Syndromic disease

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 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 looks at whether giving red blood cell transfusions only when a child is bleeding or not getting enough oxygen (instead of using a fixed hemoglobin level) can reduce organ damage and improve brain development. It involves 228 children under 6 years old on ECMO life sup…

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

This Phase 2 trial tests a combination of pembrolizumab (Keytruda) and quavonlimab against other drug combos in 302 people with stage IV colorectal cancer that has a specific genetic feature (MSI-H or dMMR). The goal is to see if the new combo shrinks tumors better and for longer…

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

This study is testing a new hip implant called the OR3O Dual Mobility System in 177 people who need hip replacement surgery. The goal is to see if the implant is safe and lasts longer without needing another surgery. Researchers will follow participants for up to 10 years to chec…

This study compares the standard drug azacitidine alone versus azacitidine combined with either lenalidomide or vorinostat in 282 patients with higher-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). The goal is to see if any combination improves re…

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 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 tests a device that uses heat to ablate (destroy) a small part of the ovary in women with polycystic ovary syndrome (PCOS) who have trouble getting pregnant. The goal is to restore ovulation without needing daily medication. About 195 women will be randomly assigned to…

This Phase 3 trial compares the drug niraparib to the standard chemo temozolomide in 450 adults with newly diagnosed, aggressive glioblastoma that has a specific genetic marker (MGMT unmethylated). Participants take the assigned drug daily during radiation, then continue until th…

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 phase 1 trial is testing a combination of total marrow and lymphoid irradiation (TMLI) plus two chemotherapy drugs, fludarabine and melphalan, given before a donor stem cell transplant. The goal is to find the safest dose of radiation that can kill cancer cells while prepari…

This phase 2 trial tests whether adding the drug pomalidomide to standard chemotherapy (daunorubicin and cytarabine liposome) helps people with a newly diagnosed, aggressive form of acute myeloid leukemia (AML) linked to prior bone marrow disorders. About 50 participants will rec…

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 whether luspatercept works better than epoetin alfa for treating anemia in adults with a certain type of low-risk MDS who have not needed blood transfusions or prior anemia drugs. About 360 participants will receive one of the two drugs, and researchers will trac…

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 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 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 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 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 phase 2 trial tests whether the supplement fisetin can improve lung disease in people with common variable immunodeficiency (CVID). Twenty participants will receive either fisetin or a placebo on four days over a month. The study measures changes in immune cells and lung fun…

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 giving a higher radiation dose to a brain area called the subventricular zone (where stem cells live) can delay glioblastoma from coming back. About 60 adults with newly diagnosed glioblastoma will receive standard radiation and chemo, but half will get e…

This study tests whether benralizumab can improve lung structure and function in people with severe eosinophilic asthma who still have symptoms despite standard treatments. About 75 adults aged 18-70 will receive either the drug or a placebo for 48 weeks, followed by a 4-week che…

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 phase 3 trial compares two types of stem cell transplants for children, adolescents, and young adults with acute leukemia or myelodysplastic syndrome (MDS). One uses a half-matched family donor (haploidentical), the other a fully matched unrelated donor. The goal is to see w…

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 early-stage trial tests an experimental drug called BI 3000202 in 16 adults with rare type 1 interferonopathies, such as Aicardi-Goutières syndrome. Participants take a low dose for 4 weeks, then a higher dose for 36 weeks. The main goal is to see if the drug is safe and how…

This study tests an experimental drug called AG-946 (tebapivat) in 87 adults with lower-risk myelodysplastic syndromes (MDS) who have anemia. The goal is to see if the drug can raise hemoglobin levels or help patients go without blood transfusions for at least 8 weeks. Participan…

This early-phase trial tests a drug called lintuzumab-Ac225 in 18 adults with high-risk myelodysplastic syndrome (MDS) that did not improve after standard treatment. The drug is an antibody that seeks out cancer cells and delivers a radioactive payload to kill them. The main goal…

This study tests whether weekly infusions of Alpha-1 MP (a protein replacement) can slow lung damage in 345 people with emphysema caused by Alpha-1 antitrypsin deficiency. Participants receive one of two doses or a placebo for three years, with lung CT scans tracking changes. The…

This phase 2 trial tests a drug called VK-2019 in 13 patients with Epstein-Barr virus (EBV)-positive nasopharyngeal cancer that has returned or spread and has no standard treatment. VK-2019 works by blocking a key viral protein, EBNA1, which the virus needs to survive. The main g…

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 early-stage trial is testing an experimental drug called APX005M in 32 children with brain tumors that have come back or are hard to treat, including a type of brain stem glioma. The drug works by activating the immune system to attack cancer cells. The main goals are to fin…

This early study tests the safety of TheraSphere PCa, tiny radioactive glass beads injected into the prostate to treat localized prostate cancer. The trial involves 36 men with favorable intermediate-risk cancer and aims to find the maximum safe radiation dose. Researchers will m…

