Metabolic disease

This trial tests an experimental therapy called PM577a, which uses prime editing to correct a common genetic mutation that causes Wilson disease. The treatment is given as a single intravenous infusion and aims to restore the liver's ability to remove excess copper. The study inc…

This early trial tests a gene therapy called NGGT006 in people with homozygous familial hypercholesterolemia, a rare inherited condition causing extremely high LDL cholesterol and early heart disease. The therapy uses a harmless virus to deliver a working copy of the LDL receptor…

This study looks at people with early-stage gastrointestinal cancers that have a mismatch repair deficiency, a feature that can make tumors respond well to immunotherapy. Part A is a registry that tracks all such patients, whether they have surgery or immunotherapy. Part B follow…

Researchers at Sheba Medical Center are studying a tele-diabetes remote monitoring system for children and teens with type 1 diabetes. The system analyzes real-time glucose data from continuous monitors and sends automated WhatsApp alerts when it detects high or low patterns. The…

This phase 3 trial tests whether an experimental drug called MK-8748 can treat diabetic macular edema (DME), a condition where fluid builds up in the retina and blurs vision. About 960 adults with DME will receive either MK-8748 or the standard treatment aflibercept via eye injec…

This study tests a drug called veverimer in 150 adults with moderate-to-severe chronic kidney disease (CKD) and a condition called metabolic acidosis, where acid builds up in the blood. The goal is to see if the drug can raise blood bicarbonate levels and improve physical functio…

This study looks at the long-term safety of inclisiran, a cholesterol-lowering injection, in children with a genetic condition that causes very high cholesterol. About 195 kids who completed earlier inclisiran studies will receive the drug for an extended period. Researchers will…

Researchers at the University of South Carolina are testing a program that pairs nurses with community health workers to coach adults who have chronic conditions like diabetes, high blood pressure, heart disease, kidney disease, or a prior stroke. Each patient joins with a carepa…

This study tests an oral drug called nizubaglustat in children and teens aged 4 and older with Niemann-Pick type C disease, a rare genetic disorder that affects movement and thinking. Participants will take the drug or a placebo for 18 months to see if it slows the disease and im…

This early-phase trial will test whether a special form of resveratrol (JOTROL) is safe and tolerable for people with Parkinson's disease. About 30 participants will receive either a low dose, high dose, or placebo daily for three months. The study will also measure how the drug …

This early-stage trial tests a new CRISPR-based treatment called CTX310 for people with severe cholesterol and triglyceride problems that don't improve with standard therapies. The treatment uses a tiny fat bubble to deliver gene-editing tools that turn off a gene linked to high …

Researchers compare empagliflozin and dapagliflozin, two similar diabetes drugs, to see which works better at preventing death, heart problems, and kidney damage in adults with type 2 diabetes, heart failure, or chronic kidney disease. The trial uses electronic health records to …

This study tests two ways to treat proliferative diabetic retinopathy, a serious eye problem caused by diabetes. One group gets eye injections of faricimab plus laser treatment; the other gets vitrectomy surgery with laser. Researchers will track vision and how many extra treatme…

This trial tests an investigational medicine called ATR 1072 in adults with PRKAG2 syndrome, a genetic condition that causes abnormal heart muscle thickening and rhythm problems. The drug is designed to reduce the activity of the faulty PRKAG2 gene. Researchers are evaluating its…

Researchers are testing whether a home-use pulsed electromagnetic therapy device can slow cognitive decline in people with mild to moderate Alzheimer's disease, Lewy body dementia, or vascular dementia. About 48 participants will use the device three times a day for 15 minutes ov…

This study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare condition that causes muscle weakness and heart problems. Participants will receive either the drug or a placebo for 72 weeks. The main goal is to see if the drug…

This study tests whether taking a daily probiotic (Lab4P) for 12 weeks can improve memory, thinking speed, and blood sugar control in overweight adults aged 18-40 with prediabetes (mildly high blood sugar). Seventy participants will be randomly assigned to receive either the prob…

Researchers are testing whether measuring phenylalanine at home once a week, rather than once a month, helps adults with classic phenylketonuria keep their levels in a healthier range. The trial enrolls 36 adults aged 18 to 65 who follow a low-protein diet and use special amino a…

Researchers are testing whether adding bempedoic acid to high-intensity statin and ezetimibe therapy within 72 hours after a heart procedure helps adults with acute coronary syndrome reach very low LDL cholesterol. Participants receive either the three-drug combination or the sta…

This study tests a one-time gene therapy called surabgene lomparvovec (Sura-vec) for diabetic retinopathy, a common eye problem from diabetes that can lead to blindness. About 576 adults with moderate-to-severe non-proliferative diabetic retinopathy will receive either the gene t…

Researchers are comparing two sequences of treatment for metastatic castration-resistant prostate cancer that carries certain DNA repair gene mutations. Participants receive either carboplatin first and olaparib second, or olaparib first and carboplatin second, switching after th…

Researchers are testing a combination of two drugs, doxecitine and doxribtimine, in adults with thymidine kinase 2 (TK2) deficiency, a rare genetic condition that causes muscle weakness and breathing problems. The study will enroll about 15 adults with moderate to severe disease.…

Researchers are testing a 12-week online program that teaches adults with type 2 diabetes and excess weight to follow a low-fat vegan diet. Participants meet weekly with a physician and a registered dietitian for education and support. The team measures body composition, blood su…

This phase 3 trial asks whether survodutide, an investigational weekly injection, can help adults with type 2 diabetes lower their blood sugar more than a placebo. Around 600 participants, who also follow diet and exercise or take standard diabetes medications (including insulin)…

This study is testing the long-term safety and effectiveness of two experimental drugs, pozelimab and cemdisiran, taken together for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 202 participants who completed a prior study will continue treatment to see…

This study tests whether a 6-week self-management program, done with a caregiver's help, can improve diabetes control and thinking skills in adults aged 55+ who have both diabetes and mild cognitive impairment. About 309 participants will attend weekly group sessions and provide …

This large phase 3 trial is testing whether adding vicadrostat to the existing drug empagliflozin can reduce the risk of heart problems in adults with type 2 diabetes, high blood pressure, and heart disease. About 11,800 participants will take either the combination or a placebo …

This study is for people who completed a previous trial of KL1333 (napazimone) for primary mitochondrial disease. It will test the drug's long-term safety and whether it helps with fatigue and daily activities. About 140 participants will take the pill twice daily for an extended…

This trial tests whether a single daily pill that combines treatments for high blood pressure, high cholesterol, and type 2 diabetes can lower the risk of heart disease and dementia. The study involves over 8,000 adults aged 50 to 75 in China who have all three conditions. Partic…

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 Phase 3 trial tests an oral drug called nizubaglustat (AZ-3102) in people aged 4 and older with Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. The study lasts 18 months and compares the drug to a placebo to see if it is safe and can slow disease prog…

This study tests a drug called crinecerfont in 20 children aged 3 months to 4 years with classic congenital adrenal hyperplasia (CAH). The goal is to see if the drug is safe and tolerable. Participants must already be on stable hydrocortisone treatment. The study is not yet recru…

Researchers are testing a new once-weekly injection called NNC0487-0111 in about 400 Japanese adults with overweight or obesity. Half the participants receive the drug and half receive a placebo, assigned by chance. The main goal is to measure how much body weight changes over th…

This phase 2 trial tests an experimental drug called vamifeport in 84 adults with HFE-related hereditary hemochromatosis, a genetic condition causing iron buildup. The drug aims to reduce liver iron levels, potentially offering an alternative to regular blood removal. Participant…

This trial is testing an experimental once-daily pill called ASC30 in adults who are overweight or have obesity and also have type 2 diabetes. The study aims to see if ASC30 can help with long-term weight loss and improve blood sugar control compared to a placebo. Participants wi…

This study tests a single dose of a new drug, ALN-4324, to see if it improves how the body uses insulin in people with type 2 diabetes. About 40 adults who are on a stable dose of metformin will take part. The main goal is to measure changes in insulin sensitivity using a glucose…

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

This phase II trial is testing whether a combination of venetoclax and rituximab works better than current standard treatments (ibrutinib plus rituximab or zanubrutinib alone) for people with previously untreated Waldenstrom's macroglobulinemia, a rare type of blood cancer. The s…

Researchers are testing whether the drug daratumumab can treat kidney diseases caused by monoclonal gammopathy of renal significance (MGRS), a group of conditions where abnormal antibody proteins damage the kidneys. The trial enrolls 30 adults with biopsy-confirmed MGRS. Particip…

This study is testing a new approach for people with obesity and type 2 diabetes. It combines two procedures done through the mouth: one uses mild electric pulses to regenerate the lining of the upper intestine, and the other reduces stomach size by stitching it like a sleeve. Th…

Diabetic nephropathy is a common kidney complication of diabetes that current drugs can only slow, not reverse. Researchers are testing human umbilical cord mesenchymal stem cells, given by injection at increasing doses, to see if they are safe and can reduce inflammation and rep…

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

Cystinosis is a rare inherited disease that causes kidney failure and poor growth due to cystine buildup in cells. This study follows 330 patients taking cysteamine, a drug that lowers cystine levels, to track long-term effects, detect new complications, and support genetic resea…

This Phase 3 study tests a drug called nucresiran in 1250 people with ATTR amyloidosis, a rare disease where abnormal proteins damage the heart. Participants receive either nucresiran or a placebo every 6 months. The goal is to see if the drug reduces deaths and heart-related hos…

This study tests whether tirzepatide, a drug used for type 2 diabetes, can help people with type 1 diabetes. Researchers will measure how the body responds to insulin and meals before and after 6 weeks of treatment. 44 adults with type 1 diabetes who use an automated insulin pump…

This study is testing a milder type of stem cell transplant for people with non-cancerous blood, immune, or metabolic disorders. The goal is to safely get donor cells to grow in the patient's body while reducing harsh side effects. About 220 participants will receive a reduced-in…

This study tests an oral drug called NEO212 in people with specific brain tumors (astrocytoma, glioblastoma) or cancers that have spread to the brain. The goal is to find the safest dose and see if it helps control tumor growth. About 134 adults will take NEO212 alone or with sta…

This study tests whether a drug called AMX0035 can improve how the body responds to insulin in adults with type 1 diabetes. Participants take either AMX0035 or a placebo daily for 24 weeks. Researchers measure insulin sensitivity using a glucose clamp test and track blood sugar c…

This study tests a new medication, CX11, for people with type 2 diabetes who need better blood sugar control despite taking metformin. About 240 adults will receive different doses of CX11 or a placebo for 24 weeks to see if it safely lowers blood sugar. The goal is to find the b…

