Disorder of development or morphogenesis

This early-stage trial tests a personalized cancer vaccine for people with bladder or stomach cancer who still have tiny amounts of cancer DNA in their blood after surgery. The vaccine is made from unique proteins in each person's tumor and aims to train the immune system to find…

This registry is following 500 adults in Portugal who have severe aortic stenosis and are getting a new type of heart valve replacement called the Medtronic Evolut FX+ TAVI system. The goal is to see if using a standardized procedure makes the treatment safer and more effective. …

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

This study tests a program to help young adults with congenital heart disease transition from pediatric to adult care. The program includes a nurse check-in, a diagnosis summary, an education day, and a handover video call. Researchers will track whether this reduces hospital vis…

This study is for people whose melanoma has come back after standard treatment. Researchers want to see if giving two immunotherapy drugs (ipilimumab and nivolumab) before surgery can shrink the tumor and improve outcomes. About 25 adults will receive two cycles of these drugs, t…

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

This study 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 compares a new heart valve, the DurAVR®, to standard TAVR valves in up to 1650 people with severe aortic stenosis. The valve is designed to mimic natural blood flow. Participants are randomly assigned to get the new valve or a standard one and are followed for up to 10…

This study compares two versions of the drug ocrelizumab given as a shot under the skin in people with multiple sclerosis. The goal is to see if the new test version works the same as the current one. About 182 participants with relapsing or primary progressive MS will take part.

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

This early-phase study tests a new oral drug, RLY-8161, designed to block a specific cancer-driving protein called NRAS. It is for people with advanced melanoma or other solid tumors that have an NRAS mutation and have not responded to standard treatments. The main goals are to f…

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

This study tests 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 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 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 whether clemastine fumarate, a common allergy medicine, can help repair the protective coating around nerves (myelin) in people with multiple sclerosis. About 74 adults with relapsing-remitting MS will receive either the drug or a placebo, and advanced MRI scans …

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

This phase 2 trial tests whether NLY01, a drug similar to those used for diabetes, can slow brain and retinal shrinkage in people with multiple sclerosis. About 120 adults with stable MS will receive either NLY01 or a placebo for 96 weeks. The main goal is to see if the drug redu…

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

This study compares two methods for creating an arteriovenous fistula (AVF) in people with end-stage kidney disease who need dialysis. The newer endovascular method uses a needle through the skin, while the traditional surgical method requires an incision. Researchers will enroll…

This early-stage trial is testing a new oral drug, PF-07799933, in people aged 16 and older with advanced solid tumors that have a specific BRAF gene change. The drug is given alone or combined with other medicines like binimetinib or cetuximab. The study aims to find safe doses …

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

This study tests a new 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 whether giving two immunotherapy drugs (nivolumab and ipilimumab) before eye removal surgery is safe and feasible for people with high-risk uveal melanoma, a rare eye cancer. Fifteen participants will receive the drugs, then have their eye removed. Th…

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

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

This early-stage trial tests a personalized cell therapy for children with recurrent neuroblastoma or newly diagnosed DIPG, two aggressive cancers. The treatment uses the child's own immune cells (dendritic cells and T cells) and stem cells, trained to attack their specific tumor…

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

This early-stage trial tests a new treatment called CC-97540, which uses a patient's own immune cells engineered to target and destroy harmful immune cells. The study includes 120 people with relapsing or progressive multiple sclerosis or refractory myasthenia gravis. The main go…

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

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

This early-stage study tests a new oral drug, DEG6498, in about 100 adults with advanced solid tumors that have not responded to standard treatments. The main goals are to find a safe dose and understand side effects. Participants take the drug once daily and are closely monitore…

This study compares aspirin and clopidogrel to prevent leaflet thrombosis (blood clots on the valve) in 254 people who had a transcatheter aortic valve replacement (TAVR) for severe aortic stenosis. After 4 weeks of both drugs, participants take either aspirin or clopidogrel alon…

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

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

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

This Phase 3 trial tests 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 phase 3 trial tests whether the drug tebentafusp can prevent uveal melanoma from coming back in high-risk patients after their initial treatment. About 290 people with a specific genetic marker (HLA-A*02:01) will either receive weekly infusions of tebentafusp or be observed …

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

This 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 early-phase trial tests a single dose of YTB323, a CAR-T cell therapy, in 28 adults with non-active progressive multiple sclerosis. The study aims to check safety and track disability changes over time. Participants must be 18–60 and able to undergo lumbar punctures and MRIs…

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

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

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

This study tests a new drug called CYH33 in people with rare conditions that cause abnormal tissue growth and blood vessel malformations (PROS and PRVM). The trial has two phases: phase I finds the safest dose, and phase II checks if the drug shrinks growths. About 141 adults and…

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

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

This study tests an experimental treatment called OBX-115 for people with advanced melanoma or non-small cell lung cancer that has spread or stopped responding to standard therapy. OBX-115 uses a patient's own immune cells (tumor-infiltrating lymphocytes) that are modified in the…