This study compares a new three-drug combination (NABPLAGEM) against a standard two-drug chemo regimen for people with advanced pancreatic cancer that has spread and carries a BRCA or PALB2 gene mutation. The trial enrolls 10 participants whose cancer worsened after initial treat…

This study looks at the long-term safety of a weekly intravenous treatment for people with emphysema caused by alpha-1 antitrypsin deficiency. About 290 adults who completed a prior study will receive the drug for 2 more years. The main goal is to monitor side effects, not to cur…

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 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 phase 3 trial tests whether a new drug, depemokimab, works as well as the current treatment mepolizumab for adults with a rare disease called EGPA, which causes inflammation of blood vessels. The study involves 163 participants and aims to see if depemokimab can help patient…

This Phase 2 trial tests whether N-Acetylcysteine (NAC), an antioxidant, can help people with ME/CFS (chronic fatigue syndrome). Researchers will give 95 participants different doses of NAC or a placebo for four weeks, then measure brain glutathione levels and markers of oxidativ…

This pilot study is testing whether the drug luspatercept can help people with lower-risk myelodysplastic syndromes (MDS) need fewer blood transfusions. Ten adults with MDS will receive the drug by injection. The main goal is to see if it is safe and reduces transfusion needs.

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 donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…

This phase 3 study tests whether ianalumab, a biologic drug, can reduce disease activity and symptoms in people with active Sjogren's syndrome. About 506 adults with a recent diagnosis (within 7.5 years) and positive anti-Ro/SSA antibodies are randomly assigned to receive ianalum…

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

This early-phase trial tests a gene therapy called MVX-220 in 12 children and adults with Angelman syndrome. The therapy delivers a working copy of the UBE3A gene via a single injection into the fluid around the brain. Researchers are primarily checking safety, but also looking f…

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

This early-stage study tested the safety and best dose of vorinostat when given with the chemotherapy drug temozolomide in 83 adults with aggressive brain tumors called malignant gliomas. The goal was to see if adding vorinostat could make the tumor cells more sensitive to chemot…

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

This study 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 phase II trial is studying a combination of chemotherapy (Hyper-CVAD) and the targeted drug dasatinib, with or without a donor stem cell transplant, in 97 adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL). The goal is to see how well this approa…

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 phase 3 trial tests whether an oral antioxidant called N-acetylcysteine (NAC) can slow the loss of vision in people with retinitis pigmentosa, an inherited eye disease that causes gradual blindness. About 485 adults aged 18 to 65 are taking NAC or a placebo for several years…

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

This study was designed to test whether adding olutasidenib to standard chemotherapy drugs could help control certain blood cancers in people with an IDH1 gene mutation. The trial was withdrawn before enrolling any participants, so no results are available.

This phase 2 trial tests whether the drug TPST-1495 can reduce the number and size of polyps in the small bowel and colon of people with familial adenomatous polyposis (FAP), an inherited condition that greatly raises colon cancer risk. About 38 participants will take the drug by…

This study tests whether starting luspatercept (Reblozyl) at the maximum approved dose can help people with low-risk myelodysplastic syndromes (MDS) go without red blood cell transfusions for at least 8 weeks. About 106 participants who currently need regular transfusions will re…

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

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

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

This study tests whether giving a drug that blocks a key inflammatory protein (TNF-alpha) within 6 hours after a cardiac arrest can help more people survive. The trial includes 208 adults who remain unconscious after their heartbeat is restored. The goal is to see if the drug imp…

This early-phase trial tests whether adding special immune cells called veto cells to a stem cell transplant can help donor cells grow in the patient without causing severe graft-versus-host disease (GVHD). The study includes 16 people with various blood cancers or bone marrow fa…

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

This study looks at the long-term safety and effectiveness of a drug called sonelokimab for people with psoriatic arthritis. About 1560 adults who finished an earlier study will receive the drug as a shot under the skin. Researchers will track side effects and how well the drug c…

This study tests whether combining two immunotherapy drugs (nivolumab and ipilimumab) with radiation therapy can control or shrink tumors in people with colorectal or pancreatic cancer that has specific genetic features (MSS or MSI-high). About 84 adults with advanced disease are…

This study tests whether low-dose sirolimus can safely improve blood cell function in adults with RUNX1 familial platelet disorder, a genetic condition that raises bleeding and leukemia risks. Six participants receive the drug and are monitored for side effects and changes in blo…

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

This study 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 study offers continued treatment with the drug ceritinib to people with ALK-positive cancers who were already benefiting from it in a previous Novartis study. The main goal is to track safety and side effects over time. About 233 participants will receive the drug as long as…

This study tests a combination of two drugs, venetoclax and ASTX727, in people with high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) who haven't had treatment before. The goal is to find the safest dose and see if the combo can shrink or control …

This study tests a gene therapy called NGN-401 in 33 girls with Rett syndrome, a rare genetic disorder that affects development and movement. The therapy delivers a working copy of the MECP2 gene to try to improve skills and daily function. Researchers will measure success by whe…

