Disorder of development or morphogenesis

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

Researchers are testing whether adding terfenadine to standard first-line disease-modifying therapy is safe and may improve outcomes for adults with relapsing-remitting multiple sclerosis. About 100 participants aged 18 to 65 will be randomly assigned to receive either terfenadin…

This early-stage trial tests whether a stool transplant (FMT) can help cancer patients who develop severe diarrhea or colon inflammation from immune-checkpoint inhibitor drugs. About 40 patients with various cancers will receive the transplant via colonoscopy. The main goal is to…

This study tests a daily oral drug called alpelisib in people aged 2 and older with PIK3CA-related overgrowth spectrum (PROS), a rare condition causing abnormal tissue growth. The goal is to see if the drug can shrink overgrown tissue and improve symptoms. About 104 participants …

This early-phase trial is testing an experimental drug called BI-1808, alone or with the immunotherapy pembrolizumab (Keytruda), in people with advanced cancers like ovarian cancer, T-cell lymphoma, and melanoma. The main goals are to check safety, find the best dose, and see how…

This early-stage trial is testing whether an oral probiotic called R-5780 is safe and tolerable when taken alongside a standard PD-1 checkpoint inhibitor in people with advanced solid tumors (like melanoma or certain skin cancers). About 33 adults whose cancer has not responded t…

This study tests a new drug called AU-007 in people with advanced solid tumors that have spread or cannot be removed by surgery. The drug is designed to help the immune system fight cancer. About 159 participants will receive AU-007 alone or with other medicines to find the safes…

This phase 2 trial tests enasidenib, a daily pill that targets the IDH2 gene mutation, in 40 adults with rare cancers of the nasal cavity or skull base that have spread or come back. Participants take the drug once daily in 28-day cycles, and researchers will track how long the c…

Researchers at a Korean hospital are following 210 adults who receive a cementless total hip replacement using the collarless Actis femoral stem. The study focuses on Asian patients, who tend to be younger at surgery and more often need the operation because of lost blood supply …

This study tests a new medicine called empasiprubart against the standard treatment (IVIg) for people with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disease that causes weakness and numbness. About 218 adults who have used IVIg before will receive either t…

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 daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare condition that causes muscle weakness and heart problems. Participants will receive either the drug or a placebo for 72 weeks. The main goal is to see if the drug…

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

This phase 2 trial is testing 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.…

Researchers are comparing two sequences of treatment for metastatic castration-resistant prostate cancer that carries certain DNA repair gene mutations. Participants receive either carboplatin first and olaparib second, or olaparib first and carboplatin second, switching after th…

Researchers are testing a combination of two drugs, doxecitine and doxribtimine, in adults with thymidine kinase 2 (TK2) deficiency, a rare genetic condition that causes muscle weakness and breathing problems. The study will enroll about 15 adults with moderate to severe disease.…

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 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 zilovertamab vedotin in children and young adults whose blood cancers (like certain leukemias and lymphomas) or solid tumors (neuroblastoma, Ewing sarcoma) have come back or not responded to treatment. The goal is to see if the drug is safe and can …

This phase 3 trial tests whether empasiprubart, given by IV infusion, can help adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder that causes weakness and disability. The study will enroll 160 participants. For the first 6 months, some get the …

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 study tests whether adding the drug naxitamab to standard chemotherapy is safe and effective for children and young adults newly diagnosed with high-risk neuroblastoma, an aggressive nerve cell cancer. Ten participants aged 1 to 21 will receive the combination and be monitor…

This study is testing whether a drug called vorinostat is safe and helpful for people with Pitt Hopkins syndrome, a rare genetic condition that affects development. Five participants will each receive a placebo and two different doses of the drug in a special 'N-of-1' design, mea…

This early-stage study is testing a new drug called BGB-B2033, alone or with other drugs, in people with advanced liver, stomach, or lung cancers. The goal is to see if it is safe and how well it works. About 392 participants will take part.

This study is for people who completed a previous trial of KL1333 (napazimone) for primary mitochondrial disease. It will test the drug's long-term safety and whether it helps with fatigue and daily activities. About 140 participants will take the pill twice daily for an extended…

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 phase 2 trial tests a drug called BMS-986504 in people with advanced solid tumors that have lost a gene called MTAP. The drug is given alone or with other cancer medicines. The study aims to see if the drug shrinks tumors and how safe it is. About 260 adults will take part.

This early-phase trial tests whether giving different drug combinations after surgery, based on how well the tumor responded to initial treatment, can reduce the risk of melanoma returning. About 50 adults with stage III or IV BRAF-mutant melanoma will receive encorafenib, binime…

This study compares a stem cell transplant (using a person's own cells) against the best available medications for people with relapsing multiple sclerosis that hasn't responded to treatment. About 156 participants will be randomly assigned to one of the two approaches and follow…

This early-stage trial is testing a new drug called BL-M14D1 in about 120 people with advanced small cell lung cancer or other neuroendocrine cancers that have spread or cannot be removed. The drug works by homing in on a protein called DLL3 found on these cancer cells and delive…

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

This phase 2 trial tests whether a 3-day course of high-dose corticosteroids (methylprednisolone or prednisone) can help heal ring-enhancing brain lesions in people with multiple sclerosis. Thirty adults with MS and a specific type of active lesion will be randomly assigned to re…

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 study tests an oral drug called NEO212 in people with specific brain tumors (astrocytoma, glioblastoma) or cancers that have spread to the brain. The goal is to find the safest dose and see if it helps control tumor growth. About 134 adults will take NEO212 alone or with sta…

This phase 3 trial tests whether taking darovasertib and crizotinib after standard treatment can delay or prevent uveal melanoma from returning. The study enrolls adults with high-risk, non-metastatic uveal melanoma who have completed local therapy such as surgery or radiation. P…

