Disorder of development or morphogenesis

Researchers are testing a new enzyme replacement therapy called GNR-055 in people with Hunter syndrome (MPS II), a rare inherited disorder that causes harmful sugar molecules to build up in the body. The trial enrolls about 32 patients, including those who have never had enzyme t…

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 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 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 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 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 two drugs, nivolumab and cabozantinib, together in 60 people with advanced or metastatic non-clear cell kidney cancer. The goal is to see how well the combination shrinks tumors. Participants take cabozantinib daily and receive nivolumab infusions every two weeks…

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 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 II trial tests whether combining lenvatinib and everolimus can shrink or slow advanced carcinoid tumors that cannot be removed by surgery. The study enrolls 36 adults with confirmed unresectable carcinoid tumors. Both drugs are taken orally and work by blocking enzymes…

This phase 2 trial is testing whether a combination of two drugs, enfortumab vedotin and pembrolizumab, can shrink tumors in people with advanced melanoma that has worsened after standard therapy. The study will enroll 60 adults with unresectable stage III or IV melanoma. Researc…

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

This study tests whether Empagliflozin (Jardiance), a drug used for diabetes and heart failure, can help adults born with heart defects who now have a weakened heart. Forty participants will receive either the drug or a placebo for one year. Researchers will measure changes in he…

This study tests a new medicine called PYC-001, given as an injection into the eye, for people with a genetic condition that damages the optic nerve (OPA1 optic atrophy). The main goal is to check if the treatment is safe and tolerable. About 18 adults will receive a single dose,…

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

This 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 early study is testing a new device called the ReNew Hip Implant in 15 people aged 14 to 64 with hip cartilage damage from conditions like osteoarthritis or hip impingement. The implant is surgically placed to repair the cartilage. Researchers are checking if it is safe and …

This phase 2 trial tests whether adding an experimental pill (GB1211) to the immunotherapy drug pembrolizumab (Keytruda) works better than Keytruda alone for people with advanced melanoma or head and neck cancer. The study will enroll 92 participants and measure how many patients…

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 study tests whether adding tocilizumab (a drug used for arthritis) to standard immunotherapy can reduce severe side effects in people with advanced melanoma, lung cancer, or bladder cancer. About 35 participants will receive all three drugs. The goal is to see if the combina…

Researchers are testing whether natural killer (NK) cells from a universal donor can be safely given with standard chemotherapy drugs and an immune-targeting antibody to children and young adults with neuroblastoma that has returned or stopped responding. The NK cells are modifie…

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 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…

Fanconi anemia is a genetic condition that can damage bone marrow and lead to low blood cell counts, causing anemia, bleeding, or infections. Researchers are testing whether a daily pill called eltrombopag can improve blood counts in people with this condition. The trial enrolls …

This early-phase study tests whether 30 minutes of moderate exercise (like walking or cycling) right before standard immunotherapy can improve treatment for certain skin cancers (melanoma, squamous cell carcinoma, Merkel cell carcinoma). About 22 adults will exercise before each …

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 …

Researchers test whether giving mitomycin plus carboplatin before surgery helps people with BRCA1 or BRCA2 mutations and locally advanced triple-negative breast cancer. The trial compares this combination, followed by paclitaxel, against standard chemotherapy. About 30 adults wit…

This international registry study looks at a small device called a transcatheter pulmonary flow restrictor, which doctors implant in the lung arteries of young children with congenital or acquired heart disease. The goal is to see whether the device safely and effectively control…

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 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 study tests a drug called selumetinib in adults 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 these tumors or slow their growth. Participants take the drug twice daily and are monitored …

This early-phase trial tested the safety and best dose of two drugs—riluzole and sorafenib—given together to people with advanced solid tumors or melanoma that had spread and stopped responding to standard treatments. The goal was to find the highest safe dose and see if the comb…

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

Researchers are testing an experimental antibody called ONC-392 in people with advanced solid tumors, including non-small cell lung cancer and melanoma. The trial gives ONC-392 alone or with pembrolizumab, an approved immunotherapy, to see what doses are safe and how the body pro…

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

This 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 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 phase II trial tests whether combining two drugs, atezolizumab and bevacizumab, can shrink rare solid tumors like appendix cancer, anal cancer, and mesothelioma. About 133 participants with various rare cancers will receive the drugs intravenously. The goal is to see if the …

This study tests the safety and dosing of ocrelizumab in 23 children and teens (ages 10–18) with relapsing-remitting multiple sclerosis over 2 years. The goal is to find the best dose for a future larger study. Participants receive the drug and are monitored for side effects and …

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 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 follows 300 people with blocked leg arteries who received a treatment using a balloon coated with paclitaxel and vitamin E. The goal is to see if the balloon keeps the artery open and prevents the need for repeat procedures over one year. Participants are adults with m…

This early-phase trial is testing a personalized cell therapy called lifileucel in 10 people with melanoma that has spread to the brain and is no longer responding to standard immunotherapy. Doctors remove a piece of the tumor, grow the patient's own immune cells in the lab, and …

This trial is testing whether adding hydroxychloroquine to two targeted cancer drugs (dabrafenib and trametinib) can help children and young adults with certain brain tumors that have come back or not responded to treatment. The study includes patients with specific genetic chang…

This early-phase trial tests a new treatment for people with advanced melanoma that has stopped responding to standard immunotherapy. The treatment uses a patient's own immune cells (TILs) that have been engineered to produce a protein called IL15, combined with a drug called ace…

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 early-phase trial is testing an experimental drug called TTX-080, which blocks a protein (HLA-G) that tumors use to hide from the immune system. The study will give TTX-080 alone or combined with other cancer drugs (pembrolizumab, cetuximab, or FOLFIRI plus cetuximab) to abo…

This study tests an experimental drug called TransCon IL-2 β/γ, which aims to boost the immune system to fight cancer. It is given alone or with other treatments like pembrolizumab (Keytruda) or chemotherapy. The trial includes 320 adults with advanced solid tumors that have not …

This early-phase study is testing a new drug called JZP815 in people with advanced or spreading solid tumors that have specific changes in the MAPK pathway. The main goals are to check the drug's safety, find the right dose, and see if it can shrink tumors. About 332 adults who h…

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 early-phase trial tested two oral drugs, cediranib and selumetinib, in 19 people with advanced solid tumors (including melanoma) that had stopped responding to standard treatments. Cediranib works by cutting off blood supply to tumors, while selumetinib blocks a key growth s…

This study looks at people with severe aortic stenosis (a narrowed heart valve) who don't yet have symptoms. Doctors usually wait until symptoms appear before replacing the valve, but by then the heart may already be damaged. The researchers use MRI scans to find hidden scarring …

People with MEN1 syndrome inherit a high risk of developing multiple pancreatic neuroendocrine tumors. Surgery is the only curative option but causes major complications. Researchers are testing high-dose, high-precision MR-guided radiotherapy to see if it can shrink these tumors…

This early-phase trial tests two drugs together—MK-2206 and hydroxychloroquine—in people with advanced solid tumors, melanoma, kidney, or prostate cancer. The goal is to find the safest dose and see how the combination affects cancer cell growth. About 62 participants will take b…

Researchers are studying whether daily injections of vosoritide can safely improve long-term growth in children with hypochondroplasia, a genetic condition that causes short stature. The trial enrolls about 140 children who have already taken vosoritide in earlier studies and who…

Researchers are testing tividenofusp alfa (DNL310), an experimental enzyme replacement therapy, against the standard treatment idursulfase in children and young adults with mucopolysaccharidosis type II (MPS II). The study includes participants with and without brain involvement,…