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 phase 2 trial tests the drug talazoparib in 150 people with advanced cancers that have not responded to standard treatments. Participants must have specific gene changes in BRCA1, BRCA2, or related DNA repair genes. The drug works by blocking an enzyme that helps cancer cell…

This study looks at how safe and effective the drug everolimus is for Taiwanese adults with tuberous sclerosis complex (TSC) who have kidney tumors called angiomyolipomas. Four participants will take the drug and be monitored for side effects and tumor shrinkage. The goal is to c…

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

This study 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 trial tests a personalized treatment plan for people with advanced colorectal cancer that has spread to the liver and stopped responding to standard treatments. The approach combines a liver-directed chemotherapy (HAIC), a targeted therapy (fruquintinib or cetuximab), and an…

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 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-stage trial tests a new drug called MP0533 in adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or not responded to treatment. The drug is designed to help the body's immune cells find and attack cancer cells. The study will …

This Phase 2 study tests an experimental drug called SPY002-072 in 285 adults with moderately to severely active rheumatoid arthritis, psoriatic arthritis, or axial spondyloarthritis. Participants receive either the drug or a placebo, and researchers measure changes in disease ac…

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

This phase 3 study tests guselkumab (Tremfya) in 453 adults with active psoriatic arthritis who did not respond to or could not tolerate a prior anti-TNF drug. Participants receive either guselkumab or a placebo injection. The main goal is to see if guselkumab reduces joint swell…

This phase 3 trial tested guselkumab, a biologic drug given as a shot, in over 1,000 people with active psoriatic arthritis who had not responded well to other treatments. The goal was to see if it reduces symptoms like joint pain and swelling and slows joint damage seen on X-ray…

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 study is testing the long-term safety of secukinumab, a biologic drug, in people with polymyalgia rheumatica (PMR). It includes 161 patients who already completed a 52-week study of secukinumab and had a relapse after stopping treatment. The main goal is to see how safe and …

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 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 study tests whether bimekizumab works better than risankizumab for adults with active psoriatic arthritis (PsA), a condition causing joint pain and skin lesions. About 684 participants will receive one of the two drugs for 16 weeks. The main goal is to see who gets at least …

This phase 4 trial tests whether guselkumab (TREMFYA) can reduce spine-related symptoms in people with active psoriatic arthritis who have not used biologic drugs before. About 411 participants will receive either guselkumab or a placebo as a shot under the skin. The main goal is…

This phase 3 trial tests whether nipocalimab can safely reduce disease activity in adults with moderate to severe Sjogren's disease. About 655 participants receive either nipocalimab or a placebo, along with standard care, for 48 weeks. The main goal is to see if the drug improve…

This early-stage trial tests a new drug called JNJ-89853413 in 64 people whose acute myeloid leukemia (AML) or myelodysplastic neoplasms (MDS) have come back or stopped responding to treatment. The main goal is to check safety and find the right dose, with a second part looking a…

This study tests an experimental drug called JNJ-77242113 (icotrokinra) against a placebo in 552 adults with active psoriatic arthritis who have not tried biologic treatments before. The main goal is to see if the drug reduces joint swelling and tenderness after 16 weeks. Partici…

This study follows 159 children aged 2 to 17 who have already completed a prior ustekinumab study for Crohn's disease, ulcerative colitis, or psoriatic arthritis. The goal is to collect long-term safety data by monitoring side effects, lab results, and injection-site reactions. P…

This study monitors long-term safety in 120 cancer patients who are already benefiting from treatments like JNJ-75348780, safimaltib, or ibrutinib in earlier trials. Participants have blood cancers, solid tumors, or prostate cancer. The goal is to track serious side effects over …

This study tests if a new 2mL auto-injector or pre-filled syringe delivers the same amount of the drug ianalumab into the body as the current 1mL syringe. About 155 adults with rheumatoid arthritis, Sjögren's disease, or lupus will receive the drug both ways to compare how much g…

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

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

This study is testing two injectable drugs, ustekinumab and guselkumab, in 50 children with active juvenile psoriatic arthritis. The goal is to see how well the drugs work, how they move through the body, and if they are safe. Participants will receive one of the drugs and be mon…

This study tests an oral medication (decitabine/cedazuridine) in people with BAP1 gene mutations who have early-stage mesothelioma that is not yet causing symptoms. The goal is to see if the drug can stop the cancer from growing or shrink it. About 9 participants will receive tre…

This early-phase study tests a vaccine called SurVaxM in 35 children with certain brain tumors that have come back or are hard to treat. The vaccine is designed to teach the immune system to find and destroy tumor cells that have a protein called survivin. The goal is to see if i…

This study follows about 111 people with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex who are taking Epidyolex (a CBD oral solution) for seizures. Researchers track how many stay on the drug for a year and how many seizure-free days they have. It's an o…

This study tests a new way to treat children and young adults with high-risk blood cancers like leukemia and lymphoma. Doctors take blood cells from a family donor, remove certain T cells that can cause a harmful immune reaction, and then transplant the modified cells. The goal i…

