Disorder of development or morphogenesis

This study tests an experimental drug called ATV-1601 in people with moderate to severe HHT, a condition that causes frequent nosebleeds and anemia. The trial has two parts: first, participants receive either the drug or a placebo to check safety and effectiveness; then, all elig…

This trial tests whether a new transcatheter heart valve (Myval) works as safely and effectively as two existing valves (Sapien and Evolut) in people with severe aortic stenosis, a condition where the heart's main valve narrows. About 768 patients who are eligible for valve repla…

This Phase 2 trial tests whether combining pembrolizumab (an immunotherapy) with aldesleukin (IL-2) can shrink tumors in people with advanced melanoma or kidney cancer that hasn't responded to prior treatments. About 78 adults will receive two cycles of the combo in the hospital,…

This early-phase trial is exploring whether a radioactive drug called [211At]MABG, given in several smaller doses, is safe and tolerable for children with neuroblastoma that has relapsed or not responded to treatment. The study will include about 10 participants aged 1 year and o…

This early-stage study tests a new medicine called PF-08046033 in adults with advanced lung, esophageal, or skin cancers that have spread or can't be removed. The main goal is to check safety and find the right dose. Researchers will also look for early signs that the drug may he…

This study tests a new drug called BMS-986340, alone or with other cancer treatments, in people with advanced solid tumors like lung, colon, or breast cancer. The goal is to find safe doses and check for side effects. About 1,100 adults with tumors that have worsened after standa…

This study looks at whether the diabetes drug pioglitazone can improve how the liver processes energy in people with nonalcoholic fatty liver disease (NAFLD) and type 2 diabetes. Researchers will measure liver mitochondrial function using special tracers before and after 16 weeks…

This study tests whether adding the drug dinutuximab to standard chemotherapy and other treatments helps children with high-risk neuroblastoma live longer without their cancer returning. About 478 children and young adults up to age 30 will be randomly assigned to receive either …

This Phase III trial tests whether giving radiation in three smaller doses (FSRS) works better than a single large dose (SRS) for cancer that has spread to the brain. About 269 adults with lung, breast, melanoma, kidney, or gastrointestinal cancers will be randomly assigned to on…

This study tests whether exercise using electrical stimulation can improve muscle strength and endurance in people with lower limb paralysis from conditions like spinal cord injury, stroke, or multiple sclerosis. Participants will use devices that send electrical signals to their…

This early-phase trial is testing a new drug, FOG-001, in about 595 people with advanced or metastatic solid tumors, including colorectal, prostate, and liver cancers. The drug is given alone or with other cancer treatments to see if it is safe and shrinks tumors. The study is cu…

This study tests whether adding a new drug (RO7268489) to the standard MS treatment ocrelizumab can slow disability progression in adults with progressive multiple sclerosis. About 360 participants will receive either the new drug or a placebo, and researchers will track walking …

This study tests a drug called AGA2115 in 80 adults with osteogenesis imperfecta (brittle bone disease) types I, III, or IV. Participants receive either the drug or a placebo as a shot under the skin. The main goal is to see if the drug increases bone density in the spine after 1…

This trial asks whether giving children a low-dose ketamine infusion after heart surgery can protect their brains from damage. Children aged 1 to 60 months undergoing surgery for certain congenital heart defects will receive either ketamine at one of two doses or no ketamine. Res…

This Phase 3 trial tests whether privosegtor, a neuroprotective drug, can improve vision recovery in people having their first episode of optic neuritis. About 210 adults aged 18 to 50 will receive either privosegtor or a placebo, both alongside standard steroid treatment. The ma…

This study tests whether adding the drug pembrolizumab to a treatment that uses a patient's own immune cells (called TIL therapy) can shrink tumors in people with advanced melanoma. About 53 adults aged 18 to 72 who have already tried at least one other treatment will take part. …

This early-phase trial is testing an experimental oral drug called GIM-531, alone or with an immunotherapy (anti-PD-1), in 117 adults with advanced solid tumors that have stopped responding to standard treatments. The goal is to see if the drug is safe, tolerable, and can help th…

This early-phase trial is testing a new treatment called KITE-363 for people with certain autoimmune nerve diseases that have not responded to standard therapies. KITE-363 is a type of CAR T-cell therapy that uses a patient's own immune cells, modified to target and attack faulty…

This study tests whether adding an antibody drug called hu14.18K322A to standard chemotherapy helps children with high-risk neuroblastoma, a rare but aggressive childhood cancer. About 144 children and teens aged 18 months to 18 years will receive the combination over up to 12 cy…

This Phase 3 trial tests whether a simple injection of frexalimab every 4 weeks works as well as the standard IV infusion for adults with relapsing or non-active progressive multiple sclerosis. About 160 participants will be randomly assigned to one of the two methods. The study …

This early-stage trial tests two experimental pills (PF-07799544 and PF-07799933) taken twice daily at home by adults with advanced solid tumors that have a specific gene change called BRAF V600. The study aims to find safe doses and see if the drugs can shrink tumors. About 124 …

This study compares two standard procedures—minimally invasive TAVR and open-heart surgery (SAVR)—for replacing a diseased aortic valve in people with a bicuspid aortic valve. About 1,200 participants aged 50 and older with severe aortic stenosis will be randomly assigned to one …

This study follows 550 patients whose first TAVI heart valve has failed. Researchers will compare a second TAVI procedure (redo TAVI) with open-heart surgery or medication alone. The goal is to see which approach works best and why, helping doctors make better decisions for futur…

This study looks at whether people with multiple sclerosis can safely switch from their current anti-CD20 therapy to a new under-the-skin version of ocrelizumab. About 100 participants will be followed for up to 48 weeks to see if brain lesions stay stable and if they are satisfi…

This early-stage trial tests a new type of cell therapy for children whose embryonal tumors (rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, or Wilms tumor) have returned or not responded to standard treatments. The therapy uses the patient's own immune cells, engineered to bette…

This early-phase trial tests the safety of CRD3874-SI, a drug that activates the STING pathway to boost the immune system against cancer. About 81 adults with advanced solid tumors (sarcoma, Merkel cell carcinoma, and others) will receive weekly IV infusions at increasing doses. …

This trial investigates whether giving standard combination immunotherapy (ipilimumab and nivolumab) in the early morning (before 10 AM) versus the afternoon (after 1 PM) improves tumor response in people with advanced melanoma. About 170 participants with metastatic or inoperabl…

This phase II trial is testing whether the combination of cabozantinib (a targeted therapy) and nivolumab (an immunotherapy) can shrink or stabilize advanced melanoma and head and neck cancers that have stopped responding to prior immunotherapy. Researchers are also checking if t…

This study tests a new treatment for adults with certain cancers (pancreatic, kidney, breast, melanoma, or ovarian) that have a protein called CD70. Doctors take a sample of your blood, modify your white blood cells in a lab to recognize and attack CD70, and then give them back t…

This Phase 3 trial tests an oral drug called orelabrutinib against a placebo in about 990 people with non-active secondary progressive multiple sclerosis (MS). The goal is to see if the drug can delay worsening disability over 24 to 60 months. Participants are randomly assigned t…

This trial investigates different types of steroid injections for back, neck, and related pain to see which are most effective and safe. It compares various steroids, doses, and injection sites, including epidural and facet joint injections. The study measures pain relief and fun…

This study tests whether a low dose of glucagon, given before exercise, can prevent blood sugar drops in adults with type 1 diabetes who use an automated insulin pump. Eighteen participants will exercise after a meal, comparing glucagon to a standard glucose drink. The goal is to…

This program provides continued access to lucerastat for people with Fabry disease who were previously enrolled in a clinical trial of this drug. The goal is to allow these patients to keep receiving the treatment after the original study was stopped. It focuses on maintaining tr…

This early-stage study is testing a new drug called RO7121932 in 129 people with multiple sclerosis (MS). The goal is to check if the drug is safe and how the body handles it when given as a single or multiple doses, either through a vein or under the skin. Participants are not o…

This study tests a new drug called AZD9793 in people with advanced liver cancer that has spread. The drug is an antibody designed to help the body's immune cells find and attack cancer cells. The main goals are to check if the drug is safe and if it can shrink tumors. About 304 a…

This phase II trial is testing whether adding the drug naxitamab (Danyelza) to standard chemotherapy improves outcomes for children newly diagnosed with high-risk neuroblastoma. About 93 participants will receive naxitamab during five cycles of induction chemo. The goal is to see…

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 trial asks whether clamping the umbilical cord at 120 seconds after birth is better than clamping at 30 seconds for babies born with congenital heart disease. About 500 infants with a prenatal heart defect diagnosis will be randomly assigned to one of the two timings. Resear…

This early-phase study tests a new treatment called MQ710, a modified virus injected directly into tumors, alone or with the immunotherapy drug pembrolizumab. It is for people with advanced solid tumors (like skin cancer or head and neck cancer) that have not responded to standar…

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 trial tests whether a six-month program of supervised resistance exercise and personalized nutritional support can help adults with stage II to IV melanoma preserve muscle mass and quality while starting immunotherapy. Participants are randomly assigned to either the exercis…

This early-stage study tests a new drug called KUP-101A in 21 people with advanced skin cancers (like melanoma or Merkel cell carcinoma) that have not responded to other treatments. The main goals are to find the safest dose and check for side effects. Researchers will also measu…

This study tests a new treatment called DOC1021 for people with advanced melanoma that has not responded to standard therapy. DOC1021 is a personalized vaccine made from a patient's own blood and tumor cells, designed to teach the immune system to attack the cancer. The trial wil…

This study follows 255 patients of any age (weighing over 3 kg) who receive a device called the Occlutech PDA Occluder to close a common heart defect called patent ductus arteriosus (PDA). The device is inserted through a blood vessel, avoiding open-heart surgery. Researchers wil…

This Phase 2 study tests an oral drug called RLY-2608 in adults and children with overgrowth conditions (like CLOVES or Klippel-Trenaunay syndrome) caused by PIK3CA gene mutations. The drug is designed to block the faulty protein driving abnormal tissue growth. The trial will enr…

This trial tests an experimental drug called STAR0602, which is designed to help the immune system attack cancer cells. It is being studied in people with advanced solid tumors that have not responded to standard treatments. The drug is given alone or combined with chemotherapy, …

This phase 3 trial compares a once-weekly growth hormone injection (lonapegsomatropin) to a daily one (somatropin) in 186 prepubertal children with growth failure due to Turner syndrome, SHOX deficiency, being small for gestational age, or idiopathic short stature. The goal is to…

