Syndromic disease

This trial tests a new approach to face transplantation that aims to reduce the need for lifelong, high-dose immunosuppressive drugs. The method combines a bone marrow cell infusion with a single low-dose immunosuppressant, potentially lowering the risk of infection, cancer, and …

This study tests a new approach to prevent leukemia from coming back after a stem cell transplant. It uses donor T cells that have been engineered to recognize and attack a specific target (HA-2) on leukemia cells. The trial enrolls adults with acute myeloid leukemia (AML) or mye…

This study looks at whether taking just one blood thinner (a P2Y12 inhibitor) is as safe and effective as taking two blood thinners (dual antiplatelet therapy) for people with stable heart disease who have a drug-coated balloon procedure without a stent. About 2,170 adults will b…

This study tests whether patients can safely stop the drug tacrolimus earlier than usual after a stem cell transplant. Tacrolimus is given to prevent graft-versus-host disease (GVHD), but newer methods have lowered GVHD risk. The trial will enroll 50 people with blood cancers and…

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 trial tests whether two approved drugs, dabrafenib and trametinib, can treat rare cancers that have a specific genetic change called BRAF V600. These drugs are already used for melanoma and lung cancer, but researchers want to see if they help people with other cancer types.…

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 personalized radiotherapy approaches for people with lung cancer, brain metastases, or other solid tumors. It aims to see if adapting radiation based on how a patient responds is safe and effective. About 45 adults will participate, receiving either extra radiati…

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 early-phase trial is testing a new drug, FOG-001, in about 595 people with advanced or metastatic solid tumors, including colorectal, prostate, and liver cancers. The drug is given alone or with other cancer treatments to see if it is safe and shrinks tumors. The study is cu…

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-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 an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the dru…

This study tests a drug called BPC2001 to prevent acute graft-versus-host disease (aGVHD), a serious complication where donor immune cells attack the patient's body after a stem cell transplant. Fifty adults with blood cancers like leukemia will receive six weekly doses of BPC200…

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 trial tests whether a structured six-month lifestyle modification program can improve penile blood flow and erectile function in men with metabolic syndrome-associated erectile dysfunction. Participants receive dietary counseling, exercise guidance, weight management, smokin…

This trial tests whether giving patients special immune cells, called TAA-T, after a stem cell transplant can help prevent high-risk blood cancers like acute myeloid leukemia from coming back. The cells are designed to recognize three common tumor markers and attack any remaining…

This trial is testing whether an experimental drug called PBSS1113, taken as a pill alongside the standard medicine azacitidine, can help people with acute myeloid leukemia (AML) or a related condition called myelodysplastic syndrome (MDS). The study includes adults who have eith…

This study tests whether exemestane, a drug typically used for breast cancer, can help women with polycystic ovary syndrome (PCOS) ovulate. Participants take either a low or high dose of exemestane or a placebo for 5 to 10 days. Researchers track ovulation through blood tests and…

This study tests a daily pill called olutasidenib given after a stem cell transplant to people with certain blood cancers (like AML or MDS) that have an IDH1 mutation. The goal is to see if the drug is safe and tolerable as maintenance therapy. About 31 participants will take the…

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 a new oral medication, upadacitinib, in children aged 1 to 17 with active systemic juvenile idiopathic arthritis (sJIA), a rare and serious form of arthritis that can affect the whole body. About 90 participants will receive either upadacitinib or the standard in…

This trial tests a new approach for glioblastoma, an aggressive brain cancer. A device implanted in the skull uses ultrasound to temporarily open the blood-brain barrier, allowing immune-boosting drugs to reach the tumor. The study involves 25 adults who have completed radiation,…

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 study tests whether the drug CardiolRx can prevent another episode of pericarditis (inflammation around the heart) in people who have had repeated attacks. About 110 adults who have been stable on an IL-1 blocker for at least a year will stop that medication and receive eith…

This Phase 2 trial tests IHL-42X, a combination of two drugs (dronabinol and acetazolamide), in people with obstructive sleep apnea. The study aims to see if the drug can reduce the number of breathing pauses during sleep and improve daytime symptoms. Participants will receive di…

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 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 AZD9793 in people with advanced liver cancer that has spread. The drug is an antibody designed to help the body's immune cells find and attack cancer cells. The main goals are to check if the drug is safe and if it can shrink tumors. About 304 a…

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 is testing a pacemaker lead placed in a specific area of the heart called the left bundle branch area. The goal is to see if this placement is safe and helps the heart beat more naturally in people with slow heart rates. About 140 participants will get the lead and be …

This study tests an experimental drug called IMVT-1402 in 180 adults with moderate to severe Sjögren's disease. Participants receive either the drug or a placebo as a weekly injection under the skin for 24 weeks. The goal is to see if the drug can reduce overall disease activity …

This study tests an experimental drug called DISC-0974 in about 150 adults with myelofibrosis or myelodysplastic syndrome (MDS) who also have anemia. The goal is to see if the drug can reduce the need for blood transfusions and increase red blood cell levels. The study is in earl…

This early-phase trial is testing a new drug called Debio 1562M in about 134 people with acute myeloid leukemia (AML) that has come back or not responded to other treatments. The study first checks safety and finds the right dose, then looks at whether the drug can shrink the can…

This study tests whether a medicine called Panzyga can prevent major infections in people with autoimmune or rheumatic conditions who have low antibody levels after receiving B-cell depletion therapy. About 360 adults will receive either Panzyga or a placebo, and researchers will…

This phase 3 study is testing an oral drug called NNZ-2591 in 180 children aged 3 to 12 with Phelan-McDermid syndrome, a rare genetic condition that causes developmental delays and other challenges. The main goal is to see if the drug is safe and tolerable over the long term. Res…

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 trial investigates whether taking capsules containing freeze-dried fecal matter from healthy donors—known as fecal microbiota transplantation (FMT)—can improve pregnancy outcomes and metabolic health in overweight or obese women with polycystic ovary syndrome (PCOS) who are …

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 study tests a breathing machine strategy that adjusts pressure based on lung measurements to protect the right side of the heart in 180 adults with moderate to severe lung failure from pneumonia. The goal is to see if this method reduces heart strain and complications compar…

This study tests several experimental treatments for progressive supranuclear palsy (PSP), a rare brain disease that affects movement and thinking. About 440 people with early-stage PSP will receive one of the treatments or a placebo. The goal is to see if any of the treatments c…

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 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 phase 3 trial tests glepaglutide, a synthetic hormone given as a twice-weekly injection, in 90 adults with short bowel syndrome who rely on IV nutrition. The goal is to see if the drug can safely reduce the amount of IV support needed. Participants will be randomly assigned …

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 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 new way to give Alpha1-Proteinase Inhibitor—a medicine that helps protect the lungs in people with Alpha-1 Antitrypsin Deficiency. Instead of getting it through a vein at a clinic, participants will receive it as a weekly injection under the skin. The trial wil…

This trial tests whether a mobile app called NutriSteppe can improve blood sugar control and weight loss in adults with type 2 diabetes or obesity who are already taking GLP-1 medications like semaglutide. Over 12 weeks, half the participants will use the app for personalized mea…

This early-phase trial is testing whether dronabinol, a synthetic form of THC, can reduce the severity of obstructive sleep apnea. 120 adults with moderate to severe sleep apnea will take the drug for two weeks. Sleep studies before and after treatment will measure changes in bre…

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 an injected drug called KPL-387 in 165 people with recurrent pericarditis, a condition where the sac around the heart becomes inflamed again and again. The goal is to see if KPL-387 can reduce flare-ups and pain better than a placebo. Participants must have sympt…

This early-stage trial is testing a new drug called CLN-978 in 36 adults with active, moderate to severe Sjogren's disease. The main goal is to see if the drug is safe and tolerable, while also checking how it affects immune cells in the blood. Participants receive the drug as a …