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 early-phase study tests whether adding a drug called S-Gboxin to standard treatment can help people with glioblastoma, a fast-growing brain cancer. The drug works by cutting off the energy supply to cancer cells. Ten patients with recurrent or progressive disease will receiv…

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 study tests a pill called ruxolitinib for people whose immune system attacks their bone marrow, causing conditions like severe anemia. The drug aims to stop this attack and help the bone marrow recover. Participants take the pill twice daily for up to 6 months and are follow…

This early-phase study tests a new drug called uproleselan combined with two standard chemotherapy drugs for children and teens with acute myeloid leukemia or related blood cancers that have returned or not responded to treatment. The drug works by blocking a protein that helps c…

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 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 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 tests a combination of two chemotherapy drugs (CPX-351) plus a targeted drug (quizartinib) in people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The goal is to find the best dose and see if the combo helps control the cancer. About 52…

This phase 2 trial tests the drug olaparib in 14 adults with acute myeloid leukemia or myelodysplastic syndrome that has returned or not responded to treatment, and whose cancer has a specific change in the IDH gene. Olaparib is a pill that may block enzymes cancer cells need to …

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 a new blood-filtering method called adaptive blood purification (ABP) for people with septic shock. The treatment aims to remove inflammatory substances from the blood. 276 adults in intensive care will be randomly assigned to receive either standard care or stan…

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 looks at whether taking a yerba mate extract can help improve health markers in overweight adults. Eighty people aged 40-65 will take either the extract or a placebo for 90 days. Researchers will measure changes in weight, blood sugar, cholesterol, and inflammation to …

This early-phase trial is testing whether adding an experimental drug called Q702 to two standard leukemia drugs (azacitidine and venetoclax) is safe for people with relapsed or refractory acute myeloid leukemia (AML). About 17 adults whose leukemia has not responded to or has re…

This phase 3 trial compares two treatments for people with a bone marrow disorder called myelodysplastic syndrome (MDS) who have low red blood cell counts (anemia). One group gets lenalidomide alone, the other gets lenalidomide plus epoetin alfa, a drug that stimulates red blood …

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 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 planned to test a new cell therapy (CD19-BCMA CAR-T) for people with connective tissue diseases like lupus and rheumatoid arthritis. The goal was to see if it is safe and can control the disease. However, the study was withdrawn before enrolling any participants, so no…

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

This study looks at the long-term safety of a CBD gel (ZYN002) applied to the skin for children, teens, and young adults with Fragile X syndrome. Participants from two earlier studies will use the gel twice daily for 52 weeks. The main goal is to check for side effects, with a se…

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 looks at whether switching to a plant-based diet can improve heart health and reduce inflammation in people who have had a kidney transplant. Twenty-five participants who are at least three months post-transplant will follow a plant-based diet for 16 weeks after a two-…

This study tests whether giving surfactant (a natural substance that helps lungs stay open) through a small airway device placed above the vocal cords, along with standard breathing support (CPAP), is better than CPAP alone for late preterm and early term infants with respiratory…

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

This study tests whether infusions of cord blood stem cells can help people with moderate to critical COVID-19 pneumonia. The trial includes 65 adults with COVID-19-related acute respiratory distress syndrome (ARDS). Researchers will compare stem cell treatment plus standard care…

This study tests a combination of two chemotherapy drugs (CPX-351 and gemtuzumab ozogamicin) in about 50 adults with acute myeloid leukemia (AML) that has returned or not responded to treatment, or with high-risk myelodysplastic syndrome (MDS). The goal is to see if the combo is …

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

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

This study tests the drug bimekizumab in 89 adults with moderate to severe plaque psoriasis, some of whom also have psoriatic arthritis. Researchers will measure changes in gene activity in skin samples after 48 weeks of treatment. The goal is to understand how the drug affects t…

This early-phase trial is testing whether adding the drug venetoclax to standard chemotherapy before and after a bone marrow transplant can help prevent cancer from coming back in people with high-risk acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or related blood…

This early-stage trial tests a combination of two drugs, venetoclax and azacitidine, in people with a serious type of myelodysplastic syndromes (MDS) who have not had treatment before. The goal is to find the safest dose and see how well the drugs work together to control the dis…

This early-phase study is testing a drug called TAK-243 in people whose acute myeloid leukemia or myelodysplastic syndrome has come back or is not responding to treatment. The goal is to find the best dose and check for side effects. The trial is currently on hold.