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 phase 2 trial is testing a drug called tebentafusp in 19 people with a rare eye cancer (uveal melanoma) that has spread to the liver. Participants receive weekly infusions for 6 months, then may have surgery to remove any remaining tumors. The goal is to see if the drug can …

This study provides continued access to dabrafenib and/or trametinib for up to 100 patients with melanoma, lung cancer, or other solid tumors who were benefiting from these drugs in a prior Novartis or GSK trial. The main goal is to track long-term side effects and safety over ti…

This study is for people who have both an autoimmune disease (like rheumatoid arthritis, lupus, or multiple sclerosis) and an advanced cancer that has spread or cannot be removed by surgery. It tests the safety and effectiveness of the immunotherapy drug nivolumab, given alone or…

This early-stage trial tests a personalized cell therapy called lifileucel for people with advanced uveal melanoma (a rare eye cancer) or certain sarcomas (connective tissue cancers). The treatment involves removing immune cells from the patient's tumor, growing them in a lab, an…

This study tests whether adding a fecal microbiota transplant called LND101 to standard immunotherapy can help stop advanced melanoma from growing or spreading. About 128 people with stage IV or unresectable melanoma will receive either immunotherapy alone or immunotherapy plus L…

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 early-phase trial tests SL-28, a cell therapy made from donor immune cells, in 60 people with advanced solid tumors (like lung, pancreatic, or breast cancer) that haven't responded to standard treatments. The study aims to find a safe dose and see if the therapy can shrink t…

This study tests whether giving two medicines (acetaminophen and ibuprofen) together works better than ibuprofen alone to close a common heart blood vessel in extremely premature infants (born before 27 weeks). About 310 babies will take part. The goal is to see if the combo redu…

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 phase 2 trial compares two treatment combinations for people with BRAF-V600 mutant melanoma that has spread to the brain. One group gets a targeted therapy (encorafenib and binimetinib) plus an immunotherapy (nivolumab), while the other gets two immunotherapies (ipilimumab a…

This early-phase trial tests a new drug called BI 3810944 in adults with advanced solid tumours or melanoma who have no other treatment options. The study aims to find a safe and tolerable dose, and to see if the drug can shrink tumours. Participants receive the drug every three …

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 special stent designed to keep a small heart vessel open in babies under 6 months old who have a congenital heart defect. The goal is to improve blood flow to the lungs and delay or avoid open-heart surgery. About 35 infants will receive the stent and be monito…

This study is testing a new experimental drug called GI-101, given alone or with pembrolizumab or lenvatinib, in people with advanced or metastatic solid tumors (cancers that have spread). The goal is to see if the drug is safe and can shrink or control tumors. About 317 adults w…

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 study compares two surgical approaches for hip dysplasia: the standard periacetabular osteotomy (PAO) alone versus PAO combined with hip arthroscopy. Researchers will enroll 106 people aged 15-55 with mild or no arthritis. The goal is to see if adding arthroscopy leads to be…

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

This study tests 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 early-phase trial tests a new treatment for children and young adults with medulloblastoma or similar brain tumors that have come back or not responded to standard therapy. The treatment uses the patient's own immune cells, modified to recognize and attack tumor cells, deliv…

This study will test a special balloon coated with the drug paclitaxel to treat long blockages in the leg arteries. About 300 people with severe leg pain or wounds will receive the balloon treatment during a standard angioplasty procedure. Researchers will track how well the arte…

Researchers are testing whether combining SX-682, an experimental drug that blocks CXCR1 and CXCR2, with proton craniospinal irradiation is safe and effective for people with leptomeningeal disease from melanoma or non-small cell lung cancer. Participants take SX-682 by mouth twi…

This study tests whether a soft, FDA-approved belly band can help premature babies on breathing support develop stronger abdominal muscles. Researchers will measure muscle thickness with ultrasound and see if babies reach full oral feedings earlier. About 40 premature infants bet…

This study looks at how a highly targeted form of radiation called stereotactic radiosurgery (CyberKnife) works alongside newer treatments like immunotherapy and targeted therapy for people with melanoma that has spread to the brain. Researchers will observe 50 patients over time…

This Phase 2b study tests whether the drug IMVT-1402 can help adults with CIDP, a nerve disorder causing weakness and numbness. About 162 participants will receive either the drug or a placebo weekly for up to 52 weeks. The main goal is to see if the drug keeps the disease from w…

This study compares two daily pills, ozanimod and fingolimod, to see which works better at preventing MS relapses in children and teens aged 10 to 17. Participants will be randomly assigned to one of the two drugs and monitored for about two years. The goal is to find a more effe…

This study is testing whether giving radiation therapy along with immunotherapy before surgery can improve outcomes for people with sinonasal or anorectal mucosal melanoma, two rare and aggressive cancers. About 40 participants will receive combination immunotherapy (such as ipil…

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 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 tests a drug called tebentafusp in people with a specific type of advanced eye cancer (uveal melanoma) that has spread to other parts of the body. The drug is designed to help the immune system attack cancer cells. Researchers will also use a blood test to measure tumo…

This early-phase study tests a new radioactive drug, 225Ac-ETN029, in 116 adults with advanced DLL3-positive cancers like small cell lung cancer or neuroendocrine tumors. The main goal is to check safety and find the right dose, while also seeing if the drug can shrink tumors. Pa…

This early study tests a new treatment called azercabtagene zapreleucel (azer-cel) for people with B-cell mediated autoimmune disorders like multiple sclerosis and neuromyelitis optica. The treatment uses a patient's own immune cells that are modified in a lab to target and destr…

This study tests whether adding AGuIX gadolinium-based nanoparticles to standard brain radiation can better control brain metastases that are at high risk of coming back. About 134 adults with certain cancers (like melanoma, lung, breast, or colorectal) that have spread to the br…

