Syndromic disease

This early-stage trial is testing an experimental cell therapy for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) that has returned or not responded to standard treatments. The therapy uses a patient's own immune cells, modified to recognize …

This trial tests a new, lower-intensity combination of chemotherapy and radiation before a donor stem cell transplant for people with high-risk blood cancers or aplastic anemia. The goal is to see if this approach is safe enough to allow the donor cells to take hold and cure the …

This phase II trial tests a vaccine designed to protect stem cell transplant recipients from cytomegalovirus (CMV), a common virus that can cause serious illness in people with weakened immune systems. The vaccine uses a modified, harmless virus to deliver three CMV proteins, tra…

This study tests a new two-drug combination (pacritinib plus azacitidine) for people with moderate to very high risk myelodysplastic syndrome (MDS), a bone marrow disorder. The first part finds the safest dose of pacritinib, and the second part checks how well the combination wor…

This study tests whether a lower dose of the drug cyclophosphamide, given after a stem cell transplant from a partially matched donor, can reduce serious infections in the first 100 days while still preventing graft-versus-host disease (a common complication where donor cells att…

This phase 1 trial is testing whether amivantamab, an antibody drug given by IV, is safe and tolerable for people with high-grade malignant brain tumors like glioblastoma. The study enrolls about 12 patients with recurrent glioblastoma or certain molecular subtypes. Participants …

This study compares two types of corneal transplant surgery (UT-DSAEK and DMEK) in 160 people with Fuchs dystrophy, a condition that clouds the cornea. It also tests whether a drug called rho-kinase inhibitor can reduce loss of important eye cells after surgery. The goal is to fi…

This study looks at two different ways to use ventilators for children with acute respiratory distress syndrome (ARDS), a serious lung condition. The goal is to see which method helps children recover faster and breathe better. About 160 children will take part, and the results w…

This study tests a new treatment called Orca-T for people with advanced blood cancers like leukemia. It uses a special mix of stem cells and immune cells from a donor to help the body accept the transplant while reducing serious side effects like graft-versus-host disease. The go…

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

This 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 study tests whether giving the drug anakinra early can prevent severe cytokine release syndrome (CRS) in children and young adults with B-cell acute lymphoblastic leukemia (B-ALL) who are receiving CAR-T cell therapy. Participants receive anakinra at the first sign of fever,…

This study looks at the long-term safety of relacorilant, a drug for people with Cushing syndrome. It includes 125 adults who previously completed a relacorilant study and may benefit from continued treatment. Researchers will track side effects over time to see if the drug is sa…

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

This early-phase trial tested whether combining two anti-cancer drugs, pomalidomide (taken as a pill) and liposomal doxorubicin (given through an IV), is safe and tolerable for people with advanced or hard-to-treat Kaposi sarcoma. The study enrolled 62 adults, including those wit…

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 early-phase trial is testing a radioactive drug called 131I-TLX101, given alongside standard care, for people newly diagnosed with glioblastoma, an aggressive brain cancer. The study involves 12 participants and aims to find the safest dose and check for side effects. It is …

This study tests whether using a special camera (OCT) inside the heart's arteries during a heart attack procedure leads to better outcomes than the standard X-ray method. About 3000 people having a heart attack will be randomly assigned to one of the two approaches. The goal is t…

This trial tests whether adding gentle oscillations to standard nasal continuous positive airway pressure (NCPAP) improves breathing support for newborns with respiratory distress syndrome. Premature infants who need NCPAP will be randomly assigned to receive either oscillatory N…

This study tests a vaccine called AADvac1 in 146 people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement and thinking. The vaccine aims to target a protein linked to the disease and slow its progression. Participants will receive injections o…

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

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

This study tests whether a newer drug, benralizumab, works as well as the current standard, mepolizumab, for people with a rare disease called EGPA that inflames blood vessels. About 140 adults with active EGPA will receive either drug for a year, along with their usual steroids.…

This study tests whether the drug ixekizumab is safe and effective for children with certain types of juvenile arthritis (enthesitis-related arthritis and juvenile psoriatic arthritis). About 101 children aged 2 to 17 years will receive either ixekizumab or the standard drug adal…

This Phase 2 trial tests whether the drug LM11A-31 can slow the progression of Progressive Supranuclear Palsy (PSP), a rare brain disorder that affects movement and balance. About 147 people with early-stage PSP will receive either the drug or a placebo twice daily for several mo…

This study tests a drug called sonelokimab in 960 adults with active psoriatic arthritis who have not used biologic treatments before. The goal is to see if it reduces joint pain and swelling better than a placebo. Participants receive injections over several months, and research…

This study tests an experimental drug called LP352 to see if it can safely reduce seizures in children and adults with developmental and epileptic encephalopathies (DEE), a group of severe epilepsy syndromes. About 320 participants will receive either LP352 or a placebo, and rese…

This trial tests whether rituximab, an infusion drug that calms certain immune cells, can help people with idiopathic membranous nephropathy and nephrotic syndrome. Participants receive either rituximab or a placebo, and the study tracks how many achieve a significant reduction i…

This phase 3 study is testing the long-term safety and effectiveness of LP352 (Bexicaserin) in 324 children and adults with developmental and epileptic encephalopathy (DEE), including Dravet and Lennox-Gastaut syndromes. Participants who completed earlier studies will receive LP3…

This early trial is testing whether the drug olaparib can shrink tumors in people with newly diagnosed BRCA-mutant ovarian, fallopian tube, or peritoneal cancer before they have surgery. Fifteen participants will receive olaparib for two cycles, then undergo standard chemotherapy…

This study tests a new device that uses freezing (cryotherapy) to treat obstructive sleep apnea in adults who cannot use standard treatments like CPAP machines. About 25 people with moderate to severe sleep apnea will receive the procedure. The main goal is to check safety and se…

This phase II trial is testing whether adding the drug asciminib to standard treatment can improve outcomes for people newly diagnosed with Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL). The study will enroll 55 participants and involves several phases o…

This study is testing a combination of two oral medications, Onureg and Venclyxto, in people with a high-risk form of myelodysplastic syndromes (a blood cancer) who cannot have a stem cell transplant. The goal is to find the safest and most effective dose of Onureg when used with…

This study tests whether baricitinib, a daily pill, is safe and works for children aged 1 to under 18 with systemic juvenile idiopathic arthritis (sJIA), a chronic inflammatory disease. About 58 children will receive either baricitinib or a standard injected drug (tocilizumab) to…

This Phase 3 study tests whether a gel containing patidegib can lower the number of new basal cell carcinomas (a type of skin cancer) on the face of adults with Gorlin syndrome. Participants apply the gel or a placebo to their face twice daily for a year. The study will compare h…

This phase 2 study tests two drugs, sunitinib and temsirolimus, in 137 people with advanced rare tumors that have no cure. The goal is to see if these drugs can shrink tumors or slow their growth. Participants take one of the two drugs, and researchers measure how long the cancer…

This pilot study tests a new surgical procedure for people with anterior shoulder instability, where the shoulder repeatedly dislocates. The procedure uses a semitendinosus tendon graft to create a sling around the subscapular tendon and form a new labrum, aiming to hold the shou…

This trial is testing an oral drug called AG-120 (ivosidenib) in adults with myelodysplastic syndrome (MDS) or a related early form of leukemia that carries a specific IDH1 gene mutation. The drug is designed to block the mutated enzyme that drives abnormal blood cell growth. Res…

This study tests a new way to treat adults with a specific type of leukemia (Ph+ ALL) that has come back or not responded to treatment. The approach combines low-intensity chemotherapy with two targeted drugs, ponatinib and blinatumomab, to kill cancer cells and boost the immune …

This study tests the long-term safety and effectiveness of an experimental drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). Participants who completed a previous VX-670 study can join. The drug is given through a vein, and researchers will monitor side effects a…

This early-phase trial tests a drug called DR-18 in 40 adults with acute myeloid leukemia or myelodysplastic syndrome whose cancer returned or persisted after a stem cell transplant. DR-18 is a modified immune protein designed to boost the body's natural defenses against cancer c…

This trial is testing an oral drug called enasidenib (AG-221) in people with myelodysplastic syndrome (MDS) who have a specific genetic change called an IDH2 mutation. The drug is designed to block the mutated enzyme and help restore normal blood cell production. Participants rec…

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

This study tests a culturally tailored sleep program for Latino couples where one partner has sleep apnea and is starting CPAP therapy. The program includes three telehealth sessions teaching sleep skills and support strategies. Researchers want to see if it improves CPAP use and…

This phase II study tests a vaccine called SurVaxM, designed to train the immune system to attack glioblastoma cells, when given alongside standard chemotherapy (temozolomide). The trial includes 66 patients with newly diagnosed, survivin-positive glioblastoma or gliosarcoma. The…

This study tests two drugs, vorinostat and azacitidine, together in people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The goal is to find the best dose and see if the combination is safe and effective. About 135 participants will take part in this early-…

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 specific timing of chemotherapy drugs (busulfan and fludarabine) before a donor stem cell transplant, followed by cyclophosphamide after the transplant, to treat high-risk blood cancers like leukemia and lymphoma. The goal is to reduce the chance of the transpl…

This early pilot study will test the safety of a procedure called extracorporeal photopheresis (ECP) in just 3 adults with stiff person syndrome. ECP involves filtering a person's blood, treating it with light and a drug, then returning it to the body. The goal is to see if patie…

This Phase 3 trial tests a drug called GTX-102 (apazunersen) in 129 children with Angelman syndrome, a genetic condition causing severe developmental delays. The study compares the drug to a sham procedure to see if it improves cognitive function and other symptoms over about 11 …

This study tests whether adding an experimental drug called NanO2 to standard radiation and chemotherapy can help people with newly diagnosed glioblastoma, an aggressive brain cancer. NanO2 is designed to deliver more oxygen to tumors, which may make radiation work better. About …

This trial tests whether an investigational drug called SHR-1314 can reduce joint pain, swelling, and skin symptoms in adults with active psoriatic arthritis. Participants receive either SHR-1314 or a placebo injection over 24 weeks, with follow-up for safety. The study aims to s…

This trial is testing whether mebendazole, a drug commonly used to treat intestinal parasites, can help children with brain tumors called gliomas. The study combines mebendazole with standard chemotherapy drugs to see if it is safe and effective. It includes children and young ad…