This study tests whether whole-body electromyostimulation (WB-EMS), a technology that stimulates muscles with mild electrical pulses, can lower resting blood pressure and improve other heart risk factors in overweight adults aged 50+ with mild hypertension. Over 12 weeks, 28 part…

This early-stage trial is testing the safety and potential benefit of pembrolizumab, an immunotherapy drug, in 71 children whose brain tumors (like DIPG, high-grade gliomas, or medulloblastoma) have come back or not responded to standard treatments. The drug works by helping the …

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

This trial aimed to test the drug ASTX727 (Inqovi) alone or with donor immune cells in people with blood cancers who had a stem cell transplant. The goal was to see if it could control cancer that remained or came back. However, the study was withdrawn before enrolling any patien…

This phase 3 study tests whether the drug ianalumab can reduce disease activity in people with active Sjogren's syndrome. About 275 adults with the condition will receive either ianalumab or a placebo shot under the skin. The main goal is to see if the drug improves symptoms and …

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

This phase 2 study tests a drug called MAS825 in 17 people with rare genetic conditions that cause severe inflammation. The goal is to see if the drug can prevent disease flares better than a placebo. Participants will be monitored for safety and effectiveness over several period…

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

This study tests whether adding venetoclax to ASTX727 (a two-in-one pill) works better than ASTX727 alone for people with chronic myelomonocytic leukemia or related bone marrow cancers with too many immature cells. About 132 adults will be randomly assigned to one of the two trea…

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

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

This early-phase study tests a stem cell transplant method for people with advanced blood cancers like leukemia. Instead of using strong drugs to prevent complications, doctors give specially selected immune cells (regulatory T-cells) along with regular T-cells to help the body a…

This Phase 3b study is testing whether the drug benralizumab (Fasenra) can allow people with severe eosinophilic asthma to safely reduce their daily maintenance medications. About 504 participants aged 12-75 in China will try to step down from drugs like inhaled steroids or long-…

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 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 adding a myo-inositol supplement to the standard fertility drug letrozole helps women with polycystic ovary syndrome (PCOS) achieve pregnancy. About 168 women will receive either the supplement or a placebo alongside letrozole for up to 5 treatment cycles…

This early-phase trial is testing a drug called AZD6738 in 52 people with myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) whose disease got worse after standard therapy. The drug works by blocking a DNA repair pathway that cancer cells rely on, causing th…

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

This early-phase trial tests a new targeted drug called pivekimab sunirine (PVEK) alongside a standard chemotherapy regimen (FLAG-Ida) in 30 adults with newly diagnosed, high-risk acute myeloid leukemia or related blood cancers. PVEK works like a smart bomb, attaching to a protei…

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

This study 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 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 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 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 early-stage trial tested a personalized DNA vaccine designed to train the immune system to attack glioblastoma, a fast-growing brain cancer. Nine patients with a specific type of glioblastoma (unmethylated MGMT) received the vaccine along with an immune booster. The main goa…

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 phase II trial tests the safety and effectiveness of a drug called XmAb20717 in 140 people with advanced rare cancers, including mesothelioma, lymphoma, and small cell lung cancer. The drug is given by IV. The main goal is to see if tumors shrink. The trial is currently susp…

This phase 3 trial tests the long-term safety of GTX-102 (apazunersen) in 255 people with Angelman syndrome, a genetic disorder causing developmental delays and seizures. Participants have already completed an earlier GTX-102 study and will continue receiving the drug. The main g…

This study compares two breathing support strategies—prone positioning and high-frequency ventilation—against standard care in 600 children with severe acute respiratory distress syndrome. The goal is to see which approach helps children spend more days off a ventilator within 28…

This study tests whether combining two drugs, azacitidine and quizartinib, can help people with myelodysplastic syndromes or related blood cancers that have specific genetic changes (FLT3 or CBL mutations). The trial involves 30 adults and aims to find the best dose and see how w…

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 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 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 a 6-month home-based program called FIT Families, designed to help African American teens with obesity and their caregivers lose weight. The program includes coaching sessions, family counseling, and small prizes for completing tasks. Researchers will measure cha…

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

This early-stage trial tests a single oral dose of BMN 349 in 6 adults with a genetic form of alpha-1 antitrypsin deficiency (PiZZ or PiMZ/MASH). The main goal is to check safety and how the drug moves through the body. Participants receive either BMN 349 or a placebo and are clo…

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

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

This study tests a drug called luspatercept in 6 adults with lower-risk myelodysplastic syndromes (MDS) or related blood cancers who have anemia. The goal is to see if the drug can reduce or eliminate the need for regular blood transfusions. Participants receive luspatercept plus…

This phase 3 trial tests imatinib, a targeted cancer drug, with two different chemotherapy backbones in children and adolescents with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The goal is to see if a less intense chemo regimen works as well as the s…

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 study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

This study tests whether giving a personalized dose of a drug called r-ATG before a stem cell transplant helps the immune system recover faster and reduces serious side effects. It includes children and adults with acute leukemia or myelodysplastic syndrome (MDS) receiving a dono…

This early-phase trial is testing whether adding the drug venetoclax to a standard treatment regimen can help adults with a specific type of leukemia (Philadelphia chromosome-positive ALL). The study involves 20 participants and aims to find the safest dose of venetoclax when com…

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 phase 3 study tests the long-term safety of ianalumab in 612 people with Sjögren's syndrome who already completed a year of treatment in earlier trials. Participants receive ianalumab or placebo injections for up to several more years. The goal is to see if the drug remains …

