Metabolic disease

This early-stage trial tests a single intravenous infusion of NGGT002, a gene therapy designed to deliver a working copy of the PAH gene to adults with classic phenylketonuria (PKU). The goal is to see if this approach can safely lower phenylalanine levels in the blood, which cou…

This trial is testing an experimental therapy called CD22 CAR T cells in adults with certain types of B cell lymphomas that have come back or not responded to previous treatments. The therapy involves collecting a patient's own immune cells, modifying them to recognize and attack…

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

This phase 2 study tests an oral drug called nemtabrutinib in about 490 people with blood cancers like chronic lymphocytic leukemia, non-Hodgkin lymphoma, and Waldenstrom's macroglobulinemia. The goal is to see if the drug can shrink tumors and control the cancer, while also chec…

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

This study tests a new medicine called HM15275 in people with type 2 diabetes. The goal is to see if it can lower blood sugar levels (HbA1c) safely over 36 weeks. About 180 adults who manage their diabetes with diet, exercise, or metformin will take part. Half will get the drug, …

This early-stage trial is testing a new drug called DNL952 in 32 adults with late-onset Pompe disease. The main goal is to check if the drug is safe and how the body processes it. Participants will receive the drug through an IV infusion at different doses.

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

This study looks at whether the diabetes drug pioglitazone can improve how the liver processes energy in people with nonalcoholic fatty liver disease (NAFLD) and type 2 diabetes. Researchers will measure liver mitochondrial function using special tracers before and after 16 weeks…

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

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

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

This study tests a new medicine called ersodetug for people whose tumors cause dangerously low blood sugar that standard treatments can't control. About 16 adults with insulin-producing tumors will receive the drug alongside their usual care. The main goal is to see if ersodetug …

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

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

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

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

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

This study tests a program that delivers fresh produce to the homes of pregnant women with type 1 or 2 diabetes who also struggle to afford healthy food. Participants also get group classes on diabetes, nutrition, and cooking, plus help finding other resources. The goal is to see…

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

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

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

This trial tests whether a daily 250 mg supplement of lactoferrin, a protein found in milk, can improve kidney health in people with type 2 diabetes and chronic kidney disease. The study will compare lactoferrin against a placebo, alongside standard diabetes care, to see if it re…

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

This study tracks 8 Korean patients with paroxysmal nocturnal hemoglobinuria (PNH) who are taking Danicopan alongside standard C5 inhibitors like eculizumab or ravulizumab. The goal is to monitor safety and check if the drug works as expected in everyday medical practice. It is a…

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

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

This study tests a new approach for teens (ages 10-17) recently diagnosed with type 1 diabetes. They will use an inhaled insulin called Afrezza along with a long-acting injected insulin. The goal is to see if this combination helps keep blood sugar in a healthy range and is safe …

This early-stage study tests an experimental drug called EXS73565 in about 50 adults with certain blood cancers (like lymphoma or leukemia) that returned or didn't respond to standard treatments. The main goal is to check safety and find the right dose, while also seeing if the d…

This study tests whether an AI app called SnapD, which recognizes Thai food and estimates nutrition, can help people with type 2 diabetes and overweight improve their blood sugar control. Some participants will also use a continuous glucose monitor (CGM) that tracks sugar levels …

This pilot study tests whether intermittent use of a continuous glucose monitor (CGM), along with regular medical visits, diabetes education, and psychological support, can improve blood sugar control in children and adolescents with high-risk type 1 diabetes in the Dominican Rep…

This study tests whether a low dose of glucagon, given before exercise, can prevent blood sugar drops in adults with type 1 diabetes who use an automated insulin pump. Eighteen participants will exercise after a meal, comparing glucagon to a standard glucose drink. The goal is to…

This program provides continued access to lucerastat for people with Fabry disease who were previously enrolled in a clinical trial of this drug. The goal is to allow these patients to keep receiving the treatment after the original study was stopped. It focuses on maintaining tr…

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

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

This study tests whether a 12-week program of supervised foot and ankle exercises can help people with diabetes who have nerve damage in their feet. The exercises are done in groups at primary care clinics. Researchers will measure changes in symptoms, foot movement, and quality …

This study tests a one-time gene therapy called ECUR-506 in baby boys under 9 months old with a severe form of OTC deficiency, a genetic disorder that prevents the body from breaking down ammonia. The goal is to see if the treatment is safe and can reduce dangerous ammonia levels…

This phase 2 trial tests whether dulaglutide, a drug used for type 2 diabetes, can improve small blood vessel function in adults with type 1 diabetes. The study involves 47 participants aged 18-40 with long-standing type 1 diabetes. Researchers measure blood flow and insulin sens…

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

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

This study tests a new digital tool called P3-EX that helps doctors create personalized exercise plans for patients with heart disease risk factors like high blood pressure, diabetes, and obesity. Researchers will recruit 24 doctors and 48 patients to compare the new tool with a …

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

This study tests the long-term safety and effectiveness of a drug called plozasiran in adults with very high triglycerides (a type of fat in the blood). About 869 people who completed earlier studies will receive injections every 3 months for 2 years. The goal is to see if the dr…

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

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

This study tests whether an automated insulin delivery system (CamAPS FX) can help women with type 1 diabetes maintain better blood sugar control before and during pregnancy, compared to their usual insulin routine. The trial will enroll 305 women aged 18-45 who are either planni…

This study is testing a new wearable device that continuously measures both glucose (sugar) and ketone levels in adults with type 1 diabetes. The device is worn for 15 days and its readings are compared to standard lab tests. The goal is to see if it is accurate and safe enough f…

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

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

This 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 study tests if a new insulin that is injected just once a week works as well as daily insulin shots to control blood sugar in people with type 2 diabetes. About 586 adults who have never used insulin before will take either the weekly insulin or a standard daily insulin for …

This study tests a drug called cosibelimab in 80 people with advanced skin cancer who also have a kidney transplant or a blood cancer. The drug works by helping the immune system attack cancer cells. Researchers will check if the tumors shrink and monitor for side effects.

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-phase trial tests a single intravenous dose of a gene editing therapy called LNP.UCD.ABE in 7 children with severe urea cycle disorders. The therapy aims to correct the genetic defect using a lipid nanoparticle to deliver a base editor. The main goal is to check safety…

This study tests whether a nutrition program that provides twice-daily meal delivery for up to 90 days after hospital discharge, plus diet education, can reduce food insecurity in people with type 1 or type 2 diabetes. About 160 hospitalized patients who screen positive for food …

This study tests a drug called CSL300 in people with end-stage kidney disease who are on dialysis and have signs of inflammation. The goal is to see if it can reduce the risk of heart attacks or heart-related death. About 3110 adults will take part, and half will get the drug whi…

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

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

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

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

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

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

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

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

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

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

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

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

This early-stage study is testing a new treatment called GNTI-122 for adults who were recently diagnosed with type 1 diabetes. The therapy is made from a person's own blood cells and is designed to help rebalance the immune system. Sixteen participants will receive either a low o…

This study tests whether an internet-based depression treatment called cognitive behavioral therapy (iCBT-D) can prevent insulin resistance from getting worse in adults with HIV who are on antiretroviral therapy and have depression. Researchers will compare the online therapy pro…