This study looks at whether using a CPAP machine during pregnancy can reduce the chance of developing high blood pressure disorders like preeclampsia. About 1,500 pregnant women with mild to moderate sleep apnea will be randomly assigned to use CPAP or not. The goal is to see if …

This study tests an oral drug called ONC201 in people whose acute leukemia or high-risk myelodysplastic syndrome (MDS) has returned or not responded to standard treatments. The trial has two phases: Phase I finds the safest dose, and Phase II checks if the drug shrinks or control…

This study tests a simplified, accelerated HIV care model called ACCELERATE. It combines rapid telehealth appointments, a pre-approved HIV medication, and a free 30-day starter supply to help people with HIV who have fallen out of care get back on track. The goal is to see if thi…

This study explores whether regular aerobic exercise, specifically walking about 300 minutes per week, can change certain biomarkers in the blood that are linked to cancer risk. Participants are adults who have completed all cancer therapy and have either clonal hematopoiesis (a …

This early-phase study tests a new way to deliver total body radiation before a stem cell transplant for people with acute leukemia or myelodysplastic syndrome. The goal is to reduce radiation to the lungs to prevent serious lung damage. Twenty participants will receive intensity…

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 study tests an investigational drug called relutrigine in 160 children with a severe form of epilepsy called developmental and epileptic encephalopathy (DEE). The goal is to see if the drug can safely reduce monthly seizure frequency compared to a placebo. After the initial …

This phase 3 trial is testing whether adding the immunotherapy drug atezolizumab to standard chemotherapy can help prevent cancer from coming back in people with stage III colon cancer that has a specific DNA repair defect (dMMR). About 712 participants will receive either chemo …

This study is testing an experimental drug called ACE-86225106 in adults with advanced solid tumors that are difficult to treat or have stopped responding to standard therapies. The drug works by blocking a protein that helps cancer cells repair themselves, potentially slowing or…

This study tested a combination of the immunotherapy drug blinatumomab with either standard chemotherapy or dasatinib and prednisone in adults aged 65 and older with acute lymphoblastic leukemia (ALL). The goal was to see if the combination could improve survival and control the …

This study tests an experimental drug, HT-6184, in adults with a bone marrow disorder called myelodysplastic syndrome (MDS) who have low red blood cell counts (anemia). The goal is to see if the drug can improve blood counts and reduce the need for transfusions. Participants take…

This study tests the long-term safety of an experimental drug called UCB0107 (bepranemab) in people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement and balance. Participants who completed a prior study receive regular infusions of the drug. …

This trial is investigating whether an experimental drug called XTMAB-16 can safely control pulmonary sarcoidosis over the long term. The study includes people who have already completed a previous XTMAB-16 trial and are on a stable steroid dose. Researchers are monitoring side e…

This early-stage trial tests a single injection of RB001, a gene therapy, directly into the fluid around the brain of children with Phelan-McDermid syndrome caused by SHANK3 gene changes. The study includes up to 8 children aged 3 to 17 and primarily checks whether the treatment …

This trial tests whether a simple 10-hour time-restricted eating schedule, combined with health education, can improve early-stage cardiovascular-kidney-metabolic (CKM) syndrome in adults aged 30–79 who do not yet need medication. Participants in the intervention group eat all th…

This early-stage trial is testing an experimental drug called belzutifan in people with advanced solid tumors that have not responded to standard treatments. Belzutifan works by blocking a protein (HIF-2α) that tumors use to survive in low-oxygen conditions. The main goal is to f…

This study tests an experimental oral drug called LY3410738 in people with advanced blood cancers such as acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) that have specific IDH1 or IDH2 gene mutations. The drug is designed to block the mutated IDH proteins that he…

This trial investigates the long-term safety of ecopipam, a drug that blocks dopamine D1 and D5 receptors, in children, adolescents, and adults with Tourette syndrome. Participants who completed a prior study may enroll and receive ecopipam for up to 24 weeks or longer. The main …

This study tests whether adding a PCSK9 inhibitor (a powerful cholesterol-lowering drug) to high-intensity statin therapy can prevent future cardiovascular events in people who have recently had a heart attack or unstable angina and underwent a stent procedure. About 1,200 partic…

This study compares two approaches to help people with moderate-to-severe obstructive sleep apnea stay on CPAP therapy. One group receives usual care, where patients reach out if they have trouble. The other group adds active monitoring and extra support only for those who strugg…

This study investigates whether tirzepatide, a drug used for diabetes and weight loss, can reverse biological aging in people with HIV who are virally suppressed and have obesity. Participants receive tirzepatide for 25 weeks after a diet program. Researchers measure epigenetic a…

This study tests whether giving patients hyperbaric oxygen (breathing pure oxygen in a pressurized chamber) after an umbilical cord blood stem cell transplant helps the donated stem cells grow faster. The trial enrolls people with blood cancers like leukemia or lymphoma who lack …

This study investigates whether diagnosing obstructive sleep apnea (OSA) early and starting CPAP therapy in patients hospitalized for chronic obstructive pulmonary disease (COPD) can reduce the number of patients who are readmitted to the hospital within 30 days. Participants are…

This study investigates whether using CPAP therapy for six months can reduce average 24-hour blood pressure in people with moderate-to-severe obstructive sleep apnea who also report excessive daytime sleepiness. Participants are adults aged 18-75 with elevated blood pressure and …

This study investigates whether diagnosing and treating obstructive sleep apnea with CPAP therapy early, while patients are still in the hospital for heart problems, can reduce the chance of being readmitted within 30 days. The trial enrolls adults with heart failure, heart attac…

This study is a long-term extension of an earlier trial, testing the safety of a drug called RAY121 in people with six different immune system disorders, including antiphospholipid syndrome and immune thrombocytopenia. Participants have already completed four doses of RAY121 and …

This study tests whether combining two drugs, luspatercept and lenalidomide, can improve anemia in people with lower-risk myelodysplastic syndrome (MDS) who lack a specific chromosome change. Participants take lenalidomide daily by mouth and receive luspatercept as a shot under t…

This phase 1 trial tests whether the drug tagraxofusp can help prevent relapse in people with certain blood cancers (acute myeloid leukemia, myelodysplastic syndrome, myelofibrosis, or chronic myelomonocytic leukemia) who have recently received a stem cell transplant. Participant…

This study tests the drug anakinra in Chinese patients with two related inflammatory conditions: Systemic Juvenile Idiopathic Arthritis (SJIA) in children and Adult-Onset Still's Disease (AOSD) in adults. Participants receive a daily injection under the skin for 48 weeks. The goa…

This study follows 300 people who receive a hip replacement using either the Vivacit-E or Longevity liner, both made of a special plastic designed to last longer. The goal is to see how well these liners hold up over time, measuring how many are still in place without needing rep…

This trial tests whether adding two drugs—vorinostat and low-dose azacitidine—after a stem cell transplant can help prevent relapse in children with certain myeloid cancers. The study includes children aged 1 to 21 with acute myeloid leukemia or related conditions. Researchers ar…

This trial investigates whether a single dose of the experimental drug DAW2020, taken before sleep, can improve how the brain responds to airway blockages in people with moderate-to-severe obstructive sleep apnea. The study measures changes in the arousal threshold—the point at w…

This trial tests whether using a special camera inside the arteries (optical coherence tomography, or OCT) is better than standard pressure-wire measurements for deciding which non-blocked arteries need stenting in people who have just had a heart attack. About 1,425 participants…

This trial tests whether a custom-made plastic implant (ultra-high molecular weight polyethylene) works better than a standard titanium implant for people with chronic jaw dislocation. Participants have a jaw that repeatedly dislocates and has not improved with conservative treat…

This phase 1 study tests whether adding a highly targeted, high-dose radiation boost (stereotactic body radiotherapy, or SBRT) to standard radiation and chemotherapy can safely treat head and neck cancer that has come back or spread. The trial includes two groups: patients whose …

This study tests a program called PIXI that coaches parents of infants diagnosed with rare neurogenetic disorders (such as Fragile X, Angelman, or Down syndrome) during the first year of life. The program combines education about the disorder, guided parent-child interaction acti…

This study tests a device that uses freezing (cryoablation) on the tongue and soft palate to treat moderate to severe obstructive sleep apnea. It is for adults aged 22 to 70 who cannot or will not use standard treatments like CPAP or mouthguards. The goal is to see if the procedu…

This study follows children with Phelan-McDermid syndrome (a rare genetic disorder causing developmental delays) who received a single dose of RB001 gene therapy injected into the brain. Researchers will monitor them for up to 5 years to see if the treatment is safe and whether i…

This study tests whether taking agave inulin, a natural fiber, for eight weeks can improve blood sugar control and increase healthy gut bacteria in adults with type 2 diabetes. Participants receive either 10 grams of agave inulin or a placebo daily. Researchers measure changes in…

This trial tests a digital tool that helps doctors and patients manage cancer risks linked to hereditary syndromes like BRCA-related breast/ovarian cancer and Lynch syndrome. The tool provides personalized recommendations during primary care visits. Researchers compare care gaps,…

This phase 2 trial tests whether adding lab-grown umbilical cord blood cells (dilanubicel) to a standard cord blood transplant helps people with blood cancers like leukemia recover their blood and immune cells faster. Participants receive chemotherapy and radiation to prepare the…

This trial tests a drug called ivarmacitinib in people with Behçet's disease who have not improved with standard treatments. Ivarmacitinib blocks a key inflammation pathway (JAK-STAT) to reduce symptoms. Participants take the drug daily for 24 weeks, and researchers monitor their…

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 study tests whether a short, standardized 4-minute heart-healthy talk is as effective as a full 60-minute personalized session with a dietitian for lowering LDL cholesterol and improving diet. Participants are adults aged 30–80 with rheumatoid arthritis, psoriatic arthritis,…

This study investigates whether applying sound waves (extracorporeal shockwave therapy) to the abdomen, alongside a low-calorie diet and aerobic exercise, can reduce abdominal fat and improve metabolic health in adults with abdominal obesity. Researchers will measure changes in w…

This trial tests whether taking hydrocortisone once a day (as a dual-release pill) leads to more stable blood sugar levels compared to the standard three-times-a-day regimen in adults who have both insulin-treated diabetes and Addison's disease. Participants will try both schedul…

