Disorder of development or morphogenesis

This study looks at the long-term safety of a daily pill called lucerastat for adults with Fabry disease, a rare genetic disorder. About 107 people who completed a previous study will take the drug and be monitored for side effects over several years. The goal is to see if lucera…

This phase II trial tests pembrolizumab, an immunotherapy drug, in 57 patients with desmoplastic melanoma, a rare skin cancer. The drug helps the immune system attack cancer cells. The study includes patients whose tumors can be surgically removed (cohort A) and those whose canno…

This phase 2a trial tests a nasal spray called foralumab in 54 people with non-active secondary progressive multiple sclerosis (SPMS). The spray contains an antibody that aims to calm overactive immune cells in the brain. Participants will receive either a low dose, a high dose, …

This phase 2 trial tests whether a daily pill called miransertib can slow or stop the abnormal tissue overgrowth seen in Proteus syndrome, a rare genetic condition. About 38 people aged 3 and older will take the drug for up to 4 years. Researchers will measure changes in foot ove…

This phase 3 trial tests whether the immunotherapy drug ipilimumab is better than high-dose interferon alfa-2b at preventing melanoma from coming back in patients whose cancer was removed by surgery. Over 1,600 people with high-risk stage III or IV melanoma took part. The study m…

This study follows 165 children who previously took dabrafenib and/or trametinib for certain brain tumors. Researchers want to see if these drugs are safe over the long term and how they affect growth. The children continue their treatment and are monitored for side effects and d…

This phase III trial compares two sequences of treatment for advanced melanoma with a BRAF mutation. One group starts with immunotherapy (ipilimumab and nivolumab) followed by targeted therapy (dabrafenib and trametinib) if needed; the other group starts with targeted therapy the…

This phase II trial tests whether adding a growth factor (GM-CSF) to the immunotherapy drug ipilimumab helps people with advanced melanoma live longer. About 245 patients with stage III or IV melanoma that cannot be surgically removed are randomly assigned to get ipilimumab alone…

This study tests whether adding a drug called sargramostim to the standard immunotherapy combination of nivolumab and ipilimumab helps people with advanced melanoma live longer. About 600 patients with stage III or IV melanoma that cannot be surgically removed are participating. …

This study tests a new drug called ARV-806 in adults with advanced solid tumors that have a specific KRAS G12D mutation. The drug aims to break down the mutated protein that helps tumors grow. The trial has two parts: first, finding a safe dose in small groups, then testing that …

This phase 2 trial tests whether adding tuvusertib (a drug that blocks a DNA repair enzyme) to avelumab (an immunotherapy) can slow cancer growth in people with advanced Merkel cell cancer that didn't respond to prior treatment. About 50 participants will receive either the combi…

This phase III trial compares the drug pembrolizumab (MK-3475) to the current standard treatments—high-dose interferon or ipilimumab—in 1,301 patients with high-risk melanoma that has been surgically removed. The goal is to see if pembrolizumab is better at preventing the cancer …

This phase 2 trial compares two ways of taking the targeted drugs dabrafenib and trametinib for advanced BRAF-mutant melanoma: continuously versus with planned breaks. About 280 people with stage III or IV melanoma that cannot be surgically removed are participating. The goal is …

This phase II trial studies whether giving the immunotherapy drug pembrolizumab both before and after surgery is more effective than giving it only after surgery for patients with stage III-IV high-risk melanoma. About 313 participants will be randomly assigned to one of two trea…

This study investigates whether combining the medication lovastatin with intensive reading tutoring can improve reading abilities in children and young adults (ages 8-20) with Neurofibromatosis Type 1 (NF1) who have reading disabilities. Participants first receive either lovastat…

This early-stage study tests a new drug called belvarafenib, alone or with other drugs (cobimetinib and nivolumab), in people with advanced melanoma that has a specific NRAS gene change and has stopped responding to standard immunotherapy. The main goals are to check safety and f…

This study tests whether injecting botulinum toxin (Botox) into fat pads around the heart's veins during cardiac surgery can prevent atrial fibrillation (AFib), a common and serious complication. About 220 adults undergoing heart surgery will receive either Botox or a placebo inj…

This phase 2 trial tested the drug ivosidenib in children and young adults (ages 1 to 21) with solid tumors, lymphomas, or histiocytic disorders that have an IDH1 mutation and have either come back after treatment or not responded. The goal was to see if the drug could shrink or …

This early-stage trial is testing a new type of cell therapy for children with neuroblastoma that has returned or is hard to treat. The therapy uses the patient's own immune cells, which are modified in the lab to recognize and attack a protein called PHOX2B found on neuroblastom…

This study tests a behavioral program called Individualized Developmental Care (NIDCAP) for newborns with complex congenital heart disease. The program aims to reduce stress from the hospital environment by supporting parent involvement, creating a soothing setting, and adjusting…

This trial investigates whether adding metformin, a common diabetes drug, to the immunotherapy pembrolizumab (Keytruda) can improve treatment for people with advanced melanoma. The study focuses on reversing metabolic problems inside tumors that may weaken immune cells. Participa…

This trial investigates whether combining atezolizumab (a drug that boosts the immune system) with bevacizumab (a drug that blocks blood vessel growth) can shrink tumors in people with advanced melanoma that cannot be removed by surgery or has spread. The study focuses on patient…

This early-phase trial is testing whether the immunotherapy drug nivolumab can help people with melanoma or lung cancer that has spread to the leptomeninges (the lining of the brain and spinal cord). The drug is given both through a vein and directly into the spinal fluid via a l…

This study aimed to test a finger prosthetic system called Point Mini in 5 children aged 5-15 with partial hand loss. The device uses ratcheting mechanical digits to help with gripping and lifting tasks. However, the trial was withdrawn before any participants were enrolled, so n…

This study tests a new oral drug called IPN01194 in 36 adults with advanced solid tumours (melanoma, colorectal, pancreatic, or head and neck cancer) that have not responded to standard treatments. The goal is to find the right dose and check if it is safe, while also looking for…

This study tests a combination of two drugs, neratinib and valproate, in people with advanced solid tumors that have not responded to standard treatments. The first part finds the safest dose, and the second part focuses on cancers with a RAS mutation, like certain colon and panc…

This study tests a new device called the DurAVR™ THV System, a replacement heart valve placed through a small tube in an artery, for people with severe aortic stenosis (a narrowed heart valve). The trial involves 150 participants and aims to see if the device is safe and works we…

This early study tests a new device called the DurAVR™ heart valve in 15 people aged 65 and older with severe aortic stenosis, a condition where the heart's aortic valve becomes narrowed. The valve is implanted through a catheter (TAVR) without open-heart surgery. The main goals …

This study tests whether a robotic exoskeleton called ReWalk can help people with complete spinal cord injury below T4 walk and improve their health. Fifty participants will either use the exoskeleton or receive standard physical therapy. The goal is to see if the device improves…

This phase 3 study tests whether mitapivat, an oral medication, can safely reduce the number of blood transfusions needed by adults with transfusion-dependent alpha- or beta-thalassemia. Participants receive either mitapivat or a placebo for 48 weeks. The main goal is to see if m…

This phase 3 trial is testing whether the immunotherapy drug nivolumab (Opdivo) can prevent melanoma from coming back in people who have had their stage IIB/C melanoma completely removed. About 790 adults and children are randomly assigned to receive either nivolumab or a placebo…

This study tests a daily cream called tapinarof, already used for psoriasis, in 6 adults with palmoplantar keratoderma — a condition that causes painful thickening of skin on the palms and soles. Researchers want to see if the cream can clear or improve the skin and check for sid…

This phase II trial tests whether adding cabozantinib to the immunotherapy drug nivolumab can prevent mucosal melanoma from returning after surgery. About 101 patients with resected mucosal melanoma will receive either nivolumab alone or nivolumab plus cabozantinib. The study aim…

This phase 3 study tests whether venglustat, an experimental oral drug, can slow heart thickening better than current standard treatments in 104 adults with Fabry disease. Participants are randomly assigned to venglustat or usual care (enzyme replacement or migalastat) for 18 mon…

This study aimed to test a new treatment called CABA-201, a CAR T-cell therapy made from a patient's own immune cells, for people with multiple sclerosis (MS). The goal was to see if it was safe and could control the disease by targeting and resetting the immune system. However, …

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

This Phase 3 study compares fenebrutinib, an experimental oral drug, to teriflunomide, an approved MS therapy, in 751 adults with relapsing multiple sclerosis. The goal is to see if fenebrutinib better reduces relapse rates and delays disability progression. Participants take eit…

This Phase II study tested a combination of two drugs—peginterferon alfa-2b and temozolomide—in 29 children with high-risk melanoma that had spread or come back. The main goal was to see how well the tumors shrank and to check for side effects. The study also looked at how long c…

This study tests a drug called entrectinib in children with advanced solid tumors or brain tumors that have come back or haven't responded to other treatments. The trial has two parts: first finding the best dose, then seeing how well it works in children whose tumors have specif…

This Phase 2 trial tests whether combining bevacizumab (Avastin) with atezolizumab (Tecentriq), with or without cobimetinib (Cotellic), can shrink or control melanoma that has spread to the brain. The study enrolls 29 adults with untreated brain metastases who are not taking ster…

This study tests a yearly injection called Debio 4326 in 56 children with central precocious puberty, a condition where puberty starts too early (before age 8 in girls, 9 in boys). The goal is to see if it can safely suppress puberty hormones back to childhood levels for a full y…

This large study compared two treatments for severe aortic stenosis (a narrowed heart valve) in 1,746 patients at intermediate risk for surgery. One group got a less invasive procedure called TAVI (a new valve inserted through a tube in the artery), and the other had open-heart s…

This phase 3 trial compares two drugs for primary progressive multiple sclerosis (PPMS), a form of MS that steadily worsens. About 985 adults with PPMS will receive either daily oral fenebrutinib or intravenous ocrelizumab (plus matching placebos) to see which better delays disab…

This study planned to test a new CAR-T cell therapy called LCAR-AIO in adults with relapsed or treatment-resistant neurological autoimmune diseases, including multiple sclerosis and myasthenia gravis. The goal was to check safety and find the right dose. However, the trial was wi…

This study tests whether combining an experimental drug (GSK2636771) with the immunotherapy pembrolizumab (Keytruda) can help control metastatic melanoma in patients whose tumors have lost a protein called PTEN. The trial involves up to 41 adults with advanced melanoma that hasn'…

This trial tests an experimental drug called TRK-950 in combination with several standard anti-cancer treatments for people with advanced solid tumors, including colorectal, bladder, ovarian, and other cancers. The main goal is to find safe doses and understand side effects. Part…

This Phase 3 trial tests whether the oral drug remibrutinib works better than teriflunomide at reducing relapses and slowing disability in people with relapsing multiple sclerosis. About 1,000 adults aged 18–55 with active MS are taking part. The study is double-blind, meaning ne…

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

This study looks at children with congenital heart disease who take the blood thinner rivaroxaban after a Fontan procedure. The goal is to see how often serious or minor bleeding happens. Researchers will collect data from regular doctor visits for up to 2 years. No extra tests o…

