Metabolic disease

Atransferrinemia is a rare inherited condition in which the body cannot make enough transferrin, a protein that moves iron through the blood. Without it, iron builds up in organs and red blood cells cannot form properly. Researchers are testing intravenous infusions of apotransfe…

Researchers are following 64 people with cerebral adrenoleukodystrophy (CALD) for 15 years after they received a one-time gene therapy called eli-cel in earlier studies. The therapy uses the patient's own stem cells, modified to carry a working copy of the gene that is faulty in …

This study tests an experimental drug called ALN-APP in 200 adults with cerebral amyloid angiopathy (CAA), a condition that causes bleeding in the brain. The drug is given via spinal injection and aims to slow disease progression and reduce new brain bleeds. Participants will be …

This study tests a device called the iLet Bionic Pancreas, which automatically adjusts insulin delivery based on continuous glucose monitor readings, in people with cystic fibrosis-related diabetes. About 150 participants aged 14 and older will either use the bionic pancreas or t…

This study tests a higher dose of the drug aflibercept (8 mg) given as an eye injection every 4 weeks to people with wet age-related macular degeneration (nAMD) or diabetic macular edema (DME). The goal is to see if this dosing schedule is safe and works well for those who have a…

This study tests a new gene therapy called LY3884961 in 32 people with moderate to severe Parkinson's disease who have a specific genetic change (GBA1 mutation). The treatment is given as a single injection into the fluid around the brain and spinal cord. The main goal is to chec…

This study looks at how well a new insulin called icodec controls blood sugar in adults with type 2 diabetes who are switching from their current daily insulin. About 214 participants in Italy will use icodec as prescribed by their doctor for 22 to 30 weeks. Researchers will meas…

This study tests whether the Ask Me 3® educational program, which encourages patients to ask three key questions during doctor visits, can improve blood sugar control in adults with type 2 diabetes. Participants are randomly assigned to receive the program plus standard care or s…

This study tests a new treatment called cipaglucosidase alfa/miglustat in children (0 to under 18 years) with late-onset Pompe disease. The goal is to see if it is safe and how well it works. Participants either have never had enzyme therapy before or have been on it for at least…

This study explores whether eating alkalizing fruits and vegetables, delivered to your home, can help manage metabolic acidosis in people with chronic kidney disease. The usual treatment, sodium bicarbonate, often causes side effects and doesn't always work. The trial will see if…

This study tests whether triheptanoin, a special oil, can reduce major clinical events (like severe low blood sugar or heart problems) in children with long-chain fatty acid oxidation disorders (LC-FAOD), a rare condition where the body can't break down certain fats for energy. A…

This study tests a new medicine called PYC-001, given as an injection into the eye, for people with a genetic condition that damages the optic nerve (OPA1 optic atrophy). The main goal is to check if the treatment is safe and tolerable. About 18 adults will receive a single dose,…

Researchers are following people with chronic kidney disease who received up to four injections of REACT, a therapy made from their own kidney cells, in earlier studies. The goal is to track long-term safety and see how long any effects on kidney function last. About 50 participa…

This study tests an experimental drug called BI 764524, given as an eye injection, in 190 adults with moderate to severe diabetic retinopathy. Participants receive different numbers of injections over a year, and doctors compare eye damage improvements against a sham procedure or…

This study gives the drug metreleptin to 29 people with partial lipodystrophy, a rare condition that causes high blood fats and diabetes. Participants have already shown improvement from the drug in earlier studies. The goal is to keep providing the treatment and check its long-t…

This study tests AIDANET, an automated insulin delivery algorithm, in 40 adults with type 1 diabetes. Participants will use the system in three modes: fully automatic (no meal announcements), hybrid (with meal announcements), or a mix. The goal is to see which mode keeps blood su…

This study looks at whether the MCV test (which measures red blood cell size) is as good as or better than the standard ferritin test for guiding phlebotomy (blood removal) therapy in people with hereditary hemochromatosis, a condition causing too much iron absorption. About 622 …

This study gives the drug metreleptin to people with generalized lipodystrophy, a rare condition that causes severe fat loss and leads to high blood fats and diabetes. Participants, aged 6 months and older, have already received metreleptin in past NIH studies but cannot get it i…

Fanconi anemia is a genetic condition that can damage bone marrow and lead to low blood cell counts, causing anemia, bleeding, or infections. Researchers are testing whether a daily pill called eltrombopag can improve blood counts in people with this condition. The trial enrolls …

This early-stage study tests an experimental drug called ALN-4324 in two groups: healthy overweight volunteers and overweight patients with type 2 diabetes. The goal is to check the drug's safety and how well it controls blood sugar. Participants receive either the drug or a plac…

This study looks at how well a new once-weekly insulin, called insulin icodec, works for people with type 2 diabetes who are switching from a daily insulin. Researchers will track blood sugar control, treatment satisfaction, and how well patients stick to the new regimen over abo…

Steroid injections for knee arthritis can temporarily raise blood sugar in people with type 2 diabetes. This trial tests whether giving a short course of NPH insulin right after the injection can prevent those high blood sugar episodes. Adults with type 2 diabetes and knee osteoa…

Researchers test whether giving mitomycin plus carboplatin before surgery helps people with BRCA1 or BRCA2 mutations and locally advanced triple-negative breast cancer. The trial compares this combination, followed by paclitaxel, against standard chemotherapy. About 30 adults wit…

This study is for people with PMM2-CDG, a rare genetic disorder, who have already taken the experimental drug GLM101 in a previous trial. The goal is to see if GLM101 is safe and effective over a longer period. Participants will receive weekly infusions of GLM101 at the same dose…

This trial tests whether a multilingual interactive voice response (IVR) system can help adults with type 2 diabetes and low health literacy adjust their insulin doses. Participants use a feature phone to report blood sugar levels and receive algorithm-guided dose recommendations…

This study compares two ways of managing gout: a supported self-management approach where patients monitor their own urate levels with a finger-prick test and adjust medication via a smartphone app, versus usual care where a doctor manages treatment. The goal is to see if the sel…

Researchers are testing two doses of an experimental eye implant called EC-104 against the standard Ozurdex implant in adults with diabetic macular edema. The trial enrolls people whose swelling did not improve enough with anti-VEGF injections but who have tolerated steroid eye t…

This early study tests whether QLS-111 eye drops can improve blood flow to the back of the eye in people with non-proliferative diabetic retinopathy or certain types of glaucoma. Fourteen participants will use the drops twice daily for two weeks. The main goal is to measure chang…

This study tests a program called 'Type 1 Doing Well' for youth aged 10–13 with type 1 diabetes and their parents. The program uses a smartphone app, goal-setting, and family meetings to build on the teen's diabetes strengths. Researchers want to see if it improves blood sugar co…

This study tests a device that uses radiofrequency energy to calm overactive kidney nerves, which can drive high blood pressure. About 1,900 adults with uncontrolled hypertension will receive the procedure and be followed for years. The goal is to see if this approach safely and …

This phase 2 study tests acalabrutinib, a targeted drug, in 107 people with Waldenström macroglobulinemia, a rare blood cancer. The goal is to see how well the drug shrinks tumors and controls the disease. Participants either had prior treatment or are new to therapy and cannot t…

Researchers at Stanford University are testing whether a program that combines weekly healthy food deliveries with group lifestyle coaching helps Latino patients with type 2 diabetes. The study enrolls adults who receive care at partner clinics, have type 2 diabetes, and report f…

Researchers are testing whether Chronocort, a modified-release hydrocortisone taken twice daily, controls hormones as well as standard glucocorticoid therapy in people aged 16 and over with congenital adrenal hyperplasia. The trial assigns participants to either Chronocort or the…

This study tests whether cognitive behavioral therapy for insomnia (CBT-I) can help lower blood sugar levels in people with prediabetes. About 300 adults with both prediabetes and insomnia will receive either CBT-I or a patient education program. The main goal is to see if improv…

Researchers are testing whether a structured primary care program can help adults aged 40 to 90 with higher heart or diabetes risk reach recommended prevention targets. The program trains doctors and nurses, uses routine data to flag at-risk patients before visits, and provides d…

This Phase 2 study tests a new injectable drug called maridebart cafraglutide in 409 adults with type 2 diabetes. The main goal is to see if it improves blood sugar control compared to a placebo. Researchers will also check if it helps with weight loss. The trial is active but no…

This study is testing two simple tools to help women with type 2 diabetes better manage their condition and understand reproductive health risks. The tools include a personalized medication list for patients and an alert for doctors during checkups. Researchers will track blood s…

This trial tests whether trained village health workers, using a smartphone app, can provide effective type 2 diabetes care in rural Lesotho. 252 adults with diabetes will either receive community-based care (lifestyle advice, metformin, statins) or be referred to a clinic. The g…

This study tracks 132 people with PNH who are taking Fabhalta to see how safe it is and what side effects occur. Researchers will monitor infections and check for serious red blood cell breakdown if the drug is stopped. The goal is to better understand the drug's risks in everyda…

This study tests two methods for adjusting basal insulin in people with type 2 diabetes who fast during Ramadan. One method follows standard guidelines, while the other uses a personalized carbohydrate fasting test. The goal is to see which approach better controls blood sugar an…

Researchers are studying how the length of bypassed intestine during gastric bypass affects weight loss, complications, and metabolic changes in people with severe obesity. Participants will have different lengths of intestine bypassed and will provide blood and stool samples bef…

Researchers compare a very low calorie ketogenic diet with a Mediterranean-style low-calorie diet in 40 adults who have type 2 diabetes, obesity, and non-alcoholic fatty liver disease. Participants follow one of the two diets for several weeks. The study measures changes in weigh…

This long-term study tests the safety and effectiveness of bitopertin (DISC-1459) in people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), rare conditions that cause severe pain from sunlight exposure. About 230 participants who previously took bitoper…

This early-phase trial tests VX-264, a therapy made from stem cells that produce insulin, in 7 people with long-standing type 1 diabetes. The goal is to see if it is safe and can help control blood sugar. If successful, it may reduce or replace the need for insulin injections.

