Syndromic disease

This early-phase trial tests a personalized treatment for people with blood cancers like leukemia. Doctors take a patient's own T cells, modify them in the lab to recognize unique cancer mutations, and infuse them back after a short course of chemotherapy. The goal is to see if t…

This study tests whether clemizole HCl (EPX-100) can safely reduce seizures when added to current treatments for Lennox-Gastaut syndrome, a severe form of epilepsy. About 260 children and adults aged 2 to 55 will receive either the drug or a placebo. The main goal is to measure c…

This study tests a new drug called LMP744 in people whose glioblastoma has come back after standard treatment. The drug is designed to kill cancer cells by blocking a key growth signal and interfering with DNA repair. Participants will receive the drug through a vein for 5 days e…

This study tests a stem cell transplant using blood stem cells from a half-matched family donor (haploidentical) for people with severe aplastic anemia, related MDS, or PNH. Participants receive chemotherapy and a single radiation dose before the transplant, plus a drug to preven…

This study tests whether a new cord blood stem cell product, Omidubicel, can improve transplant outcomes for people with severe aplastic anemia or a related bone marrow disorder. Participants receive a combination of stem cells from a family member and specially processed cord bl…

This Phase 3 trial compares a low-dose combination of prednisone and methotrexate to standard high-dose prednisone for people with active cardiac sarcoidosis. Researchers hope the combo works just as well but causes fewer side effects and improves quality of life. The study plans…

This study tests a new way to do stem cell transplants for people with severe aplastic anemia or other bone marrow failure diseases. Doctors give a smaller number of donor white blood cells along with the stem cells to try to reduce serious side effects. Up to 120 patients aged 4…

This study tests an oral drug called pacritinib in people aged 12 and older with myelodysplastic syndromes (MDS) or related blood cancers (MDS/MPN). The goal is to find the best dose and see if it can shrink or control the disease. Participants take capsules twice daily for up to…

This study tests whether combining two immune-suppressing drugs, rituximab and cyclosporine, can safely reduce protein leakage in the urine of people with membranous nephropathy, a kidney disease where the immune system attacks the kidneys. About 30 adults with persistent high pr…

This study tests the drug abemaciclib, typically used for cancer, in people with neurofibromatosis type 1 (NF1) who have atypical neurofibromas that cannot be surgically removed. The goal is to find a safe dose and see if it can shrink or stabilize these tumors. Participants take…

This study tests an experimental drug called BAY 3401016 in 60 adults aged 18 to 45 with Alport syndrome, a rare genetic condition that damages kidneys, hearing, and eyes. The drug works by blocking a protein thought to cause kidney injury, with the goal of slowing the loss of ki…

This study follows 30 people with Alagille syndrome who are taking odevixibat (Bylvay) in their daily lives. The goal is to see if the drug helps them avoid serious surgeries like liver transplant or bile duct repair over the long term. Researchers will also track weight, height,…

This registry will follow 20 people in China with progressive familial intrahepatic cholestasis (PFIC) who are taking odevixibat (Bylvay) as part of their normal care. The goal is to see how safe the drug is over the long term and how well it controls symptoms like severe itching…

This early-stage trial tests imatinib, a drug already used for certain cancers, in adults with a harmful RUNX1 gene mutation. The mutation causes easy bleeding and a high risk of blood cancers. The study aims to find the best dose and see if imatinib can improve platelet function…

This study tests a new biologic drug called ianalumab combined with a standard platelet-boosting drug (TPO-RA) in people with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. The trial includes 164 adults who have tried at l…

This registry will follow 10 people with progressive familial intrahepatic cholestasis (PFIC) in South Korea who are taking odevixibat (Bylvay). The goal is to see how safe and effective the drug is over the long term in real-world use. Researchers will track side effects and how…

This study tests a temporary device that stimulates the diaphragm (the main breathing muscle) in ICU patients on breathing machines. The goal is to see if it helps patients breathe more on their own and recover from lung injury. About 30 adults with severe breathing failure will …

This study looks at how well and how safely the drug Ilaris (canakinumab) works for people with Schnitzler's syndrome, a rare condition that causes hives, fever, joint pain, and fatigue. About 5 participants will receive the drug and be monitored for up to 48 weeks. The goal is t…

This early-phase trial is testing whether adding the drug emapalumab to standard medications can safely prevent graft-versus-host disease (GVHD) in 15 adults with leukemia or myelodysplastic syndrome receiving a reduced-intensity stem cell transplant. Emapalumab blocks a protein …

This trial tests a real-time algorithm-driven system that gives doctors feedback on ventilator settings to protect the lungs of critically ill patients. The study will include 208 adults on breathing machines with acute respiratory distress syndrome (ARDS). The goal is to see if …

This phase 2 trial tests a powerful mix of short-course radiation, chemotherapy (mFOLFOX6), an immunotherapy (PD-1 antibody), and a targeted drug (cetuximab or bevacizumab, depending on genetic markers) in people with high-risk rectal cancer that is mismatch repair proficient (pM…

This study follows about 665 people with myelodysplastic syndromes, beta-thalassemia, or myelofibrosis who have already taken luspatercept in earlier trials. Researchers want to see how safe the drug is over many years by tracking side effects, disease progression, and survival. …

This study investigates whether two medications—acetazolamide and eszopiclone—can improve sleep apnea outcomes when taken after soft palate surgery. The trial includes adults with moderate to severe obstructive sleep apnea who cannot use CPAP or oral appliances. Researchers will …

This early-stage trial is testing a new combination of chemotherapy drugs (thiotepa, busulfan, and fludarabine) followed by a stem cell transplant for people with certain blood cancers like leukemia and myelodysplastic syndrome. The goal is to see if this approach can reduce the …

This Phase 3 trial tests whether adding immunotherapy and a targeted drug to short-course radiotherapy and chemotherapy improves outcomes for people with high-risk rectal cancer. About 204 participants will receive either the standard treatment or the intensified combo. The study…

This study tests a new way to prepare donor stem cells (called Orca-Q) for people with blood cancers like leukemia or MDS who need a transplant. The goal is to see if this engineered graft is safe and helps the body accept the new cells with fewer complications. About 300 partici…

This Phase 3 trial tests whether DYNE-101 can improve muscle function and daily life in 150 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo by IV every few weeks for 48 weeks. The study measures how quickly people can stand from a ch…

This study tests a drug called risankizumab in children with juvenile psoriatic arthritis, a condition causing joint pain and swelling. About 40 kids will receive either risankizumab or the standard drug adalimumab for 24 weeks, with responders continuing for up to 2 years. The g…

This study tests whether a stem cell transplant from a healthy donor can help people with severe primary immunodeficiencies. Participants aged 4 to 75 receive chemotherapy before the transplant to prepare their body. The goal is to see if the procedure is safe and can improve imm…

This early-stage trial tests whether an experimental drug called UDP-003 is safe in healthy volunteers and people with heart disease. Researchers will give single or multiple doses of the drug or a placebo and monitor for side effects. The study includes 84 participants and is th…

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

This pilot study tests whether the drug colchicine can reduce artery plaque in 120 heart attack survivors who have a specific genetic change (TET2-CHIP). Participants take colchicine or standard care for 12 months. The goal is to see if this genetic marker can guide personalized …

This Phase 3 trial tests whether adding the drug venetoclax to standard chemotherapy helps adults with newly diagnosed acute myeloid leukemia (AML) or a related high-risk condition. About 650 participants aged 18 to 75 will receive either venetoclax or a placebo alongside chemo. …

This study looks at a newly recognized disease called KSHV Inflammatory Cytokine Syndrome (KICS), caused by a virus that can also lead to certain cancers. Researchers aim to learn more about the disease and test experimental treatments, including antiviral and chemotherapy drugs,…

This Phase 3 trial tests the drug benralizumab (Fasenra) in children aged 6 to 17 with two rare eosinophilic diseases: EGPA and HES. The study aims to see if the drug is safe, how it works in the body, and whether it can help control the disease. Fourteen children will receive in…

This early-phase trial is testing a new drug called MTS109, made with mRNA technology, for people with moderate to severe autoimmune diseases like lupus and scleroderma. Fifteen participants will receive several injections over a month to see if it is safe and tolerable. The goal…

This trial tests whether sirolimus (rapamycin), an immune-suppressing drug, can safely raise platelet counts in people with antiphospholipid antibodies and persistent low platelets. Participants are randomly assigned to receive sirolimus or a placebo for six months, and those who…

This study follows 300 adults with Parkinson's disease, essential tremor, or dystonia who receive deep brain stimulation (DBS). DBS uses a device implanted in the chest to send electrical pulses to brain areas controlling movement. Researchers will track symptom severity, medicat…

This study tests whether a digital support program (called DEEPER) helps heart patients maintain their health after completing cardiac rehab. About 306 adults with heart disease or heart failure will be split into two groups: one gets standard follow-up care, the other gets stand…

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

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

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

This study tests an oral drug called KT-621 in 264 adults with moderate to severe eosinophilic asthma that is not well controlled by current treatments. Participants will take either KT-621 or a placebo daily for several weeks. Researchers will measure changes in lung function an…

This phase 2 trial tests whether adding vedolizumab to standard drugs (cyclophosphamide and tacrolimus) can prevent graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers like leukemia and myelodysplastic syndrome. GVHD occurs when donor immu…

This study tests whether a personalized breathing machine strategy can improve survival in people with acute respiratory distress syndrome (ARDS), a severe lung condition. About 1,100 adults on ventilators will be randomly assigned to either standard care or a precision approach …

This early-phase trial tests a new treatment called CD64 CAR T cells for adults with acute myeloid leukemia (AML) that has come back or not responded to standard therapy. The treatment uses a patient's own immune cells, modified in a lab to target and attack leukemia cells. The m…

This early-stage trial compares a new investigational drug, CG-0255, to the established blood thinner Plavix in 136 healthy adults aged 18-55. The goal is to see if CG-0255 is processed by the body similarly to Plavix and has the same effect on preventing blood clots. Participant…

This Phase 3 study tests whether the oral drug zasocitinib (TAK-279) can improve symptoms of active psoriatic arthritis in people who have never used biologic treatments. About 1,088 adults will receive either zasocitinib, an active comparator, or a placebo for up to 60 weeks. Th…