This study tests an investigational drug called rozanolixizumab in adults with MOG antibody-associated disease (MOGAD), a rare condition where the immune system attacks the brain and spinal cord. The main goal is to see if the drug can prevent or delay relapses (flare-ups) compar…

This early-stage trial tests a new antibody, BAY 3375968, designed to help the immune system fight advanced solid tumors. It works by reducing regulatory T cells that can suppress immune activity. The study will test the drug alone and combined with pembrolizumab in about 129 peo…

This phase 3 trial tests an experimental drug called ALXN1850 in 124 adolescents and adults with hypophosphatasia, a rare genetic bone disease. Participants receive either the drug or a placebo by injection under the skin. The main goal is to see if the drug improves walking dist…

This phase II trial tests two drugs—olaparib and pembrolizumab—together in 12 people with advanced uveal melanoma, a rare eye cancer that has spread. The goal is to see if the combination can shrink tumors or slow the disease. The study is active but no longer recruiting, and res…

This trial tests a fracture liaison service (FLS) — a coordinated care program for people aged 50 and older who have had a hip or spine fracture. The program follows best-practice standards to ensure patients receive bone density tests and appropriate osteoporosis management. Res…

This study tests a drug called erdafitinib in children and young adults (ages 1 to 21) whose cancers have come back or are not responding to treatment and have specific changes in FGFR genes. The drug works by blocking enzymes that help cancer cells grow. The main goal is to see …

This study tests a new device called VCFix for treating a broken bone in the spine (vertebral compression fracture). Ten adults with a single recent fracture will receive the implant to see if the procedure is safe and works as intended. The goal is to gather early data to plan a…

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

This phase 3 trial tests whether a shot (subcutaneous) of ublituximab works as well as the standard IV infusion for people with relapsing multiple sclerosis. About 360 participants are enrolled. The main goal is to compare drug levels in the blood over 24 weeks, along with safety…

This study tested two drug combinations in 42 people with advanced uveal melanoma, a rare eye cancer that has spread. The goal was to see if adding a second drug (GSK2141795) to trametinib could better slow cancer growth. Participants were randomly assigned to receive either tram…

This early-phase study tests the safety and best dose of a drug called veliparib when given with two chemotherapy drugs (carboplatin and paclitaxel) in people with advanced solid tumors that have spread or can't be removed. It includes patients who also have liver or kidney probl…

This study tests a stem cell transplant method for people with acquired or inherited bone marrow failure. Donor stem cells are specially processed to remove certain immune cells, which may lower the risk of graft rejection and graft-versus-host disease. The goal is to see if this…

This study looks at whether a one-year treatment with Ocrevus can improve walking and balance in people with relapsing multiple sclerosis, compared to other standard treatments. Sixty adults aged 18-65 who can walk at least 25 feet will be split into two groups. Their walking abi…

This early-stage study tests whether a mix of immune-boosting drugs and targeted radiation can help the body fight advanced solid tumors that cannot be removed by surgery. About 14 adults with certain types of skin cancer, sarcoma, or breast cancer will receive the treatment dire…

This study tests a combination of three drugs—dabrafenib, trametinib, and navitoclax—in people with BRAF mutant melanoma or other solid tumors that have spread or can't be removed by surgery. The goal is to find the best dose and see how well the drugs shrink tumors. About 75 adu…

This phase II trial tests whether intensive blood pressure management (targeting systolic BP below 120 mmHg) is better than standard care (below 140 mmHg) for patients with advanced kidney or thyroid cancer starting anti-angiogenic TKI therapy. The study enrolls 61 participants a…

This study is a long-term follow-up for people with relapsing multiple sclerosis who previously completed a ublituximab trial. It aims to see if the drug remains safe and effective over time. About 1100 participants will receive ublituximab infusions, and researchers will track r…

This phase 2 trial is testing a combination of two immunotherapy drugs, nivolumab and ipilimumab, in 798 people with over 50 types of rare cancers. The goal is to see if these drugs can shrink tumors by helping the immune system attack cancer cells. Participants receive the drugs…

This study tests two different tendon grafts (kneecap tendon vs. quadriceps tendon) for ACL reconstruction, with or without an extra stabilizing procedure called lateral extra-articular tenodesis (LET). The goal is to see which approach best prevents graft failure in 1,272 young,…

This study tests a drug called ulixertinib in children and teens whose advanced cancers have a specific genetic change in the MAPK pathway. The goal is to see if the drug can shrink or control the tumor. About 20 participants with various solid tumors, lymphomas, or related disor…

This study looks at whether doctors can safely stop standard anti-PD-1 immunotherapy early in people with advanced melanoma that cannot be surgically removed. By using PET/CT scans and tumor biopsies as guides, the goal is to reduce treatment side effects and burden without harmi…

This phase 2 trial is testing the drug larotrectinib in children and young adults (ages 1 to 21) with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have a specific genetic change called an NTRK fusion. The cancers have either come back after treatment…

This Phase 2 trial tests whether giving a mix of targeted drugs and immunotherapy before and after surgery can reduce the risk of melanoma returning in people with high-risk, resectable tumors. About 95 participants will receive vemurafenib, cobimetinib, and atezolizumab in diffe…

This early-stage study tests whether a mix of three drugs—tacrolimus, nivolumab, and ipilimumab—can shrink or control certain skin cancers in people who have had a kidney transplant. The main goal is to see if the cancer responds without causing the body to reject the transplante…

This study tested the drug ensartinib in 13 children and young adults (ages 1–21) whose cancers had come back or did not respond to treatment and had specific ALK or ROS1 gene changes. The goal was to see if the drug could shrink or stop tumor growth. While the drug targets cance…

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

This study tests a liquid medicine called mirabegron in children aged 6 months to 3 years who have bladder problems due to nerve damage (neurogenic detrusor overactivity). The goal is to see if the medicine helps the bladder hold more urine safely, reducing leaks and pressure. Ch…

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

This study tests a service designed to help people with osteoporosis take their medications as prescribed. Participants receive education, reminders, and regular follow-up calls. The goal is to see if this support improves medication adherence and reduces fracture risk over 18 mo…

This phase 3 trial tests whether the immunotherapy drug avelumab can keep Merkel cell carcinoma from returning in patients whose cancer has spread to lymph nodes and who have already had surgery or radiation. About 100 adults will receive either avelumab or a placebo every two we…

This early-stage study tests a combination of two drugs, avutometinib and defactinib, in children and young adults (ages 3 to 30) with advanced or recurrent solid tumors that have specific genetic changes. The main goal is to find the safest dose with the fewest side effects. The…

This phase 3 study tests setrusumab, a monthly IV infusion, in 6 Japanese children with osteogenesis imperfecta (types I, III, or IV), a condition that causes fragile bones and frequent fractures. The main goal is to see if the drug lowers the number of fractures, including spine…

This trial studies nearly 1,000 people with severe aortic stenosis and blocked coronary arteries who need both a valve replacement (TAVI) and artery stenting (PCI). Researchers want to see if doing the stenting before or after the valve replacement leads to fewer deaths, heart at…

This early-phase study tests a new drug, RO7247669, that targets two immune system checkpoints (PD-1 and LAG-3) to help the body fight cancer. About 170 adults with advanced solid tumors (including melanoma, lung cancer, and esophageal cancer) who have run out of standard options…

This study tests whether adding hydroxychloroquine (a malaria drug) to standard immunotherapy (nivolumab with or without ipilimumab) can help shrink advanced melanoma tumors. About 41 adults with stage III or IV melanoma that cannot be removed by surgery are participating. The go…

This study tests whether infliximab, a drug used for inflammatory conditions, can treat colitis (colon inflammation) caused by immune checkpoint inhibitors in people with stage III or IV skin cancer. About 42 participants will receive either infliximab or steroids, and researcher…

This phase 2 trial tests a combination of two drugs—naxitamab (an antibody) and GM-CSF (an immune booster)—in children and adults with high-risk neuroblastoma that has not responded well to previous treatments. The goal is to see if this therapy can shrink or eliminate tumors in …

This study compares the standard fixed-interval dosing of ocrelizumab (every 24 weeks) with a personalized approach where infusions are given only when B cell levels rise above a certain threshold. The goal is to see if the personalized schedule is just as good at preventing rela…

This study was designed to test two immunotherapy drugs, nivolumab and ipilimumab, in children and young adults with cancers that returned or didn't respond to treatment and had many genetic mutations. The goal was to see if the drug combination was safe and could shrink tumors. …

This study tests if a new growth hormone medicine (somapacitan) given once a week works as well as the standard daily growth hormone (Norditropin) for children who are very short due to being born small, or having Turner syndrome, Noonan syndrome, or unknown causes. About 412 chi…

This study looks at the long-term safety and effects of an experimental drug called DNL310 for people with Hunter syndrome (MPS II), a rare genetic disorder. About 99 participants who completed earlier studies will receive the drug for up to 5 years. Researchers will monitor side…

This study tests an oral drug called DFMO in children and young adults with high-risk neuroblastoma who are currently in remission. The goal is to see if taking DFMO for two years can prevent the cancer from coming back. The trial involves 441 participants and focuses on safety a…

This early-phase trial tests several new drug combinations in people with advanced solid tumors (like stomach, ovarian, or liver cancer) that have spread and stopped responding to standard treatments. The goal is to find the safest dose and see if the drugs can help control the c…

This phase II trial is testing two different three-drug combinations in people with advanced BRAF-mutated melanoma that has spread or cannot be removed by surgery. One group receives nivolumab (immunotherapy) plus dabrafenib and trametinib (targeted therapy); the other receives n…

This phase 2 trial tests the drug cabozantinib in children and young adults with rare cancers like sarcomas, Wilms tumor, and others that have come back or not responded to treatment. Cabozantinib works by blocking enzymes that help tumors grow and form blood vessels. The study a…

This study is a long-term extension of earlier trials testing the drug riliprubart in adults with CIDP, a condition where the immune system attacks the nerves, causing weakness and numbness. About 300 participants who completed a prior riliprubart study will continue receiving th…

This phase 3 trial tests whether rituximab, a cheaper drug, works as well as ocrelizumab for active multiple sclerosis. About 600 adults with relapsing or progressive MS will receive one of the two drugs. The main goal is to see if rituximab can prevent new brain lesions as effec…

This study tests a tumor vaccine combined with daily low-dose chemotherapy for children whose high-risk neuroblastoma has returned or not responded to standard treatment. The vaccine is designed to train the immune system to recognize and attack neuroblastoma cells, while the low…

This study tests whether combining two drugs, DFMO and etoposide, can help children and young adults (up to age 30) whose neuroblastoma has come back or not responded to standard treatment. About 131 participants will receive the drug combination to see if it delays cancer growth…

This study looks at whether the drug ofatumumab can slow down thinning of the retina (the light-sensitive layer at the back of the eye) in people with relapsing-remitting multiple sclerosis (MS). Researchers will follow 75 adults with MS who are either starting or already taking …