This trial is testing a new heart valve called SAPIEN X4/X4S in over 1,200 people with severe aortic stenosis, a condition where the heart's aortic valve narrows. The valve is inserted through a catheter, avoiding open-heart surgery. Researchers are measuring how many patients di…

This early-stage study tests a new drug called JL19001 in people with advanced solid tumors or a type of lymphoma that has come back or not responded to treatment. The main goal is to find a safe dose and check for side effects. About 14 participants will receive the drug as a sh…

This trial tests whether combining bexmarilimab with nivolumab can help people whose cancers no longer respond to immunotherapy. It focuses on two cancers: non-small cell lung cancer and melanoma. The study first finds the safest dose of bexmarilimab to give with a fixed dose of …

This study tests whether a bivalent vaccine, taken with a sugar called beta-glucan and a protein (GM-CSF), can help prevent high-risk neuroblastoma from returning in children who are currently in remission. The treatment works by training the immune system to recognize and attack…

This study tests whether oral antibiotics (linezolid or amoxicillin) can effectively treat newborns at risk of congenital syphilis, compared to a standard penicillin shot. The goal is to find a safe, outpatient-friendly option that avoids long hospital stays. The trial involves 9…

This study looks at whether the experimental drug remibrutinib can reduce inflammation in the brain and spinal cord of people with relapsing multiple sclerosis (MS). Participants, aged 40 to 70, will have PET scans, MRIs, and blood and spinal fluid samples taken before and six mo…

This early-phase trial tests MGC026, a biologic drug given by IV, in 250 adults with advanced solid tumors that have not responded to standard treatments. The study has two parts: first, finding a safe dose, then expanding to see if the drug shrinks tumors. Participants receive u…

This trial tests whether methylphenidate, a standard ADHD medication, can reduce attention and hyperactivity problems in children and adolescents with KBG syndrome, a rare genetic condition often accompanied by ADHD-like symptoms. Participants receive alternating blocks of the dr…

This study tests a new treatment called afamitresgene autoleucel in children and young adults aged 2 to 21 with advanced cancers that have a specific marker (MAGE-A4). The treatment uses the patient's own immune cells, modified to attack the cancer. The main goals are to check sa…

This study tests an oral drug called fenebrutinib in 12 children and adolescents aged 10 to 17 with relapsing multiple sclerosis. The goal is to see how the drug moves through the body, how safe it is, and whether it reduces new brain lesions seen on MRI. Participants can choose …

This phase 3 trial is testing whether a daily pill called remibrutinib works as well as the standard infusion or injection ocrelizumab for people with relapsing multiple sclerosis. About 360 adults aged 40–70 who are stable on ocrelizumab will either switch to remibrutinib or sta…

This study tests a special heart valve that can be adjusted to fit children as they grow. It involves 36 kids aged 1.5 to 16 years with congenital heart disease who need a new pulmonary valve. The valve is placed during surgery and can later be expanded without another operation.

This early-stage trial tests an oral drug called SVG103 (Paxalisib) in 15 adults with rare brain conditions (FCD-II, TSC, or HME) that cause seizures. The main goal is to check safety and side effects, while also seeing if it reduces seizure frequency. Participants take the drug …

This study is testing a device called the WavelinQ EndoAVF System that creates a connection between an artery and a vein (AV fistula) in people who need dialysis. The goal is to make it easier to start and continue dialysis with fewer complications. About 280 adults with end-stag…

This phase 2 trial is testing whether adding hydroxychloroquine—a drug commonly used for malaria—to a MEK inhibitor (tunlametinib) and an immunotherapy (pucotenlimab) can improve outcomes for people with advanced NRAS-mutant melanoma. The study aims to see if this combination can…

This early-stage trial tests two experimental mRNA therapies (mRNA-4106 and mRNA-4200) in 42 people with advanced solid tumors like melanoma, lung, or liver cancer. Some participants receive the mRNA therapy alone, others get it combined with the immunotherapy drug pembrolizumab.…

This phase 2 trial is testing an experimental drug called patritumab deruxtecan (HER3-DXd) in 740 people with advanced solid tumors, including melanoma, lung, breast, and other cancers. The drug is given by IV every three weeks. The main goal is to see if the drug shrinks tumors.…

This study looks at whether giving radiation to the lymph nodes after surgery can lower the chance of melanoma returning in people who are already receiving immunotherapy. About 168 adults with high-risk melanoma that has spread to a sentinel lymph node will take part. The goal i…

This study looks at whether people with early relapsing multiple sclerosis (MS) can stop taking ocrelizumab (Ocrevus) after two years without their disease coming back. All 123 participants will receive ocrelizumab for the first two years. Then, some will switch to a placebo whil…

This early-phase trial tests a personalized RNA nanoparticle vaccine made from a patient's own tumor. The goal is to help anti-PD-1 immunotherapy work better in people with advanced melanoma or soft tissue sarcoma whose cancer has progressed on standard treatment. Up to 18 partic…

This study tests a compact heart-lung machine called Cardiohelp in 50 children with severe heart failure. The device supports the heart for up to 30 days while kids recover or await a transplant. The trial also compares two blood thinners to see which causes fewer bleeding or clo…

This study tests a drug called ifinatamab deruxtecan (I-DXd) in children with solid tumors that have returned or not responded to treatment. The drug is an antibody-drug conjugate that targets cancer cells directly. The trial aims to check safety and see if tumors shrink in about…

This study tests a drug called PRX-102 (pegunigalsidase alfa) in 22 children and teens aged 2 to 18 with Fabry disease, a rare genetic disorder that causes pain and organ damage. The drug is an enzyme replacement therapy given by infusion every two weeks. Researchers will monitor…

This trial tests whether pegtibatinase, a lab-made version of the CBS enzyme, can safely lower homocysteine levels in people with classical homocystinuria (HCU), a rare genetic condition that causes harmful buildup of homocysteine and methionine. The study includes children and a…

This early-stage trial is testing an experimental drug called MBS8(1V270), given alone or with the immunotherapy pembrolizumab, in people with advanced solid tumors that have not responded to standard treatments. The main goals are to check safety and find the right dose. About 1…

This study investigates whether aggressive lipid-lowering therapy can reduce plaque buildup in the heart arteries of melanoma patients starting immune checkpoint inhibitors (ICIs). Participants with plaque detected on a heart CT scan are randomly assigned to either usual care or …

This trial compares standard cleft palate surgery alone versus surgery augmented with platelet-rich fibrin (PRF) or gentamicin-loaded PRF in 90 children and adolescents with cleft palates. The goal is to see if these biological adjuncts improve wound healing, reduce wound breakdo…

This study tests whether taking a daily probiotic supplement for six months can improve immune function in people with relapsing-remitting multiple sclerosis (MS). Eighty adults aged 18-55 will be randomly assigned to receive either the probiotic or a placebo, alongside their usu…

This phase 3 trial tests whether the oral drug orelabrutinib can slow disability progression in people with primary progressive multiple sclerosis (PPMS). About 705 adults aged 18–60 with PPMS will receive either orelabrutinib or a placebo for up to 5 years. The study measures ti…

This early-stage trial tests a new drug called GAIA-102 in children whose neuroblastoma or other solid tumors have not responded to standard treatments. The study has four groups, testing GAIA-102 alone or combined with other cancer drugs. The main goal is to check safety and fin…

This trial tests a new model of care called PEN-Plus, which brings specialized treatment for severe chronic diseases like diabetes, heart conditions, and sickle cell disease to first-level hospitals in Zambia. The goal is to train local health workers and set up clinics to manage…

This study tests whether adding an experimental immune-boosting drug (N-803) to standard chemoimmunotherapy can help children and young adults whose neuroblastoma has returned or not responded to treatment. About 54 participants will receive the combination to see if it is safe a…

This Phase 3 trial tests pegtibatinase, an enzyme given as a shot, in 70 people aged 12–65 with classical homocystinuria. The goal is to see if it safely lowers high homocysteine levels when added to standard care. Participants receive either the drug or a placebo for 24 weeks.

This study tests a new drug called PTH-IA for Jansen's Metaphyseal Chondrodysplasia (JMC), a very rare genetic disorder that causes severe bone problems, short stature, and kidney issues. About 12 adults and children will receive the drug to see if it is safe and works. The goal …

This trial is testing an investigational drug called ARV-6723, given as a pill, in people with advanced solid tumors. The drug is designed to help the immune system recognize and attack cancer cells. The study will test ARV-6723 alone and in combination with pembrolizumab, an imm…

This study compares two types of fat infusions (mixed oil vs. soybean oil) given to extremely preterm infants to improve nutrition and reduce the risk of bronchopulmonary dysplasia (a chronic lung disease) and neurodevelopmental impairment. About 230 babies born before 28 weeks o…

This study tests a new drug, T3011, in 42 people with advanced melanoma that has spread. The main goals are to check safety and see if tumors shrink. Participants must have measurable tumors and be in good overall health.

This study tests two different doses of an intravenous immune globulin (IGIV 10%) as maintenance therapy for adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder causing weakness and numbness. About 161 participants will receive either a higher o…

This study offers access to a radioactive therapy called 131I-MIBG for people with neuroblastoma or pheochromocytoma that has returned or stopped responding to standard treatments. The therapy is designed to seek out and deliver radiation directly to tumor cells. Researchers are …

This Phase 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either ma…

This trial is testing an experimental therapy called STX-003, which uses mRNA to produce a protein that may help the immune system fight cancer. It is given intravenously, either alone or with the immunotherapy drug pembrolizumab (Keytruda). The study involves adults with advance…

This study tests a drug called ifinatamab deruxtecan (I-DXd) in people with 13 types of advanced solid tumors that have come back or spread. The trial aims to see how well the drug shrinks tumors and what side effects it causes. About 520 participants will receive the drug intrav…

This trial tests an experimental drug called KLS-1, a form of zinc aspartate enriched with a specific zinc isotope, in adults with certain solid tumors (like melanoma, prostate, or pancreatic cancer) or chronic lymphocytic leukemia (CLL). The goal is to find the safest and most e…

This early study tests a new treatment called S103 (BCMA-CAR T cells) in 9 adults with progressive or hard-to-treat multiple sclerosis (MS). The main goal is to see if the treatment is safe and to find the best dose. Researchers will also look for early signs that it might help c…

This Phase 2 trial tests a new treatment called GC101 TIL for people with advanced melanoma that has not responded to standard therapies. The treatment uses immune cells taken from the patient's own tumor, grown in a lab, and then given back to fight the cancer. About 98 particip…