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 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 phase 1b trial is testing whether ixazomib, a drug already used for multiple myeloma, is safe for people with multiple sclerosis. The study will enroll 72 adults with relapsing-remitting or progressive MS. Participants will receive either ixazomib or a placebo for up to 24 m…

This early-stage study aims to develop a personalized treatment for children with syndromic craniosynostosis, a group of rare genetic disorders where skull bones fuse too early. The approach uses a nano-engineered gel to deliver custom genetic medicine directly to the affected ar…

This trial is testing a robotic exoskeleton called ABLE Kids to see if it is safe and practical for children who have trouble walking due to conditions like cerebral palsy, spinal cord injury, or muscle weakness. Fifteen children will use the device in up to ten one-hour training…

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 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 trial tests whether a monitor that measures muscle response can guide the dose of the muscle relaxant atracurium in intensive care unit patients who need a continuous drip for more than 48 hours. Researchers will compare a group whose dose is adjusted using the monitor to a …

This international study is evaluating a minimally invasive procedure to close a type of heart defect called a perimembranous ventricular septal defect (PmVSD) — a hole between the heart's lower chambers. The procedure uses a small device inserted through a catheter to plug the h…

This study tests a combination of two drugs, trametinib and azacitidine, for children newly diagnosed with juvenile myelomonocytic leukemia (JMML), a rare blood cancer. Lower-risk patients get just these two drugs, while higher-risk patients also receive standard chemotherapy. Th…

This study tests whether specially grown donor immune cells (called cytotoxic T lymphocytes) can safely and effectively control BK and JC virus infections in people with weakened immune systems, such as those with cancer, HIV, or organ transplants. About 100 participants will rec…

This trial tests whether short sprint interval training, involving repeated 5-second high-intensity cycling bursts, can improve walking, strength, fatigue, and cognitive speed in people with multiple sclerosis. Participants are randomly assigned to either the sprint program or th…

This study tests a home-based cardiac rehabilitation program delivered through a custom app for people who have had a heart valve procedure. Participants who choose not to attend center-based rehab are randomly assigned to usual care, the app with self-guided content, or the app …

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

This 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 two oral drugs, AMXT 1501 and DFMO, given alongside standard treatments for people with advanced solid tumors (breast cancer or melanoma) that have worsened after prior therapies. The goal is to find a safe dose and see if the combination helps shrink tumors or s…

This trial tests whether traditional open-heart surgery to replace a narrowed aortic valve works as well as a less invasive catheter-based approach in patients with severe aortic stenosis who are otherwise at low surgical risk. Researchers will compare rates of death, stroke, and…

This study is for children newly diagnosed with high-risk neuroblastoma, a rare and aggressive nerve cancer. It uses genetic testing to choose personalized chemotherapy, then tests whether adding the drug DFMO to standard immunotherapy helps prevent relapse. About 500 children wi…

This study tests whether a weekly under-the-skin antibody treatment (XEMBIFY) provides similar blood levels as a standard IV infusion (Gamunex-C) given every 3 weeks in people with CIDP, a chronic nerve disorder. About 40 adults who are stable on IVIG will first receive Gamunex-C…

This study tests whether gonadal tissue removed from children with disorders of sex development (DSD) can be frozen and stored to preserve fertility. The tissue is already being removed for medical reasons, such as preventing cancer. Researchers will check the tissue for viable g…

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

This 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 study tests whether adding the immunotherapy drug sintilimab to standard chemotherapy can help prevent a rare type of skin cancer (mucosal melanoma) from coming back after surgery. About 220 adults with PD-L1-positive, completely removed tumors will be randomly assigned to g…

This early-phase study tests whether a new cell therapy called B7-H3 CAR T cells is safe and can help children and young adults whose solid tumors have returned or stopped responding to standard treatments. About 40 participants will receive their own immune cells that have been …

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 study is testing the safety of Replagal, an enzyme replacement therapy, in 5 Indian children and adults with Fabry disease who have not taken it before. Participants receive an infusion every 2 weeks for about a year. The main goal is to track side effects and serious reacti…

This trial compares two surgical techniques for treating camptodactyly, a condition where fingers are bent and can't fully straighten. Children and adolescents with a finger contracture of 30 degrees or more will be randomly assigned to receive either a radial longitudinal flap o…

This study tests whether giving two strong medications one after the other—rituximab then cladribine—is safe and works well for people with relapsing-remitting multiple sclerosis (MS). Sixty adults aged 18 to 50 with active MS within the last year will receive the treatment and b…

This early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. T…

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 study tests a new pill called tipifarnib combined with an FDA-approved drug, naxitamab, for people with neuroblastoma that has come back or not responded to treatment. The goal is to see if the combination is safe and helps shrink tumors or slow the disease. The trial is for…

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 Phase 3 trial tests whether the drug claseprubart (DNTH103) can prevent relapses in adults with CIDP, a nerve disorder that causes weakness and disability. About 256 participants will receive either the drug or a placebo. The main goal is to see how long it takes for symptom…

This study tests a new drug called BNT326, given alone or with other cancer immunotherapies, in about 980 adults with advanced solid tumors that have spread or come back after prior treatment. The goal is to find the best dose and see if it is safe and effective. It is an early-s…

This early-phase trial is testing a new drug called KIN-2787 (exarafenib), alone or combined with another drug (binimetinib), in 400 adults with advanced solid tumors that have BRAF or NRAS mutations. The main goals are to check safety, find the best dose, and see if the drug can…

This early-phase study tests a new drug called DSB2455 in adults with advanced cancers (breast, ovarian, prostate, pancreatic, or brain metastases) that have a specific DNA repair problem. The main goals are to check safety and see if the drug shrinks tumors. About 180 participan…

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…

Some people are born with only one working lower heart chamber, a condition often managed with a Fontan operation. Over time, that single ventricle can weaken, leading to Fontan circulatory failure, where the heart struggles to pump enough oxygen-rich blood. This trial tests whet…

This study tests a new device that creates a connection between an artery and a vein using a small needle puncture instead of open surgery. The goal is to provide a reliable access point for dialysis in people with kidney failure. About 126 adults will be enrolled and followed fo…

This phase 2 trial tests whether injecting two experimental immune drugs, L19IL2 and L19TNF, directly into tumors can help people with advanced melanoma that no longer responds to standard immunotherapy. Participants receive one of the injected drugs or a combination of both, alo…

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 study tests the drug migalastat in 8 children aged 2 to 12 with Fabry disease, a rare genetic disorder. The goal is to see if the drug is safe, how it moves through the body, and if it helps protect kidney function. Participants will take the medicine for 12 months.

