Metabolic disease

Researchers test MNV-201, a cell therapy for children with Pearson syndrome, a rare mitochondrial disease that stunts growth and harms organs. The treatment uses the child's own blood stem cells, enriched with mitochondria from donor placenta, given as a single infusion. The tria…

Researchers are testing a gene therapy called VV-14300 in adults with long-standing type 1 diabetes whose blood sugar remains poorly controlled despite using an automated insulin delivery system. The therapy uses a harmless virus to deliver the glucokinase gene into muscle, where…

Researchers are testing two experimental gene therapies, VV-14299 and VV-14300, in adults with long-standing type 1 diabetes whose blood sugar remains poorly controlled despite using an automated insulin pump. The therapies are injected into muscle and are designed to work togeth…

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

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

This study tests a faster version of a procedure called renal denervation, which uses energy to calm overactive nerves in the kidneys to lower blood pressure. About 130 adults with uncontrolled high blood pressure will receive the quicker procedure. The goal is to see if it works…

Researchers are testing whether a nasal spray made from exosomes, tiny particles released by donor umbilical cord or placental stem cells, is safe and can help people with non-arteritic ischemic optic neuropathy (NAION), a condition that causes sudden vision loss when blood flow …

Researchers are testing how well people with overweight or obesity, with or without type 2 diabetes, tolerate switching from semaglutide to an experimental drug called zenagamtide. All 60 participants receive both drugs as injections. The study measures side effects and how the b…

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

This study 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 tests a device called EchoMark/EchoSure that is implanted during fistula surgery for dialysis patients. The device uses automated ultrasound to monitor healing every two weeks. The goal is to see if it helps the fistula mature faster and reduces the need for additional…

Researchers test whether a specialized cardiometabolic clinic can improve heart function and reduce coronary inflammation in people with type 2 diabetes and established cardiovascular disease. Participants are randomly assigned to the clinic or usual care. The clinic provides ind…

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 study tests whether higher doses of the drug Respreeza/Zemaira can slow lung damage better than the standard dose in adults with emphysema caused by Alpha-1 antitrypsin deficiency. About 270 participants will receive weekly IV infusions for 3 years. The goal is to find the o…

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…

Researchers are testing a teleconsultation care pathway for older adults with hypertension, diabetes, and dyslipidemia at a primary care clinic in Kuala Lumpur, Malaysia. The study compares virtual visits with usual in-person care to see if the virtual approach is feasible and co…

Researchers are testing whether a home-use antibacterial light device, used alongside normal brushing and flossing, can reduce gum bleeding and plaque in people with type 2 diabetes or pre-diabetes. The trial enrolls 150 adults with at least 20 teeth who get dental care through c…

This expanded access program makes the experimental drug elamipretide available to people with genetically confirmed rare mitochondrial diseases, including Barth syndrome, when the condition is serious or life-threatening and no other satisfactory treatments exist. Participants r…

Researchers are testing whether a once-daily combination pill can lower both blood pressure and blood sugar in people who have essential hypertension and type 2 diabetes. The phase 3 trial enrolls about 276 adults who stop their existing blood pressure and diabetes medicines, exc…

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 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 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 early-stage study tests a gene therapy for people with alpha-1 antitrypsin deficiency, a condition that can damage the lungs. The treatment is given once through a vein and aims to produce a special protein that protects the lungs. Researchers will check safety and measure p…

This study tests a gene therapy called DFT383 in 30 children aged 2 to 5 with nephropathic cystinosis, a rare disease that damages kidneys and other organs. The treatment aims to fix the genetic problem so children may no longer need daily cysteamine medication. Researchers will …

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 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 will test a program based on WHO guidelines to improve diabetes screening and care in 10 primary health centers in Nigeria. About 2,800 adults with or at risk for diabetes will take part. The goal is to see if the program helps more people get diagnosed and treated.