This trial tests a combination of two drugs, sirolimus and all-trans retinoic acid (ATRA), for people with autoimmune anemia that has not responded to standard treatments. Autoimmune anemia occurs when the immune system attacks red blood cells, causing fatigue and other symptoms.…

This trial tests whether combining roxadustat with retinoic acid can help people with low-risk myelodysplastic syndromes (MDS) whose disease hasn't responded to initial treatment. The goal is to improve blood cell counts and reduce the need for transfusions. Participants will tak…

This study tests whether a soup made from boiled legume seed coats can help control blood sugar in people with prediabetes or type 2 diabetes. Participants drink the soup daily for 30 days. Researchers measure changes in blood sugar, cholesterol, and markers of inflammation and o…

This phase I trial tests the safety of adding ruxolitinib to a standard combination of dasatinib and dexamethasone for adults aged 40 and older with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The study starts with a low dose of ruxoli…

This trial tests whether adding a radioactive antibody (90Y-DOTA-anti-CD25 basiliximab) to standard chemotherapy and targeted radiation can better prepare the bone marrow for a stem cell transplant in people with high-risk acute leukemia or myelodysplastic syndrome. The antibody …

This study tests a new approach for people receiving a stem cell transplant from a partially matched family donor. The donor's immune cells (T cells) are modified in the lab to include a 'suicide gene' that can be activated by a drug if the cells attack the patient's body, causin…

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

This trial tests whether specially modified T cells from a partially matched donor can help patients recover their immune system faster after a stem cell transplant. The T cells are engineered with a 'suicide gene' that allows doctors to destroy them if they cause graft-versus-ho…

This study compares a rapid weaning schedule to a slower one for newborns being treated with morphine or methadone for neonatal opioid withdrawal syndrome (NOWS). The goal is to see if a faster wean can reduce the number of days babies need opioid medication. The trial includes f…

This study tests a non-invasive pneumatic vest designed to apply pressure to the chest wall in patients with acute respiratory distress syndrome (ARDS) caused by COVID-19 who are on mechanical ventilators. The goal is to see if the vest can improve oxygen levels in the blood. Up …

This study tests a single dose of PBGM01, a gene therapy delivered directly into the fluid around the brain and spinal cord, in children with GM1 gangliosidosis. The therapy uses a harmless virus to carry a working copy of the GLB1 gene, which is missing or faulty in these patien…

This phase 3 trial tests a biologic drug called olokizumab in 125 people with polymyalgia rheumatica, a condition causing muscle pain and stiffness. The drug is given as a shot every two weeks. The main goal is to see if it can control symptoms and allow patients to take lower do…

This trial tests whether a home-based tele-rehabilitation program is as effective as traditional center-based cardiac rehabilitation for people who have recently had a heart attack (acute coronary syndrome). Participants will either complete 20 sessions at a rehab center or 20 se…

This study tests whether a device can detect when a patient's breathing slows after surgery and then play a recorded voice telling them to breathe. The device uses standard monitors already used in hospitals. The study includes patients who have or may have sleep apnea and are st…

This study tests whether anakinra (Kineret), a daily injection, can reduce monthly attacks in Chinese patients with Familial Mediterranean Fever (FMF) who do not respond to standard colchicine treatment. Three patients aged 2 years and older will receive anakinra for 6 months, wi…

This trial investigates the best way to treat children with acute myeloid leukaemia (AML) by comparing different chemotherapy drugs and doses. It tests whether adding up to three doses of the targeted drug gemtuzumab ozogamicin to standard chemo is safe and effective. The study a…

This trial tests a combination of donor immune cells (targeting a common virus) and the drug pembrolizumab (Keytruda) in 58 people with glioblastoma, an aggressive brain cancer. The goal is to see if this approach can shrink tumors and help patients live longer. The study has two…

This study tests whether involving patients in managing CPAP machine alerts—using a connected watch and app—can improve how often they use the device. It includes 556 adults with sleep apnea who are at risk for heart problems and likely to struggle with CPAP adherence. The goal i…

This study tests whether giving people different taste-based advice helps them reduce their free sugar intake to less than 5% of daily energy. Participants are adults in southern England who consume more than 10% of their energy from free sugars. They are randomly assigned to adv…

This trial tests a new drug called Cadonilimab in 28 people with advanced colorectal cancer that has not responded to standard immunotherapy. The drug is a bispecific antibody that targets two immune checkpoints (PD-1 and CTLA-4) to potentially reactivate the immune system agains…

This study tracks the safety of the drug Samsca (tolvaptan) in 300 Japanese patients with low sodium levels caused by SIADH. Researchers will record any side effects or special situations that occur during routine use. The goal is to confirm the drug's safety in real-world settin…

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

This was a planned early-stage trial to test whether anti-CD19 CAR-T cells could safely help children with multi-drug resistant nephrotic syndrome, a serious kidney condition. The study was withdrawn before any patients were enrolled, so no data on safety or effectiveness were co…

This study looks at whether lower doses of the chemotherapy drug CPX-351 can help older patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that have come back or not responded to other treatments. The goal is to control the disease while being gentler o…

This phase 2 trial tests whether lamivudine, an antiviral drug, can improve symptoms in people with Rett syndrome, a rare genetic disorder that affects movement, communication, and behavior. Ten participants will take the drug by mouth for a set period, and researchers will measu…

This study planned to test a new treatment combining CYNK-001 (natural killer cells from donated cord blood) with interleukin-2 in adults whose glioblastoma had returned and could be surgically removed. The goal was to see if this combination was safe and could extend the time be…

This early-phase trial tests an investigational biologic called EA5 in adults with antiphospholipid syndrome (APS) who have had two or more blood clots despite standard blood-thinning therapy. The study enrolls about 12 participants and gives EA5 by intravenous infusion followed …

This early-phase trial tests a new cell therapy called BEN301 for several autoimmune diseases, including scleroderma and rheumatoid arthritis. The therapy uses specially engineered immune cells (CAR-Treg cells) to calm the overactive immune system. The study involves 24 adults an…

This phase 2 trial tests whether eltrombopag, a drug already approved for other blood disorders, can improve blood cell counts in people with low-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) who have a mutation in the TET2 gene. Participants take…

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

This study tests a technique that delivers two cancer drugs, bevacizumab and carboplatin, directly into the brain's arteries for people with recurrent glioblastoma or anaplastic astrocytoma. The goal is to see if this method is safe and can slow tumor growth better than standard …

This study compares two types of surgery to create dialysis access in older adults (60+) with end-stage kidney disease. One method connects a vein directly to an artery (fistula), the other uses a synthetic tube (graft). The goal is to see which leads to fewer infections and more…

This early-stage trial tests a new surgical approach for recurrent glioblastoma, an aggressive brain cancer. After standard tumor removal, surgeons place a piece of abdominal tissue (omentum) into the brain cavity to bypass the blood-brain barrier. The study involves 10 adults an…

This study tests whether adding the drug itacitinib to the usual two-drug regimen (cyclophosphamide and tacrolimus) can better prevent graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers. GVHD is a serious complication where donor cells at…

This study tests a stem cell transplant from a matched unrelated donor for people with advanced blood cancers or blood disorders. Patients get chemotherapy and radiation before the transplant to prepare their body, then take drugs to prevent the donor cells from attacking their o…

This phase 2 study tests the drug atrasentan in 103 adults with various kidney diseases that cause protein leakage, including IgA nephropathy and FSGS. Participants take a daily tablet to see if it reduces protein in urine, a sign of kidney damage. The goal is to find a new way t…

This study compares two common fertility treatments—letrozole alone versus clomiphene citrate plus tamoxifen—in 240 women with infertility due to ovulation problems. The goal is to see which approach leads to more ovulations and pregnancies. Participants will be monitored with ul…

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

This study tests whether a drug-coated balloon is as safe and effective as a drug-eluting stent for people with large coronary artery disease. About 1,400 participants from multiple countries will be randomly assigned to one of the two treatments. The goal is to see which approac…

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

This early-phase trial tests a single injection of AAV-MECP2 gene therapy into the spinal fluid of 8 girls aged 4-10 with Rett syndrome, a severe neurodevelopmental disorder. The goal is to see if the treatment is safe and can reduce disease severity. The study is not yet recruit…

This study tests a new drug called obinutuzumab in 100 children aged 1-18 with refractory nephrotic syndrome, a kidney condition causing swelling and protein loss. The drug targets immune cells to reduce disease flares and dependence on steroids and other medications. Researchers…

This study tests a new medicine, NS-229, in 45 adults with a rare disease called eosinophilic granulomatosis with polyangiitis (EGPA), which causes inflammation in blood vessels. The goal is to see if NS-229 can help patients achieve remission while lowering their daily steroid d…

This study tests whether adding myoinositol or pioglitazone to metformin improves pregnancy rates in women with PCOS undergoing ICSI (a type of IVF). 150 women will take one of three drug regimens for one month before the procedure. Researchers will track fertilization, pregnancy…

This study tests whether adding cabergoline to osilodrostat works better than osilodrostat alone for controlling Cushing's disease. About 50 adults with active Cushing's disease will receive either the combination or the single drug. The goal is to see if the combo can lower cort…

This trial tests a 12-month lifestyle medicine program for 1000 adults with obesity and related conditions like diabetes or high blood pressure. Participants get regular visits with a dietitian and a doctor, plus optional behavioral health support. The goal is to see if the progr…

This study was designed to test whether an inhaled drug called ZYESAMI could help people with severe COVID-19 avoid getting worse and needing a ventilator. It was a Phase 3 trial, meaning it was in the final stage of testing. However, the study was withdrawn before any participan…

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

This study tests whether a lower dose of the steroid prednisone can treat relapses of nephrotic syndrome in children as effectively as the standard dose. Fifty children aged 1 to 18 with active relapses will be randomly assigned to either the reduced-dose or standard-dose regimen…

This study tests an experimental drug called imetelstat in 46 adults with high-risk myelodysplastic syndromes or acute myeloid leukemia that did not improve after standard therapy. The goal is to see if the drug can improve blood cell counts and control the disease. Participants …

This study was designed to test whether adding an experimental drug and special immune cells to standard care could help critically ill adults with severe pneumonia, especially those with sepsis or lung failure. The trial was withdrawn before enrolling any participants, so no res…