This Phase 3 trial tests a new injection called UBT251 in 360 adults with type 2 diabetes who have not been able to control their blood sugar with diet and exercise alone. Participants will receive either a weekly shot of UBT251 (at different doses) or a placebo for several weeks…

After a liver transplant, patients take tacrolimus to stop their immune system from rejecting the new organ, but the drug can cause kidney problems, high blood pressure, diabetes, and other side effects. Researchers at the University of Alberta are comparing a once-daily extended…

This study tests two ways to help Chinese immigrants with type 2 diabetes control their blood sugar: watching educational videos, or videos plus a community health worker. 540 adults in NYC will join. The goal is to see if these tools lower HbA1c levels and boost confidence in ma…

This phase 3 trial is testing whether adding baxdrostat to the standard drug dapagliflozin can better prevent heart failure and cardiovascular death in people with type 2 diabetes who already have heart disease and high blood pressure. About 11,300 participants will receive eithe…

This pilot study tests a temporary, flexible tube called a duodenal-jejunal bypass sleeve. It is placed in the upper intestine using an endoscope and left for 3 months to help with weight loss and improve metabolic syndrome. The study involves 20 adults with obesity or excess wei…

This Phase 3 trial tests a new drug called nucresiran in 125 people with a rare inherited condition that damages nerves (hATTR-PN). The drug is given as a shot under the skin every 6 months and aims to slow nerve damage and improve quality of life. Researchers will compare it to …

This study tests a procedure called pulsENDO that uses mild electric pulses to regenerate the lining of the duodenum (the first part of the small intestine). The goal is to improve blood sugar control and reduce fat buildup in the liver for people with type 2 diabetes and fatty l…

This early-stage trial tests a single injection of UBT38006 in 43 healthy adult men to see if it is safe and how the body processes it. Participants receive either the drug, a placebo, or an active comparator (insulin degludec). The goal is to gather safety data and measure drug …

This study tests whether injecting sodium thiosulfate directly into abnormal calcium deposits can shrink them and reduce pain. It includes people with three rare conditions: systemic sclerosis, dermatomyositis, and a genetic disorder called iPPSD2. Participants receive up to 11 i…

Researchers are testing an oral drug called PORT-77 in people with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), rare conditions that cause severe pain after sunlight exposure. The trial enrolls about 225 adults and adolescents aged 12 and older. It compa…

This study tests a procedure called pulsENDO that uses mild electric pulses to regenerate the lining of the first part of the small intestine (duodenum) in people recently diagnosed with type 2 diabetes. The goal is to see if this can help control blood sugar levels without needi…

This study follows 500 adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive heart condition caused by abnormal protein buildup. Participants take acoramidis as part of their usual care, and researchers collect data from hospitals and a European registry. The …

This Phase 3 trial is testing a new weekly injection called enicepatide for weight loss in 1600 people who are obese or overweight and also have type 2 diabetes. The drug works by mimicking natural hormones that help control appetite and blood sugar. Participants will receive eit…

This study tests an oral medication called remibrutinib for adults with moderate-to-severe papulopustular rosacea, a skin condition causing red bumps and pustules on the face. The drug works by blocking a protein involved in inflammation. Researchers will measure whether it reduc…

This phase 3 study compares an experimental weekly injection called UBT251 to the approved drug semaglutide in adults with type 2 diabetes whose blood sugar is not well controlled by oral medications like metformin. The main goal is to see if UBT251 is at least as effective as se…

This study tests a drug called teplizumab in people aged 1 to 25 who were recently diagnosed with type 1 diabetes. The goal is to see if teplizumab can help control blood sugar and reduce the need for insulin at mealtimes. Participants will receive either teplizumab or a placebo …

This early-stage trial tests an experimental drug called RO7663498, given as an injection into the eye, for people with diabetic retinopathy. The main goal is to check safety and how the drug moves through the body. About 30 participants will receive multiple doses to see if it i…

Inavolisib treats certain PIK3CA-mutated, HR-positive, HER2-negative breast cancers but can raise blood sugar. Researchers at Tianjin Medical University Cancer Institute and Hospital are studying whether matching blood sugar monitoring and preventive metformin to each patient's r…

This study tests a daily pill called enlicitide decanoate in 153 children and teens with heterozygous familial hypercholesterolemia (HeFH), a genetic condition causing very high cholesterol. The goal is to see if the drug safely lowers LDL cholesterol more than a placebo. Researc…

This study is testing the long-term safety of an oral drug called Emrusolmin (TEV-56286) in 200 adults with multiple system atrophy (MSA), a rare and serious brain disorder. Participants who completed a prior trial will take the drug for about 100 weeks. The main goal is to see h…

This pilot study looks at whether closely monitoring and managing blood sugar levels can help people with pancreatic cancer. About 50 participants will wear a continuous glucose monitor during chemotherapy. Half will get intensive glucose management from an endocrinologist, while…

Researchers are testing whether a daily probiotic supplement can improve blood sugar, blood pressure, and body composition in people over 65 who have prediabetes or type 2 diabetes and known heart disease. Participants take one probiotic capsule each morning for 60 days, while a …

This early-stage trial is testing an experimental drug called LP-168, taken as a pill, in adults with B-cell lymphoma that has come back or not responded to standard treatments. The study aims to find the safest dose and check for side effects in about 200 participants. It is too…

This Phase 2 trial tests an experimental drug called LY3457263 in 240 adults with type 2 diabetes who still have high blood sugar despite taking stable doses of semaglutide or tirzepatide. Participants receive weekly injections of either the new drug or a placebo for about 9 mont…

This study tests a new medicine called UBT251, given as a weekly shot, in 300 adults with type 2 diabetes. Some participants will get UBT251, others will get a placebo or an existing drug (semaglutide). The goal is to see if UBT251 can safely lower blood sugar and help with weigh…

This Phase 2 trial tests two experimental drugs, ALN-ANG3 and evinacumab, in 270 people with diabetic kidney disease. The drugs block a protein called ANGPTL3 to lower blood fats. Researchers will measure changes in urine protein and kidney function over time to see if the drugs …

This early-stage trial tests an experimental drug called RO7823653, given as an eye injection alone or with an approved drug (faricimab), for people with diabetic macular edema—a condition where diabetes causes fluid buildup and vision loss in the center of the eye. The study wil…

This study tests a new drug, CNP-103, in people aged 12 to 35 who were diagnosed with stage 3 type 1 diabetes within the last 6 months. The goal is to see if the drug is safe and can help control the disease. Participants will receive either the drug or a placebo, and researchers…

This study tests a gene therapy for GM1 gangliosidosis, a fatal disorder that destroys nerve cells. The therapy uses a harmless virus to deliver a working gene, helping the body produce a missing enzyme. Up to 54 children with Type I (ages 6-12 months) or Type II (ages 1-12 years…

This study tests a new drug called mibavademab for people with generalized lipodystrophy, a rare condition where the body lacks fat tissue. The goal is to see if the drug can improve blood sugar, lower blood fats, and reduce liver fat. About 28 children and adults will take part …

This phase 3 study tests aleniglipron, an oral medication taken once daily, for helping adults with obesity or overweight and type 2 diabetes manage their weight over the long term. Participants receive either aleniglipron or a placebo for 76 weeks. The main goal is to see how mu…

This study tests a new oral drug, BGB-16673, designed to destroy a protein called BTK that helps certain blood cancers grow. The trial includes about 645 people with various B-cell cancers (like lymphoma and leukemia) that have come back or not responded to prior treatments. The …

This study tests a new once-weekly injection called NNC0662-0419 in 270 adults with type 2 diabetes. It compares the drug against a placebo and an approved medication, semaglutide, to see how well it controls blood sugar and promotes weight loss. The trial is currently recruiting…

This study looks at whether changing the types of carbohydrates people with type 1 diabetes eat can improve blood sugar control. Researchers will compare a diet with more whole grains and beans to one with mostly refined grains. 80 adults will be monitored to see if the diverse c…

This trial investigates whether ECC4703, a potential new treatment for non-alcoholic fatty liver disease (NAFLD), changes how the body processes two other drugs: sulfasalazine and pitavastatin. Healthy adults aged 18 to 65 will take these drugs alone and then with ECC4703 to meas…

This phase 3 trial is testing a once-weekly injection called enicepatide in 300 Chinese adults who are obese or overweight, with or without type 2 diabetes. The drug works by mimicking two gut hormones to reduce appetite and improve blood sugar control. Participants will receive …

This study tests whether switching from tafamidis to acoramidis can raise blood levels of transthyretin (TTR) in adults with transthyretin amyloid cardiomyopathy (ATTR-CM). The drug acoramidis stabilizes TTR to prevent harmful amyloid clumps that stiffen the heart. Researchers wi…

This study tests a next-generation automated insulin delivery system (Omnipod M) in up to 40 adults with type 1 diabetes. The goal is to see if the system safely improves blood sugar control by reducing dangerously low and high levels. Participants will use the device during hote…

Researchers are testing whether adding fingolimod to standard diabetes care can improve blood sugar control in adults with type 2 diabetes. The trial enrolls 40 people aged 18 to 70 whose blood sugar is not fully controlled on no medication or one oral drug. Half receive fingolim…

This study looks at whether people who take GLP-1 receptor agonists (like Ozempic or Wegovy) should continue them after having bariatric surgery. Researchers will compare weight loss, blood sugar control, gut health, and hunger in 150 adults who either keep taking the drug or sto…

This study tests a new pill that combines two drugs (laroprovstat and rosuvastatin) to lower LDL or 'bad' cholesterol. About 600 adults with high cholesterol who have never taken cholesterol-lowering medication will receive either the combination, one of the drugs alone, or a pla…

This phase 3 trial is testing whether an experimental drug called AZD6234, given as a weekly injection, can help adults with obesity or overweight lose more weight when added to their current incretin-based therapy. The study includes about 500 participants who either have type 2…

This study tests whether empagliflozin, a diabetes medicine, can reduce protein leakage in urine and help protect the kidneys in people with very advanced chronic kidney disease (eGFR between 10 and 20). About 34 adults with type 2 diabetes will take either empagliflozin or a pla…

This study tests an experimental drug called RBD5044 in 120 adults with mixed dyslipidemia, a condition where both cholesterol and triglycerides are high. Participants receive either the drug or a placebo twice over 12 months to see if it safely lowers triglyceride levels. The go…

This phase 3 trial tests whether the experimental drug SHR-1918 can safely lower LDL cholesterol and triglycerides in 900 adults with hyperlipidemia who are already on statins. Participants are randomly assigned to receive SHR-1918 or a placebo. The study aims to see if the drug …

