a specific genetic form of mitochondrial disease. About 220 participants will take either the drug or a placebo twice daily for 52 weeks.…

This study tests whether a personalized form of transcranial magnetic stimulation (TMS) can improve motor and non-motor symptoms in people with Parkinson's disease, multiple system atrophy, or progressive supranuclear palsy. Fifty participants will receive 10 days of targeted TMS…

This study tests whether a non-invasive brain stimulation technique called TMS can help with apathy—a common lack of motivation—in people with Parkinson's disease. Sixty participants will receive TMS to two different brain areas on separate visits and perform a task where they ch…

This study tests a drug called golexanolone in 84 people with primary biliary cholangitis (PBC) who suffer from significant fatigue and cognitive problems. Participants receive either the drug or a placebo for up to 28 days. The goal is to see if the drug is safe and can improve …

This study tests whether nerve gliding exercises, tendon gliding exercises, or a combination of both can reduce pain and improve hand function in people with mild to moderate carpal tunnel syndrome. Participants will be randomly assigned to one of three groups and followed for ch…

This study tests a new type of lens called Clareon TruPlus, used during cataract surgery to replace the eye's natural lens. Researchers want to see how well it improves vision and how satisfied patients are up to 3 months after the procedure. The study involves 40 adults over 40 …

This study investigates whether breathing a 50:50 mix of nitrous oxide (laughing gas) and oxygen can reduce pain and anxiety during insertion of a central venous catheter, a common but uncomfortable procedure. Adults aged 18 to 65 who have never used nitrous oxide will be randoml…

This study looks at how well vaccines for flu, COVID-19, shingles, hepatitis B, and pneumonia work in people with certain blood cancers like chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphomas. Researchers will measure immune responses through blood samples before and af…

This study aims to better understand and treat people with certain inherited metabolic or genetic disorders. Researchers will use standard medical tests like blood work and imaging to diagnose and care for participants, who may also join other related studies. The goal is to expa…

This study uses a non-invasive brain stimulation technique called transcranial magnetic stimulation (TMS) to examine how nerve pathways from the brain to the legs and trunk are affected in people with Parkinson's disease. Researchers will measure brain activity and link it to wal…

This study aims to find genetic causes of rare diseases in children aged 2 to 18 who live outside the United States and have limited access to genetic testing. Researchers will collect blood, saliva, or cheek swab samples from affected children and their family members. The goal …

This study aims to learn more about Hyper IgE syndrome, a rare immune disorder that causes skin and lung infections. Researchers will follow 600 patients and their relatives over time, examining their health and genetics. The goal is to better understand the disease and improve c…

This study collects blood stem cells and immune cells from healthy volunteers and patients with primary immune deficiencies or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advan…

This study tests a new MRI method that uses an inhaled gas to create detailed images of lung ventilation in people with cystic fibrosis. Researchers will track 30 adults with mild lung disease over a year, comparing the MRI results to standard breathing tests. The goal is to see …

This study follows healthy girls ages 8-14 and teens with irregular periods to learn how their menstrual cycles become regular after their first period. Researchers also include girls at risk for PCOS and compare them to women with and without PCOS. The goal is to understand norm…

This study explores whether a small accelerometer worn on the thigh can objectively measure how well treatments work for neurogenic orthostatic hypotension—a condition causing dizziness and fainting upon standing. Participants wear the device for one week on placebo and one week …

This study collects blood and tissue samples from 2000 people—some with cancer and some without—to create a blinded reference set. Researchers will use these samples to evaluate how well new blood tests can detect many types of cancer early. The goal is to improve the accuracy of…

This study aims to learn more about methylmalonic acidemia (MMA) and related disorders by observing people with these conditions over time. Researchers will track complications, perform tests like blood draws and MRIs, and look for new genetic causes. The goal is to better unders…

This study provides ongoing check-ups for people who received a donor stem cell transplant at the NIH at least three years ago. Researchers will monitor for late side effects, disease return, and overall health. Participants will have yearly visits that may include blood tests, b…

This study from the National Human Genome Research Institute aims to better understand Congenital Disorders of Glycosylation (CDG), a group of rare genetic conditions that affect how the body attaches sugars to proteins and fats. Researchers will examine up to 200 participants, i…