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 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 study tests the long-term safety and effects of JR-141, an enzyme replacement therapy, in people with mucopolysaccharidosis II (MPS II), a rare genetic disorder. Participants who completed an earlier JR-141 trial receive the drug again by IV infusion. Researchers track side …

This UK trial tests whether cladribine tablets, taken for just 8-10 days a year over two years, can slow the loss of hand function in people with advanced multiple sclerosis (MS). The study includes 204 participants with significant disability (EDSS 6.5-8.5). Half receive cladrib…

This trial tests an experimental enzyme replacement called GC1130A in children with Sanfilippo syndrome type A, a rare genetic disease that damages the brain. The enzyme is given through a small device placed under the scalp that delivers it directly into the fluid around the bra…

This phase 2 trial is testing whether adding a personalized mRNA cancer vaccine (mRNA-4157) to the immunotherapy drug pembrolizumab can better prevent melanoma from coming back after surgery compared to pembrolizumab alone. The study involves 267 people with high-risk melanoma th…

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

This trial tests an experimental drug called JR-441 in children with mucopolysaccharidosis type IIIA (MPS IIIA), a rare genetic condition that harms the brain and body. Researchers give the drug as a weekly intravenous infusion to see if it is safe and tolerable. They also measur…

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 early-stage trial tests a combination of three drugs—lenalidomide, dinutuximab, and isotretinoin—in children whose neuroblastoma has not responded to treatment or has come back. The main goal is to find the safest dose of lenalidomide when used with the other drugs. Research…

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 whether giving the immunotherapy drug nivolumab before and after surgery for stage III melanoma can help prevent the cancer from returning. About 67 adults with resectable melanoma will receive one dose of nivolumab before surgery, then after surgery they will ge…

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 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 trial tests whether JR-141, an enzyme replacement therapy given as a weekly IV infusion, can safely manage mucopolysaccharidosis type II (MPS II) over the long term. Researchers will follow about 27 people who completed an earlier JR-141 study, tracking side effects, develop…

This study tests whether adding two targeted pills (encorafenib and binimetinib) to standard immunotherapy (pembrolizumab) works better than immunotherapy alone for people with advanced melanoma that has a BRAF gene mutation. About 257 adults with untreated, advanced or spreading…

This early-stage trial tests an experimental mRNA-based cancer vaccine (mRNA-4157) in 161 people with solid tumors. The vaccine is designed to train the immune system to attack cancer cells. Some participants receive the vaccine alone, while others get it with the immunotherapy d…

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

This early-stage study tests a new injectable drug called LYN101 in 44 people. First, healthy volunteers get a single dose to check safety. Then, postmenopausal women with low bone mass get multiple doses to see how the drug affects bone turnover markers. The main goal is safety,…

This study tests a new drug called INCB099280 in 73 people with advanced solid tumors who have not received immunotherapy before. The goal is to see if the drug can shrink tumors and to check for side effects. The study focuses on safety and how well the drug works in the short t…

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 trial compares two treatment strategies for people with relapsing-remitting multiple sclerosis: starting with high-efficacy drugs early versus starting with standard first-line therapies and switching if needed. The study involves 900 participants across multiple sites and t…

This early-phase trial tests whether adding an oral drug (decitabine/cedazuridine) to the immunotherapy nivolumab can help the immune system better fight mucosal melanoma, a rare and aggressive skin cancer. The study includes 8 adults with advanced disease that cannot be surgical…

This early-stage trial tests FF-10850, a liposome-wrapped version of the chemotherapy drug topotecan, in 96 adults with advanced solid tumors like ovarian, cervical, and Merkel cell carcinoma. The main goals are to find the safest dose and understand side effects. Researchers hop…

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 whether combining high-dose interleukin-2 with low-dose ipilimumab, followed by nivolumab, can shrink tumors in people with advanced melanoma that has stopped responding to prior immunotherapy. About 29 participants with inoperable stage III or IV melanom…

This study is testing a new type of intraocular lens (IOL) that can be adjusted after cataract surgery using laser energy, potentially allowing the eye to focus at different distances. Researchers will enroll 85 adults with cataracts in both eyes to check safety and how well the …

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

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

This study tests a new three-drug combination (tazemetostat, dabrafenib, and trametinib) in people with advanced BRAF-mutated melanoma whose cancer worsened despite prior BRAF/MEK inhibitor therapy. The goal is to find the best dose and see if the combo can slow tumor growth. Abo…

This phase 2 trial is testing whether a combination of two drugs, talazoparib and temozolomide, can shrink tumors in people with rare cancers that have spread. The study includes 14 participants with advanced rare solid tumors, such as pheochromocytoma or paraganglioma, who have …

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

This trial asks whether starting with highly effective MS drugs right away is better than starting with milder drugs and switching to stronger ones only if needed. Researchers will follow about 800 adults with relapsing-remitting MS for several years, using MRI scans and disabili…

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 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 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 compares two different TAVI devices (Evolut FX and Sapien 3 Ultra Resilia) used to replace a narrowed heart valve in people with aortic stenosis. About 1,346 patients will be randomly assigned to get one device or the other. Researchers will track deaths, strokes, valv…

This study tests whether high-intensity interval training (HIIT) can improve cardiovascular health in adults who had surgery to repair coarctation of the aorta, a narrowing of the main artery. Participants are randomly assigned to do HIIT at home, HIIT in a supervised clinic, or …

This study looks at people with severe aortic stenosis who need a new heart valve (TAVI). Normally, doctors do a coronary angiogram first to check for blocked arteries. But this test can cause kidney damage or bleeding, especially in older patients. The trial will compare the usu…

This trial is testing three different drug combinations given before surgery for advanced melanoma. The goal is to see which combination best shrinks the tumor before it is removed and helps prevent the cancer from coming back after surgery. The study involves adults with resecta…

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 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 tests a new tool called the Snouda Metabolic Score (SMS) that helps doctors identify the specific metabolic problems driving Type 2 Diabetes in each patient. 150 adults will follow a personalized 24-week lifestyle and nutrition plan based on their SMS results. The goal…

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 trial is investigating whether adding a short course of targeted radiation to a standard immunotherapy combination (ipilimumab and nivolumab) can improve outcomes for people with metastatic melanoma. Participants will receive the two immunotherapy drugs, and some will also r…

This study tests a new growth hormone medicine called somapacitan, given once a week, in 47 children who are very short due to being born small for gestational age, or having Turner syndrome, Noonan syndrome, or idiopathic short stature. The main goal is to see if it is safe and …

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

This study is testing a new type of hip resurfacing device made entirely of ceramic, designed for people with hip osteoarthritis, necrosis, or dysplasia. The goal is to see if it is as safe and effective as existing metal-on-metal devices, but without releasing metal ions into th…

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 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 combination of three immunotherapy drugs (INCMGA00012, INCAGN02385, and INCAGN02390) in 12 adults with advanced Merkel cell carcinoma that continued to grow after standard treatment. The drugs are given by IV to help the immune system fight the cancer. The main…

This trial is testing a new type of heart valve made from a material that the body absorbs over time. It is designed for children and young adults who need surgery to fix a congenital heart defect affecting the passage from the heart to the lungs. The valve is implanted during su…

This early-stage trial is testing an experimental oral drug, TYRA-200, in people with advanced bile duct cancer (intrahepatic cholangiocarcinoma) and other solid tumors that have specific changes in the FGFR2 gene. The study aims to find a safe and effective dose and to see wheth…

This trial compares two types of transcatheter heart valves—one designed to sit above the natural valve opening (supra-annular) and one that sits within it (annular)—in people with severe aortic stenosis and a small aortic annulus (≤23 mm). The goal is to see which valve leads to…