This study tests a new medicine called tulisokibart for people with active psoriatic arthritis, a condition causing joint pain and skin plaques. Researchers will compare different doses of the drug to a placebo in 140 adults to see if it improves symptoms like joint tenderness an…

This early-phase study tests the drug lorlatinib in children and young adults (ages 1 to 21) with newly diagnosed high-grade glioma, a fast-growing brain tumor, that has a specific genetic change (ALK or ROS1 fusion). The goal is to see if the drug can shrink or control the tumor…

This study looks at how well setmelanotide works in real-world settings for people with Bardet-Biedl syndrome, a rare genetic condition that causes severe obesity and constant hunger. Researchers will track changes in body mass index, cholesterol, liver fat, and quality of life i…

This study tests a once-weekly injection called NNC0487-0111 in 300 adults with overweight or obesity and moderate-to-severe obstructive sleep apnea who use a breathing device. The goal is to see if the drug helps with weight loss and reduces breathing pauses during sleep. Partic…

This Phase 3 trial tests whether the experimental drug zasocitinib (TAK-279) can reduce joint swelling and pain in people with active psoriatic arthritis. About 600 adults will receive either the drug or a placebo for up to 60 weeks. The main goal is to see if more people on the …

This Phase 2 study tests atacicept, a drug that may reduce immune system attacks on the kidneys. About 250 adults with various autoimmune kidney diseases (like IgA nephropathy or membranous nephropathy) will receive weekly injections. Researchers will check safety and measure cha…

This study tests a drug called deucravacitinib in children aged 5 to 17 with active juvenile psoriatic arthritis, a condition causing joint pain and swelling. The trial aims to see if the drug can control the disease and prevent flare-ups. About 60 participants will receive eithe…

This study tests whether tofacitinib, a drug already used for rheumatoid arthritis, is safe and helpful for people with Sjogren's disease. About 60 adults with mild-to-moderate Sjogren's will take the pill twice daily for 28 weeks. Researchers will track side effects and measure …

This study compares two stomach-acid-lowering drugs—tegoprazan and rabeprazole—in about 3,300 heart disease patients who take blood thinners and are at high risk for stomach bleeding. The goal is to see if tegoprazan works as well as rabeprazole at preventing serious gut problems…

This study is testing whether stem cells taken from a person's own bone marrow and then given back through the veins and nose can improve brain and nerve function. It includes 500 people with various conditions like stroke, Parkinson's, Alzheimer's, and nerve damage. The goal is …

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

This Phase 3 trial is testing whether a combination of two drugs, balcinrenone and dapagliflozin, works better than dapagliflozin alone to prevent heart failure events and cardiovascular death. About 4,800 adults with heart failure and moderate-to-severe kidney impairment who rec…

This phase II trial tests whether adding targeted total marrow irradiation to standard chemotherapy before a stem cell transplant can improve outcomes for people with high-risk acute myeloid leukemia, chronic myeloid leukemia, or myelodysplastic syndromes. The study will enroll 3…

This study tests a once-weekly injection called NNC0487-0111 in 300 adults with overweight or obesity and moderate-to-severe obstructive sleep apnea who do not use a CPAP machine. The goal is to see if the drug helps with weight loss and reduces breathing pauses during sleep. Par…

This study tests two ways to treat a slow heart rate caused by overactive nerves, without using a permanent pacemaker. One method uses freezing (cryoablation) and the other uses burning (radiofrequency) to calm the nerves. Sixty adults aged 18 to 60 with symptoms like fainting or…

This study tests whether a single dose of an experimental immune cell therapy called agenT-797, given alongside standard care, can lower the risk of death in adults with severe pneumonia and breathing failure. About 90 people in intensive care will receive either agenT-797 or a p…

This early-phase trial is testing a new drug called HC-7366 in 18 adults with acute myeloid leukemia (AML) that has come back or not responded to other treatments. The main goal is to find a safe dose and check for side effects. Researchers hope this drug may offer a new option f…

This study is for adults with recurrent glioblastoma, an aggressive brain cancer. It tests whether adding a precise type of radiation (stereotactic radiosurgery guided by a special PET scan) to a device that delivers electric fields to the brain (TTFields) helps people live longe…

This study tests whether a single dose of tocilizumab, an anti-inflammatory drug, can help children and adults with sickle cell disease recover faster from acute chest syndrome—a serious lung condition. Participants receive either the drug or a placebo, and researchers measure ho…

This Phase 2 trial is testing a stem cell transplant from a donor for people with various blood cancers and bone marrow disorders. The transplant uses strong chemotherapy or radiation to wipe out the diseased marrow, then replaces it with healthy donor cells. A new combination of…

This study tests whether Miebo eye drops, used for one month before and 1.5 months after LASIK surgery, can reduce dry eye symptoms in people who already have dry eye disease. One hundred adults will use the drops four times daily. The goal is to see if eye dryness after surgery …

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

This study compares two medicated stents (tiny mesh tubes) used to open blocked heart arteries in people having a heart attack or unstable angina. About 2,100 participants will receive either a sirolimus-eluting stent (ihtDEStiny) or an everolimus-eluting stent (Xience). Research…

This Phase 2 study tests a new drug called ADX-038, alone or with another drug (telitacicept), in 30 Chinese adults with kidney diseases caused by an overactive complement system (IgA nephropathy, C3G, or IC-MPGN). The goal is to see if the treatment is safe and can help control …

This study tests a new drug called GEn-1124 in 52 adults with acute respiratory distress syndrome (ARDS), a severe lung condition. The drug is given as an IV infusion twice daily for 5 days, starting as soon as possible after diagnosis. The main goal is to check if the drug is sa…

This study tests whether giving caffeine citrate to moderately preterm infants for 28 days after hospital discharge can prevent apnea (breathing pauses) that lead to sick visits. About 340 babies in Zambia will receive either caffeine or a placebo daily. Researchers will also che…

This study tests whether adding sarilumab (a drug that calms inflammation) to a slow steroid taper can help people with early polymyalgia rheumatica achieve lasting remission. About 300 adults aged 50 and older will receive either sarilumab or a placebo, along with a 52-week pred…

This phase 3 trial tests a drug called plonmarlimab in 30 adults with a severe immune overreaction (macrophage activation syndrome) linked to rheumatic diseases that hasn't responded to prior treatment. Participants receive weekly IV infusions for 8 weeks. The main goal is to see…

This study tests an experimental tablet, BI 764198, in adults and some teens with four types of kidney disease that cause protein in the urine. Participants take the drug or a placebo daily for 20 weeks while continuing their usual care. The main goal is to see if the drug lowers…

This early-phase trial is testing whether combining two oral drugs, eltanexor and venetoclax, is safe and effective for people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that has returned or not responded to prior treatment. The study will enroll 60 adult…

This phase 3 trial compares a once-weekly growth hormone injection (lonapegsomatropin) to a daily one (somatropin) in 186 prepubertal children with growth failure due to Turner syndrome, SHOX deficiency, being small for gestational age, or idiopathic short stature. The goal is to…

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

This early-phase trial tests a cell therapy called Orca-T combined with standard drugs to prevent graft-versus-host disease (GVHD) in 24 adults with advanced blood cancers (like leukemia or MDS) receiving stem cell transplants from partially matched donors. The main goal is to se…

This study tests whether spesolimab, given as an infusion, can help close skin ulcers in adults with pyoderma gangrenosum, a rare inflammatory skin condition. About 90 participants will receive either spesolimab or a placebo, along with corticosteroids. The trial lasts about 1.5 …

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

This study tests a new drug called CCS1477 (inobrodib) in people with advanced blood cancers like leukemia, lymphoma, and myeloma that have not responded to standard treatments. The drug is given as an oral capsule, either alone or combined with other cancer medicines. The goal i…

This study tests whether a stem cell transplant from a healthy donor can treat GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of leukemia. Participants aged 6 to 70 with GATA2 mutations will receive chemotherapy or radiation before t…

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

This phase 2 trial is testing a new treatment called Orca-T for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Orca-T is a personalized cell therapy made from a donor's blood that includes stem cells and immune cells. The goal is to see if it can impr…

This early-stage study tests a new drug (GSK5926371) in people with lupus and similar autoimmune diseases. The drug is designed to target and calm specific immune cells that attack the body. The main goal is to check if the drug is safe and how the body handles it, with 54 adults…

This study tests doing two heart procedures at the same time for people with atrial fibrillation. First, doctors use pulsed field energy to fix the heart's rhythm. Then, they implant a small device to close off a part of the heart where clots can form. The goal is to see if this …

This study tests whether starting inclisiran early during a hospital stay for a heart attack can lower bad cholesterol more than a placebo, when both are added to standard care. About 300 adults hospitalized for a heart attack will receive either inclisiran or a placebo shot at t…

This study follows 300 adults with severe eosinophilic asthma who are prescribed benralizumab as part of their normal care in Germany. Researchers will track changes in asthma control, lung function, and medication use over one year. The goal is to see how the drug performs outsi…

This Phase 3 study tests an experimental drug called ION582 in 158 children and adults with Angelman syndrome, a genetic disorder causing severe developmental delays. The drug is given as a spinal injection and aims to improve expressive communication and cognitive skills. Partic…

This Phase 2 trial tests whether adding belimumab to standard rituximab treatment helps more people with primary membranous nephropathy achieve remission. The study enrolls 58 adults with confirmed disease. Belimumab targets new immune cells, while rituximab depletes existing one…

This phase 3 trial tests a weekly injection called eloralintide in 800 adults with moderate-to-severe obstructive sleep apnea who are also overweight or have obesity. The study looks at whether the drug can improve sleep apnea symptoms and help with weight loss. Participants are …

This study tests whether a weekly under-the-skin antibody treatment (XEMBIFY) provides similar blood levels as a standard IV infusion (Gamunex-C) given every 3 weeks in people with CIDP, a chronic nerve disorder. About 40 adults who are stable on IVIG will first receive Gamunex-C…

This study tests an experimental drug called NIO752 in 300 people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement and balance. Participants are randomly assigned to receive either the drug or a placebo. After the main study, everyone can rec…

This study tests a new medicine called MAS825 for people with Still's disease, a rare inflammatory condition causing fever, rash, and joint pain. About 20 children and adults with active disease will receive the drug to see if it safely controls symptoms. The goal is to improve d…