This study screens up to 2,000 people who have or may have immune disorders that make them prone to infections, along with their healthy relatives. Researchers will use medical history, physical exams, blood tests, and genetic testing to better understand these conditions. The go…

This study looks at whether a Mediterranean diet can improve gut health in children with cystic fibrosis. Twenty kids aged 3 and older will follow this diet for 6 months. Researchers will check changes in gut bacteria and inflammation markers. The goal is to offer better diet adv…

This study uses special PET scans to measure inflammation in the brains of people with Alzheimer's, Parkinson's, ALS, and other neurodegenerative diseases. Researchers will compare these scans to those from healthy volunteers to see if inflammation levels differ. The goal is to d…

This study looks at how well people with Anderson-Fabry disease can exercise and how their hearts respond to stress. Researchers will use treadmill tests, echocardiograms, and heart scans to measure heart function during rest and exercise. The goal is to find better ways to detec…

This study at the National Institutes of Health looks at how infections can worsen symptoms in people with mitochondrial disease, a group of disorders that affect energy production in cells. Researchers will evaluate participants' immune systems through blood tests, physical exam…

This early-phase study will give 9 healthy adults a single dose of the experimental HIV drug VH4524184, plus a tiny radioactive version to track it. Researchers will measure how much of the drug gets into the blood, how it's broken down, and how it leaves the body through urine a…

This study is testing whether a skin biopsy can detect abnormal proteins linked to Parkinson's disease in people with essential tremor, a common movement disorder. Researchers will follow 300 participants aged 50-85 for two years to see who develops Parkinson's. The goal is to fi…

This study is looking for the genes that cause certain immune system problems. Researchers want to understand why some people get sick and others don't, even in the same family. Up to 5,000 people with immune disorders and their relatives will give blood or cheek cell samples for…

This study aims to learn more about how lupus (SLE) develops and changes over time by observing patients and their relatives. Up to 2000 people aged 3 and older with known or suspected lupus, along with their family members, will provide medical history, blood and urine samples, …

This study follows up to 1,000 adults with certain slow-growing blood cancers (CLL, SLL, LPL, WM) to track how the diseases change over time. Participants have regular check-ups and blood draws every 6 to 24 months, and may also have optional biopsies or scans for research. No tr…

This study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

This study looks for better ways to find harmful bacteria in the lungs of people with cystic fibrosis. Traditional sputum tests are becoming harder to use because newer CF treatments reduce mucus. Researchers will collect samples like saliva, blood, urine, and breath from 300 par…

This study looks for genetic changes linked to progressive familial intrahepatic cholestasis (PFIC) in adults with unexplained bile flow problems. Researchers will collect health information and blood samples from 150 participants across Spain during a single visit. The goal is t…

This study follows people with inherited BAP1 gene mutations, which raise the risk of mesothelioma and other cancers. Researchers will track participants over many years, collecting medical history, blood samples, and scans to understand how these mutations lead to cancer. The go…

This study watches people with blood cancers like CLL who take BTK inhibitors or venetoclax to see how these drugs affect heart rhythm. Researchers will use EKGs, stress tests, and heart monitors to track abnormal rhythms and sudden death risk. The goal is to better understand an…

This study looks at how leishmaniasis infections progress and respond to treatment. It involves up to 289 people aged 3 to 100 with known or suspected leishmaniasis. Researchers will collect blood and tissue samples to learn more about the disease and test different treatments. T…

This pilot study looks at how belzutifan, a drug already used for some tumors, changes the production of adrenaline-like hormones in people with pheochromocytoma and paraganglioma (PPGL). Over 5 days, researchers measure key substances in the hormone-making process to see if belz…

This study follows children and adults with medullary thyroid cancer (MTC), often linked to a genetic condition called MEN2. Researchers will track how the cancer grows and affects health over time using regular check-ups, blood tests, and imaging. No treatment is given, but part…

This observational study is gathering medical and genetic information from up to 1,250 people with known or suspected genetic conditions, as well as their relatives. Researchers will use advanced data analysis to find new insights into how genes cause disease. Participants may pr…