This study is testing a new heart valve called ACURATE neo2 in 51 patients with severe aortic stenosis, a condition where the heart's main valve is narrowed. The valve is placed using a tube through an artery, avoiding open-heart surgery. Researchers are tracking how many patient…

This trial tests whether a personalized training program called 'Kleuter Extra' can improve executive functioning—skills like attention, self-control, and planning—in preschoolers aged 4 to 6 who have severe congenital heart disease. Many children with this condition face thinkin…

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 trial investigates whether a single or repeat infusion of zoledronate, or daily oral alendronate, can maintain the bone density improvements achieved with denosumab in postmenopausal women with osteoporosis. Participants who have been on denosumab for at least three years wi…

This trial is for people with advanced cancers who have already responded well to 6 months of standard immunotherapy. It compares continuing the same drugs at the usual frequency versus giving them every 3 months. The goal is to see if the reduced schedule can prevent cancer prog…

This study tests a one-time gene therapy for children with Hurler syndrome, a severe genetic disorder. Doctors take the child's own blood stem cells, add a working copy of the missing gene, and put them back. The goal is to help the body produce the enzyme it lacks, potentially s…

This study tests an oral drug called infigratinib in children with hypochondroplasia, a condition that causes short stature. The trial has two parts: first, all children receive the drug to see if it safely increases their growth rate; then, some will switch to a placebo to confi…

This study looks at whether giving an extra balloon inflation after placing a new heart valve (TAVI) helps the valve open more evenly and work better. About 146 people with severe aortic stenosis will be randomly assigned to receive this extra step or not. The goal is to see if i…

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 study tests whether choline, a vitamin-like nutrient, can improve memory, thinking, and behavior in children aged 2.5 to 5 years with Fetal Alcohol Spectrum Disorders (FASD). Participants receive choline daily for either 3 or 6 months. Researchers measure changes in memory, …

This trial tests a long-acting injection of leuprolide acetate in children with central precocious puberty, a condition where puberty starts too early. The goal is to suppress puberty-related hormones, slowing or stopping early development. The study includes children aged 4 to 9…

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 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 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 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 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 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 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 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-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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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-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 2 trial is testing a new drug called NVG-2089 in 60 adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a condition where the immune system attacks nerves. The drug is designed to mimic a natural protein (IVIg) to reduce inflammation and improve nerve…

This study tests a new heart valve called the NAVITOR, placed using a thin tube (catheter) without open-heart surgery. It includes 434 people with severe aortic stenosis who are at intermediate or low risk for surgery. The main goals are to check for leaks around the valve at 30 …

This early study is testing a new balloon-expandable heart valve device (TAVI) in 8 people with severe aortic stenosis who are at increased surgical risk. The goal is to see if the device can be safely delivered and perform as intended. Because this is a first-in-human study with…

This study compares two bone cements used to treat painful spinal fractures caused by osteoporosis. One cement includes a softener (V-Flex) to make it less rigid, while the other is standard cement (V-Steady). The goal is to see if the softer cement reduces the chance of new frac…

This study tests whether the immunotherapy drug avelumab can help prevent Merkel cell carcinoma, a rare skin cancer, from returning after initial treatment. About 122 adults with stage I to III Merkel cell carcinoma will receive either avelumab or a placebo. The main goal is to s…

This study tests three ways emergency responders help children breathe: a bag-mask, a throat tube, or a breathing tube. It includes 3,000 children under 18 with cardiac arrest, severe injury, or breathing failure. The goal is to see which method leads to more days alive and out o…

This study looks at whether injecting special stem-like cells called Muse cells directly into the ovaries can help women aged 28 to 70 who are in perimenopause. The cells may repair tissue, reduce inflammation, and improve hormone balance. Researchers will track safety, hormone l…

This early-phase trial tests a new type of immune therapy for children with neuroblastoma that has come back or not responded to treatment. Researchers take a patient's own T cells, add new genes to help them recognize and kill neuroblastoma cells and survive longer, then infuse …

This study tests whether high-dose nicotinamide (vitamin B3) is safe and can help people with dominant optic atrophy, a rare genetic disease that slowly damages the optic nerve and causes vision loss. Researchers will give 25 adults 3 grams of nicotinamide daily and monitor for s…

This study looks at whether people over 50 with a stable, inactive form of secondary progressive multiple sclerosis can stop taking their disease-modifying drugs without getting worse. Researchers will track 250 participants for 2 years to see if stopping treatment leads to more …

This study tests whether adding radiation therapy (either external beam or a targeted radioactive drug called Lutathera) to the immunotherapy avelumab can help control metastatic Merkel cell carcinoma. About 19 adults with advanced disease will receive avelumab every two weeks fo…

This phase 2 trial tests a gel containing sirolimus (PTX-022) on 15 people with visible venous malformations on the skin. The goal is to see if the gel is safe and can improve the appearance and symptoms of these abnormal veins. Participants apply the gel daily for 12 weeks, and …

This early-stage trial tests a combination of three drugs—sorafenib, cyclophosphamide, and topotecan—in children and young adults with neuroblastoma that has relapsed or not responded to treatment. The main goal is to find the highest safe dose of sorafenib when given with the tw…

This study tests whether a special fish-oil emulsion given through an IV can improve outcomes for newborns with a diaphragm defect that causes severe lung pressure. About 40 babies will receive either the fish-oil emulsion or a standard soy-oil one. Researchers will measure lung …

This study tests whether a low dose of pazopanib, a drug currently used for cancer, can reduce severe nosebleeds and improve anemia in people with hereditary hemorrhagic telangiectasia (HHT). About 70 participants will take either the drug or a placebo daily for 24 weeks. Researc…

This early-stage trial tests a radioactive drug called CLR 131 in 30 children and young adults with solid tumors, lymphoma, or brain tumors that have come back or not responded to standard treatments. The main goals are to find a safe dose and see if the drug can shrink tumors. P…

This study looks at a special balloon coated with medicine that is used to open up blocked dialysis fistulas in the arm. The goal is to see if it keeps the fistula working longer and safely. About 213 people took part in this global study.

This early-phase trial tests a new type of cell therapy for children and young adults with solid tumors that have come back or not responded to standard treatments. The therapy uses a patient's own immune cells, which are trained in the lab to recognize and attack multiple cancer…

This study tests whether N-acetyl cysteine (NAC), a supplement, can protect the brain in people with progressive multiple sclerosis (MS). About 98 adults with primary or secondary progressive MS will receive either NAC or a placebo for 15 months. Researchers will measure brain sh…

This study looks at whether switching from anti-CD20 drugs (like ocrelizumab) to cladribine can help restore immune function and reduce infections in people with relapsing multiple sclerosis. About 70 participants will be followed for 2 years. The goal is to see if the switch imp…

This study looks at the long-term safety and effects of a daily injection called BMN 111 in children with achondroplasia, a common form of dwarfism. It includes 30 children who have already taken the drug for two years in a previous study. The goal is to see if the drug remains s…

This early-phase trial tests whether a patient's own bone marrow stem cells, injected into the spinal fluid, can repair nerve damage caused by progressive multiple sclerosis. The study involves 18 adults with secondary or primary progressive MS. Researchers will measure changes i…

This early-stage trial tests a new approach for advanced melanoma that has not responded to standard treatments. Nine patients will receive a modified virus (TILT-123) designed to deliver immune-boosting signals directly into tumors, along with their own tumor-fighting immune cel…

This study looks at whether the drug bevacizumab can help people with a rare condition called hereditary hemorrhagic telangiectasia (HHT) who have severe liver problems leading to high heart output and possible heart failure. Researchers will check heart function before and after…

