Metabolic disease

This study tests a new drug called KK8123 for adults with X-linked hypophosphatemia (XLH), a rare genetic condition that causes low phosphate levels and bone problems. The trial has two parts: first, finding the right dose, then checking safety and how well it works over time. Ab…

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

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

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

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

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

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

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

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

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

This early-phase study tests a new treatment called OPF-310, which uses pig islet cells enclosed in a protective capsule, for people with unstable type 1 diabetes who still experience severe low blood sugar episodes despite using an automated insulin pump and continuous glucose m…

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 early-stage trial tests a new mRNA medicine called IN026 in 16 adults with refractory gout — gout that doesn't get better with standard treatments. The drug is given as an IV infusion and is designed to lower uric acid levels and shrink uric acid crystals (tophi). The main g…

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 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 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 study tests whether a minimally invasive procedure called endoscopic sleeve gastroplasty (ESG), which reduces stomach size by 70%, can help reverse fatty liver disease (MASH) in people with obesity. Researchers will compare ESG plus lifestyle changes to lifestyle changes alo…

This study tests whether a 4-month structured telerehabilitation program, delivered via video calls and an app, can reduce deaths and hospital readmissions in heart patients who have Post-Intensive Care Syndrome (PICS). About 326 adults aged 30-75 who were in the ICU for at least…

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This early study tests whether combining electrical stimulation cycling with blood flow restriction cuffs is safe and doable for people with chronic spinal cord injury. Six adults will try the 20-minute exercise sessions over 6 weeks. The goal is to see if this approach can be us…

This phase 3 trial tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. Participants are randomly assigned to receive either the active treatmen…

This study looks at the long-term safety of an experimental medicine called mRNA-3927 for people with propionic acidemia, a rare genetic disorder that prevents the body from breaking down certain proteins and fats. About 50 participants who were in an earlier study will continue …

This pilot study tests a new procedure called PSAM (Pylorus-Sparing Antral Myotomy) for weight loss in 30 adults with obesity. The procedure involves cutting some stomach muscles to slow down how fast food leaves the stomach, which may help people feel full longer and lose weight…

This study tests whether giving ICU patients extra protein and activating their muscles early (using electrical stimulation and movement) can prevent severe muscle loss. About 40 critically ill adults on ventilators will take part. The goal is to see if this approach helps patien…

This study tests whether a high-phenolic olive oil can improve metabolic health better than a low-phenolic version. Over 8 weeks, 80 adults with overweight/obesity and metabolic issues will consume one of the two oils. Researchers will measure changes in blood fats, blood pressur…

This study tests a blood filtering procedure called rheopheresis to treat calciphylaxis, a rare condition causing painful skin wounds in people on dialysis. About 138 participants will receive either rheopheresis or a sham procedure to see if it helps wounds heal completely withi…

This study tests an experimental mRNA therapy called mRNA-3927 for propionic acidemia, a rare genetic disorder that causes dangerous metabolic crises. The trial involves about 77 participants of all ages, starting with older children and adults. Researchers will check safety, fin…

This study tests a special clinic designed to help people with severe mental illness (like schizophrenia or bipolar disorder) lose weight and improve their metabolic health. About 84 participants will either receive care at this clinic or standard care for 12 months. The main goa…

This study looks at whether time-restricted eating (eating only during certain hours) can improve blood pressure and insulin sensitivity in people who work night shifts. Night shift workers are at higher risk for heart disease and type 2 diabetes. Researchers will compare 4 weeks…

This study tests whether taking a daily oil rich in linoleic acid (a type of omega-6 fat) for 8 weeks can improve insulin resistance in adults aged 20-60 who have this condition plus at least one other cardiometabolic risk factor (like abdominal obesity or high blood pressure). P…

This study tests whether a special high-intensity interval training (HIIT) program can improve heart health in school-age children who are at high risk for cardiovascular disease. The program involves short bursts of intense exercise, done several times a week. Researchers will t…

This study tests whether adding stair climbing to a brisk walking routine lowers blood pressure more than walking alone. 450 adults with high blood pressure, overweight, and a sedentary lifestyle will be split into three groups: stair walking plus brisk walking, brisk walking onl…

This study follows adults aged 30-65 who previously tried a fasting-mimicking diet (FMD) to see if they can maintain fat loss and heart health improvements for one more year. Participants will continue FMD cycles every three months. The goal is to check if these benefits last and…

This program offers extra stem cells from a half-matched family donor to patients who are getting a cord blood transplant for serious blood cancers or immune disorders. The goal is to help the bone marrow recover more quickly while the cord blood cells take over permanently. It i…

This study is testing whether umbilical cord blood stem cells from unrelated donors can safely and effectively restore blood cell production in people with serious conditions like leukemia, immune deficiencies, and inherited blood disorders. The main goal is to see how quickly th…

This study tests whether following exercise and healthy eating guidelines can improve heart health and reduce metabolic problems in breast cancer survivors. Forty-five postmenopausal women who had breast cancer will be split into three groups: exercise only, exercise plus dietary…