This study enrolls people with endocrine tumors (such as thyroid, parathyroid, adrenal, or pancreatic tumors) who are scheduled for surgery or biopsy. Researchers collect samples of the tumor, nearby healthy tissue, blood, and urine to analyze genetic and molecular changes. The g…

This study looks at children with suspected or confirmed genetic and metabolic disorders to find new disease-causing gene mutations. Researchers will analyze blood samples for DNA and metabolites, and in some cases take a small skin sample. The goal is to better understand these …

This study follows 155 adults with CADASIL, a genetic condition that narrows small blood vessels and raises the risk of strokes and dementia, over nine years. Researchers will use brain scans, blood tests, and other exams to track how the disease changes blood vessels and thinkin…

This study examines how common eczema treatments, including antibiotics and bleach baths, change the types and amounts of bacteria on the skin. Researchers will compare people with eczema to healthy volunteers. The goal is to understand the skin microbiome better, not to test a n…

This study aims to uncover genetic changes that cause autoimmune diseases in people native to Peru. Researchers will collect blood, urine, and stool samples from 300 participants, including patients with conditions like lupus and rheumatoid arthritis, their family members, and he…

This study aims to understand how HIV behaves differently in the blood compared to lymph nodes and bone marrow. Researchers will take samples from 400 people living with HIV and some without HIV to measure virus levels and immune system changes. The goal is to learn more about wh…

This study aims to learn more about Hermansky-Pudlak Syndrome (HPS), a rare inherited disease that causes light skin/eye color, bleeding problems, and often deadly lung scarring. Researchers will follow 600 people with HPS and their family members to track how the disease progres…

This registry study follows about 100 people with FOP, a rare disease where soft tissue turns to bone. Some participants take palovarotene (an approved treatment), while others do not. Researchers will track side effects, flare-ups, and daily function over time to see how well th…

This study follows people with a rare genetic condition called DICER1 syndrome, which raises the risk of certain childhood cancers like pleuropulmonary blastoma. Researchers will collect medical histories, genetic samples, and tissue from up to 1,500 participants and their relati…

This study looks at whether people with a curved spine (sagittal imbalance) from lumbar spinal stenosis can have their spine naturally straighten after a simple decompression surgery, without needing a more complex fusion procedure. Researchers will measure spine alignment change…

This study follows 900 people with bronchiectasis—a condition where airways are damaged and prone to infection—to learn why they get sick repeatedly. Researchers will collect medical history, lung function tests, and blood, urine, and sputum samples from patients and their family…

This study follows people with propionic acidemia (PA), a rare metabolic disorder, to track how the disease affects the body over time. Researchers will collect medical history, blood, urine, and other samples, and perform imaging and heart tests during annual hospital visits. Th…

This study aims to understand how genetics and environmental factors contribute to melanoma, a serious skin cancer. Researchers will enroll up to 3,000 people with a personal or family history of melanoma or related conditions. Participants will fill out questionnaires, provide b…

This study collects blood, urine, saliva, and tissue samples from nearly 6,000 people who have or may have cancer, including rare inherited forms. The samples are stored and used for research to understand the genetic and molecular basis of cancer. No new treatment is being teste…

This international observational study will follow 50,000 people with Alzheimer's disease or related dementias for many years. Researchers will collect real-world data from routine doctor visits to track how the disease progresses and how treatments are used and tolerated. The go…

This study aims to uncover the genetic causes of hereditary leiomyomatosis renal cell cancer (HLRCC), a condition that can lead to painful skin bumps, uterine fibroids, and kidney tumors. Researchers will study people with known or suspected HLRCC and their family members to iden…

This study follows 500 people with geographic atrophy, an advanced form of age-related macular degeneration that causes permanent vision loss. Participants receive avacincaptad pegol (IZERVAY) as part of their routine care. Researchers will collect data on treatment patterns, sid…

This study is observing up to 3,500 people with immune system problems, including rare genetic conditions like chronic granulomatous disease and severe combined immunodeficiency. Researchers will track how these diseases change over time through blood tests, imaging, and checkups…