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

This study tests a drug called PRX-102 (pegunigalsidase alfa) in 22 children and teens aged 2 to 18 with Fabry disease, a rare genetic disorder that causes pain and organ damage. The drug is an enzyme replacement therapy given by infusion every two weeks. Researchers will monitor…

This trial tests whether pegtibatinase, a lab-made version of the CBS enzyme, can safely lower homocysteine levels in people with classical homocystinuria (HCU), a rare genetic condition that causes harmful buildup of homocysteine and methionine. The study includes children and a…

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

This study tests two higher doses (3.0 mg and 4.5 mg) of the diabetes drug dulaglutide in teenagers aged 10 to 17 with type 2 diabetes. The goal is to see if these doses are safe and help control blood sugar and weight. Participants will receive weekly injections for about 8 mont…

This Phase 2 trial tests whether a drug called 2-HOBA can improve the function of HDL (good cholesterol) in people with familial hypercholesterolemia, an inherited condition causing very high cholesterol. Researchers will give 72 participants either 2-HOBA or a placebo for six we…

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

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

This study looks at whether wearing a continuous glucose monitor (CGM) for two weeks after leaving the hospital helps people with diabetes who are starting insulin for the first time. About 150 adults with type 1 or type 2 diabetes will be randomly assigned to use either a CGM or…

This Phase 2 trial tests whether a new drug called icovamenib can help people with type 2 diabetes who still have high blood sugar despite taking other medications. About 60 adults aged 18 to 70 will receive either icovamenib or a placebo daily for 12 weeks. The main goal is to s…

This study is tracking how well the drug zanubrutinib works and its safety in people with Waldenström macroglobulinemia, a rare blood cancer. Researchers are especially interested in patients with specific genetic mutations and those from racial and ethnic minority groups. The st…

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

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

This study tests a medicine called iptacopan in children aged 2 to under 18 who have a rare blood disease called PNH. The goal is to see how the drug works in their bodies and if it is safe. About 12 children will take the medicine, and doctors will monitor side effects and blood…

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

This Phase 3 trial tests pegtibatinase, an enzyme given as a shot, in 70 people aged 12–65 with classical homocystinuria. The goal is to see if it safely lowers high homocysteine levels when added to standard care. Participants receive either the drug or a placebo for 24 weeks.

This study looks at how well a combination of three cholesterol-lowering medicines (bempedoic acid, ezetimibe, and a statin) works in real-world patients with high cholesterol or mixed lipid problems. About 2,000 adults at high or very high risk will be followed for up to a year.…

This study tests whether a 12-week sleep-focused behavioral program can help adults with type 1 diabetes improve their sleep and blood sugar control. Participants learn sleep hygiene, stress management, and routines to target 7-9 hours of quality sleep. The study tracks sleep wit…

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

This study tests whether personalized marketing messages and discounts can encourage people with prediabetes or type 2 diabetes to switch from refined grains to whole grains when shopping for groceries online. About 216 adults aged 45-70 from the Philadelphia area who already sho…

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

This study is testing a new implantable insulin pump (MIIPS 2020) in 40 adults with type 1 diabetes whose blood sugar is hard to control with standard insulin shots or external pumps. The pump is placed inside the body and delivers insulin directly, aiming to reduce dangerous hig…

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

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

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

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

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

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

This trial tests whether a low-calorie diet combined with intensive lifestyle coaching can help adults with type 2 diabetes achieve remission—meaning blood sugar levels return to normal without medication. Participants are randomly assigned to either the intervention or standard …

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

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

This study tests whether empagliflozin (Jardiance), a drug used for diabetes, can lower uric acid levels and reduce inflammation in people with gout. Sixty adults with gout and high uric acid will take either the drug or a placebo daily for 12 weeks. Researchers will measure chan…

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

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

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

This study tests whether helping people with diabetes find local resources (like food or housing help) and pairing them with a peer supporter can improve blood sugar and blood pressure. About 694 adults with diabetes who have trouble controlling their condition will be randomly a…

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

This study tests two experimental drugs, pozelimab and cemdisiran, together in 35 adults with paroxysmal nocturnal hemoglobinuria (PNH) whose red blood cell destruction is not well-controlled by current C5 inhibitors. The goal is to see if the combination can lower hemolysis (bre…

This trial tests whether extending nightly sleep by about 2 hours in adults who habitually sleep less than 6.5 hours can improve insulin sensitivity and reduce cardiometabolic risk. Participants are overweight or obese adults aged 18-45 without diabetes or sleep disorders. They a…

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

This trial tests whether a specific breathing exercise, called resonant frequency breathing with heart rate variability biofeedback, can improve how the body handles sugar in people with prediabetes. The training aims to calm the body's stress response by increasing heart rate va…

This study looks at how food insecurity affects heart and metabolic health in people with HIV. Researchers will test a program that combines peer support and a mobile health app to help reduce food insecurity and improve insulin resistance. About 200 adults with HIV from a clinic…

This trial tests whether an educational program using continuous glucose monitors (CGMs) can help older adults with type 2 diabetes use insulin more safely. Participants aged 75 and older who are at high risk for low blood sugar will either attend group education sessions with CG…

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

This trial is testing whether semaglutide, a medication used for obesity, can improve heart health in young adults (ages 18-30) who have both type 1 diabetes and obesity. Participants will receive either semaglutide or a placebo for a year. The study will measure changes in body …

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 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 3 study tests whether a new oral drug called elecoglipron can help adults with type 2 diabetes better manage their blood sugar when added to their current insulin and other diabetes medications. About 600 participants will receive either elecoglipron or a placebo daily…

This trial is testing whether an experimental drug called BGM0504 can help people with early-stage type 2 diabetes and obesity achieve better blood sugar control and weight loss than a standard treatment, semaglutide. Participants will receive either BGM0504 or semaglutide as a o…

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

This study tests whether meals designed for South Asian and Filipino cultures, following diabetes guidelines, can improve blood sugar control in people with type 2 diabetes. Thirty participants will wear a continuous glucose monitor for a month while eating their usual diet and t…

This study tests a treatment called physiologic insulin resensitization (PIR) in 120 adults with advanced chronic kidney disease and type 2 diabetes. The goal is to see if PIR can improve kidney function better than standard care. Participants will be randomly assigned to receive…

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

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

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

This study compares a new insulin pump system, Omnipod 6, to the current Omnipod 5 in 200 people with type 1 or type 2 diabetes who have poor blood sugar control. Participants will use both pumps for a set time to see which one helps keep blood sugar in a healthy range more effec…

This study tests a new automatic insulin pump (Omnipod M) in 350 adults with type 2 diabetes who already use insulin. The pump adjusts insulin delivery on its own to keep blood sugar in a healthy range. The goal is to see if it improves blood sugar control and is safe to use.