This trial investigates an experimental drug called GUB-UCN2 for people with obesity and type 2 diabetes. The study includes healthy volunteers and those with the conditions, testing the drug alone or alongside standard care. The main goal is to check safety and how the drug beha…

This study looks at whether eating cottonseed oil just three times a week can help lower cholesterol and reduce heart disease risk. Researchers will give 75 overweight or at-risk adults meal replacement shakes with different amounts of cottonseed oil for 56 days. They will measur…

This study tests whether montelukast, a medicine used for asthma, can help protect the kidneys and blood vessels in 50 teens and young adults (ages 14-24) with type 2 diabetes. The drug works by blocking harmful inflammatory chemicals linked to diabetes complications. Researchers…

This study compares two telehealth approaches—digital health coaching and remote patient monitoring—to see which works better for managing type 2 diabetes in people with physical disabilities and at least one other chronic condition. About 5,000 participants will be randomly assi…

This early-phase trial tests a drug called ALT001, a nerve repair protein, in 60 people with multiple system atrophy (MSA), a rare and progressive brain disorder. The study is open-label, meaning everyone knows they are getting the drug, and it focuses on safety and whether the d…

This study tests a new eye injection called KIO-104 for people with macular edema, a condition where fluid builds up in the eye and blurs vision. About 28 adults will receive multiple doses to see if the drug is safe and helps reduce swelling. The goal is to find a steroid-free o…

Researchers are testing an experimental oral drug called MWN109 in adults with type 2 diabetes. The trial compares several doses of MWN109 against a placebo and against semaglutide tablets, a standard treatment, to see which works better at lowering blood sugar over 20 weeks. Abo…

This phase 3 study tests a new oral drug, elecoglipron, against the approved pill semaglutide in adults with type 2 diabetes who also have increased heart risk. The goal is to see which works better for lowering blood sugar and body weight over about a year. Participants take one…

This study tests whether the drug tirzepatide, given once weekly, helps adults with obesity (but without diabetes) lose weight and avoid developing type 2 diabetes. About 3,000 participants will receive either tirzepatide or standard care for up to 5 years. The goal is to see if …

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

This study tests whether a proactive, pharmacist-led telephone program can help people who have had a heart attack reach safer cholesterol levels. Participants receive pharmacist calls, repeat blood tests, and medication adjustments based on national guidelines. The goal is to se…

This early-stage study tests a new drug, ALT001, in 20 people with a rare brain disease called multiple system atrophy-cerebellar type (MSA-C). The main goal is to check if the drug is safe and tolerable. Researchers will also look for early signs that it might help control the d…

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

Researchers are testing whether low-dose interleukin-2 (IL-2) can help people recently diagnosed with type 1 diabetes preserve their own insulin production. The trial enrolls 24 people aged 16 to 45 who still make some insulin. Participants first receive a two-month course of cyc…

This study tests a once-weekly injection called CagriSema (a mix of cagrilintide and semaglutide) in 80 children and adolescents with type 2 diabetes. The goal is to see if it lowers blood sugar and body weight better than a placebo. The trial lasts about 15 months, with everyone…

This study compares two drugs, ATG and teplizumab, to see if they can delay or prevent type 1 diabetes in people who are at high risk. About 60 participants aged 4 to 35 will receive either two infusions of ATG or 14 daily infusions of teplizumab. Researchers will monitor them fo…

This phase 3 trial is testing a new daily pill called elecoglipron for weight loss in 4500 adults who are obese or overweight, with or without type 2 diabetes. Participants will take the pill or a placebo for 72 weeks, alongside diet and exercise. The main goal is to see how much…

This study follows about 1,875 people aged 6 and older with type 1 diabetes who use the iLet Bionic Pancreas, an automated insulin delivery system. Over 12 months, researchers will track serious events like severe low blood sugar and diabetic ketoacidosis to see how the device pe…

This study is testing whether a continuous glucose monitor (CGM) can help older adults with type 2 diabetes who do not use insulin achieve better blood sugar control. Participants will either wear a CGM every two months or use standard finger-stick monitoring for six months. The …

This study tests an experimental drug, YOLT-203, in 36 children and adults with primary hyperoxaluria type 1 (PH1), a rare genetic condition that causes painful kidney stones and kidney damage. The goal is to see if a single dose can safely lower oxalate levels in urine and blood…

This early-phase trial is testing a new type of CAR T cell therapy called Azer-cel, which is made from healthy donor cells rather than the patient's own cells. It aims to treat adults with relapsed or refractory non-Hodgkin lymphoma, B-cell acute lymphoblastic leukemia, and relat…

This study tests a new weekly injection called eloralintide in 900 adults with obesity or overweight who are already taking a weekly incretin drug (like Ozempic or Wegovy) but still have excess weight. Participants will receive either eloralintide or a placebo for about 80 weeks …

This phase 2 trial tests an experimental injection called MWX203, given alone or with another drug called inclisiran, in people with mixed dyslipidemia whose blood fat levels stay high despite statin therapy. The study enrolls 216 adults in China who receive the injection under t…

This study tests a new once-weekly medicine called eloralintide to help people with obesity or overweight and type 2 diabetes lose weight. About 1035 adults will receive either the drug or a placebo for around 75 weeks. Researchers will measure changes in body weight, fat, waist …

This study tests a once-daily pill called orforglipron in adults with type 2 diabetes who plan to fast during Ramadan. The goal is to see if the drug helps control blood sugar and weight without causing harm. About 130 participants will take the pill for up to 48 weeks, with clin…

This study tests two experimental drugs, macupatide and eloralintide, alone or together, to help adults with obesity or overweight and type 2 diabetes lose weight. About 200 participants will receive the drugs or a placebo for 48 weeks. The main goal is to see how much weight the…

This study tests an injectable drug called solbinsiran in 60 adults with severe hypertriglyceridemia (very high blood fats). Participants receive two shots of either the drug or a placebo. The goal is to see if solbinsiran safely lowers triglycerides and related fats better than …

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

This early-stage trial tests an investigational drug called MWN105, given as a weekly injection for 8 weeks, in Chinese adults who are overweight or obese but do not have diabetes. The study aims to see if the drug is safe and tolerable, and how it moves through the body. Partici…

This early-stage trial tests a new drug called LY4152199 in about 215 adults with certain types of blood cancer (like follicular lymphoma or diffuse large B-cell lymphoma) that have already been treated. The drug is designed to help the body's immune cells attack the cancer. The …

This phase 2 trial tests whether donanemab, an antibody given by IV, can slow worsening of thinking and daily function in people with early cognitive decline and Lewy body dementia. The study enrolls 350 participants who also have signs of two brain proteins (amyloid and alpha-sy…

This study tests whether a four-week education program can help adults with type 1 diabetes dose mealtime insulin using continuous glucose monitor (CGM) data instead of traditional carbohydrate counting. Participants are randomly assigned to either the C-Carbs program or usual ca…

This Phase 2 trial tests a drug called rezpegaldesleukin in people recently diagnosed with type 1 diabetes (within 100 days). The goal is to see if it can help the body keep producing some insulin, reducing the need for injected insulin. 66 participants will receive either the dr…

This study looks at whether SGLT2 inhibitors, a class of diabetes and kidney drugs, can improve blood flow through the heart's small arteries in women with HIV who also have diabetes or chronic kidney disease. Eighty women aged 45-75 will be randomly assigned to health education …

This study tests a new drug called AZD4956, alone or with other cancer drugs, in people with advanced or spreading solid tumors that have a specific DNA repair defect (HRR deficiency). The main goals are to check safety, find the best dose, and see if the drug can shrink tumors. …

This study tests whether a structured, goal-directed care plan can help people with diabetic kidney disease better manage their kidney and heart health. About 100 adults with type 2 diabetes and kidney disease will either receive this focused care or standard treatment. The goal …

This study tests a once-monthly injection called berobenatide to help adults with overweight or obesity lose weight. About 954 participants will receive either the drug or a placebo for 20 months. The goal is to see if the drug is safe and effective for weight loss, including in …

This study tests whether giving sodium bicarbonate (baking soda) intravenously can reduce major kidney problems or death in critically ill patients with metabolic acidosis and acute kidney injury. About 660 adults in intensive care will receive either sodium bicarbonate or a stan…

This study tests whether giving patients electrical muscle stimulation and high-protein shakes soon after a type of stroke (aneurysmal subarachnoid hemorrhage) can prevent muscle loss and improve recovery. Thirty adults will receive the combo therapy or standard care. The main go…

This study tests whether baricitinib, a pill, can help preserve the body's ability to make insulin in people newly diagnosed with type 1 diabetes. About 300 children and young adults (ages 1 to under 36) will take either baricitinib or a placebo for 60 weeks. The goal is to see i…

This trial tests whether verapamil, a blood pressure drug, can protect the pancreas cells that make insulin in children and teens newly diagnosed with type 1 diabetes. Participants aged 10 to 18 will take verapamil or a placebo daily for 24 weeks. Researchers will measure how muc…

This phase 2 trial tests whether photobiomodulation, a type of light therapy, can improve healing of foot ulcers in people with diabetes. Researchers will compare standard wound care alone against standard care plus light therapy applied to the ulcer and to blood vessels. The stu…

This study tests a once-weekly injection called NNC0487-0111 in 630 adults who are overweight or obese and have type 2 diabetes. Participants receive either the active drug or a placebo for about a year. The main goal is to see how much weight they lose, with additional checks on…

This study helps older adults with type 2 diabetes talk to their doctors about switching from sulfonylurea drugs, which can cause dangerous low blood sugar and heart problems, to newer, safer options. About 220 participants will get either a simple question sheet to guide their n…

This trial tests two oral diabetes drugs, metformin and glyburide, in pregnant women with gestational diabetes who need medication to control blood sugar. The study asks whether one drug leads to fewer treatment failures, meaning blood sugar stays in a healthy range. Researchers …

This Phase 3 trial tests a new drug called KAI-9531, given as a weekly injection, in 1700 adults who are overweight or obese and have type 2 diabetes. The study compares KAI-9531 to a placebo to see if it leads to greater weight loss and better blood sugar control over 76 weeks. …

This study tests a new drug, pegozafermin, in 1,350 adults with MASH (a type of fatty liver disease) and moderate liver scarring (stage F2 or F3). The goal is to see if the drug can improve scarring or resolve liver inflammation without making the disease worse over 52 weeks. Par…

This study tests a new medicine called dotinurad against the standard drug allopurinol in 250 adults with tophaceous gout (gout with hard lumps under the skin). The main goal is to see if dotinurad lowers uric acid levels better after 24 weeks. Researchers will also check if toph…

This phase 2 trial tests an injectable drug called ION775 in 72 adults with high or severely high triglycerides. The goal is to see if it safely lowers fasting triglyceride levels. Participants continue standard care while receiving the study drug.