This study will track 100 people with severe aortic stenosis who receive the Hydra THV heart valve through a less invasive procedure (TAVR). The goal is to see how well the valve works and how safe it is in everyday practice. Participants will be followed for short- and mid-term …

This study tests two experimental drugs, BNT326 and BNT327, in people with advanced non-small cell lung cancer that has spread or come back. The trial has three parts to find the best dose and check safety, and will compare the combination to standard treatments like pembrolizuma…

This phase 3 trial tests two different doses of Panzyga, an intravenous immunoglobulin therapy, in children aged 2 to 17 with CIDP, a rare nerve condition causing weakness and disability. The study aims to see which dose better controls symptoms and prevents relapses, using a dis…

This Phase 1 trial tests IDOV-Immune, a genetically engineered virus designed to infect and destroy cancer cells while boosting the immune system. It is for adults with advanced solid tumors (like colorectal, pancreatic, or lung cancer) that have not responded to standard treatme…

This study tests a small device implanted on the vagus nerve in the neck to see if it can help repair myelin (the protective coating around nerves) in people with relapsing-remitting multiple sclerosis. About 60 adults aged 22–50 will take part. Two-thirds will receive active sti…

This trial tests whether the direction of the needle bevel (up or down) during fistula puncture affects the risk of complications in people on hemodialysis. About 240 adults with end-stage kidney disease who are starting dialysis with a new arteriovenous fistula will be randomly …

This early-stage trial is testing whether a combination of two natural substances, deoxycytidine and deoxythymidine, is safe for people with telomere biology disorders. These rare genetic conditions cause premature aging, bone marrow failure, and lung scarring. Up to 36 participa…

This early-phase study tests an experimental cell therapy called YTB323 in about 28 people with relapsing multiple sclerosis whose disease is still active despite taking strong medications. The goal is to see if YTB323 is safe and can control the disease. Participants receive a s…

This early-stage study tests whether giving children with high-risk solid tumors a special type of immune cell (called TAA-T) is safe. The cells are designed to recognize and attack proteins found on tumor cells. Up to 36 children whose cancer has not responded to standard treatm…

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 trial tests whether an eight-week digital cardiac rehabilitation program using the Peloton app can improve physical activity and recovery in people who have had a transcatheter aortic valve replacement (TAVR). Participants are randomly assigned to either the digital rehab pr…

This study is testing whether giving lower, less frequent doses of the cancer drug atezolizumab can still keep enough drug in the blood to fight cancer. About 30 adults with certain advanced cancers (like lung cancer or melanoma) will start with standard doses, then switch to a l…

This early-stage trial tests an oral drug called ALE1 for hypophosphatasia (HPP), a rare genetic bone disease. The study will first check safety and how the drug moves through the body in healthy volunteers, then in adult HPP patients. It is a small, placebo-controlled study to f…

This pilot study explores whether people with melanoma or non-small cell lung cancer that has spread to the brain can safely skip stereotactic radiosurgery (focused radiation) after having a brain tumor surgically removed. Instead of radiation, participants receive systemic thera…

This study tests a drug called mitapivat in children aged 1 to 17 with alpha- or beta-thalassemia who need regular blood transfusions. The goal is to see if mitapivat can reduce the number of transfusions needed compared to a placebo. The study involves 54 participants and lasts …

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 will follow 30 people with Alagille syndrome who are taking odevixibat in their daily lives. The goal is to see how safe the drug is over the long term by tracking any side effects. It is not testing if the drug works, but rather monitoring its real-world safety.

This study is for children and young adults (up to age 30) with a rare type of soft tissue cancer called non-rhabdomyosarcoma. It tests whether adding the drugs pazopanib and selinexor to standard chemotherapy can improve outcomes. Participants receive treatment tailored to their…

This early-phase study tests a new drug (PLN-101095) alone or with pembrolizumab in adults whose advanced solid tumors have stopped responding to standard immunotherapy. About 124 participants will receive the treatment to check safety and early signs of tumor shrinkage. The goal…

This phase 2 study tests belzutifan, a daily pill that blocks a protein called HIF-2α, in people with advanced rare cancers like pheochromocytoma/paraganglioma, pancreatic neuroendocrine tumors, VHL disease-associated tumors, and certain other solid tumors. The goal is to see if …

This trial tests whether a six-month program of high-intensity exercise delivered through an immersive virtual reality headset can improve mobility, strength, and thinking skills in people with multiple sclerosis (MS). Participants will be randomly assigned to either the VR exerc…

This trial is testing whether vitamin D supplements can improve bone density, body composition, strength, walking speed, and physical performance in people hospitalized with neurological disorders like stroke, spinal cord injury, or traumatic brain injury. Participants will recei…

This study tests an implanted electrical stimulation device designed to improve movement, standing, and walking in people with paralysis from spinal cord injury, stroke, multiple sclerosis, or traumatic brain injury. The device sends electrical signals to muscles in the trunk and…

This early-phase trial is testing whether adding sitagliptin, a diabetes drug, to the immunotherapy pembrolizumab is safe and can help people with advanced kidney cancer or melanoma whose cancer has stopped responding to standard treatment. The study will first find the safest do…

This phase II trial is testing an experimental drug called IBI363 in people with advanced melanoma that has worsened after at least one prior treatment. The study aims to see whether IBI363 is safe and can shrink tumors or slow their growth. About 180 participants will receive IB…

This study tests whether a monthly injection of leuprorelin, a drug that blocks puberty hormones, can safely slow or stop early puberty in children diagnosed with central precocious puberty. About 43 children—girls under 9 and boys under 10—will receive the injection every 4 week…

This trial compares two single antiplatelet therapies—ticagrelor versus aspirin—in people who have just undergone transcatheter aortic valve implantation (TAVI) for severe aortic stenosis. The goal is to see which drug better prevents serious complications like stroke, heart atta…

This trial investigates whether adding core stability training and intrinsic foot muscle exercises to conventional physical therapy can improve balance, joint position sense, and foot function in people with chronic ankle instability. Eighty adults aged 18 to 44 with a history of…

This study explores whether being physically active can help melanoma patients avoid or lessen the immune-related side effects of immunotherapy drugs like pembrolizumab and nivolumab. Researchers will track the activity levels of 160 adults starting immunotherapy and monitor them…

This trial tests a robotic knee exoskeleton combined with visual biofeedback (like a video game) to help children aged 4–21 who have genu recurvatum (knee hyperextension) or crouch gait (bent-knee walking). Over five rehab sessions, the device assists knee movement while the chil…

This trial is testing whether combining two drugs—selumetinib and APG-115—can help people with neurofibromatosis type 1 (NF1) who have nerve sheath tumors that are either pre-cancerous or already cancerous. The study aims to find a safe dose and see if the combination can shrink …

This trial investigates whether adding dried chicory root fiber to standard immunotherapy helps people with advanced melanoma. The fiber is meant to increase beneficial gut bacteria, which may in turn improve immune cell activity and the body's response to treatment. About 70 par…

This trial compares two minimally invasive surgical techniques for repairing ventral hernias (bulges in the abdominal wall). One technique, called eTEP, works entirely outside the abdominal cavity, while the other, IPOM, places a mesh inside the cavity. Researchers want to know w…

This trial compares two surgical techniques for repairing medium-to-large ventral hernias (bulges in the abdominal wall). One group receives robotically-assisted, minimally-invasive surgery; the other receives traditional open surgery. Researchers are measuring how long patients …

This phase 3 study tests a new weekly growth hormone injection (inpegsomatropin) against an existing one (givopegsomatropin) in 84 girls with Turner syndrome who have short stature. The goal is to see which works better for increasing growth over 52 weeks. Participants are prepub…

This study looks at postmenopausal women with severe osteoporosis who have finished a course of either romosozumab or teriparatide. Researchers want to see which follow-up drug—zoledronate, denosumab, teriparatide, romosozumab, or abaloparatide—best maintains or improves bone den…

This trial tests a new 3D printed dental appliance designed to correct an underdeveloped upper jaw in children aged 8-13 with class III malocclusion (where the lower jaw sticks out). The device, called a reverse twin block, is worn in the mouth to gently guide jaw growth over six…

This trial investigates whether giving oral antibiotics before elective colorectal surgery reduces complications like infections and bowel leaks in children. About 74 children scheduled for procedures such as colostomy closure or pull-through for conditions like Hirschsprung dise…

This study tests whether injecting sodium thiosulfate directly into abnormal calcium deposits can shrink them and reduce pain. It includes people with three rare conditions: systemic sclerosis, dermatomyositis, and a genetic disorder called iPPSD2. Participants receive up to 11 i…

This study tests whether a handheld device that strengthens the muscles used for breathing and swallowing can improve speech in children aged 5 to 17 with velopharyngeal dysfunction, a condition where the soft palate does not close properly, causing nasal-sounding speech. Partici…

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 phase 3 trial tests whether a combination of two experimental drugs (futibatinib and zimberelimab) plus standard chemotherapy can help people with advanced biliary tract cancer live longer compared to current standard immunotherapy combinations. The study enrolls about 784 a…

This study investigates penile transplantation as a way to replace a severely damaged or missing penis with a donor organ, aiming to restore appearance, sensation, and sexual function. It includes men aged 18-69 who have lost their penis due to trauma, surgery, or birth defects. …

This phase 1 trial tests whether giving the immunotherapy naxitamab more slowly, alongside standard chemotherapies irinotecan and temozolomide plus sargramostim, can reduce side effects in children with relapsed or refractory neuroblastoma. The study enrolls about 18 patients who…

This phase 1 trial tests a drug called 177Lu-DTPA-omburtamab, which delivers radiation directly to tumor cells in children and teens with recurrent or refractory medulloblastoma or other central nervous system (CNS) tumors that have spread. The drug targets a protein called B7-H3…

This trial tests a new device called the P3 Occluder System designed to close a patent foramen ovale (PFO) — a small opening between the upper chambers of the heart. The study includes adults aged 18 to 65 who have had a stroke possibly linked to a PFO. Participants receive the i…

This study is testing the safety and performance of the Navitor TAVI system, a device used to replace a narrowed heart valve (aortic stenosis) without open-heart surgery. About 1,000 participants who are eligible for the procedure will receive the Navitor valve and be followed fo…

This study tests a surgically implanted device that uses mild electrical pulses to stimulate muscles in the legs and trunk. The goal is to help people with paralysis from spinal cord injury, stroke, or other conditions exercise, stand, step, and improve balance. Ten participants …