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 Phase 3 study tests whether fenfluramine (ZX008) can safely reduce seizures in 87 children and adults with CDKL5 deficiency disorder, a rare genetic condition causing severe epilepsy. Participants receive either the drug or a placebo, followed by an open-label extension wher…

This early-stage trial tests whether adding the immunotherapy drug ipilimumab to the chemotherapy drug decitabine is safe and tolerable for people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that has come back or stopped responding to treatment. About 54 p…

This phase 3 trial looks at whether people with severe sarcoidosis who are in remission should stop or continue taking infliximab. 32 adults who have been on infliximab for at least 6 months and are stable on low-dose steroids will be randomly assigned to stop or continue the dru…

This study tests a combination of three drugs—ruxolitinib, dexamethasone, and etoposide—for children and young adults with hemophagocytic lymphohistiocytosis (HLH), a rare condition where the immune system attacks the body. The trial includes two groups: newly diagnosed patients …

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

This early-stage trial tests whether combining a precise form of radiation (stereotactic radiosurgery) with two immunotherapy drugs (MBG453 and spartalizumab) is safe for people whose glioblastoma has come back. The study includes 16 adults with recurrent GBM. The main goal is to…

This study tests a small implant placed inside the eye to help drain fluid and lower pressure in people with open-angle or narrow-angle glaucoma. It is for adults whose eye pressure is not well controlled with eye drops. The main goal is to see if the implant safely reduces press…

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 tests an experimental drug, BMS-986278, in about 1057 adults with progressive pulmonary fibrosis, a condition where lung scarring worsens over time. Participants receive either the drug or a placebo to see if it can slow lung function decline and is safe. The trial is …

This study tests a new drug called bexmarilimab, given together with standard treatments, for people with certain blood cancers (MDS, CMML, or AML). The goal is to first find a safe dose and then see if the combination helps shrink or control the cancer. About 181 adults are taki…

This study tests a new medicine called crovalimab for people with a rare disease called atypical hemolytic uremic syndrome (aHUS), which causes blood clots and kidney damage. The trial includes 83 adults and teens who receive the drug to see if it can control the disease and impr…

This phase 3 study is testing the long-term safety and effectiveness of an experimental drug called AOC 1001 (del-desiran) for people with myotonic dystrophy type 1 (DM1), a genetic muscle disorder. The trial enrolls 230 adults who have completed a prior AOC 1001 study and will r…

This phase 3 study tests whether deucravacitinib, a daily pill, can reduce joint pain and swelling in people with active psoriatic arthritis who have never used biologic drugs. About 670 participants will receive either the drug or a placebo for 16 weeks, with a long-term extensi…

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 phase II trial tests whether giving a short course of azacitidine or decitabine before standard chemotherapy can improve outcomes for children with newly diagnosed acute myeloid leukemia (AML). The study enrolls 206 participants and focuses on tolerability, changes in DNA me…

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

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

This study tests a device called Infrasensor that may help doctors quickly identify people with severe heart artery blockages. About 350 adults with chest pain will use the device for 5 minutes, and results will be compared to standard heart catheterization. If accurate, it could…

This study tests an AI tool called AIDP that analyzes MRI scans to help diagnose Parkinson's disease and two similar conditions (MSA and PSP). Researchers at 21 sites will compare the AI's diagnosis to that of movement disorder specialists. The goal is to see if the tool can impr…

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 phase IIb trial tests whether a vaccine called Tri-Ad5, combined with an immune booster N-803, can prevent colon cancer in people with Lynch syndrome. The vaccine targets proteins found in precancer and cancer cells, aiming to train the immune system to destroy them. 186 par…

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 the Shingrix vaccine in over 2,000 adults with autoimmune rheumatic diseases like rheumatoid arthritis and lupus, who are at higher risk for shingles. Researchers will compare vaccinated patients to unvaccinated ones to see if the vaccine is safe and triggers a s…

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

This trial tests a new inflatable cushion called the BathMat, designed to help reposition patients lying on their stomachs (proned) in the ICU. Proning helps patients with severe breathing problems get more oxygen, but repositioning them every few hours currently requires at leas…

This study tests whether a device called RELiZORB™ can help critically ill adults with multiple organ failure better tolerate tube feedings. The device breaks down fats in the feeding to make them easier to digest. Researchers will compare how well patients meet their nutrition g…

This study compares four treatments—clonazepam, capsaicin, photobiomodulation (light therapy), and alpha-lipoic acid—against a placebo for burning mouth syndrome. Researchers will enroll 150 adults with daily oral burning for over 3 months. The goal is to see which approach best …

This study tests whether drinking a ketone supplement (Ketone-IQ) before bed can improve sleep quality and reduce sleep apnea severity in 30 adults who already use a CPAP machine. Participants will try two different doses and compare effects to a placebo. The goal is to find a si…

This study looks at whether auto-captioning glasses are easy and helpful for adults with NF2-related schwannomatosis who have hearing loss. Eighteen participants will use the glasses for 12 weeks and report their experience through surveys and interviews. The goal is to see if th…