This study tests a drug called lanreotide in 10 people with advanced or metastatic paraganglioma or pheochromocytoma, rare tumors that release hormones. The goal is to see if lanreotide can slow tumor growth compared to before treatment. Participants receive injections every 4 we…

This study tests a daily combination of sirolimus, celecoxib, and low-dose chemotherapy in 46 children whose solid or brain tumors have returned or not responded to standard treatment. The goal is to see if this approach can shrink tumors or stop them from growing. While not a cu…

This early-phase trial tests a new treatment for people with advanced melanoma that cannot be removed by surgery or has spread. The therapy uses a patient's own immune cells (tumor-infiltrating lymphocytes) grown in a lab and then given back along with a drug called ANV419 to hel…

This early-stage trial is testing whether a combination of two drugs, naporafenib and trametinib, can shrink or control advanced solid tumors that have a specific genetic change called RAS Q61X. About 86 people whose cancer has not responded to standard treatments will receive th…

This study compares two ways to place dental implants in people with bone loss. One technique places the implant at the gum level (tissue-level), while the other places it at the bone level and requires a second surgery to uncover it. Researchers will track bone changes, implant …

This study looks at two kinds of plastic used in total knee replacement surgery: one with vitamin E and one without. The goal is to see which one lasts longer, causes less wear, and helps patients move better. About 605 adults with knee arthritis or similar problems took part, an…

This study tests whether adding the drug abatacept to standard care can prevent graft-versus-host disease (GVHD) in children receiving stem cell transplants from unrelated donors. GVHD occurs when donor cells attack the patient's body, causing serious illness. The trial will enro…

This phase 3 study tests an oral medication called IMU-838 against a placebo in about 1,100 adults with relapsing multiple sclerosis (MS). The goal is to see if the drug can reduce the number of relapses and new brain lesions over 72 weeks. Participants must be 18-55 years old wi…

This study tests a medicine called setrusumab in people with osteogenesis imperfecta (brittle bone disease). The goal is to see if it can lower the number of bone fractures. About 183 participants with types I, III, or IV of the disease who have had recent fractures will receive …

This Phase 3 trial tests whether IMU-838 (vidofludimus calcium) can reduce relapses and brain lesions in adults with relapsing multiple sclerosis. Over 1,100 participants are randomly assigned to receive either the drug or a placebo for up to 72 weeks. The study is active but no …

This phase 3 trial tests whether ocrelizumab (Ocrevus) can slow disability progression in adults with primary progressive multiple sclerosis (PPMS). Over 1,000 participants receive either the drug or a placebo. The main goal is to see if the drug delays worsening of hand function…

This study tests a new medicine called setrusumab against standard bone-strengthening drugs (bisphosphonates) in children aged 2 to under 7 with osteogenesis imperfecta (brittle bone disease) types I, III, or IV. The goal is to see if setrusumab can lower the number of bone break…

This study tests whether a device called Sonablate can safely use high-intensity focused ultrasound (HIFU) to treat vein problems like varicose veins, leg ulcers, and certain birth defects. About 30 adults with these conditions will receive the treatment, which does not involve c…

This study is tracking 200 people with blocked leg arteries who are receiving a special balloon coated with the drug paclitaxel (GENOSS PCB). The balloon is inflated inside the artery to open it and release the drug, which helps prevent the artery from narrowing again. Researcher…

This study tests whether giving two weekly injections together can improve growth in children aged 2 to 11 with achondroplasia, a common cause of dwarfism. The drugs, navepegritide and lonapegsomatropin, are designed to increase height. The trial will measure growth rate and moni…

This Phase 2 trial tests whether infusions of special immune cells (tolerogenic dendritic cells) can reduce brain lesions and relapses in people with relapsing-remitting multiple sclerosis. The 45 participants also take standard MS drugs. The study aims to see if this cell therap…

This phase 3 trial tests whether upadacitinib (Rinvoq) can help people with Takayasu arteritis, a rare disease that inflames large arteries. About 56 adults who have had a recent relapse despite steroids will receive either upadacitinib or a placebo, along with a steroid taper. T…

This study compares two newer self-expanding heart valves and a minimalist approach (using only local anesthesia, avoiding extra tubes) against standard care for people with severe aortic valve stenosis. About 836 participants will be randomly assigned to receive either the new v…

This Phase 2 trial tests a genetically modified herpes virus (RP1) alone or with the immunotherapy drug nivolumab in people with advanced solid tumors, including melanoma, non-small cell lung cancer, and certain skin cancers. The study aims to see if the combination is safe and c…

This study is testing a radioactive drug called Lutathera in teenagers aged 12 to 17 who have rare tumors of the digestive system or adrenal glands. The goal is to see if the drug is safe and how much radiation reaches organs. Eleven teens are taking part, and researchers are wat…

This early-stage trial tests a gene therapy called AAV9.hMCOLN1co in one child with Mucolipidosis Type IV (MLIV), a rare genetic disorder. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety, but researchers will also look for any si…

This study tests an experimental drug called AGA2118 in postmenopausal women aged 55 to 80 who have low bone density but no history of fractures. The goal is to see if the drug can safely increase bone mineral density over 12 months compared to a placebo. About 379 women will par…

This phase 2 trial is testing whether one year of pembrolizumab (an immunotherapy drug) works as well as the standard two-year course for people with advanced melanoma that cannot be surgically removed. The study involves 29 participants in Brazil's public health system and measu…

This study is testing whether the drug ocrelizumab (Ocrevus) can improve signs of inflammation in the spinal fluid of people newly diagnosed with relapsing multiple sclerosis (MS) or a high-risk first attack (clinically isolated syndrome). Thirty participants will receive ocreliz…

This study tests whether giving the immunotherapy drug pembrolizumab before and after surgery can help prevent head and neck mucosal melanoma from coming back. About 60 adults with resectable tumors will receive pembrolizumab, then surgery and possibly radiation, followed by more…

This study tests a new, less invasive heart valve (SAPIEN 3) for people with severe aortic stenosis who are at low risk for standard surgery. About 1,000 participants will be randomly assigned to get the new valve or traditional open-heart surgery. The goal is to see if the new v…

This study tests whether giving a short course of chemotherapy plus immunotherapy before surgery can improve outcomes for people with Merkel cell carcinoma, a rare skin cancer. About 36 adults with operable tumors will receive one cycle of retifanlimab and platinum-etoposide befo…

This phase 2 trial tests a new drug called roginolisib against standard treatments in 85 adults with advanced uveal melanoma, a rare eye cancer. The study aims to see if roginolisib helps people live longer and improves their quality of life. Participants are randomly assigned to…

This early-phase trial tests a new approach to treat advanced solid tumors by giving patients a large dose of their own immune cells that are specially selected to attack cancer. The cells are taken from the tumor, multiplied in the lab, and then infused back. Some patients also …

This study compares two procedures used to increase blood flow to the lungs in newborns with congenital heart disease: a surgical shunt or a stent placed via catheter. About 300 babies will be randomly assigned to one method and followed for their first year. The goal is to see w…

This Phase 3 trial tests a new gene therapy called OTL-203 against the standard stem cell transplant for children with Hurler syndrome, a rare genetic disorder. The therapy uses the child's own blood stem cells, modified to produce the missing enzyme, aiming to improve survival a…

This expanded access program provides a genetically modified herpes virus (vusolimogene oderparepvec) combined with the immunotherapy drug nivolumab to adults with advanced melanoma that has worsened after anti-PD-1 treatment. The goal is to offer potential benefit to patients wh…