This study tests an experimental drug called GTX-102 (apazunersen) in 60 people with Angelman syndrome, a genetic disorder causing developmental delays and seizures. Participants range from 1 to 65 years old and are grouped by age and genetic type. The goal is to see if the drug …

This early study tests a device called TheraSphere GBM in 36 people whose glioblastoma has come back after prior treatment. The device delivers tiny radioactive beads directly to the tumor to try to control it. The main goal is to check if the procedure is safe, not yet whether i…

This study tests a radioactive antibody (211At-BC8-B10) that seeks out and attacks cancer cells, followed by a donor stem cell transplant for people with high-risk leukemia or myelodysplastic syndrome (MDS) that has returned or not responded to treatment. The goal is to find the …

This study tests a new experimental drug called EP0042 in adults with advanced acute myeloid leukemia (AML) or related blood cancers. The goal is to find a safe dose that might work against the cancer, either alone or with other treatments. About 70 participants will take part to…

This study compares two ways of giving the drug bimekizumab to people with active psoriatic arthritis or axial spondyloarthritis: as an IV infusion or as a shot under the skin. The goal is to see if the IV method works just as well as the shot. About 392 adults who are suitable f…

This study tests whether the drug THRV-1268 can safely shorten the heart's electrical recovery time (QT interval) in people with Long QT Syndrome Type 2, a genetic condition that raises the risk of dangerous heart rhythms and sudden cardiac arrest. About 64 participants will take…

This Phase 2 trial tests whether adding the experimental drug silevertinib to standard chemotherapy (temozolomide) helps people with a specific type of newly diagnosed glioblastoma live longer without their cancer worsening. The study includes 162 participants whose tumors have c…

This phase 3 trial tests whether the drug dalcetrapib can reduce the risk of major heart problems like heart attacks in people who have a specific genetic makeup (AA genotype) and have recently had a heart-related event. About 2,000 participants will receive either dalcetrapib or…

This Phase 2 trial tests a combination of horse anti-thymocyte globulin, cyclosporine, steroids, and growth factors in 140 adults with aplastic anemia or low-risk myelodysplastic syndrome (MDS). The goal is to see if this immune-suppressing and blood-cell-boosting approach can im…

This phase 2 trial tests whether ruxolitinib, a drug already approved for certain blood disorders, can help regrow hair in people with APECED, a rare immune condition that often causes severe alopecia areata. About 70 participants aged 12 to 75 will take the pill twice daily for …

This study tests a drug called bimekizumab in children aged 2 to 17 with specific types of juvenile arthritis (enthesitis-related arthritis and juvenile psoriatic arthritis). The goal is to see how the drug moves through the body and if it is safe. About 40 children will take par…

This phase 3 trial tests whether the drug bimekizumab can clear or nearly clear the pustules and skin lesions of palmoplantar pustulosis (PPP) on the hands and feet. About 320 adults with moderate to severe PPP will receive either bimekizumab or a placebo for 16 weeks, with an op…

This study tests whether a 20-day fasting schedule before hip replacement surgery can reduce complications like infections. 130 adults will either follow the fasting plan or receive standard care. Researchers will measure changes in gut bacteria and immune markers to see if fasti…

This study tests the drug lenalidomide in 42 adults with Behçet's syndrome who have stubborn mouth ulcers. Participants take 10 mg of lenalidomide daily for 12 weeks, then stop for 4 weeks of observation. The main goal is to see if the ulcers completely disappear by week 12.

This study tests whether adding enasidenib to standard treatment (ASTX727) improves outcomes for people with a high-risk form of myelodysplastic syndrome (MDS) that has a specific gene mutation (IDH2). About 54 adults who have not had prior cancer therapy will be randomly assigne…

This study is testing a device called the Genio® System for people with moderate to severe obstructive sleep apnea who cannot use CPAP machines. The device is implanted and stimulates the tongue to keep the airway open during sleep. Researchers will track how well it reduces brea…

This study tests a new drug called KPL-387 in 80 people with well-controlled recurrent pericarditis (inflammation around the heart). The goal is to see if patients can safely switch from their current treatments to KPL-387 alone and still prevent flare-ups. The trial will measure…

This study is testing a new approach for people with high-risk acute leukemias or myelodysplastic syndrome. Before a donor stem cell transplant, patients receive a radioactive antibody (211At-BC8-B10) designed to target and kill cancer cells while sparing healthy tissue. The goal…

This study tests an experimental drug called ultevursen for people with retinitis pigmentosa caused by a specific gene mutation (USH2A). The drug is injected into the eye and may help slow vision loss. The trial involves 81 participants, some of whom will receive a sham (fake) in…

This study tests whether adding AGuIX gadolinium-based nanoparticles to standard brain radiation can better control brain metastases that are at high risk of coming back. About 134 adults with certain cancers (like melanoma, lung, breast, or colorectal) that have spread to the br…

This early-stage trial tests a special type of immune cell (CIML NK cells) combined with a low dose of IL-2 in people whose blood cancer (like AML or MDS) came back after a stem cell transplant. The goal is to find a safe dose and see if it can put the cancer back into remission.…

This early-phase trial tests whether specially trained natural killer (NK) cells, called CIML NK cells, along with interleukin-2 (IL-2), can safely prevent leukemia from coming back after a stem cell transplant. The study enrolls 15 adults with high-risk acute myeloid leukemia, m…

This phase 2 trial tests a combination of three drugs—carfilzomib, cyclophosphamide, and dexamethasone—in 20 people newly diagnosed with POEMS syndrome, a rare disorder affecting nerves and blood. The goal is to see if the regimen can reduce abnormal proteins and improve symptoms…

This study tests whether custom 3D-printed titanium implants can safely and effectively rebuild facial bones in 30 adults with defects from injury, cancer surgery, or birth conditions. Each implant is designed from CT scans to match the patient's unique anatomy. Researchers will …

This study tests a new device called Freesolve, a temporary magnesium scaffold that props open blocked heart arteries and then dissolves over time. About 1,859 people with up to two new blockages will receive either this scaffold or a standard permanent stent. The goal is to see …

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

This early-phase study tests a new drug called SL-401 combined with two other drugs (azacitidine and venetoclax) in people with certain blood cancers—acute myeloid leukemia, myelodysplastic syndrome, and blastic plasmacytoid dendritic cell neoplasm. The main goal is to find the s…

This study looks at whether people with a type of throat cancer linked to the Epstein-Barr virus can safely receive lower doses of radiation after initial chemotherapy. The goal is to see if reducing radiation can still control the cancer while causing fewer long-term side effect…

This study tests a new approach for people newly diagnosed with a specific, aggressive leukemia (Ph+ ALL). It combines a single infusion of specially engineered immune cells (CAR-T cells) with a daily targeted pill (olverembatinib). The goal is to see if this powerful one-two pun…

This study compares two devices used during cataract surgery to treat open-angle glaucoma. About 194 adults with mild to moderate glaucoma and cataracts will receive either the ELIOS device or a competitor device. The main goal is to see which one lowers eye pressure more effecti…

This study tests whether a non-invasive device that gently stimulates a nerve in the ear can help people with psoriatic arthritis. The device, called taVNS, is used for 20 minutes daily over 10 days. Researchers will measure changes in disease activity, pain, quality of life, and…

This early-phase trial is testing the drug asciminib as a maintenance treatment for adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) who have already received a stem cell transplant or CAR T-cell therapy. The goal is to see if asciminib is safe …

This study is testing a device called the Indigo Aspiration System to remove blood clots from leg arteries in people with acute limb ischemia, a condition where a clot suddenly blocks blood flow. About 300 participants will undergo a procedure where the device sucks out the clot.…

This early-stage trial is testing whether combining two drugs—niraparib and irinotecan—can safely treat advanced solid tumors that have mutations in DNA repair genes like BRCA. The study will enroll 24 adults with various cancers, including breast, ovarian, pancreatic, and others…

This study is for people who have both an autoimmune disease (like rheumatoid arthritis, lupus, or multiple sclerosis) and an advanced cancer that has spread or cannot be removed by surgery. It tests the safety and effectiveness of the immunotherapy drug nivolumab, given alone or…

This early-phase study is testing an experimental oral drug called AZD3632 in about 84 people with advanced blood cancers (acute leukemias or myelodysplastic syndromes) that have certain genetic changes. The main goals are to check the drug's safety and find the right dose, while…

This phase 2 trial is testing whether a combination of three drugs—bevacizumab, erlotinib, and atezolizumab—can safely shrink or stabilize advanced kidney cancer in people with hereditary leiomyomatosis and renal cell cancer (HLRCC) or sporadic papillary renal cell cancer. The dr…

This phase 2 trial tests clofutriben, a drug that lowers cortisol, in 1500 adults with hard-to-control type 2 diabetes and signs of high cortisol. Participants receive either clofutriben or a placebo daily for 24 weeks. The study measures changes in blood sugar and cortisol level…

This study tests an experimental oral drug, GSK5460025, alone or with other cancer drugs, in adults whose solid tumors have specific genetic changes (dMMR or MSI-H). The goal is to see if the drug can shrink tumors and to check its safety. About 47 people with advanced cancers th…

This study looks at whether having regular motivational conversations after leaving the hospital for alcohol withdrawal can help people stay sober longer. About 104 adults who want to quit drinking completely will be split into two groups: one gets standard care, the other also g…

This study tests the long-term safety and effectiveness of an oral drug called zasocitinib (TAK-279) in adults with active psoriatic arthritis, a condition causing joint pain and skin patches. About 1,182 participants who completed a one-year parent study will take either a lower…

This trial tests whether a special alert system called ICU Beacon can help doctors in intensive care units detect early signs of organ failure. The study includes about 1,962 adult ICU patients and compares units using the software plus standard care to those using standard care …

This study tests whether empagliflozin, a diabetes drug, can prevent death, new dialysis, or lasting kidney damage in people with cardiorenal syndrome type 1—a condition where heart failure suddenly worsens kidney function. About 200 hospitalized adults will receive either empagl…

This Phase 2 study is testing a new drug called ADX-038 in 45 adults with complement-mediated kidney diseases, such as IgA nephropathy and C3G. The drug is an siRNA that targets the complement system, which is overactive in these conditions. Researchers are checking if it is safe…