This study tests whether removing only the largest affected lymph node (instead of all nearby lymph nodes) after 6 weeks of standard immunotherapy works just as well for stage III melanoma. About 1500 adults with stage IIIB-D melanoma will be randomly assigned to one of the two s…

This study tests whether a drug called enavogliflozin can prevent major heart problems or worsening heart failure in people who have had a heart valve replacement (TAVR) and still have heart failure. About 1,040 participants will receive either the drug or a placebo, in addition …

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

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

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

This 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 follows 30 adults in Taiwan with relapsing-remitting multiple sclerosis who are starting fingolimod (Gilenya), a daily pill that helps control the disease. Over 12 months, researchers will watch for specific side effects like slow heart rate, vision problems, liver iss…

This Phase 3 trial is testing whether a powerful combination of two treatments works better than standard immunotherapy alone for people with advanced melanoma that has not been treated yet. The experimental approach uses lifileucel, a personalized therapy made from a patient's o…

This study tests a new device called EmStop that captures debris during a heart valve replacement procedure (TAVR) for aortic stenosis. About 663 people will be randomly assigned to receive either the EmStop device or a standard device. The goal is to see if EmStop reduces the ri…

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 personalized treatment for advanced melanoma using a patient's own immune cells, called tumor-infiltrating lymphocytes (TIL), which are boosted with a protein called CD40L. After a short course of chemotherapy to make room, the enhanced cells are infused back a…

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

This trial tests whether a new breathing support method called NIV-NAVA helps very preterm infants (born at 23-28 weeks) avoid breathing failure after being taken off a ventilator. About 478 infants will be randomly assigned to receive either NIV-NAVA or standard non-synchronized…

This study tests a new drug called XTX301 in people with advanced solid tumors that have not responded to standard treatments. The trial has two parts: first, finding a safe dose, and then testing that dose in specific cancer types. Researchers will monitor side effects and wheth…

This phase 2 trial is testing whether adding common diabetes drugs (metformin or rosiglitazone) to standard immunotherapy (nivolumab or pembrolizumab) can improve treatment for several advanced solid tumors, including melanoma, lung cancer, and liver cancer. About 72 participants…

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

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

This early-phase trial tests a new drug called BI-1206, given alongside the immunotherapy pembrolizumab, in adults with advanced solid tumors that have stopped responding to prior anti-PD-1/PD-L1 treatment. The study aims to find the safest dose and check for early signs of tumor…

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

This study tests whether using just one Perclose device to close the artery after TAVR is as safe and effective as the usual two devices. Researchers will measure bleeding time and complications in 300 adults undergoing TAVR. The goal is to see if a simpler approach can reduce pr…

This study tests whether a home monitoring program can reduce complications and deaths in infants who had heart surgery. Parents will measure their child's heart rate, oxygen levels, and weight at home and send the data daily via a secure app. The program includes alerts for worr…

This study tests a monthly injection called pelacarsen to see if it can slow the worsening of calcific aortic valve stenosis, a condition where the heart valve becomes stiff and narrow. About 502 adults aged 50-79 with high levels of lipoprotein(a) and mild-to-moderate valve dise…

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

This study tests a personalized vaccine made from a patient's own tumor proteins, given alone or with the immunotherapy drug pembrolizumab. The goal is to see if the combination is safe and helps the immune system attack advanced solid tumors. About 132 people with various advanc…

This phase 3 trial tests an experimental drug called sepofarsen in 32 people with Leber congenital amaurosis (LCA), a rare genetic condition that causes severe vision loss from birth. The drug is injected into one eye, while the other eye gets a placebo, to see if it safely impro…

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 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 early-stage trial is testing a new way to give ocrelizumab (Ocrevus) for multiple sclerosis — as a simple shot under the skin instead of a long IV infusion. About 75 adults with MS will receive increasing doses to see if the new form is safe and tolerable. If it works, it co…

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 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 early-stage trial is testing whether the drug cemiplimab (Libtayo) is safe and effective when given before surgery to remove Merkel cell carcinoma or cutaneous squamous cell carcinoma. About 36 people with stage I–II cancer will receive the drug every three weeks for up to n…

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

This study follows 230 children with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC) who are taking Livmarli, a drug to reduce bile buildup and itching. Researchers will monitor side effects, liver function, and long-term outcomes like need for transplan…

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

This early-stage study tests a new drug called azirkitug, alone or with other cancer medicines, in people with advanced solid tumors like lung, head/neck, and pancreatic cancers. The goal is to find safe doses and check side effects. About 694 adults will take part worldwide, rec…

This study tests an experimental mRNA therapy called mRNA-4359, given alone or with standard immunotherapy drugs, in people with advanced solid tumors (like melanoma, lung cancer, or colorectal cancer). The main goals are to find a safe dose and see if the treatment can shrink tu…