Researchers are testing how well people with overweight or obesity, with or without type 2 diabetes, tolerate switching from tirzepatide to a new drug called zenagamtide. The trial aims to find which dose of zenagamtide works best after people have been on a stable dose of tirzep…

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

This Phase 3 trial compares a new once-daily formulation of trientine tetrahydrochloride (TETA 4HCl) to the standard drug D-penicillamine for people newly diagnosed with Wilson's disease. About 38 adults and children (ages 8 and up, weighing at least 55 lbs) will be randomly assi…

This study tests a new device that uses sound waves (lithotripsy) to break up hard calcium deposits in the carotid arteries of the neck before placing a stent. About 185 people with heavily calcified carotid arteries will be enrolled. The main goal is to see if the procedure is s…

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 early-phase trial is testing a new type of immunotherapy called CAR T-cells for people with a specific kind of stomach and esophageal cancer that has spread to the lining of the abdomen. The treatment involves collecting a patient's own immune cells, modifying them in a lab …

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

This trial tests whether adding a second procedure to a standard weight-loss operation leads to greater weight loss and better metabolic health in adults with obesity. The standard procedure, endoscopic sleeve gastroplasty (ESG), uses stitches to make the stomach smaller. The add…

This study tests whether adding a natural gut hormone called gastrin to islet cell transplants can help people with hard-to-control type 1 diabetes produce their own insulin and stabilize blood sugar. About 20 adults aged 18-68 with unstable type 1 diabetes will receive donor isl…

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

This study 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 early-stage trial tests a daily pill called APG-3288 in 180 adults with blood cancers like leukemia or lymphoma that have come back or not responded to standard treatments. The main goals are to find the safest dose and check for side effects. Researchers will also look for …

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

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

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

This study looks at whether starting automated insulin delivery (AID) early in children aged 7–16 with type 1 diabetes improves blood sugar control, reduces stress for patients and families, and prevents long-term complications compared to standard insulin injections. One hundred…

This study follows 350 people with type 1 diabetes in France who use the Control-IQ insulin pump system. Researchers want to see how safe and effective the system is over 12 months, focusing on blood sugar control and quality of life. Participants must be at least 6 years old and…

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 early study tests a one-time gene therapy injection under the retina for children with CLN2 Batten disease who already receive enzyme therapy. The main goal is to check safety in 16 participants, with a secondary look at whether it can preserve vision. It is a first-in-human…

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 study tests whether a 6-month program of problem-solving training, community health worker support, and exercise coaching at a YMCA can improve fitness and heart health in 210 adults with type 2 diabetes, obesity, and low income. Participants are compared to those who receiv…

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

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

This study tests 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 single intravenous dose of a gene therapy called AB-1009 in 12 adults with late-onset Pompe disease. Participants must have been on enzyme replacement therapy for at least 6 months. The main goal is to check safety and side effects, while also seeing if the tre…

The STEP-1 trial tests a device called the RESET System in adults with type 2 diabetes and obesity whose blood sugar is not well controlled. The device is placed in the small intestine during an endoscopy and is designed to alter how the body absorbs nutrients. Participants are r…

This study tests whether the antibiotic rifampin can safely lower high calcium levels in the blood and urine of people with a rare genetic mutation in the CYP24A1 gene. The condition can cause kidney stones and other problems. Sixty participants aged 6 months to 65 years will tak…

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…

Researchers are testing an engineered interleukin-2 drug called MDNA11 in adults with advanced solid tumors. The trial gives MDNA11 alone or with pembrolizumab, an immune checkpoint inhibitor, to see which doses are safe and how the body processes the drug. It also looks for earl…

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

Researchers are running a registry to collect real-world data on a range of Abbott vascular devices, including stents, catheters, plugs, guidewires, and imaging tools. Adults who have a planned or completed procedure using one of these devices can take part. The registry follows …

Researchers are testing whether denosumab, given as injections under the skin, can help heal acute Charcot foot in people with diabetes. The trial enrolls adults aged 18 to 80 with type 1 or type 2 diabetes and a newly diagnosed Charcot foot, which causes a red, swollen, warm foo…

This study tests an experimental drug called ARO-DIMERPA in 78 adults with mixed hyperlipidemia (high cholesterol and triglycerides). The drug is given as a shot under the skin, and researchers are checking if it is safe and how well it lowers fats in the blood. The trial is stil…

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

This study tests whether teplizumab, a drug already approved in the US, can safely delay the start of full-blown type 1 diabetes in Japanese people aged 8 to 34 who are at high risk (Stage 2). Participants will receive a 14-day IV treatment and be monitored for side effects and h…

This study tests a new drug called SAB-142 in people recently diagnosed with type 1 diabetes. The goal is to see if it can help the body keep making its own insulin for longer, slowing the disease. About 159 people aged 5 to 40 will receive either the drug or a placebo, and resea…