This study tests whether combining a liver-directed chemotherapy (HEPZATO KIT) with an immunotherapy drug (Opdualag) is safe and effective for people with metastatic melanoma that has spread to the liver. About 15 participants who have not received prior systemic treatment will r…

This early-stage study tests a drug called codrituzumab in children and young adults (ages 1 to 21) whose solid tumors have come back or not responded to treatment and carry a specific protein called GPC3. The main goal is to find a safe dose and see how the body handles the drug…

This study looks at children aged 2 to 18 having minimally invasive heart surgery to fix simple defects. Researchers compare two approaches: giving sugammadex in doses adjusted to real-time nerve monitoring, versus letting muscle relaxation wear off naturally. The goal is to see …

This study tests a new way to deliver radiation therapy for people with lung cancer, melanoma, or kidney cancer that has started to grow again in a few spots despite treatment. The approach uses a PET scan to find the most active parts of the tumor and delivers a higher radiation…

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

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

This trial tests two ways to correct flat head syndrome in infants: caregiver-led repositioning and a custom-fit helmet. Researchers will measure head shape changes in treated infants and compare them with a group of typically developing infants. Infants with torticollis will als…

This study tests a combination of two immunotherapy drugs (nivolumab and ipilimumab) along with two anti-rejection medications (sirolimus and prednisone) in kidney transplant recipients who have advanced skin cancer that cannot be surgically removed or has spread. The goal is to …

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 phase 2 trial tests a new treatment called LM103 TILs, made from a patient's own immune cells, for advanced melanoma that hasn't responded to standard therapies. About 92 adults will be randomly assigned to receive either the TIL therapy or standard care. The study aims to s…

This Phase 3 trial tests whether giving children azathioprine or rituximab immediately after their first MOGAD attack reduces relapses and long-term disability compared with waiting until a second attack. The study enrolls children aged 6 to 17 who have had a first inflammatory e…

This study tests a combination of darolutamide, hormone therapy, and a lower dose of the chemotherapy drug docetaxel in men with metastatic hormone-sensitive prostate cancer. The goal is to see if this approach can keep PSA levels very low (below 0.2 ng/mL) at 24 weeks while redu…

This trial tests whether a daily probiotic pill can improve gut health and lower heart disease risk in 250 people living with HIV who drink heavily. Heavy drinking changes gut bacteria, which may raise inflammation and heart disease risk. Researchers will measure changes in gut b…

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 tests an experimental oral drug called VMD-102 in people with advanced solid tumors, including liver, lung, kidney, skin (uveal melanoma), and colorectal cancers that have not responded to standard treatments. The drug works by blocking a specific protein (PKC epsilon)…

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 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 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 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 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 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 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 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 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 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 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 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 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 early-access program tests whether a weekly infusion of tralesinidase alfa directly into the brain's fluid spaces is safe and tolerable for children with Sanfilippo syndrome type B, a rare genetic disorder that causes severe neurological decline. The study enrolls about 10 c…

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 early-stage trial tests a new drug called MST-0312, designed to help the body's immune system fight cancer more effectively. It is being studied alone or with another cancer drug (pembrolizumab) in people with several types of solid tumors, including lung, melanoma, bladder,…

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 a drug called afatinib in 25 adults with Fanconi anemia who have advanced head and neck cancer that cannot be removed by surgery. Afatinib is a pill that blocks a protein helping cancer grow. The goal is to see if it can shrink tumors and control the disease. Res…

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 study tests whether a CT scan 3 months after TAVI (aortic valve replacement) can help doctors decide who needs blood thinners and who can stop them. Currently, patients take aspirin for life, which can cause bleeding. The trial will enroll 2,500 people and compare this perso…

This Phase 2 study tests whether a Traditional Chinese Medicine (TCM) herbal formula can improve muscle strength and bone health in pre-frail adults aged 65 and older, compared to the nutritional supplement HMB. Over 24 weeks, 90 participants will take either the TCM powder or HM…

This study tests a new drug called Becotatug Vedotin in children aged 2 to 18 with certain solid tumors that have come back or spread. The drug targets a protein called EGFR found on many cancer cells. The main goal is to see if the drug is safe and to find the best dose, while a…

This early-phase trial is testing a new treatment called LauT-1 for people with advanced melanoma or sarcoma that has not responded to standard therapies. LauT-1 is made by taking a patient's own immune cells, modifying them in a lab to better recognize and attack their cancer, a…

This small pilot study tests whether cutting a fibrous strand at the end of the spinal cord (filum terminale) can relieve symptoms of occult tethered cord syndrome better than medical management alone. Twenty people aged 2 to 80 who have not improved with standard care will be ra…

This study tests whether a high-energy diet helps infants with congenital heart disease grow and develop their brains better after heart surgery. Researchers will compare high-energy versus standard nutrition in 160 babies aged 0-6 months. The goal is to see if the special diet i…

This study compares two flapless surgical techniques used alongside braces to see which better improves gum and bone changes. Forty adults with mild to moderate crowding will receive either a soft tissue graft or a bone graft during their orthodontic treatment. Researchers will m…