This study planned to test a combination of immunosuppressive drugs (methotrexate, rituximab, bortezomib, IVIg, and folic acid) in people with hypophosphatasia whose bodies had stopped responding to asfotase alfa due to antibodies. The goal was to see if suppressing the immune sy…

This study tests whether the drug eflornithine, taken as a pill after immunotherapy, can help prevent high-risk neuroblastoma from coming back in children. Twenty children under 18 who have completed standard treatment will take eflornithine for maintenance. Researchers will trac…

This study tests whether the immunotherapy drug nivolumab, alone or combined with ipilimumab, can shrink melanoma tumors that have spread to the brain. About 76 adults with stage IV melanoma and at least one brain metastasis will receive treatment. The main goal is to see how man…

This early-stage study tests a new treatment called BOXR1030 for people with advanced solid tumors that have a specific marker (GPC3). The treatment uses specially designed immune cells given after chemotherapy to help fight the cancer. The main goals are to check safety and find…

This trial compares two types of replacement heart valves used in a procedure called TAVI for people with a narrowed aortic valve. One valve expands with a balloon, the other expands on its own. The study will track about 1,960 patients to see which valve leads to fewer deaths or…

This study tests a new surgical method called RADAR for creating blood vessel connections needed for dialysis in 200 people with end-stage kidney disease. The goal is to see if this technique leads to fewer blockages and better long-term function compared to the standard approach…

Researchers test whether an AI algorithm called AK-AVS can spot moderate-to-severe aortic stenosis, a narrowing of the heart's aortic valve, in older adults who have not been diagnosed. The study reviews about 4,000 routine electrocardiograms (ECGs) already collected during prima…

Researchers at Mayo Clinic are testing whether advanced PET and MRI brain scans can measure inflammation in people with multiple sclerosis and other inflammatory demyelinating diseases. The study includes about 200 participants, both patients and healthy controls. Participants re…

This study tests whether a new type of PET scan (18F-MFBG) can detect neuroblastoma tumors as accurately as the current standard scan (123I-MIBG). About 84 children and adults with newly diagnosed, high-risk neuroblastoma will receive both scans to compare results. If the new sca…

This study tests whether special lights and cameras can help find early signs of mouth cancer in people at high risk. About 338 participants with precancerous spots or conditions like Fanconi anemia will have their mouths examined with fluorescence imaging, which makes abnormal c…

Researchers at a Turkish hospital are studying how errors in bone density scans (DXA) affect doctors' decisions about osteoporosis. They will review scans from 530 adults, identify common positioning mistakes, correct them, and compare the original and corrected results. The goal…

This trial tests whether an AI tool called DeepBMD can identify people at high risk for osteoporosis using CT scans they've already had for other reasons. Researchers will compare the AI's findings with the standard bone density test (DXA) to see if it's accurate. If it works, th…

This trial tests an experimental imaging tracer called [11C]-CS1P1, which is designed to attach to a protein involved in brain inflammation. The study will first check the tracer's safety and how it moves through the body in healthy volunteers, then compare its signals in people …

This trial is developing an artificial intelligence system that reads electrocardiograms (ECGs) to detect congenital heart disease in children. Researchers are collecting ECG data from thousands of children, including those with common and rare heart defects, to train a deep-lear…

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 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 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 study tests whether a simple checklist can help general practitioners (GPs) better diagnose developmental dysplasia of the hip (DDH) in babies during routine 6-week checkups. About 172 GP practices in England will be split into two groups: one using the checklist and one con…

Researchers test whether flecainide, an antiarrhythmic drug, can prevent atrial arrhythmias in adults who have a patent foramen ovale (PFO) closed after a cryptogenic stroke. Participants receive flecainide or standard care for 3 to 6 months. An insertable cardiac monitor records…

This trial tests whether a year of twice-weekly supervised exercise can improve bone mineral density in women. Researchers will measure bone density and blood markers linked to bone turnover. The goal is to see if regular physical activity can help prevent osteoporosis and relate…

This study tests if teaching children and parents about avoiding certain chemicals (like BPA) can actually lower the levels found in kids' urine. About 700 children aged 6 to 8 in Belgium and Spain will take part in a school program over two and a half years. The goal is to see i…

This study tests a program called PREVENT designed to reduce hip fractures in long-term care homes. The program uses a risk calculator based on residents' health records to identify those at high risk, then trains healthcare staff on the latest fracture prevention recommendations…

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 tests whether a weekly dose of everolimus (a drug that targets aging pathways) and/or a fun exercise program called 'Football Fitness' can prevent the rapid bone loss that happens after menopause. 136 healthy women aged 45-60 will be assigned to one of four groups: exe…

This study compares three care models to prevent falls and fractures in older adults recovering from a broken bone in a skilled nursing facility. About 3,780 participants across 42 facilities will receive either medication reduction, bone health management, or both. The goal is t…

This study tests whether special slip-resistant shoes can help dairy workers avoid slipping on wet, slippery floors. About 22 full-time employees at a Danish dairy will wear different shoes and rate how slippery they feel each day. The goal is to find footwear that reduces fall r…

Researchers are testing whether a 12-week computerized cognitive training program can improve thinking and change brain activity in people with multiple sclerosis. The study enrolls 180 adults with MS who have mild to moderate disability. Participants train for one hour a day, th…

Researchers are testing whether wearing a lightweight robotic exoskeleton at home can improve quality of life for people with multiple sclerosis who have walking difficulties. The study includes 28 adults who use a walking aid and live at home. Participants wear the Keeogo exoske…

This early study tests whether short-term fasting (eating less than 200 calories per day for 3 days) before standard immunotherapy is safe and doable for people with advanced or metastatic skin cancer. Ten participants will receive their usual PD-1/PD-L1 inhibitor drugs along wit…

This study tests whether two special diets—a modified Paleolithic elimination diet and a time-restricted ketogenic diet—can improve quality of life, fatigue, and mood in 162 people with relapsing-remitting multiple sclerosis. Participants are assigned to one of the diets or stand…

This study is testing whether an online course can help people with multiple sclerosis, long COVID, fibromyalgia, or cancer-related fatigue adopt healthier diets and self-care habits. Researchers will measure changes in quality of life, fatigue, and mental health. The goal is to …

Guillain-Barré syndrome can leave people with weak arms and hands, making daily tasks hard. This trial tests whether adding robot-assisted arm training to usual therapy helps adults in the sub-acute phase of recovery. Participants use the Armeo Power 2 exoskeleton for 20 sessions…

Researchers are testing whether a suture-based closure device helps patients recover faster after heart procedures that use a large tube placed in the femoral vein. The trial enrolls adults undergoing procedures such as atrial fibrillation ablation, leadless pacemaker insertion, …

This study tests a virtual behavioral therapy (Functional Behavioral Training) for children aged 2-12 with genetic syndromes like Fragile X, Angelman, or Rett syndrome who have challenging behaviors. The therapy teaches parents how to identify what triggers problem behaviors and …

This study tests if giving the drug dinutuximab over 4 hours or less is safe and practical for children with high-risk neuroblastoma. Currently, standard infusions take 10-20 hours and require a hospital stay. The goal is to see if a shorter infusion can be used in an outpatient …

People on hemodialysis receive needle sticks through a fistula or graft several times a week, and the pain and anxiety can build over time. This trial tests whether slow, deep breathing combined with lemongrass aromatherapy reduces pain and anxiety during needle insertion. Resear…

This study was designed to see if giving nitrous oxide (laughing gas) helps pregnant women feel less pain when a balloon is placed to start labor. It planned to compare nitrous oxide with plain oxygen. However, the study was withdrawn before any participants were enrolled, so no …

This study tests a new type of cycling called eccentric cycling for people with multiple sclerosis. Researchers want to see if it improves muscle strength, walking ability, fatigue, and quality of life better than standard cycling. Sixty adults with MS who can walk independently …

This trial tests a wearable device that uses mild electrical pulses to stimulate the leg muscles of people with multiple sclerosis who have foot drop. The goal is to see if using the device for several months improves walking quality, physical activity, and participation in daily…

This study tests a smartphone app called MS CATCH that lets people with multiple sclerosis regularly report their mood to their neurologist. The goal is to improve how doctors screen and treat depression in MS. About 100 adults with MS and mild-to-moderate depression are taking p…

This study looks at whether people with melanoma prefer getting their cancer medicine as a shot under the skin or through an IV. About 100 patients will try both ways and say which they like better. The goal is to make treatment more convenient and comfortable for patients.