This study looks at families where non-medullary thyroid cancer runs in the family. Researchers want to learn how the disease develops, find genes that might increase risk, and figure out the best way to screen for it early. Participants will have exams, imaging, and blood tests,…

This study follows up to 3,500 people with sickle cell disease to learn more about how the condition progresses and what complications arise. Participants receive their usual medical care outside the hospital and visit the clinic for check-ups and research tests. No experimental …

This study aims to find the genetic causes of inherited immune problems that affect white blood cells called lymphocytes. Researchers will review medical records and collect blood samples from up to 5,000 people with suspected immune disorders, as well as their relatives. The goa…

This study aims to learn more about the genetics and natural history of autoinflammatory diseases, which cause repeated fevers and inflammation. Researchers will study up to 5,000 people, including patients, their relatives, and healthy volunteers. Participants provide blood and …

This study aims to understand the genetic and biological causes of Proteus syndrome, a rare condition causing abnormal growth of bones, skin, and other tissues. Researchers will follow up to 1,500 participants over time, using medical exams, imaging, and blood tests to track how …

This study explores a new type of MRI that uses an inhaled gas to create images of lung ventilation in children ages 6-17. Researchers will compare results between children with mild cystic fibrosis and healthy children to see if the technique is feasible and sensitive to early l…

This study collects extra blood, urine, and tumor samples from children and adults with certain cancers to build a large tissue repository. Researchers will analyze these samples to learn more about the genetic and molecular makeup of these tumors. The goal is to support future s…

This study will carefully examine the physical and mental health of 100 adults admitted to NHS mental health wards. Researchers will review medical notes, do physical exams, and use questionnaires to measure conditions like bipolar disorder, schizophrenia, depression, and persona…

This study follows adults who have recently received a cochlear implant to track how their ability to distinguish sounds and understand speech changes over time. Participants complete at-home sound tests frequently during the first three months, then return for lab tests up to on…

This study aims to learn more about heart and blood vessel diseases by observing people who have them or are at risk. Researchers will collect samples and perform tests like imaging and stress tests on up to 5,000 participants, including healthy volunteers and relatives of affect…

About 1 in 4 women have their water break before labour starts. Most go into labour within 48 hours, but some need to be induced. This study tests whether deliberately breaking the leftover membrane when a woman is admitted to the hospital can shorten the time to labour and reduc…

This study looks at how mucus plugs in the lungs affect breathing in people with severe asthma. Researchers will use special MRI scans to see air flow in blocked areas. Only 5 adults with severe asthma will take part, and the goal is to better understand lung function, not to tes…

This study looks at whether anxiety or depression makes asthma harder to control and aims to build a model that predicts when a serious asthma attack might happen. Researchers will follow 256 people with asthma for up to two years, tracking their symptoms, mental health, and lung…

This study looks at the causes and effects of food allergies and related conditions like eczema and eosinophilic esophagitis. Researchers will collect blood, skin, and genetic samples from up to 1,800 people over many years. The goal is to find biomarkers and pathways that explai…

This study investigates how the eye's focusing ability changes after 20 minutes of looking at a screen up close (25 cm) compared to looking at a distant object (10 m). Healthy adults aged 18-35 will perform focusing tasks while a wavefront sensor measures their eye's response. Th…

This study tracks the safety of Fycompa injection in people with epilepsy who have partial-onset seizures (age 4 and older) or primary generalized tonic-clonic seizures (age 12 and older). Researchers will record any side effects and measure drug levels in children aged 4 to 12. …

This study will test how the heart drug empagliflozin is absorbed and processed in 10 children with Duchenne muscular dystrophy, ages 8 to 18. The goal is to find the best dose for protecting the heart in this rare disease. Participants will take the drug by mouth and have blood …

This study investigates whether the quality of parent-adolescent communication at the start of psychiatric inpatient treatment predicts how much symptoms improve six months later. Researchers will track 60 adolescents hospitalized for depression, anxiety, or other mental disorder…

This study follows people with rare genetic disorders that cause harmful substances to build up in the body, leading to brain damage. Researchers will monitor up to 200 participants over time using exams, surveys, and lab tests. The goal is to better understand how these diseases…