This study tests a cancer pill called venetoclax in 14 Japanese adults with a rare blood cancer (Waldenström macroglobulinemia) that has come back or not responded to treatment. Participants take the drug by mouth daily, with doses slowly increased. The main goal is to see how ma…

This study tests a drug called BBP-418 (Ribitol) in 81 people aged 12 to 60 with limb girdle muscular dystrophy type 2I (LGMD2I), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo for 36 months to see if it slows the di…

This study is for people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), rare conditions that cause severe pain and skin reactions to sunlight. The purpose is to check the long-term safety of an oral medication called dersimelagon. About 301 participant…

This Phase 3 trial tests a gene therapy called DTX301 for people with late-onset OTC deficiency, a rare genetic disorder that causes dangerous ammonia buildup. The study involves 37 participants and compares the gene therapy to a placebo. The goal is to see if the treatment can h…

This study tests whether delivering healthy food boxes, remote health monitoring, and one-on-one coaching can help low-income Black adults with type 2 diabetes better control their blood sugar. The trial enrolls 304 participants in the Deep South and tracks changes in HbA1c level…

This study follows 17 people with Pompe disease who have already taken part in earlier trials of avalglucosidase alfa (Nexviadyme). It aims to check the drug's long-term safety and effectiveness by giving it every two weeks as an IV infusion. The study will continue until the dru…

This study looks at whether semaglutide, a medicine for type 2 diabetes, can slow or prevent diabetic eye disease over 5 years. About 1500 adults with type 2 diabetes will receive either semaglutide or a placebo injection once a week, in addition to their usual diabetes medicatio…

This Phase 3 study tests whether a daily pill combining obicetrapib and ezetimibe, or obicetrapib alone, can lower LDL (bad) cholesterol more than placebo in 300 people with type 2 diabetes or metabolic syndrome. Participants continue their standard cholesterol medications. The m…

This study tests an experimental oral medication, KAI-7535, taken once daily to help people with obesity or overweight lose weight. About 320 adults with at least one weight-related condition (like high blood pressure or sleep apnea) will receive either the drug or a placebo for …

This study observes how well insulin icodec controls blood sugar in 245 people with type 1 diabetes who have never used it before. Participants take the insulin as prescribed by their doctor, and researchers track changes in HbA1c and treatment satisfaction over about 22 to 30 we…

Researchers are testing tividenofusp alfa (DNL310), an experimental enzyme replacement therapy, against the standard treatment idursulfase in children and young adults with mucopolysaccharidosis type II (MPS II). The study includes participants with and without brain involvement,…

This study looks at how ketogenic diets impact heart health in adults with epilepsy. Researchers will measure cholesterol, blood pressure, weight, and artery thickness over 12 months. Participants are adults 18 or older who are already on a keto diet or starting the modified Atki…

This study looks at whether eating 2 cups of frozen raspberries daily for 8 weeks can improve blood sugar, cholesterol, and gut health in overweight or obese adults with early signs of metabolic syndrome. Researchers want to see if the berries' natural compounds help by changing …

This Phase 3 trial tests a new medicine called MET097, given as a weekly injection, to help adults with overweight or obesity and type 2 diabetes lose weight. About 999 people will take part, with two-thirds getting the real drug and one-third getting a placebo. The study lasts a…

This study tests the long-term safety and effects of JR-141, an enzyme replacement therapy, in people with mucopolysaccharidosis II (MPS II), a rare genetic disorder. Participants who completed an earlier JR-141 trial receive the drug again by IV infusion. Researchers track side …

This Phase 2 trial tests two experimental injections, HRS-7249 and SHR-1918, in 108 adults with severely high triglycerides who are at high risk of acute pancreatitis. The study aims to see if these drugs can safely lower fat levels in the blood and reduce the chance of a pancrea…

This trial tests an experimental enzyme replacement called GC1130A in children with Sanfilippo syndrome type A, a rare genetic disease that damages the brain. The enzyme is given through a small device placed under the scalp that delivers it directly into the fluid around the bra…

This study tests whether taking 50 grams of blueberry powder daily for 8 weeks can improve gut bacteria and metabolic health in overweight or obese adults. Researchers will measure changes in blood sugar, cholesterol, and gut microbes. The goal is to see if blueberries can help m…

This trial tests an experimental drug called JR-441 in children with mucopolysaccharidosis type IIIA (MPS IIIA), a rare genetic condition that harms the brain and body. Researchers give the drug as a weekly intravenous infusion to see if it is safe and tolerable. They also measur…

Researchers are testing a digital program called Centering Appetite in African American adults with type 2 diabetes and binge eating. The program includes web-based lessons to help people relearn their body's hunger and fullness signals, along with diabetes education and a Fitbit…

This study tests a new medicine called cagrilintide to help people who are overweight or have obesity and type 2 diabetes lose weight. About 330 adults will receive either cagrilintide or a placebo for around 18 months. The main goals are to see how much weight they lose and how …

This study looks at whether a team of clinical pharmacists and community health workers can help people with type 2 diabetes improve their blood sugar levels using continuous glucose monitors (CGMs). About 318 adults aged 25-75 with high blood sugar will be enrolled. The goal is …

This study will test whether an artificial intelligence (AI) system can help community doctors better manage patients who have high blood pressure along with diabetes or high cholesterol. About 6,800 adults will be split into two groups: one where doctors use the AI tool to guide…

This trial tests whether an AI system that flags at-risk Medicare Advantage members and suggests care actions, reviewed by a nurse, can reduce overall healthcare spending over a year. About 9,900 participants in Utah are assigned to either this AI-supported navigation or usual ca…

This study tests a new medicine, EYP-1901, given as an eye injection to people with diabetic macular edema (DME), a condition that causes blurry vision in people with diabetes. The goal is to see if EYP-1901 works as well as the standard treatment (aflibercept) but with fewer inj…

This study tests whether simple text messages and online activities can encourage people with Type 2 Diabetes to get more preventive dental care, like cleanings, instead of waiting for expensive treatments. Researchers will track dental insurance claims from 10,000 adults over 18…

This phase 3 trial tests EYP-1901, a new eye injection, against the standard treatment aflibercept for diabetic macular edema (DME), a condition that causes vision loss in people with diabetes. The study will enroll 240 participants and compare how well each drug improves vision …

This phase II trial tests whether adding the targeted drug acalabrutinib to standard chemotherapy (bendamustine and rituximab) improves outcomes for people with untreated Waldenstrom's macroglobulinemia, a rare blood cancer. About 63 participants will receive the three-drug combo…

This study follows about 200 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are taking pegcetacoplan (Aspaveli/Empaveli) as part of their routine care. Researchers will track blood markers, symptoms, and side effects for up to 3 years to see how well the drug works out…

This trial tests whether JR-141, an enzyme replacement therapy given as a weekly IV infusion, can safely manage mucopolysaccharidosis type II (MPS II) over the long term. Researchers will follow about 27 people who completed an earlier JR-141 study, tracking side effects, develop…

This phase 2 trial tests two oral drugs, abrocitinib and ritlecitinib, in 78 people aged 12-35 who were diagnosed with type 1 diabetes within the last 100 days. The goal is to see if these JAK inhibitors can help preserve the body's ability to produce insulin, measured by a stimu…

This phase 2 trial tests whether theophylline, a drug used for asthma, can help people with pseudohypoparathyroidism lose weight and improve blood sugar control. The study includes 29 obese participants aged 13 and older. Researchers will measure changes in body mass index and gl…

This Phase 3 trial tests whether bitopertin, a daily pill, can help people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) spend more time in sunlight without pain. About 183 participants aged 12 and older will take bitopertin or a placebo for 6 months. …

This study is checking if a new insulin pump (NMX8) and continuous glucose monitor (DS5/Simplera Sync) are safe for people with type 1 or type 2 diabetes. About 400 participants, ages 7 to 85, will first wear the sensor for up to 40 days, then use the pump for about 90 days while…

This early-stage study tests a gene therapy called AT845 for adults with late-onset Pompe disease, a condition that weakens muscles. The treatment aims to deliver a working copy of the GAA gene to muscle cells. Eleven participants who have been on standard enzyme replacement ther…

This study tests whether theophylline, a drug used for asthma, can help children and young adults with pseudohypoparathyroidism—a rare genetic condition causing early obesity, hormone problems, and short stature. Researchers will check for weight loss, better blood sugar control,…

This early-stage study tests a new drug, RO7497372, given as an eye injection for people with diabetic macular edema (DME), a condition that causes vision loss. The trial involves 151 participants and aims to check the drug's safety, how long it stays in the eye and blood, and it…

This study tests whether adding a new drug called AZD0780 to the common statin rosuvastatin can lower 'bad' LDL cholesterol more than rosuvastatin alone. About 76 adults with high cholesterol will take either AZD0780 or a placebo pill, plus rosuvastatin, for 12 weeks. Neither par…

This trial tests whether nutrition counseling delivered over video calls can help people with spinal cord injury eat healthier and lower their risk of obesity, high cholesterol, and diabetes. Participants, who are at least one year post-injury and overweight, will receive six ses…

This study investigates whether omega-3 fatty acids, natural fats found in fish and flaxseed, can help manage blood sugar and reduce inflammation in people with type 2 diabetes and heart disease. Participants take omega-3 supplements or a placebo for 28 days, and researchers meas…

This Phase 3 trial tests whether a new drug, tigulixostat, is better than the standard gout medication febuxostat at lowering uric acid levels in Chinese adults with gout. About 600 participants will take one of the two drugs for 24 weeks, then switch to tigulixostat for another …

This study compares a new insulin that is injected once a week (insulin icodec) to a standard daily insulin (insulin glargine) in about 877 adults with type 1 diabetes. The goal is to see if the weekly insulin controls blood sugar just as well as the daily one. Participants will …

This study tests whether providing medically tailored meals along with intensive lifestyle coaching can help adults with HIV and type 2 diabetes or prediabetes better manage their weight and blood sugar. About 200 participants will be randomly assigned to receive either the meals…

This study tests whether giving two immune-calming drugs (rituximab followed by abatacept) can better preserve the body's ability to make insulin in people newly diagnosed with type 1 diabetes, compared to rituximab alone. About 74 participants aged 8 to 45 will receive the drug …

This phase 3 trial tests whether adding enlicitide decanoate to the standard statin rosuvastatin lowers LDL cholesterol more than placebo in 975 adults with high cholesterol. Participants take the drugs or matching placebos for 8 weeks. The main goal is to see the percent change …

This study tests a video-based healthy lifestyle program for adults aged 50 to 74 who are overweight and have conditions like prediabetes, type 2 diabetes, or high blood pressure. Participants use a tablet, scale, and activity tracker at home, and some get extra support from a co…

This trial tests whether community pharmacies can run a produce prescription program for adults with type 2 diabetes or hypertension. Participants receive a monthly card loaded with funds to buy healthy foods like fruits, vegetables, beans, nuts, and seafood at their usual grocer…

This study looks at whether taking omega-3 fatty acid supplements can help lower very high triglyceride levels in pregnant women. High triglycerides during pregnancy can increase the chance of having a large baby. The study involves 40 pregnant women with elevated triglycerides a…