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 phase 2 trial tests whether combining interferon-gamma (a protein that boosts the immune system) with donor immune cells can help patients whose acute myeloid leukemia or myelodysplastic syndromes have returned after a stem cell transplant. The study will enroll 45 adults an…

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 trial tests whether methylphenidate, a standard ADHD medication, can reduce attention and hyperactivity problems in children and adolescents with KBG syndrome, a rare genetic condition often accompanied by ADHD-like symptoms. Participants receive alternating blocks of the dr…

This clinical trial is testing whether adding the drug venetoclax to standard chemotherapy before a stem cell transplant can improve outcomes for people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The study will enroll 324 participants and aims …

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 drug called ARO-DM1 in 78 adults aged 18-65 with type 1 myotonic dystrophy, a genetic muscle disorder. The goal is to check safety and how the body processes the drug. Participants receive either ARO-DM1 or a placebo by IV or injection. It is an early-stage…

This study tests a program called LAGOM designed to improve heart health and quality of life for people with psychotic disorders. 644 adults from six clinics in Gothenburg will either receive the LAGOM program or standard care. The program includes regular check-ups, motivational…

This trial tests whether adding a low-dose blood thinner (rivaroxaban) to standard antiplatelet therapy for the first three months after a heart stent procedure can reduce the risk of future heart attacks, strokes, and other cardiovascular events in high-risk patients. Participan…

This study tests a combination of two drugs, azacitidine and enasidenib, in people with a high-risk blood cancer called myelodysplastic syndrome (MDS) that has a specific genetic change (IDH2 mutation). The goal is to see if the drugs can stop cancer growth and improve outcomes. …

This early-stage trial tests an oral drug called SVG103 (Paxalisib) in 15 adults with rare brain conditions (FCD-II, TSC, or HME) that cause seizures. The main goal is to check safety and side effects, while also seeing if it reduces seizure frequency. Participants take the drug …

This trial asks whether GLP-1 medications, commonly used for type 2 diabetes and weight loss, can better control psoriatic arthritis symptoms than nutrition counseling alone. Adults with psoriatic arthritis who also have obesity and type 2 diabetes will be randomly assigned to re…

This Phase 3 study tests whether telitacicept, a biologic drug given as a shot, can reduce disease activity in adults with active primary Sjögren's disease. About 250 participants will receive either telitacicept or a placebo for 48 weeks. The main goal is to see if the drug impr…

This early-stage trial tests a new gene-editing medicine called TSRA-196 in 72 adults with a severe form of alpha-1 antitrypsin deficiency (the PiZZ genotype) who have lung or liver problems. The treatment is given by IV infusion and aims to raise levels of a protective protein. …

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 study tests whether eating only during a specific window each day (time-restricted feeding) can improve metabolic health in postmenopausal women who have metabolic syndrome or are prediabetic. Researchers will measure changes in insulin sensitivity, body weight, and blood fa…

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 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 phase 2 trial tests a drug called emapalumab in 10 people with APECED, a rare disease where the immune system attacks the body. The drug aims to reduce gut inflammation (enteritis) that causes severe symptoms. Participants receive 7 IV doses over 6 months, and researchers tr…

This study is testing the long-term safety and effectiveness of the drug filgotinib in children and adolescents aged 8 to 17 with juvenile idiopathic arthritis. Participants must have already completed a previous filgotinib study and shown improvement. The goal is to see if the d…

This phase 2 trial tests the drug momelotinib in 57 people with VEXAS syndrome, a rare inflammatory condition that may also involve a bone marrow disorder called MDS. Participants receive momelotinib daily, and doctors check if symptoms improve and if steroid doses can be lowered…

This early-stage trial is testing an oral form of the cancer drug azacitidine (called ASTX030) for people with myelodysplastic syndrome (MDS). The goal is to see if the pill version reaches similar levels in the blood as the standard injection. About 40 adults with MDS will take …

This trial is investigating whether combining an immunotherapy drug (a PD-1 inhibitor like sintilimab or pembrolizumab) with progesterone therapy can help young women with early-stage, mismatch repair-deficient (MMRd) endometrial cancer achieve complete remission while preserving…

This study tests a drug called vadadustat in 1,100 hospitalized adults with acute respiratory distress syndrome (ARDS) caused by infection. Participants take either a low or high dose of vadadustat or a placebo daily for up to 14 days. The goal is to see if the drug reduces death…

This early-stage trial tests an investigational drug called ABBV-319 in 36 adults with lupus or Sjogren's disease. The drug is designed to deplete overactive B cells, which are part of the immune system that cause inflammation and symptoms. Participants receive two doses by IV, 2…

This study tests an experimental drug called R289 in about 86 adults with a type of blood cancer called lower-risk myelodysplastic syndromes (MDS). Participants must have tried other standard treatments without success. The main goal is to check the drug's safety and side effects…

This pilot study tests whether adding powder made from Chardonnay grape skins and seeds to the daily diet can improve gut and heart health. The study includes postmenopausal women aged 45-70 with excess weight and high triglycerides. Participants will consume the powder daily, an…

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 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 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 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 trial asks whether cooling the body to 33-34°C after a cardiac arrest treated with ECMO improves survival and brain function compared to keeping a normal temperature. It includes adults aged 18-75 who had an out-of-hospital cardiac arrest with certain heart rhythms. Particip…

This phase 3 trial tests whether taking the drug olaparib for one year works as well as taking it for two years to keep ovarian cancer from coming back. The study includes 880 people with advanced ovarian cancer (stage III or IV) who have a BRCA mutation or similar DNA repair pro…

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 study tests a new treatment for people with blood cancers like leukemia, lymphoma, and multiple myeloma that have returned or not responded to treatment. Participants receive chemotherapy followed by specially engineered natural killer (NK) cells from donated cord blood. The…

This study looks at the long-term safety and effectiveness of a drug called iptacopan for people with two rare kidney diseases: C3 glomerulopathy and IC-MPGN. About 225 adults who have already completed a previous iptacopan study will take the drug for an extended period. The goa…

This early-stage trial tests a single dose of a gene therapy called NVC-001 in 21 adults with a genetic form of dilated cardiomyopathy (LMNA mutation). The therapy uses a harmless virus to deliver a modified gene into heart cells, aiming to protect the heart muscle. The main goal…

This trial compares two weight-loss surgeries in people with severe obesity (BMI 50-60). One is the standard Roux-en-Y gastric bypass, the other a newer sleeve gastrectomy with duodenojejunal bypass. The goal is to see which procedure leads to healthier hormone responses after me…

This study tests a new drug called PTH-IA for Jansen's Metaphyseal Chondrodysplasia (JMC), a very rare genetic disorder that causes severe bone problems, short stature, and kidney issues. About 12 adults and children will receive the drug to see if it is safe and works. The goal …

This Phase 3 study tests whether the drug apremilast (Otezla) can safely reduce joint inflammation and pain in children aged 5 to 17 with active juvenile psoriatic arthritis. About 60 participants will receive either apremilast or a placebo pill for 16 weeks. The main goal is to …

This study looks at two ways to treat Lennox-Gastaut syndrome (LGS), a severe form of childhood epilepsy: adding more anti-seizure medications or undergoing palliative surgery (like a vagus nerve stimulator or corpus callosotomy). Researchers will track thousands of children acro…

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 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 trial tests whether adding a single injection of the anti-inflammatory drug meloxicam to a standard jaw joint washout procedure (arthrocentesis) improves outcomes for people with painful jaw joint problems. Half of the 50 adult participants will receive the combined treatmen…

This early-phase trial tests the safety of a new drug called karonudib in up to 9 adults with blood cancers like leukemia who have no standard treatment options left. The study aims to find a safe dose and look for any signs that the drug works. It is too early to know if karonud…

This study tests whether the drug nerandomilast can slow lung scarring in people with autoimmune diseases like rheumatoid arthritis or scleroderma. About 400 adults whose lung function hasn't improved with standard treatment will take either nerandomilast or a placebo twice daily…