This trial tests whether the type and intensity of exercise affect blood sugar control in pregnant women with gestational diabetes. Participants will complete four separate exercise sessions: low-intensity resistance training, low-intensity aerobic training, moderate-intensity re…

This Phase 3 trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. The goal is to see if a single dose can keep blood counts stable so patients can stop their regular enzyme replacement or substrate reduction therapy. The study will enroll 45 people who…

This early-stage trial tests UX016, a drug designed to help people with GNE myopathy, a rare genetic muscle-weakening disease. Researchers will give the drug or a placebo to 24 adults to see if it is safe and improves muscle strength. The study is not yet recruiting.

This study compares two lifestyle programs for people with type 2 diabetes or prediabetes: one using a ketogenic diet and the other a low-fat diet. Over 12 months, researchers will track changes in weight, blood sugar, blood pressure, and heart health. The goal is to see which di…

This trial tests a procedure to remove and freeze ovarian tissue from pre-pubertal children who face cancer treatments that may harm their fertility. The goal is to store the tissue so it can be used later to restore fertility and hormone function. Researchers will follow partici…

This study follows adults with chronic kidney disease who are on dialysis and starting the phosphate binder Nephoxil for high blood phosphorus. Researchers track patients for 48 weeks to see how many reach a target phosphorus level of 3.5 to 5.5 mg/dL, using either a low or stand…

This study tests an oral medication called BI 1815368 in 300 adults with center-involved diabetic macular edema, a common cause of vision loss in diabetes. Participants take tablets twice daily for about 11 months, and doctors compare vision changes between those on the drug and …

This study looks at whether a 12-week structured diet and exercise plan can help adults aged 50 and older with obesity and insulin resistance lose fat and improve muscle function. Participants will receive all meals and exercise three times a week under supervision. The goal is t…

This study tests a new medicine called dotinurad against the standard drug allopurinol for lowering uric acid in adults with gout. About 500 people who have had at least two gout flares in the past year will take either dotinurad or allopurinol for 24 weeks. The main goal is to s…

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 is testing the safety of Replagal, an enzyme replacement therapy, in 5 Indian children and adults with Fabry disease who have not taken it before. Participants receive an infusion every 2 weeks for about a year. The main goal is to track side effects and serious reacti…

This Phase 3 study tests an experimental drug called pegozafermin in 762 adults with liver scarring (cirrhosis) caused by MASH, a fatty liver disease. Participants receive weekly injections of the drug or a placebo for about two years. The goal is to see if the drug can improve l…

This study tests a new drug called dotinurad in 90 adults with gout who cannot take common medications like allopurinol or have not responded to other treatments. The goal is to see if dotinurad can safely lower uric acid levels over 24 weeks. Participants are randomly assigned t…

This early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. T…

This trial is testing whether an investigational wound gel called VES-0-D can help heal mild-to-moderate diabetic foot ulcers better than standard wound care. Adults with type 1 or type 2 diabetes and a foot ulcer will receive either the gel or a standard dressing, applied every …

This phase 3 trial tests whether a subcutaneous (under-the-skin) injection of pegloticase works as well as the standard intravenous (IV) infusion for people with uncontrolled gout. About 270 adults will receive either the shot or the IV, both with weekly methotrexate. The goal is…

This study tests a new medicine called BI 3812465 for people with diabetic macular edema, a condition that causes swelling in the center of the eye and can lead to vision loss. The trial has two parts: first, a small group gets low to high doses to check safety, then a larger gro…

This study tests a drug called maridebart cafraglutide (MariTide) in people who are overweight or obese and also have type 2 diabetes. The goal is to see if the drug can help with long-term weight loss and blood sugar control, and to check its safety over time. Participants recei…

This trial tests whether a 12-week Nordic walking program, which uses poles to engage more muscles and burn more calories than regular walking, improves cardiovascular fitness in overweight or obese adults with type 2 diabetes and recent heart problems. Participants are randomly …

This study tests a new daily pill called elecoglipron in adults with type 2 diabetes and reduced kidney function. Participants also take dapagliflozin, a standard diabetes medicine. The goal is to see if elecoglipron improves blood sugar control, body weight, and blood pressure c…

This 76-week study tests whether mazdutide, a weekly injection already used for diabetes, can slow memory and thinking decline in 420 adults aged 50-75 with type 2 diabetes and early dementia. Half receive the drug, half a placebo. Researchers will measure changes in thinking ski…

This study tests a new drug, atumelnant, in 150 adults with classic congenital adrenal hyperplasia (CAH). The goal is to see if it can safely control hormone levels and allow patients to take lower doses of their usual steroid medications. Participants will receive either atumeln…

This trial tests whether trained community health workers can help adults with high blood pressure, diabetes, or high cholesterol address social needs like transportation, healthy food, housing, and job opportunities. Participants from clinics will be connected to a health worker…

This study tests whether giving a low dose of anti-thymocyte globulin (ATG) followed by either adalimumab or verapamil can help preserve the body's ability to make insulin in people aged 9 to under 21 who were recently diagnosed with type 1 diabetes. The main goal is to see if th…

This early-stage trial tests a single IV dose of VERVE-102, a gene-editing drug designed to permanently lower LDL (bad) cholesterol by turning off a specific gene in the liver. It involves 85 adults with inherited high cholesterol or early heart disease who still need lower chole…

This phase 3 trial tests a drug called plozasiran in 288 adults with severe hypertriglyceridemia (very high blood fats) who have had at least two pancreatitis attacks. Participants receive either the drug or a placebo every three months. The main goal is to see if the drug can pr…

This early-phase study tests a new drug called DSB2455 in adults with advanced cancers (breast, ovarian, prostate, pancreatic, or brain metastases) that have a specific DNA repair problem. The main goals are to check safety and see if the drug shrinks tumors. About 180 participan…

This study tests a smartphone app called DailyDose that gives personalized advice on insulin doses and carbohydrate intake for people with type 1 diabetes who use multiple daily injections. The app uses data from continuous glucose monitors and insulin pens. Researchers want to s…

This study tests whether eating peanuts and peanut products daily for six months can improve blood sugar, blood pressure, thinking skills, and gut health in people with prediabetes. Researchers will enroll 72 adults from diverse racial backgrounds. Participants will eat different…

This study tests a daily oral medication called atumelnant in children aged 1 to 18 with classic congenital adrenal hyperplasia, a genetic condition that disrupts hormone production. The goal is to see if the drug can safely normalize hormone levels and allow patients to lower th…

This study tests whether giving pasteurized donor human milk to full-term babies of mothers with diabetes during the first five days of life can reduce the number of infants admitted to the neonatal unit for low blood sugar. About 1,444 mothers and their newborns will be randomly…

This Phase 2b study tests whether the experimental drug MAR001 can lower high triglycerides and remnant cholesterol in adults. About 216 participants will receive either MAR001 or a placebo by injection. The main goal is to see if MAR001 reduces triglyceride levels more than plac…

This trial tests whether applying perinatal tissue allografts to chronic diabetic foot ulcers, along with standard wound care, leads to more ulcers healing completely within 12 weeks compared to standard care alone. Adults with type 1 or 2 diabetes who have a nonhealing foot ulce…

This study tests whether a daily dose of freeze-dried wild blueberry powder can improve blood sugar, blood pressure, and brain function in women with prediabetes. Thirty overweight women aged 20-65 will take either the blueberry powder or a placebo for six weeks. Researchers will…

This study tests the drug migalastat in 8 children aged 2 to 12 with Fabry disease, a rare genetic disorder. The goal is to see if the drug is safe, how it moves through the body, and if it helps protect kidney function. Participants will take the medicine for 12 months.

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 trial compares an experimental once-weekly injection, zenagamtide, with a standard daily insulin injection, insulin glargine, in adults with type 2 diabetes who also have higher cardiovascular risk. Participants are already taking 1 to 3 oral diabetes medications but need be…

This study follows people with chronic kidney disease who previously received an experimental treatment called REACT, which uses a patient's own kidney cells to try to repair damaged kidneys. Researchers will track participants for years to see if the therapy causes any delayed s…

This phase 2 trial tests whether adding an experimental drug called AT673 to the standard diabetes medication semaglutide leads to greater weight loss and better blood sugar control in adults with type 2 diabetes who are overweight or obese. Participants receive either a low or h…

This study tests a treatment called VX-880 for people with type 1 diabetes who have dangerous low blood sugar episodes. The therapy uses donor cells that can produce insulin, aiming to let participants stop taking insulin shots and avoid severe hypoglycemia. About 52 adults with …

This phase 2 trial is testing a combination of three drugs—zanubrutinib, bendamustine, and rituximab—in people with previously untreated Waldenström macroglobulinemia, a rare type of blood cancer. The study will enroll 56 participants to see how well the treatment shrinks or cont…

This trial tests whether a Mediterranean diet, enriched with extra virgin olive oil or walnuts, can lower the chance of stroke and slow cognitive decline in people with cerebral amyloid angiopathy (CAA) or CADASIL, two conditions that affect blood vessels in the brain. Participan…

This early-stage study tests if implanting a small piece of the participant's own leg nerve into a key brain area is safe and feasible for people with early Parkinson's disease or Multiple System Atrophy. Seven people will take part; four will get the nerve graft and three will h…

This study is testing whether a healthy diet centered around beef can reduce inflammation and improve metabolic health better than a standard U.S.-style diet based on USDA guidelines. Researchers will enroll 56 adults with metabolic syndrome or pre-diabetes and assign them to one…

This study tests an experimental drug called elecoglipron, taken alone or together with the existing medication dapagliflozin, in adults with type 2 diabetes whose blood sugar is not well controlled. About 800 participants will receive either the new drug, a placebo, or a combina…

This phase 3 study tests whether taking two diabetes medications together—elecoglipron and dapagliflozin—works better than either drug alone for controlling blood sugar in adults with type 2 diabetes. The trial enrolls about 2,000 people whose diabetes is not well managed by life…

This study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…

This study tests a drug called dorzagliatin in 15 adults with cystic fibrosis who have trouble controlling their blood sugar. Participants take the drug or a placebo for 7 days each, in a crossover design. The goal is to see if dorzagliatin improves glucose tolerance and insulin …

This study tests whether group care—where patients meet together with health workers for education and check-ups—can improve control of high blood pressure and diabetes in Nepal. Over 1,100 adults from 37 health facilities will be assigned to standard care, low-intensity group ca…