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

This study looks at how well the Omnipod 5 Automated Insulin Delivery System works in real-world settings for people with type 1 diabetes. About 152 participants aged 2 and older will use the system for the first time. The goal is to see if it helps keep blood sugar levels in a h…

This early-stage trial tests an oral drug called ALE1 for hypophosphatasia (HPP), a rare genetic bone disease. The study will first check safety and how the drug moves through the body in healthy volunteers, then in adult HPP patients. It is a small, placebo-controlled study to f…

This study tests a new drug called AP306 in 168 adults on maintenance hemodialysis who have high phosphate levels (hyperphosphatemia). Participants take one of several fixed doses of AP306 or a placebo daily for 8 weeks. The main goal is to see if AP306 safely lowers blood phosph…

This study tests whether combining two drugs, teclistamab and daratumumab, can help people newly diagnosed with both multiple myeloma and light chain amyloidosis. About 30 adults will receive the treatment for up to 24 cycles. The goal is to see if the combo improves survival wit…

This study compares continuous glucose monitors (CGMs) to standard fingerstick testing in 180 pregnant women with type 2 diabetes. The goal is to see if CGMs reduce the number of large babies and improve blood sugar control. Participants will use either a CGM or traditional finge…

This study tests whether pemafibrate, a drug that lowers triglycerides, can prevent the worsening of narrowed brain arteries in people who have already had a stroke or mini-stroke. About 270 participants will be randomly assigned to receive either pemafibrate plus standard care o…

This early-stage study tests a new drug, AMG 127, to see if it is safe for people. It involves 162 healthy adults and people with type 2 diabetes. Researchers will give single or multiple doses and watch for side effects. This is a first step to see if the drug can be developed f…

This study tests whether the diabetes drug metformin can prevent blood sugar increases caused by statins in people with schizophrenia. Researchers will give 400 patients either metformin or a placebo alongside atorvastatin for six months. The goal is to see if metformin helps kee…

This study tests an experimental drug called WAL0921 in 96 adults with kidney diseases that cause protein in the urine, including diabetic nephropathy and several rare conditions. Participants receive either the drug or a placebo as an IV infusion every two weeks for 14 weeks, th…

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

This expanded access program provides bitopertin (DISC-1459) to patients aged 12 and older with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) who have no satisfactory treatment options in the US. The goal is to offer access and gather safety information. Pa…

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

This trial tests whether an artificial intelligence system can help optimize insulin pump settings remotely for people with type 1 diabetes. The AI analyzes continuous glucose monitor and pump data to suggest personalized adjustments, which a doctor then reviews before sending to…

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

This study tests the drug acoramidis in people with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart, weakening it. Over 36 months, researchers measure whether acoramidis improves heart function and structure using cardiac …

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

Black individuals often have lower levels of natriuretic peptides, heart hormones that help regulate metabolism. This trial tests whether raising those hormone levels with a drug called sacubitril/valsartan can improve insulin sensitivity and energy expenditure. About 200 Black a…

This trial tests whether denosumab, a drug already used for bone conditions, can protect the beta cells that produce insulin in people with early type 1 diabetes. Researchers believe the same biological pathway that denosumab targets in bones may also help preserve beta cell func…

This trial tests whether a diet adapted to Puerto Rican culture can lower the risk of heart disease, type 2 diabetes, and obesity. Researchers will compare culturally tailored nutrition advice—emphasizing traditional healthy foods and limiting unhealthy ones—with standard healthy…

This study tests a new drug called ARC-02, given together with rituximab, in adults with B-cell non-Hodgkin lymphoma whose cancer has not responded to at least two prior treatments. The main goals are to check the safety of ARC-02 and find the best dose, while also looking at whe…

This study tests a 10-week virtual program called Acceptance Based Coping (ABaCo) for Hispanic/Latinx military patients with type 2 diabetes. The program, delivered by a bilingual health coach, teaches skills to cope with diabetes-related stress and improve self-care. Researchers…

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

This trial tests whether GlycoQuest, a video game that connects to a continuous glucose monitor and adapts using artificial intelligence, can help children aged 6 to 17 who were diagnosed with type 1 diabetes in the past three months. The game includes a glucose-responsive avatar…

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

This phase 2 trial tests an investigational injection called SYH2070 in adults with mixed hyperlipidemia—a condition where both cholesterol and triglycerides are high. Participants must already be on stable statin therapy. The study compares three doses of SYH2070 against a place…

This trial tests whether adding a specific probiotic (Bacillus subtilis and Enterococcus faecium) to standard lipid-lowering therapy can reduce triglyceride levels and inflammation in people hospitalized with pancreatitis caused by very high triglycerides. About 180 adults with m…

This trial tests whether adding an antibiotic-impregnated bone graft substitute (Cerament G) to standard surgery improves healing of heel bone infections (calcaneal osteomyelitis) in people with diabetes. Thirty participants will be randomly assigned to receive either partial rem…

This study tests whether a structured exercise and nutrition program can slow the loss of muscle mass, strength, and function in adults aged 65 and older with type 2 diabetes and obesity who are taking semaglutide. Participants receive either standard health advice or a 26-week p…

This study investigates whether adding melatonin to standard diabetes care can improve symptoms of diabetic peripheral neuropathy, a common complication causing pain and numbness in the feet and hands. The trial includes 60 adults aged 40-75 with type 2 diabetes and diagnosed ner…

This study looks at whether replacing some wheat flour with chickpea flour in bread can lower blood sugar rises after eating. Healthy adults and people with type 2 diabetes will eat bread made with finely or coarsely ground chickpea flour, and their blood sugar will be tracked co…

This study looks at whether eating lunch and dinner at a subsidized community dining site encourages older adults with prediabetes to walk more after meals and improves their blood sugar levels. Participants, aged 60 to 79, will spend six days eating at the community site and six…

This study compares two exercise programs—high-intensity interval training (short bursts of fast stair climbing) and continuous endurance training (steady walking)—in adults with prediabetes. The goal is to see which approach better improves blood sugar control, body mass index, …

This study tests whether a behavioral program — time-restricted eating, nutrition education, and reducing sedentary time — can prevent the unhealthy fat buildup that often occurs during chemotherapy for breast cancer. Researchers will measure changes in visceral and muscle fat us…

This study tests whether a structured nutrition counselling program can improve diet, blood sugar, blood pressure, and quality of life in adults who have both type 2 diabetes and hypertension. About 308 participants from 14 hospitals in Ethiopia will either receive six individual…

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

This study tests a web-based program called Web-SUCCEED that helps veterans with chronic illness and their informal caregivers work together to manage the condition. The program includes three weekly online modules and brief phone calls with staff, focusing on stress coping and c…

This study tests a smartphone program called Smart Walk, designed specifically for African American women who are not very active. The program includes an app, virtual coaching, and text messages to encourage more physical activity. Researchers want to see if it helps women exerc…

This study investigates whether giving phosphate through an IV can reduce the severity of acute pancreatitis in adults. Researchers want to know if low phosphate levels make the disease worse and if replenishing it helps. Participants receive either standard care or IV phosphate …

This study investigates whether canagliflozin, a diabetes medication, can improve cognitive function in people with type 2 diabetes who also have mild cognitive impairment. The trial uses brain scans to measure changes in blood flow after insulin inhalation, along with standard c…

This study tests whether personalized nutrition counseling, guided by continuous glucose monitor (CGM) data, helps people with type 2 diabetes better control their blood sugar. Participants are randomly assigned to receive either CGM-based nutrition advice, standard nutrition edu…