This study looks at whether people with relapsing-remitting multiple sclerosis (RRMS) who are over 40 and have been stable on strong anti-CD20 drugs for at least 3 years can safely switch to milder treatments. The goal is to see if this de-escalation approach prevents disease act…

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 study compares two treatment schedules for postmenopausal women with severe osteoporosis. One group gets romosozumab for 12 months then denosumab for 12 months. The other group cycles between the two drugs every 6 months for 2 years. Researchers will measure bone density and…

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

This study tests whether 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 Phase 3 study tests whether the drug remibrutinib can slow disability worsening in people with secondary progressive multiple sclerosis (SPMS). About 1,275 adults aged 18-65 with SPMS and recent disability progression will receive either remibrutinib or a placebo. The main g…

This early-stage trial tests an experimental drug called SAR445877, alone or with other cancer drugs, in adults with advanced solid tumors that have spread or cannot be removed. The study has two parts: first, finding a safe dose, and second, checking if the drug shrinks tumors. …

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 trial tests a new drug called N17350, a modified enzyme injected directly into tumors, in adults with advanced solid tumors that have not responded to standard treatments. The study aims to find the safest and most effective dose, and to see if the drug can shrink or stop tu…

This phase 2 trial tests a drug called AGA2115 in 48 Chinese adults and adolescents with osteogenesis imperfecta (types I, III, IV), a condition that makes bones brittle. Participants receive one of three dose schedules by injection under the skin. The study checks for side effec…

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 study follows children and teens with osteogenesis imperfecta (brittle bone disease) who previously received romosozumab in an earlier trial. Researchers will monitor 71 participants for side effects over time. The goal is to see if the drug is safe for long-term use in this…

This study tests whether a new combination of drugs (fianlimab and cemiplimab) works better than an already approved treatment (Opdualag) for people with advanced melanoma that cannot be removed by surgery or has spread. About 560 adults with previously untreated melanoma will re…

This study tests whether giving the drug cemiplimab before lymph node surgery can prevent Merkel cell carcinoma from spreading to nearby lymph nodes. About 135 adults with early-stage cancer will receive either the drug or a placebo before their scheduled biopsy. The goal is to s…

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

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

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

This study tests whether 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 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 24-month study tests whether adding umbilical cord stem cells (AdiaVita) to glutathione therapy helps improve autism symptoms in children ages 3-12. About 100 kids will be randomly assigned to get either glutathione alone or glutathione plus stem cell infusions. Parents and …

This early-stage study tests a new drug called CBA-1205 in people with advanced solid tumors (like liver cancer or melanoma) that have not responded to other treatments. The main goal is to check the drug's safety and find the best dose. About 66 participants will receive the dru…

This study looks at whether high-intensity exercise and a high-fiber diet are practical and helpful for people with advanced melanoma who are receiving immunotherapy. About 40 adults will be assigned to exercise, diet changes, both, or standard care. The goal is to see if these l…

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

This study tests whether a caregiver-led telehealth program can help autistic children ages 5 to 12 who still have bowel accidents after completing treatment for bedwetting. About 150 children will take part. The program teaches caregivers strategies to manage encopresis, with th…

This study tests whether different oxygen targets (higher vs. lower) improve outcomes in 39 preterm infants with bronchopulmonary dysplasia and pulmonary hypertension. Each infant will experience both oxygen levels in a crossover design. The goal is to see which level reduces dan…

This study tests whether changes in spinal fluid biomarkers can predict which drug or drug combination works best for people with progressive multiple sclerosis (MS). Participants will take one or two of five drugs (cilostazol, leucovorin, pirfenidone, dantrolene, or pioglitazone…

This study is testing a device called WRAPSODY CIE, a mesh tube placed in veins used for dialysis. The goal is to see if it can keep these veins from narrowing or blocking over time. Researchers will follow 250 people on dialysis for up to 3 years to check how well the device wor…

This early-phase study tests a chemotherapy drug (gemcitabine) given as a mist to breathe in. It is for people whose solid tumors have spread to the lungs and who have no standard treatment options. The main goal is to find the safest dose and watch for side effects. Up to 44 par…

This pilot study tests whether a surgical procedure called lymphovenous bypass (LVB) can prevent lymphedema (chronic swelling) when done at the same time as lymph node removal in melanoma patients. About 20 adults with melanoma who need axillary or inguinal lymph node dissection …

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 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 study tests an oral drug called infigratinib in infants and children under 3 years old with achondroplasia, the most common form of dwarfism. The goal is to see if it safely improves growth and body proportions. The trial has several phases to find the right dose and then co…

This Phase 3 trial is testing a modified dosing schedule of ublituximab (BRIUMVI) in 800 people with relapsing multiple sclerosis. The study has three parts: one open-label part to see if the new regimen reduces brain lesions, a placebo-controlled part to measure drug levels, and…