This study tests two online programs designed to help people with multiple sclerosis (MS) understand and manage their fatigue. Researchers will enroll 2,000 adults with MS across the U.S., including veterans, and compare the programs to usual care alone. The goal is to find pract…

This study tests whether a six-week, nurse-led virtual group program can help adult women with Multiple Sclerosis (MS) find more meaning and purpose in life, and improve their quality of life. Participants attend weekly online group sessions focused on reflection and values explo…

This study tests a special laser to safely lift eyebrows, tighten loose skin under the chin, and reduce wrinkles on the face and jawline. About 40 adults aged 45 to 85 with mild to moderate skin sagging will receive up to three treatments. The goal is to see if the laser can impr…

This pilot trial tests whether people with multiple sclerosis can safely and satisfactorily use a telemedicine platform for balance rehabilitation at home. After completing intensive in-hospital rehabilitation, participants either follow a 12-week home telerehabilitation program …

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 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 tests whether a supervised, home-based exercise program can help people with metastatic cancer who are starting oral targeted therapy. Participants are randomly assigned to either a weekly 60-minute session with a trained coach or to a booklet with exercise recommendat…

This trial tests whether custom-designed sensorimotor insoles, worn daily for six months, can improve balance and walking in people with multiple sclerosis. Fifty adults with MS (mild to moderate disability) will be randomly assigned to receive the insoles or continue their usual…

People on hemodialysis need their fistula punctured with a needle three times a week, which can be painful and anxiety-provoking. This trial compares three approaches: usual care, applying an ice pack to a specific point on the hand before the needle, and using a numbing gel. Res…

This pilot study explores whether robot-assisted walking devices can be used in schools to help children who cannot walk independently. The devices attach to a walker and power leg movements, allowing children to stand and walk upright. The study aims to identify the best ways to…

This study tests whether a wearable device, used at home alongside pelvic floor therapy, can help people with multiple sclerosis manage bladder problems. Twenty women with MS will use the device for three months. The main goal is to see if the approach is practical and easy to us…

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 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 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 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 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 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 tests a cognitive rehabilitation program called CIRCuiTS, adapted for people with Multiple Sclerosis (MS). Twenty-four participants will either start the program right away or after a 13-week wait. The program involves up to 36 hours of therapist-led and independent se…

This study looks at whether improving diet quality and reducing calories can help physical function, thinking, pain, fatigue, mood, and anxiety in 100 adults with relapsing-remitting or secondary progressive multiple sclerosis. Participants receive prepared meals and behavioral s…

This study tests whether a 4-week telerehabilitation program can improve balance and quality of life in 36 people with multiple sclerosis or Parkinson's disease. Participants do balance exercises at home while monitored by a hospital physiotherapist. The results are compared to s…

This study tests whether a 9-week online cognitive behavioral therapy program can improve sleep in people with multiple sclerosis (MS). About 550 participants in Norway will either receive the digital therapy or digital education about insomnia. The main goal is to see if the the…

This study tests whether educational programs about exercise, Mediterranean diet, and mental strategies can improve quality of life and reduce fatigue in women with relapsing-remitting multiple sclerosis. About 60 participants will be assigned to either supervised in-person sessi…

This study tests a set of virtual reality exergames in the Metaverse to help people with neurological conditions like multiple sclerosis, Parkinson's disease, stroke, and traumatic brain injury improve their cognitive skills. 25 participants will try games like virtual cooking an…

This study tests whether a type of brain stimulation called rTMS can reduce depression in people with autism. Twenty-four participants aged 13 to 26 will receive either real or sham (fake) stimulation to see if it helps. The goal is to find a new way to treat depression that hasn…

This study tests whether venglustat can reduce nerve pain in the arms and legs, and belly pain, in people with Fabry disease. Participants are 16 or older and have not had Fabry treatment for at least 6 months. The trial lasts 12 months, with visits every 3 months, and compares v…

This early-phase study tests a new type of proton therapy called Pro-GRID for people with large tumors (over 7 cm) that are hard to treat. The goal is to see if the treatment is safe and can shrink tumors or ease symptoms. About 11 adults with sarcoma or melanoma are taking part.

This study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …

This study will test whether taking a daily DHA (omega-3) supplement and doing therapeutic exercise three times a week can improve strength, fatigue, thinking, and quality of life in 100 people with multiple sclerosis. Participants will be randomly assigned to one of four groups:…

This study tests a gamified app called MITA designed to improve language and thinking skills in children with autism aged 2 to 5. Sixty children will be randomly assigned to either use the app alongside their usual therapy or continue with standard care alone. The goal is to see …

This early-stage study tests whether a novel light therapy, designed to reset the body's internal clock, is safe and can reduce fatigue in people with progressive multiple sclerosis. Twenty participants will receive the light therapy while researchers monitor side effects and mea…

This study tests a 6-week group program called Working Out Dads (WOD) to see if it helps reduce mental health struggles in fathers of children aged 0-4. About 293 dads with mild to moderate distress will either join the WOD peer-support group or receive usual care. The goal is to…

This study tests a 6-month online program designed to help people with multiple sclerosis become more physically active. Participants receive interactive video lessons, a step tracker, and one-on-one video chats with a coach. The goal is to see if the program increases daily acti…

Researchers are sequencing the gut microbiomes of people with a wide range of conditions, from autism and Alzheimer's to Crohn's disease and multiple sclerosis, to see whether bacterial patterns differ by disease. The study also enrolls healthy participants for comparison. By mat…

This early-phase trial tested a special PET/CT imaging agent, Zirconium Zr 89 Crefmirlimab Berdoxam, to see if it can measure immune activity in tumors of patients with advanced melanoma. Six adults on immunotherapy plus hydroxychloroquine received scans before and after starting…

This study looks at oxygen levels in the kidneys of premature babies born before 32 weeks who have a heart condition called patent ductus arteriosus (PDA). Researchers want to see how kidney oxygen relates to the severity of the heart condition and whether it can predict kidney i…

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…

Researchers are running an international registry to follow adults who had an ischemic stroke or transient ischemic attack and also have a patent foramen ovale, a small opening between the upper chambers of the heart. The registry collects real-world information on how these pati…

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 follows 126 melanoma survivors who are receiving immunotherapy after surgery. Researchers will track side effects and quality of life for up to 24 months. They will also collect blood samples to look for markers that might predict who gets side effects. The goal is to …

This study reviews medical records of 72 American Indian patients treated with immunotherapy for cancers like lung cancer, melanoma, and kidney cancer. Researchers compare their outcomes—such as how long the cancer is controlled and side effects—to those of White patients. The go…

Researchers are studying how advanced and metastatic melanoma is diagnosed and treated in everyday Spanish clinics. The study follows about 400 adults with stage III, metastatic, or unresectable melanoma from their first diagnosis. No experimental treatment is given; instead, the…