This study compares three diets for weight loss in adults with type 1 diabetes who are overweight or obese. Participants will follow either a moderate carbohydrate restriction, an early time-restricted eating plan, or a standard calorie-restricted diet. The goal is to see which a…

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

This trial compares two ways of monitoring blood sugar in adults recently diagnosed with type 2 diabetes (within the last 3 months). Half of the 80 participants use a real-time continuous glucose monitor (CGM) that provides constant feedback, while the other half use standard fin…

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

This study tests an experimental oral drug called MZE782 in adults with phenylketonuria (PKU), a genetic disorder that prevents the body from breaking down an amino acid called phenylalanine (Phe). High Phe levels can cause brain damage. The trial compares two doses of MZE782 aga…

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

This study investigates penile transplantation as a way to replace a severely damaged or missing penis with a donor organ, aiming to restore appearance, sensation, and sexual function. It includes men aged 18-69 who have lost their penis due to trauma, surgery, or birth defects. …

This trial investigates whether time-restricted eating (TRE) — eating all food within an 8-10 hour window each day — can improve kidney function in people with type 2 diabetes who have elevated protein in their urine (microalbuminuria). All 25 participants follow the same TRE sch…

This study tests a program to help new mothers with chronic conditions like high blood pressure, diabetes, or depression transition smoothly from obstetric to primary care after giving birth. The program includes automatically scheduling a primary care visit, sending personalized…

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

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

This study compares two types of walking exercise for people with type 2 diabetes: walking on a downhill slope versus walking on flat ground. Over 8 weeks, participants will walk twice a week on a treadmill under supervision. Researchers will measure changes in walking distance, …

This study tests whether Taiwan green propolis, a natural bee product, can improve blood lipids and body fat in adults with metabolic dysfunction-associated steatotic liver disease (MASLD), a common fatty liver condition linked to obesity and high cholesterol. About 60 participan…

This study explores whether a short-term low-energy diet (using meal replacements) can help adolescents aged 12–17 with obesity and type 2 diabetes lose weight and potentially achieve diabetes remission. Participants follow the diet for 12 weeks, then gradually reintroduce food o…

This trial investigates whether exosomes—tiny particles from human umbilical cord stem cells—can safely improve blood sugar control in adults with type 2 diabetes. Participants receive weekly intravenous infusions of the exosome product or a control for eight weeks. The study mea…

This study tests whether very low doses of radiation delivered to the whole brain are safe and can help preserve thinking and memory in people with Alzheimer's disease or certain dementias with inflammation. Participants receive an initial course of 10 radiation sessions, followe…

This study tests whether a simple breathing exercise, called Inspiratory Muscle Strength Training (IMST), can safely lower blood pressure in pregnant women during their third trimester. Participants do 5 to 8 minutes of breathing against light or moderate resistance each day for …

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

This trial studies two experimental drugs, UIC202601 and UIC202602, designed to treat high blood pressure and high cholesterol. It enrolls healthy adults aged 19 to 55 to see how the drugs interact when taken together, focusing on safety and how the body processes them. Participa…

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

This early-stage trial tests a single dose of an experimental oral drug called GenSci144 in healthy adults. The drug aims to block a transporter in the gut to reduce phenylalanine levels, which could help manage phenylketonuria (PKU). The study focuses on safety and how the body …

This pilot study investigates whether taking myo-inositol (a vitamin-like substance) along with folic acid can improve blood sugar control and reduce oxidative stress in women diagnosed with gestational diabetes. About 40 pregnant women will be randomly assigned to receive either…

This study tests an experimental drug called THN391, given as an injection into the eye, for people with diabetic macular edema (DME) — a condition where fluid builds up in the retina due to diabetes, causing vision loss. The trial involves 21 adults aged 18 to 80 with DME and vi…

This study tests whether a real-time continuous glucose monitor (CGM) helps pregnant women with type 2 diabetes keep their blood sugar in a healthy range better than standard finger-prick testing. About 240 women in early pregnancy will either wear the CGM device for most of thei…

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 a pulse electric field ablation system can help adults with type 2 diabetes whose blood sugar is not well controlled by oral medications. The study enrolls 20 people aged 18 to 70 with a specific range of blood sugar levels. Researchers will measure chang…

This study tests a new type of stem cell transplant for children with primary immune deficiencies or inherited metabolic disorders. The transplant uses stem cells from a half-matched family donor, which are specially processed to remove certain immune cells. The goal is to see if…

This early-stage trial tests an experimental drug called PL54 in adults aged 18 to 55 with phenylketonuria (PKU), a genetic disorder that prevents the body from breaking down the amino acid phenylalanine. PL54 is an enzyme designed to help lower high blood phenylalanine levels, w…

This study looks at whether adding GLP-1 receptor agonists or SGLT inhibitors to insulin helps adults with type 1 diabetes manage their blood sugar better. Researchers will track 300 participants for one year, measuring changes in HbA1c and other health markers. The goal is to se…

This study tests whether a 12-week personalized exercise program done at home and guided by a mobile app and wearable device can improve blood sugar control and physical fitness in adults with type 2 diabetes. Participants are 40 to 69 years old, newly diagnosed or with diabetes …

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

This early-stage trial tests whether TIX100, a daily oral pill, is safe and tolerable in healthy adults who are overweight or obese. Over 28 days, participants receive either TIX100 or a placebo, and researchers monitor for side effects and how the drug moves through the body. Th…

This study investigates whether combining intranasal insulin (delivered as a nose spray) with high-intensity exercise can improve brain blood flow, insulin sensitivity, and thinking skills in older adults with type 2 diabetes. Participants aged 55–80 will receive either insulin o…

This study tests whether montelukast, a common allergy medication, can lower protein levels in urine and improve kidney and blood vessel function in people with type 1 diabetes who have early signs of kidney disease. Inflammation is thought to play a role in diabetic kidney damag…

This study compares continuous glucose monitoring (CGM) to standard fingerstick testing for pregnant women with type 2 diabetes. Researchers want to see if CGM leads to better blood sugar control and fewer complications for both mother and baby. Participants wear a CGM device or …

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

This study tests whether a continuous glucose monitor (CGM) paired with a coaching app helps pregnant women with type 2 diabetes achieve better blood sugar control compared to standard care. Participants use a Dexcom G7 CGM sensor and receive guidance through a mobile app and car…

This phase 2 trial tests whether dapagliflozin, a diabetes drug, is safe and can lower high urine protein levels in people with renal AL amyloidosis—a rare condition where abnormal proteins damage the kidneys. About 20 participants will take the drug daily for 6 months and be mon…

This study tests whether semaglutide, a diabetes drug, is safe and tolerable for people with type 2 diabetes who are on dialysis. About 100 participants will either receive weekly semaglutide injections or standard care for 26 weeks. The goal is to see if patients can stick with …

This study tests whether a fully closed-loop insulin system (which automatically adjusts insulin delivery) works better than a standard insulin pump for people with type 2 diabetes. Eighty adults who have used an insulin pump for at least six months will try both methods for 12 w…

This study tests a drug called pegcetacoplan in 12 to 17-year-olds with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The drug aims to control the disease by blocking part of the immune system. The trial will check safety, how the…