This study tests a new artificial tear called ABBV-444 against an existing brand, Refresh Optive, in 250 adults with dry eye disease. Participants use the assigned eye drops for 90 days. The goal is to see if the new formula improves eye comfort and surface health better than the…

This study tests whether a very low dose of naltrexone, a drug usually used for addiction, can reduce pain and other symptoms in people with Complex Regional Pain Syndrome (CRPS). Forty adults with CRPS will receive either the drug or a placebo for six months. The main goal is to…

This study tests an 8-week program called PhYoMind, which blends physiotherapy, yoga postures, breathing exercises, and mindfulness, against home exercise alone in 40 adults with fibromyalgia. The goal is to see if the combined approach reduces disability, pain, fatigue, and stre…

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 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 study tests a medicine called tenapanor in children aged 6 to 12 who have irritable bowel syndrome with constipation (IBS-C). The goal is to see if it safely improves bowel movements and reduces belly discomfort. About 72 children will receive either the drug or a placebo fo…

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

This study is testing the long-term safety of tenapanor (Ibsrela) in children aged 6 to 17 with irritable bowel syndrome with constipation (IBS-C). About 150 kids who completed a prior study will continue taking the drug for up to one year. Researchers will monitor side effects a…

This phase 3 trial tests tenapanor, a drug that helps with constipation and belly pain, in 180 teenagers aged 12 to 17 with irritable bowel syndrome and constipation (IBS-C). Participants take either a low or high dose of tenapanor or a placebo twice daily for 12 weeks. The goal …

This study tests whether a virtual program called Lin Health can improve daily life for people with fibromyalgia. Researchers at Mayo Clinic will follow 300 adults with fibromyalgia to see if online behavioral health support reduces symptoms and improves function. The goal is to …

This study tests whether a gentle nerve-stimulating earpiece can help newborns going through opioid withdrawal. The device is used up to four times a day for 20 days, just before each morphine dose. Researchers want to see if it reduces the number of days babies need morphine and…

This study tests a culturally tailored counseling and support program for Chinese and Korean American caregivers of relatives with dementia. The program includes family counseling sessions and online peer support groups. Researchers will measure whether it reduces stress, depress…

This study explores whether individualized transcranial magnetic stimulation (TMS) can improve symptoms in adults with irritable bowel syndrome (IBS). Participants receive 10 TMS sessions over two weeks, with brain scans and stool tests before and after to understand how the trea…

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

This study 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 aimed to see if a one-hour session of inhaled nitrous oxide (laughing gas) could reduce pain in people with fibromyalgia. It was designed for adults aged 18-75 with moderate to severe pain. However, the study was withdrawn before any participants were enrolled, so no r…

This study tests whether two 90-minute pain science education sessions can help 54 adults with fibromyalgia living in rural Spain. The sessions aim to change how patients think about pain, using simple language and metaphors. Researchers will measure pain sensitivity, daily funct…

This Phase 2A study tests three drugs—sivopixant, acetazolamide, and SASS-001—in 60 adults with sleep apnea that has a central component (where the brain fails to signal breathing). The goal is to see if these drugs can reduce the number of breathing pauses during sleep. Particip…

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 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 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 tests whether a numbing injection near the glossopharyngeal nerve, combined with topical anesthesia, can help people recover faster after surgery for obstructive sleep apnea. The trial involves 80 adults with moderate to severe sleep apnea who are scheduled for palatop…

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 a 12-week tele-rehabilitation program using mindful and conscious movement (like adapted yoga and breathing exercises) to conventional low-intensity exercise and usual care for people with chronic fatigue syndrome or post-COVID syndrome. The goal is to see if …

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

This study planned to compare a mindfulness-based eating awareness program with the standard low-FODMAP diet for people with irritable bowel syndrome (IBS). The goal was to see which approach better improves IBS symptoms. However, the study was withdrawn before enrolling any part…

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 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 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 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 study looks at whether teenagers and young adults with Polycystic Ovary Syndrome (PCOS) have more fat in their liver than those without PCOS. Researchers will use advanced imaging and tests to measure liver fat, insulin resistance, and cholesterol problems in 80 participants…

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 observes 80 children with Marfan syndrome to understand how the condition impacts their quality of life, self-image, and ability to manage their health. Participants fill out questionnaires about their well-being and medication habits. The goal is to identify who might…

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 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 study follows 125 people with Parkinson's disease or related conditions over two years. Researchers use special brain scans to measure changes in a chemical called acetylcholine, which is linked to walking problems, falls, and thinking skills. The goal is to understand how t…

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

This study 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 follows 154 people taking Epidiolex (cannabidiol) for seizures caused by Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. Researchers will monitor for signs of chronic liver injury and fibrosis over time using blood tests and liver scans. The go…

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

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

This study 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 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 collects blood and bone marrow samples from up to 550 people with blood cancers like leukemia, lymphoma, and myelodysplastic syndrome. Researchers will study the cells, genes, and proteins in these samples to better understand how these cancers develop and to find new …