This study tests whether giving people a live gut bacterium called Oxalobacter formigenes can lower the amount of oxalate in their urine. High urinary oxalate is a major risk factor for calcium oxalate kidney stones. Forty adults with a history of such stones who lack this bacter…

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

This 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…

Researchers are testing whether a single injection of an experimental gene therapy, 4D-150, can improve vision and reduce the need for regular eye injections in adults with diabetic macular edema. The trial compares 4D-150 to aflibercept, a standard treatment given as repeated in…

This study tests whether a specially designed health behavior program can help people with metabolic syndrome and chronic kidney disease reduce their waist size and improve kidney function. Researchers will compare the program to usual care in 160 adults, measuring changes in wai…

Researchers at Kaiser Permanente are testing a six-session medical nutrition therapy program for adults with type 2 diabetes and high blood sugar. Registered dietitians deliver personalized diet and behavior-change support to about 600 patients in San Bernardino County. The trial…

Researchers compare two metabolic surgery techniques, sleeve and bypass, in adults with type 2 diabetes and moderate obesity. The goal is to see which operation leads to better blood sugar control and remission of diabetes. Thirty patients who have not reached their blood sugar t…

This Phase 2 trial tests the long-term safety and effectiveness of HRS-7249, an investigational injection that targets a protein called APOC3 to lower triglycerides. The study includes people who previously took part in an earlier HRS-7249 trial, plus new participants aged 18 to …

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

This study 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 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…

Researchers are investigating whether giving vitamin C to critically ill patients can lower high triglyceride levels. The study looks back at records of 2,000 patients admitted to an intensive care unit, comparing those who received intravenous vitamin C with those who did not. H…

This phase 3 trial compares an experimental drug, MK-8748, to the standard treatment aflibercept for diabetic macular edema (DME), a condition where fluid buildup in the retina blurs vision. About 1,100 adults with diabetes and vision loss from DME receive either MK-8748 or aflib…

This study tests whether omega-3 fatty acids from fish oil or camelina oil can reduce inflammation and improve heart health in 324 adults over 55 at moderate to high cardiovascular risk. Participants take daily supplements for 12 weeks, and researchers measure changes in blood ma…

This study will test a new way to manage heart disease risk using a digital, nurse-led remote monitoring program. About 750 adults with heart conditions will use home devices and answer questionnaires. The goal is to see if this approach helps more patients reach their cholestero…

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 trial tests whether a lipid score card helps people who have had a heart attack and their doctors follow guidelines for lowering LDL cholesterol. The score card tracks the patient's current LDL level, how well they take their cholesterol medication, and what treatment decisi…

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

This observational study follows up to 2,000 adults with ATTR-CM, a serious heart condition, who are starting treatment with acoramidis as part of their normal care. Researchers will track patient characteristics, how the drug is used, and its effects on heart function and qualit…

This trial tests whether clazakizumab, an experimental anti-inflammatory drug, can lower a blood marker of inflammation called hsCRP in adults who have elevated levels and are at increased risk for heart problems. Participants must have conditions like established heart disease, …

This master protocol supports several early-phase studies testing different amyloid-targeting drugs in healthy volunteers and people with Alzheimer's disease. The main goals are to check safety and tolerability, and to see whether these drugs can remove amyloid plaques from the b…

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 early-stage trial is evaluating an experimental drug called KRRO-121, given as a shot under the skin, to see if it is safe and tolerable. The study first tests KRRO-121 in healthy adults, then in people with urea cycle disorders—a group of inherited conditions that impair th…

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

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

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

Researchers are following people with Alagille syndrome or progressive familial intrahepatic cholestasis who take Livmarli (maralixibat) for up to five years. The study tracks liver transplants, biliary surgery, deaths, growth, bleeding, and fractures to see how the drug performs…

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 trial tests whether a culturally tailored, Spanish-language onboarding and support program helps Latino youth aged 10–20 with type 1 diabetes adopt and consistently use the Medtronic MiniMed 780G automated insulin delivery system. Participants receive one-on-one nutrition su…

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 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 compares two treatments for gestational diabetes: oral metformin pills and injectable insulin. Researchers want to see if metformin is as good as insulin at preventing problems for mothers and babies, like high birth weight or low blood sugar. About 1,500 pregnant wome…

Researchers are testing petrelintide, an experimental once-weekly injection, in adults with overweight or obesity and type 2 diabetes. The trial compares petrelintide to a placebo injection, alongside a reduced-calorie diet and increased physical activity. The main goal is to mea…