This phase 3 trial is testing whether atorvastatin, a common cholesterol drug, is safe and can reduce inflammation in children with Kawasaki disease who also have coronary artery abnormalities. The study will enroll 9 children and give them different doses of atorvastatin for 6 w…

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 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 early-phase trial tests a new drug called ADCT-701 in adults with rare cancers like neuroendocrine tumors, adrenal cancer, and malignant peripheral nerve sheath tumors. The goal is to find the safest dose and see if it can shrink tumors or slow disease. Participants receive …

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

This study tests a new drug called PEEL-224, alone or with standard chemotherapy, in children and young adults whose solid tumors have not responded to treatment or have returned. The first part finds the safest dose, and the second part measures how well the drug combination shr…

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

This early-phase study tests a new drug (IOV-3001) designed to boost immune cells in people with advanced melanoma that has not responded to prior treatments. About 42 adults will receive the drug alongside a personalized cell therapy called lifileucel. The main goals are to chec…

This study tests a drug called tebentafusp combined with treatments directed at the liver (like embolization or chemo-embolization) for people with a rare eye cancer (uveal melanoma) that has spread to the liver. About 109 adults with a specific immune type (HLA-A*0201) will part…

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

This Phase 2 trial tests an experimental mRNA therapy called mRNA-1195 in 180 adults aged 18 to 55 with relapsing multiple sclerosis. Participants receive either the mRNA shot or a placebo, and researchers monitor safety and measure new brain lesions on MRI scans over about 2.5 y…

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

This study tests a new drug called riliprubart against standard IVIg treatment in 160 adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder causing weakness and numbness. Participants currently on IVIg will be randomly assigned to receive either r…

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

This early-stage trial is testing a new drug called AB821 in 50 adults with advanced melanoma or other solid tumors that respond to immune therapies. The main goal is to check safety and find the right dose. Participants receive AB821 by IV every 2 or 3 weeks for up to two years.

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

This study compares two different devices (Allegra and Sapien) used to replace a failing surgically implanted aortic heart valve without open-heart surgery. About 104 adults with a deteriorated biological aortic valve will be randomly assigned to get one of the two devices. The m…

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

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

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

This study is testing the drug pembrolizumab in Japanese children with solid tumors or lymphomas, and in Japanese adults with advanced Merkel cell skin cancer. The main goals are to check the drug's safety, how the body processes it, and whether tumors shrink. About 20 participan…

This study follows 180 people who receive the CeraFlex PFO Closure System, a device that closes a small hole in the heart called a patent foramen ovale (PFO). The goal is to see how well the device works in everyday medical practice, especially for patients with migraines or stro…

This study tests a new drug, AZD6750, that helps the immune system fight cancer. It is for adults with advanced solid tumors like melanoma, lung, or breast cancer that have not responded to other treatments. The goal is to find the safest dose and see if it shrinks tumors when us…

This study tests a drug called riliprubart in 140 adults with chronic inflammatory demyelinating polyneuropathy (CIDP) that hasn't improved with standard care. Participants receive either riliprubart or a placebo for up to 111 weeks. The main goal is to see if the drug reduces di…

This Phase 3 trial tests a one-time cell therapy called IMA203 for people with advanced melanoma that has not responded to prior treatment. IMA203 is made from a patient's own immune cells and is designed to target cancer cells. The study compares IMA203 to standard treatments li…

This study tests a drug called tebentafusp in 19 people with a rare eye cancer (uveal melanoma) that is too large to remove without taking the whole eye. The goal is to see if the drug can shrink the tumor by at least 20% so that less invasive surgery might be possible. Participa…

This study tests a new drug, [212Pb]VMT01, that delivers a tiny dose of radiation directly to melanoma cells. It is being tested alone or with the immunotherapy nivolumab in 300 people with advanced melanoma that has worsened after prior treatment. The goal is to find safe doses …

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

This study is checking how well a device called the Cera Vascular Plug works and if it is safe. The plug is used to block blood flow in certain blood vessels to treat conditions like aneurysms, abnormal connections between arteries and veins, and other problems. About 132 adults …

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 study tests whether placing a small balloon in the uterus for 7-10 days after septum removal surgery can prevent internal scar tissue (adhesions). 320 women will be randomly assigned to receive the balloon plus hormones or hormones alone. The main goal is to see if the ballo…

This study tests a new treatment for people with stage IV melanoma that has spread to the brain. It combines a chemotherapy drug (temozolomide) with specially grown immune cells (NK cells) from a healthy donor. The goal is to see if this combination is safe and can shrink brain t…

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

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

This study looks at whether getting standard immunotherapy in the morning (before 11 am) or afternoon (after 12 pm) changes how well it fights advanced or metastatic solid tumors. Researchers think the body's internal clock (circadian rhythm) might affect immune activity. About 1…