This study tests whether a purine-rich diet can lower disease markers in people with AICA-ribosiduria, a rare genetic condition causing severe disability and epilepsy. Ten participants will follow a diet with 160 mg of purines per day. Early results in one patient showed promise,…

Researchers at Johns Hopkins University are studying whether neuromuscular ultrasound and MRI can serve as reliable markers of swallowing muscle problems in people with inclusion body myositis (IBM). The study compares imaging and clinical findings in 30 participants, including p…

This study looks at two different lab tests (live-cell and fixed-cell) to see which one is better at diagnosing MOGAD, a rare autoimmune disease that affects the brain and nerves. Researchers will test 240 adults who are suspected of having MOGAD, along with people who have other…

The ORACLE study watches people who have finished treatment for early-stage solid tumors, such as lung, breast, colon, and pancreatic cancers. Researchers want to see if a blood test called Guardant Reveal can find tiny traces of tumor DNA that might signal the cancer is coming b…

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 is testing a smartphone app that records and analyzes speech to help diagnose and monitor neurodegenerative conditions like dementia, Parkinson's disease, multiple sclerosis, and motor neuron disease. Researchers will compare speech patterns from 150 people with these …

Researchers at Yale University are testing a PET imaging tracer called [18F]3F4AP to see whether it can measure demyelination, the loss of the protective myelin sheath around nerve fibers. The study includes healthy volunteers and people with conditions such as multiple sclerosis…

This study tests whether a newer type of CT scan, called photon-counting CT, can accurately measure blood flow to the heart muscle and detect scarring or fibrosis. Researchers will compare the CT results with cardiac MRI and invasive coronary angiography in 100 patients with aort…

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 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 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 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 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 an artificial intelligence program that analyzes heart rhythm data from a small, wearable patch to estimate how well the heart pumps blood. Researchers will compare the AI's results to standard ultrasound in 2,000 adults. If accurate, this could offer a simpler, …

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 whether a quick blood test (CT-ADP) can help doctors detect and fix leaks around a new heart valve during a minimally invasive TAVI procedure. Currently, doctors often use general anesthesia and an ultrasound probe in the throat to check for leaks, but this study…

This study checks if a computer tool called BBCT-hip can accurately predict the risk of breaking the thigh bone (femur) when a person falls. Researchers will enroll 300 postmenopausal women with osteoporosis—150 who have already had a fracture and 150 who have not. All will get a…

Researchers are testing whether sulforaphane, a substance found in broccoli and other cruciferous vegetables, can prevent melanoma in people who have multiple atypical moles and a prior history of melanoma. Participants take sulforaphane or a placebo by mouth. The trial measures …

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 study tests if giving very premature babies (born before 30 weeks) slightly less fluid (135 mL per kg per day) compared to a standard amount (165 mL per kg per day) can prevent bronchopulmonary dysplasia (BPD), a chronic lung disease. About 750 babies will be enrolled across…

Hereditary hemorrhagic telangiectasia (HHT) causes tangled blood vessels that lead to frequent, hard-to-stop nosebleeds and low hemoglobin. Researchers are testing whether intravenous bevacizumab, given once or twice a year after nasal electrocoagulation, reduces nosebleed severi…

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 phase 2 trial tests whether a single dose of cabergoline can prevent breast engorgement, milk leakage, and tenderness after abortion or pregnancy loss at 12-18 weeks. About 348 participants will receive either cabergoline or a placebo right after the procedure and report sym…

This study tests whether balance training tailored to each person's difficulty level is feasible for people with multiple sclerosis during a three-week inpatient rehabilitation stay. Participants are randomly assigned to either standard group training or individually tailored bal…

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…

Researchers are testing whether warming the wrist with a forced-air warmer helps doctors place arterial catheters on the first try in babies and toddlers having heart surgery. The trial includes 126 children aged 2 years or younger. Half receive local warming before the procedure…

Researchers are testing whether a home-based program combining adapted exercise and mindfulness training can improve walking ability and sleep quality in people with rare neurodegenerative diseases such as leukodystrophy, ataxia, and adrenomyeloneuropathy. The study enrolls about…

Researchers are testing whether a nerve block called a PENG block reduces pain after periacetabular osteotomy, a major hip realignment surgery. In the study, 90 adults scheduled for this surgery receive either ropivacaine, a local anesthetic, or a saline placebo injected around s…

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 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 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 looks at how two parts of cannabis, THC and CBD, affect thinking and bladder symptoms in people with Multiple Sclerosis (MS). Thirty adults with MS will take either THC or CBD oil for 15 weeks and complete tests and questionnaires. The goal is to see if one compound he…

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 cannabidiol (CBD) can safely improve behavior, mood, sleep, and daily function in people with Sanfilippo syndrome, a rare genetic disorder. Thirty-five participants will receive either CBD or a placebo, then switch after a break. Caregivers will report on…

This study tests a virtual psychological support program called HeartGPS for mothers whose babies are diagnosed with congenital heart disease before birth. About 210 mothers and their infants will take part, with some receiving the program plus usual care and others only usual ca…

This study tests whether a short, intense cycling workout, known as high-intensity interval training (HIIT), can help people with multiple sclerosis (MS) walk farther and feel better in daily life. The trial includes 30 adults with MS at different disability levels, from mild to …

This trial tests whether botulinum toxin type A injections can improve lower-lip asymmetry in children with hemifacial microsomia, a condition where the lower lip moves unevenly during smiling or speaking. The study enrolls children under 18 with this condition and a crooked-mout…

Children having corrective heart surgery through a chest incision often need strong pain medicine. This trial compares two ultrasound-guided nerve blocks, a parasternal intercostal plane block and an erector spinae plane block, both using a mix of bupivacaine and lidocaine. All c…