This study tests whether a new drug, ABP 692, works as well as the approved drug Ocrevus for people with relapsing-remitting multiple sclerosis (RRMS). About 152 adults with RRMS will receive either ABP 692 or Ocrevus to compare how the drugs move through the body and how well th…

This study tests a new drug called Cetrelimab (JNJ-63723283) in 234 people with advanced solid tumors like lung, melanoma, bladder, and colorectal cancers. The drug works by blocking PD-1, a protein that stops the immune system from attacking cancer cells. The trial has two parts…

This study tests whether a daily fingolimod capsule works better than a weekly interferon injection to prevent multiple sclerosis relapses in children. About 240 kids with MS will take one of the two treatments for 2 years, followed by a 5-year extension where everyone gets fingo…

This early-phase trial tests an experimental drug called HFB200603, either alone or with the immunotherapy tislelizumab, in 83 adults with advanced solid tumors (kidney, lung, melanoma, stomach, or colon cancer). The main goal is to find a safe dose and check for side effects. Re…

This phase 3 trial tests whether satralizumab can prevent relapses in people with MOGAD, a rare disease where the immune system attacks the brain and spinal cord. About 152 participants aged 12 and older will receive either the drug or a placebo. The main goal is to see if satral…

This study looks at whether adding the drug abaloparatide (Tymlos) to the standard treatment denosumab (Prolia) can improve bone density more than denosumab alone in postmenopausal women with osteoporosis. Seventy women who have already been on denosumab will either continue with…

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

This study tests a next-generation heart valve (Navitor) in 333 people with severe aortic stenosis who are too high-risk for open-heart surgery. The goal is to see if the valve is safe and works well at 30 days, focusing on survival and reducing leaks around the valve. The valve …

This early-phase trial is testing whether taking high-dose vitamin B5 (pantothenic acid) can help standard immunotherapy work better for people with advanced melanoma that cannot be surgically removed. The study includes 12 participants who have not had prior treatment for their …

This early-phase study tests a new oral drug called E7386 in about 60 adults with advanced solid tumors (like desmoid tumors) that have not responded to other treatments. The main goals are to find the safest dose and check for side effects. Researchers will also watch for any tu…

This Phase 3 study tests a new drug called JR-141 against the current standard treatment (idursulfase) in 86 people with Hunter syndrome (MPS II). The goal is to see if JR-141 can better reduce harmful substances in the brain and improve thinking skills. Participants can switch t…

This study aimed to see if romosozumab (Evenity) could improve bone density and muscle mass in postmenopausal women with osteoporosis undergoing lumbar spine surgery. Participants would have received monthly injections of romosozumab or weekly alendronate pills for 12 months. How…

This study tests whether adding focused radiation (stereotactic radiosurgery) to a combination of targeted drugs (binimetinib and encorafenib) and an immunotherapy (pembrolizumab) can better control melanoma that has spread to the brain. It includes 10 adults with a specific gene…

This early-stage study tests a new treatment called TAR-210 in 5 Japanese patients with a type of bladder cancer that has specific genetic changes (FGFR mutations). The treatment is placed directly into the bladder to deliver the drug erdafitinib. The main goal is to see if it is…

This study tests a gene therapy called RGX-121 in children aged 4 months to 5 years with Hunter syndrome, a rare genetic disease that affects the brain and body. The therapy delivers a working copy of the missing gene to the central nervous system. Researchers will measure improv…

This early-phase study tests a personalized cell therapy for people with certain cancers (bladder, melanoma, head/neck, ovarian, lung, esophageal, stomach, or sarcoma) whose tumors have a specific marker called MAGE-A4. The treatment involves taking a patient's own immune cells, …

This early-phase trial tests an experimental antibody called GEN1042, given alone or with the cancer drug pembrolizumab (and sometimes chemotherapy), in people with advanced solid tumors like lung, colon, melanoma, head and neck, or pancreatic cancer. The main goals are to check …

This study tests whether adding two experimental vaccines (IO102/IO103) to the standard drug combination of nivolumab and relatlimab can shrink or control advanced melanoma that cannot be removed by surgery. About 43 adults with untreated stage III or IV melanoma will receive the…

This study looks at how well the Harmony Transcatheter Pulmonary Valve (TPV) works when used by doctors in everyday practice. It involves 174 people with congenital heart disease who need a new pulmonary valve. The main goal is to see if the valve works properly without needing a…

This early-phase trial tests whether giving stem cells (from a donor) during heart-lung bypass surgery is safe for infants under 6 months with certain congenital heart defects. Seventeen infants will receive the cells while undergoing heart repair. The main goal is to check for s…

This phase 2 study tests a personalized immune cell therapy called tumor-infiltrating lymphocytes (TIL) in 20 people with advanced solid cancers (like stomach, colon, or pancreatic cancer) that have not responded to standard treatments. Patients first receive chemotherapy to prep…

This phase III trial tests whether the immunotherapy drug pembrolizumab can prevent Merkel cell cancer from returning after it has been surgically removed. About 280 adults with stage I-III Merkel cell cancer are randomly assigned to receive either pembrolizumab or standard obser…

This phase 3 trial compares a subcutaneous (under-the-skin) injection of the drug combination nivolumab and relatlimab to the standard intravenous (IV) infusion in people with advanced melanoma that cannot be removed by surgery. The study aims to see if the shot delivers enough d…

This trial tests a vaccine designed to help the immune system recognize and attack neuroblastoma, a type of childhood cancer. The vaccine contains two antigens found on neuroblastoma cells, plus an adjuvant to boost the immune response. Participants also take an oral supplement c…

This phase 2 trial tests whether a patient's own immune cells, called tumor-infiltrating lymphocytes (TIL), can shrink tumors in people with metastatic uveal melanoma, a rare and aggressive eye cancer. Participants receive chemotherapy to prepare their immune system, then an infu…

This study tests whether giving three rounds of very strong chemotherapy with stem cell rescue works better than one round for children under 10 with high-risk brain tumors. The goal is to improve survival without adding too many side effects. About 250 children will take part.

This study compares two types of TAVR devices (self-expanding vs. balloon-expandable) in over 1,100 people with severe aortic stenosis and a small aortic annulus. The goal is to see which device leads to fewer deaths, strokes, or hospitalizations, and which performs better over t…

This study follows 150 people with long-chain fatty acid oxidation disorders (LC-FAOD) to check the long-term safety of their treatment, including for pregnant women and their babies. Researchers track serious side effects and disease complications. The goal is to better understa…

This phase II trial is testing the drug dinaciclib in 72 people with stage IV melanoma that has spread. The drug works by blocking enzymes that help cancer cells grow. Researchers are looking at side effects and whether it helps patients live longer or slows tumor growth.

This study tests a special tube called a stent graft to repair damage in the upper part of the main artery from the heart (the ascending aorta). It is for people with bulges (aneurysms), tears (dissections), or other lesions that usually need open-heart surgery. The goal is to se…

This phase 3 trial tests whether CYB704, a proposed biosimilar to the MS drug Ocrevus, works the same way in the body. About 183 people with relapsing multiple sclerosis will receive either CYB704 or Ocrevus. Researchers will compare drug levels, MRI scans, and side effects to se…

This study follows about 200 people with relapsing-remitting multiple sclerosis (RRMS) who are starting ozanimod (Zeposia®) for the first time. Researchers want to see how long patients continue the medication in everyday practice and how well it controls their disease. The study…

This Phase 3 trial tests remibrutinib, an oral tablet, against teriflunomide in 1000 adults with relapsing multiple sclerosis. The goal is to see if remibrutinib better reduces yearly relapse rates and delays disability. Participants will then have the option to continue remibrut…

This study tests whether a two-drug combination can help the immune system attack ovarian cancer that has returned after standard treatments. The drugs are E7777, which removes certain immune cells that block the body's defenses, and pembrolizumab, which reactivates cancer-fighti…

This study tested two different doses of the immunotherapy drug ipilimumab, either alone or combined with high-dose interferon, in 88 people with advanced melanoma that could not be surgically removed. The goal was to see if the combination could delay cancer growth better than i…

This study looks at whether people with relapsing-remitting MS who have not had a relapse in the past year but have high levels of a nerve damage marker (NfL) in their blood would benefit from switching to the drug ofatumumab. About 136 participants will either switch to ofatumum…

This study tests a new drug called ALXN1850 in children aged 2 to 12 with hypophosphatasia, a rare genetic condition that weakens bones. The trial compares the drug to a placebo to see if it improves bone health and movement. About 30 children who have not received prior treatmen…

This pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…

This study compares a new biosimilar drug (GME751) to the standard immunotherapy Keytruda in 322 adults with stage II or III melanoma that has been surgically removed. The goal is to see if the biosimilar stays in the body and works as well as Keytruda in preventing the cancer fr…

This early-phase trial tests the safety of combining two cancer drugs, nivolumab and cabozantinib, in people with HIV who have advanced solid tumors. Only 8 participants were enrolled. The goal is to see if the combination is safe and can shrink or stabilize tumors.

This study planned to test a new surgical approach for people with pulmonary vein stenosis, a condition where the veins carrying blood from the lungs to the heart become narrowed. The procedure involved transplanting a donor's left atrium and pulmonary veins, followed by lifelong…

This study tests the long-term safety and effectiveness of pegtibatinase in people with classical homocystinuria (HCU), a rare genetic disorder that prevents the body from breaking down certain amino acids. About 100 participants who completed earlier studies will receive the dru…

This early-stage trial is testing an experimental drug called AMG 355, given alone or together with the immunotherapy Keytruda, in 77 adults with advanced solid tumors (like lung, colon, stomach, or melanoma) that have not responded to standard treatments. The main goals are to c…

This study tests a drug called trametinib in children with a rare blood cancer (juvenile myelomonocytic leukemia) that has returned or not responded to treatment. The drug works by blocking certain enzymes that help cancer cells grow. The goal is to see if it can shrink or contro…

This phase 2 study tests the drug entrectinib in over 500 people with advanced solid tumors that have specific gene changes (NTRK, ROS1, or ALK fusions). The goal is to see if the drug can shrink tumors and control the disease. Participants have various cancer types, including lu…

This study follows about 1,900 people with relapsing multiple sclerosis who have already taken ofatumumab in a previous Novartis trial. Participants continue receiving ofatumumab injections every 4 weeks to monitor long-term safety, side effects, and how well the drug controls re…

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

This study tests a drug called romosozumab against standard bone-strengthening medicines (bisphosphonates) in children and teens with osteogenesis imperfecta, a condition that causes fragile bones and frequent fractures. The goal is to see if romosozumab can reduce the number of …

This study tests if Prolia can improve bone density in people whose bones have weakened from taking steroids (glucocorticoids). About 102 adults in China will receive the drug and be monitored for 12 months. The main goal is to see if bone mass in the lower spine increases.