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 early-stage trial tests a radioactive drug that seeks out and delivers radiation directly to cancer cells with a specific protein (somatostatin receptor). It is for adults with advanced lung, kidney, head and neck, digestive tract, or adrenal gland tumors that cannot be remo…

This phase 2 trial tests a new approach for children and young adults with relapsed or hard-to-treat neuroblastoma. Patients receive donor immune cells (NK cells) along with standard chemotherapy and an antibody therapy. The goal is to see if this combination can shrink tumors or…

This trial tests a one-time gene therapy called UX111 for children with Sanfilippo A, a rare genetic disorder that causes severe brain damage. The therapy delivers a working copy of the missing gene to cells. Researchers will measure whether it reduces harmful substances in the b…

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

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

This phase 2 trial is testing a drug called ONC206 in 90 people with advanced pheochromocytoma or paraganglioma, rare tumors of the adrenal glands. Participants must have tumors that cannot be removed by surgery and have not responded to or cannot have standard treatments. The st…

This phase 2 trial tests the drug avapritinib in 50 adults with advanced solid tumors that have mutations in the CKIT or PDGFRA genes. The drug blocks enzymes that help cancer cells grow. The main goal is to see how many patients' tumors shrink or disappear. Researchers will also…

This early-phase study tests whether dapagliflozin, a diabetes medicine, is safe for children with brain or solid tumors that have come back or not responded to other treatments. About 20 children will receive the drug alongside standard care. The goal is to check for side effect…

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

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

This Phase 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 early-phase study tests whether adding chemotherapy directly into the spinal fluid (intrathecal topotecan) followed by maintenance pills is feasible for children under 6 with high-risk embryonal brain tumors. About 15 children will receive this treatment after standard high-…

This study looks at whether taking a standard immunotherapy drug (PD-1 inhibitor) on an intermittent schedule works as well as taking it continuously for people with advanced melanoma that cannot be removed by surgery. About 614 participants will be randomly assigned to one of th…

This study tests a new drug called IDE196 in people with certain cancers that have specific genetic mutations (GNAQ/11 or PRKC fusions). The cancers include metastatic uveal melanoma, cutaneous melanoma, and colorectal cancer. The trial has two phases: first to find safe doses, t…

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 early-phase trial is testing a new drug called enzelkitug, alone or with standard immunotherapies, in people with advanced solid tumors that have not responded to other treatments. The main goals are to check safety, find the right dose, and see if the drug shrinks tumors. A…

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

This study looks at ways to prevent bone loss when older adults lose weight. It will test if a combination of resistance exercise and a bone-strengthening drug (alendronate) can protect bone density. About 900 overweight or obese older adults with low bone mass will take part for…

This phase II trial is testing whether combining two drugs, binimetinib and imatinib, can shrink tumors in people with advanced KIT-mutant melanoma that cannot be removed by surgery. The study will enroll 25 participants with stage III or IV melanoma. The goal is to see how many …

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

This early study tests whether giving an IL-17 blocker (secukinumab) alongside restarting immunotherapy can lower the risk of severe immune-related side effects in people with advanced melanoma. Only 4 participants who previously had side effects like colitis or rash will be enro…

This program provides access to two experimental drugs, doxecitine and doxribtimine, for people with thymine kinase 2 deficiency (TK2d), a rare genetic disorder that can cause severe muscle weakness and early death. It is for children and adults who have a confirmed TK2 gene muta…

This study is for people with high-risk melanoma who have tiny bits of tumor DNA still in their blood after surgery. It compares a two-drug immunotherapy combo against a single drug to see which better clears these hidden cancer cells. About 54 participants will be randomly assig…

This Phase II trial tests a bivalent vaccine (with an immune booster) plus two different schedules of oral β-glucan in 94 people with high-risk neuroblastoma who are in complete remission. The goal is to see which schedule triggers stronger antibodies against the cancer, potentia…

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 program provides the experimental drug ulixertinib to people with advanced solid tumors that have certain gene mutations (MAPK pathway). It is for patients who have not responded well to or have run out of standard treatments. The goal is to control the disease and possibly …

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

This phase II trial tests a combination of four drugs—chidamide, dinutuximab beta, irinotecan, and temozolomide—in 27 children whose neuroblastoma has returned or not responded to standard treatment. The goal is to see how well the regimen controls the cancer and to monitor side …

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 is testing a combination of two drugs—a targeted radioactive drug (177Lu-DOTATATE) and a PARP inhibitor (olaparib)—in children and teens aged 3 to 18 with certain solid tumors that have come back or not responded to treatment. The goal is to see if the combination is s…

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

This study tests whether the drug nipocalimab can delay or prevent relapses in adults with CIDP, a rare nerve disorder that causes weakness and numbness. Participants first receive nipocalimab, and those who improve are then randomly assigned to continue the drug or switch to a p…

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 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 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 montelukast, an asthma drug, in 28 premature infants at high risk for bronchopulmonary dysplasia (BPD), a serious lung condition. Researchers want to see if the drug is safe and how it moves through the body. The infants receive montelukast or a placebo daily for…