This study looks at how the types of bacteria in the mouth change in people with severe gum disease as they go from having some teeth left to having no teeth, and then after getting dental implants. Researchers will collect microbial samples and analyze DNA to see if different im…

This study looks at whether isotretinoin, a common acne medicine, changes how well people can smell. Researchers will test the sense of smell in 54 adults before and after at least 4 weeks of treatment. The goal is to understand if the drug's drying effect on the nose can reduce …

This study investigates whether a protein called lumican, found in the blood, can identify postmenopausal women who have fragile bones but normal bone density scans. About 100 women will have their bone density, muscle strength, and blood lumican levels measured. The goal is to s…

This study follows 300 adults aged 30 and older who have or may have a BAP1 gene mutation, which raises the risk for mesothelioma and other cancers. Researchers will use advanced CT scans, blood tests, and minimally invasive camera procedures to look for early signs of cancer. Pa…

This study observes about 170 children and adults with achondroplasia in the United States over roughly 5 years. Researchers will collect health records and questionnaires to track growth, medical issues, surgeries, and quality of life. The goal is to better understand the condit…

This study investigates how changes in kidney cell metabolism relate to the rate of kidney function decline in people with ANCA vasculitis and other kidney diseases. Researchers will analyze kidney biopsy samples and blood/urine tests from 146 participants over one year. The goal…

This study creates a large registry of people with Rett syndrome, a rare genetic disorder that causes severe intellectual and physical disabilities. Researchers will collect information from doctors and caregivers to understand how the disease progresses over time. The goal is to…

This study aims to understand what happens in the brain when someone thinks about or attempts suicide. Researchers will use brain scans, sleep monitoring, blood tests, and questionnaires to compare four groups: people with recent suicidal thoughts or attempts, those with past sui…

This study is creating a large collection of health information and biological samples (like blood, saliva, and eye tissue) from people with and without retinal diseases. Researchers aim to use this database to better understand conditions like age-related macular degeneration an…

This study enrolls 324 parent-child pairs to see if a specially designed educational website helps parents feel more supported during hypospadias care. Parents are randomly assigned to either the study website or a basic information site. The goal is to learn how to improve commu…

This study screens males with Duchenne muscular dystrophy (ages 0 to under 25) for antibodies against AAV8, a virus used in some gene therapies. The goal is to find out how many have these antibodies and to identify who might be eligible for future gene therapy trials. No treatme…

This study looks at how the brains of children with anxiety disorders work differently from healthy children. Researchers will use brain scans and thinking tasks to track changes over a year, before and after standard treatments like therapy or medication. The goal is to find pat…

This study collects blood, bone marrow, and tissue samples from people with HIV, KSHV, or certain cancers to help researchers learn more about these diseases. Participants must be 18 or older and may have HIV, KSHV, or related conditions. No treatment is given; the goal is to gat…

This study follows 600 people with GATA2 deficiency, a genetic disorder affecting the immune system and other body systems. Researchers aim to understand the full range of health problems it causes and why it affects people differently. Participants will have yearly check-ups for…

This study looks at a new tool called the Functional Muscle-Bone Incongruity Index (FKUI) that combines handgrip strength and bone density measurements. Researchers want to see if this combined approach gives a fuller picture of muscle and bone health than looking at each measure…

This study screens 360 adults with neurodegenerative disorders like ALS, frontotemporal dementia, and progressive supranuclear palsy. Researchers will use medical history, physical exams, memory tests, movement analysis, MRI scans, and other tests to find common features and diff…

This study at Necker Hospital in Paris is checking how well patients with inherited metabolic diseases follow their daily oral medication routines. About 200 patients (children aged 7 and up, teens, and adults) will fill out a questionnaire during a regular visit. The goal is to …

This study explores whether a computer algorithm can analyze resting-state MRI scans taken before surgery to predict how long a person with glioblastoma multiforme (a type of brain cancer) might survive. The goal is to see if the algorithm can accurately classify patients as shor…

This study is creating a large collection of medical data and blood samples from 450 adults with autoimmune diseases (rheumatoid arthritis, lupus, or scleroderma) and 50 healthy volunteers. Researchers will use advanced lab techniques to find unique biological markers that could …