This study tests a new medicine called AR882 to see if it can lower uric acid levels in people with gout. About 750 adults with gout will receive either AR882 or a placebo for 12 months. The main goal is to see if AR882 helps more patients reach a safe uric acid level and reduce …

This trial tests a device called Lixelle, which attaches to a standard dialysis machine to filter out beta-2 microglobulin, a protein that builds up in the blood of people on long-term dialysis and can cause painful joint and bone deposits. The study includes 40 adults on thrice-…

This study looks at the long-term safety of a daily pill called lucerastat for adults with Fabry disease, a rare genetic disorder. About 107 people who completed a previous study will take the drug and be monitored for side effects over several years. The goal is to see if lucera…

Researchers are testing whether having mango before or after a meal changes how the body handles blood sugar and inflammation in people with prediabetes. The study involves 33 adults aged 20 to 60 who are overweight and have elevated fasting glucose or HbA1c. Participants will co…

This study follows overweight and obese adults aged 45-65 to see if a tailored program of counseling, supervised exercise, and digital tools helps them lose weight and improve heart health over 18 months. Participants are compared to a group that only gets general written advice.…

Researchers are testing whether a specially fermented milk drink with probiotic bacteria can improve symptoms of irritable bowel syndrome (IBS) and metabolic syndrome. The study will include 80 adults with IBS or at least one sign of metabolic syndrome, such as high blood pressur…

This trial tests whether a combination of dapagliflozin, saxagliptin, and metformin can help control blood sugar in 600 adults with type 2 diabetes who have controlled cancers. Participants take the drugs by mouth for at least 90 days. Researchers measure blood sugar and insulin …

This early-stage trial is testing whether a rosemary extract supplement can improve markers of oxidative stress and inflammation in people with type 2 diabetes. Forty adults with stable diabetes will take either the extract or a placebo for 12 weeks. The study focuses on blood bi…

This study tests a new drug called EYE103 (Restoret) against an existing treatment, ranibizumab (Lucentis), for people with diabetic macular edema, a condition that causes vision loss in diabetes. About 984 participants will receive injections into the eye every 4 weeks for a yea…

This study looks at whether providing medically tailored groceries can help people with type 2 diabetes and high blood pressure keep up healthy eating habits and control their blood sugar and blood pressure. About 80 adults who already took part in a meal delivery study will rece…

This study tests a new tool called the Snouda Metabolic Score (SMS) that helps doctors identify the specific metabolic problems driving Type 2 Diabetes in each patient. 150 adults will follow a personalized 24-week lifestyle and nutrition plan based on their SMS results. The goal…

This study tests whether a diabetes care program using continuous glucose monitors (CGM) and remote patient monitoring can help adults with uncontrolled type 2 diabetes improve their blood sugar levels and reduce diabetes-related distress. Participants will wear a CGM that shows …

This study tests whether giving people with type 2 diabetes and food insecurity monthly vouchers for fruits and vegetables can improve blood sugar control. Participants receive $100 per month in produce-only gift cards along with dietary education for five months, then switch to …

This trial tests whether adjusting diabetes medications to keep blood sugar levels in the normal range can preserve the body's insulin-producing beta-cells and slow the natural worsening of type 2 diabetes. Adults aged 40 to 75 with type 2 diabetes will be assigned to either usua…

This pilot trial tests whether the timing of carbohydrates and protein across meals can improve glucose metabolism in people with prediabetes or type 2 diabetes who are overweight or obese. Participants follow two different isocaloric diets—one with a high-protein breakfast and h…

This study tests three different non-drug approaches to help older adults in nursing homes manage their type 2 diabetes. One group uses a device that sends mild electrical pulses to the leg muscles, another does supervised exercise, and the third receives health education. The ma…

This study tests whether taking potassium supplements can slow down kidney function loss in people with moderate-to-severe chronic kidney disease (CKD) and high blood pressure. About 532 adults with CKD stage 3b or 4 will receive potassium or a placebo. The goal is to see if pota…

This study tests whether drinking 15 ml of apple cider vinegar twice a day for two weeks can improve blood sugar stability and cholesterol in adults with type 2 diabetes. Thirty-eight participants will compare the vinegar to plain water using a continuous glucose monitor. It's a …

This study is testing a procedure called duodenal mucosal resurfacing (DMR) to see if it can safely improve blood sugar control in people with type 2 diabetes who are not on insulin. About 18 adults will be randomly assigned to receive DMR or a sham (fake) procedure. The study wi…

This trial tests whether an insomnia treatment can improve markers of heart health in adults with insomnia and risk factors for cardiovascular disease. Participants receive either a cognitive-behavioral therapy program for insomnia or a sleep education control. The study measures…

This trial asks whether real-time continuous glucose monitoring (CGM) can improve blood sugar control in adults with type 2 diabetes who do not use insulin. Participants will either use a CGM device or stick to standard self-monitoring with a blood glucose meter. The study tracks…

This trial tests whether providing monthly food vouchers for farmers markets and mailed food stock boxes, alongside diabetes education, can help African American adults with type 2 diabetes and food insecurity achieve better blood sugar control. Participants are randomly assigned…

This trial tests whether proglumide, an older drug that blocks a specific receptor, can safely reduce pain and slow or reverse the scarring seen in chronic pancreatitis. The study includes adults with confirmed chronic pancreatitis and compares proglumide against a placebo over 1…

This study looks at whether adding menopausal hormone therapy to a GLP-1 drug (semaglutide) improves blood sugar control in early postmenopausal women who have or are at risk for type 2 diabetes. About 96 women with menopausal symptoms and a BMI of 27 or higher will be enrolled. …

This trial tests whether a monthly injection of lerodalcibep can safely lower LDL cholesterol in children aged 6 to 17 with heterozygous familial hypercholesterolemia, an inherited condition that causes dangerously high cholesterol. Participants receive either the drug or a place…

This trial tests whether a low-calorie or low-carbohydrate diet can help people with type 2 diabetes achieve remission—meaning blood sugar returns to normal without medication. One hundred adults aged 20–80 with type 2 diabetes and a BMI of 27 or higher will follow a structured d…

This study tests whether student pharmacists can help older Asian adults (age 50+) with uncontrolled type 2 diabetes improve their blood sugar through lifestyle coaching, education, and adherence support. Participants will receive non-pharmacological guidance from student pharmac…

This study tests whether a digital platform called Haibu Diabetes helps children with type 1 diabetes manage their condition better. The platform collects health data from devices and shares it with patients, caregivers, and doctors through a single dashboard. Researchers will co…

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

This study tests an experimental drug, NPI-001 (AT-001), in people with hereditary cystatin C amyloid angiopathy (HCCAA), a rare genetic condition that causes repeated brain bleeding and stroke. The trial includes about 15 participants aged 12 and older who carry the specific L68…

This study tests whether semaglutide, a drug already used for obesity and type 2 diabetes, can reduce liver scarring in people with non-alcoholic fatty liver disease (NAFLD). Participants are adults aged 40 to 80 who are overweight or have type 2 diabetes. The trial will also val…

This study investigates whether adding two common drugs—valsartan (for high blood pressure) and celecoxib (for arthritis pain)—to standard metformin therapy can improve blood sugar control in people with type 2 diabetes. The trial enrolls adults with obesity, high blood pressure,…

This study tests whether transferring gut bacteria from a healthy donor (fecal transplant) can improve blood sugar control in 16 adults with type 2 diabetes. Participants receive either the transplant or a placebo, and researchers measure insulin sensitivity and blood sugar level…

This study tests a new treatment called NXC-201 CAR-T for people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. The therapy uses a patient's own immune cells, modified to target and destroy the cells producing these proteins. The trial is open to …

This phase 2b trial tests whether adding ALXN2220 to eplontersen works better than eplontersen alone for adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart. About 326 participants will receive either the combinati…

This trial tests a culturally adapted program called Health Without Barriers/Salud Sin Barreras for teens aged 11–19 and their families in Northern Colorado. The program combines mindfulness, nutrition education, physical activity, and parenting support over several sessions. Res…

This phase 2 trial tests whether a daily low dose of colchicine, an anti-inflammatory drug used for gout, can reduce inflammation and heart disease risk in adults with type 1 diabetes. 102 participants aged 35–80 will take either colchicine or a placebo for 26 weeks, with an opti…

This study tests whether people with type 2 diabetes who take their usual metformin on the morning of surgery have better blood sugar control and fewer problems like infections. Participants are randomly assigned to receive either metformin or a placebo before their operation. Th…

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

This study investigates whether SGLT2 inhibitors, a class of diabetes medications, can slow cognitive decline in people aged 50 and older with type 2 diabetes. Participants are randomly assigned to receive either standard diabetes care plus an SGLT2 inhibitor (empagliflozin or da…

This study tests whether a smart order set in electronic health records can help primary care doctors better manage blood sugar in people with type 2 diabetes who use insulin. About 150 participants will use continuous glucose monitors, and doctors will get decision support from …

This study looks at whether a special program called Primary Care Connect (PC2) helps cancer survivors who also have conditions like high blood pressure, diabetes, or high cholesterol keep their heart risks under control during cancer treatment. About 266 adults with early-stage …

This phase 2 trial tests an oral drug called venglustat, given alongside the standard enzyme therapy Cerezyme, in adults with Gaucher disease type 3 (a rare genetic disorder affecting the body and brain). The study has four parts: first, it checks spinal fluid biomarkers to disti…

This study tests whether a culturally tailored plant-based diet, guided by community health workers, can help Latino adults with obesity lose weight and improve metabolic health. Forty participants will be randomly assigned to either the plant-based diet program or standard healt…

This study is testing different combinations of diet and exercise to find the best weight loss plan for people with prediabetes who also have physical or mobility disabilities. Thirty participants will start with either a high or reduced carbohydrate diet. After four weeks, those…

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

This study will test whether sending reminders by text, email, or secure patient portal messages helps people with heart disease or diabetes refill their statin prescriptions. About 22,000 adults who have not refilled their statin in the last 3 months will receive one of these me…

This trial tests whether a chelation therapy infusion—a cocktail that removes toxic metals from the blood—can reduce the risk of heart attacks, strokes, and leg amputations in people with diabetes and critical limb ischemia (severe blockage in leg arteries). Half of the 50 partic…

This study tests whether giving financial incentives — including monthly cash, rewards for buying healthy food, and bonuses for lowering blood sugar — can improve diabetes control in adults who also struggle with food insecurity. Participants receive diabetes education and are ra…

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

This study tests a program called Share plus for adults aged 60+ with type 1 diabetes and their care partners. The program helps them better use continuous glucose monitor data together. Researchers want to see if it improves blood sugar control and reduces diabetes-related stres…