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

This study examines how common obstructive sleep apnea (OSA) is in adult survivors of Hodgkin lymphoma who received chest radiation. Researchers will compare 220 survivors to matched controls using sleep tests, brain scans, and heart assessments. The goal is to understand links b…

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 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 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 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 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 tests a new way to find unstable plaques in the heart arteries of people who come to the emergency room with chest pain but are not having a heart attack. Researchers use a simple calcium scan to group patients by risk, then follow up with special PET scans to see if p…

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 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 at Duke University tests whether a special MRI scan using inhaled xenon gas can detect changes in lung function over time in people with progressive pulmonary fibrosis. Sixty adults with various types of lung scarring will have MRI scans before starting anti-fibrotic m…

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 Johns Hopkins study looks at whether a leaky blood-brain barrier and immune changes contribute to cognitive problems in people with ME/CFS (chronic fatigue syndrome), both those who got sick before the pandemic and those who developed it after COVID-19. Researchers will use …

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 follows over 1,300 people with psoriatic arthritis who are starting either guselkumab (Tremfya) or an IL-17 inhibitor as part of their routine care. Researchers will track how long patients continue each treatment and monitor joint swelling and tenderness. The goal is …

This study looks at how the liver burns fat and makes sugar in people with NASH (a serious liver disease linked to obesity) who are planning to have weight-loss surgery. Researchers will use special scans to measure liver function before and after surgery in 30 adults. The goal i…

This study is a large registry that follows nearly 16,000 people with psoriasis to monitor the long-term safety of several treatments, including biologic drugs like infliximab and ustekinumab. Participants receive their usual care while researchers track side effects, disease act…

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

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

This study looks at why people with HIV and certain cancers caused by KSHV (Kaposi sarcoma herpesvirus) may lose bone density faster. Researchers will review past CT scans and health records from 40 adults treated at the NIH between 2005 and 2020. The goal is to find factors link…

This study follows 23 people with tumor-induced osteomalacia (TIO), a rare condition where tumors cause weak bones. The goal is to see how well the drug burosumab works over time and to monitor any tumor changes. Participants continue their usual care, and no new treatments are g…

This study follows 1000 Chinese patients with myotonic dystrophy type 1 (DM1) to understand how the disease affects multiple body systems and leads to disability. Researchers will collect health data and blood samples every 3 to 6 months for several years. The goal is to identify…

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 looks at skin cells (fibroblasts) from people with Marfan syndrome and other genetic conditions that cause thoracic aortic aneurysms (a bulging of the main artery in the chest). Researchers want to find out how these cells behave differently and what genes or proteins …

This study is testing a digital health tool to help more people of Ashkenazi Jewish descent get tested for BRCA gene mutations, which increase cancer risk. Over 5,400 participants will use a web portal to learn about testing and decide whether to proceed. The goal is to see if th…

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

This study looks at how stress affects the brain and body in 70 teenagers (ages 11-17) with chronic widespread pain. Participants will have an MRI scan while experiencing mild heat pain, and provide saliva samples to measure stress hormones. The goal is to understand the link bet…

This study tests whether showing parents an infographic about obstructive sleep apnea (OSA) symptoms helps more children get diagnosed. About 200 parents of children with OSA symptoms will either see the infographic or receive usual care. Researchers will compare how many childre…

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

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

This study looks at over 110,000 people who had hip replacement surgery to see if a longer or shorter hip stem works better. It compares death rates, how often the hip implant needs to be replaced, and patient-reported pain and function. The goal is to find out which stem length …

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

This study 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 tracks 378 people with moderate to severe dry eye disease and healthy volunteers to see how signs, symptoms, and biomarkers change after treatment with either Vevye or Xiidra eye drops. The goal is to find better ways to measure the disease and its response to therapy.…

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 study tests whether a drug called racecadotril can reduce fluid leakage in skin blisters, as a model for what happens in the lungs during Acute Respiratory Distress Syndrome (ARDS). ARDS is a severe lung injury with no effective drug treatments, where patients' lungs fill wi…

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 study observes 367 Chinese patients with atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots and kidney damage. Researchers will track how patients are treated in routine care and monitor their health for up to 12-24 months. The goal is to bet…

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 looks at how sleep apnea might cause prediabetes to turn into diabetes. Researchers will use CPAP machines and niacin infusions in 300 overweight, prediabetic adults with sleep apnea to measure changes in blood chemicals. The goal is to understand the biological steps …

This small study tests whether the supplement N-acetylcysteine (NAC) can reduce oxidative stress and inflammation in people with obstructive sleep apnea who are already using standard PAP therapy. Researchers want to see if the effects differ between men and women. The study invo…

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 creates a registry for up to 500 people with CDKL5 Deficiency Disorder (CDD), a rare genetic condition that causes seizures and developmental delays. Patients and their caregivers will provide information about symptoms, treatments, and quality of life over several yea…

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…