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

This study tests an experimental oral drug, BMS-986368, for treating muscle stiffness (spasticity) in people with multiple sclerosis. About 200 participants will receive one of three doses of the drug or a placebo. The goal is to see if the drug safely reduces muscle tightness an…

This study tests whether giving a numbing medicine (bupivacaine) as a mist before surgery can lower pain and the need for extra painkillers in children aged 1-7 having cleft palate repair. Half the children get the mist, half get a placebo, and neither the doctors nor families kn…

This early-stage study tests the safety of an inhaled steroid called ciclesonide in 30 premature babies who are on breathing machines and at risk for a chronic lung condition called bronchopulmonary dysplasia. Researchers will closely monitor side effects like high blood sugar, g…

This study tests whether playing soft music or using a musical mobile can reduce pain and stress in infants when chest tubes are removed after heart surgery. Fifty-four babies aged 2 to 12 months will be randomly assigned to hear music, see a musical mobile, or receive standard c…

This trial tests a new enema device designed for people with spina bifida who have poor sphincter tone, which often causes leakage during daily bowel cleanouts. Participants use the device with their regular enema and compare their experience to their usual method. The goal is to…

This study tests a drug called DT402 (a form of MDMA) in 20 adults with autism spectrum disorder. The goal is to see if it can reduce the severity of social and communication problems. Participants will take the drug and report their experiences using simple rating scales. It is …

This study tests a group program to help teens (ages 13-17.5) with Prader-Willi Syndrome and their families learn practical ways to handle irritability, meltdowns, and anxiety. Ten participants will attend weekly 90-minute sessions. The goal is to see if the program improves emot…

This study compares a new radiation technique called lattice radiation therapy (LRT) to standard stereotactic body radiation therapy (SBRT) for people with solid tumors that have spread to soft tissues. The goal is to see if LRT shrinks tumors better and helps the immune system f…

This pilot trial tests a new approach to support cancer patients and their caregivers. The intervention combines social prescriptions—connecting people to community resources—with short online learning modules and regular symptom check-ins. The goal is to see if this package can …

This study tests whether an AI-based website that gives personalized diet tips and recipes can help cancer patients who experience taste changes during therapy. About 198 adults with certain cancers (like multiple myeloma, melanoma, or GI cancers) will either get the AI tool or a…

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 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 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 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 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 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 study tests whether balance rehabilitation that changes how patients see (closing eyes, using a mask, or projecting moving dots) can improve walking stability better than standard rehab with eyes open. It includes 40 adults with chronic nerve damage who have trouble with bal…

This study tests whether psilocybin (the active ingredient in magic mushrooms) can safely reduce depression in adults with COPD, ALS, MS, or atypical Parkinson disease who have at least 6 months to live. Participants receive either two moderate-to-high doses (15mg then 25mg) or t…

This study tests whether using mixed reality (a blend of real and virtual environments) can improve hand strength, coordination, and quality of life in people with multiple sclerosis. Thirty participants will either receive standard physiotherapy or standard therapy plus mixed re…

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 collects moles and blood samples from 50 adults to analyze the genetic mutations that cause moles to form. Researchers will compare DNA from mole tissue with normal DNA from blood to find specific changes. No treatments or interventions are given—the goal is purely to …

This observational study aims to better understand primary mitochondrial myopathy, a rare muscle disease. Researchers will follow 1300 patients and healthy controls, measuring muscle strength, balance, and daily function over time. The goal is to develop and validate tools to tra…

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.…

Researchers are studying whether blood levels of luteinizing hormone (LH) and human chorionic gonadotropin (hCG) are linked to the presence, severity, and long-term outcomes of endocrine disorders such as osteoporosis, thyroid disease, and adrenal gland problems. The study enroll…

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…

Researchers at University Hospital Essen are studying donated tissue from people with hereditary hemorrhagic telangiectasia (HHT), a genetic condition that causes abnormal blood vessel tangles called telangiectasias. The study compares tissue with and without these malformations,…

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 examines how a type of immune cell called gamma delta T cells adapt and function in critically ill patients, both with and without sepsis. Researchers will compare these cells in healthy people, non-septic ICU patients, and septic ICU patients. The goal is to understan…

This study looks at how body awareness (knowing where your body is in space), trunk (core) stability, and arm function are related in people with multiple sclerosis (MS). Researchers will measure these abilities in 50 adults with MS and compare them to healthy volunteers. The goa…

This study works to improve questionnaires that measure pain, daily activities, and physical function for people with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas. Participants aged 5 and older will give feedback on existing surveys through group discussions or…

This study looks at 50 adults with AL amyloidosis who achieved complete remission after initial chemotherapy. It checks if tiny amounts of remaining disease (called minimal residual disease, or MRD) affect organ recovery and survival. Researchers also test whether a stem cell tra…

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

This study tests whether a structured shared decision-making process helps patients with severe aortic stenosis choose between two valve replacement options: TAVR (less invasive) and SAVR (surgery). 140 patients aged 70+ will be randomly assigned to either the structured approach…

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

This study is observing 400 people aged 12 and older who have noncirrhotic portal hypertension (NCPH) or are at risk for it. NCPH is a liver condition that increases blood pressure in the liver's blood vessels without causing cirrhosis, and often has no early symptoms. Researcher…

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…

Researchers at the University of Miami are studying whether advanced eye imaging devices can reveal useful information about the eye in health and disease. The study has two parts: one observes the eyes of healthy people and people with conditions such as multiple sclerosis, dry …

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 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 study looks at facial features of males aged 2 to 40 with creatine transporter deficiency (CTD), a genetic disorder that causes intellectual disability, seizures, and behavioral issues. Researchers will examine photos of participants to see if they share common facial traits…

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 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…

People born with single-ventricle heart disease often have the Fontan operation, which reroutes blood flow but leaves them with reduced exercise capacity. Researchers at Hacettepe University are measuring lung function and breathing muscle strength alongside treadmill exercise te…