This study tests a culturally tailored education program for African Americans with type 2 diabetes. The program teaches resilience and self-management skills through group sessions led by nurses, plus support groups and booster sessions. Researchers are tracking blood sugar leve…

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

This study is testing whether a daily semaglutide tablet can safely reduce the risk of heart attacks, strokes, and related deaths in over 21,000 adults with type 2 diabetes who have never had a heart attack or stroke. Participants take either semaglutide or a placebo pill for sev…

This phase 3 study tests whether venglustat, an experimental oral drug, can slow heart thickening better than current standard treatments in 104 adults with Fabry disease. Participants are randomly assigned to venglustat or usual care (enzyme replacement or migalastat) for 18 mon…

This study tests whether a short-term program combining health coaching and medication can help people with type 2 diabetes achieve remission without surgery. It involves 118 adults diagnosed within the last 5 years, recruited from a primary care clinic in Hamilton, Ontario. The …

This study is testing the long-term safety and effectiveness of an experimental drug called XH-S003 in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that can cause fatigue, blood clots, and other serious problems. About 26 people who have already ta…

This study tests whether switching to avalglucosidase alfa is safe and more effective for people with late-onset Pompe disease whose condition has worsened on the standard treatment, alglucosidase alfa. Participants receive biweekly infusions of the new drug and are monitored for…

This study is testing whether a combination of two drugs, sotagliflozin and volagidemab, can improve blood sugar control in adults with type 1 diabetes. The 24 participants will receive both drugs or a placebo in a crossover design. The goal is to see if this approach can help ma…

This Phase 3 trial tests whether a daily injection of metreleptin (a lab-made leptin hormone) can improve blood sugar and fat levels in people with partial lipodystrophy, a rare condition where fat is distributed abnormally. About 69 participants will receive either metreleptin o…

This study compares two types of weight-loss surgery (gastric bypass and gastric banding) with an intensive diet and exercise program for controlling type 2 diabetes in moderately obese adults aged 25 to 55. The goal is to see which approach works best for improving diabetes and …

This study was designed to see if taking metformin or salsalate on the morning of surgery could help control blood sugar and reduce complications in people with type 2 diabetes. It planned to enroll patients having elective surgery who regularly take metformin. However, the trial…

This study looks at whether using a continuous glucose monitor (CGM) along with a mobile app can help people with type 2 diabetes control their blood sugar better than standard care. Sixty adults will be randomly assigned to either the digital tool group or usual care, and their …

This study tests whether time-restricted eating (eating all meals within an 8-10 hour window each day) can help people with type 2 diabetes better control their blood sugar and improve heart health. About 60 adults with type 2 diabetes will either follow their usual care or add t…

This study tests whether using a blood marker called transferrin saturation to guide bloodletting (removing blood) improves quality of life in people with HFE haemochromatosis, a genetic condition causing iron buildup. 239 adults already on maintenance bloodletting will be random…

This study tests whether an AI-based counseling program on a smartphone can help women with gestational diabetes manage their condition. 80 pregnant women will either get standard nutrition advice or AI-powered guidance. The goal is to see if the AI approach leads to healthier bi…

This study tests a new program where schools, families, and doctors work together to help children manage type 1 diabetes. About 30 kids will take part to see if the program is practical and well-liked. The goal is to improve daily diabetes care at school.

This study tests a one-time gene therapy infusion (FBX-101) given after a standard stem cell transplant in 6 children with infantile Krabbe disease, a severe genetic disorder affecting the nervous system. The therapy uses a harmless virus to deliver a working copy of the GALC gen…

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

This study tests whether monthly home visits from trained health agents can help people in poor, high-altitude communities in Peru better control their high blood pressure and diabetes. Over 1,000 participants will either receive these visits for a year or get usual care. The goa…

This study tests whether using a continuous glucose monitor (CGM) along with remote dietitian support helps people with type 2 diabetes better control their blood sugar. About 160 adults across Australia will use a CGM and receive online dietary advice for several months. The goa…

This study tests the long-term safety and effectiveness of pegtibatinase in people with classical homocystinuria (HCU), a rare genetic disorder that prevents the body from breaking down certain amino acids. About 100 participants who completed earlier studies will receive the dru…

This study follows 1,600 people with type 1 diabetes, including young children aged 2-6, who use hybrid closed-loop insulin pumps (Medtronic, Tandem, or Omnipod). These devices automatically adjust insulin based on blood sugar readings but still require users to enter meal inform…

This study is testing whether a real-time continuous glucose monitor (CGM) can help 200 adults with type 2 diabetes who use insulin in Saudi Arabia. Participants wear a CGM device that tracks blood sugar levels continuously. The main goal is to see if it improves HbA1c, a measure…

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

This study tests a culturally tailored medical nutrition therapy for African patients with type 2 diabetes. The program includes personalized meal plans, nutrition counseling, and group education. Researchers will measure blood sugar levels and diet adherence after 6 months, and …

This study looks at whether supervised exercise classes or doing physical activity on your own works better for controlling blood sugar in adults with type 2 diabetes. About 120 people whose diabetes is not well controlled with diet or non-insulin medications will take part. Part…

This study tests a drug called crinecerfont in adults with classic congenital adrenal hyperplasia (CAH), a genetic condition that requires lifelong steroid treatment. The goal is to see if crinecerfont can safely reduce the daily steroid dose needed while keeping hormone levels u…

This Phase 2 trial tests whether adding carfilzomib to ibrutinib improves outcomes for people with Waldenström's macroglobulinemia, a rare blood cancer. About 99 participants will receive either the combination or ibrutinib alone. The goal is to see if the combo leads to higher r…

This trial tests an AI chatbot called Gout Buddy that gives personalized diet advice to people with gout. 110 participants will either use the chatbot or receive standard care. The goal is to see if the chatbot improves dietary knowledge, eating habits, medication adherence, and …

This study tests a community-based program to help Black and Latino adults with diabetes, prediabetes, high blood pressure, or obesity. Participants receive lifestyle coaching, help connecting to health and social services, and digital skills training. The goal is to see if this …

This study tests whether an AI tool that shows patients how their blood sugar control compares to others, combined with home monitoring, can improve risk perception and blood sugar levels in adults with poorly controlled type 2 diabetes. About 360 participants aged 36–65 will be …

This early-stage trial tests a new drug called VSA012 in about 50 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study includes people who have not used complement inhibitors before or who still have low hemoglobin …

This study compares a lower dose of insulin (5 units) plus dextrose to the standard dose (10 units) for treating mild to moderate high potassium in emergency patients. Researchers want to see if the lower dose works just as well while causing fewer side effects like low blood sug…

This study tests whether a mobile app that trains girls to manage food cravings can help them eat more fruits and vegetables. About 150 girls aged 14-18 will use the app twice a week for a year. Researchers will track diet quality, weight, and blood sugar to see if the app leads …

This study follows 60 adults with Fabry disease who are receiving pegunigalsidase-alfa, a newer enzyme replacement therapy. Researchers want to see how well it works in everyday medical practice, especially for kidney function. Participants will be treated for 2 years at speciali…

This phase 2 trial tests whether ixekizumab (Taltz), a drug already approved for psoriasis, can help people newly diagnosed with type 1 diabetes keep making their own insulin. The study involves 127 adults aged 18-45 who were diagnosed within the last 100 days. Participants recei…

This study tests whether giving pregnant women a daily vitamin B12 supplement (50 micrograms) from early pregnancy until six months after birth helps their children grow and think better. Researchers are enrolling 800 pregnant women in Nepal who are at risk of low B12 levels. The…

This study tests a new daily pill, aleniglipron, in 58 adults with type 2 diabetes who are overweight or obese. The main goal is to check if the drug is safe and tolerable at different doses. Researchers will also measure weight loss and blood sugar changes. The study is not yet …

This study tests whether a diet with more protein (25% of daily calories) helps children aged 7-14 with overweight or obesity lose weight and reduce diabetes risk. Over 10 weeks at a lifestyle camp, one group eats a higher-protein diet while another eats a lower-protein diet, bot…

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

This study planned to test whether a virtual weight control program (WW, formerly Weight Watchers) could help adults with type 2 diabetes and obesity lower their blood sugar levels. Participants would have used the WW SmartPoints system and attended weekly online workshops. Howev…

This study looks at whether using an over-the-counter continuous glucose monitor (CGM) every 30 or 90 days can help people with type 2 diabetes who do not use insulin better manage their blood sugar. About 188 participants will wear the Dexcom Stelo device periodically over six m…

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

This study tests whether a pre-filled syringe is as safe as the traditional vial when giving the medicine bevacizumab for certain eye diseases. About 120 people with wet age-related macular degeneration, diabetic macular edema, or branch retinal vein occlusion will receive inject…

This early-stage trial is studying whether an experimental drug called AZD6793 changes how the common diabetes medicine metformin works in people with type 2 diabetes. About 28 participants will take both AZD6793 and a placebo alongside their usual metformin to compare effects on…

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

This study compares two ways of giving faricimab, a drug injected into the eye, to treat diabetic macular edema (DME). One group gets injections on a fixed monthly schedule, while the other uses a 'treat and extend' approach where the time between injections is gradually lengthen…

This study tests whether regular warm-water baths can improve blood sugar control and brain energy use in healthy adults aged 65 and older who are at risk for Alzheimer's disease. Over 10 weeks, 60 participants will soak in warm water three times a week. Researchers will measure …

This study looks at whether doing light aerobic exercise five days a week can improve heart rate variability—a sign of heart health—in adults with type 1 diabetes. Over one year, 35 participants were split into two groups: one exercised regularly, the other less often. Researcher…

This study looked at whether time-restricted eating (eating only during a set window each day) helps overweight or obese people with prediabetes return to normal blood sugar levels. Over 12 months, 142 participants either followed this eating pattern or received standard care. Th…

This study tests a daily pill (obicetrapib plus ezetimibe) to see if it can reduce fatty plaque buildup in the arteries of 300 adults with heart disease. Participants must already be on cholesterol-lowering therapy but still have high LDL cholesterol and measurable plaque. The tr…

This study follows 433 people with chronic pancreatitis who undergo a surgery called TPIAT, where the entire pancreas is removed to stop pain and the patient's own insulin-producing islet cells are infused back to lower diabetes risk. Researchers track pain relief, quality of lif…

This study tests whether a smartphone app called Hedia Diabetes Assistant can help adults with type 1 diabetes keep their blood sugar in a healthy range more often. About 154 people with poorly controlled diabetes will either use the app plus their usual care or just usual care f…

This study tests a new enzyme replacement therapy called avalglucosidase alfa in babies with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and breathing problems. The treatment is given through an IV every other week for up to 4 years. …