This study was designed to see if adding two immune treatments (NAI and iNKT cells) to standard care could help critically ill adults with severe pneumonia, sepsis, or lung failure recover. It planned to enroll ICU patients and follow them for 90 days. However, the study was with…

This study is testing a custom-made artificial heart valve, called TRICENTO, in 15 people with carcinoid heart disease who have severe leakage of the tricuspid valve and cannot have surgery. The valve is delivered through a catheter, avoiding open-heart surgery. The goal is to se…

This study looks at whether a step-by-step care plan (called TheraPPP) helps patients with severe breathing failure (ARDS) survive and spend less time on a ventilator. The plan includes proven treatments like protective ventilation, paralysis, and prone positioning. About 20,000 …

This study tests whether offering small prizes (phone credit) can encourage young people aged 15-30 with HIV to take their medication every day. 560 participants in Uganda are split into groups with different reward rules. The goal is to see if these low-cost incentives improve a…

This phase 2 trial tests giving chemotherapy (carboplatin and paclitaxel) plus the immunotherapy drug nivolumab before surgery for people with Pancoast tumors, a rare type of lung cancer. After surgery, additional treatment is tailored based on whether the tumor was fully removed…

This study tests whether adding the drug venetoclax to standard azacitidine treatment helps people with a severe form of myelodysplastic syndrome (MDS) live longer. MDS is a bone marrow disorder that can lead to leukemia. About 500 adults with newly diagnosed high-risk MDS will r…

This study looks at whether eating almonds every day for 12 weeks can improve gut health and lower inflammation in people with metabolic syndrome. About 80 adults aged 35-60 with conditions like high blood pressure, high blood sugar, or unhealthy cholesterol levels will participa…

This study tests whether a drug called Angiotensin II can raise blood pressure more effectively than standard medications in people with liver cirrhosis who have septic shock. Researchers will measure how well the heart and blood vessels work together over 24 hours. The trial inv…

This study was designed to test whether IC14, a drug that blocks a key inflammatory signal, could help people with acute respiratory distress syndrome (ARDS). The plan was to give IC14 or a placebo to about 100 ICU patients and see if it reduced time on a ventilator. However, the…

This study tests whether adding the targeted drug pazopanib to standard chemotherapy (temozolomide) can safely control glioblastoma, an aggressive brain cancer. About 51 adults who had surgery and chemoradiation will receive the combination. The main goal is to find the best dose…

This clinical trial is testing different treatment strategies to improve outcomes for older adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) who are getting a stem cell transplant. The study compares a new pre-transplant drug (Vyxeos) with stan…

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 tests a new way to do stem cell transplants for children and young adults (up to age 22) with blood cancers like leukemia. Doctors remove certain immune cells from donated stem cells to lower the risk of graft-versus-host disease, a serious complication. The goal is to…

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

This study tests a drug called tezepelumab in 42 adults with EGPA, a rare disease that causes inflammation in blood vessels and can damage organs. The goal is to see if the drug helps patients reach remission, meaning no active disease and low steroid use. Participants receive ei…

This study tests whether using two eye drops together—Vevye (cyclosporine) and Flarex (a steroid)—can safely improve symptoms in adults with moderate to severe dry eye disease. Sixty participants will use Vevye twice daily and Flarex four times daily for one month. The goal is to…

This study tests whether a program combining exercise, diet advice, and behavioral support can improve fitness and health in adults aged 60-75 who are at high risk for heart disease. Participants in the program receive supervised high-intensity exercise and counseling, while all …

This study tests whether a 6-week exercise program, done twice a week, can improve physical and mental health in 100 adults with severe mental illness. Participants are randomly assigned to either usual care or usual care plus the exercise program. The goal is to see if exercise …

This study tests whether taking vitamin B1 tablets for a year can reduce the size and number of colon polyps in people with familial adenomatous polyposis (FAP), a genetic condition that raises colon cancer risk. Ten adults with FAP who still have their colon will take vitamin B1…

This study planned to test a new hip replacement device (the HYPE stem) in people with hip arthritis or other hip damage. The goal was to see how well the device works and if it is safe. However, the study was withdrawn before any participants were enrolled, so no results are ava…

This early-phase study tests a new treatment for glioblastoma, an aggressive brain cancer. The approach uses a patient's own immune cells, modified in the lab to better recognize and attack the tumor, combined with chemotherapy. The main goal is to check the safety of this therap…

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

This study tests whether stem cells from donated umbilical cords are safe and can help people with myelodysplastic syndrome (MDS), a bone marrow disorder. About 40 participants with moderate to severe symptoms will receive the stem cells and be monitored for side effects and impr…

This study planned to test a procedure called bariatric embolization for people with morbid obesity who cannot have standard weight-loss surgery. The procedure involves injecting tiny particles into blood vessels near the stomach to reduce blood flow, which may lower hunger hormo…

This study tests a procedure for people with early-stage osteonecrosis (bone death) of the hip. Doctors drill into the hip bone, inject a concentrated sample of the patient's own bone marrow, and add a special bone graft material called Genex. The goal is to see if this helps bon…

This early-phase study tests CPX-351 (Vyxeos), a two-drug chemotherapy, in 20 adults aged 18-70 with higher-risk myelodysplastic syndrome (MDS) who are eligible for a stem cell transplant. The main goal is to see if the drug is safe and tolerable, with secondary goals of measurin…

This study tests a device that stimulates a nerve in the neck to keep the airway open during sleep. It is for adults with moderate to severe obstructive sleep apnea who cannot use a CPAP machine. The goal is to see if the device is safe and improves breathing and sleep quality.

This study tests an experimental drug called AMX0035 in 12 adults with Wolfram syndrome, a rare genetic disorder that causes diabetes and vision loss. The goal is to see if the drug can help preserve the pancreas's ability to make insulin and to check its safety over up to 4 year…

This study tests a new blood filtering therapy for adults in the ICU with septic shock, a life-threatening condition caused by severe infection. The treatment aims to remove harmful substances from the blood to help patients recover faster. The trial includes 15 participants and …

This study tests a stem cell transplant method for people with blood cancers like leukemia or lymphoma. The approach uses chemotherapy, total body radiation, and a drug called cyclophosphamide after the transplant to prevent graft-versus-host disease, where donor cells attack the…

This study follows 9 people with Fanconi Anemia who already received a gene therapy that adds a working FANCA gene to their blood stem cells. Researchers will check their health and blood counts for 15 years to see if the treatment remains safe and keeps working. No new treatment…

This pilot study tests whether deep brain stimulation (DBS) of specific brain pathways can safely reduce seizures in people with Lennox-Gastaut syndrome, a severe form of epilepsy. Five participants receive the electrical stimulation as an add-on to their usual treatment. The goa…

This study tests whether ambrisentan, a drug approved for lung blood pressure, can help patients with hepatorenal syndrome—a serious kidney problem that can occur in advanced liver cirrhosis. About 54 hospitalized adults will receive either ambrisentan or the standard drug terlip…

This study compares taking two blood thinners for just 1 month versus the standard 12 months after getting a heart stent. It involves over 3,700 patients with acute coronary syndrome. The goal is to see if the shorter regimen reduces bleeding risks without increasing heart proble…

This trial tests an implantable ultrasound device that temporarily opens the blood-brain barrier, allowing chemotherapy to reach brain tumors. It includes 57 adults with recurrent glioblastoma who have already tried 1-2 treatments. The goal is to find a safe dose and see if this …

This study tests whether maslinic acid, a natural compound found in olive skins, can improve metabolic health by activating brown fat—a type of fat that burns calories. Fifty adults with pre-metabolic syndrome or mild obesity will take maslinic acid or a placebo for 12 weeks. Res…

This study compares stem cell transplants from two types of donors for people with leukemia or myelodysplastic syndromes (MDS): younger non-family members (age 40 or under) and older first-degree relatives (over 50). The goal is to see which donor leads to better overall survival…

This study was designed to see if adding hydroxychloroquine to standard treatment (aspirin and heparin) could help women with antiphospholipid syndrome have a healthy full-term pregnancy. It planned to enroll pregnant women before 14 weeks of gestation. However, the trial was wit…

This phase 2 trial tests whether adding blinatumomab (an immunotherapy) to standard targeted therapy (dasatinib) can improve treatment for adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The goal is to achieve a deep molecular response without…

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 study tests a new way to do stem cell transplants for 20 children with blood cancers or myelodysplasia who don't have a perfect donor match. Doctors remove certain immune cells from a parent's partially matched stem cells before transplant to help the new cells grow faster a…

This study tests whether adding everolimus to the existing drug lonafarnib can better control progeria, a rare disease that causes rapid aging in children. About 80 children with confirmed progeria will take both pills by mouth. The first part finds the safest dose of everolimus,…

This study tests the long-term safety and effectiveness of pegcetacoplan in 100 people with two rare kidney diseases, C3 glomerulopathy and IC-MPGN. Participants previously completed a one-year trial and saw benefit from the drug. The goal is to see if pegcetacoplan can keep redu…

This phase 2 trial tests whether pembrolizumab (Keytruda) can shrink or control advanced solid tumors that have certain gene mutations (BRCA1, BRCA2, POLE, POLD1). The study enrolled 21 adults with cancers that have spread or come back. Researchers measure how many patients see t…

This early-phase trial tests an experimental drug called radiprodil in 30 people with tuberous sclerosis complex or focal cortical dysplasia type II whose seizures have not responded to at least two other medications. The study is open-label, meaning everyone gets the drug, and w…

This study is observing about 881 people with rheumatoid arthritis, ankylosing spondylitis, psoriatic arthritis, or psoriasis who are taking Remsima (a biologic drug) either as a shot under the skin or through an IV. The goal is to track any serious side effects over time. Resear…

This phase 1 trial tests whether the oral drug Inqovi (decitabine/cedazuridine) can help prevent myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) from returning after a stem cell transplant. About 22 adults who have had a reduced-intensity transplant will …

This phase 2 study tests whether the drug ruxolitinib can help prevent acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) from coming back after a stem cell transplant. About 64 older adults in remission will take ruxolitinib as maintenance therapy. The goal is to see…

This study tests a new gene therapy for boys with Hunter syndrome, a genetic disorder that damages the brain and body. The therapy uses the child's own blood stem cells, modified to produce the missing enzyme, and aims to stop disease progression. Five boys aged 3 to 22 months wi…