This study follows 100 adults with hereditary hemorrhagic telangiectasia (HHT) who start taking blood thinners (anticoagulants or antiplatelets). Researchers compare the three months before treatment to the first three months on treatment, tracking bleeding events, iron levels, a…

This study tests whether giving choline supplements to pregnant women who drink heavily can reduce the negative effects of alcohol on their baby's growth and brain development. Choline is an essential nutrient that supports cell membranes and brain function. The trial compares da…

This study investigates whether cleaning the mouths of very low birth weight premature infants with human milk several times a day can reduce harmful bacteria and lower the risk of lung complications like pneumonia and bronchopulmonary dysplasia. Researchers will follow 168 babie…

This early-stage study tests a new drug called N-3C01, given alone or with an immunotherapy, in people with advanced solid tumors that have spread or cannot be removed. The main goals are to check safety, find the right dose, and see if the combination shrinks tumors. About 114 a…

This trial tests a 12-week neurocardiac rehabilitation program for children, adolescents, and young adults with moderate to complex congenital heart disease. The program combines home-based physical exercise, telehealth psychoeducation for parents and children, and computerized c…

This study tests whether giving a tetanus-diphtheria or polio vaccine during standard immunotherapy can help the immune system better attack melanoma tumors. About 25 adults with advanced melanoma will receive one of these vaccines after four cycles of checkpoint inhibitor therap…

This trial tests a single injection of MELPIDA, a gene therapy that delivers a working copy of the AP4M1 gene directly into the spinal fluid of children with SPG50. SPG50 is a rare genetic condition that causes muscle stiffness, intellectual disability, and developmental delays. …

This study looks at Portuguese adults with relapsing multiple sclerosis who are taking ofatumumab (Kesimpta). It compares those who started the drug within three years of diagnosis to those who started later. The goal is to see if earlier treatment leads to less disease activity,…

This trial investigates a Functional Balance Intervention (FBI) — a structured exercise program — for people aged 55 to 80 with multiple sclerosis. The program includes agility, strength, balance, and dual-task exercises done twice a week for four months, either in a lab or at ho…

This phase 1 trial tests a new drug called AWT020, given alone or with other cancer treatments, in people with advanced solid tumors such as non-small cell lung cancer, colorectal cancer, kidney cancer, and melanoma. The main goals are to check the drug's safety, find the best do…

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 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 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 study is testing whether stem cells taken from a person's own bone marrow and then given back through the veins and nose can improve brain and nerve function. It includes 500 people with various conditions like stroke, Parkinson's, Alzheimer's, and nerve damage. The goal is …

This trial investigates whether combining two drugs, camrelizumab and famitinib, can help people with rare head and neck cancers that have spread or cannot be removed by surgery. The study includes four types of these cancers and aims to see how safe and effective the treatment i…

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 early-stage trial is testing whether specially engineered immune cells (called CAR-T and CTL cells) and a dendritic cell vaccine can safely treat people with neurofibromatosis or schwannomatosis, conditions that cause nerve tumors. The study will enroll 100 participants aged…

This study tests a device called Vessel-X® that uses a special container to deliver bone cement into fractured vertebrae caused by osteoporosis. The goal is to reduce cement leakage and improve safety compared to standard procedures. About 146 adults aged 40 to 95 with spinal fra…

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 phase 1 trial tests an experimental drug, DXC006, in people with advanced solid tumors (like small cell lung cancer and neuroblastoma) or blood cancers (like multiple myeloma) who have not responded to standard treatments. DXC006 is an antibody-drug conjugate designed to del…

This trial investigates whether giving very preterm infants (born at 24-29 weeks) a daily supplement of two fatty acids—ARA and DHA—starting within three days of birth can reduce their risk of bronchopulmonary dysplasia (BPD), a chronic lung disease common in premature babies. Ha…

This study tests whether custom 3D-printed titanium implants can safely and effectively rebuild facial bones in 30 adults with defects from injury, cancer surgery, or birth conditions. Each implant is designed from CT scans to match the patient's unique anatomy. Researchers will …

This study is following 35 adults who have received a Tipmed revision hip prosthesis during routine care. Researchers will track how well the hip works and any complications over 24 months. The goal is to see if the device performs safely and effectively in real-world use.

This study tests a daily pill called vibegron to see if it safely improves bladder control in children aged 2 to 18 with neurogenic detrusor overactivity (NDO), a condition where the bladder contracts too often due to nerve damage. Participants must already use a catheter to empt…

This trial tests whether a bioactive glass material can effectively fill the mastoid bone after surgery for cholesteatoma, a non-cancerous but destructive ear growth. Adults having their first cholesteatoma operation will receive the glass filler to stabilize the repair and preve…

This study tests a new device that delivers a special type of radiation directly into skin tumors. The goal is to see if it can shrink or control the cancer. The study is for adults with certain types of skin cancer, including squamous cell carcinoma and basal cell carcinoma. Par…

This study tests two types of branch endografts (Zenith t-Branch and Terumo Arch Branch) to treat aortic aneurysms in the chest and abdomen. The devices are placed inside the aorta to reinforce the weakened vessel wall and keep blood flowing to vital organs. Researchers will trac…

This study tests a new device called CHORDS that catches loose debris during a heart valve replacement procedure (TAVR). The debris can travel to the brain and cause a stroke. The trial will compare CHORDS to an existing device in 240 people with severe aortic stenosis. Participa…

This study follows 140 patients in Indonesia who received the HeartR PDA Occluder, a device that closes a common heart defect called Patent Ductus Arteriosus (PDA). The goal is to see if the device works well and is safe over 12 to 24 months. Patients must be at least 6 months ol…

This early-phase trial is testing a new treatment called MEM-288, a virus designed to infect and kill cancer cells while also boosting the immune system. The study involves 40 adults with advanced solid tumors, including lung, skin, and breast cancers, who have not responded to s…

This Phase 2 trial tests whether adding thymosin alpha 1 to the immunotherapy drug toripalimab can help elderly patients (60+) with advanced melanoma. The study will enroll 55 participants and measure how many patients' tumors shrink or disappear. Researchers also want to see if …

This study tests a new treatment called IOV-4001 for people with advanced melanoma or non-small-cell lung cancer. The treatment uses a patient's own tumor-fighting immune cells, which are collected, genetically modified to remove a 'brake' called PD-1, and then infused back after…

This study tests whether giving oxybutynin syrup early (starting at 3-6 months old) can improve bladder function in boys born with posterior urethral valves, a condition that blocks urine flow. Fifty boys who had valve surgery will be randomly assigned to receive either oxybutyni…

This phase 2 trial tests whether specially trained donor immune cells, called CIML-NK cells, can be safely given to 20 children and young adults (ages 1-39) with relapsed or refractory neuroblastoma. The cells are made from a half-matched donor and infused alongside standard dinu…

This study tests a new treatment made from donor immune cells (CAR-NK cells) that are designed to attack two targets on melanoma cells. It is for adults with advanced melanoma that has spread or cannot be removed and has not responded to standard therapies. The goal is to find a …

This study tests a lifestyle program designed to help teens and young adults (ages 15-25) with congenital heart disease become more physically active. Participants will receive a Fitbit, a personalized exercise plan, and education on healthy living. The goal is to see if the prog…

This study tests whether giving two steroids (hydrocortisone and fludrocortisone) to high-risk patients before heart surgery can lower the chance of serious complications like kidney injury and lung problems. The trial will include 196 adults with a EuroSCORE II above 4% who are …

This study tests whether a combination of umbilical cord stem cells and glutathione can safely improve autism symptoms in 100 people aged 3 and older. Participants receive either the stem cell product plus glutathione, or a placebo plus glutathione, with the option to switch grou…

This study is testing whether a common heart valve procedure called TAVI can be performed safely in hospitals that do not have on-site cardiac surgery backup. About 1,600 patients with severe aortic stenosis will be randomly assigned to get TAVI either at a center with or without…

This study is tracking 145 people who have a hole in their heart (atrial septal defect) and received the CeraFlex closure device. Researchers want to see how well the device works in real-world settings, focusing on safety and successful closure of the hole at 6 months. The devic…

This study is testing a treatment called electrosclerotherapy for people with low-flow venous malformations (abnormal blood vessels present from birth). The goal is to shrink the malformation by at least 30% and reduce pain. About 65 participants will receive the treatment and be…

This trial tests whether an AI tool called icobrain ms can help radiologists detect signs of multiple sclerosis (MS) activity on brain MRI scans. The study involves over 1,300 people with MS or early signs of it, who are already on treatment. The AI analyzes scans and creates str…

This trial is testing whether a new radioactive tracer, called 68Ga-SorB, can help doctors see certain solid tumors on PET/CT scans. The tracer targets sortilin, a protein that is often found on cancer cells. About 50 adults with melanoma, liver, lung, breast, pancreatic, or ovar…

This trial is developing an AI-powered device that listens to children's heart, breath, and bowel sounds to identify signs of common diseases like congenital heart disease, bronchopneumonia, and abdominal issues. It will involve 30,000 children aged 0-18, some with these conditio…

This trial evaluates a new continuous glucose monitoring (CGM) system in adults with type 1 diabetes. Participants will wear the sensor for 14 days while their blood sugar is measured against standard lab tests to see how accurate and safe the device is across different glucose l…

This trial is investigating whether a new radioactive tracer called 18F-grazy-02, used with PET/CT scans, can safely and effectively monitor how well immunotherapy is working in people with lymphoma or solid tumors. The tracer targets granzyme B, a protein released by immune cell…

This study investigates whether a blood test measuring clotting and clot-busting markers can predict complications in patients undergoing aortic valve replacement. Researchers will follow 750 adults with aortic stenosis who receive either surgical or catheter-based valve replacem…

This study tests whether a rapid finger-prick blood test for syphilis and HIV can be used in a prenatal clinic for pregnant people at risk. The test gives results in five minutes, potentially speeding up diagnosis and treatment. Participants will provide feedback on their experie…

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 tests a new radioactive tracer called 68Ga-GPC3 that targets a protein found on liver cancer cells. About 40 adults with suspected or confirmed hepatocellular carcinoma will receive both this new tracer and a standard tracer (18F-FDG) for PET/CT scans. Researchers will…

This study investigates whether using a lower dose of iodine contrast dye in CT scans can still produce high-quality images needed to plan a heart valve replacement procedure (TAVR). Normally, 100 ml of contrast is used, but reducing the dose may help avoid kidney injury and save…