This study tests whether levodopa, a drug used for Parkinson's, can slow blood vessel changes in the eyes of people with diabetes. Researchers will give the drug or a placebo to 244 patients with early diabetic retinopathy and measure retinal electrical activity and imaging over …

This study tests a rapid drug desensitization (RDD) protocol for adults with phenylketonuria (PKU) who have had allergic reactions to Palynziq. Over one day, patients receive gradually increasing doses to retrain their immune system. The goal is to see if they can safely restart …

This study tests whether a hybrid closed-loop insulin pump system (mylife CamAPS FX) can improve blood sugar control and quality of life in 30 adults with type 1 diabetes who have chronically high blood sugar and psychological challenges. Participants either start the pump immedi…

This study tests a special balloon designed to open up hardened leg arteries in people with peripheral artery disease. About 58 participants with moderate to severe calcium buildup will receive the balloon treatment. Researchers will check if the balloon successfully widens the a…

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

This study tests whether high-dose nicotinamide (vitamin B3) is safe and can help people with dominant optic atrophy, a rare genetic disease that slowly damages the optic nerve and causes vision loss. Researchers will give 25 adults 3 grams of nicotinamide daily and monitor for s…

This study is testing whether daily shakes made from bilberries and oats can lower LDL cholesterol and improve exercise capacity in people who have had a heart attack or have type 2 diabetes. 900 participants will drink either a bilberry shake, an oat shake, a combination shake, …

This study looks at whether treating patients at home for conditions like infections, heart failure, diabetes, and COPD can lead to better health outcomes and satisfaction compared to traditional hospital stays. Nearly 10,000 adults will receive hospital-level care at home, inclu…

This study tests a higher dose (8mg) of aflibercept in 40 adults with proliferative diabetic retinopathy (PDR) who do not have swelling in the center of the retina. The goal is to see if this treatment can improve the severity of the disease and maintain vision over 96 weeks. Par…

This study tests an experimental drug called iopofosine I 131 in people with certain blood cancers (like Waldenstrom macroglobulinemia, multiple myeloma, and lymphomas) that have come back or not responded to standard treatments. The drug delivers radiation directly to cancer cel…

This study was designed to test whether a program called Lifestyle MIND can help people with serious mental illness (like schizophrenia or bipolar disorder) prevent or manage type 2 diabetes. The program includes group exercise and health classes twice a week for 10 weeks. Howeve…

This study tests a mobile phone program (MOSAIC) that changes over time to fit each person's needs, helping adults manage type 2 diabetes. About 547 participants will be assigned to either the phone program or printed materials for 16 months. Researchers will check blood sugar le…

This study tests whether a drug called finerenone can reduce scarring and fat buildup in the heart muscle of people with diabetic kidney disease and heart failure. About 40 adults will take the pill daily for 6 months and get advanced heart scans to see if their heart structure i…

This phase 2 study tests pembrolizumab (Keytruda) in 40 men with metastatic castration-resistant prostate cancer that has specific genetic flaws (mismatch repair deficiency or CDK12 inactivation). The goal is to see if the drug can shrink tumors or slow the cancer. Researchers wi…

This study tests whether adding the drug abatacept to standard care can prevent graft-versus-host disease (GVHD) in children receiving stem cell transplants from unrelated donors. GVHD occurs when donor cells attack the patient's body, causing serious illness. The trial will enro…

This study tests a new way for cancer doctors and primary care doctors to work together to help cancer survivors manage other health problems like high blood pressure, diabetes, and high cholesterol. About 800 adults with certain cancers and at least one of these conditions will …

This study tracks 73 adults with homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder causing extremely high cholesterol and early heart disease. Researchers will compare major cardiovascular events (like heart attacks and strokes) during the first three years…

This study tests whether a specially designed app can help women who had high blood pressure or diabetes during pregnancy get the recommended heart check-ups after giving birth. About 400 women from Oslo University Hospital will either use the app or receive standard care. The go…

This study tests a drug called Aramchol for people with nonalcoholic steatohepatitis (NASH), a serious fatty liver disease that can lead to scarring. About 150 adults with NASH and liver scarring (stages 1-3) will take Aramchol or a placebo for up to 72 weeks. The goal is to see …

This study tests whether a 12-week program of very-low-calorie diet or high-intensity exercise can improve blood sugar control in adults aged 40-70 who are newly diagnosed with type 2 diabetes and overweight. Participants are randomly assigned to diet, exercise, or standard educa…

This study compares two weight loss surgeries for people with severe obesity: sleeve gastrectomy with transit bipartition (SG+TB) and Roux-en-Y gastric bypass (RYGB). About 320 participants will be followed for two years to see which surgery leads to greater weight loss and fewer…

This early-stage trial tests a gene therapy called AAV9.hMCOLN1co in one child with Mucolipidosis Type IV (MLIV), a rare genetic disorder. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety, but researchers will also look for any si…

This early-phase study tests an experimental oral drug (GSK4418959) in adults with advanced solid tumors that have specific genetic changes (dMMR or MSI-H). The drug works by blocking a protein (WRN) that these tumors need to survive. The study aims to see if the drug, alone or w…

This study tests a drug called teplizumab in 20 children under 8 who have early signs of type 1 diabetes (Stage 2). The goal is to check if the drug is safe and how the body handles it. Teplizumab works by calming the immune system to protect insulin-making cells. If successful, …

This early-stage trial tests a single dose of VTX-801 gene therapy in 4 adults with Wilson's disease, a rare condition where copper builds up in the body. The goal is to see if the treatment is safe and can help control copper levels, possibly allowing patients to stop their usua…

This study tests a new medicine called PRAX-562 in 77 children with rare genetic epilepsies (SCN2A or SCN8A). The goal is to see if it safely reduces motor seizures. The trial has two parts: a double-blind phase where some children get the drug and some get a placebo, followed by…

This study tests if weight-loss surgery (sleeve gastrectomy) can help people with newly diagnosed type 2 diabetes and a BMI of 30-42 achieve diabetes remission without medication. About 30 adults will be randomly assigned to either surgery or standard medical care and followed fo…

This study looks at whether adding the drug insulin detemir to a low-calorie diet helps obese adults with type 2 diabetes lose more weight and improve brain function related to food reward. About 240 people will be split into two groups: one on diet only, the other on diet plus d…

This study tests whether an electronic alert in the medical record can help doctors prescribe better cholesterol-lowering medications for heart disease patients with high LDL. About 400 patients from a single hospital will be randomly assigned to have their doctor receive the ale…

This study tests whether regular exercise and cognitive training, alone or together, can improve physical and mental function in adults aged 60+ who have had a stroke and also have high blood pressure or diabetes. Over 400 participants from five Chinese cities will take part in 1…

This study tests whether fenofibrate, a drug typically used to lower blood fats, can slow the worsening of diabetic retinopathy (eye damage) in adults with type 1 diabetes. Over 400 participants take either fenofibrate or a placebo daily for three years. The goal is to see if the…

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that can cause dangerous blood clots. Researchers want to know how often PNH appears in people under 50 who have had a stroke, mini-stroke, or brain vein clot with no clear cause. The study will screen 200 partici…

Researchers are testing whether a brain imaging measurement can predict how strongly the body reacts to low blood sugar. The study enrolls healthy adults aged 18 to 40 with a body mass index between 20 and 30. Participants undergo two procedures: a brain scan to measure glial ace…

This study tests a new way to screen for type 2 diabetes by measuring sugar levels in fingernails. Researchers will compare results from 100 adults (half with known diabetes, half without) to standard blood tests. The goal is to see if this simple, non-invasive method can accurat…

This study tests whether special lights and cameras can help find early signs of mouth cancer in people at high risk. About 338 participants with precancerous spots or conditions like Fanconi anemia will have their mouths examined with fluorescence imaging, which makes abnormal c…

Researchers at Beijing Anzhen Hospital are studying whether a computer model that combines two types of heart scans can predict 12-month survival in people with light-chain cardiac amyloidosis, a condition where abnormal proteins stiffen the heart. The study enrolls 49 patients w…

This study is testing a mixed meal test to better diagnose a specific type of diabetes (type 3c) that occurs in people with pancreatic cancer or chronic pancreatitis. Researchers will enroll 775 adults aged 30 to 84 who have new-onset diabetes and one of these pancreatic conditio…

This study will test if artificial intelligence (AI) can spot a rare heart condition called ATTR-CM in people with heart failure. Researchers will use AI to analyze electrocardiograms (ECGs) and echocardiograms (Echo) from 150 adults. The AI's findings will be checked against a s…

This study is testing whether artificial intelligence can detect transthyretin amyloid cardiomyopathy (ATTR-CM), a serious and often underdiagnosed heart condition, earlier than usual. Researchers will use AI algorithms to analyze routine ECGs and heart ultrasounds from up to 1.5…

This study looks for tiny particles in the blood, called exosomes, that might signal the start of type 1 diabetes. Researchers will compare children recently diagnosed with type 1 diabetes to healthy children, all aged 12 to 18. The goal is to find a better way to catch the disea…

This study looks at whether two blood markers, PLIN2 and RAB14, can accurately diagnose NASH (a serious liver disease) and advanced scarring, without needing a liver biopsy. Researchers will test these markers in 828 adults with NASH, type 2 diabetes, or obesity. If successful, t…

This study is testing whether an artificial intelligence (AI) program can spot early signs of diabetic retinopathy—a condition that can lead to blindness—in people with prediabetes and diabetes. Researchers will take pictures of the retina and use the AI to analyze them. The goal…

This study explores whether a community-based cooking program can help African-American adults at risk for heart disease cook and eat healthier. About 43 participants from Washington, D.C. will take surveys, attend cooking classes led by a chef, and track their eating habits. The…

Women who develop gestational diabetes are far more likely than other women to develop type 2 diabetes within a few years after giving birth. This trial tests whether a smartphone app that delivers personalized coaching on diet, exercise, sleep, and mental well-being can help pre…

Researchers at Cornell University are testing whether a produce prescription program helps adults who have diet-related conditions and trouble affording food. Participants receive weekly boxes of fresh local produce for 8 to 24 weeks and attend eight nutrition education classes. …

This large Phase 3 study tests whether inclisiran, a twice-yearly injection, can prevent major heart problems like heart attacks, strokes, and heart-related death in people at high risk who have not yet had such an event. Over 14,000 participants are receiving either inclisiran o…

This study tests whether screening children with a high genetic risk for type 1 diabetes, along with regular follow-up and family education, leads to an earlier diagnosis of the disease. Researchers will track 600 children to see if this approach prevents severe symptoms and diab…

This study tests whether sending personalized screening invitations based on a person's race, ethnicity, and diabetes risk, plus offering phone navigation for those who don't respond, leads to more people getting tested for diabetes compared to generic invitations or usual care. …