This trial tests a gene therapy for Fanconi anemia, a rare genetic disorder that causes bone marrow failure and increases cancer risk. Participants receive their own stem cells that have been genetically corrected with a lentiviral vector to fix the faulty FANCA gene. The study e…

This study compares two blood sugar targets for overweight or obese women with gestational diabetes. One group aims for stricter goals (fasting under 90 mg/dL, after meals under 120 mg/dL), while the other follows standard targets (fasting under 95 mg/dL, after meals under 140 mg…

This study investigates whether taking spermidine, a natural compound found in wheat germ, can improve blood fat levels and body weight in overweight or obese adults with high cholesterol. Participants take either spermidine capsules or a placebo daily for one month. The goal is …

This trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare and life-threatening genetic disorder that damages the nervous system. The treatment uses a lentivirus to deliver a working copy of the ARSA gene directly into the spinal fluid and bloodstream. Up to 1…

This clinical trial tests a new type of immunotherapy called 4SCAR-T, which uses a patient's own immune cells engineered to recognize and attack three different markers (GD2, PSMA, and CD276) on neuroblastoma cancer cells. The study enrolls children and adults (ages 1 to 65) with…

This study compares a nasal high-flow device to standard non-invasive ventilation (NIV) for treating people who come to the emergency room with a severe COPD flare-up and high carbon dioxide levels. Participants are randomly assigned to receive one of the two breathing supports f…

This study tests whether regular phone calls from a trained peer coach can help older adults (55+) with type 2 diabetes in rural Kentucky improve their self-care. Participants are divided into groups that receive weekly or bi-weekly coaching calls to discuss goal-setting and diab…

This trial investigates whether people with diabetic macular edema can safely monitor their vision and eye health at home instead of making frequent hospital visits. Participants use a home vision tester and a portable eye scanner every four weeks, with results sent to their doct…

This pilot study will enroll 150 pregnant individuals to see if a continuous glucose monitor (CGM) can accurately diagnose gestational diabetes. Participants will wear a small sensor on their arm that measures blood sugar continuously. The results will be compared to the standard…

This study is testing whether a diagnostic test called KidneyIntelX can help primary care doctors improve care for people with type 2 diabetes and chronic kidney disease. The test predicts the risk of kidney function worsening. Researchers will see if using this test leads to mor…

This trial evaluates an artificial intelligence system designed to automatically detect signs of diabetic retinopathy in retinal photographs. Researchers will compare the AI's performance against experienced ophthalmologists using a large set of eye images from adults with the co…

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

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

This observational study is testing whether a combination of non-invasive measurements—including blood pressure monitoring, ultrasound, blood and urine tests, and a light-based technique called photoplethysmography—can detect early damage to blood vessels and organs in adults at …

This trial evaluates a new continuous glucose monitoring (CGM) system in adults with type 1 diabetes. Participants will wear the sensor for 14 days while their blood sugar is measured against standard lab tests to see how accurate and safe the device is across different glucose l…

This study tests whether a radioactive tracer called Florbetaben F18 can reliably detect amyloid deposits in the heart using PET scans. Researchers will scan 15 people with and without cardiac amyloidosis twice to see if the results are consistent. The goal is to improve diagnosi…

This Phase 1 trial is testing a new radioactive tracer called 99mTc-p5+14 to see how it spreads through the body and how much radiation it delivers. The study includes healthy volunteers and people with AL or ATTR systemic amyloidosis, a condition where abnormal protein deposits …

This trial tests whether an artificial intelligence algorithm, called EchoNet-LVH, can help identify patients who may have cardiac amyloidosis — a rare but serious condition where abnormal protein builds up in the heart. The AI analyzes routine echocardiograms for subtle signs th…

This trial tests whether an artificial intelligence system called VeriSee can help doctors diagnose age-related macular degeneration and diabetic retinopathy from standard eye photos. The AI analyzes the images and provides instant results for the ophthalmologist to consider. Res…

This study tests a small, noninvasive device that analyzes breath to detect high and low blood sugar events in children and young adults with diabetes. Participants aged 7–25 who already use a continuous glucose monitor will provide breath samples during normal, high, and low blo…

This study collects blood samples from healthy people, those with benign diseases, and cancer patients to develop a multi-analyte blood test. The test looks for DNA methylation markers that may indicate the presence of various cancers, especially liver cancer. Researchers aim to …

This study explores whether a simple finger-prick blood sugar test done during a routine dental visit can help find people with advanced gum disease who have undetected high blood sugar. Adults with severe periodontitis but no known diabetes will be tested at the dental clinic an…

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

This study tests whether a device that shines light through the toe (photoplethysmography) can quickly detect peripheral artery disease in people with diabetes. The new method measures how long it takes blood to refill the toe after a heartbeat, and researchers compare it to the …

This study tests a program called PINPOINT that helps women with obesity or diabetes get screened for cervical cancer. About 20 women who are behind on screening will be offered options like self-collection or a Pap smear. The goal is to see if the program is practical and well-l…

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

This study tests whether adalimumab, an anti-inflammatory drug, can prevent brain changes linked to Parkinson's disease in 108 people with REM sleep behavior disorder (RBD). RBD often precedes Parkinson's. Participants will receive adalimumab or a placebo every two weeks for two …

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

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

This study tests a family-oriented version of the Diabetes Prevention Program for Black families in Southwest Georgia. Parents with prediabetes and their children aged 8-15 will learn healthy eating and exercise habits together. The goal is to help parents lose weight and lower t…

This phase 3 trial tests whether acoramidis can prevent or delay transthyretin amyloidosis (ATTR) in 587 adults who carry a faulty gene but have no symptoms yet. ATTR causes sticky plaques to build up in the heart and nerves, leading to heart failure and nerve damage. Acoramidis …

This trial tests whether a single polypill combining three medications—finerenone, empagliflozin, and losartan—can help prevent heart failure and maintain exercise capacity in people with type 2 diabetes who are at high risk. Participants will take either the polypill or the same…

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

This trial tests a 4-week diet and exercise program called Small Steps for Big Changes in adults with prediabetes. Participants will attend supervised exercise sessions and learn to cut sugar and refined carbs while increasing vegetables. The study aims to see if this program can…

This trial compares two blood-thinning drugs—aspirin and cilostazol—to see which one better prevents major heart and blood vessel events in people with type 2 diabetes who are already at high risk. About 1,200 participants will be randomly assigned to take one of the two medicati…

This study adapts a diabetes prevention program for Hispanic adolescents aged 12-16 with obesity, delivering it via telehealth using a Fitbit, chatbot, and online lessons. The goal is to see if this approach is feasible and acceptable for reducing type 2 diabetes risk. Researcher…

This pilot study tests whether a family-centered health and wellness coaching program can help prevent type 2 diabetes in South Asian and Black African/Caribbean families in Ontario, Canada. Each family dyad includes an adult caregiver and a youth aged 14–24. Participants receive…

This study is testing whether a digital tool that encourages self-reflection and healthy habits can prevent type 2 diabetes. Over 40,000 adults without diabetes will use the app or receive usual care for three years. Researchers will track how many people develop diabetes in each…

This trial tests whether a community-based screening program using advanced eye imaging (OCT, fundus photography, and pressure checks) can prevent vision loss from age-related macular degeneration, diabetic retinopathy, and glaucoma better than simple vision tests alone. About 60…