This study tests a drug called tipifarnib in children and young adults (ages 1 to 21) whose cancers have a specific change in the HRAS gene. The cancers are advanced or have come back after treatment. The goal is to see if tipifarnib can shrink tumors or stop them from growing. A…

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

This early-phase trial is testing whether a ketogenic diet (low carbs, high fat) can safely help immunotherapy drugs work better in people with advanced melanoma or kidney cancer. About 60 participants will follow the diet while receiving standard immunotherapy. The main goals ar…

This study is for children with hypochondroplasia, a condition that causes short stature and bone differences. Researchers are testing a drug called infigratinib to see if it is safe and helps improve growth over the long term. Participants must have already completed a previous …

This Phase 2 trial tests JK07, a lab-made protein designed to help heart cells repair and pump better. About 282 adults with chronic heart failure will receive either JK07 or a placebo. The study looks at safety and whether JK07 can improve heart function in two groups: those wit…

This study tests a new device called the Occlutech Flex II PFO Occluder, which is used to close a small hole in the heart (PFO) that can cause strokes. The trial involves 450 people who have had a stroke with no clear cause and a PFO. Researchers want to see if the new device wor…

This study tests a non-invasive device that uses low-frequency electromagnetic fields to improve communication between immune cells. 120 adults with lupus, rheumatoid arthritis, or multiple sclerosis will receive either active or sham sessions three times a week for 12 weeks. The…

This study tests whether adding telitacicept to standard therapy helps adults with refractory chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve condition that causes weakness and numbness. About 76 participants will receive either standard care alone or standard c…

This Phase 1 trial compares a new drug called RPH-035 to the approved MS drug Ocrevus. The study involves 180 adults with relapsing-remitting or secondary progressive multiple sclerosis who have flare-ups. Researchers are checking if RPH-035 behaves similarly in the body and has …

This study looks at whether taking selumetinib with a low-fat meal reduces stomach problems in teenagers with neurofibromatosis type 1 (NF1) who have tumors that cannot be removed by surgery. About 24 teens will take the drug under both fed and fasted conditions to compare drug l…

This study tests whether a less invasive heart valve replacement (TAVR) is safe and effective for people with moderate aortic stenosis, a condition where the heart's aortic valve is partially blocked. About 650 participants will receive the TAVR device and be monitored for compli…

This study compares two procedures for severe aortic valve stenosis: transcatheter aortic valve implantation (TAVI), a less invasive approach, and standard open-heart surgery. It will follow 4,000 high-risk patients for up to 24 months to see which method leads to fewer deaths, h…

This early-stage trial tests a new approach for children and young adults with neuroblastoma that has come back or not responded to standard treatments. Doctors take a patient's own immune cells (T-cells), genetically modify them in the lab to recognize and attack a protein calle…

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

This phase 2 trial studies a combination of two immunotherapy drugs—talimogene laherparepvec (T-VEC) and pembrolizumab—in 43 adults with advanced melanoma that has worsened despite prior anti-PD1/L1 therapy. T-VEC is a modified virus that infects and kills cancer cells while also…

This Phase 2 trial tests fenebrutinib, an oral drug, in 109 people with relapsing multiple sclerosis. Participants receive either the drug or a placebo for 12 weeks, and researchers measure new brain lesions on MRI scans. The goal is to see if fenebrutinib can reduce disease acti…

This phase 2 trial tests whether giving selpercatinib before standard surgery can shrink tumors in people with RET-altered thyroid cancer. About 30 participants with advanced or recurrent disease will receive the drug and then undergo surgery. The goal is to see if the drug impro…

This study compares two daily pills—fenebrutinib and teriflunomide—in about 746 adults with relapsing multiple sclerosis. The goal is to see which one better reduces relapses and delays worsening disability. Participants take one of the two drugs for several years, with an option…

This study compares two ways to place stents in the left main coronary artery when there is a blockage at the opening of a branch. One method places a stent only in the blocked branch, while the other extends a stent from the main artery into the branch. Researchers will track 10…

This study tests whether a Fracture Liaison Service (FLS) helps people aged 50 and older who have had a hip or spine fracture. Half of the 400 participants get the FLS, which includes bone health checks, medication guidance, diet and exercise advice, and regular phone follow-ups …

This phase 2 trial tests whether adding infliximab (a drug used for arthritis) to standard immunotherapy (pembrolizumab or nivolumab+relatlimab) can prevent severe immune-related side effects in people with advanced melanoma. About 36 patients who have not had prior treatment wil…

This study tests a personalized treatment for age-related osteoporosis using the drug teriparatide. Researchers aim to match treatment to each patient's bone turnover rate using simple blood tests, avoiding the need for painful bone biopsies. The trial involves 60 adults aged 45 …

This study tests a small device called the BLOOM Micro-Occluder to close a common heart hole (PDA) in premature babies. About 55 babies weighing between 600 and 2500 grams will be enrolled. The goal is to see if the device is safe and works well over 6 months.

This study tests two newer multiple sclerosis drugs, ofatumumab and siponimod, against an older one, fingolimod, in 129 children aged 10 to 17. The goal is to see if the newer drugs are at least as good at preventing MS relapses. Participants take one of the three drugs for two y…

This study tests a new drug called DAY101 (tovorafenib) against standard chemotherapy for children and young adults with a type of brain tumor called low-grade glioma that has a specific gene change (RAF alteration). The goal is to see if the new drug works better at shrinking tu…

This early-phase trial tests a combination of two drugs—pembrolizumab (an immunotherapy that helps the immune system fight cancer) and ziv-aflibercept (which cuts off blood supply to tumors)—in people with advanced solid tumors like melanoma, colorectal, ovarian, and kidney cance…

This study compares two ways to replace a narrowed heart valve in people with severe aortic stenosis who are at intermediate risk for surgery. About 1,400 participants will be randomly assigned to get either a less invasive TAVI procedure or open-heart surgery (SAVR). The main go…

This phase 1 trial is testing a new treatment called GD2CART for children and young adults with osteosarcoma or neuroblastoma that has returned or does not respond to standard treatments. GD2CART is made by taking a patient's own T cells (a type of immune cell), adding a new gene…

This study tests a drug called selumetinib in 32 Chinese children and adults with neurofibromatosis type 1 (NF1) who have nerve tumors that cannot be surgically removed. The goal is to check the drug's safety and how well it works at shrinking these tumors. Participants take the …

This study tests whether adding an experimental drug (CMP-001) to the immunotherapy pembrolizumab can better shrink melanoma tumors before surgery and help prevent them from coming back. About 60 adults with stage III or recurrent melanoma that can be surgically removed will rece…

This study tests a drug called selumetinib in children and young adults with low grade glioma that has come back or not responded to other treatments. The drug works by blocking certain enzymes that help tumor cells grow. The goal is to find the best dose and see if it can shrink…

This phase 3 trial compares the experimental drug frexalimab to the approved drug teriflunomide in about 1,655 adults with relapsing multiple sclerosis. The main goal is to see if frexalimab reduces the number of relapses more effectively. Participants receive either frexalimab i…

This study tests a new drug called ensartinib in people with advanced melanoma that has a specific genetic abnormality (ALK). The goal is to see if the drug can shrink tumors or stop them from growing for at least 24 weeks. Participants must be 18 or older and have already tried …

This study tests a new long-acting growth hormone (lonapegsomatropin) given once a week, compared to the standard daily growth hormone shot, in 48 prepubertal children with Turner syndrome who have not had growth hormone before. The goal is to see if the weekly shot is safe and h…

This study tests an experimental drug called frexalimab in people with a type of progressive multiple sclerosis (MS) that is no longer causing relapses. The goal is to see if the drug can delay worsening disability compared to a placebo. About 943 adults aged 18 to 60 are taking …

This study was designed to see if hyperbaric oxygen therapy or computerized cognitive training could help adults with Fetal Alcohol Spectrum Disorder (FASD) think and function better. It planned to enroll 0 participants and was withdrawn before starting, so no results are availab…

This study tests whether taking teriparatide (a bone-building drug) for one month before lumbar fusion surgery helps surgeons place screws more securely in patients with osteoporosis. About 192 participants will be randomly assigned to receive either the drug or no pretreatment. …

This study tests a custom-made drug for one child with a rare, severe brain disease called CONDBA, caused by a specific gene mutation. The drug aims to slow or stop brain damage by targeting the faulty gene. Researchers will track changes in movement, coordination, and quality of…

This study tests whether propranolol, a drug often used for high blood pressure, can safely treat chylothorax—a buildup of lymphatic fluid—in children after open heart surgery. About 50 children aged 7 days to 18 years with congenital heart disease will receive either propranolol…

This study tests whether adding olaparib to the standard chemotherapy temozolomide helps control advanced pheochromocytoma or paraganglioma—rare neuroendocrine tumors that have spread or can't be removed surgically. About 46 adults with these cancers will be randomly assigned to …

This study was designed to test the safety and tolerability of a new drug called JK07 in people with heart failure and a specific type of high blood pressure in the lungs. It planned to enroll about 20 to 30 adults aged 18 to 85. However, the study was withdrawn before any partic…

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

This early-phase study tests a flavored, drinkable version of the chemotherapy drug irinotecan (VAL-413) combined with another drug (temozolomide) in children and young adults up to age 30 whose solid tumors have come back. The main goal is to find the safest dose and see how the…

This study looks at whether actively treating a heart condition called patent ductus arteriosus (PDA) in premature babies is better than simply monitoring it. About 482 infants born between 22 and 28 weeks of pregnancy will be followed to see which approach reduces the risk of de…

This study looks at whether taking teriparatide (Forteo) for 24 months can improve bone structure in 100 men and women with osteoporosis. Researchers will use a special CT scan to see changes in bone thickness and microarchitecture at the wrist and shin. The goal is to understand…

This study tests a new maintenance dosing schedule for ofatumumab, a drug used to control relapsing multiple sclerosis (MS). Researchers want to see if the new schedule is as safe and effective as the current approved dose. About 196 adults with relapsing MS will take part, and t…

This study is testing a new combination of two immunotherapy drugs (BGB-A445 and tislelizumab) in about 113 people with advanced bladder, kidney, or skin cancer that has spread. The goal is to see if the combo can shrink tumors and how safe it is. Participants must have already t…

This Phase 2 trial tests whether a combination of three drugs—dasatinib, quercetin, and nicotinamide riboside—can improve bone health in 120 adults aged 60-90 with osteopenia or osteoporosis. The goal is to see if these 'senolytic' drugs can clear aging cells from the skeleton an…

This study looks at how teriparatide, a bone-building drug for severe osteoporosis, affects bone structure in postmenopausal women. Researchers will use a special scanner to see changes in bone thickness and tiny connecting rods at the wrist and ankle over 24 months. The goal is …

This study tests an experimental drug called KL590586, taken as a capsule, in people with advanced solid tumors that have specific RET gene changes. The trial has two phases: Phase I checks safety and the right dose, while Phase II looks at how well the drug shrinks tumors. Up to…

This study tests whether a less invasive procedure called TAVR (replacing the aortic valve through a tube in an artery) is as safe and effective as open-heart surgery for people with severe aortic stenosis who are otherwise healthy enough for surgery. About 2,200 participants wil…