This early study tests a new freezing treatment for people who recently had a heart attack or unstable chest pain. The treatment uses a special balloon to freeze high-risk plaque in the heart's arteries that could rupture and cause another heart attack. The goal is to stabilize t…

This early-stage trial tests the safety and best dose of combining two drugs—everolimus and midostaurin—for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or is hard to treat. The drugs work by blocking faulty signals that help cance…

This early-phase study tests a new drug called ERAS-601, alone or with other cancer treatments, in adults with advanced solid tumors that have not responded to standard therapies. The main goals are to find safe doses and check for side effects. About 90 participants will take pa…

This study looks at whether a 26-week behavioral weight loss program called 'Paso a Paso' is practical for Mexican and Central American adults with fatty liver disease and overweight. Researchers will track attendance, weight loss, liver stiffness, and diet changes in 50 particip…

This study tests a drug called ivabradine in 429 adults with septic shock, a life-threatening condition where infection causes dangerously low blood pressure. Many patients develop a fast heart rate, which can worsen outcomes. Ivabradine slows the heart without weakening its pump…

This study tests whether an implantable heart monitor with remote follow-up can help doctors decide when to prescribe blood thinners to patients who have had a heart attack and developed new atrial fibrillation. The goal is to reduce bleeding complications compared to the standar…

This study tests whether adding the drug sodium valproate to a standard jaw-washing procedure can lower inflammation in people with a painful jaw joint condition. Ten female participants with jaw clicking and pain will receive the drug intravenously during the procedure. Research…

This study tests whether taking fecal microbiota transplantation (FMT) pills can improve symptoms of major depressive disorder (MDD) and irritable bowel syndrome (IBS). About 133 adults with MDD, with or without IBS, will either receive FMT or continue their usual treatment. Rese…

This study tests an oral drug called emavusertib (CA-4948) in adults with acute myeloid leukemia (AML) or a high-risk bone marrow disorder (MDS) that have not responded to or returned after prior treatment. The goal is to find the safest and most effective dose and to see if the …

This study tests whether a stem cell transplant from a healthy donor can safely treat people with severe T-cell problems. Participants receive chemotherapy and antibody therapy before the transplant, followed by donor stem cells. The goal is to see if the new immune system can co…

This study tests a drug called vosoritide in 56 children with short stature caused by certain genetic conditions. The drug targets the growth plate to help children grow faster. Participants are observed for 6 months, then treated with daily injections for 12 months to check safe…

This early-phase study tests a new treatment called Prula-cel for people with autoimmune diseases like lupus, scleroderma, and vasculitis. Prula-cel uses donor immune cells engineered to target and destroy faulty immune cells that attack the body. The goal is to find a safe dose …

This study tests a stem cell transplant using donors who are not a perfect match for patients with blood cancers like leukemia and lymphoma. Doctors give special drugs after the transplant to help prevent the donor cells from attacking the patient's body. The goal is to see if th…

This trial tests whether a procedure called catheter ablation works better than anti-arrhythmic drugs for people with structural heart disease who have had a dangerous fast heart rhythm (ventricular tachycardia). About 162 participants will be randomly assigned to either ablation…

This study tests a gene therapy called RGX-111 for people with MPS I, a rare genetic disorder that damages the brain and body. The therapy delivers a working copy of the missing IDUA gene directly into the fluid around the brain. The trial aims to check if the treatment is safe a…

This trial tests whether the drug ribociclib can reach brain tumors in people with recurrent high-grade glioma or meningioma. About 48 participants take the drug before their scheduled tumor-removal surgery. The goal is to measure how much drug gets into the tumor and surrounding…

This phase 2 trial tests whether clemastine, a common allergy medication, can improve thinking, motor skills, and social abilities in people with Williams syndrome. The study includes 30 participants aged 6 to 30 and uses a double-blind, placebo-controlled design to measure safet…

This study looks at whether a special type of CT scan, called dual energy CT, can better detect carcinoid tumors in the digestive system. About 71 people with signs or confirmed carcinoid tumors will get this scan during a routine check-up. The goal is to see if this imaging meth…

This trial is testing a new imaging tracer called FLT, which is used with a PET scan to measure how fast tumors are growing. The study includes people with various cancers or those awaiting a biopsy for a suspicious mass. The goal is to see if FLT PET imaging can help find and di…

This study will compare two ways to diagnose sarcoidosis, a disease that causes inflammation and lumps (granulomas) in the lungs. About 469 people with suspected stage I or II sarcoidosis will be randomly assigned to receive either a newer method called EBUS-guided cryobiopsy or …

This study looks at whether a protein called ApoC3 in the blood can help identify insulin resistance in women with polycystic ovary syndrome (PCOS), a common cause of infertility and other health issues. Researchers will measure ApoC3 levels in 200 women with PCOS and compare the…

This study tests whether two specialized eye imaging devices—the LipiView II and Keratograph 5M—can detect changes in tear film stability after using Miebo eye drops in people with dry eye. Twenty adults with normal tear film will have their eyes measured before and at several ti…

This study tests whether a compact 3-lead ECG device can accurately detect acute coronary syndrome (heart attack) in adults arriving at the emergency department with chest pain. Participants receive a HeartBeam ECG recording within 10 minutes of the standard 12-lead ECG. The goal…

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

This study planned to compare a newer blood test (presepsine) with a standard one (lactate) for diagnosing severe sepsis and septic shock in emergency department patients. The goal was to see if presepsine could detect these life-threatening infections more accurately. However, t…

This study is testing whether a blood test called Monocyte Distribution Width (MDW) can quickly identify patients with bacteria in their blood (bacteremia) in the emergency department. Researchers will observe 50,000 patients who have blood cultures ordered, comparing MDW results…

This study is testing a new finger-prick blood test that measures troponin levels in about 10 minutes, compared to the usual 2-hour lab test. Researchers hope it will help diagnose heart attacks faster and reassure patients who are not having one, allowing them to be discharged s…

Li-Fraumeni syndrome is a rare genetic condition that greatly increases the risk of developing many types of cancer. This pilot study will test three advanced MRI techniques—whole body STIR MRI, DW-MRI, and PET-MRI—in 30 people with the condition to see how well they can detect v…

This study is testing a new device called Gastric Alimetry that records the stomach's electrical activity through electrodes placed on the belly. Researchers want to see if it can reliably detect abnormal stomach rhythms in people with conditions like gastroparesis and functional…

This study enrolls 800 patients having heart attacks to test an artificial intelligence model that reads standard 12-lead ECGs. The AI aims to detect which artery is blocked and how severely blood flow is reduced, at the moment of a heart catheterization. If accurate, this could …

This study checks how well a new home sleep test system called SleepView+ can diagnose moderate to severe obstructive sleep apnea in people ages 12 and older. About 200 participants will first use the device during an overnight lab sleep study to compare results, then try it at h…

This study planned to test a new wearable wireless device that wraps around a child's abdomen to monitor central sleep apnea. It would have involved 15 children aged 1 month to 5 years already undergoing standard sleep studies. The device's accuracy and comfort would have been co…

This early-stage study tests an experimental vaccine designed to prevent cancer in adults with BRCA1 or BRCA2 gene mutations. The vaccine is given alone or with another drug, followed by a mild electric pulse to help it enter cells. Researchers want to see if it is safe and wheth…

This trial tests whether an educational program on sustainable diet and breastfeeding, given before delivery, can increase the number of mothers who exclusively breastfeed for up to six months. The study includes 429 pregnant women and also measures the environmental impact of di…

This study tests whether a dedicated care coordinator can help people with psoriasis or psoriatic arthritis reduce their risk of heart disease. About 520 adults aged 40-75 who do not already take cholesterol medication will receive support to manage risk factors like high cholest…

This study tests whether a 6-month program combining Bollywood-style dance classes and a fitness tracker can increase physical activity and improve metabolic health in South Asian Indian immigrant women aged 40-65 who are not very active. The goal is to see if this culturally tai…

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 is testing a digital health platform called STELA that creates personalized lifestyle plans to reduce risk factors for heart disease, like obesity, high cholesterol, and insulin resistance. About 100 adults without existing heart disease will use the platform for a yea…

This study tests whether coaching for Latino mothers on how to feed and put their babies to sleep can prevent obesity in the first two years of life. About 256 mothers and their healthy full-term infants will receive either group coaching or text-based support. The goal is to hel…

This study tests whether doing specific mouth and tongue exercises can help people with obstructive sleep apnea who cannot use a CPAP machine. Forty-five adults will be randomly assigned to real exercises, sham exercises, or unsupervised exercises for 10 weeks. The main goal is t…

This study compares two types of hands-on therapy—Stecco Fascial Manipulation and Rolfing Structural Integration—for people with piriformis syndrome, a condition that causes buttock and leg pain. Forty-two adults aged 25 to 45 with at least four weeks of pain will receive four we…

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 …

This study explores whether art therapy can help people with progressive supranuclear palsy (PSP) feel better and improve their daily life. Ten participants will take part in art sessions, and researchers will measure changes in quality of life, mood, and caregiver stress. The go…

This study tests whether microcurrent therapy—a very mild electrical stimulation—can reduce pain and improve daily life for people with fibromyalgia. About 50 adults will use a device at home alongside their usual pain care. The goal is to see if this simple, self-administered tr…

This trial tests whether a small device that stimulates the vagus nerve through the ear can improve quality of life for people with long COVID who also meet the criteria for ME/CFS (chronic fatigue syndrome). Participants will use the device daily for six weeks, and researchers w…

This trial tests whether slow intravenous ketamine infusions can safely reduce pain in people with fibromyalgia. Researchers will measure pain levels before and after treatment, and track how long any relief lasts. They will also look for traits that predict who responds best, ai…

This early study tests whether a non-invasive brain stimulation technique called TMS can help reduce pain, fatigue, and brain fog in people with fibromyalgia. About 30 adults will receive multiple TMS sessions and undergo MRI scans to see how the brain changes. The goal is to gat…

This study tests the long-term safety and effectiveness of an experimental drug called AXS-14 for people with fibromyalgia, a condition causing widespread pain and fatigue. About 300 adults who previously took part in a related study will receive AXS-14 for 52 weeks. Researchers …

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 follows children and adolescents with fetal alcohol spectrum disorder (FASD) who have already completed a dog-assisted therapy program. Researchers want to see if improvements in social skills, behavior, emotional well-being, and quality of life persist over 12 months.…