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

This study is testing a new type of artificial lens, called the Clareon TruPlus, for people having cataract surgery. The lens is placed inside the eye to replace the cloudy natural lens and help restore clear vision. About 122 adults with cataracts and astigmatism will receive th…

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 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 pembrolizumab, a drug that helps the immune system fight cancer, in children aged 6 months to 18 years with advanced melanoma, solid tumors, or lymphoma that has spread, returned, or not responded to other treatments. The main goal is to see if the drug can shrin…

This early-phase trial is testing a new drug called IACS-6274, either alone or combined with other cancer drugs, in 54 adults with advanced solid tumors (such as melanoma, ovarian, or head and neck cancers). The main goal is to find the safest dose and understand side effects. Th…

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…

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

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

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

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

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

This study looks at a new needle that glows under special light to help surgeons find and sample very small, high-risk breast lesions during surgery. The goal is to see if this method gives more accurate and faster results compared to standard techniques. The study involves 300 w…

This study tests a new glowing needle that helps doctors find and sample very small abnormal areas inside breast ducts during surgery. The needle uses a special coating that lights up, making it easier to see and remove the right tissue. Researchers will compare how well this met…

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

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

This study tests whether a new type of PET scan using a radioactive tracer called 18F-FCH can more accurately locate overactive parathyroid glands or tumors before surgery. About 193 adults scheduled for parathyroid removal will receive both the new scans and a standard 4D-CT sca…

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

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

This study tests whether adding folic acid to iodized salt can raise folate levels in women of childbearing age in Zambia, a country without mandatory folic acid fortification. Two hundred fifty non-pregnant, non-lactating women aged 18–45 will use the fortified salt instead of r…

This study tests different combinations of an online program called CARRII, designed to help Native American women reduce their risk of alcohol-exposed pregnancy. Over 500 participants will be randomly assigned to one of eight versions of the program for three months. The goal is…

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

This study tests whether people with multiple sclerosis can learn to prevent falls by practicing on a treadmill that unexpectedly slips or trips. 64 participants aged 45 and older will be split into two groups: one gets the slip-and-trip training, the other just walks on the trea…

This phase 3 trial tests whether two different eye treatments can prevent vision loss in people who have had radiation therapy for a type of eye cancer called uveal melanoma. The study will compare faricimab injections given every 3 months, a fluocinolone acetonide implant placed…

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

This study tests an investigational drug called zelpultide alfa to see if it can prevent bronchopulmonary dysplasia (BPD), a serious chronic lung disease, in extremely premature babies (born between 22 and 27 weeks). About 366 high-risk infants will receive either the drug or a p…

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

This study tests whether exercise paired with simple mental tasks (like counting backwards) can improve walking, balance, and thinking in people with multiple sclerosis. Forty adults with MS will do supervised workouts twice a week for 8 weeks. The goal is to find better rehab st…

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

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

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

This study is testing a smartphone app called MedlyPeds that lets children with heart disease send their weight, blood pressure, and heart rate to their doctors from home. The app also asks about symptoms and sends personalized tips. The goal is to see if the app is easy to use a…

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 whether a 16-week behavioral therapy called Pivotal Response Treatment (PRT) works better when done at a center or at home for young children with autism who have language delays. Researchers will compare both groups to children receiving usual care. The goal is …

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

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

This study tests a new manual standing wheelchair designed for children with spina bifida. Thirty kids aged 8 to 17 will try the chair in lab and simulated home, school, and community settings. The goal is to see if it helps them move and stand better than their current wheelchai…

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

This study compares two surgical techniques—perforator flap versus skin graft—for repairing the wound left after removing a stage II melanoma. Researchers want to see which method leads to faster wound healing and better quality of life. Forty adults with melanoma on the trunk, a…

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

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

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

This study follows 350 people with stage III-IV melanoma during their first two years after diagnosis to understand how their quality of life and mental health change. It also tests whether cognitive behavioral therapy for cancer distress (CBT-C) can help reduce stress, improve s…

This study tests a 5-week online program called WE BEAT that teaches teens with congenital heart disease skills to handle stress and bounce back from tough times. About 390 teens aged 12-17 will be randomly assigned to the program or usual care, and researchers will measure chang…

This study tests a medical device called VCFix to treat broken vertebrae (spinal bones) from compression fractures. The goal is to reduce pain and improve movement in 103 adults aged 21-85 with a single recent fracture. The device is placed into the bone to restore height and sta…

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

This study tests if the Ulthera System, a non-invasive ultrasound device, can safely improve sagging skin on the lower face, under the chin, and neck. About 218 adults with mild to moderate skin laxity will receive treatment or no treatment, and results will be measured at 90 day…