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,…

Researchers are studying how people with relapsing multiple sclerosis use ofatumumab in everyday practice in Germany and Japan. The study looks at insurance claims from about 5,000 adults to see how many stay on the treatment and how regularly they take it. It also tracks how oft…

Researchers are checking whether tests of muscle strength, walking, balance, and fine motor skills give consistent results in people with polyneuropathy. About 520 adults with various forms of nerve damage, plus healthy volunteers, take part. Participants complete physical tests,…

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

Researchers at Karolinska Institutet are surveying adults in Sweden who have Chiari malformation type 1, a condition where part of the brain pushes into the spinal canal. The study asks how these patients rate their health-related quality of life compared with the general Swedish…

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 looks at children having surgery to remove a common neck lump called a thyroglossal duct cyst. Some kids will have a small drain placed during surgery, and some will not. The goal is to see if using a drain reduces problems like infection, fluid buildup, or the need fo…

Researchers are collecting blood and skin samples from people with ALS, people with MS, and healthy volunteers. They turn skin cells into stem cells and grow them into 3D organoids that mimic the brain and spinal cord. The goal is to see whether secretions from human amniotic mes…

This study aims to find blood-based biomarkers that signal the presence and size of plexiform neurofibromas in people with Neurofibromatosis type 1 (NF1). Researchers will collect blood samples and use whole-body MRI to compare biomarker levels in 200 participants with different …

This study is testing a new video game designed to help teenagers with congenital heart disease learn how to manage their own healthcare. The game teaches skills like talking to doctors, understanding medical information, and keeping track of appointments and medications. About 1…

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

This long-term study follows over 1,500 people of all ages with hypophosphatasia (HPP), a rare bone disease. Researchers will collect information on how the disease progresses, its impact on daily life, and the long-term safety and effectiveness of the treatment asfotase alfa. Th…

Researchers in Uzbekistan are studying common respiratory and heart conditions in children from birth to age 15. They will analyze medical records and examine blood samples to understand immune and antioxidant function in affected children. The goal is to develop and test improve…

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 looks at whether measuring IL-6 and lactate in cord blood can detect early signs of infection or inflammation in full-term newborns. Researchers will compare 160 women whose fetal heart rate patterns suggest possible inflammation with those whose patterns are normal. I…

Researchers are studying the natural fidgety movements of infants to see if these movements can help predict cerebral palsy and other developmental issues. They will follow 350 babies, including healthy full-term infants and those at higher risk due to medical conditions, trackin…

This study looks at whether analyzing tumor DNA in the blood can help monitor melanoma after surgery. Researchers will collect blood samples from 136 patients before and after surgery, and every 3-4 months, to see if changes in DNA levels predict when a treatment stops working. T…

This study follows 200 adults in Western Denmark who are having a transcatheter aortic valve implantation, a procedure to replace a narrowed heart valve. Researchers will measure quality of life before the procedure and at follow-up using a standard questionnaire. They will also …

Researchers at Amsterdam UMC are studying tumor samples from 180 people with advanced melanoma or pancreatic cancer that has stopped responding to immunotherapy. Participants give one extra tumor biopsy and a blood sample. The team will analyze the tumor's full genetic code and t…

This observational study follows about 2,400 people with relapsing-remitting multiple sclerosis (MS) across Germany for up to six years. It asks whether loss of volume in the thalamus—a deep brain region—tracks with worsening disability, measured by the EDSS scale. Using standard…

This study is testing whether a blood test (liquid biopsy) can detect a rare but serious nerve tumor called MPNST earlier than current methods in adults with Neurofibromatosis Type 1 (NF1). Researchers will compare the timing of tumor detection by the blood test versus standard i…

This study will interview 35 adults in Sweden who have Fabry disease but are not yet receiving treatment. The goal is to learn how they experience their quality of life, daily activities, and healthcare support. By listening to patients' own stories, researchers hope to identify …

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 aims to see if a structured education program based on the Health Belief Model can improve osteoporosis awareness and health beliefs in women aged 45 and older who have at least one risk factor for the disease. About 74 women will be randomly assigned to either receive…

This study is a long-term follow-up for 100 people who completed a previous trial of bel-sar, an experimental treatment for early choroidal melanoma (a type of eye cancer). Participants will not receive any new treatment but will be monitored for about 5 more years to check for s…

This study explores whether a brief, moderate-intensity walk can alter brain function and blood flow in people with multiple sclerosis. Participants will undergo MRI scans before and after either treadmill or overground walking at a brisk pace. The goal is to see if even a single…

This study follows 300 people with multiple sclerosis to see whether advanced MRI scans, eye tests, and blood markers can better predict when disability will worsen. Current tools are not accurate enough to guide early treatment for each individual. The researchers will compare t…

This observational study follows thousands of people with multiple sclerosis (MS) over many years to identify factors that predict how the disease will progress. Researchers collect detailed clinical data, MRI scans, and quality-of-life questionnaires from participants treated at…

This study looks at tumor samples from over 3,000 people with advanced cancers to find specific biomarkers—unique features of each cancer. The goal is to help doctors choose the most promising targeted therapy or clinical trial for each patient. Participants are adults with vario…

This study uses a new, child-friendly brain imaging technique called OPM-MEG to understand how immune-related brain diseases (like autoimmune encephalitis and multiple sclerosis) and other brain conditions affect children aged 2 to 25. Standard scans often miss problems with memo…

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 follows 500 people diagnosed with uveal melanoma (a rare eye cancer) for five years after treatment. Participants fill out questionnaires about their physical and mental well-being, treatment side effects, and decision-making. The goal is to learn how different treatme…

This study follows 50 adults in the UK with advanced BRAF V600 mutation-positive melanoma who are starting treatment with encorafenib and binimetinib. Instead of a controlled trial, it observes how these drugs work in real-world settings, focusing on patients' quality of life ove…

This study was designed to see if a test using acetylcholine could predict whether people with congenital heart disease and high blood pressure in the lungs would develop worsening high blood pressure after surgery to close a heart shunt. The test would have measured how well the…

This study looks at two different oxygen level targets for newborns who have trouble breathing or a condition called pulmonary hypertension (high blood pressure in the lungs). About 54 babies born at 35 weeks or later are randomly assigned to one of two oxygen goals. Researchers …

This study looks at whether a heart valve replacement procedure called TAVI can be safely performed in hospitals that do not have on-site cardiac surgery backup. It involves 657 patients with severe aortic stenosis who are at high risk for surgery. The same experienced doctors pe…

This study checks whether two easy-to-use risk calculators (OST and MORES) can accurately predict osteoporosis in Turkish men aged 50-75, compared to the standard DEXA bone density scan. Researchers will enroll 140 men to see how sensitive and specific these tools are. If they wo…

This observational study follows 36 people with neurologic conditions like traumatic brain injury, cerebral palsy, multiple sclerosis, or Alzheimer's who are already receiving intranasal MuSE stem cell or exosome therapy as part of their routine care. Researchers track safety and…

This study looks for a biomarker to identify people at risk of bone loss from glucocorticoid medications like prednisolone. 36 healthy volunteers will take either prednisolone or a placebo for seven days. Researchers will measure bone turnover markers in blood and collect bone, m…

This study is looking for the genetic causes of bicuspid aortic valve disease, a common heart condition where the aortic valve has two flaps instead of three. Researchers will analyze DNA from 11,000 participants, including patients and their relatives, to find gene changes linke…

This study gathers genetic and health information from over 6,400 people with bicuspid aortic valve, a common heart defect. Researchers aim to find the genes responsible for the condition and why some patients later develop serious aortic disease. No treatment is given; the goal …