This trial compares two surgical techniques for treating a painful jaw condition called disc displacement without reduction, where the jaw's cushioning disc slips out of place and limits mouth opening. Participants will receive either arthroscopic lysis and lavage, a procedure th…

This early-stage study tests a new treatment called NT-175 for people with advanced solid tumors (like lung, colon, or breast cancer) that have a specific mutation (TP53 R175H) and a certain immune marker (HLA-A*02:01). NT-175 is made from a patient's own immune cells, which are …

This trial investigates whether placing infants with severe acute respiratory distress syndrome (ARDS) on their stomachs (prone positioning) after surfactant treatment can improve their oxygen levels. The study involves 14 mechanically ventilated infants, comparing oxygenation an…

This trial tests whether giving surfactant through a small tube while babies breathe with high-flow nasal cannula support can reduce the need for invasive ventilation. The study includes infants born 2-6 weeks early who have respiratory distress. Researchers will compare outcomes…

This Phase 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either ma…

This early-stage trial is testing whether an oral form of arsenic trioxide can safely help people with low-risk myelodysplastic syndromes (MDS) who have not improved with standard treatments. The study will first find the safest dose, then check how well it works after several cy…

This Phase 3 trial tests a new inhaled medication called APC-0101 in 520 premature babies born between 26 and 33 weeks who have respiratory distress syndrome. The drug is a surfactant that helps keep tiny air sacs in the lungs open. Some babies receive the active drug through a s…

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 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 empagliflozin, a drug used for diabetes, can reduce chronic inflammation in people with obesity and metabolic syndrome. Researchers will measure immune cells and blood vessel function in 74 adults. The goal is to find a new way to lower heart disease risk…

This study tests whether a behavioral program that reduces evening light exposure and encourages finishing meals at least four hours before bedtime can improve how the body handles sugar. Overweight adults aged 18-45 who habitually sleep less than 6.5 hours per night will be rand…

This study is for people with a rare form of myelodysplastic syndromes (MDS) that has a specific genetic change called IDH1 mutation. Participants have not had prior treatment with certain drugs. They will be randomly assigned to receive either ivosidenib (a daily pill) or azacit…

This program allows doctors to request NanO₂, an investigational drug that may help deliver oxygen to tissues, for patients with serious or life-threatening conditions like glioblastoma, respiratory distress, or ischemic stroke. It is for patients who have exhausted other treatme…

This study tests a drug called TAK-226 (Elritercept) in 27 Japanese adults with lower-risk myelodysplastic syndromes (MDS) who need regular blood transfusions. The goal is to see if the drug can help them go without transfusions for at least 8 weeks. Participants will receive inj…

This study tests whether a ketogenic (high-fat, low-carb) diet can help people with newly diagnosed glioblastoma, an aggressive brain cancer, live longer. About 170 adults will be randomly assigned to either a ketogenic diet or standard dietary guidance, while all receive standar…

This study looks at how well the medication pegcetacoplan works and how safe it is for people with two rare kidney conditions: C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Researchers will follow about 150 patients who are…

This trial tests whether orlistat, a weight-loss medication, can improve hormonal and metabolic features in women with polycystic ovary syndrome (PCOS). Women aged 18-45 with PCOS will receive either orlistat or a placebo, alongside standard lifestyle advice, for three months. Th…

This study compares two common ovarian stimulation protocols used during IVF in women with polycystic ovary syndrome (PCOS). The PPOS protocol uses a progestin hormone to suppress ovulation, while the antagonist protocol uses a different drug. Researchers will track which method …

This trial tests whether a drug called ATLG, used before a stem cell transplant, can help the immune system recover better and reduce viral infections compared to the standard drug ATG. The study involves adults with blood cancers like leukemia, lymphoma, or MDS who are receiving…

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 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 early-stage trial tests a new treatment called QUAIL-100, made from a live weakened bacteria, in children and young adults with high-risk leukemia or myelodysplastic syndrome who have already had a stem cell transplant. The main goal is to check safety and side effects. Only…

This early-stage trial is testing whether a combination of two natural substances, deoxycytidine and deoxythymidine, is safe for people with telomere biology disorders. These rare genetic conditions cause premature aging, bone marrow failure, and lung scarring. Up to 36 participa…

This early-stage trial tests a single injection of AXV-101 gene therapy into one eye of 12 children and teens (ages 4-17) with Bardet-Biedl syndrome type 1, a rare genetic condition that causes progressive vision loss. The main goal is to check safety and find the right dose, whi…

This study tests whether Systane PRO Preservative Free eye drops are as effective as Blink Triple Care Preservative Free in relieving dry eye symptoms. Adults with mild to moderate dry eye will use one of the two drops four times a day for 30 days. Researchers will measure change…

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 early-stage trial is testing a new antibody drug called GEN3018 in people whose acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) has come back or stopped responding to treatment. The main goal is to check safety and find the right dose. Up to 78 parti…

This study tests a device called Aortix, a small pump placed in the main artery, to help remove excess fluid in people hospitalized with acute heart failure who are not responding well to diuretic medications. About 320 participants will be randomly assigned to receive either the…

This study tests an experimental inhaled drug called PUL-042 in 100 people with blood cancers or who have had a stem cell transplant and have a viral lung infection. Participants receive either the drug or a placebo three times over six days. The goal is to see if PUL-042 can red…

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 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 early-phase trial is testing a new biologic drug called XmAb657 in 60 people, including healthy volunteers and those with autoimmune diseases like scleroderma, Sjögren's disease, and idiopathic inflammatory myopathies. The main goal is to check safety and how the body handle…

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 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 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 will follow 30 people with Alagille syndrome who are taking odevixibat in their daily lives. The goal is to see how safe the drug is over the long term by tracking any side effects. It is not testing if the drug works, but rather monitoring its real-world safety.

This study investigates whether changes in retinal blood vessels can serve as a marker for cerebral small vessel disease (SVD), a common condition in older adults that increases stroke and dementia risk. The trial includes people with SVD-related conditions like CADASIL, sporadic…

This study tracks the safety and effectiveness of the arthritis drug Xeljanz XR in about 200 adults with moderate to severe rheumatoid arthritis, psoriatic arthritis, or ankylosing spondylitis. Participants take the drug as prescribed by their doctor, and researchers record any s…

This study looks at the safety and effectiveness of the drug Xeljanz in children aged 2 to 17 with active juvenile idiopathic arthritis or juvenile psoriatic arthritis. It is an observational study in Korean clinics, meaning doctors prescribe the drug as usual and researchers mon…

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 investigates whether combining bariatric surgery with a GLP-1 receptor agonist (semaglutide) leads to greater and more sustained weight loss than surgery alone in people with obesity. Researchers will follow 400 participants across multiple centers in China for up to t…

This trial tests whether olezarsen, an experimental drug given as a weekly injection, can lower dangerously high blood fat levels in children with familial chylomicronemia syndrome (FCS). FCS is a rare genetic condition that prevents the body from breaking down fats, leading to s…

This trial tests whether an antibody called IC14 can reduce dangerous lung inflammation in people with acute respiratory distress syndrome (ARDS), a severe condition where the lungs fill with fluid. Hospitalized patients on a ventilator receive either IC14 or a placebo, and resea…

This study tests whether a simple isometric wall squat exercise can lower blood pressure in adults with both hypertension and obstructive sleep apnea treated with CPAP. Each participant will complete one session of wall squats and one rest-only session, with blood pressure monito…

This trial investigates whether adding core stability training and intrinsic foot muscle exercises to conventional physical therapy can improve balance, joint position sense, and foot function in people with chronic ankle instability. Eighty adults aged 18 to 44 with a history of…