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

This study tests whether Pilates, a gentle mind-body exercise, can help manage pain and other symptoms in people with hypermobile Ehlers-Danlos syndrome or hypermobility spectrum disorder. One hundred adults with persistent pain will be randomly assigned to either Pilates classes…

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 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 compares giving the cancer drug nivolumab (Opdivo) as a shot at home versus in a clinic for people with various advanced cancers. The goal is to see if home treatment improves patients' overall care experience and quality of life. About 50 adults will receive the drug …

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 compares two dental devices—the Leaf expander and the Hyrax expander—in 30 children aged 6 to 12 who have a crossbite (when upper teeth sit inside lower teeth). The main goal is to see which expander causes less pain during the first week. Researchers will also measure…

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

This study tests whether teaching new mothers how to do acupressure on themselves can help them breastfeed exclusively for longer. 360 mothers will either get standard care or also learn acupressure within 48 hours after giving birth. Researchers will track breastfeeding rates at…

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

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

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

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

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

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

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

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

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

This study 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 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 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 follows 450 babies born with serious heart defects to find early signs of developmental delays by 6 months of age. Researchers want to understand why over half of these children face learning, thinking, or behavior challenges later in life. The goal is to identify risk…

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

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

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

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

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

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

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

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

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

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

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

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

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

This study will follow 300 children with idiopathic short stature (ISS) — meaning they are shorter than expected for no known medical reason. Researchers will measure their height, weight, and other health factors over time to create a detailed picture of natural growth. The goal…

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 aims to understand how reproductive disorders, such as delayed or early puberty, are inherited. Researchers will collect blood samples and medical histories from 850 people with these conditions and their family members. No treatment is provided; the goal is to identif…

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 observational study follows 150 people with MEHMO syndrome or related conditions to better understand how the disease progresses. Researchers will collect medical history, imaging, and lab samples to find biological markers that could help monitor the disease. No treatment i…

This study collects information from 200 adults in Russia who have neurofibromatosis type 1 (NF1) with plexiform neurofibromas. Researchers will look at patients' age, symptoms, and medical history to better understand the disease. No new treatments are being tested; the goal is …

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 is a long-term registry that collects health information from people with Fabry disease, a rare genetic disorder. It does not test any new treatments; instead, it follows up to 9,000 patients worldwide to better understand how the disease progresses and how current tre…

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

This study tracks 70 skin cancer patients to see how long it takes to get insurance approval for off-label neoadjuvant therapy (treatment before surgery). Researchers will measure approval times, out-of-pocket costs, and whether delays lead to worse surgical outcomes. The goal is…

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 pilot study looks at how different types and intensities of exercise affect muscle recovery in people taking statins compared to those not on statins. Researchers will test cycling and resistance training at various levels, measuring muscle damage markers and pain sensitivit…

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

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

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

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

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

This study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

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 is testing whether monthly blood tests for a nerve damage marker (neurofilament light chain) can detect hidden disease activity in people with relapsing-remitting multiple sclerosis (MS) earlier than standard yearly MRI scans. The goal is to reduce the need for gadolin…

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

This study from Mayo Clinic is looking at how T cells (a type of immune cell) change during anti-PD-1 immunotherapy in people with advanced melanoma, lung cancer, or other solid tumors. Researchers will collect blood and tissue samples from 500 participants to measure specific ma…

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 enrolls 400 adolescents and young adults (ages 18-39) recently diagnosed with various cancers. Participants are randomly assigned to either choose which health-related questions they answer or receive a fixed set of questions. The goal is to see if giving patients a ch…

This study is observing 55 children and adults in Russia who have hypophosphatasia (HPP), a rare genetic bone disease. Researchers will track symptoms, lab results, and quality of life to learn how the disease naturally progresses. No treatments are being tested—the goal is to ga…

This study looks at how to reduce the long and frustrating journey to a diagnosis for people with developmental abnormalities. Researchers will review past cases, collect new blood or skin samples, and use advanced genetic testing. The goal is to understand why some people remain…

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 how much of the multiple sclerosis drug BRIUMVI gets into breast milk. About 16 breastfeeding women with relapsing MS will provide milk samples after their regular treatment. The goal is to understand drug exposure for nursing infants, not to test a new treatm…

This registry study follows pregnant women with multiple sclerosis (MS) who have taken BRIUMVI, comparing them to those who haven't taken the drug. The goal is to see if BRIUMVI increases the risk of major birth defects. Researchers will enroll about 728 participants across two g…

This study follows about 50,000 people who survived childhood cancer (diagnosed before age 21 between 1970 and 1999) and 10,000 of their siblings. Researchers want to understand how cancer treatments like radiation, chemotherapy, and surgery affect health years later. By comparin…

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

This study tracks 770 adults with advanced solid cancers (like lung, head and neck, melanoma, or bladder cancer) who are receiving immunotherapy. Researchers collect tumor and blood samples over time to find biological markers that predict how well the treatment works or causes s…