This study looks at freezing ovarian tissue in girls with Turner syndrome, a condition that often causes early ovary failure and infertility. Researchers want to find out which patients are most likely to have healthy eggs in their tissue by checking their genes, hormones, and me…

This study looks at whether treating obstructive sleep apnea (OSA) with a breathing device (CPAP) lowers blood pressure more in people who are very sleepy compared to those who are not. Researchers will analyze data from about 10,000 adults with OSA from previous studies. The goa…

This study will examine lymph nodes from 5 people with relapsing-remitting multiple sclerosis who are starting ocrelizumab treatment. The goal is to see how effectively the drug removes B cells from lymph nodes, since these cells are known to be important in MS but are not well s…

This study was designed to test a wearable sensor system that tracks how people walk on different surfaces like grass, pavement, and stairs. It aimed to include healthy volunteers and people with conditions such as Parkinson's disease, dementia, stroke, multiple sclerosis, or low…

This small pilot study is testing whether a 30-minute stationary bike ride on the same day as immunotherapy can help the treatment work better for people with skin cancers like melanoma. The study involves 12 patients and focuses on whether this exercise plan is practical and saf…

This study collects tumor tissue, blood, and saliva from 2500 people with various solid cancers to analyze their genetic makeup. The goal is to understand why some tumors become resistant to targeted therapies. Samples may also be grown in special mice to study how genetics affec…

This study compares two dental devices—the quadhelix and mini-hyrax expanders—in 24 children with cleft lip and palate who need their upper jaw widened before bone graft surgery. Researchers will use 3D scans and models to measure how much the jaw expands. The goal is to see whic…

This study examines whether removing the breathing tube (extubation) in the operating room versus later in the intensive care unit improves outcomes for newborns undergoing surgery for myelomeningocele, a type of spina bifida. Researchers will review records of 60 babies to compa…

This study aims to find a combination of markers—from genetics, immune system, and brain scans—that are linked to how severe multiple sclerosis (MS) becomes. Researchers will follow 120 people with MS and their siblings who also have the condition. Participants undergo neurologic…

This study looks back at the medical records of about 4 million people who had physical or occupational therapy for muscle and joint problems. Researchers want to see if different ways of giving therapy lead to different results. No new treatments are tested—the goal is to learn …

This study follows 125 adults aged 18-75 who are getting a hip replacement with a new type of implant (the Trident II shell). The main goal is to see how long the implant lasts without needing another surgery. Researchers will also track patients' pain and function using a standa…

This study aims to uncover the genetic roots of aortic aneurysms and valve disease by analyzing tissue and blood samples from 3,000 participants. Researchers will look for new disease-causing genes and factors that affect disease severity. The goal is to build a biorepository to …

This study follows 1000 patients who had a standard aortic valve replacement (TAVR) to see how their heart rhythms change over a year. Researchers will track abnormal heartbeats using ECG monitors. The goal is to learn which patients are at risk for rhythm problems and how they r…

This study tracks 42 people with ocular melanoma who received radiation therapy via eye plaque brachytherapy. Researchers want to see if using lower radiation doses can still keep the cancer from coming back while reducing harmful side effects. The goal is to find the safest effe…

This study tests a digital tool called AsthmaOptimiser that helps doctors assess asthma control and find ways to improve treatment during a single visit. About 746 adults with asthma who have had recent flare-ups will take part. The goal is to see if the tool can identify better …

This study looks at whether eating a high-fiber diet can change the gut bacteria of people with advanced melanoma who are receiving standard immunotherapy. About 50 participants will be randomly assigned to either a high-fiber or a healthy control diet for a set period. The main …

This study compares virtual (telehealth) care to standard in-person visits for 120 adults recently diagnosed with multiple sclerosis. The goal is to see if virtual care can prevent disability progression just as well, while also being more convenient and cost-effective. Participa…

This study uses a non-invasive breathing test called forced oscillometry to measure lung function in 600 newborn infants, both full-term and premature, with and without lung conditions like respiratory distress or bronchopulmonary dysplasia. The goal is to better understand how t…

Researchers at Arkansas Children's Hospital are studying how mitochondria work in children with autism spectrum disorder. They aim to identify distinct patterns of mitochondrial dysfunction that may relate to developmental delays. The study involves up to 5 visits with blood draw…

This study looks at whether common osteoporosis medications also improve muscle health in adults aged 65 and older. Researchers will measure changes in muscle mass, strength, and walking speed over time. The goal is to better understand the link between bone and muscle health in …

The Brain Health Registry is an online project that collects information about memory, mood, sleep, diet, and exercise from adults over 18. Participants also take simple cognitive tests online. The goal is to build a large database to speed up research into brain diseases like Al…

This study brings together medical teams that treat children with cleft lip and/or palate. They will collect information on speech, hearing, dental health, and quality of life from about 1,000 patients. The goal is to compare results across centers and find ways to improve care. …

This observational study plans to enroll 120,000 people with various chronic diseases like pancreatitis, diabetes, and arthritis. Researchers will collect blood samples and health data to study how genetics and lifestyle affect disease progression. The goal is to find common dise…

This study is a registry that tracks 900 people with aortic valve stenosis who had either a surgical aortic valve replacement (SAVR) or a transcatheter aortic valve replacement (TAVR). The goal is to compare how safe and effective these two procedures are over time. Researchers a…

This study followed 900 adults who had their aortic valve replaced to see how safe and effective the procedure is in routine care. Researchers tracked deaths from any cause and heart-related deaths. The goal is to understand real-world outcomes beyond controlled trials.

This study aims to understand what parents go through when their child is born with a large or giant congenital melanocytic nevus (a type of birthmark). Researchers will interview 30 parents in France to learn about their experiences and find ways to better support them. The stud…

This study observes 106 Austrian patients with relapsing multiple sclerosis who are already taking Ofatumumab (Kesimpta). Researchers compare those who started the drug early versus later, tracking disease activity over two years. The goal is to see if early treatment leads to fe…

This study watches 100 people with Fabry disease who are already taking enzyme replacement therapy as part of their regular care. Researchers want to see how the treatment affects their health over time by checking things like kidney function and pain levels. The study does not t…

This study aims to find the genetic cause of PHACE syndrome, a rare condition. Researchers will analyze DNA from 50 people diagnosed with PHACE. Participants must also have an eye exam. The goal is to identify genes that may be responsible for the syndrome, which could lead to be…

This study was designed to measure levels of a protein called SGLT2 in heart muscle samples taken during valve replacement surgery in people with aortic stenosis. Researchers wanted to compare SGLT2 levels between those with and without diastolic heart failure to better understan…

This study looks at whether people who get a new heart valve through a tube in their leg (TAVI) can safely go home just 24 hours later. Researchers will track 585 patients for complications like stroke, heart attack, or need for a pacemaker. The goal is to see if early discharge …

This study is looking for new genes that may cause certain types of congenital heart disease, specifically transposition of the great arteries. Researchers will collect DNA from up to 300 children with these heart defects and their parents to find genetic changes. The goal is to …

This study looks at whether using an AI tool to analyze brain MRIs helps doctors spot signs of multiple sclerosis (MS) activity more accurately. About 750 people with MS or early warning signs will be split into two groups: one where doctors use the AI tool, and one where they re…

This study is testing whether rehabilitation that combines physical and mental exercises (called dual-task training) can help people with stroke, multiple sclerosis, Parkinson's disease, or long COVID. Researchers will design and try out different dual-task strategies in small gr…

This study enrolls 800 people with severe aortic stenosis who are scheduled for a TAVI procedure (a less invasive heart valve replacement). Researchers will compare participants from different countries using CT scans of the heart and blood tests to measure inflammation. The goal…