This trial tests whether adding a real-time continuous glucose monitor (CGM) to a YMCA healthy lifestyle program helps adults with prediabetes improve their glucose levels, diet, and physical activity. Participants attend health coaching sessions and YMCA classes, with some weari…

This pilot study will test whether providing Mediterranean diet groceries plus virtual nutrition classes helps overweight adults at risk for type 2 diabetes improve their diet and lose weight. Forty participants in the US South will be randomly assigned to receive either weekly g…

This trial tests whether giving women who had gestational diabetes a continuous glucose monitor and a FitBit can help them adopt healthier habits and prevent type 2 diabetes. Participants will wear the sensor for up to 14 days and use the tracker to monitor their activity. The st…

This trial tests whether a comprehensive lifestyle program, called HELMS, can improve the health of overweight women and their children. The program starts before conception and continues through pregnancy and the first 18 months of the child's life, including health screenings, …

This trial tests whether adding recreational basketball to the standard National Diabetes Prevention Program (NDPP) helps adults aged 18-44 with prediabetes lose more weight, lower blood sugar, and become more active. Participants are randomly assigned to either the standard prog…

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

This trial tests a wearable insole system that monitors foot pressure, temperature, and movement, giving real-time alerts to help users shift weight and avoid injury. The study includes people with type 1 or 2 diabetes who have nerve damage and a recent history of healed foot ulc…

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

This study looks at whether giving children with a genetic risk for celiac disease a low-gluten diet (less than 3 grams per day) during their first three years of life can lower their chance of developing the disease by age 7. About 1,100 children from Sweden are taking part. The…

This study tests whether the BCG vaccine, normally used for tuberculosis, can prevent type 2 diabetes in people with pre-diabetes. Researchers in Guinea-Bissau will give two doses of BCG or a placebo to 200 adults and track their blood sugar levels. The goal is to see if the vacc…

This study tests a virtual behavioral therapy (Functional Behavioral Training) for children aged 2-12 with genetic syndromes like Fragile X, Angelman, or Rett syndrome who have challenging behaviors. The therapy teaches parents how to identify what triggers problem behaviors and …

This study tests a group counseling program called Healing HEARTS to help people with conditions like obesity, skin disease, cancer, HIV, diabetes, or chronic pain deal with internalized stigma. Participants will either join the counseling program or a peer support group. The goa…

This pilot study tests MPowerHub, a smartphone app that helps adults with type 2 diabetes make sense of data from continuous glucose monitors and activity trackers. The app shows combined trends and prompts daily questions about diet, sleep, stress, and medication. Researchers wa…

This study tests whether adding mental health care to standard diabetes treatment improves mood and blood sugar control in adults with type 1 diabetes. Eighty participants will be randomly assigned to usual care or a collaborative care model that includes a behavioral health cons…

This study compares high-effort versus low-effort resistance exercise sessions to see which better controls blood sugar in people with prediabetes or type 2 diabetes. Fifteen participants will complete both types of sessions plus a no-exercise control day, with blood sugar monito…

This study looks at how dried figs affect blood sugar and insulin in adults with pre-diabetes. Five participants will eat figs or a calorie-matched control breakfast, and researchers will measure blood glucose, insulin, and fig polyphenol levels over 24 hours. The goal is to see …

This pilot study tests a mobile phone program called STRIDE for adults with type 2 diabetes and a close friend or family member. The program includes monthly coaching calls and text message support to help with medication and goals. The study will enroll 180 people to see if the …

This pilot study tests a training program designed to improve eHealth literacy—the ability to find and use health information online—among Bangladeshi adults with type 2 diabetes in Ancona, Italy. The program, co-designed with patients and community members, includes four modules…

This study tests a personalized workplace intervention for 124 Dutch workers with Parkinson's disease, cerebellar ataxia, hereditary spastic paraparesis, or slowly progressive neuromuscular/mitochondrial disorders. A trained facilitator helps employees and their managers identify…

This study tests a remote education program called REST, designed to help adults aged 65 and older with insulin-treated diabetes start and keep using continuous glucose monitors (CGMs). The goal is to reduce dangerous low blood sugar episodes and improve overall glucose control. …

This trial tests whether giving older adults with diabetes and other chronic conditions audio recordings of their clinic visits helps them take a more active role in their own care. Participants either receive these recordings for a year or standard after-visit summaries. The stu…

This study tests a non-invasive light therapy called photobiomodulation (low-level laser) for people with diabetic peripheral neuropathy—nerve damage causing pain, tingling, and numbness in the feet. 36 adults with type 2 diabetes and foot pain for at least 6 months will receive …

This study tests whether a prebiotic supplement called galacto-oligosaccharide (GOS) can lower blood sugar spikes after eating. Fifty healthy adults will drink either a GOS supplement or a glucose control beverage, and their blood sugar and insulin levels will be measured over se…

This study tests a self-guided digital program called covivio to help adults with type 2 diabetes manage emotional distress tied to their condition. About 250 participants will either use the app alongside their usual care or continue with usual care alone. The goal is to see if …

This study tests new vitamin and mineral supplements called EasiVits for children aged 1-16 with inborn errors of metabolism (IEM). These supplements have less carbohydrate and no flavor, so they can be used with different diets and kids can add their own taste. The goal is to se…

This study compares a new numbing gel (chloroprocaine HCl 3%) to standard eye drops plus a shot for pain relief during eye injections. About 236 adults with conditions like diabetic macular edema or age-related macular degeneration will take part. The goal is to see if the gel wo…

This study is testing whether a 12-week physiotherapy and exercise program can improve physical fitness and quality of life in adults with Fabry disease. Fifteen participants will do regular exercise sessions using body weight, resistance bands, and increased daily activity. The …

This study looks at whether online community support and telemedicine can improve quality of life for people with Parkinson's disease, Lewy body dementia, and related conditions, as well as their caregivers. About 632 participants will receive palliative care training for their d…

This study was designed to see if two drugs, empagliflozin and anakinra, could help people with prediabetes who experience low blood sugar after eating. The trial planned to give a single dose of each drug before a test meal and measure symptoms. However, the study was withdrawn …

This study tests whether venglustat can reduce nerve pain in the arms and legs, and belly pain, in people with Fabry disease. Participants are 16 or older and have not had Fabry treatment for at least 6 months. The trial lasts 12 months, with visits every 3 months, and compares v…

This study tests if anakinra is better than prednisone for treating gout flares in people with advanced chronic kidney disease or who have had a kidney transplant. Gout causes sudden, severe joint pain. Standard treatments can be risky for these patients. The study measures pain …

This study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …

This study tests a new way of caring for children under 10 with diabetes. The clinic uses play, nutrition advice, mental health support, and parent groups to help families feel calmer and more in control. About 100 children and their families will take part to see if this approac…

This study looks at people with late-onset Pompe disease to measure antibodies against a virus used in gene therapy and against the standard enzyme replacement therapy. It also checks for biomarkers in blood and urine. About 119 teenagers and adults will give samples over 2 years…

The Austrian LEAD study follows a large group of people to understand how lung, heart, and metabolic health change over time. Researchers measure lung function with spirometry and track body composition and metabolic markers. By observing this group, they aim to learn how these c…

Researchers in Argentina are developing and testing a self-administered questionnaire to measure how hereditary transthyretin amyloidosis affects patients' physical, emotional, and social quality of life. The study includes input from neurologists, a cardiologist, a psychologist,…

This study checks if a Turkish version of a questionnaire about managing low blood sugar (hypoglycemia) is accurate and reliable. Researchers will ask 200 adults with type 2 diabetes who have had low blood sugar to fill out the questionnaire. The goal is to make sure the scale wo…

This early-stage study looks at how taking a new tablet called CX11 with or without food changes the amount of drug in the blood. It involves 32 healthy adults and does not aim to treat any disease. The results will help researchers decide how to give the drug in future studies f…

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

This study aims to identify genetic, biochemical, and clinical factors linked to disease severity in people with Gaucher disease and other lysosomal storage disorders. Researchers will evaluate up to 1,000 participants, including patients, carriers, and healthy controls, to bette…

This study is looking at how nerve pain (neuropathic pain) affects the ability to care for oneself and overall quality of life in people with type 2 diabetes. Researchers will measure pain levels and quality of life in 128 adults with type 2 diabetes. The goal is to better unders…

This study looks at the genetic and molecular factors behind type 2 diabetes, obesity, and related complications like kidney disease. Researchers will collect health information, blood and urine samples, and perform tests like eye exams and ultrasounds from over 2,300 adults, foc…

This long-term study looks at people and families with a higher chance of developing blood or lymph node cancers like leukemia or lymphoma. Researchers collect medical histories, genetic samples, and sometimes perform exams to find inherited genes or environmental triggers. The g…

This long-term study follows over 3,700 adults in Baltimore to understand why some people age healthier than others. Researchers are looking at how race, income, and neighborhood affect risks for diseases like diabetes, heart disease, and memory loss. Participants visit mobile he…

This pilot study looks at biomarkers in the blood of people with fragile sarcolemmal muscular dystrophy, a condition that makes muscle membranes weak. Researchers will collect blood samples at rest and after exercise during four 5-day hospital stays. The goal is to better underst…

This pilot study is testing a device called LabClasp that measures blood sugar without needles. Researchers want to see if regular, non-invasive glucose checks can help predict gestational diabetes and preeclampsia in pregnant women. The study plans to enroll 100 women with at le…

This study follows 500 obese adults to see how different factors like diet, exercise, stress, and sleep affect weight loss. Researchers collect blood tests, measurements, and questionnaires at the start and during follow-up. The goal is to find patterns that could lead to more pe…

Researchers are checking whether tests of muscle strength, walking, balance, and fine motor skills give consistent results in people with polyneuropathy. About 520 adults with various forms of nerve damage, plus healthy volunteers, take part. Participants complete physical tests,…

This study looked at whether people who donate a kidney have a higher chance of developing gout later in life compared to similar healthy people who did not donate. Researchers reviewed health records from 50,000 donors and matched non-donors in Canada over many years. The goal i…

This study looks at how to help VA medical centers offer virtual programs that prevent heart disease, diabetes, and depression in women Veterans. Researchers are testing two different support strategies at 20 VA sites to see which works better. The study does not involve patients…

This study investigates why brown fat, which burns calories to generate heat, stops working when it's warm. Researchers will use cold vests on 8 healthy and obese adults to see how genes and proteins change. The goal is to find new targets for treating obesity and related metabol…