This study looks at whether adding rubber bands (elastics) to standard clear plastic retainers helps keep an underbite correction stable after orthodontic treatment. Researchers want to see if this simple addition can reduce the need for future jaw surgery. The trial involves 42 …

This early-phase trial tests an investigational drug called ABBV-706, alone or combined with other drugs, in about 288 adults with advanced solid tumors like small cell lung cancer, high-grade brain tumors, and neuroendocrine cancers. The main goals are to check safety, find the …

This study tests a special balloon coated with the drug paclitaxel to treat blockages in arm fistulas used for dialysis. The balloon is inflated to open the narrowed blood vessel and release the drug to help keep it open. Researchers will check if the fistula stays open without n…

This study tests a new device called the Navitor TAVI system to replace a stiff, narrowed heart valve without open-heart surgery. It includes 1,500 people with severe aortic stenosis who are at low or intermediate risk for surgery. The goal is to see if the device is safe and eff…

This study compares a single-stage surgery using implants coated with an antibiotic-loaded hydrogel to the standard two-stage surgery for treating chronic hip prosthesis infections. The single-stage approach aims to remove the infected implant and place a new one in the same oper…

This study compares a new medicine, ALXN1850, to the current standard treatment (asfotase alfa) in 43 children aged 2 to 12 with hypophosphatasia, a rare genetic condition that weakens bones. All children have been on the standard treatment for at least 6 months before joining. T…

This pilot study tested whether adding low-dose radiation (brachytherapy) to standard immunotherapy could help shrink tumors in people with advanced melanoma, kidney cancer, or urothelial cancer. Only 5 participants were enrolled, and the main goal was to see how many responded t…

This study tested a new device called the ACURATE neo2, which replaces a narrowed heart valve without open-heart surgery. About 1,900 people with severe aortic stenosis received either the new device or a standard one. The goal was to see if the new device is safe and works as we…

This phase 2 trial tests whether bevacizumab, a drug that blocks blood vessel growth, can reduce chronic bleeding and iron-deficiency anemia in people with hereditary hemorrhagic telangiectasia (HHT). HHT causes abnormal blood vessels that bleed easily. The study involves 33 adul…

This study tests a drug called mirdametinib in 114 adults and children with a genetic condition called NF1 that causes nerve tumors (plexiform neurofibromas) that cannot be removed by surgery and cause serious problems. The drug works by blocking a protein that helps tumors grow.…

This early study is testing a new device called the DurAVR™ THV System, which is a replacement heart valve placed through a small tube in the leg (TAVI procedure). The study includes 40 people with severe aortic stenosis or a failed surgical valve. The main goal is to see if the …

This phase 2 trial tests a procedure called transarterial chemoembolization (TACE) for people with uveal melanoma that has spread to the liver. Doctors inject a chemotherapy drug (carmustine) mixed with an oily contrast agent (Lipiodol) directly into the liver's blood supply, the…

This study is testing a less invasive procedure called TAVR to replace the aortic valve in 150 people who have a bicuspid aortic valve (a valve with two flaps instead of three) and severe narrowing. The goal is to see if it is safe and works well for people who are at low risk fo…

This study is testing a less invasive procedure using stent-grafts to treat various types of aortic disease, including aneurysms and dissections. It involves 170 participants who are considered too high-risk for traditional open surgery. The goal is to see if this endovascular ap…

This study tests two ways to surgically repair spina bifida in the womb using a tiny camera. The goal is to close the spinal defect and reverse brain changes caused by the condition. About 110 pregnant women carrying babies with spina bifida will take part. The two methods are co…

This study tests a personalized approach to dosing two immunotherapy drugs (ipilimumab and nivolumab) for people with advanced melanoma. Instead of giving everyone four doses, doctors will check after two doses: patients who show early benefit may stop, while those who don't can …

This phase 3 trial tests whether the oral drug masitinib can slow disability worsening in people with progressive multiple sclerosis who do not have relapses. About 800 participants will receive either masitinib or a placebo for 96 weeks. The main goal is to see if masitinib dela…

This early-phase trial is testing whether adding an experimental drug called VX15/2503 to standard immunotherapies (ipilimumab or nivolumab) can help treat stage IIIB-D melanoma that can be removed by surgery. About 41 participants will receive the drug combination before surgery…

This study tests a personalized approach for children with non-high-risk neuroblastoma, where treatment is guided by the tumor's biology and how it responds over time. Some children may only need monitoring instead of immediate therapy, potentially avoiding harsh side effects. Th…

This study is testing the long-term safety of a daily oral drug called zagociguat in 44 adults with MELAS, a rare genetic disease that affects energy production in cells. All participants previously completed a lead-in study of the same drug. Researchers will monitor side effects…

This study follows about 1,300 people with multiple sclerosis who are already taking ocrelizumab to see how safe and effective it is over many years. Participants continue their usual treatment, and researchers track side effects, disability changes, and brain scans. The goal is …

This early-phase study tests a radioactive drug (177Lu-PP-F11N) that seeks out and attaches to cancer cells in people with advanced medullary thyroid cancer and certain other neuroendocrine tumors. The goal is to see if the drug can help image tumors and deliver targeted radiatio…

This trial tests a new hip resurfacing system in 238 adults with hip osteoarthritis or mild dysplasia. The device is designed to cap the damaged joint rather than replace the entire hip. Researchers will compare pain, function, and safety to standard total hip replacement over tw…

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

This study looks at a procedure done before birth to help babies with a severe heart defect called hypoplastic left heart syndrome (HLHS). The goal is to improve blood flow through the heart so that the baby may have a better chance at a two-sided heart repair after birth. The st…

This study tests whether a higher dose of the drug ocrelizumab can better control relapsing multiple sclerosis (MS) and delay disability compared to the standard dose. About 864 adults with relapsing MS will receive either the higher or standard dose every 24 weeks. The main goal…

This study is testing the drug ocrelizumab in 927 people with progressive multiple sclerosis (MS), a form of the disease that steadily worsens. Participants receive infusions every 24 weeks for about 4 years. The main goal is to see if the drug can stop or slow disability progres…

This phase 2 study tests the drug alpelisib in 206 children and adults with PIK3CA-related overgrowth spectrum (PROS), a rare condition causing abnormal tissue growth. Participants receive either alpelisib or a placebo for 16 weeks to see if the drug can shrink growths by at leas…

This study follows 60 adults with Fabry disease who are receiving pegunigalsidase-alfa, a newer enzyme replacement therapy. Researchers want to see how well it works in everyday medical practice, especially for kidney function. Participants will be treated for 2 years at speciali…

This early-phase trial is testing a new drug called NTX-1088, which targets a protein on cancer cells, either alone or combined with the immunotherapy pembrolizumab. The study involves 90 people with advanced solid tumors that have spread or cannot be removed. The main goals are …

This phase 2 trial is testing the drug ibrutinib in 18 people with stage IV melanoma that has not responded to previous treatments. Ibrutinib works by blocking certain enzymes that help cancer cells grow. The study aims to see if the drug can shrink tumors and how long it keeps t…

This study tests a surgically implanted bionic hand system for people who have lost an arm below the elbow. The device aims to connect directly to nerves so users can control it more naturally and even feel sensations. Fifteen participants will be followed to see if the system is…

This study checks the long-term safety of RGX-111, a gene therapy for people with MPS I (a rare genetic disorder). It follows 21 participants who already received the therapy in an earlier trial. Researchers will monitor side effects and measure changes in thinking and behavior o…

This trial tests a nurse-led health program for women aged 45 to 64 in Hong Kong. The program uses a 5-step approach (ask, advice, assess, assist, arrange) to help women manage conditions like high blood pressure, anxiety, and menopause symptoms. Researchers will compare the prog…

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

This phase 2 trial tests whether theophylline, a drug used for asthma, can help people with pseudohypoparathyroidism lose weight and improve blood sugar control. The study includes 29 obese participants aged 13 and older. Researchers will measure changes in body mass index and gl…

This phase II trial is testing whether a combination of two drugs, rigosertib and pembrolizumab, can help people with advanced melanoma that no longer responds to standard immunotherapy. The study involves 7 participants with unresectable or metastatic melanoma. The goal is to se…

This early-phase study tests a personalized cell therapy for children and young adults (up to age 26) with solid tumors that have come back or not responded to standard treatment. The therapy uses the patient's own immune cells, modified to recognize and attack a protein called B…

This early-phase trial tests a combination of three treatments—radioactive iodine (131-I-MIBG), an immunotherapy drug (nivolumab), and an antibody (dinutuximab beta)—in children whose high-risk neuroblastoma has come back or not responded to standard therapy. The main goal is to …

This study is testing a new surgical heart valve (Model 400) in over 1,300 people with aortic valve disease. The valve is designed to replace the damaged aortic valve and improve blood flow. Researchers are checking how safe the valve is and how well it works over one year, looki…

This study tests whether theophylline, a drug used for asthma, can help children and young adults with pseudohypoparathyroidism—a rare genetic condition causing early obesity, hormone problems, and short stature. Researchers will check for weight loss, better blood sugar control,…

This phase 2 trial tests theophylline, a drug used for asthma, in 34 children aged 2 to 12 with pseudohypoparathyroidism, a genetic condition causing obesity, short stature, and hormone resistance. The study aims to see if theophylline can help with weight loss, slow bone growth …

This early-stage trial tests a new treatment called GD2 CAR T cells in 12 children and teens (ages 1-16) with neuroblastoma that has come back or not responded to standard therapy. The treatment involves taking a patient's own immune cells, modifying them to better recognize and …

This study compares two treatments for metastatic melanoma that has spread: high-dose Interleukin-2 (IL-2) alone versus IL-2 combined with targeted radiation (SBRT). The goal is to see if adding radiation helps shrink tumors better than IL-2 alone. The trial enrolled 44 adults wi…

This study follows 41 children with Sanfilippo A (MPS IIIA) who previously received UX111 gene therapy in earlier trials. Researchers will monitor safety and how well the therapy controls the disease over time, using tests like the Bayley cognitive scale. No new gene therapy is g…

This phase 2 trial tests the drug trametinib in children and young adults (ages 1 month to 25 years) with low-grade glioma or plexiform neurofibroma that has not responded to prior treatment. Participants take a daily oral dose for up to 18 cycles. The study aims to see if the dr…

This phase II trial tested a combination of three drugs—pembrolizumab (Keytruda), dabrafenib (Tafinlar), and trametinib (Mekinist)—in 16 people with advanced melanoma that could not be removed by surgery. The goal was to see if a shorter course of targeted therapy, followed by im…

This study tests an inhaled form of interleukin-2 (IL-2), a protein that boosts the immune system, in people whose cancer has spread to the lungs. The goal is to find the best dose and see if it can shrink lung tumors. About 70 adults with cancers like melanoma, sarcoma, or kidne…

This study planned to test whether adding surgery, radiation, or electrochemotherapy could help control melanoma tumors that did not shrink with encorafenib + binimetinib combination therapy. It was designed for adults with BRAF-mutated melanoma who had been on the drugs for at l…

This early-stage study tests an experimental cell therapy called P-CD19CD20-ALLO1 in 60 adults with multiple sclerosis. The main goal is to see if it is safe and how the body handles it. Researchers will also measure how long the cells last and how they affect the immune system.