This study tests a three-session training program to help older adults (65+) who use insulin learn to use a continuous glucose monitor (CGM). Participants will be randomly assigned to the program or standard training. The goal is to see if the program improves how often they wear…

This study tests a cream called roflumilast 0.15% for itching in people with mild to moderate atopic dermatitis (eczema). About 40 participants aged 12 and older will apply the cream once daily for 4 weeks. Researchers will track how patients describe their itch and other skin se…

This study tests whether a gamified education program can improve health literacy and quality of life in children aged 8-12 with type 1 diabetes. Half of the 52 participants will receive the gamified program plus standard education, while the other half gets standard education al…

This trial tests whether kangaroo care—holding a diaper-clad baby skin-to-skin on the chest—can reduce pain during a routine heel-prick blood test in newborns receiving phototherapy for jaundice. It compares two approaches: kangaroo care given by the mother versus by a nurse. The…

This study tests whether eating protein and fat (scrambled eggs) before carbohydrates (toast) can reduce blood sugar spikes in women with gestational diabetes. Thirty-five pregnant women will eat the same breakfast in two different orders on separate days, acting as their own con…

This study tests a program called enCompass Humana, which provides coaching and resources to unpaid caregivers of adults with both cancer and diabetes. The goal is to see if the program is practical and helpful for caregivers, improving their coping skills and reducing loneliness…

This pilot study tests a new flexible bandage called exciflex that delivers mild electrical stimulation to chronic wounds that won't heal normally. The study includes 16 veterans with diabetes or spinal cord injury who have lower leg wounds. The bandage is designed to work for up…

This pilot study tests a care model called Patient Priorities Care (PPC) for older African Americans with heart disease plus other chronic conditions like diabetes or COPD. The approach shifts focus from treating each disease separately to aligning care with what matters most to …

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

This study looks at whether a common diabetes medication (GLP-1 RA) can improve muscle strength and bone health in women over 65 with type 2 diabetes. About 20 participants will take the drug as part of their regular care and undergo tests like grip strength and bone density scan…

This trial tests whether taking Ceylon cinnamon supplements for six months can reduce pain and improve blood sugar, cholesterol, and body weight in adults with painful diabetic peripheral neuropathy. Participants are randomly assigned to receive either cinnamon capsules or a plac…

This trial tests a short, child-friendly psychoeducation program called Education & Care in RARE for children and teens aged 5–20 with any rare disease. The program uses simple, resource-oriented materials to teach kids about their condition and build coping skills. Researchers w…

This study tests whether a supervised cardiac rehabilitation program is feasible and beneficial for people with cardiac amyloidosis, a condition where abnormal protein builds up in the heart. Participants attend three one-hour exercise sessions per week for 12 weeks. The study me…

This study investigates whether low-frequency pulsed electromagnetic field (PEMF) therapy can improve vibration perception and protective sensation in people with diabetic foot ulcers. Forty-two participants with type 2 diabetes and Wagner grade 1 ulcers will receive either real …

This study tests whether a supplement called Normast3 can reduce nerve pain and improve nerve function in adults with diabetic polyneuropathy, a common complication of diabetes. About 40 participants will take either Normast3 or a placebo twice daily for 12 weeks, while researche…

This study tests a personalized program combining exercise and nutrition advice to help reduce fatigue in people with indolent (slow-growing) lymphomas, such as chronic lymphocytic leukemia and follicular lymphoma. Participants receive an individualized exercise plan and dietary …

This study tests whether pairing teens with type 1 diabetes with a young adult peer mentor can help them become more physically active and improve their quality of life. The 12-week program includes in-person, virtual, and text-based support. Researchers will measure how well the…

This study will test a new screening program for 3,000 people turning 50 in the Ascoli Piceno province of Italy. Participants will fill out questionnaires and get a blood test to check their risk of developing type 2 diabetes and cardiovascular disease. The goal is to see if this…

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

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

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

This study aims to learn more about insulin resistance and diabetes by collecting blood and skin samples from up to 1,200 participants, including patients with severe insulin resistance and their family members. Researchers will study genetics and how insulin works in the body to…

This study is testing whether a mobile app called Fabla-diabetesMM, which lets people record audio diaries, is easy and helpful for older adults with type 1 or 2 diabetes who also have other health conditions. Thirty participants will use the app and answer questions about their …

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

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

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

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

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

This study is testing whether a continuous glucose monitor (CGM) can predict blood sugar problems in people who are at risk for type 1 diabetes but have no symptoms. Researchers will follow 75 people aged 5 to 39 who have diabetes-related autoantibodies. They will compare CGM dat…

This study looks at anxiety related to cancer screenings in adults with Fanconi anemia, a rare condition that raises cancer risk. Researchers will observe 20 participants during their regular screening visits, noting body language, worries, and the clinic environment. No treatmen…

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

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

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

This study follows up to 30 children (up to age 16) with a rare genetic condition called methylmalonic acidemia (MMA), which affects how the body processes food. The goal is to observe how the disease progresses over time by tracking specific blood tests and health signs. This in…

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

This study looks at whether tiny particles released by fat cells, called adiposomes, carry harmful fats that damage blood vessels in people with obesity and type 2 diabetes. Researchers will test if a 12-week aerobic exercise program or weight-loss surgery can reduce these harmfu…

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

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

This study looks at how medium-chain and long-chain fatty acids affect metabolism in people with Medium-Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) and healthy individuals. Researchers will measure ketone bodies, resting metabolic rate, and other factors after participants co…

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

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

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

This study enrolls up to 15,000 children with known or suspected endocrine or metabolic disorders, along with their family members. Researchers will collect medical records, blood, saliva, and other samples to identify genetic changes linked to these conditions. The goal is to be…

This study collects blood samples from 10,000 healthy adults using both a fingerstick and a traditional needle draw from the arm. The goal is to compare lab results from the two methods and see how well fingerstick samples hold up after a few days of storage. The findings could h…

This study looks at how exercise changes the amount of sugar the liver makes in people with type 2 diabetes and fatty liver disease. Researchers will measure liver sugar production during rest and exercise in 12 adults aged 25-55. The goal is to better understand how exercise hel…

This early-stage trial is testing how the experimental drug repinatrabit interacts with several common medications, including birth control pills, metformin, and methotrexate. The study involves 48 healthy volunteers and will measure drug levels in the blood to see if combining t…

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

This early-stage study tests the safety of a new drug called AZD4248 in healthy volunteers and people with chronic kidney disease and type 2 diabetes. Participants receive either the drug or a placebo, and researchers monitor side effects and how the drug moves through the body. …

This study looks at how the body processes repinatrabit (JNT-517), an oral tablet for phenylketonuria, in people with moderate liver impairment or severe kidney impairment. It compares these results to those from healthy volunteers of similar age, weight, and sex. The goal is to …

This study investigates whether people who had carpal tunnel surgery might have a higher chance of having ATTR amyloidosis, a condition where abnormal proteins build up in tissues, potentially affecting the heart. Researchers will biopsy tissue from about 180 patients who had car…

This study follows 106 men who have had surgery for prostate cancer to see how well they recover bladder control, sexual function, and overall health. Participants will receive standard care plus optional exercise programs. The goal is to better understand recovery timelines and …