This study aims to find biological markers that can track the progression of Niemann-Pick type C (NPC), a rare genetic disorder that damages the nervous system. Researchers will evaluate 900 patients of any age through regular checkups, spinal taps, eye exams, and brain scans. Th…

This study follows people with autoimmune lymphoproliferative syndrome (ALPS) and their relatives over many years. Researchers will collect blood and tissue samples, track symptoms, and study genetics to better understand why the immune system attacks the body. The goal is to lea…

This study collects wisdom teeth and surrounding tissue that are normally thrown away after oral surgery. Researchers will use these samples to learn more about oral health and disease. Up to 3,000 people aged 16 to 50 who need their wisdom teeth removed can join. The study also …

This study aims to learn more about diseases that affect the salivary glands, which produce saliva. Researchers will collect medical data, blood, saliva, and tissue samples from people with these conditions, their relatives, and healthy volunteers. The goal is to better understan…

This trial investigates whether a caudal nerve block, a common pain-relief method for children, increases the chance of a fistula (an abnormal opening) after surgery to correct hypospadias, a birth defect where the urine opening is on the underside of the penis. About 80 boys age…

This study looks at the bacteria, fungi, and viruses living on the skin, in the gut, and in the mouth of people with Netherton syndrome, a rare condition that damages the skin barrier and often causes allergies. Researchers will compare these microbes to those of healthy voluntee…

This study follows up to 500 people with polyostotic fibrous dysplasia or McCune-Albright syndrome to learn how these conditions change over time. Researchers will collect medical data and tissue samples to better understand the disease. The goal is to improve future diagnosis an…

This study looks at people with unusual or repeated infections and their family members to find the genetic and biochemical causes of immune system problems. Researchers will collect blood, saliva, urine, and other samples over time to better understand these conditions. The goal…

This study looks at why people with tuberous sclerosis, a rare genetic condition, develop non-cancerous skin tumors. Researchers will examine up to 400 adults, perform skin exams, and take small tissue samples (biopsies) from tumors. The goal is to identify the cells and genetic …

This study reviews 10 years of medical records from 600 adults who had thyroid surgery at Minia University Hospital. The goal is to find out which patients are most likely to develop low calcium levels after surgery, a common complication. By identifying key risk factors, doctors…

This study aims to understand how heart problems develop in people with Duchenne and Becker muscular dystrophy, as well as in carriers. Researchers will collect cardiac MRI scans and clinical data from 1,000 participants to build a registry. Using advanced image analysis and deep…

This study aims to find the genes that keep airways healthy and understand how genetic mutations cause primary ciliary dyskinesia (PCD), a rare disease that leads to serious lung problems. Researchers will study people with suspected PCD and healthy family members. The goal is to…

This study investigates whether a small RNA molecule called tRF-31 is linked to the severity of age-related cataracts. Researchers will collect lens tissue from 200 adults undergoing routine cataract surgery and measure tRF-31 levels. The goal is to see if higher or lower levels …

This study enrolls 600 adults with Huntington disease to examine specific genetic markers (SNPs) linked to the disease. Participants provide a blood sample and answer questions about their health and medications. The goal is to better understand the genetics of Huntington disease…

This study looks at how a simple monitor called the Pleth Variability Index (PVI) changes during laparoscopic-assisted vaginal hysterectomy. Researchers will track PVI at six key moments when the patient's body position changes, such as tilting the head down or raising the legs. …

This study collects blood, urine, and skin samples from people with rare genetic diseases whose genetic cause is not yet known. The goal is to build a biobank that researchers can use to confirm whether certain gene variants are responsible for these diseases. By identifying new …

This study explores whether a blood test that detects circulating tumor DNA (ctDNA) can help doctors personalize treatment for people with five types of advanced cancer: non-small cell lung cancer, gastrointestinal stromal tumors, colorectal cancer, biliary tract cancer, and ovar…

This study looks at children under 15 with neurofibromatosis type 2 (NF2), a rare genetic condition that causes nerve tumors. Researchers want to see if skin and mouth lesions can help diagnose NF2 earlier than current methods, which rely on hearing, nerve, and eye symptoms. By e…