This study tests whether mirabegron (a bladder medication) and tadalafil (used for erectile dysfunction) can improve blood sugar control in people with prediabetes and obesity. About 96 adults will take one or both drugs or a placebo for 14 weeks. The goal is to see if these drug…

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 Phase 3 study tests whether a new drug called CagriSema helps people with severe obesity (with or without type 2 diabetes) lose more weight than the existing drug semaglutide. About 2,500 participants will receive either CagriSema or semaglutide for 83 weeks. The main goal i…

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 trial tests whether taking 150 mg of grape seed extract (MegaNatural BP) daily for 6 weeks can lower blood pressure and improve heart health in adults aged 40 to 60 with elevated blood pressure and prediabetes. Researchers will measure changes in blood pressure, blood vessel…

This phase 3 trial is testing whether an experimental once-daily tablet called elecoglipron can help Asian adults with obesity or overweight lose weight, when combined with diet and exercise. The study includes people with or without type 2 diabetes. Participants will receive eit…

This trial tests a mobile health program called The 3Ms 2.0 for primary caregivers of Black adolescents with type 1 diabetes. The program uses interactive online sessions, video clips, and text messages to encourage daily parental monitoring of diabetes care. Researchers will com…

This trial tests whether repetitive transcranial magnetic stimulation (rTMS) can reduce or delay the involuntary movements, called dyskinesia, that many Parkinson's patients develop after taking levodopa. Researchers will apply targeted magnetic pulses to a brain region involved …

This trial asks whether aggressively lowering blood phosphate levels in people with end-stage kidney disease on dialysis can reduce heart attacks, strokes, and death. Participants are randomly assigned to either a liberal phosphate target or an intensive target aimed at normal le…

This trial tests whether a silicone-based or a hydrocolloid patch placed under insulin pumps or glucose monitors can prevent contact dermatitis from coming back in children aged 2-18. Participants will use different patches and attend visits for skin exams, ultrasounds, and quest…

This study tests whether a structured, multidisciplinary obesity care program—which may include the weight-loss drug semaglutide—helps adults with obesity lose more weight than usual care. Adults aged 18-65 with a BMI of 30 or higher will be followed for 18 months to see if they …

This trial tests whether using portable HEPA air cleaners at home can reduce exposure to fine air pollution particles and, in turn, lower systolic blood pressure in people with metabolic syndrome. Participants in Qatar will use either a real HEPA filter or a sham filter for four …

This trial tests whether starting a comprehensive, guideline-based treatment plan immediately after a heart attack can reduce the chance of another heart attack, stroke, or death. The plan targets key risk factors like high cholesterol, high blood pressure, and diabetes using a c…

This study tests whether using a diabetes management app alongside personalized nutrition counseling from a dietitian can help adults with insulin-requiring diabetes improve their blood sugar control, cholesterol, physical activity, and overall quality of life. Participants will …

This trial asks whether empagliflozin, metformin, or a combination of both can improve how muscles produce energy and respond to blood flow in people newly diagnosed with type 2 diabetes. About 54 men with type 2 diabetes will be randomly assigned to one of three groups: empaglif…

This trial tests whether using a diabetes-specific questionnaire during annual check-ups can improve the quality of care for people with type 2 diabetes. About 2,000 adults with type 2 diabetes from 30 general practices in Denmark will take part. Half of the practices will use th…

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 16 weeks of exercise training, liraglutide treatment, or both can improve blood vessel function and insulin sensitivity in adults with type 2 diabetes. Participants are randomly assigned to one of three groups and are studied before and after the interven…

This trial tests whether a smartphone app called SPARK can help women with gestational diabetes manage their condition. Participants receive standard care, and some also use the app to support healthy eating, physical activity, and blood sugar tracking. The study looks at blood s…

This trial tests three different ways to reconnect the digestive tract after stomach cancer surgery to see which one best controls blood sugar in people who also have type 2 diabetes. Participants will be randomly assigned to one of three surgical procedures. The main goal is to …

This trial tests a new model of care called PEN-Plus, which brings specialized treatment for severe chronic diseases like diabetes, heart conditions, and sickle cell disease to first-level hospitals in Zambia. The goal is to train local health workers and set up clinics to manage…

Researchers at Johns Hopkins University are studying whether neuromuscular ultrasound and MRI can serve as reliable markers of swallowing muscle problems in people with inclusion body myositis (IBM). The study compares imaging and clinical findings in 30 participants, including p…

Researchers at Ankara Etlik City Hospital are studying whether the perfusion index, a blood-flow reading taken with a standard pulse oximeter, can predict the severity of diabetic ketoacidosis in adults arriving at the emergency department. The study will follow 140 patients aged…

Researchers on Réunion Island are measuring blood sugar levels in 2,000 adults to estimate how many people have diabetes or pre-diabetes. Participants give a fasting blood sample and drink a 75g glucose solution, then give a second blood sample two hours later. The study also che…

This study tests a special radioactive tracer called [68Ga]Ga-FAPI-46 for PET scans in 40 people with transthyretin cardiac amyloidosis (ATTR-CM), a condition where abnormal proteins build up in the heart. The goal is to see if this scan can measure scar-like activity in the hear…

Researchers are building AI models that score standard physical and cognitive screening tests from video and audio, and that predict results a person did not obtain. The study enrolls adults 18 and older and follows them for life, linking records to a long-term aging study to che…

This study is testing whether a special PET/CT scan using an amino acid tracer can better detect tumors in people with amino acid transport disorders, primary hyperparathyroidism, or glioma. About 500 adults will receive a single injection of the tracer and then get scanned. The …

This trial tests whether PET/CT scans using special radioactive tracers can detect amyloid protein buildup in various diseases, including cardiac amyloidosis. Researchers will give each participant a single injection of one of several tracers, then perform imaging to measure how …

This study tests whether a special imaging agent called 18F-FPyCO/SMBT-1 can help diagnose Parkinson's disease and related conditions by detecting a brain enzyme called MAO-B. About 200 adults with suspected or confirmed Parkinson's will receive a single injection of the agent an…

This study tests whether PET/CT scans using two radioactive tracers, 18F-92/AV45 and 11C-PIB, can detect beta amyloid buildup in the brain. Researchers will enroll 500 adults with suspected or known neurodegenerative diseases linked to amyloid. Each participant receives one injec…

This study is testing three new radioactive tracers that light up alpha-synuclein clumps in the brain during PET/CT scans. Researchers want to see if these scans can accurately diagnose Parkinson's disease and related disorders. 500 adults with suspected or confirmed Parkinson's …

This study is testing a new imaging agent called [18F]FT8 to see if it can help doctors better see and diagnose amyloidosis, a disease where abnormal proteins build up in organs like the heart. The study will involve 20 patients with amyloidosis and 5 healthy volunteers. Research…

This study is testing whether a combined autoantibody test on a drop of blood from a finger prick (dried on special paper) can detect type 1 diabetes as accurately as the standard blood draw from a vein. The researchers will enroll 106 children newly diagnosed with type 1 diabete…

This study tests a new way to measure the maximum amount of insulin a person's pancreas can produce. Participants first undergo the standard hyperglycemic clamp, then take a single dose of alpelisib and consume a standardized meal to see if the simpler test gives similar results.…

This study tests whether a cardiac imaging technique called a PYP scan can detect transthyretin amyloidosis in people aged 65 and older who have heart failure with preserved ejection fraction, also known as diastolic heart failure. The researchers will scan 515 participants to se…

This early-stage study tests a new radioactive tracer called SST001 that lights up on PET scans. Researchers want to see if it is safe and can help tell the difference between multiple system atrophy (MSA) and Parkinson's disease. The study will include 30 healthy volunteers and …

This trial tests whether adding an AI analysis of eye photos, an eye pressure check, and a virtual reality visual field test to standard diabetic eye screening can help detect glaucoma. About 2,000 people with diabetes who are already getting AI-based screening for diabetic eye d…

This study tests whether a quantitative analysis of bone scintigraphy (SUVmax) can better track amyloid burden in the hearts of patients with transthyretin cardiac amyloidosis who are being treated with tafamidis. The current Perugini score is limited in assessing disease severit…

This trial asks whether a home sleep apnea test can be a better first step than an overnight lab study for diagnosing obstructive sleep apnea in people with dementia or mild cognitive impairment. Sleep apnea is common in these conditions and can worsen thinking problems, but many…

This study tests whether a continuous glucose monitor (CGM) worn on the arm can diagnose gestational diabetes as well as the standard oral glucose tolerance test (OGTT). Pregnant women of Chinese or Indian ethnicity will wear the sensor for up to 10 days and also undergo the OGTT…

This trial is testing whether an artificial intelligence (AI) model, called MONA DR, can accurately detect diabetic retinopathy—a common eye complication of diabetes that can lead to blindness—from retinal photographs. The study will enroll 100 adults with diabetes and compare th…

Researchers are testing a weekly family-based lifestyle program called Family DPP+ to see if it helps parents with prediabetes lose weight and adopt healthier habits. The trial enrolls African American and Hispanic parents and their children in Georgia. Parents attend sessions fo…

Researchers in Pakistan are testing whether a mobile app that uses artificial intelligence to send personalized heat-risk alerts can reduce heat-related illness symptoms in adults with chronic conditions such as hypertension, diabetes, kidney disease, heart disease, or obesity. T…

This trial tests whether two natural supplements—quercetin (from foods like apples and onions) and coenzyme Q10 (a substance your body makes)—can reduce inflammation and improve metabolic health in older adults. Over 12 weeks, participants aged 50 to 75 who are sedentary will tak…

This pilot study will test whether teaching people aged 50 to 75 how to cook healthy meals can reduce their risk of type 2 diabetes and dementia. Forty participants at higher risk will either attend weekly cooking classes with at-home practice or receive nutrition handouts. The s…

This phase II trial tests whether a special form of nicotinic acid (a B vitamin) can reverse prediabetes and prevent type 2 diabetes. About 390 adults with prediabetes will take either a low or high dose of the drug or a placebo daily for 26 weeks, along with lifestyle advice. Th…

This study tests whether the drug semaglutide can prevent type 2 diabetes in women who had gestational diabetes and now have high blood sugar after pregnancy. About 252 women will receive either semaglutide or a placebo for several months. The goal is to see if the drug lowers th…

Researchers are testing whether adding dapagliflozin to a lifestyle program can normalize blood sugar in people with prediabetes and protect their kidneys. The trial enrolls 170 adults aged 35 to 75 with prediabetes. Participants receive either dapagliflozin or a placebo for six …

This study looks at whether adding supervised cycling and rowing to a Mediterranean diet improves blood sugar control better than diet alone in women with prediabetes. Thirty women will be split into three groups based on their current blood sugar levels. They will follow the die…