This study looks at how metabolic problems in type 2 diabetes might affect the way the brain and heart communicate. Researchers will measure brain activity, heart rhythm, physical fitness, and mental well-being in about 46 adults with type 2 diabetes. The goal is to see if these …

This study follows 30 people with Gaucher disease who are receiving standard medical care to better understand how the condition affects the lungs, bones, and nervous system over time. Researchers will track changes in breathing, bone density, and neurological signs, along with b…

This study is building a large international registry of people with Wilson's disease, a rare genetic condition that causes copper to build up in the body. Researchers will collect data from routine clinic visits over several years to describe the natural course of the disease in…

This study is for adults aged 18 to 55 who were diagnosed with type 1 diabetes within the last 6 months. Researchers want to see how many of them carry a specific gene called HLA-DRB1*04:01. Participants will provide a blood sample or cheek swab for genetic testing. The goal is t…

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

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

This study is testing whether a pharmacist-led program that uses continuous glucose monitors (CGMs) can help people with prediabetes better control their blood sugar and make healthier choices. About 40 adults with prediabetes will wear a CGM for 12 weeks and receive guidance fro…

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

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

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

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

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

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

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

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

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

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

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

This study watches people with blood cancers like CLL who take BTK inhibitors or venetoclax to see how these drugs affect heart rhythm. Researchers will use EKGs, stress tests, and heart monitors to track abnormal rhythms and sudden death risk. The goal is to better understand an…

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

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

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

This study uses a special PET scan tracer, [18F]MH.MZ, to measure serotonin 2A receptors in the brains of people with neurodegenerative diseases like Parkinson's. Researchers want to see if these receptors differ between patients with psychosis and healthy people of the same age.…

This study is investigating how immune cells called macrophages in fat tissue handle fat and influence metabolism. Researchers will compare people with obesity and related metabolic issues to lean individuals, using imaging, blood tests, and tissue biopsies. The goal is to unders…

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

This study investigates how immune cells in fat tissue, called macrophages, handle fats and whether this affects metabolism in people with obesity. Researchers will compare adults with obesity and type 2 diabetes or fatty liver to lean individuals, using imaging, blood tests, and…

This study aims to create a nationwide registry of Indian children with hepatic glycogen storage diseases (GSDs), a group of inherited metabolic disorders affecting the liver and muscles. By collecting genetic, clinical, and outcome data from 250 children, researchers hope to map…

This study follows 130 adults with hypophosphatasia, a rare bone disorder, to understand how they are diagnosed and what symptoms they have. Researchers hope to find ways to reduce the long delay between first symptoms and diagnosis. No treatment is being tested; this is purely a…

This study uses a special eye imaging technique called OCT angiography to look at tiny blood vessels in the retina of people with diabetic retinopathy. Researchers will measure vessel density and other vascular features, then check whether these relate to visual sharpness and blo…

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

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

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

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

This study looks at how well vaccines for flu, COVID-19, shingles, hepatitis B, and pneumonia work in people with certain blood cancers like chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphomas. Researchers will measure immune responses through blood samples before and af…

This study uses a special MRI technique called magnetic resonance spectroscopy to measure brain chemicals in adults with Type 3 Gaucher disease. The goal is to find markers that show how the disease affects the brain and how it responds to treatment. About 5 participants aged 18 …

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

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

This study is checking if the diabetes medicine exenatide (Byetta, Bydureon) increases the risk of pancreatic cancer. Researchers will look at health records from 24,000 patients across seven European countries, comparing those who started exenatide with those who started other d…

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

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

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

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

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

This pilot study is testing whether a real-time continuous glucose monitor (FreeStyle Libre 3) can reveal differences in blood sugar patterns between people with diabetes who have gastroparesis (delayed stomach emptying) and those without it. About 50 adults with type 1 or type 2…

This study looks at how energy production inside cells (mitochondria) works in people with cystinosis, a rare disease that causes cystine buildup and early aging. Researchers will measure cell energy activity in 25 patients and compare it to healthy people. The goal is to underst…

This study is collecting blood samples and health data from 15,000 symptom-free adults over 40 to understand what healthy aging looks like at the molecular level. By tracking participants over time, researchers hope to establish normal ranges for thousands of blood markers and id…

This study uses a brain imaging technique called functional near-infrared spectroscopy (fNIRS) to observe brain activity in people with urea cycle disorder (UCD) while they perform driving tasks of varying difficulty. Researchers compare these brain patterns to those of healthy v…

This study looks at whether a standard blood test measurement called red cell distribution width (RDW) is linked to HbA1C levels in people with type 2 diabetes. Researchers will measure RDW and HbA1C in 250 adults with uncontrolled diabetes at the start, then again after 3 and 6 …

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

This early-phase trial investigates whether oxidative stress—an imbalance of damaging free radicals and protective antioxidants—causes small blood vessel dysfunction in otherwise healthy women who previously had gestational diabetes. Researchers will deliver two drugs (acetylchol…

This study is collecting blood, stool, genetic information, and health records from 1,200 Medicaid patients in South Carolina — some with type 2 diabetes and some without. Researchers will analyze this data to find patterns that could predict chronic kidney disease, a common comp…

This observational study uses a painless, non-invasive eye scan called iris OCTA to measure blood flow in the iris of people with type 2 diabetes. Researchers want to see if blood-flow patterns differ between healthy eyes, diabetic eyes without complications, and those with diabe…

This study investigates whether testicular cancer survivors who received cisplatin-based chemotherapy and bone marrow transplant have hidden heart disease that standard checkups might miss. Researchers will use advanced blood tests and CT scans to measure cholesterol, plaque in t…

This observational study follows 1,000 pregnant people with type 1 diabetes who use either a commercial or an open-source automated insulin delivery (AID) system. Researchers aim to compare maternal and neonatal outcomes, blood sugar control, and emotional well-being between the …

This study investigates whether genetic differences influence how people respond to semaglutide, a GLP-1 drug used for weight loss and diabetes. Overweight or obese volunteers of Amish descent will receive weekly semaglutide injections for six weeks. Before and after treatment, r…

This observational study examines how a phenylalanine-restricted diet and PKU medications like sapropterin influence the gut microbiome in children aged 3-17. Researchers will compare stool samples from children with PKU and their healthy siblings to see if diet or drugs alter gu…

This study explores whether a person's psychological profile — Type A (competitive, ambitious) or Type B (relaxed, less stress-prone) — affects the healing of diabetic foot wounds. Researchers will compare Bortner questionnaire scores between people whose wounds heal and those wh…

This pilot study investigates whether regularly chewing a hard gum for four weeks can produce measurable changes in facial shape. Twenty healthy adults aged 20 to 40 will chew a standardized hard gum every other day, and researchers will use 3D facial imaging to track any shifts …

This study surveys adults taking GLP-1 medications (like semaglutide or tirzepatide) for diabetes or weight loss to understand their resistance training habits before and after starting the drug. The goal is to learn how often people strength train, what their workouts look like,…

This study investigates whether a person's glycolytic power — how efficiently their body uses sugar for quick energy — can predict muscle growth from resistance training. Healthy adults who have not done structured weight lifting in the past year will take a 10-second all-out cyc…