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 aims to develop and test new MRI methods for imaging the heart and blood vessels to improve diagnosis and management of heart disease. It will enroll up to 3,400 adults, including both patients with heart conditions and healthy volunteers. Participants will undergo MRI…

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 global study follows people with POLG-related disorders, a group of rare mitochondrial conditions, to understand how the disease progresses over time. By observing clinical changes in up to 300 participants of all ages, researchers aim to identify key milestones that could s…

This study is collecting information from 200 people with cerebral palsy, multiple sclerosis, stroke, fibromyalgia, or similar nerve disorders to see how the EXOPULSE Suit affects their balance and risk of falling over time. Participants will be followed routinely, and researcher…

This study is testing whether smartwatches and blood tests can measure how well children recover after surgery to remove a solid tumor. About 40 children and their caregivers will wear smartwatches and answer questionnaires before surgery and for up to a year after. Researchers w…

Trigonocephaly is a condition where a baby's skull fuses too early along the forehead, giving the head a pointed shape. Doctors often operate to relieve pressure on the growing brain, but some children may do just as well without surgery. This study follows children aged 0 to 8 w…

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 aims to understand why people with Turner syndrome often lose their hearing. Researchers will look at genetic and epigenetic markers in 150 adults aged 18-60. Participants will have hearing, balance, blood, and imaging tests. The goal is to find new ways to treat or de…

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

This study is creating a registry of up to 1,000 people with rare lymphatic system disorders. Researchers will collect information from medical records and yearly surveys to understand how these diseases progress, what treatments are used, and how they affect quality of life. The…

This study follows 400 people with mitochondrial disease and their household members to learn how infections affect them. Researchers will analyze blood samples and health records to find immune patterns linked to severe illness. The goal is to improve care and identify potential…

This study tests a special breastfeeding guideline for infants born with congenital heart disease (CHD). Researchers want to see if the guideline helps more mothers breastfeed directly and reduces stress. About 266 mother-infant pairs will take part across multiple hospitals, wit…

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 …

Researchers at the University of Rochester are testing a program called FMF Connect Pro that trains mental health providers to screen for prenatal alcohol exposure, diagnose fetal alcohol spectrum disorders, and support caregivers through a companion app. The study compares two t…

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 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 follows 450 US adults with Fabry disease who are taking migalastat or enzyme replacement therapy. Researchers will track kidney function, heart and brain events, and quality of life over time to see how well these treatments work in everyday practice.

This study looks at how things in your environment—like pollution, chemicals, diet, and sleep—might affect your hormones and reproductive health. Researchers will follow 300 people aged 8 and older who have conditions like low or high hormone levels, early or late puberty, or inf…

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 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 study uses special MRI scans to measure the flow of cerebrospinal fluid and blood in the brain and spine of people with Chiari malformation. The goal is to see how these flows differ between people with symptoms and those without. Researchers will compare these measurements …

This study is testing a new non-invasive device that uses laser speckle to measure blood flow and structure inside the eye. Researchers will compare these images with standard vision tests in 500 people with various retinal conditions. The goal is to see if this technology can be…

This study is looking for simple ways to detect early signs of multiple sclerosis and Parkinson's disease in people who drive. Researchers will test 200 drivers with these conditions using computer-based attention and reaction tests. The goal is to find quick, easy tests that cou…

This study tests whether digital education or in-person classes help people over 50 with osteoporosis take better care of themselves. Six hundred participants will be split into three groups: one gets standard information, another attends three group sessions with a physiotherapi…

This observational study in Vietnam looks at archived tumor samples from patients with several solid cancers, including liver, colorectal, gastric, breast, lung, endometrial, melanoma, and pancreatic cancer. Researchers will measure the presence of two proteins, ENPP1/cGAS and FA…

This study will measure how much of the drug mirikizumab passes into breast milk in 14 breastfeeding women who take it for inflammatory bowel disease. Participants will provide milk samples over several weeks. The goal is to understand potential exposure to nursing infants, helpi…

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 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…

Researchers are building an international registry to collect health information from people with congenital central hypoventilation syndrome (CCHS), a rare condition that affects breathing control. The study asks patients with a confirmed PHOX2B gene mutation to fill out surveys…

This study is testing whether a mobile health app system can help people with chronic conditions like spinal cord injury, cerebral palsy, spina bifida, or traumatic brain injury improve their self-management skills and wellness. About 160 participants will use the app for one yea…

This study measures thinking, memory, and attention in people with congenital central hypoventilation syndrome (CCHS), a rare condition that can reduce oxygen to the brain. Participants complete a 45-minute app-based cognitive assessment on an iPad, and parents or adult participa…

This study looks at why some adults who had the Fontan procedure as children develop serious liver scarring. Researchers will use blood tests, imaging, and liver biopsies to track changes and find early markers of disease. The goal is to better understand the process and pave the…

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 looks at whether using genetic markers from a child's skin tumor can help doctors decide how much surgery is needed. About 51 children and young adults up to age 25 with localized melanoma or similar growths will have their tumor tissue reviewed centrally. Based on ris…

This study follows about 1,400 people with coronary heart disease who undergo a stent procedure, called PCI, to open blocked arteries. Even with standard treatment, these patients still face a risk of major heart events. Researchers will measure levels of a protein called hsCRP, …

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 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 international registry follows people with alpha thalassemia, a genetic blood disorder, to learn how the disease progresses and how well fetal treatments work. Researchers will track survival to birth, developmental milestones, and health after birth. The goal is to improve …

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 registry will follow 2,500 people who receive Abbott heart devices, such as implants for holes in the heart or replacement heart valves. The goal is to check how safe and effective these devices are when used in everyday hospital practice. Data will be collected from standar…