Researchers test whether lowering systolic blood pressure to below 120 mm Hg prevents major cardiovascular events in adults with type 2 diabetes and high-normal blood pressure. The trial enrolls about 11,400 people aged 45 to 79. Participants receive either intensive treatment wi…

This study aims to develop a better system to support breastfeeding for women who had gestational diabetes, with the goal of preventing type 2 diabetes later. Researchers will work with patients, healthcare providers, and hospital leaders to design a practical strategy. Only 10 p…

This study tests whether giving adults at risk for type 2 diabetes free fresh fruits and vegetables, along with a proven lifestyle program, can help them lower blood sugar and lose weight. One hundred adults with prediabetes will either get the standard diabetes prevention progra…

Researchers are testing whether daily cottonseed oil improves cholesterol and blood fat levels compared with oils that have a similar fat makeup. The study includes 90 adults aged 25 to 75 who have elevated cholesterol or excess weight. Participants drink a breakfast shake contai…

This study tests whether the drug baricitinib can delay the onset of type 1 diabetes in children aged 1 to 36 months who are at high risk. Participants take baricitinib or a placebo daily for up to 5 years. The goal is to see if the drug can push back or prevent the need for insu…

This study looks at whether a diet rich in whole white potatoes can improve blood sugar control and blood pressure in adults aged 50-70 who are at risk for type 2 diabetes. Participants will eat either a potato-rich healthy diet or a typical Western diet for 12 weeks. Researchers…

This trial tests whether a smartphone app can help people with REM sleep behavior disorder, a condition that often precedes Parkinson's, become more physically active over two years. Participants receive personalized activity goals through the app and track their steps and exerci…

This trial tests whether a smartphone app can help 600 people with Parkinson's-related gene mutations increase their daily physical activity over two years. Participants set their own exercise goals and get feedback through the app. Researchers will measure changes in step counts…

This study tests whether regular, personalized health coaching—delivered virtually and during community screenings—can reduce the 10-year risk of heart attack or stroke in adults aged 40-75 who have at least one cardiometabolic risk factor like high blood pressure, high cholester…

This pilot study is testing whether a virtual reality (VR) cognitive training program can help protect memory and thinking skills in adults aged 45-80 with type 2 diabetes and mild cognitive impairment. Forty participants will be randomly assigned to either 16 weeks of VR-based t…

People with type 2 diabetes often develop stiff ankles, pain, and walking problems. This trial compares two physiotherapy approaches: Mulligan Mobilization with Movement, where a therapist applies a gentle glide to the ankle during active movement, and conventional physiotherapy …

Researchers are observing how high-dose vitamin C injections affect symptoms and tolerability in adults with vitamin C deficiency caused by acute or chronic diseases. The study includes about 1,000 participants and tracks changes in general and disease-specific symptoms using a s…

Researchers are testing whether a home-based program combining adapted exercise and mindfulness training can improve walking ability and sleep quality in people with rare neurodegenerative diseases such as leukodystrophy, ataxia, and adrenomyeloneuropathy. The study enrolls about…

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

People with treatment-resistant schizophrenia often take clozapine, a drug that can cause serious weight gain and metabolic problems. This pilot study tests whether a daily infusion made from the leaves of the Nitraria retusa plant can reduce those side effects. Thirty patients w…

Researchers test whether adding a predictive app to a continuous glucose monitor changes fear of low blood sugar, sleep quality, and diabetes distress in adults with type 1 or insulin-treated type 2 diabetes. Participants use the monitor alone for two weeks, then with the app for…

Researchers are testing SugarVita 2.0, a digital game that lets players make nutrition, activity, and medication decisions while a glucose simulator shows the results. The 12-week pilot enrolls 30 adults with type 2 diabetes and an HbA1c above 55 mmol/mol. The study measures chan…

This study tests whether quercetin, a natural supplement found in foods like onions and apples, can improve blood pressure and body fat in healthy adults. Researchers will give 40 participants either quercetin or a placebo for a set period. The goal is to see if this simple dieta…

Researchers are testing an experimental oral drug called BGE-102 in adults with diabetic macular edema, a condition where fluid leaks into the center of the retina and blurs vision. The trial enrolls about 180 people with type 1 or type 2 diabetes and center-involved DME. Partici…

This trial tests whether two simple distractions — watching a betta fish or playing with a hand puppet — can lower pain, fear, and anxiety in children aged 5 to 7 during a blood draw. One group will use the fish, another the puppet, and a third will receive standard care. Researc…

This study tests whether Radiesse (calcium hydroxylapatite) injections can restore facial volume and improve skin quality in people who lose facial fullness from GLP-1 weight loss drugs like semaglutide. 48 adults starting GLP-1 therapy will be randomly assigned to receive either…

This study tests a drug called N-acetyl-L-leucine for people aged 4 and older with CACNA1A gene disorders, which can cause coordination problems, dizziness, and migraines. About 60 participants will receive either the drug or a placebo, then switch, to see if it improves movement…

This study tests whether a type of talk therapy called Acceptance and Commitment Therapy (ACT) can help people with type 1 diabetes who also have eating disorders. Participants will be randomly assigned to ACT, supportive diabetes counseling, or a waitlist. The goal is to see if …

This study tests whether semaglutide, a drug used for diabetes, can help people with type 2 diabetes quit smoking. About 100 adults in Ontario who smoke at least 5 cigarettes daily will receive the drug alongside nicotine replacement therapy. The main goal is to see if a larger s…

This study tests an experimental drug called HRS-2129 in adults experiencing a sudden, painful gout flare. The goal is to see if it can safely reduce joint pain within 24 hours compared to a placebo. Researchers are also looking at how well different doses work and how quickly pa…

This pilot study will test whether a 6-week program of cognitive behavioral therapy for insomnia (CBT-I) can improve sleep and blood sugar control in 30 adults aged 40+ with type 2 diabetes and chronic insomnia. Participants are randomly assigned to CBT-I or usual care, and outco…

This trial tests whether adding nanoparticles to the standard disinfection during root canal retreatment reduces post-operative pain in people with type II diabetes. Participants are adults aged 18 to 40 with a failed root canal in a front tooth and an active infection at the roo…

This study tests a mobile health program called R2D2 designed to reduce diabetes distress in children aged 8-12 with type 1 diabetes and their parents. The program uses cognitive behavioral therapy, mindfulness, and behavioral strategies to help families cope. Researchers will me…

This study looks at whether giving a carbohydrate drink before coronary artery bypass surgery can improve insulin sensitivity and recovery in people with type 2 diabetes. About 50 participants will be enrolled. The goal is to see if this simple step can reduce complications and s…

This trial tests whether personalized repetitive transcranial magnetic stimulation (rTMS) can reduce cognitive fluctuations—sudden changes in alertness and attention—in people with dementia with Lewy bodies (DLB). Forty participants will receive both an experimental rTMS conditio…

Researchers are building a large database of cognitive, behavioral, lifestyle, and genetic information from people with neurodegenerative diseases such as Parkinson's disease, multiple system atrophy, and progressive supranuclear palsy. The goal is to find biomarkers that help do…

This study looks at how losing sleep and having a disrupted body clock (like shift workers) can make the body less responsive to insulin, a key step toward obesity and type 2 diabetes. Researchers will test 48 healthy adults aged 18-45 to see how the timing of the stress hormone …

This study looks at how a supplement called nicotinamide riboside (NR) and a low-carb, high-fat 'ketogenic' diet affect the way the body burns fat and uses energy. Researchers will measure changes in metabolism in 100 overweight or obese adults. Participants will eat a standard d…

This study tests four different HIV drug regimens in 120 healthy volunteers to see how they affect metabolism and weight. Participants take one of the drugs or a combination for four weeks, with blood tests, body scans, and optional fat biopsies. The goal is to understand why som…

Researchers are investigating how mitochondria, the energy-producing structures inside muscle cells, relate to frailty in older adults. The study includes about 120 people: older adults with type 2 diabetes, healthy older adults, and healthy young adults. Everyone does a six-week…

Doctors often give phosphate supplements to hospitalized patients with low phosphate. This study asks whether those supplements cause a temporary rise in FGF23, a hormone that controls phosphate. Researchers will measure FGF23 and phosphate in blood samples from 60 adults in inte…

This study follows 100 adults with diabetic eye disease who are already receiving standard anti-VEGF injections for swelling in the center of the retina. Researchers will use advanced vision tests and imaging to track changes over time. The goal is not to test a new drug, but to …

This study follows people who previously took part in type 1 diabetes trials to see how long their bodies continue to produce insulin. Researchers will also look at how immune system changes relate to insulin production. The goal is to gather information that could lead to better…

Researchers want to learn how the body's own remaining insulin production, measured by a marker called C-peptide, affects quality of life and health outcomes in people with insulin-treated diabetes. The study follows about 700 adults, including some from a previous diabetes study…

This observational study aims to better understand primary mitochondrial myopathy, a rare muscle disease. Researchers will follow 1300 patients and healthy controls, measuring muscle strength, balance, and daily function over time. The goal is to develop and validate tools to tra…

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 uses advanced CT scans to find out how common blood clots are inside the heart chambers of people with cardiac amyloidosis, a condition where abnormal proteins build up in the heart. Researchers will scan 200 adults diagnosed with this disease to detect clots that may …

This study aims to learn how type 2 diabetes and obesity in young people affect heart and blood vessel health over time. About 930 participants aged 12 to 25, including those with diabetes, overweight but not diabetic, and healthy lean individuals, will be followed for up to 25 y…

This study will follow 2,000 adults with metabolic syndrome or prediabetes over time to understand how these conditions progress and lead to complications. Researchers will track health records, nutrition, and physical function to find key factors that affect long-term outcomes. …

Researchers are studying whether blood levels of luteinizing hormone (LH) and human chorionic gonadotropin (hCG) are linked to the presence, severity, and long-term outcomes of endocrine disorders such as osteoporosis, thyroid disease, and adrenal gland problems. The study enroll…

This study tracks the natural course of fucosidosis, a rare genetic disorder, in up to 57 people. It collects medical history and ongoing health data without giving any experimental treatment. The goal is to better understand the disease and help design future therapies.