This study is testing a new device that uses light and sound to detect melanoma cancer cells flowing in the blood, without needing a blood draw. The goal is to see if it can find these cells faster and more sensitively than current lab tests. About 75 people with melanoma will be…

This study tests whether a machine learning algorithm can better detect critical congenital heart disease (CCHD) in newborns by combining standard oxygen saturation measurements with perfusion index data. Researchers will enroll up to 320 infants, including those with suspected o…

This study tests whether a tablet-based eye-tracking app (eVOG) can detect subtle eye movement problems in people with brain MRI abnormalities (white matter lesions). The goal is to see if these eye movement patterns can help diagnose multiple sclerosis or related conditions. Res…

This study is testing a new blood test that looks for fetal cells in the mother's blood to detect genetic conditions like Down syndrome. The test will be compared to standard diagnostic methods such as amniocentesis or newborn testing. The study involves 1,000 pregnant individual…

This study tests whether artificial intelligence can analyze standard ultrasound images of the heart to measure calcium buildup on the aortic valve. If accurate, this could help doctors diagnose severe aortic stenosis without the extra cost and radiation of a CT scan. The study w…

This study tests whether an AI system (VeriOsteo OP) can help doctors find osteoporosis earlier in adults aged 40 to 80 who are at high risk. About 1,180 participants will be randomly assigned to get either AI-guided screening or usual care. The goal is to see if AI leads to bett…

This study is testing a new blood-based method to diagnose osteoporosis and predict fracture risk. Researchers will analyze the vitality and behavior of certain blood cells from 120 healthy, osteopenic, and osteoporotic adults aged 40 and older. The goal is to see if this approac…

This study tests a sun protection program for children playing baseball in rural Utah and West Virginia. Researchers will observe whether kids on teams using the program apply sunscreen, wear hats and long sleeves, and seek shade more often than those on teams that don't. The goa…

This trial tests whether adding nitric oxide gas to the heart-lung machine during surgery can prevent acute kidney injury in newborns with congenital heart defects. Up to 60% of these babies experience kidney damage after surgery, which can lead to serious complications. The stud…

This phase 2 trial tests whether the drug quisinostat can prevent uveal melanoma from spreading in high-risk patients. About 63 people who have completed initial treatment for their eye tumor will take quisinostat capsules three times a week. Researchers will track how long parti…

This study tests whether a 12-week online ukulele learning program, called Music-4-MS, can improve cognitive function in people with multiple sclerosis (MS). Half the participants will learn to play the ukulele, while the other half will simply listen to music they enjoy. The goa…

This study tests whether using shockwave therapy on the leg artery before a TAVR heart valve procedure can reduce bleeding and other complications. It involves 100 adults with severe aortic stenosis and heavily calcified arteries. The goal is to make the procedure safer by prepar…

This study tests a drug called fezolinetant to see if it can reduce hot flashes in men with prostate cancer who are on hormone therapy (androgen deprivation therapy). About 60 men will take either the drug or a placebo daily for 4 weeks. Researchers will track hot flash frequency…

This study tests whether mentally practicing walking while watching videos of people walking can improve gait in people with multiple sclerosis. Twenty participants will either watch walking videos and imagine themselves walking, or watch nature scenes. Researchers will measure m…

This study tests whether Acceptance and Commitment Therapy (ACT), delivered through a smartphone app, can help caregivers of children with RASopathies (like Neurofibromatosis type 1 and Noonan syndrome) cope with parenting stress. The trial is fully remote and involves 70 adult c…

This trial tests whether a mild electric current applied to a brain area linked to memory can improve verbal recall in people with relapsing-remitting multiple sclerosis. Participants will receive either active or sham stimulation during cognitive tasks, and their performance wil…

This study explores whether hypnosis can help people with stroke, traumatic brain injury, or multiple sclerosis improve their attention and reduce fatigue. About 48 participants will receive hypnosis sessions alongside standard care, while researchers monitor brain activity with …

This trial tests whether daily music training—either piano practice or listening to music—can improve behavior and thinking in children with fetal alcohol spectrum disorder (FASD). Each child will do both activities for 12 weeks each, in a random order, with tests before and afte…

This study looks at two ways of giving the pain medicine ketorolac to children after heart surgery: as a steady drip or as shots every few hours. The goal is to see if the steady drip reduces the need for stronger opioid pain medicines. About 166 children aged 3 months to 5 years…

This trial tests whether closed kinetic chain exercises or Thrower's Ten exercises better reduce pain, improve mobility, and boost performance in young male bowlers with shoulder instability. Participants will follow a six-week program. The goal is to find which approach offers b…

This trial asks whether adding gentle nerve mobilization to standard PNF exercises can improve facial symmetry and movement in people with Bell's palsy, a condition causing one-sided facial weakness. Adults aged 15 to 60 with moderate to severe Bell's palsy will be randomly assig…

This study tests whether applying Mastisol, a liquid adhesive, during clubfoot casting helps babies with clubfeet get better faster and with fewer problems. Researchers will compare outcomes like how many weeks of casting are needed and how often complications like sores or swell…

This study tests a methadone-based recovery plan to improve pain control and reduce opioid use in children after heart surgery. About 500 children aged 3 to 18 will be randomly assigned to either the new methadone plan or standard care. The goal is to see if the new approach lowe…

This study tests a 16-week exercise program designed specifically for Hispanic people with multiple sclerosis who have walking problems. About 66 participants will either join the exercise group or a waitlist. The goal is to see if the program improves walking ability and quality…

This study tests whether a single injection of RE104 can reduce symptoms of depression or mixed depression and anxiety in people with adjustment disorder caused by a serious medical illness like cancer, ALS, multiple sclerosis, Parkinson's disease, or idiopathic pulmonary fibrosi…

This study tests whether combining a memory retraining technique (KF-mSMT) with aerobic exercise and virtual reality can improve learning and memory in people with multiple sclerosis (MS). 78 participants with mild-to-moderate mobility disability will be enrolled. The approach fo…

This study tests whether a digital tool combining weekly symptom surveys and a Fitbit can help monitor patients during immunotherapy for advanced melanoma. Forty participants will be randomly assigned to use the tool or receive usual care. The goal is to see if the system is easy…

This pilot study tests whether a mild electrical current applied to the spinal cord (transcutaneous stimulation) can improve leg muscle strength, walking, and bladder function in 20 children with spina bifida (myelomeningocele). The children, aged 4-17, will receive stimulation w…

This trial tests whether a device called sacral neuromodulation (InterStim X) can improve bladder, bowel, and sexual function in people with multiple sclerosis who have not responded to standard treatments. Participants who show at least 50% improvement during a test period will …

This trial tests whether sertraline, a widely used antidepressant, can reduce anxiety in children and teens aged 8 to 17 with neurodevelopmental disorders such as autism, ADHD, Fragile X syndrome, and Tourette syndrome. Participants receive either sertraline or a placebo for seve…

This trial tests whether a 10-session group program called Social Management Training can improve mental health, executive function, and social skills in adults aged 16 to 69 who have sex chromosome aneuploidies (extra or missing sex chromosomes). Participants complete questionna…

This study investigates whether cannabidiol (CBD), a compound from cannabis, can improve sleep and reduce pain in people with multiple sclerosis (MS) who also have chronic pain. About 166 participants take CBD or a placebo for several weeks while their sleep is measured in a lab …

This study tests whether a device that gently stimulates the forehead during sleep can help children aged 8 to 12 who have ADHD linked to prenatal alcohol exposure. The device, already cleared for general pediatric ADHD, is used at home by parents each night for four weeks. Resea…

This study tests whether riluzole, a drug already used for ALS, can help cancer survivors who struggle with memory, focus, and mental clarity after treatment — a condition called cancer-related cognitive impairment. Researchers will measure changes in a brain protein called BDNF …

This study tests whether a 6-month personalized exercise program can improve physical and cognitive function in people with multiple sclerosis (MS) who primarily use a wheelchair. Participants will follow a low-impact, resistance-based routine designed to build muscle without inc…

This study tests whether a quadratus lumborum block—a type of nerve block—can better control pain and reduce opioid use in children aged 6 to 18 who are having palate repair surgery with bone taken from the hip. Half of the participants receive the nerve block, while the other ha…

This study tests whether a noninvasive brain stimulation technique called tDCS can reduce depression in people with multiple sclerosis. Participants use a headband-like device at home for 30 minutes a day over 30 days, while listening to mindfulness meditation. Half receive real …

This study tests the Point Mini, a mechanical finger prosthetic, in 14 children aged 3-17 with partial hand differences from amputation or birth. Each child uses their current device for one month, then the Point Mini for two months, to compare hand function, ease of use, and how…

This study compares two ways to block pain after hypospadias surgery in children aged 6 months to 7 years. One method uses a sacral erector spinae plane block, the other a caudal epidural block. Researchers will measure pain scores, how much extra pain medicine is needed, and any…

This study tests whether a personalized mix of endurance and strength exercises, done at home with video check-ins, can improve mobility and muscle strength in people with mitochondrial myopathy. Fifteen adults with confirmed genetic mutations will follow the program for up to 12…

This study compares two drug combinations (ketamine with propofol vs. dexmedetomidine with propofol) for sedating children aged 2-12 during a heart catheterization to close simple heart defects. The goal is to see which approach causes fewer breathing problems and provides smooth…

This early-phase trial tests a new nasal ointment called TOR-582, which contains sirolimus, in 27 adults with hereditary hemorrhagic telangiectasia (HHT) who have frequent nosebleeds. Participants apply the ointment inside their nostrils twice daily for 12 weeks, with different s…

This trial tests a wearable robot called ENMS-BSF that combines soft muscles, electrical stimulation, and balance feedback to correct foot drop and foot inversion after a stroke. Sixty people who had a stroke at least six months ago and have mild-to-moderate walking problems will…

This early-phase study tests a 4-week training program that uses mixed reality to guide precise movements, like stacking virtual objects. The goal is to see if this can improve walking speed, balance, and thinking in people with multiple sclerosis. Thirty adults with MS will part…

This study compares two surgical methods for treating gynecomastia (enlarged male breast tissue): a newer endoscopic technique using small incisions versus traditional open surgery. Researchers will follow 100 men aged 18 to 60 for up to 12 months after surgery to see which metho…

This study tests whether a special laser (MLS class IV) can reduce chronic pain and fatigue in people with hypermobile Ehlers-Danlos syndrome (hEDS), a condition that causes loose joints and widespread pain. Twenty-five adults will receive 10 laser sessions over 5 weeks, with pai…

This study tests whether cognitive behavioral therapy for insomnia (CBT-I) can improve sleep and reduce fatigue in people with multiple sclerosis. About 70 adults with MS and insomnia will receive CBT-I or an active control. The goal is to see if this talk therapy approach can ea…

This study tests if pairing a gentle, non-invasive brain stimulation (tDCS) with personalized physical therapy can help people with cerebellar damage (from stroke, tumor, or degeneration) improve movement and daily function. About 30 adults aged 18-75 will participate. The goal i…

This study tests whether injections of Poly-L-lactic acid (PLLA) can improve skin laxity in people who have loose skin after bariatric surgery. Fifteen participants receive three injections in one thigh over three months, while the other thigh is left untreated. During later plas…

This study tests whether solriamfetol (Sunosi), a drug used for sleepiness, can reduce fatigue in people with multiple sclerosis who also have excessive daytime sleepiness. About 46 participants will receive either the drug or a placebo for four weeks, then switch after a washout…

This study tests whether a personalized facial rehabilitation program can help people with systemic sclerosis who have facial tightness and mouth problems. About 60 adults with this rare autoimmune disease will either receive the rehab program or standard care. The goal is to see…

This study looks at whether two different exercise programs can help reduce depression in people with multiple sclerosis (MS) who also have major depressive disorder. Researchers will enroll 146 adults aged 18 to 64. One group will do a structured exercise program, while the othe…

This study tests a walking program called Walk-BEST for people aged 70+ with osteoporosis. The program uses a sensor to improve walking and aims to reduce falls and fractures. Researchers want to see if it's practical and acceptable for 28 participants.