This phase 2 trial is investigating whether a combination of three drugs—irinotecan, cetuximab, and envafolimab—can control tumor growth when given again to people with metastatic colorectal cancer who had previously responded to similar treatments. The study enrolls adults whose…

This trial studies adults with moderate-to-severe acute respiratory distress syndrome (ARDS) who need ECMO life support. Researchers aim to classify patients into immune-based subtypes, understand why complications like kidney injury and bleeding occur, and develop a personalized…

This study tests whether combining a daily oral diabetes medication (semaglutide) with a nasal insulin spray can improve memory, thinking, and blood flow in the brain. The trial enrolls older adults with mild cognitive impairment and metabolic syndrome, a group at high risk for d…

This trial is testing whether combining two drugs—selumetinib and APG-115—can help people with neurofibromatosis type 1 (NF1) who have nerve sheath tumors that are either pre-cancerous or already cancerous. The study aims to find a safe dose and see if the combination can shrink …

This study investigates whether a standardized, full-course management pathway for ECMO can improve outcomes for adults with moderate-to-severe acute respiratory distress syndrome (ARDS). The pathway covers everything from deciding when to start ECMO through weaning and follow-up…

This phase III trial tests whether vicagrel, an experimental antiplatelet drug, can prevent major heart-related events better than the standard drug clopidogrel in people with acute coronary syndrome (such as a heart attack or unstable angina) who are undergoing a stent procedure…

This early-stage study tests an experimental drug called CND319 in healthy volunteers and people with rheumatoid arthritis or Sjögren's syndrome. The main goal is to check safety and how the body processes the drug. Researchers will also look for early signs that it might help co…

This study tests an in-ear device called WhisperPT that plays soft audio cues when a person sleeps on their back, encouraging them to shift position. It is designed for people with positional obstructive sleep apnea, where breathing problems are worse when lying face-up. The tria…

This phase 3 trial tests whether a combination of two experimental drugs (futibatinib and zimberelimab) plus standard chemotherapy can help people with advanced biliary tract cancer live longer compared to current standard immunotherapy combinations. The study enrolls about 784 a…

This trial investigates whether different doses and durations of the corticosteroid dexamethasone improve outcomes for adults with acute respiratory distress syndrome (ARDS) who are on a ventilator. Participants are randomly assigned to receive either no steroids, a low or medium…

This phase 2 trial tests fimepinostat, a drug that blocks two cancer-related pathways, in adults with Cushing disease caused by a pituitary tumor. The goal is to see if the drug can lower cortisol levels to normal within four weeks. The study compares two doses and includes patie…

This trial investigates whether giving the protein albumin together with standard intravenous fluids during the first hours of treatment can reduce deaths in people with septic shock. About 2,400 adults arriving at emergency departments with septic shock will be randomly assigned…

This study tests whether adding a drug tailored to a person's specific sleep apnea subtype can help when a mouthpiece or tongue nerve stimulator still leaves them with breathing pauses. Participants whose device did not fully control their apnea will receive either a placebo or o…

This phase IIa trial tests whether FT1, a lab-made version of a natural gut hormone, is safe and helps reduce fluid loss in adults with short bowel syndrome. Participants receive weekly injections of FT1 or a placebo for five weeks. The study measures changes in stool weight and …

This phase 1 trial tests the safety and tolerability of pacritinib, a pill that blocks JAK2 and IRAK1, in people with VEXAS syndrome—a rare, severe autoinflammatory disorder caused by a UBA1 gene mutation. Up to 15 participants will take pacritinib daily for up to 12 cycles, star…

This study investigates whether drinking a fiber-rich kodo millet porridge every day for 12 weeks can improve gut bacteria diversity and metabolic health in overweight or obese adults. Participants will consume a standardized porridge beverage while keeping their usual diet and a…

This phase 1 trial tests a new drug called GTB-3650 for adults with high-risk myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) that has come back or not responded to treatment. The drug is designed to connect natural killer cells—a type of immune cell—to cancer cel…

This study tests whether injecting a mixture of hyaluronic acid and I-PRF (a concentrate of healing factors from your own blood) into the jaw joint, after a cleaning procedure called arthrocentesis, can reduce pain and improve jaw movement in people with a displaced disc. Partici…

This study investigates whether a 12-week treadmill walking program can improve metabolic syndrome in women with Behcet disease. Participants walk for 40 minutes three times a week. Researchers measure changes in cholesterol, triglycerides, blood sugar, blood pressure, and waist …

This trial tests whether inclisiran, an injectable medication, can lower LDL cholesterol in patients who have multiple health conditions and take many drugs that interfere with standard statin therapy. Participants must be taking five or more prescription medications and have con…

This study compares delayed cord clamping (waiting at least 30 seconds after birth) with immediate clamping in preterm infants born between 32 and 37 weeks. The goal is to see if the extra time improves early health outcomes such as breathing, anemia risk, and survival. Researche…

This study looks at whether people with rheumatic diseases (like rheumatoid arthritis or lupus) can safely stop their immunosuppressant medications for a shorter time before elective shoulder replacement surgery. The goal is to see if this approach lowers the chance of disease fl…

This study tests whether giving choline supplements to pregnant women who drink heavily can reduce the negative effects of alcohol on their baby's growth and brain development. Choline is an essential nutrient that supports cell membranes and brain function. The trial compares da…

This study tests whether adding resilience training—a mix of group therapy and relaxation exercises—to standard rehabilitation helps people recover from Takotsubo cardiomyopathy (broken heart syndrome) or a recent heart attack. Participants attend two group therapy sessions and t…

This trial investigates whether injecting Botox into the upper throat muscle (cricopharyngeus) can ease swallowing problems in people with conditions like stroke, multiple sclerosis, or Parkinson's. The muscle normally relaxes during swallowing but can become too tight in these d…

This trial studies whether giving anti-seizure medication to comatose patients who have brain seizure activity after cardiac arrest can improve their recovery. Participants are randomly assigned to receive either standard care alone or standard care plus a stepwise anti-seizure t…

This trial compares three cholesterol-lowering treatments—pitavastatin alone, pitavastatin plus ezetimibe, and atorvastatin—to see how they affect blood sugar control in people with heart disease and metabolic syndrome (but not diabetes). The main focus is on insulin resistance a…

This trial tests whether intermittent dietary restrictions—either low-carb or calorie-restricted diets on two days per week—can reverse early signs of heart and metabolic problems in school-aged children aged 7 to 18. Participants have at least one risk factor like prediabetes, a…

This study tests a stem cell transplant using a reduced-intensity conditioning regimen (lower doses of chemotherapy and radiation) followed by a donor stem cell infusion. The goal is to treat various blood cancers and blood disorders while lowering the risk of graft-versus-host d…

This study aims to create a way for mothers with opioid use disorder to receive treatment (buprenorphine) while visiting their babies in the neonatal intensive care unit (NICU). Researchers will interview mothers and healthcare providers to understand barriers and then develop a …

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 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 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 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 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 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 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 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 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 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 trial explores whether a PSMA PET scan can detect changes in glioblastoma, an aggressive brain cancer, after treatment with bevacizumab. About 20 adults with IDH-wildtype glioblastoma will receive PSMA scans before and after bevacizumab therapy. The goal is to see if PSMA ex…

This study explores whether performing a cardiac MRI (CMR) before the standard invasive coronary angiography (ICA) can accurately diagnose the cause of a suspected non-ST-elevation heart attack (NSTEMI). Currently, patients with NSTEMI often undergo an invasive angiogram, and if …

This study aims to improve how doctors diagnose Cushing's syndrome, a condition caused by too much cortisol. Researchers will test the accuracy of the desmopressin stimulation test in 140 people aged 18-70 who have or may have Cushing's, plus healthy volunteers. Participants will…

This Phase I trial is testing an experimental imaging drug, 203Pb-RMX-VH-PIB, to see how it spreads through the body and how much radiation reaches different organs. The study includes adults with glioblastoma (a type of brain cancer) or pancreatic cancer. Participants receive a …