Researchers are testing a transition program for 17-year-olds with type 1 diabetes, asthma, or obesity who are preparing to move from pediatric to adult healthcare. The program includes a consultation with the teen and family, a solo visit where the teen completes a readiness che…

This study aims to understand how genes affect obesity, diabetes, and heart disease in people of African ancestry. Researchers will collect blood samples and health information from 1,100 adults, including African Americans, Afro-Caribbeans, and migrants from sub-Saharan Africa. …

This large study is looking at the genes of over 9,000 people with diabetes and kidney disease, along with their family members. The goal is to find which genes make some people more likely to get diabetic kidney disease. By understanding the genetic roots, researchers hope to de…

This study follows 207 people with primary hyperoxaluria type 1 (PH1), a rare kidney disease, to see how the condition progresses over time. Researchers are also checking the long-term safety and real-world effectiveness of the drug lumasiran. Participants are not given any new t…

Researchers are studying whether a mother's weight and blood sugar during pregnancy affect the makeup of her breast milk, and whether those differences influence how her baby grows. The study will follow pregnant women with a range of body sizes, including those with gestational …

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

This study follows 121 adults who have been cured of chronic hepatitis C to understand why some still develop liver complications like scarring or cancer. Participants are monitored for up to 10 years with regular tests, scans, and occasional liver biopsies. The goal is to learn …

This large study is testing whether a risk-scoring tool (called ENDPAC) can identify people with new-onset diabetes who are at higher risk for pancreatic cancer. About 8,800 participants aged 50-85 will have their medical records checked for age, weight changes, and blood sugar t…

This pilot study tests a program that screens older dialysis patients for common age-related problems like falls, memory trouble, and medication issues. A geriatrics team then gives personalized recommendations to the dialysis clinic. The goal is to see if this approach is practi…

This long-term study follows over 1,500 people of all ages with hypophosphatasia (HPP), a rare bone disease. Researchers will collect information on how the disease progresses, its impact on daily life, and the long-term safety and effectiveness of the treatment asfotase alfa. Th…

Researchers at a South African hospital are asking whether two questionnaires can reliably capture how patients feel after metabolic surgery for obesity. Adults scheduled for the procedure will rate their comfort, pain, nausea, and return to normal activities before surgery and a…

This study monitors heart rate variability in 150 adults with type 2 diabetes who also have heart failure, chronic kidney disease, or both. Participants will wear a 24-hour heart monitor before and after starting a standard SGLT2 inhibitor medication. The goal is to understand ho…

This pilot study tests a digital tool that doctors use during visits to help patients with prediabetes become more active in managing their health. The tool asks patients a few questions and then gives the doctor tips on how to talk about lifestyle changes and resources like heal…

This study follows 20 newborns and children diagnosed with late-onset Pompe disease through newborn screening. Researchers will track their health for up to 4.5 years to document early muscle and joint symptoms, biomarkers, and quality of life. The goal is to learn when to start …

This study looked at 41 healthy adults who regularly drink sugary sodas. Researchers replaced their sugary drinks with either stevia-sweetened or aspartame-sweetened beverages for 10 weeks. The goal was to see how these alternatives affect metabolism and gut bacteria. The results…

This study follows 5,000 people with diabetes who use a Dexcom continuous glucose monitoring (CGM) system in their daily routine. Researchers will track how the device performs when used as labeled and ask participants to complete questionnaires over four years. The goal is to un…

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

This study tests whether a clinical decision framework helps hospital doctors feel more confident when prescribing medications for older adults (65+) with chronic conditions like diabetes and high blood pressure. About 150 doctors will attend an educational session and complete s…

This study is collecting long-term, real-world data from about 6,000 adults being treated with Roche eye medications (Faricimab or the Port Delivery System with Ranibizumab) for conditions like wet age-related macular degeneration and diabetic macular edema. The goal is to unders…

This study looks back at dental implant cases to see if bone-related conditions—like vitamin D deficiency, calcium problems, or kidney disease—make implant failure more likely. Researchers will compare 192 failed implants with successful ones from similar patients. The goal is to…

This study looks at whether diabetic kidney disease and blood-sugar control before surgery can help predict a common complication called pseudophakic macular edema (PME) — swelling of the central retina — after cataract surgery in people with type 2 diabetes. Researchers will rev…

This study will use artificial intelligence (AI) to analyze eye scans from 554 adults with diabetic macular edema (DME). The goal is to see if AI can predict when a person's eye treatment needs to change. Participants will not receive any new treatment; instead, researchers will …

This study aims to find early markers of Parkinson's disease, multiple system atrophy, and Lewy body dementia by analyzing samples from the nose, blood, and urine. Researchers will compare results from 180 people with these conditions and healthy volunteers. The goal is to improv…

This study will interview 35 adults in Sweden who have Fabry disease but are not yet receiving treatment. The goal is to learn how they experience their quality of life, daily activities, and healthcare support. By listening to patients' own stories, researchers hope to identify …

This study follows 20,000 healthy Chinese adults aged 40-75 living at various altitudes to see if chronic low oxygen levels increase the risk of multiple system atrophy (MSA), a rare and fatal brain disease. Participants undergo checkups and overnight oxygen monitoring, then are …

This completed study observed 40 people with type 2 diabetes to find blood markers that could identify those at risk for a serious liver condition called NASH. Researchers measured liver stiffness and calculated scores like FIB-4 and ELF to see which patients might have liver sca…

This study gathers real-world information from pregnant women with type 1 diabetes in the United States to understand how they manage their condition. Participants share data from their continuous glucose monitors and insulin pumps, and complete online surveys. The goal is to lea…

This study tests a smartphone app designed to help adults taking GLP-1 medications for obesity or type 2 diabetes eat better. About 50 participants will use the app for 6 weeks to track their diet and learn about nutrition. The goal is to see if the app is helpful and easy to use…

This study investigates whether people with type 2 diabetes who have or are at risk for chronic kidney disease produce too much aldosterone, a hormone that regulates salt and blood pressure. Participants undergo several suppression tests—including oral sodium, saline infusion, an…

This study is screening 115 underserved children and adults who are at risk for type 1 diabetes or celiac disease. Researchers will use blood tests to find early signs of these conditions, aiming to improve how we detect them. The goal is to catch these diseases sooner, especiall…

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

This study looks at how much people trust institutions (like health organizations) when it comes to advice about physical activity. Researchers will ask 2000 volunteers questions about their trust levels, lifestyle, and background. The goal is to understand what factors influence…

This study tests a short guide called the 'Jumpstart Guide' to help doctors have better conversations with dementia patients and their families about healthcare goals. Over 1,800 patients and their caregivers are taking part. Researchers will compare whether those whose doctors u…

This long-running study follows nearly 100,000 French women, mostly teachers, since 1990 to understand what factors influence their risk of cancer, heart disease, diabetes, and other major illnesses. Participants fill out questionnaires every few years about their lifestyle, medi…

This study was designed to see how accurately patients report their own medication and healthcare use in diaries compared to official health insurance records. It planned to include people with diabetes, arthritis, kidney disease, or prostate cancer. However, the study was withdr…

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

This 24-month observational study follows 100 people aged 12–50 with Limb Girdle Muscular Dystrophy type R1 (LGMD R1). Researchers will test whether a motor function scale called NSAD and muscle fat measurements from MRI can reliably track disease progression. The goal is to vali…

This study is following 964 people with type 2 diabetes to see if three blood markers (hs-cTnI, NT-proBNP, and sST2) can predict who will die or have heart problems. Participants gave blood samples and are being tracked for years. No treatment is being tested—the goal is to learn…

This study looks at how a type of medication called PPAR agonists affects blood sugar levels in 30 adults with primary biliary cholangitis (PBC), a chronic liver disease. Researchers will measure changes in blood sugar and other metabolic markers over three months. The goal is to…

This study aims to help adults aged 40 and older in Upper Manhattan get better eye care. It will offer free eye screenings at local health centers and senior centers, and provide support like scheduling appointments and transportation. The goal is to see if this extra help leads …

This study follows over 1,000 women in Singapore from before pregnancy through their child's early years. Researchers are looking at how nutrition, lifestyle, and emotional health before and during early pregnancy may influence the child's body composition, brain development, and…

This study was designed to track how COVID-19 infection might worsen the condition of people with inherited metabolic diseases (IMD). Researchers planned to collect information from French patients with IMD who had or had COVID-19 to see how often their metabolic disease got wors…

This study was designed to see if giving a carbohydrate drink before bariatric surgery helps control blood sugar after the operation, compared to the standard fasting approach. It planned to enroll patients with high blood sugar who were scheduled for weight-loss surgery. However…

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

This study looks at why up to 40% of people with type 1 diabetes develop kidney disease, even with better blood sugar control. Researchers will use kidney biopsies and advanced imaging in 100 adults to find hidden risk factors like insulin resistance and inflammation. The goal is…

This study tests whether a remotely delivered resistance training program is practical and helpful for Black women aged 30-64 who are overweight, have prediabetes or diabetes, or have high blood pressure. About 37 women will participate from home using their own devices. The goal…

This study looks at how newer diabetes medications (SGLT2 inhibitors and GLP-1 receptor agonists) affect the eyes of people with type 2 diabetes. Researchers will monitor 40 adults who take metformin and are starting these newer drugs. The goal is to see if these drugs cause any …

Over two million people in the UK have a chronic condition like diabetes or heart disease without knowing it. This study will use information from 4.5 million hospital patients to build a digital platform that spots those at risk. The goal is to help doctors diagnose these condit…

This study is testing whether wearable devices like continuous glucose monitors and the Oura Ring can help predict how well the pancreas is working in people with prediabetes or type 2 diabetes. The goal is to develop remote tools that allow personalized treatment to preserve ins…

This large study looks back at medical records of 100,000 Chinese patients with type 2 diabetes to compare heart and kidney outcomes between those taking Ozempic and those on other diabetes drugs. Researchers will track major heart problems, strokes, and kidney events. The goal i…

This study follows over 800 people who either have type 2 diabetes or have a strong family history of it. Researchers track who develops diabetes over time using blood tests and questionnaires. The goal is to improve a risk prediction model for type 2 diabetes. The study is curre…

This study enrolls 500 adults with a sleep condition called REM sleep behavior disorder (RBD), which often leads to Parkinson's disease or related brain disorders. Researchers will track symptoms like thinking, movement, and smell to develop a rating scale. The goal is to prepare…

This study tests whether a swallowable camera capsule, controlled by magnets from outside the body, can safely and effectively examine the stomach and esophagus in 30 people preparing for weight loss surgery. Participants swallow the capsule after fasting, and a surgeon guides it…

This study directly measures how GLP-1 medications (like Ozempic) change the way people eat. Researchers use a special device to track every sip of sugary and fatty drinks in 140 adults with obesity. The goal is to understand exactly how these drugs reduce calorie intake, not jus…