This study will check if a protein called periostin in the blood is related to how much back pain limits daily activities and quality of life. Researchers will measure periostin in 38 adults with lumbar disc problems and compare it to their scores on disability and health questio…

This study is building a huge registry of information and biological samples from adults with or at risk for many types of cancer, as well as healthy volunteers. Researchers will use this resource to study what causes cancer and how to better prevent, detect, and treat it. The re…

This study is testing a new leg exoskeleton designed by the NIH to help children and young adults with muscle weakness from conditions like cerebral palsy or spinal cord injury. Researchers want to see how well the device works in different real-world settings, like walking on a …

This research study creates a registry of older adults with cancer to collect information about their health, treatments, and outcomes. Participants complete a geriatric assessment and allow researchers to follow their medical records. The goal is to better understand how cancer …

This study looks at whether taking NSAIDs (like ibuprofen) changes the level of a protein called periostin in the blood of people with degenerative disc disease. Researchers will compare three groups: patients using NSAIDs, patients not using NSAIDs, and healthy people without ba…

This study follows a large group of people with HIV over many years to track their health, treatments, and any complications. Researchers will collect medical data during regular clinic visits to better understand how the disease and its treatments affect patients over time. The …

This study is collecting live tumor samples from 2,000 cancer patients undergoing standard biopsies. Researchers will analyze these samples to understand how tumors respond to immunotherapy drugs outside the body. The goal is to develop a method to predict which patients will res…

This study follows people who previously took part in type 1 diabetes trials to see how long their bodies continue to produce insulin. Researchers will also look at how immune system changes relate to insulin production. The goal is to gather information that could lead to better…

This study follows about 50 boys under 18 with X-linked myotubular myopathy (XLMTM), a rare genetic muscle condition, to see how often they develop liver problems like cholestasis. No drugs are given—researchers simply collect health data, including liver scans, over one year. Th…

This study creates a registry of medical records, scans, and surveys from adults with neurofibromatosis type 1 (NF1) who also have a glioma brain tumor. Researchers will track diagnosis, treatments, and quality of life over time to better understand the disease. The goal is to im…

This study looks at how sleep problems might affect thinking, memory, and brain health in 65 people aged 12–25 with sickle cell disease. Participants will use a home sleep monitor and complete thinking tests during a clinic visit. The goal is to understand links between nighttime…

This study aims to better understand disorders that cause hearing to fluctuate, such as Meniere's disease. Researchers will use advanced MRI scans and blood tests to look for biomarkers linked to hearing changes. The goal is to identify different types of these disorders and pave…

This study examines whether a person's personality traits before they got sick are linked to the thinking and behavior problems seen in certain brain disorders. Researchers will ask caregivers about the patient's personality from the 10 years before the illness started. The goal …

This study follows 500 adults with lupus to see if blood and urine tests can reliably detect kidney inflammation (lupus nephritis). Participants give samples during routine visits, and doctors track their kidney health over time. The goal is to find simple, non-invasive markers t…

This study looks at anxiety related to cancer screenings in adults with Fanconi anemia, a rare condition that raises cancer risk. Researchers will observe 20 participants during their regular screening visits, noting body language, worries, and the clinic environment. No treatmen…

This natural history study aims to gather information from up to 3,000 people with genetic conditions that cause too much androgen (male-like hormones), such as congenital adrenal hyperplasia (CAH) and familial male-limited precocious puberty (FMPP). Participants undergo various …

This study is tracking 500 pregnant women with lupus to see if the drug anifrolumab is linked to birth defects or pregnancy loss. Researchers will compare women who took anifrolumab during early pregnancy to those who did not. The goal is to gather real-world safety data, not to …

This international study aims to collect genetic and health information from 1,500 people with rare inherited retinal diseases like retinitis pigmentosa. It has two parts: a registry to gather genetic and vision data, and a natural history study that follows participants over tim…

This study looks at how children with Crohn's disease or ulcerative colitis burn energy at rest. Researchers will measure resting energy expenditure using a special breathing test and compare it to standard formulas. The goal is to see if energy use is linked to disease activity …

This study uses computer models to compare four types of hip socket surgery in children aged 2 to 18 with hip dysplasia. Researchers will create 3D models from MRI scans and simulate each surgery to see how they change socket shape and volume. The goal is to understand which tech…