This study tests an experimental drug called recombinant human plasma gelsolin (rhu-pGSN) added to standard care for people with moderate-to-severe acute respiratory distress syndrome (ARDS) caused by pneumonia or other infections. About 600 hospitalized adults will receive eithe…

This study looks at whether using a CPAP machine for sleep apnea after a heart procedure (catheter ablation) can help prevent atrial fibrillation from coming back. About 658 adults with both conditions will be randomly assigned to use CPAP for a year or receive usual care. The go…

This study looks at whether adding oregano and basil leaves to the diet can lower inflammation and improve cholesterol in people who have recently had unstable angina. About 70 participants will eat these herbs and have their blood and urine tested. The goal is to see if these co…

This early-phase study tests a new drug called OM336 in 39 adults with active Sjogren's disease or idiopathic inflammatory myopathy who have not improved with at least two prior treatments. OM336 is an engineered antibody designed to target and potentially reduce harmful immune c…

This early-stage trial tests an oral drug called AG-120 in people with advanced blood cancers, like acute myeloid leukemia, that have a specific IDH1 mutation. The study aims to find the safest dose and see if the drug can control the disease. About 291 participants will take the…

This study tests whether a two-drug combination of dabigatran and ticagrelor can lower bleeding risk compared to the standard three-drug therapy (dabigatran, clopidogrel, and aspirin) in people with atrial fibrillation who have had a heart stent after a heart attack. About 1,200 …

This early-stage trial tests a new drug called BCX17725 in healthy volunteers and people with Netherton syndrome, a rare genetic skin condition. The study aims to check safety, how the drug moves through the body, and whether it can reduce skin redness and scaling. About 78 parti…

This Phase 3 trial compares elritercept to epoetin alfa in 300 adults with low- to intermediate-risk myelodysplastic syndromes (MDS) who need red blood cell transfusions. The main goal is to see if elritercept can help patients go without transfusions for at least 12 weeks while …

This phase 3 trial tests whether empasiprubart, given by IV infusion, can help adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder that causes weakness and disability. The study will enroll 160 participants. For the first 6 months, some get the …

This study is testing a new way to do stem cell transplants for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS). It combines stem cells from a half-matched donor (haploidentical) with cord blood, given at different times. The goal is to help …

This study is testing a small, leadless pacemaker placed in the heart's upper chamber (atrium) for people with sinus node dysfunction, a condition where the heart's natural pacemaker doesn't work properly. Participants receive the Aveir AR pacemaker and a loop recorder to track h…

This study is checking the long-term safety of guselkumab, a biologic drug given as a shot, in children with moderate to severe Crohn's disease, ulcerative colitis, or juvenile psoriatic arthritis. About 196 kids who already completed a prior guselkumab study and benefited from i…

This study tests a telehealth program called tele-ImPACT for infants aged 6-24 months who have an older sibling with autism, putting them at higher risk for social communication delays or language disorders. The program teaches parents strategies to support their child's communic…

This study is testing whether a lower dose of the drug cyclophosphamide, given after a stem cell transplant, can prevent graft-versus-host disease (a common complication where donor cells attack the patient's body) while still being effective. The trial involves 260 adults with b…

This study tests a drug called soquelitinib in 15 people aged 16 and older with autoimmune lymphoproliferative syndrome (ALPS). The goal is to see if the drug can shrink an enlarged spleen or lymph nodes and improve low blood cell counts. Participants take the pill twice daily fo…

This study tests a new drug called Lu AG13909 in 18 adults with Cushing's disease, a rare condition where the body produces too much cortisol. The goal is to see if the drug can safely lower cortisol levels to normal. Participants will receive the drug through an IV or injection,…

This early-phase trial tests whether adding natural killer (NK) cells from a healthy donor to the standard drugs azacitidine and venetoclax can help control acute myeloid leukemia (AML). About 32 adults with relapsed or hard-to-treat AML will receive the combination. The main goa…

This early-stage trial is testing a new drug called cirtuvivint, alone or with another drug combo (ASTX727), in people with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML). The goal is to find the safest dose and check for side effects. About 54 participants whose…

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

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

This study tests whether giving a personalized dose of Pentaglobin (an antibody treatment) after surgery helps people with severe abdominal infection (peritonitis) and sepsis recover better. Researchers will enroll 200 patients and track organ failure and survival. The goal is to…

This study tests a drug called dostarlimab in 25 people with stage II or III colon cancer that has a specific genetic feature (dMMR). The drug is given before any surgery to see if it can shrink the tumor enough that surgery may not be needed. Participants receive the drug every …

This pilot study tests a 3-month program that combines online education, music-paced brisk walking, group discussions, and phone follow-ups for 40 adults with both metabolic syndrome and chronic kidney disease. The goal is to see if the program can reduce waist circumference and …

This study tests a program called MiWeigh that helps people with obesity and related conditions like diabetes or high blood pressure lose weight. Participants get a personalized plan from a doctor, access to a website, and weekly check-ins. The goal is to see if this approach lea…

This study tests an oral drug called NNZ-2591 in 160 children aged 3 to 12 with Phelan-McDermid syndrome, a rare genetic condition. The drug is compared to a placebo to see if it improves communication and daily function. The trial is double-blind, meaning neither the families no…

This study tests a new medicine called empasiprubart against the standard treatment (IVIg) for people with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disease that causes weakness and numbness. About 218 adults who have used IVIg before will receive either t…

This study follows people with chronic myeloid leukemia (CML) who have already benefited from the drug asciminib in earlier Novartis trials. Researchers want to see how safe the drug is over the long term. About 347 participants will continue taking asciminib or other similar dru…

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

This Phase 3 trial tests whether adding baricitinib (a JAK inhibitor pill) to standard steroid treatment can help people with polymyalgia rheumatica achieve disease control without needing long-term steroids. About 140 adults aged 50+ with active PMR will receive baricitinib (2 m…

This early-stage trial tests a new drug called PRS CK STORM, made from a mix of immune and fat cells, to see if it can safely calm the 'cytokine storm'—a dangerous overreaction of the immune system—in people with severe lung infections from COVID-19, flu, or RSV. The study will e…

This study combines three chemotherapy drugs (cladribine, idarubicin, cytarabine) with a targeted drug (quizartinib) to treat acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). It includes up to 80 adults who are newly diagnosed or whose cancer has returne…

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

This study tests a new drug called sonelokimab for adults with active psoriatic arthritis who have not improved with or cannot take standard TNF-blocker medications. About 600 participants will receive either sonelokimab, a placebo, or an existing drug (risankizumab) for comparis…

This study follows 335 adults with severe eosinophilic asthma who are taking benralizumab. Researchers want to see if the drug helps patients achieve partial or complete clinical remission—meaning fewer symptoms, no need for steroid pills, and better lung function—over 12 to 24 m…

This early-phase trial tests a genetically modified virus (VSV-IFNβ-NIS) alone or with chemotherapy and immunotherapy drugs in people with blood cancers like multiple myeloma, acute myeloid leukemia, and lymphomas that have returned or not responded to treatment. The virus is des…

This study tests a new medicine, maridebart cafraglutide, for adults with obstructive sleep apnea who are overweight or obese and already use a CPAP machine. The goal is to see if the drug can reduce breathing pauses during sleep and help with weight loss over 52 weeks. About 250…

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

This study tests a combination of three drugs—ASTX727, venetoclax, and gilteritinib—for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome that has a FLT3 mutation. The drugs work in different ways to stop cancer cells from growing. The trial aims to f…

This early-phase trial tests a single inhaled dose of a lab-made protein (Rh-KGF-2) in 24 adults with moderate to severe ARDS who are on ventilators. The goal is to see if the drug is safe and tolerable, and to find the right dose. Participants are split into three dose groups, w…

This study looks at whether adding tirzepatide (a weight-loss drug) to standard ixekizumab treatment helps adults with active psoriatic arthritis who are overweight or obese. About 200 participants will receive both medications for up to 12 months. The goal is to see if this comb…

This study tests a new treatment for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or not responded to standard therapy. The treatment uses specially engineered natural killer (NK) cells designed to recognize and attack cancer cells…

This phase 3 trial tests whether maridebart cafraglutide, a drug that may help with weight loss, can reduce sleep apnea episodes in 250 adults who are overweight or obese and not using a breathing machine. Participants receive either the drug or a placebo for 52 weeks. The main g…

This study tests whether a specially designed health behavior program can help people with metabolic syndrome and chronic kidney disease reduce their waist size and improve kidney function. Researchers will compare the program to usual care in 160 adults, measuring changes in wai…

This study looks at a new genetic test called Caris Chromoseq for people with certain blood cancers (acute myeloid leukemia, myelodysplastic syndrome, or myeloproliferative neoplasms). The test scans the entire genome to help classify the cancer and guide risk assessment. Researc…

This study tests whether measuring a substance called IL-6 in the blood can quickly predict if an emergency room patient with a suspected infection will develop sepsis or septic shock. Researchers will enroll 450 adults admitted from the ER and track their outcomes for up to 30 d…

This study tests whether using a lung ultrasound score can help doctors decide when to give surfactant therapy to premature infants with respiratory distress syndrome. Surfactant is a substance that helps babies' lungs stay open, but giving it too late can reduce its benefit. The…

This study aims to see if a French version of the OSA-18 questionnaire can accurately identify obstructive sleep apnea in children. Parents of children aged 4 to 17 who are already scheduled for a sleep test (polysomnography) will fill out the questionnaire. The results will be c…

This study tests a new radioactive tracer called 68Ga-GPC3 that targets a protein found on liver cancer cells. About 40 adults with suspected or confirmed hepatocellular carcinoma will receive both this new tracer and a standard tracer (18F-FDG) for PET/CT scans. Researchers will…

This phase 2 study tests a radioactive tracer called [68Ga]Ga-PentixaFor to see if it can better identify adrenal tumors that produce excess hormones like aldosterone or cortisol. About 80 adults with known adrenal tumors and high hormone levels will receive the tracer before a P…

This study is testing a wearable sensor that measures oxygen levels in the brain and limbs of children with and without obstructive sleep apnea (OSA). The sensor, called near-infrared spectroscopy (NIRS), is compared to standard sleep study techniques. Researchers hope it could l…