This study uses data from over 30,000 Swedish adults to see if using snus (including newer white nicotine pouches) is linked to heart disease, metabolic problems, and poor mental health, independent of smoking. Researchers will compare current, former, and never-users using advan…

This study follows 96 people with end-stage kidney disease who need a brachiocephalic fistula for dialysis. Researchers collect data from exams, blood tests, and imaging to track changes in the vein over time. The goal is to learn what causes cephalic arch stenosis, a common narr…

This study looks at how fat tissue takes up sugar in people with obesity, comparing those with normal blood sugar to those with insulin resistance. Researchers will use labeled sugar infusions and PET scans to measure sugar uptake in fat and muscle. The goal is to understand why …

This study tests a new wireless sensor that continuously measures the light dose babies receive during phototherapy for jaundice. Currently, doctors check light levels only once a day with a handheld device, which can be inaccurate. The sensor aims to provide real-time data to en…

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

This study looks at whether giving extra support to community health centers helps them screen patients for non-medical needs like food or housing and refer them to community resources. Twenty health centers will be randomly assigned to get this support or not. The goal is to see…

This study looks at over 1,200 adults with type 2 diabetes and heart muscle problems to see if diabetic cardiomyopathy is a unique condition. Researchers will use detailed medical tests, imaging, and biological data to find patterns. The goal is to better understand and diagnose …

This study compares 20 women who had gestational diabetes with 20 who did not, all matched by age and weight. Researchers will measure body composition, insulin sensitivity, and other metabolic markers before and after 8 weeks of high-intensity interval training. The goal is to u…

This study follows 450 infants and their families from birth to age 12 to learn how a child's biology and home environment work together to shape eating habits and growth. Researchers collect samples, measurements, and surveys at regular visits. The goal is to understand what inf…

This pilot study compares the effects of daily erythritol (a sugar substitute) versus regular sugar on insulin resistance and blood clotting in 30 healthy adolescents aged 14-18. Participants consume either erythritol or sucrose for 5 weeks, and researchers measure changes in glu…

This study is testing a new way to care for people with rare diseases. It will use advanced genetic testing and a team of specialists to help diagnose patients faster and coordinate their care better. The study involves 136 participants with certain rare diseases and aims to redu…

This study is for people with autosomal dominant hypocalcemia types 1 or 2, rare genetic conditions that cause low blood calcium. Researchers will collect past and future health data from 95 participants to learn how the disease changes over time. The goal is to better understand…

This study uses a continuous glucose monitor (a small sensor) to track blood sugar levels in 40 adults with diabetes and end-stage kidney disease who are on dialysis. The goal is to see how glucose changes during and between dialysis sessions over two weeks, and whether those pat…

This study looks at how the bacteria in a pregnant woman's gut (her microbiome) and her nutrition affect her baby's growth and health. Researchers are following 800 young pregnant women (28 and under) in Toronto throughout pregnancy and for a year after birth. They collect stool,…

This study aims to find new genes that cause very low cholesterol levels (a condition called FHBL). Researchers are recruiting 435 people with low cholesterol and their family members to analyze their DNA. The goal is to discover new targets for understanding and potentially mana…

This study aimed to see if adding pramlintide to semaglutide (Wegovy) could change appetite and eating habits in obese people with prediabetes. It also tried to find ways to predict who would respond best to obesity medication. The trial was withdrawn before any participants were…

This study is building an online platform called DBS MatchMaker to connect doctors around the world who treat rare movement disorders with deep brain stimulation (DBS). The goal is to help doctors share knowledge and find the best ways to treat patients. Up to 500 people of all a…

This study tests whether using game-like rewards and personal coaching can encourage older adults to walk more after leaving the hospital. About 350 people aged 50 and older with conditions like high blood pressure or heart failure will wear a Fitbit to track their steps. The goa…

This study looks at whether fat in the liver, detected by ultrasound during pregnancy, can help predict blood sugar problems after delivery in women with gestational diabetes. Researchers will follow 189 women for 6-12 weeks after birth, measuring insulin resistance and other mar…

This study looks at why some people with type 2 diabetes do not get the usual health benefits from exercise. Researchers will measure how well cells produce energy before and after exercise. The goal is to find factors that make exercise less effective for certain individuals.

This study looks at how exercise training changes the way muscles and fat tissue handle insulin in people with type 2 diabetes. About 39 adults aged 30-65 who are sedentary and have type 2 diabetes will take part. Researchers will use PET scans and small tissue samples to measure…

This study looks at organ changes in people recovering from COVID-19, comparing those with and without type 2 diabetes. Researchers will use scans to measure organ size and volume. The goal is to learn more about how COVID-19 affects the body long-term in diabetic patients.

This study looks at how exercise changes the signals muscles send to the rest of the body, especially those that help burn fat. Researchers will compare obese, lean, and athletic participants to find proteins or genetic codes released from muscle during exercise. The goal is to b…

This early-phase study is testing whether combining a low-carbohydrate diet with a diabetes drug (ertugliflozin) can safely produce moderate ketone levels in healthy adults. Nine participants will follow a gradually reducing carb diet while taking the drug, all under close superv…

This study aims to find new biological markers (biomarkers) for Adult Onset Autoimmune Diabetes (AOnAD) by analyzing blood samples from 25 adults with type 1 diabetes. Researchers will measure changes in specific microRNAs from tiny particles in the blood. No treatment is given; …

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

This study was designed to learn why some children with type 1 diabetes, especially those with public insurance, do not use helpful diabetes technology like continuous glucose monitors. Researchers planned to interview and survey healthcare providers to understand barriers and fi…

This study looks at 45 people with certain changes in the WFS1 gene to figure out if they have a milder, dominant form of wolfram-like syndrome instead of the more severe recessive form. Researchers will use eye scans (OCT) to see if the gene changes are linked to specific eye pr…

This study checks how common high levels of lipoprotein(a) — a genetic risk factor for heart disease — are among hospital staff and their families. About 2800 participants will give blood samples and be followed over time to see if high levels lead to more heart problems. The goa…

This study is building a large biobank of health information and blood samples from 48,000 people with diabetes in Hong Kong. Researchers will analyze genetic and other markers to find better ways to predict serious complications like kidney failure, heart disease, and stroke. Th…

This study follows 50 obese patients who are having weight-loss surgery (laparoscopic sleeve gastrectomy). Researchers use MRI scans and blood tests before surgery and at 1, 3, and 6 months after to measure changes in pancreatic fat and insulin function. The goal is to see if los…

This study is a national registry collecting data from 1500 people with familial adenomatous polyposis (FAP) and related conditions across 28 Italian centers. It aims to better understand how these diseases progress, how they are currently managed, and what factors influence outc…

This study follows over 50,000 Chinese adults who were first surveyed between 2011 and 2014. Researchers will check how chronic lung, heart, metabolic diseases, and cancers have developed or changed over more than a decade. The goal is to understand how multiple health problems o…

This study observes heart function in 216 newborns born at 35 weeks or later. Researchers will use ultrasound to measure how the heart relaxes and fills with blood, creating normal reference values. They will also check if factors like mother's health or birth conditions affect h…

This study aims to understand how the body's response to insulin changes during pregnancy in people with type 1 diabetes. Researchers will analyze data from continuous glucose monitors and insulin pumps used by 100 participants from before pregnancy to six months after birth. The…

This study follows about 800 pregnant women in Somaliland to understand the physical, cultural, and social factors affecting their health during pregnancy, childbirth, and after birth. Researchers will also work with the community to create better contraceptive counseling materia…

This study looks at how lactose intolerance affects the gut's barrier and inflammation in 200 adults with IBS-like symptoms. Researchers will measure markers in stool samples to see if they relate to symptom severity. The goal is to better understand the link between lactose into…

This long-term study uses yearly brain scans, genetic tests, and thinking/memory assessments to track changes in nearly 1,800 people with various dementias (like Alzheimer's and vascular dementia) and over 140 healthy older adults. The goal is to better diagnose, monitor, and eve…

This study looks at differences in body composition, fitness, and temperature control during exercise between young soccer players with type 1 diabetes and healthy ones. Forty boys who have trained for at least 5 years will be monitored with thermal cameras and continuous glucose…

This registry is following 1,000 people who have trouble tolerating statins, a common cholesterol-lowering drug. Researchers will track how often treatments are changed, cholesterol levels, and quality of life over time. The goal is to better understand this condition and improve…

This study aimed to learn more about using a CT scan to check for calcium buildup in the heart arteries of people with diabetes. The goal was to improve screening and treatment decisions. However, the study was withdrawn before enrolling any participants, so no new information wa…

This study tests two new ways to help people with familial hypercholesterolemia (a genetic high cholesterol condition) get their family members screened. About 800 adults from Penn Medicine will be assigned to receive automated reminders, a patient navigator, or usual care. The g…

This study follows about 1,200 mothers from early pregnancy and their children for at least 20 years. Researchers want to learn how genetics, diet, lifestyle, and environment during pregnancy and early childhood affect the risk of metabolic and mental health conditions later in l…

This study looks at how different root canal cleaning methods—laser and ultrasound—affect the immune response in people with and without type 2 diabetes who have a tooth infection called apical periodontitis. Researchers will measure certain immune markers in fluid from around th…

This study tests three different daily doses of vitamin B12 (2.6, 10, and 50 micrograms) in 40 pregnant women and 10 non-pregnant women in Tanzania. The goal is to see how the body processes B12 during pregnancy and find the optimal dose for future larger trials. Participants tak…

This study looks at how breastfeeding changes cholesterol and heart disease risk in women with familial hypercholesterolemia (FH), a genetic condition causing high cholesterol. Researchers will follow 50 pregnant women with and without FH from late pregnancy to one year after bir…

This study follows over 5,000 Black and White men and women from age 18-30 for many years to see how their lifestyles and health habits affect their risk of heart disease later in life. Researchers measure things like blood pressure, weight, and cholesterol to understand why some…

This study followed nearly 6,000 adults aged 65 and older to learn what factors predict heart disease, stroke, and related health problems. Researchers aimed to identify risks and understand what leads to disability or death after a heart event. The study did not test a new treat…

Researchers at Arkansas Children's Hospital are studying how mitochondria work in children with autism spectrum disorder. They aim to identify distinct patterns of mitochondrial dysfunction that may relate to developmental delays. The study involves up to 5 visits with blood draw…

This study looks at whether certain genes influence how people respond to a meal and a single dose of metformin, a common diabetes medication. Over 1,000 adults without diabetes will take a special drink and then have their blood sugar and other markers measured. The goal is to u…