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 up to 1,000 people with osteogenesis imperfecta, also known as brittle bone disease, a rare condition that causes bones to break easily. Researchers aim to connect each person's genetic cause with their symptoms, disease progression, and response to current tre…

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 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 follows 819 children under 2 years old who have medium to giant congenital nevi (pigmented birthmarks). Researchers will track neurological development, melanoma risk, and quality of life over time. The goal is to create better monitoring and treatment guidelines for t…

This study is creating a registry for children with PTEN Hamartoma Tumor Syndrome (PHTS), a rare genetic condition that increases cancer risk. Researchers will follow 100 participants for 3 years to link genetic changes with health outcomes. The goal is to develop better monitori…

This study follows people with Fanconi anemia, a rare inherited condition that raises the risk of certain cancers, especially squamous cell carcinoma. Researchers will screen 200 participants aged 12 and older every year for up to 10 years using exams, blood tests, and imaging to…

This registry study will follow 2000 people with relapsing multiple sclerosis who are prescribed BRIUMVI (ublituximab-xiiy) in real-world settings. Researchers aim to track safety, effectiveness (like relapse rates), and the overall treatment experience. Participants must not hav…

This study looks at people with severe aortic stenosis, a narrowing of the heart's main valve, who are scheduled for a procedure called TAVI to replace that valve. Researchers want to see if improving blood flow through the valve also reduces fluid buildup in the lungs, a sign of…

This study looks at women who carry the gene for X-linked adrenoleukodystrophy (X-ALD), a rare inherited condition that mainly affects the nervous system. Although the disease is linked to the X chromosome, up to 80% of female carriers may develop symptoms like leg weakness and b…

This study aims to create a network of biobanks across Italy that collect and store biological samples and health data from people with rare diseases and their families. The goal is to standardize how samples are collected and shared, so researchers can access high-quality materi…

This study explores whether smartphone apps and wearable devices can objectively measure fatigue in people with multiple sclerosis (MS) and long COVID, compared to healthy volunteers. Fatigue is often reported through subjective questionnaires, which can be biased. Participants w…

This study looks at whether a standard dose of the antibiotic cefazolin, given before surgery to place a ventricular shunt, reaches high enough levels in the cerebrospinal fluid to prevent infection. Researchers will measure cefazolin in the fluid and blood of 15 adults undergoin…

This study follows 160 people with inflammatory joint disease who are starting treatment with or without glucocorticoids, or receiving a glucocorticoid injection. Researchers will measure bone turnover markers (P1NP and CTX1) in blood samples every 4 weeks and up to 6 months afte…

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

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

This study 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 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 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 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 …

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 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 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 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 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 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 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 is checking how accurate the Dexcom G7 continuous glucose monitor is for patients in the intensive care unit after major abdominal surgery or organ transplant. Researchers will place two sensors on each patient—one that is calibrated and used to guide insulin dosing, a…

This study looks at how endurance exercise (like walking or cycling) and resistance exercise (like lifting weights) affect bone health in older veterans aged 60 and over. Researchers want to see if either type of exercise causes bone breakdown and how the body adapts over time. T…

This study is building a large French database of patients with three rare skin cancers: Merkel cell carcinoma, advanced basal cell carcinoma, and cutaneous adnexal carcinomas. Researchers will track up to 9,000 participants to learn how these cancers behave, what factors affect …

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This study is a registry that collects information from routine care to see how well minimally invasive heart valve procedures work over 5 years. It includes adults over 18 who have had a transcatheter aortic valve replacement or similar treatment. No new treatments are given; th…

This study looks at how different brain regions communicate in women with and without osteoporosis. Researchers will use MRI scans and blood tests to compare brain connectivity and bone markers in 60 postmenopausal women. The goal is to better understand the link between the brai…

This study aims to find genetic factors that may cause cleft lip and palate. Researchers will analyze the DNA of 30 people with the condition using advanced sequencing. The goal is to better understand the genetic roots of this birth defect, which could inform future research.

This study is trying to figure out what makes someone more likely to develop multiple sclerosis (MS). Researchers are looking at a mix of genetic, environmental, and immune factors in 5,000 people in the US, including relatives of MS patients and others at higher risk. The goal i…

This study is for children with Morquio A, a rare genetic disease that affects bones and breathing. Researchers will use safe, non-invasive tests like MRI, X-rays, hearing tests, and walking analysis to track how the disease changes over time. The goal is to better understand the…

This study looks at how common tooth staining is in children who had very high bilirubin levels (jaundice) early in life. Researchers will check 120 children with certain liver conditions to see if their teeth are discolored and whether that affects their daily life. The goal is …

This study looks at how changing the pressure on a ventilator affects the lungs and heart of very premature babies (born before 34 weeks). Researchers will try different pressure levels and use ultrasound and imaging to see which setting works best for breathing and blood flow. T…

This study follows 30 children and young adults with Sanfilippo syndrome type C, a rare genetic disorder that causes severe brain damage. Researchers will measure changes in development and thinking skills over time using standard tests. The goal is to better understand how the d…

This study will explore whether sleep apnea makes multiple sclerosis worse. Researchers will collect sleep studies, MRIs, and lab results from 800 adults with MS to see if there is a link. The goal is to better understand how sleep problems might affect the brain and overall heal…

This study is for people with advanced cancers who are taking long-term targeted treatments. Researchers want to learn about their experiences through interviews. The goal is to find better ways to support these patients. The study is currently recruiting 75 participants.

This study looks at two ways to check for blocked heart arteries in people with severe aortic stenosis who are getting a new heart valve (TAVI). One method is the usual invasive X-ray test; the other uses non-invasive risk management. The goal is to see which approach leads to be…

This study aims to find out how many children with very short height have no known medical cause, and then search for genetic reasons in a small group. Researchers will evaluate 200 children aged 4 to 18 using a team of specialists. The goal is to better understand why some child…