This study examines how a type of immune cell called gamma delta T cells adapt and function in critically ill patients, both with and without sepsis. Researchers will compare these cells in healthy people, non-septic ICU patients, and septic ICU patients. The goal is to understan…

Researchers in Guangzhou, China, are following 20,000 pregnant women and their children to study how the mix of bacteria in the mother's body during pregnancy and in the baby's gut early in life relates to health outcomes later on. The study tracks conditions such as obesity, ast…

This study looks at 50 adults with AL amyloidosis who achieved complete remission after initial chemotherapy. It checks if tiny amounts of remaining disease (called minimal residual disease, or MRD) affect organ recovery and survival. Researchers also test whether a stem cell tra…

This study looks at how your genes and your environment—like what you eat, breathe, and experience—work together to influence common diseases such as diabetes, heart disease, and asthma. Researchers are collecting health questionnaires, genetic data, and other information from 25…

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 tests how a single injection of HRS-4729 behaves in people with mild, moderate, or severe kidney impairment compared with healthy people. Researchers measure drug levels in the blood over time to see if kidney function affects how the body processes the drug. The trial…

This study aims to learn more about lipid disorders—conditions that affect fats like cholesterol in the blood. Researchers will observe up to 2,000 people with abnormal cholesterol levels to better understand how these disorders develop and how to diagnose them. Participants will…

This early-stage trial is testing how well different tablet versions of the experimental drug elecoglipron are absorbed by the body. Healthy volunteers receive a single dose of one formulation, and researchers measure drug levels in the blood over time. The goal is to compare the…

This study looks at how fear of low blood sugar (hypoglycemia) affects how well people with diabetes follow their diet and take their medication. Researchers will measure fear levels, medication adherence, and diet adherence in 100 adults with diabetes who have had at least one l…

This study follows up to 2,500 people with thyroid problems like hyperthyroidism, hypothyroidism, or Graves' disease. Researchers will track how these conditions develop over time using standard medical tests and genetic samples. The goal is to better understand the natural histo…

This study looks at how obesity and body fat affect heart disease risk in 2,000 healthy African American men and women. Researchers will measure body fat, blood fats, and insulin function to see if risk factors differ by sex. The goal is to understand why heart disease rates are …

This study aims to better understand and treat people with certain inherited metabolic or genetic disorders. Researchers will use standard medical tests like blood work and imaging to diagnose and care for participants, who may also join other related studies. The goal is to expa…

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 people with Multiple Acyl-CoA Dehydrogenase Deficiency (MADD) over time to learn how the condition progresses and affects daily life. Researchers will collect health data and patient feedback to identify patterns and potential markers of the disease. The goal i…

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 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 at the National Institutes of Health looks at how infections can worsen symptoms in people with mitochondrial disease, a group of disorders that affect energy production in cells. Researchers will evaluate participants' immune systems through blood tests, physical exam…

Researchers at the University of Miami are studying whether advanced eye imaging devices can reveal useful information about the eye in health and disease. The study has two parts: one observes the eyes of healthy people and people with conditions such as multiple sclerosis, dry …

This study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

This study will follow 450 people with diabetes (and some without) for several years to track how their eyes change. Researchers will use advanced eye tests like visual field analysis, contrast sensitivity, and retinal imaging to see which measurements best show disease progressi…

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 facial features of males aged 2 to 40 with creatine transporter deficiency (CTD), a genetic disorder that causes intellectual disability, seizures, and behavioral issues. Researchers will examine photos of participants to see if they share common facial traits…

Researchers at Peking University First Hospital are building a prospective cohort of 2,000 people with plasma cell disorders, including multiple myeloma, amyloidosis, and related conditions. Participants give blood, bone marrow, and urine samples before and after treatment, and t…

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 better understand why some people experience dangerously low blood sugar (hypoglycemia), especially after fasting. Researchers will admit 1,500 adults to the NIH Clinical Center for controlled fasting and a series of blood tests to measure insulin and other fac…

This study follows people with rare genetic disorders that cause harmful substances to build up in the body, leading to brain damage. Researchers will monitor up to 200 participants over time using exams, surveys, and lab tests. The goal is to better understand how these diseases…

This study explores whether stem cells from taste buds can be turned into insulin-making cells. Researchers will take tiny tissue samples from the tongues of 250 healthy adults to isolate and grow these stem cells in a lab. The goal is to see if this is possible in humans, which …

This study follows 1,500 pregnant women and their children for up to 18 years to understand how a mother's health, lifestyle, and environment affect her child's growth and risk for obesity, diabetes, and fatty liver. Mothers attend visits during pregnancy and after birth, while c…

This study follows up to 10 children under 2 years old with acid sphingomyelinase deficiency (ASMD) who are already receiving olipudase alfa (Xenpozyme®) as part of their routine care. Researchers will monitor side effects and immune responses over 1 to 3 years. No new treatment …

This natural history study will look at how metabolism relates to body size and composition in 2,000 people aged 2 and older, including healthy volunteers and those with conditions like diabetes, obesity, or cancer. Participants will stay in the hospital for two days to undergo v…

This study is a long-term registry that follows over 1,200 people with Gaucher disease worldwide. It collects real-world data on how the disease progresses and how different treatments, including velaglucerase alfa, work over time. No new drugs are being tested; instead, particip…

This study follows up to 1,000 adults with certain slow-growing blood cancers (CLL, SLL, LPL, WM) to track how the diseases change over time. Participants have regular check-ups and blood draws every 6 to 24 months, and may also have optional biopsies or scans for research. No tr…

This pilot study looks at how sleep, physical activity, and meal timing in early pregnancy relate to blood sugar patterns and the risk of gestational diabetes. Researchers will track 140 pregnant women using wearable devices and an AI food-logging app. The goal is to see if real-…

This observational study will follow 2000 adults with type 2 diabetes to see how their eating habits, body composition, and grip strength change over time. Researchers hope to identify patterns that could help improve care. Participants must be 18 or older and able to complete ba…

This global study follows people with POLG-related disorders, a group of rare mitochondrial conditions, to understand how the disease progresses over time. By observing clinical changes in up to 300 participants of all ages, researchers aim to identify key milestones that could s…

This study follows 2,800 unpaid caregivers of people with chronic conditions like diabetes or inherited disorders. Researchers will survey and interview caregivers over up to 5 years to track changes in their health, stress, and social support. The goal is to understand how careg…

This study tests whether people with prediabetes and obesity change their eating and activity habits when they can see their glucose levels in real time versus when the data is hidden. Thirty-four adults will wear a continuous glucose monitor during both an open and a blinded pha…

Researchers want to understand how well people with diabetes in Reunion Island and mainland France can find, understand, and use health information. About 1,350 patients with type 1, type 2, or gestational diabetes who receive hospital care complete a one-time questionnaire calle…

This observational study monitors the long-term safety of eplontersen, a treatment for transthyretin amyloidosis, in patients who have had a liver transplant or have severe liver impairment. Researchers will track side effects, lab changes, and serious health events in about 320 …

Researchers at Joslin Diabetes Center are studying how the body processes glucose and glucagon after a meal in people who have had gastric bypass surgery. Some of these people develop low blood sugar after eating, a condition called post-bariatric surgery hypoglycemia. The study …

Calciphylaxis is a rare disorder where calcium builds up in small blood vessels in the skin, causing painful lesions. Researchers at Massachusetts General Hospital are building a biobank and patient registry to collect samples and health information from up to 300 adults with the…

Some cancer drugs cause rare but serious heart and metabolic problems. This study follows 5,000 cancer patients across several French hospitals who receive a dedicated heart checkup and ongoing follow-up through cardio-oncology programs. Researchers track how many patients benefi…

Researchers are following 10,000 registered nurses in Xiamen, China, to see how genes, lifestyle, and environment relate to common chronic diseases like high blood pressure, heart disease, stroke, diabetes, kidney disease, and some cancers. The study collects health and lifestyle…

This study looks at how certain natural chemicals (endocannabinoids) influence the development of fat cells from stem cells found in belly fat. Researchers will take small fat samples from 30 people undergoing abdominal surgery, including healthy controls, people with obesity, an…

This study looks at whether exercising in the morning or evening helps the body handle sugar better in people with prediabetes who have an extreme natural body clock. Twenty-eight adults with prediabetes and overweight will do a single cycling session at 9 AM, 5 PM, or no exercis…

This study follows 150 adults aged 70 and older with low-grade non-Hodgkin lymphoma to see how their quality of life changes during treatment. Researchers will use questionnaires to track well-being from diagnosis through one year after therapy starts. The goal is to identify whi…

Acute intermittent porphyria (AIP) is an inherited disorder that can cause sudden, severe attacks, but not everyone who carries the gene gets sick. Researchers at Mount Sinai are collecting blood, saliva, and urine samples from about 150 people in AIP families, including affected…

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

This study follows 400 people with mitochondrial disease and their household members to learn how infections affect them. Researchers will analyze blood samples and health records to find immune patterns linked to severe illness. The goal is to improve care and identify potential…

Researchers are studying whether the mix of bacteria in the gut helps explain why people with obesity and type 2 diabetes get chronic infections more often. The study follows 80 adults, some with ongoing infections and some without, for 12 months. Participants give blood, stool, …

This study aims to learn more about methylmalonic acidemia (MMA) and related disorders by observing people with these conditions over time. Researchers will track complications, perform tests like blood draws and MRIs, and look for new genetic causes. The goal is to better unders…

This study aims to see if a specially designed education module helps people with spinal cord injury (SCI) learn about their increased risk for heart disease, obesity, and metabolic problems. Researchers will enroll 250 adults with SCI and measure changes in knowledge, body weigh…

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 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 will follow about 2000 adults with a heart condition called ATTR-CM, where abnormal proteins build up and weaken the heart. Researchers want to see how patients do on a drug called vutrisiran in everyday medical care, not just in a controlled trial. They will measure s…

This study follows 450 US adults with Fabry disease who are taking migalastat or enzyme replacement therapy. Researchers will track kidney function, heart and brain events, and quality of life over time to see how well these treatments work in everyday practice.