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

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

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

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

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

This 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 aims to find the genetic causes of Ollier disease and Maffucci syndrome, rare disorders that cause cartilage tumors and increase cancer risk. Researchers will study 100 people aged 2 and older using blood tests, scans, and genetic sequencing. Participants will stay at …

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

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

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

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

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

This study looks at whether special breathing exercises can help the brains of people with multiple sclerosis or cancer, as well as their caregivers. Researchers will use MRI scans to see how breathing affects oxygen flow in the brain. The goal is to understand the connection bet…

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 monitors up to 50 people with MPS VII (Sly Syndrome) to understand how the disease changes over time. It also checks the long-term safety and effectiveness of the drug vestronidase alfa. Participants may or may not be taking the drug, and the study involves regular che…

This study looks at whether women with Ehlers-Danlos syndrome (EDS) experience more severe period pain than those without the condition. Researchers will compare pain levels and other gynecological issues in 156 women using an online questionnaire. The goal is to better understan…

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

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

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

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

This study follows 160 people with relapsing multiple sclerosis in Greece who are already taking ofatumumab (Kesimpta®) as part of their routine care. Researchers will track how many patients have no signs of disease activity—like relapses, new brain lesions, or worsening disabil…

This study looks at whether smartwatches can help monitor chronic inflammatory demyelinating polyneuropathy (CIDP), a rare nerve disease that causes weakness and numbness. 35 adults with CIDP who use subcutaneous immunoglobulin therapy at home will wear a smartwatch for 12 months…

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

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

This study is creating a large collection of blood and tissue samples from people with skin cancer and healthy volunteers. Researchers will store these samples and medical data to support future studies on skin cancer. The goal is to provide a valuable resource for translational …

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 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 is testing a device called the AlgometRx Nociometer to see if it can measure pain and other abnormal sensations in people with neurofibromatosis type 1 (NF1). The device uses a small electrode on a finger or toe to send painless signals and a camera to record changes i…

This study aims to collect DNA samples and detailed eye exam data from 1,000 people with rare inherited eye diseases like aniridia, Best disease, and albinism. Participants provide a saliva or blood sample and share their eye health records. The goal is to expand a research repos…

This study tests a new type of MRI that looks at energy use in the brain to see if it can predict how well multiple sclerosis (MS) patients will respond to a common therapy. Researchers will scan 40 adults with relapsing-remitting MS before and after they start treatment. The goa…

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 collects blood, bone marrow, and other samples along with health information from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze the participants' genes to find new genetic causes of these conditions and understa…

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

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

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

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

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

This study looks at how a pregnant woman's body handles caffeine and how much of it reaches her unborn baby. Researchers will give a small dose of caffeine to 30 women at risk of preterm delivery before a planned C-section. Blood samples from the mother, placenta, and newborn wil…

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

This study 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 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 looks at how well a non-invasive navigation system helps surgeons place the hip cup during total hip replacement. About 118 people with hip conditions like arthritis or hip dysplasia will take part. The goal is to see if the actual cup position matches the planned posi…

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

This pilot study tests whether a radioactive tracer called 89Zr-Df-crefmirlimab can help PET scans detect certain immune cells (CD8+ T cells) in the brain and spinal cord of people with multiple sclerosis (MS) or progressive multifocal leukoencephalopathy (PML). About 15 adults w…

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 follows 250 adults with multiple sclerosis (or similar conditions) for years, using yearly MRI scans and other tests to track brain and spinal cord changes. After a participant passes away, researchers examine donated brain and spinal cord tissue to see how the MRI fin…

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

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

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

This study is testing a robotic system called Epione to help doctors place needles or instruments into the bones of the pelvis and spine. The goal is to see if the robot makes these procedures more feasible, safe, and accurate compared to doing them by hand. Sixty adults who are …

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

This study uses a one-time survey to learn about the eye and body symptoms of people with congenital aniridia, a rare genetic condition that affects the iris. Researchers want to see how much patients understand about their own disease. About 100 patients of all ages will take pa…

This study looks at 600 people getting a new heart valve through a tube in the leg (TAVI). Doctors want to know if using a second tube in the arm (radial artery) instead of the groin (femoral artery) causes fewer bleeding or blood vessel problems. The goal is to find the safest w…

This study is testing whether an advanced MRI technique called Quantitative Susceptibility Mapping (QSM) can better tell apart brain lesions caused by multiple sclerosis (MS) from those due to normal aging or small vessel disease. Researchers will scan 1,000 people aged 50-70 wit…

This observational study looks at how sleep quality influences cognitive flexibility, physical activity, body awareness, and reaction time in people with multiple sclerosis. Researchers will compare those with good and poor sleep using questionnaires, computer tests, and a wearab…

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

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

This study is not testing a new drug. Instead, it will observe 800 people with relapsing multiple sclerosis in Germany who are already taking approved injectable or oral medications. Researchers will track how long patients stick with their prescribed treatment over up to four ye…

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