This study tests whether L-dopa, a drug used for Parkinson's, can improve movement and communication in children with a rare genetic disorder called CTNNB1 syndrome. The condition causes developmental delays, muscle stiffness, and trouble walking. Seven children aged 1 to 15 will…

This long-term observational study uses MRI scans to track how multiple sclerosis (MS) changes over time in up to 3750 people. Researchers will compare brain images and immune system markers in untreated patients, those on approved therapies, and healthy volunteers. The goal is t…

This study is building a large collection of blood, saliva, urine, stool, and tissue samples from people who are at increased risk of cancer, including those with genetic predispositions, survivors of childhood or adult cancer, or those with precancerous conditions. The goal is t…

This natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

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 causes of brain development disorders, including Aicardi syndrome, polymicrogyria, and Dandy-Walker malformation. Researchers will analyze genetic data from 2000 participants with these conditions, all confirmed by MRI. The goal is to use…

This study aims to improve how doctors find and understand pheochromocytomas and paragangliomas, rare tumors that can cause dangerous high blood pressure. Researchers will use advanced imaging scans and genetic testing in up to 3,000 participants to learn more about these tumors.…

This natural history study is observing up to 250 people with Smith-Lemli-Opitz syndrome and related cholesterol disorders, as well as their relatives. Researchers will track symptoms, development, and lab results over several years to find better ways to measure disease progress…

This study is following children and adults with olfactory neuroblastoma, a rare cancer of the nasal cavity, to learn more about how the disease behaves over time. Researchers will collect medical records, blood samples, and survey responses from 525 participants to track symptom…

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 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 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 follows up to 4,000 people with vasculitis (a group of diseases that cause blood vessel inflammation) and healthy volunteers over many years. Researchers collect blood, urine, imaging, and genetic samples to learn how the disease develops and changes. The goal is to fi…

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 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 observational study is recruiting 2400 people with immune-related central nervous system diseases, such as multiple sclerosis, along with healthy volunteers for comparison. Researchers will use MRI scans, lumbar punctures, blood tests, and neurological exams to track disease…

This study aims to collect information and bone samples from up to 1,000 people with bone or mineral disorders, such as tumor-induced osteomalacia or familial tumoral calcinosis. Participants receive standard medical evaluations and may provide bone tissue from surgery or a biops…

This study looks at whether freezing ovarian tissue can help preserve fertility in people aged 2-21 with conditions like Turner syndrome, galactosemia, or premature ovarian insufficiency. Participants may have surgery to remove and freeze a gonad, with a small piece used for rese…

This study aims to develop a standardized questionnaire that lets patients with venous malformations report their own symptoms and treatment outcomes. Venous malformations are abnormal veins that can cause pain, swelling, and emotional distress, and current assessments may miss w…

This study aims to understand why bones weaken with age, leading to conditions like osteoporosis and osteoarthritis. Researchers will collect bone waste and blood samples from 2,000 people undergoing orthopedic surgery, including those with and without bone disease. By analyzing …

This study is developing questionnaires to measure how visible tumors in neurofibromatosis type 1 (NF1) affect patients' appearance concerns. About 110 participants, including children, adults, and caregivers, will complete surveys and join focus groups or interviews to give feed…

This study tests whether simple reminders—called nudges—sent to doctors and patients can increase the use of genetic testing for people with certain conditions like Parkinson's disease, ALS, or heart problems. The trial will compare different ways of prompting genetic testing, su…

This study investigates how Fabry disease damages the kidneys over time, even before routine tests show problems. Researchers will examine kidney biopsies and function in 50 patients, some of whom receive enzyme replacement therapy. They aim to see if starting treatment earlier o…

This study follows 842 people with multiple sclerosis who are starting a new under-the-skin injection of ocrelizumab. Researchers will ask participants how satisfied they are with the injection after 1, 6, and 12 months. The goal is to understand patient experience, not to test t…

This study aims to learn more about RASopathies, a group of genetic conditions that can cause developmental issues, birth defects, and increased cancer risk. Researchers will follow up to 500 people of any age who have or may have a RASopathy, along with their family members, for…

This study is gathering medical information and samples from up to 5,000 people with neurosurgical conditions like brain tumors, epilepsy, and Parkinson's disease. Participants receive standard care while their data is collected for future research. No new treatments are being te…

This study follows 650 people aged 8 to 99 with androgen insensitivity syndrome (AIS) to better understand their long-term health. Researchers will track hormones, bone density, heart health, metabolism, and tumor risk through regular check-ups and scans. No treatments are being …

This study will enroll 5,000 adults with chronic heart conditions like heart failure, high blood pressure, or aortic stenosis. Participants will wear a Prolaio digital health patch that monitors heart rate, breathing, and other vital signs. The goal is to observe how these condit…

This study is a disease registry that will collect information from 200 people in Egypt who have neurofibromatosis type 1 (NF1), a genetic condition that causes tumors to grow on nerves. The goal is to understand how the disease progresses over time, what treatments patients rece…

This study investigates whether a blood marker called anti-Müllerian hormone (AMH) can predict future infertility in children and adolescents who have undergone cancer treatment. Many young patients recover normal menstrual cycles or puberty after chemotherapy, but their ovarian …

This study is creating a decision aid for people with a rare heart condition called right anomalous aortic origin of a coronary artery (R-AAOCA). The condition raises the risk of sudden cardiac death, but the best management—surgery or careful observation—isn't clear. The aid wil…

This registry study follows women with multiple sclerosis (MS) who become pregnant, comparing those who took the medication Vumerity (diroximel fumarate) with those who took other MS treatments or no treatment at all. The main goal is to estimate the risk of major birth defects i…

This study is for people aged 3 and older with neurofibromatosis type 1 (NF1), a genetic condition that often causes noncancerous nerve tumors. About half of people with NF1 get these tumors, and sometimes they turn cancerous. Researchers want to test a new method to predict whic…

This study collects biological samples—like blood, skin biopsies, and swabs—from people with or at risk for skin diseases, as well as healthy volunteers. Researchers will use these samples to study the causes of skin conditions and explore potential treatments. Up to 700 particip…

This study is a pregnancy registry that gathers information from women with chronic neurological conditions like multiple sclerosis, NMOSD, and myasthenia gravis who are planning pregnancy, currently pregnant, or within one year postpartum. Participants can contribute blood, brea…

This study looks at whether adding certain nutrients to blood cells from people with septic shock can help restore the cells' energy-making machinery (mitochondria). Researchers will collect blood samples from 55 patients in the ICU and test these nutrients in the lab. The goal i…

This study looks at patients who develop a condition called MRONJ (bone death in the jaw) after taking osteoporosis medications. When this happens around a dental implant, doctors disagree on whether to remove the implant or try to save it. The study will compare healing and bone…

This study is looking at whether advanced heart imaging, especially cardiac MRI, can help identify the cause of cardiomyopathy and predict risks like death or heart failure in a diverse group of patients. The research focuses on adults in the Bronx, including many from Hispanic a…

This study aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…

This study aims to find out whether some women diagnosed with polycystic ovary syndrome (PCOS) actually have a rare genetic condition causing severe insulin resistance. Researchers will compare 25 women with a known LMNA gene mutation (linked to lipodystrophy) to 50 women with cl…

This study screens adults with certain cancers (like melanoma, lung, or colorectal cancer) to see if they qualify for experimental treatments being tested by the NCI Surgery Branch. These treatments involve taking a patient's own immune cells, growing them in a lab, and giving th…

This study looks at whether advanced imaging can detect a hidden heart condition called cardiac amyloidosis in people with severe aortic valve stenosis. The condition can complicate treatment and recovery. Researchers will use echocardiography, CT, and SPECT/CT scans to check for…

This study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …

This study investigates whether the bacteria causing Lyme disease can survive antibiotic treatment and cause ongoing symptoms. Researchers will follow 700 participants, including those with chronic Lyme disease, recovered individuals, and healthy volunteers, using blood tests, sp…

This study aims to find out how often exocrine pancreatic insufficiency (EPI) occurs in people with Alagille syndrome who have had a liver transplant. About 30 participants aged 7 to 50 will eat a high-fat diet and collect stool samples for lab testing. The results will help doct…

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 aims to uncover the genetic causes of a group of eye conditions called MAC (microphthalmia, anophthalmia, and uveal coloboma), which can lead to vision loss or blindness. Researchers will collect DNA samples from 600 people—both those with MAC and their close relatives…

This study looks back at medical records of 500 premature babies (born before 32 weeks or under 1500 grams) to see if abnormal blood chloride levels in the first weeks of life are linked to a higher risk of death or chronic lung disease (bronchopulmonary dysplasia). Researchers w…

This study is testing whether a mobile app called Vigilant can help monitor side effects in 100 people with skin cancer or melanoma who are starting a specific type of immunotherapy. Participants will use the app to report any symptoms they experience, and researchers will track …

This study aims to create a large genetic database of people with Turner syndrome, a rare condition where a person is missing all or part of an X chromosome. Researchers want to compare the genes of women with Turner syndrome who are fertile with those who are not. Up to 500 part…