This study will test whether artificial intelligence (AI) can help doctors find and identify colorectal polyps during colonoscopy better than standard methods. About 1000 adults having a routine colonoscopy will be randomly assigned to either AI-assisted or standard colonoscopy. …

This study is testing whether artificial intelligence (AI) can predict or rule out a heart attack using data from standard blood tests. Researchers will analyze white blood cell properties from over 3,000 adults who come to the hospital with chest pain. The goal is to see if AI c…

This study tests whether a special PET scan (FET PET/CT) can better locate small pituitary tumors that cause Cushing disease. About 50 adults with confirmed Cushing disease who are scheduled for pituitary surgery will receive the scan before their operation. The goal is to see if…

This study is testing a new blood test called DENEB that looks for tiny molecules (microRNAs) to find colorectal cancer and precancerous polyps early. Researchers will collect blood samples from 2,000 people who are also getting a colonoscopy. The goal is to see if the blood test…

This study tests whether a smartphone app called Apneal can help screen for obstructive sleep apnea in people with head and neck cancer. Sleep apnea is common but often missed in these patients, and current diagnosis requires expensive equipment. Participants use the app at home …

This study compares advanced PET scans (68Ga-DOTATATE and F-DOPA) with standard imaging to locate hidden tumors causing ectopic Cushing syndrome. About 80 adults with this condition will undergo multiple scans over time. The goal is to see which scan or combination best finds the…

This study tests whether a small, single-use home monitor called Sunrise can help doctors decide how to adjust CPAP therapy for people with obstructive sleep apnea who are not getting enough benefit. About 105 adults will use the Sunrise device at home and also undergo a standard…

This study investigates whether a urine test called Nephrocheck can predict which patients with septic shock will develop severe acute kidney injury. The test measures two markers of kidney damage (TIMP2 and IGFBP7). Researchers will analyze data from previous studies involving a…

This study tests whether an AI tool that reads ECGs can safely and effectively help emergency doctors decide if a patient with chest pain is having a heart attack. About 4,670 adults with chest pain suspicious for a heart attack will be randomly assigned to either AI-assisted or …

This study looks at whether a menstrual cup can collect enough uterine tissue to screen for endometrial cancer, especially in women with Lynch syndrome who are at higher risk. About 25 participants will use a menstrual cup at home and also have a standard biopsy, then compare the…

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

This early-phase trial tests a vaccine (CMV-MVA Triplex) given to both stem cell donors and recipients to boost immunity against cytomegalovirus (CMV) and prevent active infection after a half-matched stem cell transplant. About 46 people with various blood cancers will take part…

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

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

This study is testing whether a special software that analyzes CT scans of the heart's arteries can better prevent heart attacks, strokes, and other cardiovascular events compared to usual care based on risk factors like cholesterol and blood pressure. The trial will enroll 7,500…

This study tests whether taking part in racial justice activism can prevent depression and reduce physical stress in Black and Latinx young people aged 15-20. Researchers will measure mood, stress hormones, and health markers over two years. The goal is to see if activism can be …

This early-phase study tests the safety of a drug called ONC201 (dordaviprone) in 36 adults at high risk for colorectal cancer due to familial adenomatous polyposis (FAP) or a history of many polyps. The drug aims to kill precancerous cells without harming normal ones. Researcher…

This study tests whether a non-invasive device that stimulates a nerve in the ear can help people with fibromyalgia. Participants use the device for 30 minutes daily over four weeks. The goal is to see if it reduces pain, fatigue, and other symptoms by calming the body's stress r…

This phase 3 trial tests whether the drug neridronate, given as four IV infusions over 10 days, can reduce pain and other symptoms of complex regional pain syndrome type 1 (CRPS-1). About 270 adults with CRPS in one limb will receive either the drug or a placebo. The main goal is…

This study tests a new eye drop called HUC1-394 in 150 adults with moderate to severe dry eye disease. Participants will use the drops or a placebo for a period to see if the drug reduces eye surface damage and improves symptoms. The goal is to find the best dose and frequency.

This study tests three approaches—oxygen therapy, the drug trazodone, and brief low-oxygen episodes—to see if they can improve sleep apnea in people with chronic cervical spinal cord injury. Researchers will measure changes in breathing and sleep quality in 100 participants. The …

This study tests whether online group education and lifestyle coaching can improve quality of life and health markers in women with PCOS and obesity. Forty non-pregnant women aged 18-49 will be assigned to either the virtual program or usual care. Researchers will measure changes…

This study compares three different over-the-counter lubricating eye drops in people with dry eye disease. Researchers will measure how well each drop is tolerated, how much it improves symptoms like discomfort and blurry vision, and its effect on daily visual function. The trial…

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

This study tests whether a personalized form of transcranial magnetic stimulation (TMS) can improve motor and non-motor symptoms in people with Parkinson's disease, multiple system atrophy, or progressive supranuclear palsy. Fifty participants will receive 10 days of targeted TMS…

This early-phase study tests whether MDMA-assisted therapy can reduce pain in 20 adults with fibromyalgia. Participants receive MDMA combined with psychotherapy and undergo brain scans to see how the drug affects pain and the bond between patient and therapist. The goal is to und…

This study tests a new eye drop called DFL24498 in people with dry eye disease. About 417 adults will use the drops four times a day for 12 weeks. Researchers will check if the drops improve eye surface health and reduce dryness symptoms compared to a placebo.

This study tests whether cannabidiol (CBD) can safely improve behavior, mood, sleep, and daily function in people with Sanfilippo syndrome, a rare genetic disorder. Thirty-five participants will receive either CBD or a placebo, then switch after a break. Caregivers will report on…

This study tests whether shining low-level laser light on muscles can reduce fatigue and improve quality of life in 40 adults with chronic fatigue syndrome. Participants will receive either real laser therapy or a sham treatment three times a week for eight weeks. Researchers wil…

This study tests whether core shamanism—sessions with drumming, rattling, and discussion—can help reduce pain and other symptoms in women with fibromyalgia. Twenty-five participants will attend up to five sessions while researchers measure pain, heart rate, breathing, and brain a…

This study tests whether a silicone mouth tape worn during sleep can reduce snoring and improve sleep quality in people with mild sleep apnea or simple snoring. About 100 adults with a bed partner will use the tape for a period, and both the snorer and partner will report on snor…

This study tests a new medication called PP-01 to help adults with cannabis use disorder manage withdrawal symptoms when they stop using cannabis. About 420 participants will receive PP-01, an existing drug (nabilone), or a placebo daily for 34 days, with a one-week hospital stay…

This Phase 2b study tests camlipixant, an experimental drug, in 420 adults with moderate-to-severe irritable bowel syndrome (IBS) with diarrhea or mixed bowel habits. Participants receive either camlipixant or a placebo for 26 weeks. The main goal is to see if the drug reduces we…

This study tests whether acetazolamide, a drug used for other types of sleep apnea, can help people on methadone or buprenorphine who develop central sleep apnea. Forty participants will take either the drug or a placebo for 7 days, then have an overnight sleep study. Researchers…

This study compares two drugs, pregabalin and mirogabalin, to see which works better for fibromyalgia pain. About 674 adults with moderate to severe fibromyalgia who haven't tried these medications will take one of the drugs for several weeks. The main goal is to see how many peo…

This Phase 3 trial tests whether the experimental drug paltusotine can reduce flushing and diarrhea in adults with carcinoid syndrome caused by neuroendocrine tumors. About 141 participants will receive either paltusotine or a placebo daily for 12 weeks. The study measures how mu…

This study tests whether a 12-week program of daily breathing exercises can improve lung function and reduce fatigue in adolescents with Behcet disease who have had lung surgery. Forty participants will do inspiratory muscle training at home, six days a week. Researchers will mea…

This study tests whether pregabalin, a drug used for nerve pain, can ease symptoms of irritable bowel syndrome (IBS). About 258 adults with mild-to-moderate IBS will receive either pregabalin or standard IBS treatments for 12 weeks. Researchers will measure changes in IBS severit…

This early-phase trial tests whether injecting mesenchymal stromal cells (MSCs) into the salivary glands is safe and can ease dry mouth in people with graft-versus-host disease or Sjogren's disease. Up to 36 adults will receive one injection and be monitored for pain and side eff…

This study tests whether a testosterone drug (Depo-Testosterone) can reduce fatigue and improve sexual function, mood, and body composition in young men (ages 18-54) who have survived cancer and now have low testosterone. Participants must be in remission for at least one year an…

This study tests a 9-week phone-based program called WISH that teaches positive psychology exercises to people with irritable bowel syndrome (IBS). The goal is to see if the program is practical and helpful for improving symptoms. Researchers will compare it to an educational con…

This study tests whether a protein-based eye drop (rhPRG4) can safely relieve dry eye symptoms in people with Sjögren's syndrome. About 80 adults will receive either the active drops or a placebo for 28 days. The main goal is to see if the drops can clear corneal staining, a sign…

This study investigates whether drinking plasma-activated water—water treated with light and nano gold particles to create smaller water clusters—can safely improve symptoms in people with fibromyalgia, a condition causing widespread pain. Researchers will measure changes in bloo…

This study tests whether adding a probiotic called Ecologic Barrier to standard treatment helps adults with irritable bowel syndrome (IBS) who also have stomach issues and anxiety, depression, or stress. Sixty participants will receive either standard care or standard care plus t…

This study tests whether a non-invasive brain stimulation technique called tDCS can reduce pain in people with Complex Regional Pain Syndrome (CRPS). Thirty-two adults with active CRPS will receive either real or sham stimulation over several sessions. The goal is to see if this …

This study tests a new medicine called CTH120 in 30 adult men with Fragile X syndrome. The main goal is to see if the drug is safe and tolerable, while also checking if it helps improve symptoms. Participants will receive the drug and be monitored closely for side effects and cha…

This study tests whether a personalized exercise program can improve balance and physical function in adults with rare neuromuscular disorders like Charcot-Marie-Tooth disease, facioscapulohumeral muscular dystrophy, and myotonic dystrophy type 1. Participants will receive a 12-d…

This study is testing two versions of Tears Naturale lubricating eye drops—one with preservatives and one without—in 120 people with mild to moderate dry eye disease. Participants will stop using their usual artificial tears and use only the study drops for the duration of the tr…