This study tests whether an online training and support platform can help adults with type 1 diabetes better manage their blood sugar and well-being. About 322 participants across Canada will use the platform for 12 months, either as part of their usual clinic care or on their ow…

This study tests whether an online training and peer support platform (Support-t) helps teens with type 1 diabetes prepare for the move from pediatric to adult healthcare. About 200 teens aged 14-16 will either use the platform plus usual care or just usual care. The main goal is…

This pilot study tests whether a dietary supplement called GLYLO can lower harmful compounds called AGEs in overweight or obese postmenopausal women aged 45-65. Participants take either GLYLO or a placebo for 24 weeks, and researchers measure changes in AGEs, insulin resistance, …

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 study tests two self-care methods—virtual ear acupressure and Baduanjin qigong—to prevent or reduce period pain in 145 women aged 16–35 with primary dysmenorrhea. Participants will be randomly assigned to one of three groups and practice their assigned technique for 12 weeks…

This study looks at whether cognitive-behavioral therapy (CBT), exercise training, or both can help prevent type 2 diabetes in teenage girls who are at risk. The researchers want to see if these approaches lower stress, improve mood, boost physical activity, and reduce insulin re…

This study tests whether 4 weeks of high-intensity interval training (HIIT) or time-restricted eating (TRE) can improve health markers linked to type 2 diabetes in adolescents and young adults with overweight or obesity. Forty participants will be randomly assigned to one of the …

This study tests whether a non-medication sleep program (CBT-I) can improve sleep, PTSD symptoms, and heart health in older adults with PTSD who still have sleep problems after PTSD therapy. About 167 veterans aged 50 and older will receive PTSD therapy first, then the sleep prog…

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

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

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

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

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

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

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

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

This early-stage study is testing a new drug, SNS851, in 52 healthy adults to see if it is safe and how the body processes it. Participants will receive either the drug or a placebo by injection. The goal is to check for side effects, not to treat any disease.

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

This 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 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 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 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 observational study will follow 50 adults and children who are starting long-term parenteral nutrition (IV nutrition) to measure the buildup of advanced glycation end-products (AGEs) in their skin. AGEs are harmful compounds that form when sugar binds with proteins, and they…

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 looks at how certain immune cells, called meta-inflammatory monocytes, along with body composition and diet, may contribute to insulin resistance in adolescents. Researchers will collect blood samples and health information from 175 teens aged 14-18, including those wi…

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 looks at how a person's genetic makeup influences their response to oral semaglutide (Rybelsus), a drug used for type 2 diabetes. Researchers will give 125 adults with prediabetes a 14-day course of the drug and measure blood sugar, insulin, and other factors after a s…

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 at Necker Hospital in Paris is checking how well patients with inherited metabolic diseases follow their daily oral medication routines. About 200 patients (children aged 7 and up, teens, and adults) will fill out a questionnaire during a regular visit. The goal is to …

This study is creating a large collection of blood, urine, stool, and heart tissue samples from 10,000 people with and without heart or metabolic conditions. The goal is to store these samples along with medical information to speed up future research into what causes these disea…

This study looks at whether people born with a low birth weight have a higher chance of developing non-alcoholic fatty liver disease (NAFLD) as adults. Researchers will compare 250 low-birth-weight adults with 50 normal-birth-weight adults using liver scans and other tests. The g…

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 …

This study tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk together about stem cell transplant or cellular therapy decisions. The goal is to see if the tool is easy to use and helpful for families. About 60 …

This study looks at how differences in people's gut bacteria affect the way their bodies process nutrients from blueberries. Researchers will give 125 healthy adults a single blueberry drink and measure the levels of blueberry compounds in their blood and urine over time. The goa…

This study looks at whether people with certain rare metabolic disorders that cause high ammonia levels will measure their ammonia at home every day. About 30 participants will use a special device to check their ammonia, along with temperature, heart rate, and blood oxygen. They…

This study looks at a layer of fat on the heart called epicardial fat tissue (EFT) in adults aged 50-70 from Russia and Central Asia. Researchers want to see if thicker EFT is linked to chronic diseases like heart problems and obesity. About 500 participants will have an ultrasou…

This study is collecting blood samples and health data from 10,000 hospitalized patients with metabolic diseases like diabetes, obesity, or metabolic syndrome. Researchers will analyze genetic and other markers to create a personalized risk profile for death. The goal is to impro…

This study looks at 240 adults aged 40-60 to see if being overweight or obese in midlife is linked to early signs of Alzheimer's in the brain. Participants will have metabolic tests and brain scans (MRI and PET) to measure inflammation, brain shrinkage, and protein buildup. The g…

This study is creating a large database of 8,000 adults with heart, kidney, or metabolic diseases to track their health over up to 5 years. Researchers will collect medical records, imaging, and lab data to understand how these conditions interact and affect long-term outcomes. T…

This study looks at what happens inside your fat tissue when you lose weight with or without exercise. 68 adults with obesity will lose 10% of their body weight through diet, with some also doing moderate exercise. Researchers will measure insulin sensitivity, fat breakdown, and …