This study tests a treatment called RP1, a modified herpes virus injected directly into skin tumors, in 69 people who have had an organ transplant and now have advanced skin cancer. The goal is to see if the virus can shrink tumors safely without harming the transplanted organ. P…

This early-stage study tests an oral drug called LY3410738 in people with advanced solid tumors (like bile duct cancer, cartilage cancer, or brain tumors) that have specific IDH1 or IDH2 mutations. The main goal is to find a safe dose and see if the drug can shrink tumors. About …

This early-stage trial tests an experimental drug called LY3295668 erbumine in children whose neuroblastoma has returned or not responded to treatment. The study aims to find a safe dose and see if the drug can shrink tumors, either alone or with standard chemotherapy. About 71 p…

This early-phase study tests a new treatment for advanced osteosarcoma and neuroblastoma that has not responded to standard therapy. Researchers take a patient's own immune cells (T cells), add a gene to help them recognize and attack cancer cells, and combine them with a chicken…

This phase II trial tests two drugs, pembrolizumab and lenvatinib, given before surgery to people with stage III melanoma that can be removed. The goal is to see if the combination can shrink or eliminate the tumor before the operation. Twenty-one participants will receive the dr…

This phase 3 trial tests whether combining two immunotherapy drugs, fianlimab and cemiplimab, works better than the standard drug pembrolizumab for people with advanced melanoma that cannot be surgically removed. About 1,500 adults and adolescents who have not had prior treatment…

This study offers girls from birth to age 17 the chance to freeze their ovarian tissue before medical treatments that could harm their fertility, such as chemotherapy or radiation. The tissue is removed surgically and stored. Later, if they want to have children, the tissue can b…

This study tests an experimental therapy called lifileucel for people with advanced melanoma that has spread or cannot be removed by surgery and has worsened after standard treatments. About 100 adults will receive their own tumor-fighting immune cells (TILs) grown in a lab. The …

This study tests whether a two-step drug treatment can improve bone density in people with long-term spinal cord injury who have bone loss. Participants receive either romosozumab (a bone-building drug) or a placebo for 12 months, followed by denosumab (a bone-strengthening drug)…

This Phase 3 trial tests a radioactive imaging agent called 18F-mFBG to see if it can accurately detect neuroblastoma, a type of childhood cancer. The study involves 43 participants who either have or are suspected of having neuroblastoma. Researchers will compare the new PET sca…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study tests a smartphone app called ausculto™ that records and analyzes heart sounds to detect congenital heart disease. Researchers will record heart sounds from 220 people aged 6 and older who have had recent echocardiograms. The goal is to see if the app can accurately te…

This study tests whether a special PET scan using a radioactive form of methionine (a natural amino acid) can help doctors see tumors in children and young adults with various cancers, including brain tumors and sarcomas. About 503 participants will be scanned to see if the metho…

This study tests a new radioactive tracer called 18F-DA for PET scans to see if it can find neuroblastoma and pheochromocytoma tumors more clearly than the current tracer. About 20 children and adults with known or suspected tumors will receive the tracer and be monitored for sid…

This study was designed to see if a special bone scanner (bone mineral analyser) could predict how well osteoporotic women would respond to a zoledronic acid infusion. The plan was to measure bone texture and density before and one year after treatment. However, the study was wit…

This study tests a new ultrasound device that measures bone properties in the lower leg to predict fracture risk in adults over 55. It aims to see if it works as well as or better than standard DXA scans, without using radiation. Researchers will follow 1,600 participants for thr…

This study compares a new ultra-high resolution PET/CT scanner, the NeuroEXPLORER, with standard clinical PET/CT scanners for imaging the head and neck. Researchers aim to see if the new device provides more detailed images to improve diagnosis of conditions like dementia, Parkin…

This study tests whether a new type of PET/CT scan, using a radioactive tracer called 68Ga-DOTATOC, can find neuroendocrine tumors (NETs) and similar tumors more safely and accurately than current imaging methods. About 800 people with known or suspected NETs will receive the sca…

This study tests a new dye, called cRGDY-PEG-Cy5.5-C dots, to help surgeons see cancer in lymph nodes during head and neck melanoma surgery. About 67 people will receive the dye injection around their tumor before or during surgery. The dye is not a treatment, but may improve how…

This study tests whether adding a special stain called PRAME to standard tissue analysis helps surgeons remove early-stage melanoma more accurately. About 36 adults with stage 0 to IIc melanoma will have slow Mohs surgery, and their tissue will be checked both with and without PR…

This study tests a special camera that uses light and sound to take 3D pictures of skin cancer. The goal is to see how deep and wide a tumor is before surgery. About 200 people with suspected skin cancer will take part. The images will be compared to standard lab results to see i…

This study is testing a blood-based genetic test to find cancer-driving mutations in patients with lung cancer or melanoma. The goal is to see if this simple blood draw can replace the need for a tumor biopsy, which is slower and more invasive. Researchers will check how accurate…

This study looks at whether a blood test that detects tumor DNA can find melanoma recurrence earlier than standard methods. Researchers will follow 467 high-risk patients who have had primary melanoma removed. If the test works, it could lead to faster treatment and better outcom…

This study is testing a gene signature (the Merlin Assay) to see if it can accurately predict whether melanoma has spread to nearby lymph nodes. Researchers are enrolling 1,820 newly diagnosed melanoma patients who are scheduled for a sentinel lymph node biopsy. The goal is to de…

This trial investigates whether a daily effervescent supplement containing calcium, magnesium, and citrate can prevent bone loss, magnesium deficiency, and kidney damage in adults who take proton pump inhibitors (PPIs) long-term for conditions like heartburn or GERD. Participants…

This study aimed to see if a surgical procedure called scleral buckling could prevent retinal detachment in people with Stickler syndrome, a genetic condition that raises the risk of vision loss. The plan was to treat one eye in patients aged 5 to 35 who had already lost vision i…

This study tests whether a supervised resistance exercise program can reduce the risk of osteoporosis and osteoarthritis in women aged 50-70 who have gone through menopause. Participants will do leg and core exercises twice a week for 30-40 minutes. Researchers will measure chang…

This study looks at the long-term safety and effectiveness of odevixibat in 70 people with Alagille syndrome, a rare genetic condition that affects the liver and other organs. The drug aims to reduce severe itching and improve bile flow. Participants who completed a prior 24-week…

This study compares two types of exercise—open kinetic chain (like leg raises) and closed kinetic chain (like squats)—to see which better reduces knee hyperextension in stroke survivors. Sixty adults who have had a stroke and can walk with some help will be randomly assigned to o…

This study tests whether a 24-week physical therapy program can help people with multiple sclerosis become more physically active. Forty participants will receive tailored exercise support and education. The goal is to see if this approach is feasible and helps manage MS symptoms…

This pilot study explores whether non-invasive electrical stimulation of the spinal cord, combined with hand and arm exercises, can improve upper limb function in people with multiple sclerosis. Participants use a home device with help from a partner over 8 weeks. The study focus…

This pilot study tests whether one-on-one peer support sessions can help people with relapsing-remitting multiple sclerosis stay on their prescribed medications. Sixty adults will be randomly assigned to receive either standard care or three hour-long sessions with a trained peer…

This study tests whether a single high dose of psilocybin (the active ingredient in magic mushrooms) combined with specialized talk therapy can help people with advanced cancer feel less anxious and more at peace. Fifteen adults with stage IV cancer will receive the treatment and…

This study tests a telemedicine program called FOCUS that helps advanced cancer patients manage common symptoms like trouble sleeping, worry, tiredness, and low mood. The program uses counseling techniques delivered through a phone app. Researchers will enroll 120 adults with adv…

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

This study planned to test whether a nerve block in the cheek area could reduce the need for morphine after cleft palate repair in children aged 5 months to 12 years. The experimental group would have received the nerve block, while the control group would not. However, the study…

This study tests whether monthly alexandrite laser treatments can safely shrink or improve the look of skin tumors in adults with neurofibromatosis type 1 (NF1). Ten participants will receive up to six monthly laser sessions, with some areas treated with cooling and others withou…

This pilot study tests a program called ALIGN, designed to help adults with spina bifida improve how they manage health tasks like coordinating care and handling medications. The program includes six virtual group sessions and one individual session with a clinician. Researchers …

This study tests whether a guided internet-based physical activity program can improve cognitive processing speed in people with multiple sclerosis. 280 participants who have mild walking difficulties and slowed thinking will be randomly assigned to either the exercise program or…

This study tests whether a probiotic supplement called Neuralli® MP can help adults aged 18 to 50 who have autistic traits. Participants take two capsules daily and set their own personal goals to track progress. The trial is small and open-label, meaning everyone knows they are …

This study tests whether peer health coaching—support from someone with similar experiences—can help young adults (ages 18-26) with congenital heart disease better manage their condition. Participants receive coaching via secure phone calls and texts over 6 months. The goal is to…

This study tests whether taking a special olive oil (high in a compound called oleocanthal) along with curcumin (the active part of turmeric) is safe and might help shrink skin tumors in adults with neurofibromatosis type 1 (NF1). About 23 participants take the supplements twice …

This study tests if a gentle, non-invasive nerve stimulation device can help manage symptoms of multiple sclerosis (MS), like fatigue and pain. Thirty women with MS will use the device at home for a few weeks. The goal is to see if it's practical and if it reduces symptom burden,…

This phase 2 trial tests whether a single low-dose ketamine infusion can reduce fatigue in people with multiple sclerosis. About 110 adults with MS-related fatigue will receive either ketamine or a mild sedative (midazolam) as a placebo. The study measures changes in fatigue scor…

This phase 2 study tests whether the experimental drug bezuclastinib can reduce symptoms like itching, flushing, and fatigue in people with nonadvanced systemic mastocytosis, a rare condition where too many mast cells cause chronic discomfort. About 237 adults whose symptoms are …

This study tests whether combining transcranial magnetic stimulation (TMS) with virtual reality exercises and physical therapy can improve symptoms like balance, fatigue, and thinking in people with multiple sclerosis. Thirty participants will receive both real and sham TMS in a …

This study aimed to see if freezing certain nerves during minimally invasive heart surgery could lower pain afterward. It planned to enroll adults having mitral valve or atrial septal defect repair. However, the trial was withdrawn before any participants were enrolled, so no dat…

This pilot study aimed to test whether immersive virtual reality (VR) exercises could help people with multiple sclerosis who experience dizziness, balance issues, and fatigue. Participants would have been randomly assigned to either VR-based vestibular rehab or conventional exer…

This study tests whether a home exercise program combined with a gentle brain stimulation technique (tDCS) can reduce fatigue in people with multiple sclerosis. Sixty participants will either get real tDCS or a sham version alongside their exercises. The goal is to see if the com…