This study tests whether eye drops made from a patient's own platelet-rich plasma can improve dry eye symptoms. Participants with dry eye syndrome will receive either platelet-rich plasma drops or plasma drops without platelets. The trial measures changes in vision, tear film qua…

This study tests whether acceptance and commitment therapy (ACT) helps adults with chronic non-malignant pain (pain lasting 3+ months not caused by cancer). Sixty participants are randomly assigned to ACT, cognitive behavioral therapy (CBT), or standard medical care alone. Both A…

This trial tests Gelclair, a mouth rinse that forms a protective coating over the mouth's lining, to see if it eases the painful mouth sores (oral mucositis) caused by radiation therapy for head and neck cancer. The study enrolls adults with squamous cell carcinoma who will recei…

This study tests whether combining pain neuroscience education (teaching people how the brain processes pain) with therapeutic exercise helps people with fibromyalgia or chronic low back pain. Participants attend exercise sessions three times a week and one education session week…

This pilot study explores whether a 7-day fast followed by 11 weeks of a plant-based diet can reduce skin and joint symptoms in people with psoriasis and psoriatic arthritis. Researchers will also look at changes in gut bacteria and overall quality of life. The study involves 30 …

This study explores whether a supervised, circuit-based resistance training program can help glioblastoma patients maintain muscle mass and perform daily tasks more easily. Researchers will enroll 24 adults with glioblastoma who are currently on treatment. Participants will exerc…

This study aimed to see if a saffron-based supplement called AffronEye® could improve dry eye symptoms in university staff and students. It planned to compare the supplement to a placebo over 84 days using eye tests and symptom questionnaires. However, the study was withdrawn bef…

This study looks at whether low-dose rapamycin can reduce symptoms and improve quality of life in people with ME/CFS or Long-COVID. About 150 participants will take the drug as prescribed by their doctor and complete surveys and blood tests over time. The goal is to see if the dr…

This study looks at whether supervised physical activity helps people with fibromyalgia stay active longer than just getting advice. Fibromyalgia causes pain and fatigue, and inactivity can make symptoms worse. The study involves 330 adults who are currently sedentary. Researcher…

This study tests two different mouthpieces (mandibular advancement devices) for people with obstructive sleep apnea who cannot use a CPAP machine. Twenty-eight participants will wear one of the two devices for a year, and researchers will track comfort, side effects like jaw pain…

This study looks at whether starting palliative care early can help adults with aggressive leukemias or myelodysplastic syndrome (MDS) have a better end-of-life experience. Researchers will compare where patients die (home, hospice, or hospital) and how often they need hospital c…

This study tests an online program designed to help people with inflammatory arthritis remain employed. The program includes eLearning modules, group video sessions, and consultations with an occupational therapist and vocational counselor. About 528 participants from three Canad…

This study compares giving the drug infliximab over 30 minutes versus the standard 60 minutes in children with inflammatory bowel disease (IBD). The goal is to see if the shorter infusion causes just as few allergic reactions and improves patient and nurse satisfaction. Sixty chi…

This study aimed to see if a device called Sana could improve quality of life for people with fibromyalgia, a condition that causes long-term pain. Researchers planned to compare the active device to a sham (fake) device in adults aged 18 to 65 who had fibromyalgia symptoms for a…

This study looks at whether adding a daily 30-minute muscle stimulation session can improve pain control and quality of life for people who already have a spinal cord stimulator (BurstDR) for chronic back pain. Sixty adults with failed back surgery syndrome or persistent spinal p…

This study compares two exercise programs—asynchronous telerehabilitation (remote guidance) and a standard home exercise plan—for people with piriformis syndrome, a condition causing buttock and leg pain. About 46 adults aged 18-45 with confirmed diagnosis will participate. The g…

This study tests a personalized treatment plan for adults with moderate-to-severe IBS. Participants receive a combination of medical care, dietary guidance, psychological therapy, and physical therapy, chosen in an order that suits them. The goal is to reduce IBS symptoms by at l…

This early-phase trial tests whether eye drops made from mesenchymal stem cells can safely relieve symptoms in 10 women aged 40-60 with dry eye disease that didn't improve with artificial tears. Participants use the drops twice daily for two weeks and are followed for three month…

This study tests a combination of two medicines (atomoxetine and oxybutynin) given at bedtime for 6 months to children aged 6-17 with Down syndrome and obstructive sleep apnea. The goal is to see if the treatment improves sleep quality and thinking skills. About 36 children will …

This study tests whether a ketone supplement called Kenetik can reduce withdrawal symptoms in people undergoing alcohol detox. Fifty adults with alcohol use disorder will receive either the supplement or a placebo drink during their 5-day inpatient treatment. The goal is to see i…

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 whether a home-based telenursing program, led by a specialist nurse, can improve quality of life and reduce complications like falls for people with atypical parkinsonism (MSA or PSP). About 164 participants will receive proactive and reactive remote monitoring a…

This study is testing a full-spectrum cannabis extract containing CBD and THC in 36 women with fibromyalgia, a condition causing widespread pain, fatigue, and sleep problems. Participants will receive different doses over six months to see if it improves symptoms and quality of l…

This study looks back at the medical records of 500 children and adults with cancer who received immunotherapy at the National Cancer Institute. The goal is to understand the side effects, like inflammation and immune reactions, that can happen with these treatments. By learning …

This study collects and analyzes blood, DNA, and tissue samples from 315 adults with pulmonary fibrosis, their relatives, and healthy volunteers. The goal is to learn more about the disease's causes, not to test a new treatment. Researchers hope the findings will guide future the…

This study collects small skin samples from people with primary immunodeficiency or immune regulation disorders, as well as from healthy volunteers. Researchers will use these cells to create cell lines for laboratory research. The goal is to better understand immune diseases and…

This observational study aims to measure how many people in Portugal have Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS), two rare and severe forms of epilepsy. Researchers will review existing medical records from about three public hospitals to estimate the percentage of…

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

This study is creating a registry of 500 adults with functional GI disorders like IBS and chronic constipation. Researchers will collect medical data, diet and lifestyle questionnaires, and biological samples to explore genetic, microbial, and environmental factors. The goal is t…

This study looks at how well the heart, lungs, and muscles work together in people who have had a stem cell transplant. Researchers want to find out why transplant survivors have a higher risk of heart disease. About 350 adults with blood cancers will be followed for two years af…

This study is investigating how common muscle loss, known as sarcopenia, is in people with blood cancers like leukemia, lymphoma, and myeloma. Researchers will measure body composition using DXA scans and track hospital stays, disease progression, and survival. The goal is to see…

This study looks at whether short educational videos can help people with inherited cancer gene mutations learn more about their condition. About 43 adults who carry mutations in genes like BRCA1 or BRCA2 will watch the videos and then take a quiz. The goal is to see if this simp…

This study looks at how well people with sarcoidosis take their prescribed medicines and whether that affects their disease. Researchers will follow 150 adults in Maryland and D.C. for one year, tracking lung function, symptoms, and hospital visits. The goal is to find ways to he…

This study follows 500 people who survived a cardiac arrest to understand post-cardiac arrest syndrome (PCAS), a complex condition that often leads to death or brain damage. Researchers will compare different temperature management strategies and other treatments to see which mig…

This observational study will examine how the drug mitapivat changes red blood cells in 6 people with sickle cell disease. Researchers will measure oxygen binding and cell survival markers from blood samples and muscle oxygen tests. The goal is to understand the drug's effects, n…

This registry study follows 500 adults with obstructive sleep apnea who are prescribed a mandibular advancement device (MAD) — a dental appliance worn at night to keep the airway open. Researchers will track how the device is prescribed, how often patients actually use it, and wh…

This study is collecting blood, bone marrow, and tissue samples from 5,000 people with blood cancers like leukemia and lymphoma. Researchers will analyze the samples to find genetic abnormalities that cause cancer cells to grow. The goal is to understand why these cancers develop…

This study looks at how brain networks synchronize in people with fibromyalgia, a condition causing widespread pain. Researchers will use EEG and fMRI to measure brain activity and test a gentle brain stimulation technique called HD-tDCS. The goal is to understand pain processing…

This study investigates whether people with neurodegenerative diseases—like Alzheimer's, Parkinson's, or ALS—show changes in behavior, social circumstances, or health before and after their diagnosis. Researchers will compare the records of over 61,000 patients with those of matc…

This study looks at how continuous positive airway pressure (CPAP) therapy affects body signals like oxygen levels, heart rate, and breathing in adults with obstructive sleep apnea. Researchers will analyze data from 160 people who completed a previous trial, comparing signals be…

This study aims to better understand fatigue by comparing people who have felt tired for more than a month with those who are not fatigued. Researchers will use questionnaires, physical tests, and optional brain scans to identify different types of fatigue. The goal is to gather …

This study aims to find the genes that cause inherited hearing loss by analyzing DNA from 24,000 people in large Pakistani families. Researchers will compare affected and unaffected family members to identify new deafness genes. The goal is to improve genetic testing and counseli…

This study follows children aged 6 to 17 with active juvenile enthesitis-related or psoriatic arthritis who are being treated with secukinumab. Researchers will track how long children stay on the drug, how much their symptoms improve, and whether they achieve remission. The goal…

This study follows people with Phelan-McDermid syndrome, a rare genetic condition linked to autism and intellectual disability, to map how the syndrome unfolds over two years. Researchers will measure changes in thinking, behavior, language, and motor skills, and use brain imagin…

This study looks at whether specific brain wave patterns on an EEG can predict how well the drug fenfluramine works for people with Lennox-Gastaut Syndrome (LGS), a severe form of epilepsy. Researchers will follow 20 children and adults aged 2 to 35 who are already taking fenflur…

This study looks at the genetic makeup of rare blood diseases like Castleman disease and Langerhans cell histiocytosis. Researchers will analyze blood or tissue samples from 135 participants to find genetic changes that could be targeted by existing or experimental therapies. The…

This study follows individuals with autism who carry a PTEN gene mutation, along with comparison groups, to track changes in thinking, language, and behavior over time. Researchers will also collect blood samples and medical data to build a large database for future studies. The …

This study investigates whether advanced brain imaging and electrical recordings can predict the presence and severity of autism spectrum disorder (ASD) and intellectual disability (ID) in people with tuberous sclerosis complex (TSC). Researchers will follow participants over tim…