This study follows 705 adults with Waldenström's macroglobulinemia, chronic lymphocytic leukemia, marginal zone lymphoma, or follicular lymphoma who are taking zanubrutinib (with or without obinutuzumab). Researchers will track hospital visits, quality of life, and how well the d…

This study aims to find biological markers that can track the progression of Niemann-Pick type C (NPC), a rare genetic disorder that damages the nervous system. Researchers will evaluate 900 patients of any age through regular checkups, spinal taps, eye exams, and brain scans. Th…

This registry is collecting information from 35,000 adults with type 2 diabetes, high blood pressure, heart failure, and/or chronic kidney disease. Researchers want to see how these conditions are managed in everyday medical practice, what treatments people receive, and how often…

This observational study will analyze medical records from over 52,000 people with ATTR amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers want to learn about patients' symptoms, treatments, and outcomes in everyday medical practice. No new treatm…

This study aims to find biological markers (biomarkers) that can predict whether a diabetic foot ulcer will heal or not. Researchers will collect samples like blood and wound fluid from 5,000 adults with diabetes and open foot ulcers. The goal is to develop a tool that helps doct…

This natural history study aims to gather information from up to 3,000 people with genetic conditions that cause too much androgen (male-like hormones), such as congenital adrenal hyperplasia (CAH) and familial male-limited precocious puberty (FMPP). Participants undergo various …

Researchers are building a large, diverse group of adults with varying body sizes, ethnicities, and diabetes risk. They will collect blood, stool, and fat tissue samples to study how obesity relates to diabetes and other health issues. The goal is to create a rich resource for fu…

This study is a global registry that will follow about 150 people with acute hepatic porphyria (AHP) over time. Researchers will collect information on how the disease progresses, how it is managed in real-world settings, and the safety and effectiveness of approved treatments li…

This study is testing a new non-invasive device that uses laser speckle to measure blood flow and structure inside the eye. Researchers will compare these images with standard vision tests in 500 people with various retinal conditions. The goal is to see if this technology can be…

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

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

This study follows 300 people with CLN3 Batten disease, a rare genetic disorder that causes vision loss, seizures, and decline in thinking and movement. Researchers collect samples like blood and spinal fluid to find biological markers that could be used in future treatment trial…

Researchers at Mayo Clinic are studying how the pancreas changes during and after pregnancy in women with gestational diabetes. The trial involves pregnant women who have completed their second-trimester screening and are classified as having gestational diabetes or not. After de…

This study monitors pregnancy outcomes in women who took patisiran-LNP for hereditary ATTR amyloidosis. Researchers will track birth defects, pregnancy complications, and infant health. The goal is to better understand the drug's safety during pregnancy, not to test a new treatme…

This observational study will follow 1,500 people with ATTR amyloidosis, a rare disease that damages the heart and nerves, for many years. Researchers will track how the disease progresses, how patients are treated in real-world settings, and the safety of two approved drugs, pat…

This study aims to understand how the hormone insulin controls blood sugar in different people. Researchers will test a new procedure called the graded insulin suppression test (GIST) in 15 healthy volunteers and those at higher risk for type 2 diabetes. Participants receive medi…

This study is creating a large collection of health information and biological samples (like blood, saliva, and eye tissue) from people with and without retinal diseases. Researchers aim to use this database to better understand conditions like age-related macular degeneration an…

This observational study follows 500 people with atypical parkinsonian syndromes, including multiple system atrophy, dementia with Lewy bodies, progressive supranuclear palsy, and corticobasal degeneration. Researchers track motor and non-motor symptoms and imaging changes every …

This study looks at how men and women process alcohol after sleeve gastrectomy, a weight-loss surgery. Researchers will give alcohol by mouth or through an IV, both on an empty stomach and after a meal, to see how it affects blood alcohol levels, mood, and blood sugar. They will …

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

This study follows people with propionic acidemia (PA), a rare metabolic disorder, to track how the disease affects the body over time. Researchers will collect medical history, blood, urine, and other samples, and perform imaging and heart tests during annual hospital visits. Th…

This study follows over 4,000 people in Spain who have familial hypercholesterolaemia, an inherited condition causing very high cholesterol and early heart disease. Researchers track their cholesterol levels and any heart events, such as heart attacks or strokes, over time. The g…

This study looks at whether obese children with autism spectrum disorder differ from those without autism in fitness and heart health. Researchers will review medical records of 200 children aged 6 to 18, including step test results, handgrip strength, body measurements, and bloo…

This study will follow about 579 adults who need surgery to remove part or all of their pancreas but do not have diabetes before the operation. Researchers want to see how often new diabetes develops after surgery and whether the amount of pancreas removed changes that risk. The …

This study follows 200 people with Gaucher disease over many years to learn how the condition affects the body long-term, including the nervous system, liver, and spleen. It also looks at how well current treatments like enzyme replacement therapy work over time. No new treatment…

This study is testing new magnetic resonance imaging (MRI) techniques to get clearer pictures of the heart and blood vessels without using radiation. Researchers will compare these new MRI methods to standard tests like angiography and CT scans. Up to 4,000 adults—including healt…

This study investigates why women with type 1 diabetes (T1D) frequently develop polycystic ovary syndrome (PCOS), a condition of high androgen levels. Researchers will compare five groups of 12 participants each, including women with T1D and PCOS, women with T1D alone, men with T…

This study looks at whether advanced imaging can detect a hidden heart condition called cardiac amyloidosis in people with severe aortic valve stenosis. The condition can complicate treatment and recovery. Researchers will use echocardiography, CT, and SPECT/CT scans to check for…

This early-stage trial is testing a new drug called ACI-19764 in 78 healthy adults to see if it is safe and how the body handles it. The drug targets a protein in the brain linked to inflammation. Participants will receive either the drug or a placebo, and researchers will monito…

This study follows 600 people with neurodegenerative diseases such as Parkinson's, Alzheimer's, and related conditions over 1 to 10 years. Researchers collect data from routine clinical exams, brain imaging, movement analysis, and blood tests. They use statistical tools and artif…

This study looks at whether people with mild cognitive impairment or dementia also have problems with their autonomic nervous system, which controls automatic body functions like sweating and pupil response. Researchers will use two non-invasive devices, Sudoscan and automated pu…

This study looks at whether a new tool helps young adults (ages 18-30) with type 1 diabetes and their doctors work together better to make care plans that fit daily life. Researchers will compare usual doctor visits with visits using the tool, and interview participants afterward…

This study aims to learn more about alkaptonuria, a rare genetic disease that causes a pigment to build up in bones and tissues, leading to arthritis and other problems. Researchers will evaluate 300 patients over several years using advanced medical tests. The goal is to better …

This study looks at why people with pre-diabetes or type 2 diabetes tire more quickly during leg exercises. Researchers will test a special low-load resistance training program, sometimes with blood flow restriction, to see if it improves muscle strength, blood flow, and energy u…

This early-stage trial is testing an experimental oral drug called TUL321 in healthy adults to see if it is safe and tolerable. The study will also measure how the drug moves through the body and whether food affects its absorption. TUL321 is being developed as a potential treatm…

Researchers at the University of Pennsylvania are building a large repository of blood, DNA, and family history data from people with age-related brain diseases like Alzheimer's, Parkinson's, and ALS, as well as their family members. The goal is to support studies into the geneti…

This study is testing whether a simple vision screening program in general practice can help detect vision loss early in adults over 70 who have at least one chronic condition. Participants will have three vision tests done by their GP or nurse, then visit a collaborating opticia…

This study is creating the first large database for Niemann-Pick type C (NPC), a rare disease that damages the brain, liver, and spleen. Researchers will collect blood samples and medical records from 100 people with NPC to link their genes with their symptoms. The goal is to und…

This study looks at how women with a severe form of congenital adrenal hyperplasia (classic 21-hydroxylase deficiency) become pregnant and what happens during their pregnancies. Researchers will collect information from medical records and phone interviews with 200 adult women. T…

This study aims to find out why people with diabetes often have lower vitamin C levels in their blood. Researchers will measure vitamin C in blood, urine, and cells from 5,000 adults with and without diabetes. They will also look at genes that control vitamin C transport. The goa…

This study follows people with Fanconi anemia, a rare inherited condition that raises the risk of certain cancers, especially squamous cell carcinoma. Researchers will screen 200 participants aged 12 and older every year for up to 10 years using exams, blood tests, and imaging to…

This study is looking for early signs of heart amyloidosis in Black adults who carry a specific gene change (V122I TTR) that raises their risk. Researchers will use heart MRI scans and blood tests to detect protein buildup before symptoms appear. The goal is to find ways to diagn…

This study aims to understand how environmental factors like pollution, diet, and stress contribute to human disease. Researchers will collect samples such as blood, urine, and skin from 2,000 healthy adults aged 18 and older. Participants may also complete surveys and provide ho…

This study is observing 3000 adults with various types of heart failure to learn how the disease develops and progresses. Researchers will collect medical records, blood samples, and imaging data to identify distinct patient groups. The goal is to match the right treatments to th…

This study follows people who received an investigational gene therapy in an earlier iECURE trial for a urea cycle disorder, a condition that can cause dangerous ammonia buildup. Researchers will track safety, side effects, and whether the therapy's effects last over up to 14.5 y…

This study from the National Human Genome Research Institute aims to better understand Congenital Disorders of Glycosylation (CDG), a group of rare genetic conditions that affect how the body attaches sugars to proteins and fats. Researchers will examine up to 200 participants, i…

This study looks at people with REM sleep behavior disorder (RBD), a condition where people act out their dreams and which often leads to Parkinson's disease. Researchers will use advanced brain imaging, sleep tests, and physical exams to compare 50 people with RBD to 50 healthy …

This study looks at women who carry the gene for X-linked adrenoleukodystrophy (X-ALD), a rare inherited condition that mainly affects the nervous system. Although the disease is linked to the X chromosome, up to 80% of female carriers may develop symptoms like leg weakness and b…

This observational study tests whether continuous glucose monitors (CGMs) worn from about 12 weeks of pregnancy can detect subtle blood sugar changes that might indicate a higher risk of gestational diabetes, before the usual screening at 24-28 weeks. Researchers will follow 48 p…

This study looks at adults who were diagnosed with classic congenital adrenal hyperplasia (CAH) as children, a condition that requires lifelong hormone therapy and sometimes early genital surgery. Researchers want to know if that childhood treatment, especially surgery, is linked…

This study follows a large group of people with Parkinson's disease and related conditions in France to map how the disease unfolds over time. Researchers collect detailed information on symptoms, treatments, and genetic samples at regular visits. The goal is to identify distinct…

This study uses continuous glucose monitors (CGMs) to track blood sugar levels in the days right after a kidney transplant. It compares three groups of recipients: those with diabetes, those with pre-diabetes, and those without diabetes. The goal is to see if glucose patterns dif…

This study looks at whether liver function and blood clotting tests can help doctors better understand and monitor cardiac transthyretin amyloidosis (ATTR-CA), a condition where abnormal protein builds up in the heart. Researchers will compare these tests in people with ATTR-CA a…

This study investigates how Fabry disease damages the kidneys over time, even before routine tests show problems. Researchers will examine kidney biopsies and function in 50 patients, some of whom receive enzyme replacement therapy. They aim to see if starting treatment earlier o…

This study is looking at whether a specific genetic variation, called SLC16A11, influences how well the common diabetes drug metformin lowers blood sugar. Adults with type 2 diabetes will be grouped by whether they carry this gene variant and will take extended-release metformin …

This study investigates whether a key channel in insulin-producing cells becomes less active as type 2 diabetes progresses. Researchers will give a single 10 mg dose of glipizide, a drug that blocks this channel, to people with and without type 2 diabetes and measure their insuli…

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…