This study tests whether a special laser (MLS class IV) can reduce chronic pain and fatigue in people with hypermobile Ehlers-Danlos syndrome (hEDS), a condition that causes loose joints and widespread pain. Twenty-five adults will receive 10 laser sessions over 5 weeks, with pai…

This study tests an experimental drug called MRM-3379 in males aged 13 to 45 with Fragile X Syndrome. The goal is to see if the drug is safe and can improve symptoms like learning and behavior. Participants will take the drug or a placebo for 12 weeks. The study is currently recr…

This study tests three types of injections for people with chronic neck and shoulder muscle pain caused by trigger points. It compares platelet-rich plasma (PRP), a mix of PRP and steroid, and steroid alone to see which works best. 150 adults aged 18-65 with a single active trigg…

This study tests two non-invasive treatments—connective tissue massage and vagus nerve stimulation—alone or together, in 66 women with fibromyalgia. The goal is to see if these methods improve pain, sleep, fatigue, and quality of life. Participants will be randomly assigned to on…

This study tests a non-invasive device that gently stimulates a nerve in the ear to see if it can reduce depression symptoms in people who have had a heart attack or related heart problem and a stent placed. About 120 adults will use the device at home for 8 weeks, and their mood…

This study tests whether acupuncture can make opioid painkillers more effective for people with fibromyalgia. About 45 adults who already take daily opioids will receive either real acupuncture or a sham (fake) version. Researchers will measure pain changes after an opioid dose t…

This study tests whether using a video camera (video laryngoscopy) helps doctors give surfactant—a medicine that helps lungs work—to very premature babies more successfully on the first try compared to the standard direct-look method. About 100 babies born at 28 weeks or earlier …

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

This study tests whether a non-invasive brain stimulation technique called rTMS can reduce pain and depression in people with fibromyalgia. About 36 adults with chronic pain, some also depressed, will receive either real or sham (fake) rTMS to two brain areas. Researchers will tr…

This study tests whether using a portable oxygen concentrator (a lightweight, battery-powered device) during walking or exercise helps people with interstitial lung disease (ILD) or COPD feel less breathless and be more active in their daily lives. Twenty-four participants will t…

This study tests two hands-on treatments—nerve stretches and muscle pressure release—to see which better reduces menstrual cramps, improves sleep quality, and lowers stress in women with Polycystic Ovary Syndrome (PCOS). 38 women aged 18-35 with PCOS and painful periods will be r…

This study aims to understand how and why eosinophils (a type of white blood cell) become active in conditions like allergies, asthma, and parasitic infections. Researchers will observe up to 800 people aged 1 to 100 with high eosinophil levels. No experimental treatments are giv…

This study follows healthy girls ages 8-14 and teens with irregular periods to learn how their menstrual cycles become regular after their first period. Researchers also include girls at risk for PCOS and compare them to women with and without PCOS. The goal is to understand norm…

This study follows people with inherited BAP1 gene mutations, which raise the risk of mesothelioma and other cancers. Researchers will track participants over many years, collecting medical history, blood samples, and scans to understand how these mutations lead to cancer. The go…

This study looks for genetic changes linked to progressive familial intrahepatic cholestasis (PFIC) in adults with unexplained bile flow problems. Researchers will collect health information and blood samples from 150 participants across Spain during a single visit. The goal is t…

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

This study follows 155 adults with CADASIL, a genetic condition that narrows small blood vessels and raises the risk of strokes and dementia, over nine years. Researchers will use brain scans, blood tests, and other exams to track how the disease changes blood vessels and thinkin…

This study surveys 250 informal caregivers (family or friends) of people with Erdheim-Chester disease and similar conditions. Participants complete online questionnaires about their supportive care needs, mood, and sense of purpose. The goal is to identify what caregivers need mo…

This study will analyze DNA from up to 20,000 people, including those with immune disorders and their relatives, to find genetic variants linked to conditions like atopy, autoimmunity, and primary immunodeficiency. Researchers will also study how best to return genetic results to…

This study aims to understand the causes and progression of systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD) by observing up to 2,000 participants over time. Researchers will collect medical history, blood samples, and genetic data to identify …

This study collects blood, bone marrow, skin, saliva, and stool samples from up to 1,000 healthy volunteers aged 18 and older. The samples will be stored in a database for researchers to compare with samples from people who have myelodysplastic syndromes (MDS), a group of blood d…

This study aims to uncover genetic changes that cause autoimmune diseases in people native to Peru. Researchers will collect blood, urine, and stool samples from 300 participants, including patients with conditions like lupus and rheumatoid arthritis, their family members, and he…

This study follows 900 people with bronchiectasis—a condition where airways are damaged and prone to infection—to learn why they get sick repeatedly. Researchers will collect medical history, lung function tests, and blood, urine, and sputum samples from patients and their family…

This study follows people with a rare genetic condition called DICER1 syndrome, which raises the risk of certain childhood cancers like pleuropulmonary blastoma. Researchers will collect medical histories, genetic samples, and tissue from up to 1,500 participants and their relati…

This study aims to learn more about Hermansky-Pudlak Syndrome (HPS), a rare inherited disease that causes light skin/eye color, bleeding problems, and often deadly lung scarring. Researchers will follow 600 people with HPS and their family members to track how the disease progres…

This study collects blood, urine, saliva, and tissue samples from nearly 6,000 people who have or may have cancer, including rare inherited forms. The samples are stored and used for research to understand the genetic and molecular basis of cancer. No new treatment is being teste…

This study aims to uncover the genetic causes of hereditary leiomyomatosis renal cell cancer (HLRCC), a condition that can lead to painful skin bumps, uterine fibroids, and kidney tumors. Researchers will study people with known or suspected HLRCC and their family members to iden…

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

This study follows up to 3,500 people with sickle cell disease to learn more about how the condition progresses and what complications arise. Participants receive their usual medical care outside the hospital and visit the clinic for check-ups and research tests. No experimental …

This study looks at families where non-medullary thyroid cancer runs in the family. Researchers want to learn how the disease develops, find genes that might increase risk, and figure out the best way to screen for it early. Participants will have exams, imaging, and blood tests,…

This study tests a new AI system called IntelliLung that suggests ventilator settings for ICU patients on breathing machines. Researchers will compare how often doctors' settings match the AI's recommendations. About 530 adults on ventilators will take part across multiple hospit…

This study follows up to 2000 people with immune-related kidney diseases, such as nephrotic syndrome and lupus nephritis. Researchers will collect medical history, lab tests, and blood or urine samples to better understand these conditions. The goal is to learn more about causes …

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

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

This study aims to learn more about the genetics and natural history of autoinflammatory diseases, which cause repeated fevers and inflammation. Researchers will study up to 5,000 people, including patients, their relatives, and healthy volunteers. Participants provide blood and …

This study investigates a technique called drug-induced sleep endoscopy (DISE) to examine the upper airway in adults with obstructive sleep apnea (OSA) who are considering surgery. Participants receive a mild sedative (propofol) to induce sleep, then a thin camera is passed throu…

This study aims to learn more about heart and blood vessel diseases by observing people who have them or are at risk. Researchers will collect samples and perform tests like imaging and stress tests on up to 5,000 participants, including healthy volunteers and relatives of affect…

This study looks at whether special mouth and throat exercises can improve sleep apnea by strengthening the muscles that keep the airway open. Thirty adults with sleep apnea will use myofunctional therapy devices for three months. Researchers will measure muscle pressure in the t…

This study looks at the causes and effects of food allergies and related conditions like eczema and eosinophilic esophagitis. Researchers will collect blood, skin, and genetic samples from up to 1,800 people over many years. The goal is to find biomarkers and pathways that explai…

This study evaluates a questionnaire called the Emotional Allodynia Questionnaire (AEQ), which measures how people with fibromyalgia react emotionally to everyday social cues. Researchers want to see if the tool can reliably identify fibromyalgia and tell it apart from other type…

This study follows 300 adults aged 30 and older who have or may have a BAP1 gene mutation, which raises the risk for mesothelioma and other cancers. Researchers will use advanced CT scans, blood tests, and minimally invasive camera procedures to look for early signs of cancer. Pa…

This study looks at 50 children (over 1 year old) in intensive care who are on breathing machines due to acute respiratory distress syndrome (ARDS). Researchers want to see if two methods can reliably measure airway opening pressure, which could help doctors personalize ventilati…

This study creates a large registry of people with Rett syndrome, a rare genetic disorder that causes severe intellectual and physical disabilities. Researchers will collect information from doctors and caregivers to understand how the disease progresses over time. The goal is to…

This study looks at preterm infants who need breathing support from a machine called nasal CPAP. Researchers want to find out how much oxygen actually reaches the baby's lungs by placing a small tube in the back of the mouth and using soft bands around the chest to measure lung e…

This study screens 360 adults with neurodegenerative disorders like ALS, frontotemporal dementia, and progressive supranuclear palsy. Researchers will use medical history, physical exams, memory tests, movement analysis, MRI scans, and other tests to find common features and diff…

This study explores whether a computer algorithm can analyze resting-state MRI scans taken before surgery to predict how long a person with glioblastoma multiforme (a type of brain cancer) might survive. The goal is to see if the algorithm can accurately classify patients as shor…

This study tracks 98 people with peripheral artery disease who had a stent placed in their iliac artery. Researchers want to see which patients later develop serious limb complications, like needing another procedure or amputation. They will also check if frailty plays a role. Th…

This study follows people with autoimmune lymphoproliferative syndrome (ALPS) and their relatives over many years. Researchers will collect blood and tissue samples, track symptoms, and study genetics to better understand why the immune system attacks the body. The goal is to lea…

This study aims to understand Gulf War Illness (GWI), a condition affecting up to 210,000 veterans with symptoms like fatigue, pain, and memory problems. Researchers will compare 85 Gulf War veterans—some with GWI and some without—through extensive tests including blood draws, sc…

This study aims to learn more about diseases that affect the salivary glands, which produce saliva. Researchers will collect medical data, blood, saliva, and tissue samples from people with these conditions, their relatives, and healthy volunteers. The goal is to better understan…