This study tests whether small meals containing dairy or banana can reduce bone breakdown in postmenopausal women with osteopenia (low bone density). Thirteen women will eat five different test meals or fast, then have blood samples taken over six hours to measure markers of bone…

This study looks at whether special high-dissolving foods can improve feeding quality of life for children aged 6 months to 18 years after craniofacial surgery. Half of the 160 participants will receive these foods after surgery, while the other half will follow standard care. Re…

This study tests a biodegradable tube that is placed over a cut nerve during a leg biopsy. The goal is to see if it is safe and reduces common side effects like pain, redness, and swelling. Twenty adults with various nerve conditions are taking part, and half will get the tube wh…

This early-stage trial tests whether monthly injections of Kybella (a drug that dissolves fat) can safely shrink skin tumors in people with Neurofibromatosis Type 1. Up to 15 adults will receive up to 6 monthly treatments, and researchers will compare treated and untreated tumors…

This phase II trial, called MATCH, is testing whether treating cancer based on the genetic makeup of a patient's tumor works better than standard treatments. Over 6,400 adults with advanced solid tumors, lymphomas, or multiple myeloma that have not responded to prior therapy are …

This study is not testing a new treatment. Instead, it follows people who already received experimental cell therapies (like growing immune cells from their tumors or blood) in earlier NCI Surgery Branch trials. The goal is to monitor their health for up to 15 years, as required …

This study follows over 64,000 adults who grew up on farms to see if early exposure to pesticides and other agricultural factors is linked to cancer. Participants fill out an online questionnaire and may provide biological samples. Researchers will track their health over many ye…

This study explores whether a new type of resistance exercise is practical and safe for people with low bone mass, including those with osteopenia, osteoporosis, or sarcopenia. Over two months, one group performs progressive muscle-strengthening exercises while a control group co…

This study looks at whether a fermented wheat germ supplement can help the immune system fight cancer better when taken with standard immunotherapy drugs. About 100 adults with advanced solid tumors (like lung, colon, or melanoma) will take the supplement alongside their usual tr…

This study tests whether offering online educational videos and mailed saliva genetic testing kits helps more first-degree relatives of people with BRCA mutations get tested, compared to standard care with a family letter. About 820 participants will be enrolled across several me…

This study tests whether combining standard monitoring tools with new light-based sensors can better detect a dangerous narrowing of the aorta in newborns. Infants under one month old with suspected aortic arch obstruction will be monitored using resonance Raman spectroscopy, pul…

This observational study collects extra tumor tissue from 416 people with various cancers (bladder, kidney, colorectal, head and neck, liver, lung, skin, endometrial) during routine biopsies. The goal is to develop and train a 'live tumor diagnostic platform' that can predict whe…

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

This study looks at how much of the multiple sclerosis drug ofatumumab gets into breast milk. About 24 women with relapsing MS who are breastfeeding and starting or restarting ofatumumab will provide milk samples. The goal is to measure drug concentrations, which may help guide f…

This trial tests whether a custom-made implant (patient-specific implant) or a standard occlusal splint is more accurate for positioning the upper jaw during corrective jaw surgery. Twenty adults with jaw deformities will be randomly assigned to one of the two methods. The goal i…

This study explores whether artificial intelligence can predict which children with a type of heart hole (perimembranous ventricular septal defect) will later develop complications like valve problems or blockages. Researchers will analyze past echocardiogram reports and medical …

This study was designed to compare electrical and mechanical activation of the right ventricle in adults who had surgery for Tetralogy of Fallot, a congenital heart condition. Researchers planned to use 3D mapping and imaging to see if electrical signals match the heart's physica…

This study was designed to see how a low dose of bevacizumab (Avastin) behaves in the body and whether it safely lowers a certain protein (VEGF-A) in people with kidney failure on dialysis. The goal was to gather information for a future trial on preventing dialysis access failur…

This long-running study follows nearly 100,000 French women, mostly teachers, since 1990 to understand what factors influence their risk of cancer, heart disease, diabetes, and other major illnesses. Participants fill out questionnaires every few years about their lifestyle, medi…

This natural history study follows 259 children, adolescents, and adults with Neurofibromatosis Type 1 (NF1) for up to 10 years. Researchers will track tumor growth, monitor quality of life, and perform genetic testing to better understand how the disease changes over time. The g…

This study aimed to evaluate how community cancer centers test for biomarkers in common solid cancers like breast, lung, and colorectal cancer. Researchers planned to survey pathology practices to see if they follow guidelines for key tests and how quickly results are reported. T…

This study examines how often healthcare workers in a hospital's intensive care unit (ICU) experience physical, verbal, or sexual assaults from patients or their relatives. Researchers will review medical records of 865 adults who were in the ICU and had a reported incident of vi…

This study follows 48 people with advanced melanoma who previously received the experimental drugs fianlimab and cemiplimab in an earlier study. No new treatment is given; instead, researchers track their health, cancer status, and any late side effects over time. The goal is to …

This study screens children and teens with advanced solid tumors, lymphomas, or histiocytic disorders to find genetic changes in their cancer cells. Participants undergo biopsies and scans to identify mutations that can be targeted by specific drugs. The goal is to see if matchin…

This study looks at how many women with multiple sclerosis (MS) use the drug interferon-beta during the second and third trimesters of pregnancy in Finland and Sweden. Researchers want to know if there are enough cases to study whether the drug affects pregnancy outcomes like bir…

This study was designed to see if preterm children (born at or before 28 weeks) have differences in their retinal blood vessels compared to full-term children. Researchers planned to use a non-invasive imaging device called OCT angiography to measure blood vessel density. However…

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

This study looks at whether a blood test measuring a protein called neurofilament light (sNfL) can help doctors predict and manage disease activity in people with relapsing multiple sclerosis (MS). About 489 adults with MS in Germany will be observed for 24 months. The goal is to…

This study follows 35 people with Merkel cell carcinoma, a rare and aggressive skin cancer, to see if two blood markers (T-antigen antibodies and miR-375) can detect when the cancer comes back. Currently, follow-up relies on physical exams and scans, which may miss early recurren…

This study was designed to see if different types of anesthesia could help a surgically created blood vessel connection (called a fistula) work better for dialysis patients. The plan was to compare three methods: a nerve block in the armpit, a nerve block in the neck, and local a…

This study collects tissue samples from 1,000 people having heart valve surgery to find genetic causes of calcific aortic valve disease. Researchers will compare normal and diseased tissue to understand why the disease develops. Participants must be at least 20 years old and have…

This observational study is creating a large database of up to 96,000 people with multiple sclerosis and related conditions in Italy. Researchers will collect information on patient demographics, treatments, and disease progression over time. The goal is to better understand thes…

This phase 2 trial is testing how the immunotherapy drug pembrolizumab (Keytruda) changes immune-related genes and biomarkers in people with advanced solid tumors like head and neck cancer, triple-negative breast cancer, ovarian cancer, and melanoma. Researchers will analyze bloo…

This study collects white blood cells and tumor cells from cancer patients, and white blood cells from healthy volunteers, to help researchers grow anti-cancer cells in the lab. The goal is to create experimental cell therapies for future use. Up to 7,000 participants will provid…

This study observes over 1,100 pregnant women with multiple sclerosis to see if taking the drug diroximel fumarate (VUMERITY) affects their babies' health. Researchers will compare birth defects, miscarriages, preterm births, and stillbirths among women who took DRF, other MS dru…

This study follows up to 90 children and young adults with mitochondrial liver diseases to learn how these conditions progress over time. Researchers will collect medical data and samples to better understand the diseases and find markers that predict outcomes. The goal is to imp…

This study followed children and young adults with genetic liver diseases that cause bile buildup. The goal was to track how these diseases progress over time, including the need for liver transplants or other complications. No treatments were tested; the aim was simply to learn …

This study follows 600 people with Albright hereditary osteodystrophy, a rare genetic condition affecting bones and hormones. Researchers are looking at how growth hormone treatment affects height and weight in those with a related hormone problem, and also studying thinking and …

This study looks at how bone contributes to citrate levels in urine, which may help explain why some people have low urine citrate. Researchers will study 25 adults with untreated osteoporosis, giving them potassium citrate and measuring changes in urine citrate and bone markers.…

This study tests a new imaging method to see if white blood cells enter the brain in people with multiple sclerosis, fibromyalgia, or chronic fatigue syndrome. Participants receive their own white blood cells tagged with a radioactive tracer, then undergo a brain PET/MRI scan. Th…

This study aimed to compare T cell populations between people with multiple sclerosis and healthy controls to better understand which immune cells attack the nerves. It was withdrawn before enrolling any participants, so no results are available. The goal was to learn more about …

This study looks at blood samples from 20 adults with melanoma or other skin cancers to see if certain markers in the blood can predict how well treatment works or how long someone lives. Researchers will compare these markers to standard measures like tumor response and survival…

This study tests a web-based tool called gp-multitool.de that helps general practitioners follow guidelines for older patients with multiple chronic conditions. About 659 patients from GP practices in Germany are taking part. The tool lets patients fill out questionnaires online …

This study collects tissue and blood samples from 1,200 adults with known or suspected cancer. The samples are taken during standard surgeries or biopsies, with no extra procedures. Researchers will compare cancerous and normal tissues to identify differences that could lead to n…

This study will collect and analyze genetic data from 1000 people with suspected inherited metabolic diseases, including conditions like epilepsy and mitochondrial disorders. Researchers at Karolinska University Hospital aim to improve diagnosis by using advanced genetic testing …

This study is for adults with advanced head and neck, lung, or skin cancer who have not responded to prior treatments. Researchers use special PET scans to see how two experimental drugs (BI 765063 and BI 770371) travel through the body and reach tumors. Participants receive one …

This study is looking at whether a blood test that detects circulating tumor DNA (ctDNA) can help predict if melanoma will come back or spread. Researchers are following 176 adult melanoma patients and comparing ctDNA levels with standard clinical and lab results. The goal is to …

This observational study follows 367 adults with relapsing multiple sclerosis who are taking cladribine tablets (Mavenclad) in routine care. Researchers want to find out which patient characteristics predict how long people stay on the drug and how well it works. The goal is to h…

This study tests whether a statewide educational campaign in Oregon helps people spot melanoma earlier. Over 75,000 participants, including patients and healthcare providers, receive training and digital materials. Researchers compare Oregon's results with neighboring states that…

This study looks at whether a simple bedside lung ultrasound can predict chronic lung disease in premature infants born before 32 weeks. Researchers will perform ultrasounds on 150 babies with breathing problems and track their outcomes. The goal is to see if early ultrasound sco…

This study creates a long-term registry for children with Hirschsprung's disease or anorectal malformations. It collects health data and family surveys during regular clinic visits to understand outcomes and improve care. No extra tests or procedures are needed. Up to 999 childre…

This study follows nearly 600 people with Smith-Magenis syndrome (SMS), a rare genetic condition, to track how their health, behavior, and development change over time. Researchers will perform detailed medical exams, genetic tests, and surveys to better understand the syndrome's…