This study looks at whether childhood trauma is linked to changes in stress hormones and inflammation in obese women. Researchers will measure cortisol levels in saliva and urine, along with other markers, in 102 women. The goal is to better understand how past experiences might …

This study looks at gum disease in people with rare kidney disorders like Alport syndrome, Fabry disease, and tuberous sclerosis, as well as lupus. Researchers will compare 100 participants to those with chronic kidney disease and healthy controls. They aim to understand how comm…

This study compares a smartphone chatbot to usual care for collecting family history and identifying women at high risk for hereditary cancer syndromes. Participants are English-speaking gynecology patients aged 18 and older who have not had prior genetic testing. The goal is to …

This study follows children and adults worldwide who have a rare genetic muscle disease called TNNT1 myopathy. Researchers aim to document how the disease progresses over time, including survival and motor milestones. The goal is to identify reliable measures that can be used in …

This study looks at whether certain proteins in the blood can help doctors monitor severe traumatic brain injury and predict who might benefit from hyperbaric oxygen therapy. Researchers will analyze blood samples from 150 patients already enrolled in a larger trial testing diffe…

This study follows 50 children who had a nerve-freezing procedure (cryoneurolysis) before surgery to correct a sunken chest (pectus excavatum). Researchers check for long-term nerve pain, quality of life, and sensory recovery using questionnaires. The goal is to see if the pain r…

This study creates a registry for people diagnosed with Wilson disease, a rare genetic condition that causes copper buildup in the body. Researchers will collect medical data and biological samples over time to help future studies improve diagnosis and treatment monitoring. The g…

This study investigates the specific cognitive strengths and weaknesses of children and adults with Sturge-Weber Syndrome (SWS), a rare condition affecting blood vessels in the brain, skin, and eyes. Participants complete a series of tests measuring memory, attention, language, a…

This study examines the medical records of 400 people diagnosed with various types of lymphoma at a French hospital between 1999 and 2018. Researchers want to see how long patients survived and whether the care they received matched what medical guidelines recommend. The goal is …

This study compares carbon dioxide emissions from orthopedic surgeries performed with different materials and setups. Researchers will track waste, energy use, and sterilization methods for procedures like carpal tunnel release or wound revision. The goal is to identify ways to r…

This study looks at whether a simple, non-invasive fingertip sensor (called pleth variability index, or PVI) can help doctors track blood pressure and fluid needs during skull surgery in children aged 0 to 6 years with craniosynostosis. The researchers will compare PVI readings w…

This study investigates why some children still have speech problems (velopharyngeal insufficiency) after cleft palate surgery. Researchers will use real-time MRI to look at how the soft palate moves and its muscle structure in children aged 7 to 12. By comparing children with di…

This study aims to determine how common sickle cell trait and sickle cell disease are across Uganda by analyzing up to one million blood samples. Researchers want to create a detailed map of where the condition is most prevalent, which could help improve healthcare planning. The …

This study investigates how the gut and brain communicate in people with Irritable Bowel Syndrome (IBS) who also experience anxiety or depression. Researchers will compare 100 IBS patients with anxiety or depression to 100 healthy volunteers using brain scans, stool samples, and …

This study creates a registry to collect health information from people with Autosomal Dominant Polycystic Kidney Disease (ADPKD), the most common genetic cause of kidney failure. While ADPKD was once thought to only affect adults, researchers now know it begins in childhood, but…

This study investigates whether advanced treatments for Crohn's disease also improve sleep quality. Adults with active Crohn's who are about to start a new advanced therapy will wear a Fitbit Inspire 3 for five days at three different times: before treatment, at week 4, and at we…

This study aims to better understand pre-eclampsia and intrauterine growth restriction (IUGR), serious pregnancy complications affecting about 9% of pregnancies. Researchers will collect placenta, blood, and urine samples from 500 pregnant women—both with and without these condit…

This study investigates how exercise affects the structure and function of fat tissue in adults aged 18-40 with obesity or metabolic syndrome. Participants are randomly assigned to either an exercise program or a sedentary control group. All undergo a 10% weight loss phase, then …