This study looks at 150 adults with obesity to see if there are differences in fat, muscle, and metabolism between those who were obese as children and those who became obese as adults. Participants will provide tissue samples, blood, and urine, and undergo a weight loss program.…

This observational study will enroll 8,000 adults undergoing routine health checkups. Researchers will measure hormone levels and collect health data to understand how environment, genes, and proteins interact in conditions like high blood pressure and diabetes. The goal is to cr…

This study looks at how being in hot conditions during the day, at night, or both affects the body and mind. Thirty-five healthy adults aged 18-30 will spend time in a temperature-controlled room. Researchers will measure heart function, lung capacity, blood markers of inflammati…

This study will enroll 5,000 adults with kidney disease or at risk for it, including transplant recipients. Researchers will analyze blood and urine samples to find molecular patterns that predict disease progression, complications, or treatment response. The goal is to improve r…

This study aims to find out how many adults in rural Bangladesh have metabolic fatty liver disease (MAFLD) and how it relates to their diet and metabolic health. Researchers will check 1,000 participants with physical exams, blood tests, and ultrasound. The goal is to gather basi…

This pilot study is looking at why some patients in the intensive care unit (ICU) develop severe muscle weakness after surgery. Researchers will take blood and small muscle samples from 20 high-risk patients to look for chemical changes that might cause or signal this weakness. T…

This study looks at how adding different types and combinations of dietary fiber to scones affects blood sugar, feelings of fullness, and gut bacteria. Overweight but otherwise healthy adults will eat seven different fiber-enriched scones over several days. Researchers will track…

This study uses MRI scans to look for brain differences in people with metabolic diseases compared to healthy volunteers. Researchers will track changes over time and link them to body fat and other health measures. About 126 adults will take part at Ulm University Hospital. No d…

This study looks at how severe obesity and weight-loss surgery change natural chemicals in scalp hair. Researchers will track 60 adults—some with severe obesity scheduled for bariatric surgery and some healthy-weight controls—to see if hair chemistry can signal poor metabolic hea…

This study looks at whether eating beef every day as part of a heart-healthy diet helps older adults (ages 55-70) absorb zinc better and improves how their blood vessels work. Researchers will measure zinc levels in cells and blood vessel function in 20 participants. The goal is …

This study looks at how the brain reacts to different nutrients (sugar, fat, or protein) in people with obesity compared to healthy-weight individuals. Researchers will use brain scans and blood tests to see if there are differences in brain activity and metabolism after eating. …

This study looks at how moderate wine consumption changes tiny particles called extracellular vesicles, which help cells communicate. Eight healthy adults aged 35-65 will drink red wine, white wine, or water, and researchers will analyze their blood for changes in these particles…

This study looks at how different patterns of light exposure and food intake affect the body's metabolism when sleep is reduced. Researchers will monitor 120 healthy adults aged 18-35 over 4-6 months, including three overnight clinic stays. The goal is to understand how these fac…

This study looks at the genes of 2,750 people in French Polynesia to find out why gout, inflammation, and metabolic diseases like diabetes are common there. Participants must be of Polynesian ancestry and either have gout or be part of the general population. The goal is to disco…

This study is creating a collection of blood, urine, and stool samples from 500 adults with or at risk for heart and blood vessel diseases. Researchers will use these samples to study inflammation, blood clotting, and other biological pathways. The goal is to build a resource tha…

This observational study looks at how bacteria in the gut and mouth are linked to epilepsy and whether they affect how well seizure medicines work. Researchers will collect stool, saliva, and blood samples from 1,500 people with newly diagnosed epilepsy and healthy controls. By a…

This study aims to understand how immune cells use glucose (sugar) for energy and building materials. Researchers will give 12 healthy adults a non-radioactive glucose tracer through an IV and then collect blood samples to track how different immune cells take up and process the …

The SMILE study is tracking 10,000 adults aged 18 and older to see how sarcopenia (age-related muscle loss) and metabolic diseases like diabetes influence overall health and lifespan. Researchers will collect data from medical records, tests, and surveys over time. This is an obs…

This study tests whether eating all meals within an 8-hour window (before 10 AM) for four weeks can improve how well older adults respond to seasonal flu and COVID-19 vaccines. Researchers will measure changes in immune cell activity and body composition in 24 healthy volunteers …

This study looks at how exercise and healthy eating affect heart and metabolic health in women with multiple risk factors. Researchers will follow 180 pre- and postmenopausal women for 6 months, comparing exercise alone, exercise plus diet advice, and a stretching group. The goal…

This study follows 4,094 adults in Shanghai who were part of an earlier health survey. Researchers will track them for up to 10 years to see how heart disease, kidney problems, and metabolic conditions like diabetes and obesity develop and interact. The goal is to better understa…

This study aims to find biological markers that show the balance between calories you eat and calories you burn. Researchers will study 400 adults to better understand metabolism. The goal is to develop simple tests that could one day help people manage their weight and metabolic…

This study follows 100 children under 18 who are already on a ketogenic diet for conditions like drug-resistant epilepsy, autism, chronic migraine, or brain tumors. Researchers want to see if the diet supports healthy growth, improves symptoms, and boosts quality of life. The die…