This study looks at the bacteria, fungi, and viruses living on the skin, in the gut, and in the mouth of people with Netherton syndrome, a rare condition that damages the skin barrier and often causes allergies. Researchers will compare these microbes to those of healthy voluntee…

This study follows up to 500 people with polyostotic fibrous dysplasia or McCune-Albright syndrome to learn how these conditions change over time. Researchers will collect medical data and tissue samples to better understand the disease. The goal is to improve future diagnosis an…

This study looks at why people with tuberous sclerosis, a rare genetic condition, develop non-cancerous skin tumors. Researchers will examine up to 400 adults, perform skin exams, and take small tissue samples (biopsies) from tumors. The goal is to identify the cells and genetic …

This study aims to find the genes that keep airways healthy and understand how genetic mutations cause primary ciliary dyskinesia (PCD), a rare disease that leads to serious lung problems. Researchers will study people with suspected PCD and healthy family members. The goal is to…

This study will test new methods to measure how sleep apnea affects the heart. Researchers will follow 270 adults with moderate to severe sleep apnea who use CPAP, mouth devices, or nerve stimulation. The goal is to see if these new metrics can better predict heart problems than …

This study looks at children under 15 with neurofibromatosis type 2 (NF2), a rare genetic condition that causes nerve tumors. Researchers want to see if skin and mouth lesions can help diagnose NF2 earlier than current methods, which rely on hearing, nerve, and eye symptoms. By e…

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

This research study creates a registry of older adults with cancer to collect information about their health, treatments, and outcomes. Participants complete a geriatric assessment and allow researchers to follow their medical records. The goal is to better understand how cancer …

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

This study creates a registry of medical records, scans, and surveys from adults with neurofibromatosis type 1 (NF1) who also have a glioma brain tumor. Researchers will track diagnosis, treatments, and quality of life over time to better understand the disease. The goal is to im…

This study looks at whether stimulating two nerves in the neck (the ansa cervicalis and hypoglossal nerve) can help people with obstructive sleep apnea. Researchers will measure how the airway changes during sleep in 100 adults. The goal is to understand if combining these stimul…

This study aims to better understand disorders that cause hearing to fluctuate, such as Meniere's disease. Researchers will use advanced MRI scans and blood tests to look for biomarkers linked to hearing changes. The goal is to identify different types of these disorders and pave…

This study investigates whether a gut infection called Brachyspira causes IBS symptoms by making the immune system overreact to certain foods. Researchers will use advanced imaging to identify which foods trigger reactions in the gut. Participants will eliminate those foods for f…

This study will follow 40 adults aged 18-70 who currently take tirzepatide, a medication for blood sugar and appetite control. Researchers will temporarily stop the drug for 3-4 weeks and then restart it for 6-8 weeks, using brain scans, questionnaires, and stool samples to track…

This study looks at gum disease in people with rare kidney disorders like Alport syndrome, Fabry disease, and tuberous sclerosis, as well as lupus. Researchers will compare 100 participants to those with chronic kidney disease and healthy controls. They aim to understand how comm…

This study looks at whether childhood trauma is linked to changes in stress hormones and inflammation in obese women. Researchers will measure cortisol levels in saliva and urine, along with other markers, in 102 women. The goal is to better understand how past experiences might …

This study is looking at how well current classification criteria can diagnose primary Sjögren's syndrome, a rare autoimmune disease. Researchers will compare the criteria against diagnoses made by expert doctors. The goal is to improve diagnostic accuracy for this condition.

This study follows 75 people aged 6 months or older with congenital myasthenic syndromes (CMS), a group of inherited disorders that weaken communication between nerves and muscles. Over up to 7 visits in 5 years, researchers will use physical exams, heart and lung tests, MRIs, an…

This study is analyzing inflammatory markers in the blood and spinal fluid of 300 people who are being checked for multiple sclerosis (MS). The goal is to see if certain markers can help doctors tell MS apart from other autoimmune diseases that affect the brain and spinal cord. R…

This study gathers blood and bone marrow samples from 150 adults with myelodysplastic syndrome (MDS), a blood disorder that can turn into acute leukemia. Researchers want to find biological markers that help predict which patients are likely to progress to leukemia. The goal is t…

This study tests a new way to screen patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) by analyzing their bone marrow and blood for specific biomarkers. The goal is to quickly match patients with a treatment trial that targets their cancer's unique mark…

This study aims to understand why some people are more prone to fungal infections, especially from Candida yeast. Researchers will collect blood, saliva, urine, and tissue samples from people with immune disorders, their family members, and healthy volunteers. No treatment is giv…

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

This study aims to find the best way to deliver preventive health screenings to people in underserved and rural areas. It will compare different methods, such as interactive health reports, mobile screening units, and even droid-assisted screenings, to see which improves health e…

This study is a registry that collects information from all suspected heart attack cases in Tomsk, Russia. It aims to track how many people die within 28 days and one year after a heart attack. No new treatments or drugs are being tested—just observation and data gathering.

This study looks at whether certain genes make some people more likely to develop eye damage from the drug Plaquenil, which is used for autoimmune diseases like lupus and rheumatoid arthritis. Researchers will compare people who have had eye damage from Plaquenil with those who h…

This study compares two types of CPAP machines that help premature babies breathe. One delivers a constant flow of air, the other a variable flow. Researchers will measure the babies' diaphragm activity to see if constant flow works just as well. The study involves 20 infants bor…

This study is tracking 1,500 people with cardiac sarcoidosis—a condition where inflammation affects the heart—to learn how doctors currently diagnose and treat it. Researchers will also test whether corticosteroid therapy improves outcomes. The goal is to create better guidelines…

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

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

This study looks at how ANCA-associated vasculitis affects the eyes. Researchers will use a special imaging test called OCT angiography to examine blood vessels in the eyes of 70 participants. The goal is to better understand and detect eye involvement early, which could help pre…

This study aims to find how common the KIT D816V mutation is in people with suspected clonal mast cell disease. Researchers will collect blood samples from 450 participants and test them using two sensitive methods. The results may improve understanding and diagnosis of these rar…

This study collects blood and urine samples from 200 adults with glioblastoma (a type of brain tumor) who are receiving radiation therapy. Researchers want to see if levels of two proteins, VEGF and MMP, can predict whether the tumor will come back within one year. The goal is to…

This study follows 269 people with neurofibromatosis type 2 (NF2) for up to 10 years to learn how the disease progresses. Participants will have yearly exams, MRI scans, hearing tests, and blood draws. The goal is to understand tumor growth, hearing loss, and speech or swallowing…

This study looks at whether sulfasalazine, a drug for inflammatory arthritis, makes the standard diabetes blood test (HbA1c) falsely low. That could mean diabetes goes undiagnosed or is undertreated in these patients. Researchers will use continuous glucose monitors and fasting b…

This study aims to collect information and bone samples from up to 1,000 people with bone or mineral disorders, such as tumor-induced osteomalacia or familial tumoral calcinosis. Participants receive standard medical evaluations and may provide bone tissue from surgery or a biops…

This study follows people of all ages who have or may have a RUNX1 gene variant, which can cause bleeding problems and increase the risk of blood cancers. Researchers will collect medical history, blood samples, and bone marrow over many years to understand how the condition deve…

This study tracks immune-related substances in the blood of 40 women undergoing a frozen embryo transfer. Researchers want to see how these markers change before and after the transfer, and whether they relate to pregnancy success. The goal is to better understand the role of the…

This study aims to understand how lymphangioleiomyomatosis (LAM) damages the lungs at the cellular and genetic level. Researchers will follow up to 2,000 participants, including women with LAM and healthy volunteers, using advanced imaging and lab tests. The goal is to find the p…

This study is building a large collection of blood, DNA, and tissue samples along with health records from up to 5,000 people with tuberous sclerosis complex (TSC) or a related condition called lymphangioleiomyomatosis. Participants can give blood or cheek swab samples and allow …

This study compares a smartphone chatbot to usual care for collecting family history and identifying women at high risk for hereditary cancer syndromes. Participants are English-speaking gynecology patients aged 18 and older who have not had prior genetic testing. The goal is to …

This study aims to uncover the genetic causes of pituitary and hypothalamic tumors in children. Researchers will collect blood and tumor samples from up to 2,000 participants to analyze DNA and look for inherited patterns. The study also trains doctors and tests new MRI technique…

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

This study looks at the tiny organisms living on our skin—like bacteria and fungi—and how they might differ in people with eczema (atopic dermatitis). Researchers will collect skin samples from healthy volunteers and patients aged 2 to 40 with moderate to severe eczema, as well a…

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

This study aims to learn more about Li-Fraumeni syndrome (LFS), a genetic condition that raises the risk of several cancers. Researchers will study up to 5,000 people with LFS or related cancer histories to find new genes, understand how the TP53 gene works, and identify factors …

This study looks at what happens when adults with metabolic syndrome stop taking their usual metformin medication for 14 days. Researchers want to see if a short break affects insulin sensitivity, blood sugar levels, and other health markers. Participants will be randomly assigne…

This study aims to find out how often Muir-Torre syndrome (MTS) occurs in people who already have Lynch syndrome, a genetic condition that raises cancer risk. Researchers will examine skin lesions and tumors from 150 participants to see if they are linked to MTS. The goal is to b…

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

This study looks at whether brain activity related to learning can predict how well teens respond to a program aimed at reducing cannabis use. Teens aged 14-17 get a brain scan, then attend 10 weekly virtual sessions where they report use and take drug tests. They can earn prizes…

This study is collecting blood, urine, and kidney tissue samples from 144 children and adults with a kidney condition called nephrotic syndrome. The goal is to build a large database and sample collection to study how the immune system and kidney cells interact. Researchers hope …

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

This study aims to find a marker in stool or urine that can objectively measure whether people with Irritable Bowel Syndrome (IBS) are sticking to their prescribed diet. Researchers will enroll 94 adults with moderate-to-severe IBS and track their food intake and gut symptoms. Th…

This study follows people with myelodysplastic syndromes (MDS) for up to 20 years to understand how the disease changes over time. Researchers will collect blood, bone marrow, and other samples from 1,100 participants, including healthy donors. The goal is to learn more about MDS…

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

This study works to improve questionnaires that measure pain, daily activities, and physical function for people with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas. Participants aged 5 and older will give feedback on existing surveys through group discussions or…