This trial evaluates a new chest patch sensor designed to monitor breathing movements and sleep position, which could improve the diagnosis of sleep apnea. The sensor aims to provide additional information beyond current fingertip sensors, potentially helping to distinguish betwe…

This study investigates whether tiny molecules in the blood, called microRNAs, can serve as biomarkers for obstructive sleep apnea (OSA). Researchers will compare blood samples from people with moderate-to-severe OSA and those without the condition, before and after treatment wit…

This study tests whether a lab technique called polyethylene glycol (PEG) precipitation can tell the difference between a true heart attack and a harmless condition called macrotroponin, where troponin molecules clump together and falsely signal heart damage. Researchers will com…

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 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 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 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 looks at whether a low dose of the antifungal drug liposomal amphotericin B can prevent serious fungal infections in adults with blood cancers or who have had stem cell transplants and have a prolonged low white blood cell count (neutropenia). The trial enrolls 30 pati…

This study looks at whether patients and doctors in a family planning clinic find it acceptable to discuss and start PrEP, a daily pill that prevents HIV. About 83 English- or Spanish-speaking patients aged 16 and older, along with their doctors, will take part. Patients will rec…

This trial tests whether a home-based program, led by a bilingual community health worker, can improve the diet quality of preschool-aged children in low-income Hispanic families. Parents receive coaching visits, phone calls, and text messages over six months, focusing on healthy…

This trial tests whether starting intensive blood pressure medication soon after childbirth can prevent chronic hypertension in women who had preeclampsia. Participants are randomly assigned to receive nifedipine ER when their blood pressure reaches different thresholds during th…

This study compares a new injection of liquid phase concentrated growth factor (LPCGF) to standard arthrocentesis (joint washing) for people with painful jaw clicking and limited mouth opening due to a displaced disc. Thirty-two adults aged 18-45 with unilateral disc displacement…

This European study will test a new type of spinal cord stimulation device in 200 people with persistent back and leg pain after failed back surgery. The device uses a special stimulation pattern to try to reduce pain. Researchers will track pain levels and device settings to see…

This study tests a drug called fezolinetant to see if it can reduce hot flashes in men with prostate cancer who are on hormone therapy (androgen deprivation therapy). About 60 men will take either the drug or a placebo daily for 4 weeks. Researchers will track hot flash frequency…

This Phase 3 trial tests whether fenfluramine (Fintepla) can reduce behavioral and emotional symptoms in people with Rett syndrome. About 200 participants will receive either the drug or a placebo for 14 weeks. The study measures changes in behavior, sleep, and overall condition …

This study tests whether a mobile app that plays guided imagery sessions can help reduce chronic abdominal pain in children aged 7 to 12. Participants listen to 10-15 minute audio sessions five days a week for eight weeks, alongside their usual care. The goal is to see if this ap…

This study tests a disposable silicone device that clips onto eyeglasses to trap moisture around the eyes. Thirty-five adults with mild to moderate dry eye will wear it for two hours. Researchers will measure tear film stability, tear volume, and symptom changes before and after …

This study tests whether Acceptance and Commitment Therapy (ACT), delivered through a smartphone app, can help caregivers of children with RASopathies (like Neurofibromatosis type 1 and Noonan syndrome) cope with parenting stress. The trial is fully remote and involves 70 adult c…

This trial tests whether daily music training—either piano practice or listening to music—can improve behavior and thinking in children with fetal alcohol spectrum disorder (FASD). Each child will do both activities for 12 weeks each, in a random order, with tests before and afte…

This study tests an investigational drug called CSL889 in 70 adults and adolescents with sickle cell disease who are having a painful vaso-occlusive crisis. The goal is to see if the drug can safely shorten the crisis and reduce the need for strong pain medicines. Participants wi…

This study compares two brain-training programs for adults with ME/CFS who experience brain fog. Both programs include computer games and a mild nerve stimulation device, but one focuses on processing speed and daily tasks while the other emphasizes relaxation and healthy habits.…

This Mayo Clinic study tests a smartphone app called SleepWell24 to help people with obstructive sleep apnea use their CPAP machine for at least 6 hours a night, 6 nights a week. The app connects to a smart plug and a Fitbit to track CPAP usage, sleep time, and activity. Research…

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 an online music therapy program that combines mindfulness and music to help people recovering from stem cell transplants for blood cancers. Researchers want to see if it can improve quality of life, reduce pain, fatigue, anxiety, and depression, and possibly spee…

This study tests whether extra support—like personalized mask fitting, monthly phone calls, and a tracking app—helps stroke patients with sleep apnea use their CPAP machine more consistently during rehab. About 250 participants will be randomly assigned to either this intensive s…

This study tests whether a 6-week nurse-led self-management program can reduce dialysis-related distress in people receiving hemodialysis. The program includes weekly one-on-one sessions focusing on fluid control, diet, medication adherence, and coping with symptoms. Half of the …

This trial tests whether a dietary supplement called MILAMEND Hormone Balance can improve quality of life and hormonal health in women aged 18-40 who have PCOS or likely have it. Participants take the supplement or a placebo daily for several months, tracking their menstrual cycl…

This trial tests whether closed kinetic chain exercises or Thrower's Ten exercises better reduce pain, improve mobility, and boost performance in young male bowlers with shoulder instability. Participants will follow a six-week program. The goal is to find which approach offers b…

This trial tests a program called ACT, which trains doctors and nurses to have compassionate, structured conversations with blood cancer patients and their families about end-of-life care. The goal is to help patients understand their prognosis and make informed decisions, potent…

This study tests whether a new eye drop, BUFY01, works as well as an existing one, SVS20, for people with dry eye disease. About 80 participants will use one of the two drops daily for three months. Researchers will check eye health and ask about symptoms to see if the treatments…

This study tests whether closely watching jaw movement and starting physical therapy early can prevent or reduce trismus (jaw tightness) in people receiving radiation for head and neck cancer. Thirty adults will be checked weekly during treatment and given jaw exercises if their …

This study is testing a new way to use an already-approved spinal cord stimulator to treat chronic, hard-to-manage back and leg pain. The device sends mild electrical pulses to the spine, and the new setting (called TVP-SCS) varies the timing of those pulses. Researchers will enr…

This study tests whether a non-invasive brain stimulation technique called rTMS can improve symptoms of major depression and chronic pain at the same time. Fifty-four adults with moderate depression and pain will receive 30 sessions of active rTMS over several weeks. Researchers …

This study tests whether acupressure can reduce coughing and related symptoms like shortness of breath and fatigue in lung cancer survivors. Eighty participants will either perform acupressure for 8 weeks or receive an educational booklet. Researchers will track changes in cough …

This study tests whether adding dry needling to standard physical therapy helps people with jaw pain (TMJ dysfunction). One hundred adults will either get standard rehab alone or rehab plus dry needling. Researchers will measure pain, jaw movement, and quality of life to see if t…

This study compares two methods of giving surfactant, a medicine that helps premature babies breathe. One method uses a thin tube, the other uses a small airway device. The goal is to see which method causes fewer breathing problems and complications in infants born at 29 weeks o…

This study tests an online wellness course that teaches diet, exercise, stress reduction, and self-care to people with rheumatoid arthritis, lupus, Sjogren's syndrome, and similar conditions. Researchers want to see if the program reduces fatigue and improves quality of life. Abo…

This study tests a wearable device worn on the ear that stimulates nearby nerves to change brain signals. The goal is to see if it can reduce chronic pain and ease symptoms of opioid withdrawal. Adults aged 18 to 65 with chronic pain who are tapering off opioid medication will re…

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 trial tests whether drinking hydrogen-rich water for 16 weeks can reduce fatigue and improve physical functioning in people with ME/CFS (chronic fatigue syndrome). Half of the 80 participants will receive the active supplement, while the other half gets a placebo. The study …