This study uses a special MRI technique to measure energy molecules (ATP and phosphate) in the leg muscles of people with phosphate diabetes, a condition that causes bone pain and muscle fatigue. By comparing measurements at rest and during exercise, researchers hope to uncover t…

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

This study investigates how acetaldehyde, a byproduct of alcohol, affects cells in the mouth and whether it may increase oral cancer risk. Healthy volunteers and individuals with Fanconi anemia (a condition linked to higher cancer risk) will consume a single dose of alcohol and p…

This study tests how well the adhesive parts of a future patch pump stick to the skin of children aged 7 to 17 who need insulin for diabetes. The device is a dummy—it has no electronics and just mimics the shape and weight of a real pump. Researchers will see if the patch stays a…

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

This study screens children and adolescents aged 5 to 18 who have chronic conditions like congenital heart disease, inflammatory bowel disease, celiac disease, or rheumatic diseases for lipid metabolism disorders. Participants provide a blood sample and complete a questionnaire a…

This study follows people with congenital disorders of glycosylation (CDG) — rare genetic conditions that affect how the body builds sugar chains on proteins. Researchers aim to track how the disease progresses over time, including its impact on organs and thinking abilities. By …

This study compares quality of life and psychological factors—such as anxiety, depression, stress, and social support—between pregnant women with gestational diabetes and those without. Researchers will survey 206 women aged 20–40 in India using standard questionnaires. The goal …

This study looks at how different phases of the menstrual cycle affect blood glucose changes during exercise in women with type 1 diabetes who take oral contraceptives. Participants wear a continuous glucose monitor and complete a 45-minute aerobic exercise session during both th…

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

This study follows 400 people in Norway with multiple myeloma, plasma cell leukemia, or AL amyloidosis who are receiving newer immunotherapies like teclistamab, elranatamab, or CAR-T cell therapy outside of clinical trials. Researchers will track how well these treatments control…

This study investigates how the diabetes medication empagliflozin influences the body's production of glucose, fats, and ketones in people with type 2 diabetes. Researchers will also test whether blocking the sympathetic nervous system with propranolol changes these effects. The …

This study investigates how the diabetes medication empagliflozin (an SGLT2 inhibitor) increases ketone production and fat breakdown in people with type 2 diabetes. Researchers will also test whether another drug, pioglitazone, can prevent these effects. The goal is to understand…

This study investigates why SGLT2 inhibitors, a common diabetes medication, can sometimes lead to ketoacidosis—a dangerous buildup of acids in the blood. Researchers suspect it only happens when patients also have low blood volume and low insulin levels. Adults with type 2 diabet…

This study builds a global database of people diagnosed with or at risk for Krabbe disease, a rare nerve disorder. Researchers will track symptoms, genetic mutations, and enzyme levels to find clues that predict when the disease starts and how severe it becomes. The registry also…

This study compares walking patterns between people with diabetic peripheral neuropathy (nerve damage from diabetes) and healthy adults. It also tests whether giving people real-time feedback on their walking form can immediately change how they walk. The goal is to understand th…

This study measures four specific substances in saliva—apelin, chemerin, ghrelin, and lipocalin 2—in people with and without type 2 diabetes and gum disease. Researchers want to see if these substances are linked to the presence or severity of these conditions. The goal is to fin…

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

This study investigates whether people with phenylketonuria (PKU) are more likely to develop eating disorders and obesity due to their restrictive, high-carbohydrate diet. Researchers will examine 70 PKU patients to understand the link between diet, eating behaviors, and weight. …

This study measures how common heart disease, diabetes, obesity, and infections like dengue and COVID-19 are among adults in La Reunion. Researchers will also look at possible risk factors, including gut bacteria, genetics, and social conditions. Participants provide blood, DNA, …

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

This study follows 1,000 adults with diabetes to find early signs that predict serious complications like nerve damage, eye disease, kidney problems, and heart issues. Researchers measure blood sugar control and other health markers over time. The goal is to develop better risk a…

This study looks at how blood sugar levels affect immune cells (monocytes) and kidney cells in people with type 1 diabetes and early chronic kidney disease. Researchers will compare participants with better versus worse blood sugar control to see if certain immune cells become mo…

This study is a national registry in Hungary that collects medical information from people diagnosed with systemic amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will look at patient records to understand how the disease starts, how it is diag…

This study explores whether a small accelerometer worn on the thigh can objectively measure how well treatments work for neurogenic orthostatic hypotension—a condition causing dizziness and fainting upon standing. Participants wear the device for one week on placebo and one week …

This study looks at how well people with Anderson-Fabry disease can exercise and how their hearts respond to stress. Researchers will use treadmill tests, echocardiograms, and heart scans to measure heart function during rest and exercise. The goal is to find better ways to detec…

This study follows 600 Indigenous families in Manitoba to understand how maternal health, genetics, and early-life environment contribute to obesity and type 2 diabetes in children. Pregnant women with type 2 diabetes, gestational diabetes, or no diabetes are enrolled, and their …

This study looks at children with suspected or confirmed genetic and metabolic disorders to find new disease-causing gene mutations. Researchers will analyze blood samples for DNA and metabolites, and in some cases take a small skin sample. The goal is to better understand these …

This study investigates how two days of forearm immobilization (keeping the arm in a cast or sling) changes the way muscles take up glucose and amino acids. Researchers will compare these effects in people with type 2 diabetes and healthy volunteers. The goal is to understand why…

This study investigates whether the speed at which carbohydrates are digested and absorbed affects how rewarding they feel. Researchers will give healthy adults flavored drinks sweetened with either real sugar or a no-calorie sweetener, then measure changes in brain activity, met…

This study investigates how muscle fiber type and protein makeup relate to early insulin resistance, a key step before type 2 diabetes develops. Researchers will follow 250 healthy adults aged 20-30 for up to 20 years, using muscle biopsies, scans, and wearable sensors. The goal …

This study investigates how changes in kidney cell metabolism relate to the rate of kidney function decline in people with ANCA vasculitis and other kidney diseases. Researchers will analyze kidney biopsy samples and blood/urine tests from 146 participants over one year. The goal…

This study investigates whether 6 weeks of treadmill walking with blood flow restriction cuffs on the legs can improve immune cell function in adults with type 2 diabetes. Participants walk three times per week, with some wearing inflatable cuffs that partially limit blood flow. …

This study looks at basic blood and clotting measures in children with type 1 diabetes compared to healthy children. Researchers want to see if diabetes causes changes in red blood cells or clotting that could lead to complications. The study involves children aged 1 month to 18 …

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

This research study follows 500 adolescents with type 2 diabetes to understand why some develop kidney problems. Researchers are looking at biological factors like blood sugar and blood pressure, as well as psychological and social factors such as stress, mental health, and pover…

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

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

This study creates a registry for people diagnosed with Wilson disease, a rare genetic condition that causes copper buildup in the body. Researchers will collect medical data and biological samples over time to help future studies improve diagnosis and treatment monitoring. The g…

This study examines the medical records of 400 people diagnosed with various types of lymphoma at a French hospital between 1999 and 2018. Researchers want to see how long patients survived and whether the care they received matched what medical guidelines recommend. The goal is …

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