This study looks at whether a special type of testing, called leukemia profiling, can help find personalized treatment options for children and young adults up to age 30 with recurrent, refractory, or high-risk leukemias. Researchers analyze the genetic changes in each patient's …

This study is looking at how often people with small cell lung cancer also have symptoms of Lambert-Eaton myasthenic syndrome (LEMS), a condition that affects the nerves and can cause muscle weakness. Researchers will review medical records and check for LEMS symptoms in adults d…

This study uses health records from millions of people to see if GLP-1 receptor agonists (drugs for type 2 diabetes and weight loss) are linked to a higher risk of medullary thyroid carcinoma, a rare cancer. Researchers will compare how often this cancer occurs in people taking G…

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 looks at a rare liver disease called PFIC, which causes severe itching and liver damage. Researchers compare patients who took the drug odevixibat to those who did not, using data from past studies and a patient registry. The goal is to see if odevixibat helps avoid li…

This observational study looks at adults diagnosed with obstructive sleep apnea (OSA) who visit Sohag University Hospitals. Researchers will examine the severity of their condition, their specific symptoms, and any other health problems they may have. The goal is to build a clear…

This study is investigating whether testing for a panel of 24 genes, in addition to the well-known BRCA1 and BRCA2 genes, can better estimate the risk of breast and ovarian cancer. Researchers will analyze genetic samples from people with a family history of these cancers, as wel…

This study aims to create a large, long-term research cohort of women with polycystic ovary syndrome (PCOS). By systematically collecting follow-up data, researchers hope to understand how the condition evolves across different life stages and identify key factors that influence …

This study is a patient registry that will follow people of any age with bile acid synthesis disorders who are treated with Cholbam (cholic acid). The goal is to collect information over 10 years on how the drug performs in routine clinical care, including its safety, effectivene…

This trial tests whether wearable devices can measure how the body responds to everyday challenges like changes in salt intake, meals, fasting, and exercise in adults with endocrine or metabolic disorders. Participants wear home monitoring devices and follow different diet and ac…

This study follows children aged 6 to 21 who have a CNKSR2 gene mutation, which can cause epilepsy, language difficulties, and intellectual disability. Researchers will use brain scans, EEGs, and developmental tests to track how the condition changes over time. The goal is to cre…

This study tests a questionnaire called the PAP Problems Triaging Tool, designed to identify why people with obstructive sleep apnea struggle to use their positive airway pressure (PAP) machines. Researchers will ask adults newly prescribed PAP therapy to complete the questionnai…

This study follows pregnant women with chronic constipation who took the drug prucalopride and compares them to those who did not. Researchers track health outcomes for mothers and infants for one year after birth to see if the drug is linked to birth defects or other problems. N…

This study aims to improve how doctors diagnose and treat chronic muscle and joint pain by using simple questionnaires and pain sensitivity tests. Researchers will study 250 adults with conditions like osteoarthritis, fibromyalgia, chronic low back pain, or inflammatory arthritis…

This study aims to learn more about blood sugar patterns in women with Polycystic Ovary Syndrome (PCOS) using a continuous glucose monitor (CGM). Researchers will compare women with and without PCOS to see how their blood sugar differs in daily life. They will also test whether w…

This observational study follows people who have been exposed to Andes virus, a hantavirus that can cause severe respiratory illness. Researchers collect blood samples and monitor symptoms at key points from exposure through infection and recovery. The goal is to understand how t…

This study looks at tiny particles called exosomes, taken from a patient's own platelet-rich plasma, to see if they might help repair jaw joints. Researchers will compare these exosomes with other blood products in lab tests on cells. The goal is to understand their content and p…

This study examines walking patterns in people with Parkinson's disease and similar neurological conditions, as well as healthy volunteers. Researchers aim to identify unique 'gait signatures'—speed-dependent measures of walking—that could help distinguish between different disor…

This trial investigates whether kidney transplant candidates on dialysis can safely skip routine heart screening after being placed on the waiting list. Participants are randomly assigned to either no further screening or regular annual screening for hidden coronary artery diseas…

This study looks for biological markers in the blood and tissue of adults with Marfan syndrome that could signal when an aortic aneurysm is dangerous enough to require surgery. Researchers will compare three groups of Marfan patients: those without aortic aneurysms, those with st…

This pilot study is testing whether it's possible to run a larger study that focuses on heart disease in women. Researchers will gather information from 176 women aged 25-55 in Ontario who have had certain female-specific health issues like pre-eclampsia, gestational diabetes, or…

This study investigates cognitive problems in intensive care patients with acute respiratory distress syndrome (ARDS) who develop delirium (sudden confusion). Researchers use a non-invasive EEG cap and auditory tests to measure brain activity at the bedside. The goal is to identi…

This study looks at whether people with flat feet (pes planus) are more likely to have piriformis syndrome, a condition that causes pain in the hip and lower back. Researchers will compare foot posture and pain in adults aged 18-65 who have piriformis syndrome with those who do n…

This study was designed to look back at the health of people with Cushing's disease in Western France between 1990 and 2015. The goal was to find out how many patients went into remission and how many had their disease come back after their first surgery. However, the study was w…

This Phase 2 trial tests whether dichloroacetate (DCA), a drug that affects cell energy use, can alter tumor metabolism in people with recurrent glioblastoma. Forty patients scheduled for surgery will either receive DCA or no drug for one week before their operation. Researchers …

This study investigates factors that may influence cognitive skills—like memory, attention, and problem-solving—in women diagnosed with fibromyalgia. Researchers will assess thinking abilities using a detailed cognitive test and measure how fibromyalgia affects daily life. The go…

This study explores how women with fibromyalgia experience pain using a smartphone app with animations and body maps. Participants will describe their pain by choosing and adjusting animations that match their sensations. The goal is to find better ways to measure pain quality an…

This study follows 200 adults on ECMO (a heart-lung machine) for severe breathing problems. Researchers track how many get hospital-acquired infections and measure antibiotic levels in their blood and lungs. The goal is to learn more about infections and drug dosing in these crit…

This study looks at how patients with advanced blood cancers (like acute myeloid leukemia) who are not eligible for a cure, along with their nurses and doctors, feel about the benefits of blood transfusions. Researchers will give questionnaires before each transfusion to compare …

This study will survey directors of dental programs at Italian universities to find out if sleep medicine is part of the curriculum. The goal is to understand how much future dentists learn about sleep-disordered breathing like sleep apnea. About 70 program directors will answer …

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

This study aims to identify biomarkers—biological clues in the body—that can help distinguish between Parkinson's disease and similar conditions like progressive supranuclear palsy and multiple system atrophy. Researchers will follow people with early-stage parkinsonism and healt…

This study compares stainless steel and titanium-molybdenum alloy wires for moving canine teeth in people with braces. Sixteen participants will have one type of wire on each side of their upper jaw to see which works better. The goal is to find out which wire moves teeth more ef…

This study looks at people with ALS and similar diseases like frontotemporal dementia and hereditary spastic paraplegia. Researchers want to understand how genes affect the disease and find biological markers (biomarkers) to help develop future treatments. About 708 participants,…

This study aims to create a tool that predicts which patients with inflammatory rheumatic diseases (like rheumatoid arthritis, vasculitis, or polymyalgia rheumatica) will develop serious side effects from long-term steroid use. Researchers will collect routine medical data—such a…

This study looks at how diet, genetics, and gut bacteria work together to cause digestive problems like IBS, IBD, and celiac disease. Researchers will follow about 4,000 adults with and without these conditions to find patterns. The goal is to understand why some people get sick …

This study aims to find the genetic causes of kidney diseases like FSGS and nephrotic syndrome. Researchers will collect saliva and urine samples from up to 2,050 participants, including patients and their family members, as well as healthy volunteers. The goal is to understand w…

This pilot trial tests an online decision support tool called Chart that helps local health departments assess heat-health risks and plan for extreme heat events. Thirty health departments will either use Chart or receive standard heat-health information. The study measures how w…

This study was designed to see if a new method of measuring the visual field—by projecting vision zones on the ground—helps people with retinitis pigmentosa understand their remaining sight and improve their walking rehabilitation. The trial was withdrawn before enrolling any par…

This study looks at women who have had a heart attack or unstable angina but do not have major blockages in their heart arteries. Doctors will use special imaging techniques, like IVUS and MRI, to find hidden causes such as cholesterol plaque in artery walls or problems with tiny…

This study looks at how brain blood flow and nerve signals work in veterans who served in the Gulf War (1990-1992) and have Gulf War Illness. Researchers will compare them to healthy veterans using breathing tests, ultrasound, and MRI scans. The goal is to learn more about why so…

This study uses an iPhone app to track symptoms of psoriasis and psoriatic arthritis in 1,000 adults. Participants complete surveys, take photos of their skin, and use phone sensors to measure joint function. The goal is to see if smartphones can reliably monitor these conditions…

This study gathers 35-50 international critical care experts to agree on how to classify and manage septic shock using point-of-care ultrasound. The goal is to create consensus statements that could help personalize treatment. No patients are enrolled; instead, experts complete s…

This study is testing whether a simple breath test, called fractional exhaled nitric oxide (FeNO), can help measure the severity of obstructive sleep apnea (OSA). Researchers will compare FeNO levels in 200 adults with different levels of sleep apnea to see if the test can predic…

This study aimed to measure brain blood flow and oxygen levels in critically ill patients with septic shock, and to see how common treatments like fluids and blood transfusions affect them. The researchers planned to use ultrasound to monitor blood flow in the brain. However, the…

This study follows 90 people who survived severe COVID-19 pneumonia and needed intensive care. Researchers will check their physical health, mental health, and quality of life at 3 months, 6 months, 1 year, and 5 years after leaving the ICU. The goal is to better understand the l…

This study looks at why people with antiphospholipid syndrome (APS) — a condition that raises blood clot risk — may have narrowed brain arteries. Researchers will use advanced MRI scans on 60 APS patients to check for artery narrowing and plaque buildup. The goal is to better und…

This study examines whether a low pulse pressure (the difference between top and bottom blood pressure numbers) in the first hours after a cardiac arrest is linked to early death. Researchers will analyze data from 3500 patients who had a cardiac arrest outside the hospital and a…

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

This study looks at whether diabetes or blood sugar problems can worsen thinking and memory issues in people with myotonic dystrophy type 1. Researchers will use MRI brain scans and thinking tests over 4 years to track changes. The goal is to understand why some patients have mor…