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

This study follows 300 people with Sjogren's disease (or symptoms) over time to learn how the condition starts and progresses. Participants undergo exams, saliva and tear tests, and questionnaires during several visits. No treatment is given; the goal is to gather data and sample…

This study tests whether eye-tracking tasks can detect changes in attention and learning in people with Rett syndrome who are starting a new medication called trofinetide. Researchers will compare eye movements and heart rate before and after 4 weeks of treatment in 20 participan…

This study screens children and young adults up to age 22 whose leukemia has returned or is hard to treat. By testing their blood and bone marrow, doctors can identify specific genetic markers to match them with the most promising experimental treatments. The goal is to build a r…

This study looks at whether personality traits are linked to routine lab test results in 1,000 adults getting a health check-up. Participants fill out a personality questionnaire and provide blood samples. The goal is to see if certain personality types are more likely to have ab…

This study looks at a rare inherited condition called Birt-Hogg-Dube (BHD) syndrome, which raises the risk of kidney cancer. Researchers will collect blood, saliva, and tissue samples from up to 950 participants to find the genes involved and understand how kidney tumors grow. Th…

This study follows 100 women with neuroimmune diseases (like multiple sclerosis or myasthenia gravis) through pregnancy and up to five years after. Researchers will track disease activity, pregnancy complications, and baby health to learn how these conditions affect fertility and…

This study reviews the medical records of 100 adults who were treated for Guillain-Barre Syndrome (GBS) at a single hospital in Switzerland. The goal is to better understand how often GBS occurs, what symptoms and nerve problems people have, what treatments they receive, and how …

This study aims to understand why some people get severe or unusual fungal infections due to immune system problems. Researchers will collect blood, tissue, and other samples from up to 1,200 participants over 25 years. No new treatments are being tested; the goal is to learn mor…

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

This study follows 56 young adults who have a genetic condition that raises their cancer risk. After they graduate from pediatric care at St. Jude, researchers track whether they find adult doctors and continue regular cancer screenings over 8 years. Participants fill out surveys…

This study aims to understand how the brain develops in children with Williams syndrome or 7q11.23 duplication syndrome, compared to healthy children. Researchers will use MRI scans and cognitive tests to track changes over time. The goal is to learn how these genetic variations …

This study is a disease registry that will collect information from 200 people in Egypt who have neurofibromatosis type 1 (NF1), a genetic condition that causes tumors to grow on nerves. The goal is to understand how the disease progresses over time, what treatments patients rece…

This study looks at whether dry eye disease affects the accuracy of corneal measurements needed for cataract surgery planning. 400 cataract patients will be divided into those with and without dry eye. Those with dry eye will use sodium hyaluronate eye drops for two weeks. Resear…

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

This study looks at blood samples from 350 adults with blood cancer who develop a fever and have very low white blood cells (a condition called febrile neutropenia). The goal is to find patterns of proteins in the blood that can predict if a patient will develop serious complicat…

This study reviews medical records of 500 Polynesian patients with bronchiectasis (widened airways) to understand why the condition is so common in French Polynesia. Researchers will look at age of onset, family history, infections, and CT scan patterns to see if a genetic cause …

This study looks at the long-term health of people who were cured of Cushing disease before age 21. Researchers want to understand how past high cortisol levels affect the body and mind years later. Participants will complete surveys and have check-ups every 5 years. The goal is …

This study is testing a new questionnaire called PETRA that uses pictures to help people with suspected thoracic outlet syndrome describe which daily tasks they avoid because of their symptoms. The questionnaire also asks how often they face those tasks, giving doctors a clearer …

This observational study will enroll up to one million adults to understand how health, environment, and lifestyle affect aging and disease. Researchers will link participants' clinical data with information from public records, such as pollution and social factors, to create a d…

This study is for people with a rare genetic muscle condition called congenital myasthenic syndrome (CMS). It does not test any new drug or treatment. Instead, researchers will collect information from up to 100 participants over 4 visits to better understand how the disease affe…

This study investigates how the gut and brain communicate in people with Irritable Bowel Syndrome (IBS) who also experience anxiety or depression. Researchers will compare 100 IBS patients with anxiety or depression to 100 healthy volunteers using brain scans, stool samples, and …

This study compares two methods for guiding early treatment in people with septic shock, a severe infection that can cause organ failure. The standard approach uses blood lactate levels, but this trial tests whether using the CO2 gap (a measure of blood flow) is better at reducin…

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

This study tests whether web-based tools can help people with hereditary cancer syndromes share risk information with their relatives and encourage them to get genetic testing. Researchers will enroll 625 participants (patients and their relatives) and track how many use the tool…

This pilot study at Tulane University is testing whether obstructive sleep apnea (OSA) can cause scarring (fibrosis) in the heart's upper chambers, even in people without atrial fibrillation. Researchers will use a special MRI with contrast dye to measure scarring in 60 adults wi…

This study looks at how genes play a role in lung diseases like cystic fibrosis, asthma, and pulmonary fibrosis. Researchers will examine DNA from up to 3,500 people with and without lung disease to find genetic differences. The goal is to better understand what causes these cond…

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

This study looks at how often sleep apnea occurs in people with peripheral artery disease (blocked leg arteries). Researchers will monitor 295 adults with leg artery disease using a sleep test. The goal is to better understand the connection between these two conditions, which ma…

This study is creating a central database and tissue bank for rare diseases affecting the kidneys and liver, such as ARPKD and Joubert syndrome. Researchers will collect medical information, genetic samples, and tissues from 200 participants to help doctors and scientists better …

This study is creating a registry of up to 5,000 patients with various gut movement disorders, such as GERD, gastroparesis, and IBS. Doctors will use standard criteria to make accurate diagnoses and collect patient-reported outcomes. The goal is to improve understanding of these …

This study looks at how emotional intelligence—the ability to understand and manage emotions—relates to chronic orofacial pain (pain in the face or mouth lasting over three months). Researchers will compare 88 adults with and without this pain using questionnaires about emotions,…

This study is collecting skin and blood samples from 830 people with chronic inflammatory skin diseases like psoriasis, eczema, and lupus. Researchers want to find biological markers that can predict how well a person will respond to treatment after one year. The goal is to move …

This study is for people with rare kidney diseases like Minimal Change Disease, Focal Segmental Glomerulosclerosis, and Membranous Nephropathy. Researchers will collect health information and biological samples from 1,200 participants over many years. The goal is to better unders…

This study investigates whether blood sugar patterns, measured continuously with a wearable sensor, can predict serious heart problems in the year following a heart attack. Researchers will enroll 850 people hospitalized with acute coronary syndrome and track their glucose levels…

This study checks if a French version of the MEPS/MEPI elbow score works well for children aged 4 to 17. It includes kids with and without elbow injuries to see if the score accurately measures elbow pain and function. The goal is to make sure doctors can use this tool reliably i…

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

This study is following 700 adults with myotonic dystrophy type 1 (DM1) over two years. Researchers are measuring walking speed, lung function, and muscle tissue changes to find reliable markers of disease progression. The goal is to improve future clinical trials by better under…

This study is not testing a drug or treatment. Instead, researchers are using artificial intelligence to analyze electrocardiogram (ECG) data from over 127,000 people. The goal is to create computer models that can quickly and accurately predict life-threatening heart rhythm prob…

This pilot study is testing whether fecal microbiota transplantation (FMT) — giving processed stool from a healthy donor via enema — is safe and doable in 20 patients with lymphoma or leukemia who are receiving CAR-T therapy or stem cell transplants. The goal is to see if at leas…

This study looks at 20 adults with psoriatic arthritis and fatty liver disease to see if the drug guselkumab can improve liver fat, joint swelling, and skin plaques. Participants will have two visits over 24 weeks to track changes. The goal is to understand shared disease mechani…

This study helps people with certain kidney diseases (like nephrotic syndrome and FSGS) find clinical trials that match their specific disease profile. Researchers analyze participants' molecular data to recommend targeted therapies in ongoing trials. The goal is to improve treat…

This study looks at whether bacterial biofilms (sticky layers of bacteria) on breast implants can lead to complications like chronic inflammation or Breast Implant Illness. Researchers will collect blood and discarded tissue from 1000 women—some with implants and symptoms, some w…

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

This study is for children up to 4 years old with congenital myotonic dystrophy type 1 (CDM). Researchers want to find better ways to measure how the disease affects motor skills and language, and to identify biological markers. The goal is to improve future clinical trials and c…

This study will follow 1,000,000 adults aged 18 and older for the rest of their lives. Researchers will collect health measurements and track who develops diseases, becomes frail, or dies. The goal is to find which tests best predict how well people age, so doctors can one day of…

This study uses genetic testing of brain tumors from children, adolescents, and young adults (ages 1 to 39) with newly diagnosed high-grade glioma, including DIPG. The goal is to identify specific genetic changes in each tumor and then see if patients can join a treatment trial t…

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

This phase 1 trial at M.D. Anderson Cancer Center is testing whether it is safe to collect stem cells from people with RUNX1 familial platelet disorder, a rare inherited condition that raises the risk of bleeding and leukemia. Four participants will receive drugs to mobilize stem…

This study looks at how the body's innate immune system responds to certain environmental exposures by measuring inflammation markers in blood. Researchers will collect blood samples from up to 725 healthy volunteers aged 18-45 to analyze how specific genes and proteins react. Th…

This study aims to find the genetic causes of primary aldosteronism, a condition where adrenal tumors cause high blood pressure, in Black individuals. Researchers will enroll 1150 participants, including those with difficult-to-control blood pressure or known primary aldosteronis…

This natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

This study follows 6,000 people with histiocytic disorders (like Langerhans cell histiocytosis) to understand long-term health problems, pain, mental health, and causes of death. Researchers compare participants to healthy controls to identify factors that lead to new health issu…

This study looks at blood from people with ARDS, a severe lung condition with no specific medicines. Researchers will take immune cells from the blood and test a new blocking agent in the lab to see if it can reduce lung damage. The goal is to learn more about the disease and fin…

This study investigates how exercise affects the structure and function of fat tissue in adults aged 18-40 with obesity or metabolic syndrome. Participants are randomly assigned to either an exercise program or a sedentary control group. All undergo a 10% weight loss phase, then …