This study tests whether taking kimchi powder capsules for 8 weeks can improve gut health in adults with diarrhea-predominant irritable bowel syndrome (IBS-D). Participants will be randomly assigned to receive either kimchi powder or an identical placebo, and researchers will tra…

This study tests an injected drug called brenipatide in 342 adults with irritable bowel syndrome with constipation (IBS-C). Participants will receive either the drug or a placebo for about 35 weeks. The goal is to see if brenipatide safely reduces abdominal pain and improves bowe…

This Phase 2 study tests whether brenipatide, given as an injection under the skin, can safely reduce abdominal pain and improve stool consistency in adults with diarrhea-predominant irritable bowel syndrome (IBS-D). About 531 participants will receive either the drug or a placeb…

This trial tests whether a program built around small, deliberate choices — called micro-choices — can help people with persistent fatigue, including those with long COVID. Participants attend a four-day group workshop and receive three weeks of digital support to build new habit…

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 study tests whether a device that combines hot and cold therapy with gentle compression can reduce pain, swelling, and improve hand function in people with Complex Regional Pain Syndrome (CRPS) after a hand or wrist injury. Half of the participants will use this combined the…

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 trial tests whether a specially designed website, called Mosaic, can help blood cancer patients who are about to undergo a stem cell transplant feel less distressed and have a better quality of life. Participants are randomly assigned to use either the Mosaic website or a st…

This pilot study tests whether listening to music in the evening can help people with Alzheimer's disease or related disorders fall asleep faster and behave more calmly. Researchers will compare music therapy to audiobooks in 48 adults living in secure care units. The goal is to …

This trial tests whether Yangyin Roujin Formula, a Chinese herbal blend, can reduce pain and improve quality of life in people with fibromyalgia. Sixty adults with fibromyalgia will receive either the herbal formula or a placebo for 12 weeks. Researchers will also collect blood, …

This study tests whether a nasal spray called Tyrvaya (varenicline solution 0.03 mg) can improve comfort for people who wear daily disposable contact lenses and experience dryness. Participants use the spray twice a day while keeping their lenses in. The study measures changes in…

This trial investigates whether TRYPTYR, an experimental eye drop, can reduce symptoms and improve eye surface health in adults with dry eye disease. Participants will use the drops twice daily for 30 days. Researchers will measure changes in symptom scores and eye staining patte…

This trial tests whether a therapy that blends Exposure and Response Prevention (ERP) with Habit Reversal Training (HbRT) can reduce both obsessive-compulsive and tic symptoms in adults with Tourettic OCD — a subtype where OCD and tic disorders overlap. Standard treatments usuall…

This trial tests whether sertraline, a widely used antidepressant, can reduce anxiety in children and teens aged 8 to 17 with neurodevelopmental disorders such as autism, ADHD, Fragile X syndrome, and Tourette syndrome. Participants receive either sertraline or a placebo for seve…

This study tests whether a tailored physical activity program can help older adults maintain thinking and memory after a stem cell transplant for blood cancers. Participants aged 60 and older will follow a progressive, light-to-moderate exercise plan with support from therapists …

This trial tests whether a smartphone app called STANZA, which delivers a type of talk therapy known as acceptance and commitment therapy (ACT), can improve daily function and well-being in people with fibromyalgia. Researchers will compare three groups: those using the app plus …

This study investigates whether measuring jaw muscle activity can predict dental changes caused by mandibular advancement devices (MADs) used for obstructive sleep apnea. About half of people stop using MADs due to unwanted tooth movements. Researchers will track 118 adults over …

This study tests whether a 12-week, app-guided exercise program is safe and practical for adults with primary antibody deficiencies—conditions where the immune system struggles to fight infections. Researchers will track how many people join and finish the program, how often they…

This study tests whether a structured behavioral therapy called CBIT can help children and teens aged 10–17 with chronic tics (Tourette syndrome or persistent tic disorder) gain better control over their symptoms. Participants receive 8 weekly CBIT sessions, learning to replace t…

This trial tests whether a 10-session group program called Social Management Training can improve mental health, executive function, and social skills in adults aged 16 to 69 who have sex chromosome aneuploidies (extra or missing sex chromosomes). Participants complete questionna…

This study tests whether music therapy can help newborns who are withdrawing from opioids they were exposed to before birth. Researchers will try six different music-based approaches, including parent-sung lullabies and rhythmic sounds, to see if they calm the babies and improve …

This study tests whether a device that gently stimulates the forehead during sleep can help children aged 8 to 12 who have ADHD linked to prenatal alcohol exposure. The device, already cleared for general pediatric ADHD, is used at home by parents each night for four weeks. Resea…

This study investigates which specific types of FODMAPs (carbohydrates found in many foods) cause abdominal pain in people with irritable bowel syndrome (IBS). Participants follow a low-FODMAP diet and then add back individual FODMAP subtypes to see which ones worsen pain. Stool …

This trial investigates whether diaphragmatic breathing exercises can improve sleep quality in women with polycystic ovarian syndrome (PCOS). Fifty-four women aged 25-35 with PCOS and poor sleep will practice breathing exercises three times a week for eight weeks. The study measu…

This study investigates whether a 12-week low-calorie diet can reduce tinnitus (ringing in the ears) in people who have both Behcet disease and obesity. Twenty participants will follow the diet, and researchers will measure changes in tinnitus severity, discomfort, and quality of…

This study tests whether a short breathing and attention training (BAT) session can improve how the brain handles pain in people with fibromyalgia and in healthy volunteers. Participants will rate painful and non-painful sensations before and after BAT or a control breathing exer…

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 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 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 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 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 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 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 natural history study is observing up to 250 people with Smith-Lemli-Opitz syndrome and related cholesterol disorders, as well as their relatives. Researchers will track symptoms, development, and lab results over several years to find better ways to measure disease progress…

This study aims to understand the genetic causes of brain development disorders, including Aicardi syndrome, polymicrogyria, and Dandy-Walker malformation. Researchers will analyze genetic data from 2000 participants with these conditions, all confirmed by MRI. The goal is to use…

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 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 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 aims to understand the causes and progression of Common Variable Immunodeficiency (CVID) and related inborn errors of immunity. Researchers will observe up to 500 patients over time using blood tests, imaging, and other exams. The goal is to find better ways to diagnos…

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 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 collects tissue and fluid samples from up to 5,000 cancer patients to help researchers learn more about cancer and develop new tests and treatments. Samples are taken from routine procedures or from extra biopsies done just for research. The goal is to build a resource…

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 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 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 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 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 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 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 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 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 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 will collect blood samples from 115 adults with cardiac sarcoidosis to look for tiny particles called exosomes that might show whether the disease is active or quiet. The goal is to develop a simple blood test that could help doctors monitor the condition without needi…

This study aims to build a digital early-warning system that uses machine learning to predict acute lung injury (ALI) in patients undergoing major liver surgery. By analyzing cardiopulmonary interaction characteristics, the model could help doctors identify at-risk patients early…

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 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 is developing questionnaires to measure how visible tumors in neurofibromatosis type 1 (NF1) affect patients' appearance concerns. About 110 participants, including children, adults, and caregivers, will complete surveys and join focus groups or interviews to give feed…

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 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 follows families with a history of inherited kidney cancer to learn more about the disease's genetic causes and how it progresses. Researchers will collect blood, tissue, and urine samples from affected individuals and their relatives. The goal is to identify new genes…

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 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 tests whether briefly increasing the air pressure from a CPAP machine before giving surfactant therapy helps preterm babies' lungs work better. Surfactant is a natural substance that helps immature lungs stay open, and it's often given through a thin tube. The trial wi…

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 registry is following 3,000 adults with idiopathic pulmonary fibrosis (IPF) or other chronic fibrosing interstitial lung diseases (ILDs) to understand how these conditions progress over time. Researchers collect information on symptoms, treatments, quality of life, and store…