This study aims to find genetic and metabolic markers that explain why some people develop diabetes, high blood pressure, or bone loss from glucocorticoids while others do not. Researchers will analyze blood and urine samples from up to 600 participants, including those with exce…

This study tests whether sending a short screening questionnaire directly to people with psoriasis can help detect psoriatic arthritis earlier. 18,000 participants will be randomly assigned to receive the questionnaire or not. Those with high scores will be advised to see a rheum…

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 looks at whether offering genetic testing to family members of people with hereditary breast and ovarian cancer syndrome helps more relatives learn about their own cancer risk. Researchers will follow 118 participants and their relatives to see how many choose to get t…

This study looks at how histiocytosis—a group of rare disorders—may affect memory, thinking, and brain structure. Researchers will test 13 adults with these conditions using thinking tasks and MRI scans. The goal is to better understand brain changes, not to provide treatment.…

This study was designed to see if a new type of airway device can prevent dangerous drops in oxygen levels during anesthesia in patients who are at high risk, such as those with obesity or sleep apnea. Researchers planned to compare the new device with standard ones in adults und…

This study is not testing a new drug, but rather doing the early science needed to develop one. Researchers are looking for the right targets (antigens) on adrenocortical carcinoma cells and studying how the tumor suppresses the immune system. The goal is to find a way to create …

This study looks at why sleep apnea raises the risk of diabetes. Overweight adults with and without sleep apnea will have their blood sugar control measured. Some will receive CPAP, some will get extra oxygen at night, and others will get a sham treatment. The goal is to find out…

This study brings together 60 international experts to agree on clear definitions and management strategies for intra-abdominal hypertension and abdominal compartment syndrome, serious conditions where pressure builds dangerously in the abdomen. Using a structured online survey, …

This study looks at immune system markers in the saliva and blood of 80 people with primary Sjögren's syndrome, a chronic disease causing dry eyes and mouth. Researchers will check for antibodies that may block a key enzyme and see how gum disease affects symptoms. The goal is to…

This study is observing 60 people who received a special hip replacement cup (Plasmafit Revision Structan) during routine surgery. Researchers are checking how well the cup works and how long it lasts by tracking hip function and any need for repeat surgery. No new treatment is b…

This study looks at whether measuring salt levels in the kidneys using a special MRI can help doctors predict which heart failure patients will not respond well to water pills (diuretics). About 50 adults with heart failure and kidney disease will be scanned to compare salt conte…

This study uses advanced MRI scans and genetic tests to track how neurodegenerative diseases like frontotemporal dementia and ALS spread across the brain's network. Researchers will follow 645 participants, including patients, family members, and healthy controls, for up to 2 yea…

This early-stage study tests whether the cholesterol drug atorvastatin can reduce levels of a faulty protein (p53) in people with various cancers, including solid tumors and leukemia. About 50 participants will take atorvastatin for 1 to 4 weeks before their planned surgery or tr…

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 is testing a new type of clinic that screens people with prediabetes and type 2 diabetes for several hidden health problems at the same time, including fatty liver disease, sleep apnea, and heart failure. The goal is to see how common these undiagnosed conditions are a…

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 international registry is collecting information from 2000 people who have had spontaneous coronary artery dissection (SCAD), a rare cause of heart attacks. Researchers will track participants' health over time to better understand the condition and develop best practices fo…

This study is a large international registry that will collect data from up to 130,000 people who have had a heart attack or unstable chest pain. Researchers will track treatments, outcomes, and safety to understand what works best in real-world care. No new drugs or devices are …

This study watches 40 adults who need a leadless pacemaker to see if where the device sits inside the heart changes heart rhythm or pumping strength. Researchers will use CT scans and ECGs to check heart function and valve leakage. The goal is to find the best spot for the pacema…

This study looks at how common Charles Bonnet Syndrome (CBS) is in people with vision loss from conditions like macular degeneration or glaucoma. CBS causes complex visual hallucinations without mental illness. Researchers will survey 640 participants to measure how often it happ…

This study looks at skin and blood samples from 200 people with ichthyosis (a genetic condition causing dry, scaly skin) and healthy volunteers. Researchers want to find specific markers that could help them understand the disease better and develop new treatments. No treatment i…

This study follows 120 adults with Alpha-1 Antitrypsin Deficiency, a genetic condition that can cause lung and liver problems. Researchers use liver biopsies and genetic samples over 5 years to find out who develops liver scarring or cirrhosis and why. The goal is to identify ris…

This study compares genetic differences and muscle gene activity in people who develop acute compartment syndrome after a leg fracture versus those who do not. Researchers will measure a protein called myostatin in muscle tissue. The goal is to better understand why some people g…

This pilot study aimed to see if a diet low in two specific nutrients (cysteine and methionine) could affect metabolism in patients with recurrent gliomas. Up to 30 participants were to be randomly assigned to either a normal diet or the special diet for 2, 4, or 7 days before st…

This study looks at whether mepolizumab, a drug already used to reduce asthma attacks, can also improve physical activity and exercise tolerance in people with severe asthma. Researchers will track daily steps and activity levels in 62 adults over six months. The goal is to see i…

This study aims to create a test that predicts how hospitalized COVID-19 patients will fare in the short and long term (up to one year). Researchers will enroll 600 adults from hospitals and Veterans' facilities, using medical records and lab data to forecast risks like frailty, …

This study compares two ways to predict heart and metabolic problems in people with mild-to-moderate obstructive sleep apnea: one based on inflammation in the body, and the other based on how severe the sleep apnea is. Researchers will measure things like waist size, blood pressu…

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

This study reviewed the medical records of 2500 people who received hyperbaric oxygen therapy (HBOT) for conditions like traumatic brain injury, stroke, fibromyalgia, or age-related decline. Researchers compared computerized thinking and memory tests taken before and after treatm…

This study looks at 40 people who recently had a heart attack and had one blockage treated. Researchers will use special pressure wires and optical imaging to examine other blockages that were not treated. The goal is to see how many of these leftover blockages are actually dange…

This study looks at whether pretzels made with safflower oil and soy can help postmenopausal women with metabolic syndrome maintain a healthy weight. Twenty women will eat three different types of pretzels over 14 weeks, and researchers will measure how their bodies process the s…

This study is testing a Turkish version of a questionnaire that looks at how chronic pain conditions like fibromyalgia and back pain affect sexual function and satisfaction. Researchers will ask 170 people with chronic pain and a stable partner to fill out the survey. The goal is…

This study tracks 40 people with nasal polyps and severe asthma who are taking mepolizumab, a drug that reduces inflammation. Researchers collect nose, blood, and sputum samples before treatment and at 3, 6, and 12 months to see how the drug affects symptoms, nose bacteria, and g…

This study follows 60 people with lower-risk myelodysplastic neoplasms (MDS) who need regular red blood cell transfusions. Over 6 months, participants use a smartphone app to answer weekly quality-of-life questions and take photos of their fingernails or eyelids. Researchers aim …

This pilot study aimed to see if a new care pathway could help family doctors better diagnose Irritable Bowel Syndrome (IBS) and decide when a specialist is needed. The study was withdrawn before any participants enrolled, so no results are available. It was designed for general …

This study tests whether a software tool called the Respiratory Knowledge Portal (RKP) can help ICU teams reduce complications in patients on breathing machines. Researchers will collect data from 875 patients to see if the tool improves how alarms are set, helps wean patients of…

This study tests a new, smaller feeding tube with built-in sensors to measure breathing signals in extremely premature babies (under 1000 grams). The current standard tube is too long for these tiny infants, so the new design shortens the sensor spacing to fit better. Ten babies …

This study looks at whether measuring body water with a harmless device can help detect obstructive sleep apnea (OSA) in pregnant women with severe obesity. OSA is common but often missed in this group, and can be dangerous for both mother and baby. Researchers will enroll 52 wom…

This pilot study examines proteins in the vitreous (the gel-like fluid inside the eye) of patients with pseudoexfoliation syndrome (PEX), a condition that can lead to glaucoma. Researchers will compare protein levels between eyes with and without PEX in 24 adults undergoing eye s…

This study at Boston Children's Hospital will test whether measuring thousands of proteins in a few drops of blood can predict which children with congenital heart disease will have serious complications after heart surgery. Researchers will collect blood samples from 60 children…

This study is testing whether a computer tool called MEKANOS can better predict the risk of hip fractures in people with Fibrous Dysplasia or McCune-Albright Syndrome. Researchers will take CT scans of the femur and use the tool to calculate bone strength. The goal is to improve …

This study looks at how well treatments for childhood hip conditions (like hip dysplasia and Perthes disease) work over the long term. Researchers at Mayo Clinic are following 500 adults who were treated for these conditions as children. They will check how many needed another su…

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 follows 248 adults receiving a CoreHip hip replacement to see how their hip function, pain, and leg length change over time. Participants are treated as part of routine care, and no extra tests or treatments are given. The goal is to collect real-world data on the impl…

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 screened over 16,600 adults aged 45-70 with at least one risk factor (like high blood pressure or smoking) but no known heart disease. Researchers used ultrasound and simple blood and urine tests to find hidden signs of atherosclerosis, pre-diabetes, and kidney disease…

This study aims to understand the genetic factors behind obstructive sleep apnea (OSA) and its related health issues. Researchers will enroll 5,000 adults who have been diagnosed with or are suspected of having sleep apnea. Participants will provide genetic samples and answer sle…

This study looks at whether people with Fabry, Gaucher, or Hunter disease are more likely to continue their IV treatment when it's given at home versus at a hospital. Researchers will review existing data from 222 patients in Mexico. No new treatments are given; the goal is to un…

This study looks at whether healthcare providers can use skills learned on mannequins when helping real newborns breathe. About 120 respiratory therapists will be observed in both a simulation lab and the delivery room. The goal is to see if their ability to manage a baby's mask …

This study measures how often people with myotonic dystrophy type 1 fall and what factors affect their balance. Researchers will also assess fear of falling and leg muscle strength. The goal is to better understand fall risk in this condition.

This study is testing a new blood test called AlloHeme that measures how much of a patient's own cells remain after a bone marrow transplant. The goal is to see if this test can predict whether the cancer (acute leukemia or myelodysplastic syndrome) will come back. About 307 adul…