This study looks at whether Facebook groups can help young people diagnosed with melanoma (ages 18-39) and their close relatives do more skin exams and sun protection. About 1,160 participants will join either a melanoma-focused Facebook group or a healthy lifestyle Facebook grou…

This registry follows 63 patients with choroidal melanoma or early eye lesions who were treated with the experimental drug bel-sar in earlier studies. The goal is to monitor long-term safety, side effects, and any spread of the disease over time. Participants are not receiving ne…

This early-phase study tests very small amounts of cancer drugs injected directly into accessible tumors in 15 adults scheduled for surgery. The goal is to see how the drugs affect the tumor's environment, like killing cancer cells or activating immune cells, without causing full…

This study is testing a painless, sticker-like skin test in 100 children and teens with suspicious moles. The test looks for certain genetic markers (PRAME and LINC) to see if they match the results from a standard biopsy. The goal is to learn if this noninvasive method can help …

This study collects data from 1,200 patients undergoing surgery for aortic dissection, a life-threatening tear in the heart's main artery. Researchers will track death rates, strokes, and other complications to identify the safest surgical strategies. The goal is to improve outco…

This study follows 900 patients with a life-threatening tear in the aorta (type A aortic dissection) to compare outcomes between different surgical techniques. Researchers will track deaths, strokes, heart failure, and other complications within 30 days after surgery. The goal is…

This study follows 400 patients for two years after they have endoscopic endonasal skull base surgery (surgery through the nose to reach the base of the skull). The goal is to measure their quality of life and nose function using standard questionnaires. It does not test a new tr…

This study looks at how a single dose of antibiotics given before melanoma surgery changes the bacteria in your gut. Researchers want to see if these changes affect how well the body responds to surgery and cancer treatment. The study involves 20 adults with early-stage melanoma …

This Stanford study is testing whether non-intrusive sensors placed in the home can automatically track neuropsychiatric symptoms like mood changes and agitation in older adults. Researchers will enroll 25 participants with or at risk of mild cognitive impairment or dementia, alo…

This study looks at whether the immune system mistakenly makes antibodies that attack the body's own nerves and muscles in people with multiple sclerosis, myasthenia gravis, and similar conditions. Researchers will collect blood samples (and sometimes cheek swabs) from 120 adults…

This study follows 440 people with relapsing-remitting multiple sclerosis for 5 to 10 years after their diagnosis. Researchers will use brain scans, lab tests, and genetic information to find patterns that predict how the disease will progress. The goal is to help doctors and pat…

This study follows 26 people who had hip surgery (periacetabular osteotomy) for hip dysplasia. Researchers want to see how many return to their favorite sports and how well they recover. The study collects data from medical records and patient reports, focusing on pain levels and…

This study is testing two new digital tools to monitor how multiple sclerosis (MS) progresses over time. One tool uses cameras to measure movement and balance, while the other uses eye scans to detect nerve damage. Ten people with stable MS will try these tools, and the results w…

This study looks at survival rates for people with cancers like melanoma, breast, colon, and lung cancer using data from Russian cancer registries. It includes 100,000 people of any age. The goal is to understand survival trends to help improve cancer care, not to test a new trea…

This study is looking at whether an artificial intelligence tool can help doctors decide which heart surgery patients might benefit from a drug called ulinastatin. Ulinastatin is used to reduce inflammation after surgery, but it doesn't work for everyone. Researchers will analyze…

This study follows 78 adults in Taiwan who have Fabry disease and carry a specific genetic mutation called GLA IVS4. Researchers are looking at how the disease affects the heart and other organs over time, both in people who have never been treated and those who have received aga…

This study looks at whether it's possible to screen for long-term health problems in people who survived high-risk neuroblastoma. Researchers will evaluate survivors who finished treatment at least 2 years ago, using tests for heart, nerve, and other health issues. The goal is to…

This study is creating a statewide collection of blood samples and medical information from people with various brain blood vessel diseases, such as stroke and dementia. The goal is to build a resource that scientists can use in future research to better understand and potentiall…

This study tracks about 400 people with multiple sclerosis (MS) to see how many develop antibodies against the drug natalizumab when it is given as a shot under the skin. Researchers will also look at side effects and relapse rates in those who develop antibodies. The goal is to …

This study collects tissue and blood samples from 1,600 people with various advanced cancers, along with their medical information, over the course of their treatment. Researchers will store these samples and data in a biobank for current and future studies. The goal is to better…

This study follows 96 people with end-stage kidney disease who need a brachiocephalic fistula for dialysis. Researchers collect data from exams, blood tests, and imaging to track changes in the vein over time. The goal is to learn what causes cephalic arch stenosis, a common narr…

This pilot study uses special MRI scans to check if the brain's waste-clearing system, called the glymphatic system, is not working properly in people with Clinically Isolated Syndrome (CIS), which is often the first sign of Multiple Sclerosis (MS). Researchers will compare scans…

This long-term study recruited over 12,000 non-pregnant women in Southampton, UK, and followed about 3,000 of them through pregnancy and their children's growth up to age 19. Researchers are tracking body composition, nutrition, asthma, and heart health to understand how a mother…

This study is testing a mobile app called CHAMP that lets parents of children with complex congenital heart disease enter health data and upload photos or videos from home. The goal is to see if the app helps doctors monitor the child's condition remotely and improve communicatio…

This study looks back at the medical records of 800 people who were hospitalized with dangerously low sodium levels (≤110 mmol/L). Researchers want to learn how often serious brain complications like swelling or nerve damage occur, and how treatment affects these risks. No new tr…

This study will enroll 210 adults aged 65 and older with early aortic stenosis (a heart valve narrowing) to see if an artificial intelligence tool can better predict how fast the disease worsens. Participants will have standard echocardiograms, and researchers will compare the AI…

This study looks at whether a 25-minute hydration education session plus a self-assessment worksheet can help wildland firefighters and other active people drink more fluids and stay hydrated. About 38 participants who are underhydrated will be split into two groups: one gets the…

This study tracks 80 patients with brain tumors (metastases or meningiomas) who receive stereotactic radiosurgery. Researchers use memory tests and quality-of-life questionnaires before treatment and every 3 months for a year to see if and how brain function changes. By comparing…

This study looks at how swallowing difficulties in people with multiple sclerosis (MS) relate to their nutrition and muscle function. Researchers will assess 34 ambulatory MS patients using questionnaires and physical tests. The goal is to better understand these connections to i…

This study follows 450 infants and their families from birth to age 12 to learn how a child's biology and home environment work together to shape eating habits and growth. Researchers collect samples, measurements, and surveys at regular visits. The goal is to understand what inf…

This study tests whether sending information letters to doctors and patients after a fracture can improve osteoporosis care. Over 4,200 older adults in Manitoba who had a hip, spine, arm, or wrist fracture are included. The goal is to see if these notifications increase bone dens…

This study aims to learn more about the genetic changes that cause Goltz syndrome, a rare condition that affects the skin, bones, eyes, and other organs. Researchers are collecting blood samples from up to 84 people with the condition and their parents to analyze their DNA. No tr…

This study measures certain substances in the blood (biomarkers) of infants with a rare heart defect called single-ventricle. The goal is to understand how these biomarkers relate to lung blood flow before and after a specific heart surgery (superior cavo-pulmonary anastomosis). …

This study tests whether adding automatic reminders to echocardiogram (heart ultrasound) reports can increase the number of patients with heart valve disease who see a cardiologist within six months. Researchers will randomly assign 300 patients' reports to receive no prompt, a p…

This study is testing a new way to care for people with rare diseases. It will use advanced genetic testing and a team of specialists to help diagnose patients faster and coordinate their care better. The study involves 136 participants with certain rare diseases and aims to redu…

This study follows 50 adults who had surgery for coarctation of the aorta (a narrowed main artery). Researchers want to see if exercise-induced high blood pressure causes hidden damage to the heart, arteries, or kidneys. Participants will do a stress test, heart ultrasound, and b…

This study looks at whether a high-fiber, plant-based diet and regular exercise can change the gut microbiome in people with melanoma who are receiving immunotherapy. Researchers want to see if these lifestyle changes are feasible and if they might affect how well the treatment w…

This study is testing whether a new type of MRI can detect changes in the brain that lead to memory and thinking problems in people with multiple sclerosis (MS). Researchers will scan 75 adults with MS and give them thinking and memory tests. The goal is to see if the new MRI mea…

This study looks at 224 adults who had surgery as children for a heart defect called tetralogy of Fallot. Researchers are using MRI scans and blood tests to measure scarring in the heart muscle. They want to see if this scarring is linked to serious heart problems like arrhythmia…

This study looks at whether obesity makes multiple sclerosis (MS) worse in children. Researchers will compare brain scans and blood markers between normal-weight and overweight/obese kids recently diagnosed with MS. The goal is to understand how obesity might drive inflammation a…

This study follows 302 adults with multiple sclerosis in France who are starting the drug ofatumumab as part of their normal care. Researchers will measure how patients' quality of life changes after 12 months using a special questionnaire. The goal is to understand real-world be…

This study is creating a registry of people with Hereditary Hemorrhagic Telangiectasia (HHT), a rare condition that causes abnormal blood vessels and frequent nosebleeds. Researchers will collect health information from about 1,000 adults over many years to see how the disease ch…

This study aims to develop a new way to measure how well the sinuses work in people with chronic sinusitis. About 30 adults with chronic sinusitis and a recent bacterial infection will take the drug ivacaftor. The main goal is to see if a special test can detect changes in sinus …

This study evaluates a program that aims to improve diets by making healthy foods like fish, chicken, and eggs more available and affordable for low-income households in Mozambique. Researchers will survey over 5,000 men and women to see if the program increases how often they ea…

This study was designed to compare a newer pacing method (left bundle branch area pacing) with the standard method (right ventricular pacing) in patients who developed heart block after TAVR valve replacement. The goal was to see which method better preserves heart function and r…

This study looks back at medical records of 50 people with Mucolipidosis Type IV (MLIV) to learn how the disease typically progresses. Researchers want to know when children reach or lose developmental milestones, how their movement and vision change, and what lab results look li…

This study tested two teaching methods for nursing students: a 360-degree video experience about the brain and multiple sclerosis (MS), and a standard slideshow with the same information. About 50 students from three nursing schools took part. Researchers measured how much they l…

This study follows 200 people with Parkinson's disease or multiple sclerosis over one year to see how active they are. Participants use a smartphone app called Datos Health to track steps, workouts, and heart rate. A physical therapist sets personalized exercise goals. The goal i…

This national study is looking at 500 babies and fetuses with non-immune hydrops fetalis or other birth defects to find hidden genetic causes. Researchers are using a detailed genetic test called exome sequencing to look for DNA changes that might explain these conditions. The go…

This study looks for genetic causes of non-immune hydrops fetalis, a condition where fluid builds up abnormally in a fetus, leading to high risks of stillbirth or death. Researchers will use whole genome sequencing on 500 affected fetuses or newborns and their parents. The goal i…