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 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 looks at whether simple frailty assessments can predict how older patients (60+) with acute leukemia or MDS will fare after intensive treatments like chemotherapy, CAR T-cell therapy, or stem cell transplants. Researchers will track frailty status and compare outcomes …

This study looks at how gut bacteria and diet affect symptoms in people with irritable bowel syndrome (IBS). Researchers will measure bile acids, short-chain fatty acids, and gut microbes in stool samples from 72 adults with IBS and healthy volunteers after a controlled diet. Par…

This study is for people aged 3 and older with neurofibromatosis type 1 (NF1), a genetic condition that often causes noncancerous nerve tumors. About half of people with NF1 get these tumors, and sometimes they turn cancerous. Researchers want to test a new method to predict whic…

This study looks at whether people with non-celiac wheat sensitivity (NCWS) have a higher chance of having fatty liver or liver scarring. Researchers will compare liver health in people with NCWS to those with irritable bowel syndrome, functional dyspepsia, and celiac disease. Th…

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 follows children with congenital or childhood myotonic dystrophy over time to see how their symptoms change. Researchers will test a range of assessments, from walking speed and hand strength to language and quality of life, to find the best ways to measure the disease…

This study looks at whether a new drug called UCB8600 can stop mast cells in the gut from overreacting, which may cause pain in people with irritable bowel syndrome (IBS). Researchers will take gut tissue from 60 people (some with IBS, some healthy) and treat it with UCB8600 in t…

This study surveys doctors who use the Liberant system to remove fresh blood clots from leg arteries and veins. About 50 patients aged 22 or older will be treated within 72 hours of their procedure. The goal is to confirm the device is safe and works as intended.

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 investigates whether changes in platelet counts over time can serve as a dynamic biomarker for disease severity and survival in people with antiphospholipid syndrome (APS), an autoimmune condition that increases blood clot risk. Researchers will track platelet counts f…

This study looks at whether adding certain nutrients to blood cells from people with septic shock can help restore the cells' energy-making machinery (mitochondria). Researchers will collect blood samples from 55 patients in the ICU and test these nutrients in the lab. The goal i…

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 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 is looking at whether advanced heart imaging, especially cardiac MRI, can help identify the cause of cardiomyopathy and predict risks like death or heart failure in a diverse group of patients. The research focuses on adults in the Bronx, including many from Hispanic a…

This study aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…

This study aims to find out whether some women diagnosed with polycystic ovary syndrome (PCOS) actually have a rare genetic condition causing severe insulin resistance. Researchers will compare 25 women with a known LMNA gene mutation (linked to lipodystrophy) to 50 women with cl…

This study asks whether autoimmune hemolytic anemia (AIHA) is linked to a higher risk of blood clots in people with antiphospholipid syndrome (APS). Researchers will compare the number of thrombotic events in APS patients who have AIHA versus those who do not. The study will enro…

This study is testing whether a special PET imaging tracer, [18F]PI-2620, can detect tau protein buildup in the brains of people with Parkinson's disease, including those with a genetic form linked to the LRRK2 gene. About 60 participants will undergo high-resolution brain scans …

This study creates a registry to collect medical information from people with rhizomelic chondrodysplasia punctata (RCDP) and closely related conditions. The goal is to better understand the natural history of these rare disorders and identify factors that may predict health outc…

This study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …

This study follows people with rare inherited forms of kidney stones and chronic kidney disease, such as primary hyperoxaluria, cystinuria, and Dent disease, to learn how these conditions develop over time. Researchers will measure markers of inflammation in blood and urine and t…

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 aims to find out how often exocrine pancreatic insufficiency (EPI) occurs in people with Alagille syndrome who have had a liver transplant. About 30 participants aged 7 to 50 will eat a high-fat diet and collect stool samples for lab testing. The results will help doct…

This study looks at how a Campylobacter gut infection changes the bacteria and lining of the bowel, and whether these changes can help identify who might later develop irritable bowel syndrome (IBS). Researchers will follow people with a confirmed Campylobacter infection who do n…

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 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 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 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 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 looks at whether successfully treating moderate-to-severe sleep apnea can improve memory and reduce biological markers linked to Alzheimer's disease in older adults. About 200 cognitively healthy people aged 55-85 will either receive immediate sleep apnea treatment or …

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 aims to create a large genetic database of people with Turner syndrome, a rare condition where a person is missing all or part of an X chromosome. Researchers want to compare the genes of women with Turner syndrome who are fertile with those who are not. Up to 500 part…

This study investigates whether measuring the physical effort of breathing in children with acute respiratory distress syndrome (ARDS) can predict how the disease will progress. Researchers will use advanced, non-invasive monitoring tools to track breathing drive, effort, and lun…

This study looks back at medical records of 105 people who had a minimally invasive surgery called BEVAR to treat complex aortic aneurysms. Researchers want to see if two types of stent grafts (TUVA BX and iCover) are safe and work well. No new treatments are given; the study jus…

This study follows 1,000 adults with myotonic dystrophy type 1 (DM1) over time to measure how the disease progresses. Researchers will track walking speed, hand muscle relaxation, grip strength, heart and lung function, and daily activity. The goal is to establish reliable biomar…

This study looks at how people with Down syndrome and Fragile X syndrome decide who to trust. Researchers will use eye-tracking and behavioral tasks to measure how participants read faces, behaviors, and detect deception. The goal is to understand the brain mechanisms behind trus…

This study looks at why some people with Common Variable Immunodeficiency (CVID) develop complications not caused by infections. Researchers will test whether a type of immune cell called peripheral helper T-cells (Tph) helps faulty B-cells produce antibodies that lead to these c…

This pilot study tests whether an educational video can help women with BRCA1 or BRCA2 mutations make more informed decisions about hormone replacement therapy after having their ovaries and fallopian tubes removed to lower cancer risk. Fifty premenopausal women scheduled for thi…

This study follows about 50 members of a family with a rare inherited condition called familial hypereosinophilia, where high levels of a type of white blood cell can damage the heart and nerves. Researchers will track participants over many years with yearly checkups, blood test…

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 looks at whether how well people with obstructive sleep apnea notice and interpret body signals is linked to their breathing, fatigue, shortness of breath, and sleep quality. Researchers will measure body awareness with a questionnaire and compare it with lung function…

This study follows people with SPLIS, a rare inherited metabolic condition, to map its natural history—how the disease unfolds over time. Researchers will track growth, organ function, quality of life, and survival in about 28 participants. The goal is to understand the full rang…

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 looks at brown adipose tissue (BAT), a type of body fat that may help burn energy and improve metabolism. Researchers want to understand how BAT works and how it changes when people take a drug called mirabegron. The study involves 100 adults aged 18-40 with a BMI betw…

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 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 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 study aims to learn how genes and the environment contribute to rare tumors and cancer risk in individuals and families. Researchers will collect medical histories, genetic samples, and health data from up to 500 participants of all ages. No treatment is given; the goal is t…

This study collects tissue samples, medical data, and family histories from over 1,100 people being screened for colorectal or endometrial cancer. Researchers hope to discover new genes and mechanisms that increase cancer risk, especially in hereditary cancer syndromes. The goal …

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 looks at how lying on your left side, right side, or back affects lung function in adults with acute respiratory distress syndrome (ARDS) after abdominal surgery. Researchers will use a special imaging technique called electrical impedance tomography to measure how wel…

This study looks at three blood markers — mean platelet volume (MPV), platelet-to-lymphocyte ratio (PLR), and neutrophil-to-lymphocyte ratio (NLR) — in people with antiphospholipid syndrome (APS), an autoimmune condition that raises the risk of blood clots and pregnancy complicat…

This study follows 1,500 women with mullerian anomalies — differences in the shape or structure of the uterus, cervix, or vagina. Researchers aim to learn how these variations affect fertility, pregnancy, and the success of surgical treatments. The goal is to improve diagnosis an…