This study is collecting medical data, blood, saliva, and tissue samples from up to 11,000 children, teens, and adults with known or suspected gynecologic conditions. The goal is to create a large database to better understand these conditions, including disorders of sex developm…

This study tests whether a new MRI technique, called inhomogeneous Magnetisation Transfer (ihMT), can accurately measure myelin damage and repair in people with multiple sclerosis (MS). The researchers will compare ihMT scans with standard MRI measures in 85 adults with early-sta…

This study follows 700 people with progressive multifocal leukoencephalopathy (PML), a rare brain infection caused by the JC virus. Researchers will collect blood, spinal fluid, and brain scans over time to understand how the disease progresses. No treatment is given; the goal is…

This study looks at adults with spinal deformity to understand which factors—like pain, daily functioning, mental health, and frailty—most influence quality of life. It follows 320 people receiving either conservative care, first-time corrective surgery, or a reoperation, using q…

This study looks back at medical records of 105 people who had a minimally invasive surgery called BEVAR to treat complex aortic aneurysms. Researchers want to see if two types of stent grafts (TUVA BX and iCover) are safe and work well. No new treatments are given; the study jus…

This pilot study tests whether a special MRI technique can measure wall shear stress—the force of blood against vein walls—in the pulmonary veins of infants. These veins carry blood from the lungs to the heart, and narrowing them (pulmonary vein stenosis) is a life-threatening co…

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 follows 130 adults with hypophosphatasia, a rare bone disorder, to understand how they are diagnosed and what symptoms they have. Researchers hope to find ways to reduce the long delay between first symptoms and diagnosis. No treatment is being tested; this is purely a…

This study aims to understand the quality of life of school-aged children who were treated for posterior urethral valves (PUV) in their first year of life. PUV is a rare birth defect that can affect kidney and bladder function. Researchers will use a standard questionnaire called…

This study tracks the natural course of fucosidosis, a rare genetic disorder, in up to 57 people. It collects medical history and ongoing health data without giving any experimental treatment. The goal is to better understand the disease and help design future therapies.

This study aims to create an artificial intelligence (AI) model that can predict complications in children who have a common heart defect called a ventricular septal defect (VSD) repaired with a catheter-based procedure. Researchers will analyze data from over 5,000 children's me…

This study tests a new radioactive tracer called [18F]FZTA to see if it can spot inflammation in the brain using PET scans. Researchers will compare 80 people—some with Multiple Sclerosis and some healthy—to see if the tracer lights up differently in those with MS. The goal is to…

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 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 follows up to 3,000 people who have, are at risk for, or are related to someone with a parathyroid disorder. Researchers will collect medical records, questionnaires, and samples like blood and saliva to learn what causes these conditions and how they change over time.…

This study aims to develop better MRI methods for imaging the heart and brain, and to describe heart diseases using these new techniques. It will also investigate whether gadolinium, a common MRI contrast agent, stays in the body long after use. Up to 5,000 healthy volunteers and…

This early-stage study tests whether people with advanced solid tumors can safely follow a diet low in methionine, an amino acid found in many proteins. Researchers will measure how well participants stick to the diet, check for side effects, and track changes in blood methionine…

This observational study looks at how people with relapsing-remitting multiple sclerosis (RRMS) perform when crossing obstacles while also doing a mental task. Researchers want to see if this dual-task challenge is related to levels of fatigue and disability. Thirty participants …

This study creates a detailed registry of people with hereditary hemorrhagic telangiectasia (HHT), a condition that causes abnormal blood vessels and bleeding. Researchers will track participants over time to collect information on symptoms, treatments, and disease progression. T…

22q11.2 deletion syndrome can cause heart defects, facial differences, and learning challenges, but its severity differs widely from person to person. This study analyzes DNA from 1,000 people with the deletion to identify genetic variations that might influence how the disorder …

This study looks at whether adding a high-fiber diet and short-term fasting can help melanoma patients undergoing immunotherapy. Forty participants will follow a high-fiber diet, with half also doing 72-hour fasts during each treatment cycle, while 20 others serve as a control gr…

This study aims to discover biological markers (biomarkers) that could help doctors diagnose and track neurological autoimmune diseases such as multiple sclerosis, myasthenia gravis, and Guillain-Barre syndrome. Researchers will analyze samples from 50,000 people, including patie…

This study investigates whether the multiple sclerosis medication ozanimod is linked to a higher risk of progressive multifocal leukoencephalopathy (PML), a rare but serious brain infection caused by the John Cunningham virus. Researchers will review reported cases of PML in peop…

This study tests whether a wireless sensor placed on the forehead can accurately measure oxygen levels in the brains of infants in the neonatal intensive care unit (NICU). The wireless device is compared side-by-side with the standard wired monitor in 20 newborns with conditions …

This study aims to create a new classification system for the most severe form of bronchopulmonary dysplasia (BPD), a chronic lung disease in premature infants. Researchers will use chest CT scans and bronchoscopy to gather detailed information about the lungs and blood vessels o…

This pilot study investigates whether regularly chewing a hard gum for four weeks can produce measurable changes in facial shape. Twenty healthy adults aged 20 to 40 will chew a standardized hard gum every other day, and researchers will use 3D facial imaging to track any shifts …

This observational study follows about 200 people in the United States who have chronic inflammatory demyelinating polyneuropathy (CIDP) and still experience disability despite at least three months of standard treatment. Researchers will track changes in disability scores and ex…

This study looks at whether a lung ultrasound score can help predict bronchopulmonary dysplasia (BPD), a serious lung condition, in premature babies born before 32 weeks. About 40% of extremely preterm infants develop BPD, which can cause long-term breathing problems. Researchers…

This international online study collects medical, behavioral, and developmental information from people with rare genetic changes that are linked to autism and other neurodevelopmental disorders. By partnering with families, researchers aim to build a detailed database to improve…

This study tracks 400 people who had a procedure to close a patent foramen ovale (PFO), a small hole between the heart's upper chambers that can raise stroke risk. Researchers want to see how often strokes or mini-strokes return, whether a residual shunt persists, and what factor…

This study investigates whether gut bacteria and the chemicals they produce influence inflammation in people with autoinflammatory diseases such as FMF, CAPS, and MKD. Researchers will analyze stool and blood samples from 300 patients to measure gut-derived metabolites and assess…

This study investigates whether injected GLP-1 receptor agonists, such as semaglutide, affect breastmilk production and its nutritional makeup in breastfeeding mothers. Participants will have their milk production measured before and after starting the medication, and milk sample…

This study is testing whether a non-invasive eye scan called optical coherence tomography (OCT) can help track neurological diseases like multiple sclerosis, Alzheimer's, Parkinson's, and migraine. Over two years, researchers will measure the thickness of retinal layers in 840 pa…

This study investigates how acetaldehyde, a byproduct of alcohol, affects cells in the mouth and whether it may increase oral cancer risk. Healthy volunteers and individuals with Fanconi anemia (a condition linked to higher cancer risk) will consume a single dose of alcohol and p…

This study compares two ways of giving oxygen during sedation for medical procedures done outside the operating room: high-flow nasal oxygen and a standard face mask. Researchers measure brain oxygen levels continuously to see which method keeps the brain better oxygenated. One h…

This study screens children and adolescents aged 5 to 18 who have chronic conditions like congenital heart disease, inflammatory bowel disease, celiac disease, or rheumatic diseases for lipid metabolism disorders. Participants provide a blood sample and complete a questionnaire a…

This study tracks the long-term safety and effectiveness of the growth hormone treatment Growtropin-II in children with short stature. It includes up to 5,000 children with growth hormone deficiency, idiopathic short stature, Turner syndrome, or those born small for gestational a…

This study looks at why some people with multiple sclerosis (MS) start or stay on Ocrevus Zunovo, a high-efficacy therapy, while others do not. Researchers will survey and interview 150 MS patients, along with their families and healthcare providers, to identify barriers and faci…

This study tests whether short-chain fatty acids (SCFAs) — substances naturally produced by gut bacteria — can reduce inflammation and protect thinking skills. Healthy adults receive either SCFA capsules or a placebo, then a low dose of a bacterial substance that briefly activate…

This study is a large registry that collects data from thousands of patients across Asia who have undergone transcatheter aortic valve replacement (TAVR) for severe aortic stenosis. Researchers will analyze medical records to see how well the valves perform over time and what fac…

This study creates a large registry of people diagnosed with hereditary hemorrhagic telangiectasia (HHT), a condition causing abnormal blood vessels and bleeding. Researchers will collect medical records and annual health updates for up to 10 years to understand how the disease c…

This study is a national registry in China that will track 500 women who experience an aortic dissection (a tear in the main artery) during pregnancy or within 12 weeks after giving birth. Researchers will collect information on treatments and outcomes for both mothers and babies…

This study follows 6,000 adults in France who have had their spleen removed or rendered non-functional due to surgery, radiation, or embolization. Researchers aim to identify risk factors for serious complications such as infections, cancer, and blood clots. By comparing differen…

This study examines whether two substances in the blood, Fetuin-A and COMP, are linked to the severity of knee osteoarthritis. Researchers will compare 120 people with knee osteoarthritis to 60 healthy volunteers, measuring their pain, mobility, and joint damage on X-rays. The go…

This study looks at how inflammation and energy use in the aorta differ between people with a bicuspid versus tricuspid aortic valve who have an ascending aortic aneurysm. Researchers will analyze blood and discarded surgical tissue to find molecular markers that could lead to be…

This study tests a new pulse oximeter worn on the abdomen, designed to measure oxygen levels in infants with congenital heart disease. Researchers compare its accuracy against standard fingertip monitors and direct blood measurements. The device also includes features to reduce e…

This study examines how nerve cells in the human embryonic retina and brain regions responsible for vision and higher thinking grow and connect. Researchers collect eye and brain tissue from pregnancies terminated due to medical conditions, then use advanced gene sequencing and e…

This study observes patients with high-risk stage II melanoma to see if starting anti-PD-1 immunotherapy before surgery leads to better outcomes compared to the usual timing after surgery. Researchers will track when patients begin systemic therapy and compare results between the…

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 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 compares different types of heart valve prostheses used to treat severe aortic stenosis in people with a small aortic annulus. Researchers will test the valves in the lab and then in patients using echocardiograms and a special MRI scan that measures blood flow. The go…