This study follows 250 people newly diagnosed with HIV and 161 healthy volunteers over five years to see who develops metabolic fatty liver disease (MASLD). Researchers use annual blood tests, ultrasounds, and FibroScans to measure liver fat and stiffness. The goal is to uncover …

This long-term study follows people with or at genetic risk for frontotemporal lobar degeneration (FTLD), a group of brain disorders that cause changes in behavior, language, and movement. Researchers will use brain scans, memory tests, and blood samples to measure how the diseas…

This observational study aims to measure how common obstructive sleep apnea (OSA) is in community populations, patients with conditions like hypertension or diabetes, and special groups in China. Participants will fill out a screening questionnaire, and some will undergo sleep mo…

This study looks at whether blood pressure changes during a routine exercise test can signal stiff arteries in people with Cardiovascular-Kidney-Metabolic (CKM) syndrome. Researchers will measure blood pressure before and after exercise and use ultrasound to check aortic stiffnes…

This study aims to discover biological markers (biomarkers) that could help doctors diagnose and track neurological autoimmune diseases such as multiple sclerosis, myasthenia gravis, and Guillain-Barre syndrome. Researchers will analyze samples from 50,000 people, including patie…

This study aims to characterize blood-brain barrier disruption in people newly diagnosed with glioblastoma, an aggressive brain cancer. Researchers will use advanced MRI scans, blood tests for barrier and nerve injury markers, and analysis of tumor tissue removed during surgery. …

This study uses MRI scans to compare brain structure, blood flow, and thinking skills in women with and without polycystic ovary syndrome (PCOS). Researchers want to understand whether PCOS affects overall brain health in reproductive-aged women. Participants complete two visits …

This study looks at whether screening adults with leukemia for social needs (like housing or food) and offering early social work help is practical and helpful. About 80 newly diagnosed patients will either get the screening and consultation or usual care. The goal is to see if t…

This observational study follows about 200 people in the United States who have chronic inflammatory demyelinating polyneuropathy (CIDP) and still experience disability despite at least three months of standard treatment. Researchers will track changes in disability scores and ex…

This trial compares two ways of inspecting the ileo-anal pouch during routine surveillance in adults with familial adenomatous polyposis (FAP): dye-based chromoendoscopy (spraying a blue dye) versus virtual chromoendoscopy (using special light filters). The goal is to see which t…

This study investigates whether certain proteins and other substances in the blood can help identify and track obstructive sleep apnea (OSA), a common disorder linked to heart and metabolic problems. Researchers will compare blood samples from 120 adults—some with moderate-to-sev…

This international online study collects medical, behavioral, and developmental information from people with rare genetic changes that are linked to autism and other neurodevelopmental disorders. By partnering with families, researchers aim to build a detailed database to improve…

This study investigates whether gut bacteria and the chemicals they produce influence inflammation in people with autoinflammatory diseases such as FMF, CAPS, and MKD. Researchers will analyze stool and blood samples from 300 patients to measure gut-derived metabolites and assess…

This study compares the prevalence of the HLA B leader gene in 658 people with psoriatic arthritis and a healthy control group. Researchers will also look at the related HLA B27 gene. The goal is to see whether this genetic marker, already known to affect outcomes in bone marrow …

This study creates a biobank of blood, urine, and tissue samples from people with rare kidney stone diseases—primary hyperoxaluria, cystinuria, APRT deficiency, and Dent disease—and their family members. By storing these samples, researchers hope to enable future studies that cou…

This study tracks the long-term safety and effectiveness of the growth hormone treatment Growtropin-II in children with short stature. It includes up to 5,000 children with growth hormone deficiency, idiopathic short stature, Turner syndrome, or those born small for gestational a…

This study investigates post-exertional malaise (PEM), the hallmark symptom of ME/CFS, where even minor activity triggers severe symptom worsening. Researchers will compare 50 ME/CFS patients with 50 healthy volunteers matched by age, sex, and activity level. Participants undergo…

This study tests whether short-chain fatty acids (SCFAs) — substances naturally produced by gut bacteria — can reduce inflammation and protect thinking skills. Healthy adults receive either SCFA capsules or a placebo, then a low dose of a bacterial substance that briefly activate…

This study aims to create a prediction tool for people with emphysema caused by alpha-1-antitrypsin deficiency, a rare genetic disorder. Researchers will analyze clinical data, lab results, and lung CT scans from 230 patients to identify those at highest risk of death or needing …

This study tracks how tic symptoms change in 30 youth aged 12-17 with Tourette syndrome or chronic tic disorders. Participants receive 8 sessions of behavioral therapy (CBIT) and complete daily surveys, wear a device to track physiology, and perform weekly tasks. The goal is to i…

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

This research study collects and analyzes blood and bone marrow samples from children and young adults with leukemia, myelodysplastic syndrome, or myeloproliferative syndrome. By reading the genetic instructions in these cancer cells, scientists hope to understand why the disease…

This study investigates what happens when exclusive e-cigarette users stop vaping, focusing on withdrawal symptoms like cravings, mood changes, and sleep disruption. Researchers will give participants either a nicotine patch or a placebo patch to see how nicotine affects these sy…

This study uses advanced PET scans and blood tests to examine brain inflammation and white matter damage in people with corticobasal syndrome, a rare movement and thinking disorder. Researchers will compare these brain changes across different subtypes of the condition and agains…

This study investigates whether proteins released by muscles, called myokines, can serve as early warning signs or even causes of muscle loss (sarcopenia) in people with metabolic or endocrine conditions like diabetes, obesity, and Cushing syndrome. Researchers will measure myoki…

This study aims to find the best dose of ciprofol, a new anesthetic similar to propofol but with fewer side effects, for placing a laryngeal mask airway (a type of breathing tube) in children aged 1 to 12. Children undergoing short surgeries will receive ciprofol along with a sta…

This study investigates why some patients with septic shock develop a weakened immune system after the initial crisis, making them prone to further infections. Researchers will analyze blood samples from 305 intensive care unit patients to find biological markers that indicate im…

This study looks at how common fatty liver disease is in people with Cushing's syndrome, a rare condition caused by too much cortisol. Using MRI and FibroScan, researchers will measure liver fat at diagnosis and again one year after cortisol levels return to normal. The goal is t…

This study collects medical information from people around the world who have one of four rare hereditary kidney stone diseases: primary hyperoxaluria, Dent disease, cystinuria, or APRT deficiency. By gathering data from many patients, researchers hope to better understand how th…

This study investigates whether exposure to certain chemicals found in the environment, home, or workplace can increase the risk of developing paraganglioma tumors in people who carry an inherited SDHx gene mutation. Researchers will compare chemical exposure histories between mu…

This study aims to better understand VEXAS syndrome and other autoinflammatory conditions by analyzing participants' DNA from blood, saliva, or skin samples. Researchers hope to discover new genetic variants that cause or contribute to these diseases. The study enrolls people of …

This study tests whether advanced monitoring of breathing patterns can help prevent lung damage in intensive care unit (ICU) patients who need a ventilator for reasons other than lung failure, such as coma or sepsis. Researchers will use special sensors to track airway pressure, …

This study looks at whether measuring certain immune and inflammatory markers in the blood can help predict how critically ill patients will fare. Researchers are following 200 adults in intensive care who have infections, have had major surgery, or have weakened immune systems. …

This study looks at how well current tests, including a simple breath test called nasal nitric oxide (nNO) testing, can diagnose Primary Ciliary Dyskinesia (PCD), a rare genetic condition that affects the lungs and sinuses. Researchers will collect and analyze data from people ov…

This study follows adults aged 18 and older who have a specific type of acute lymphoblastic leukemia (Ph+ ALL) and are receiving a combination of targeted drugs (BCR::ABL1 inhibitors) along with chemotherapy, steroids, or antibody therapy. Researchers will track how long patients…

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 …