This registry tracks 2,000 adults with congenital heart disease who are on the waiting list for a heart or heart-lung transplant. The goal is to understand why some patients get worse or die while waiting, while others improve enough to be removed from the list. By collecting dat…

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 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 investigates why some people with moderate aortic valve stenosis get worse while others remain stable. Researchers will analyze immune system markers and detailed heart images from about 938 participants to find patterns that predict disease progression. The goal is to…

This study investigates clonal hematopoiesis (CH) — small mutations in blood cells that can arise naturally or after cancer treatment — in adults receiving chemotherapy or radiation for solid tumors like breast, lung, or colorectal cancer. Researchers will collect blood, saliva, …

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 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 is analyzing inflammatory markers in the blood and spinal fluid of 300 people who are being checked for multiple sclerosis (MS). The goal is to see if certain markers can help doctors tell MS apart from other autoimmune diseases that affect the brain and spinal cord. R…

This study follows 3,450 patients with aortic valve narrowing (aortic stenosis) to better understand their long-term outlook. Researchers will look at ultrasound measurements and other factors to find what predicts how the disease progresses. The goal is to improve how doctors as…

This study looks at how people who have lost an arm or were born without one experience phantom limb sensations—like feeling their missing hand move. Researchers will interview participants and map these sensations to help design prosthetics that are easier to control and feel mo…

This study compares severe respiratory infections in children with bronchopulmonary dysplasia (BPD) to those without BPD. Researchers will look back at past cases and follow new cases until 2030. The goal is to learn what makes these infections more dangerous for children with BP…

This study is observing 40 adults with multiple sclerosis to see how clinical tests of balance and walking relate to detailed motion-capture measurements. Participants will attend one session where they walk on force plates and perform balance tasks while cameras track their move…

This study aims to create a smartphone system that sends personalized pain management tips to adults with multiple sclerosis or spinal cord injury who have chronic pain. Researchers will interview participants to understand their preferences for the content and timing of these no…

This study aims to uncover the genetic causes of cardiomyopathy in children by analyzing DNA from affected individuals and their families. Researchers hope to identify mutations that lead to different types of cardiomyopathy, which could improve genetic counseling and deepen unde…

This study is looking at whether a simple exercise test and other health factors can predict how well people recover after a procedure called TAVI, which replaces a narrowed heart valve. Researchers will follow 161 patients with severe aortic stenosis to see which factors before …

This study aims to collect DNA samples and medical information from 2,000 people with heterotaxy (a condition where organs are arranged abnormally) and related heart defects, along with their family members. Researchers hope to identify the genetic causes of these conditions, whi…

This study follows 100 women with neuroimmune diseases (like multiple sclerosis or myasthenia gravis) through pregnancy and up to five years after. Researchers will track disease activity, pregnancy complications, and baby health to learn how these conditions affect fertility and…

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 examines how spinal posture and movement relate to balance in 30 adults with relapsing-remitting multiple sclerosis. Researchers will measure spinal angles and mobility, then compare them with balance tests. The goal is to identify links that could inform future rehabi…

This study is looking for certain cells in the blood of people with 9 types of advanced cancers that have spread. Researchers will take one blood sample from each of 450 participants before they start a new treatment. They will use two different lab methods to see if they can fin…

This study follows 232 people with a bicuspid aortic valve (a heart valve with two flaps instead of three) over 10 years. Researchers want to learn how the condition naturally progresses and what factors lead to serious problems like valve disease or bulging of the aorta. Partici…

This observational study follows 170 people with multiple sclerosis, neuromyelitis optica, and similar conditions, plus healthy volunteers, for up to 6 years. Researchers use MRI scans, blood tests, and spinal fluid analysis to monitor brain lesions and understand how the disease…

This study aims to measure how well infants' hearts can make new muscle cells, especially in those with a heart condition called Tetralogy of Fallot. Researchers will use a special imaging technique on heart tissue that is normally removed and thrown away during surgery. The goal…

This study is creating a central database and tissue bank for rare diseases affecting the kidneys and liver, such as ARPKD and Joubert syndrome. Researchers will collect medical information, genetic samples, and tissues from 200 participants to help doctors and scientists better …

The CLUE study is a research project that follows 1,000 people with neuroinflammatory diseases like multiple sclerosis and neuromyelitis optica. Participants undergo detailed MRI and PET scans at the start and after one year to see how their brain, spinal cord, and optic nerves c…

This study looks at whether a standard shoulder X-ray can detect bone loss in the weak arm of people who have had a stroke. Researchers will measure bone thickness on X-rays from 65 stroke survivors and compare those measurements to the gold-standard DXA bone density scan. If the…

This study surveys 300 young people with spina bifida to compare how different bladder management methods—like catheters or surgery—affect their quality of life. Participants fill out questionnaires about their bladder routine and daily well-being. The goal is to identify which s…

This study checks if a French version of the MEPS/MEPI elbow score works well for children aged 4 to 17. It includes kids with and without elbow injuries to see if the score accurately measures elbow pain and function. The goal is to make sure doctors can use this tool reliably i…

This study is collecting detailed genetic and health information from 500 people with various cancers, including breast, lung, and leukemia. The goal is to create a shared database that helps researchers develop more personalized treatments. Participants provide samples and data,…

This study looks at melanoma tumors that spread between the main tumor and nearby lymph nodes (called in-transit metastases). Researchers will take biopsies from different spots and use a safe tracer to measure oxygen levels. They aim to understand how these tumors change metabol…

This observational study collects tissue samples from children undergoing surgery for polydactyly or hip dislocation, as well as from biobanks, to see which cells are best at forming cartilage. Researchers will test these cells in the lab and in animal models. The goal is to lear…

This study is investigating whether low oxygen levels (hypoxia) in melanoma tumors and nearby lymph nodes are linked to worse outcomes. Researchers will give 50 patients a safe oral tracer called pimonidazole before their standard surgery. By measuring hypoxia in the removed tiss…

This pilot study explores whether eating high-fermented foods (like yogurt and kimchi) followed by high-fiber supplements can change the gut microbiome and improve immune response in people with melanoma or lung cancer who are receiving standard immunotherapy. Researchers will tr…

This study looks at whether inflammation after a minimally invasive heart valve replacement (TAVI) can cause dangerous heart rhythm problems. Researchers will use a special PET scan to measure inflammation in the heart's electrical pathways. The goal is to better predict which pa…

This study analyzes dried blood spots from 630 newborns to find protein and RNA markers that could predict which babies with congenital CMV will develop hearing loss or other problems. Researchers will compare samples from infected babies who had complications, those who did not,…

This study follows 20 adults with multiple sclerosis who are already scheduled for a stem cell transplant. Researchers will measure levels of an immune-suppressing drug called ATLG and track how the immune system rebuilds itself afterward. The goal is to learn more about immune r…

This study looks at whether the bacteria naturally found in urine can help doctors better understand melanoma, a serious skin cancer, and predict how well patients will respond to treatment. Researchers will collect urine and stool samples from 50 people with advanced melanoma st…

This study looks at whether soaking hands in water for up to 20 minutes can be a simple screening tool for cystic fibrosis (CF) carrier status. Researchers will take photos of participants' hands and score the wrinkling, both by human observation and using a computer algorithm. T…

This study observes 1000 people with severe aortic stenosis and kidney disease who are getting a TAVI procedure. Researchers want to see how removing the blocked valve affects the kidneys' response to contrast dye used during the procedure. The goal is to understand if TAVI lower…

This study gathers medical information from people with MNGIE, a rare genetic disease that affects the nerves and digestive system. Researchers want to learn how the disease progresses and find better ways to measure it. Up to 50 patients worldwide can join, and no new treatments…

This study aims to find the genes responsible for Hirschsprung disease, a condition where nerve cells are missing from parts of the intestine. Researchers will collect DNA samples from 3,000 people with the disease and their close family members. By analyzing common and rare gene…

This study aims to collect and analyze tumor samples from 10,000 adults with advanced solid cancers (like breast, lung, or colon cancer) to create a province-wide gene database. The goal is to help doctors identify which targeted drugs or clinical trials might work best for each …

This study aims to learn how well heart scans (MRI, ultrasound, PET) can detect and predict outcomes in people with suspected or known inflammatory heart diseases. Researchers will follow 5,000 adults for one year to see how often heart involvement is confirmed and how it affects…

This study looks at how melanoma tumors process energy, specifically glucose, to see if certain metabolic patterns are linked to cancer spread. Researchers will give a special glucose tracer to 400 adults with melanoma before their surgery and analyze the tumor tissue. Participan…

This study looks at whether a structured program can help more high-risk smokers get a first lung cancer screening with a CT scan. About 500 people aged 50 to 74 who smoke or recently quit will join. The goal is to see if a dedicated network makes screening easier and more effect…

This study is examining heart valve tissue from 100 people undergoing surgery for aortic stenosis to see how often ATTR amyloid deposits are present. Researchers will use mass spectrometry to identify the type of amyloid. The goal is to better understand the link between amyloido…

This study looks at whether a special ultrasound technique, called shear wave elastography, can predict how well a child's kidney will recover after surgery to fix a blockage. Researchers will measure kidney stiffness before and after surgery in 84 children aged 6 months to 18 ye…

This study tests a new discharge protocol for preterm infants with bronchopulmonary dysplasia (BPD), a chronic lung disease. The protocol was developed by national experts to improve the transition from NICU to home. Researchers will track follow-up rates, hospital readmissions, …

This observational study aims to find biological markers (biomarkers) for RAI1-related disorders, including Smith-Magenis and Potocki-Lupski syndromes. Researchers will study 90 participants through clinical exams, blood tests, optional skin biopsies, and sleep studies. The goal …

This study will follow 300 people with aortic stenosis who are scheduled for a TAVI procedure to replace their heart valve. Researchers will use heart scans, blood tests, and checkups to measure how the heart's condition changes over time. The goal is to better understand short- …

This study aims to find small molecules called microRNAs in the blood that can predict how well two osteoporosis drugs (romosozumab and abaloparatide) work in postmenopausal women. Researchers will measure bone density and blood markers over 12 months in 42 women. The goal is to …

This study looks back at 483 patients who had small rectal neuroendocrine tumors (less than 2 cm) removed using one of two endoscopic techniques: endoscopic submucosal dissection (ESD) or endoscopic partial muscle resection (EPMR). The goal is to compare how often each method lea…

This study is recruiting 100 people who need hip replacement surgery after a fragility fracture (a break from a minor fall) and comparing them to patients having hip replacement for arthritis. Researchers will use a simple questionnaire (SARC-F) and analyze bone and muscle tissue…