This study tests whether adding a structured self-management program (DECIDE) to standard community health worker support helps 500 Philadelphia residents aged 35-75 better manage heart disease risks and meet their social needs. Participants will either receive the enhanced progr…

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 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 whether a smartphone app that shows real-time blood sugar and activity data can help adolescents and young adults with type 2 diabetes improve their glucose control. Participants use a continuous glucose monitor and Fitbit, and some receive prompts to reflect on …

Researchers are studying next-generation MiniMed glucose sensors designed to be worn for extended periods, paired with an algorithm that interprets the readings. The trial enrolls people aged 2 to 80 with type 1 or type 2 diabetes and collects sensor data over time. Investigators…

This program provides an investigational drug, ersodetug, to people with dangerously low blood sugar caused by certain tumors. It is for those who cannot join a formal clinical trial. The goal is to help control blood sugar levels when standard treatments are not enough.

This study tests whether adding evolocumab, a cholesterol-lowering drug, to standard care can slow or stop plaque buildup in heart arteries after a heart attack. About 233 adults aged 40-75 who recently had a heart attack or unstable angina will receive either evolocumab plus sta…

This study tests whether the Medtronic 780G automated insulin pump can improve blood sugar control in adults with type 1 diabetes who also have gastroparesis (stomach paralysis). Participants will use the device for three months, and researchers will measure how often blood sugar…

This study looks at whether adding baked potatoes (with skin) to a Mediterranean diet can improve blood sugar control, blood pressure, and cholesterol in adults aged 45-80 with pre-diabetes. Sixty participants will either get diet education alone or education plus pre-prepared ba…

This study tests a new drug called zodasiran in 12 teens aged 12 to 17 with homozygous familial hypercholesterolemia, a rare genetic condition causing very high cholesterol. The drug is given as a shot under the skin. The main goal is to see if it safely lowers bad cholesterol af…

This early-stage trial tests a new drug called AIR-001 in 54 adults with alpha-1 antitrypsin deficiency (AATD) who have a specific genetic type (PiZZ). The study aims to check safety and how the body processes the drug. Participants receive increasing doses of AIR-001 under the s…

This study tests a gene therapy called RP-A501 in 14 males with Danon disease, a rare genetic condition that causes heart problems. The treatment uses a harmless virus to deliver a working copy of the LAMP2 gene, aiming to improve heart muscle function and reduce thickening. Part…

This early-phase study tests a new oral drug called GS-4571 in healthy people, non-diabetic obese individuals, and non-obese people with type 2 diabetes. The main goals are to see how the drug moves through the body, how safe it is, and how food or acid-reducing drugs affect it. …

This large phase 3 trial is testing whether colchicine (a gout medication) and low-dose aspirin, taken together or alone, can prevent heart attacks, strokes, and cardiac death in 10,000 high-risk people with type 2 diabetes who have not yet had a heart-related event. Participants…

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

This 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 early-access program tests whether a weekly infusion of tralesinidase alfa directly into the brain's fluid spaces is safe and tolerable for children with Sanfilippo syndrome type B, a rare genetic disorder that causes severe neurological decline. The study enrolls about 10 c…

This study tests a nurse case management program for formerly incarcerated Black men with poorly controlled type 2 diabetes. Participants receive regular phone coaching from a nurse along with standard diabetes education. The goal is to see if this extra support improves blood su…

Researchers test whether Sanggua tablets, a traditional Chinese medicine made from mulberry leaf, bitter melon, kudzu root, Chinese yam, and cassia seed, can lower blood sugar in adults with impaired glucose tolerance or mild type 2 diabetes. The trial enrolls 84 people aged 18 t…

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

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

This Phase 3 trial tests whether a new combination pill containing laroprovstat and rosuvastatin lowers LDL cholesterol better than rosuvastatin alone. About 200 adults with high cholesterol and a history of heart disease or high risk will take either the combo or a placebo plus …

This study looks at whether eating one medium potato daily as part of a healthy Mediterranean diet affects blood sugar and heart disease risk in adults with prediabetes. About 74 people aged 25-65 with prediabetes will follow either a potato-inclusive or potato-free Mediterranean…

This study tests whether semaglutide (Ozempic), a drug used for type 2 diabetes, works well for people with spinal cord injury (SCI) who also have diabetes. SCI patients are at higher risk for diabetes, but it's unclear if standard treatments are effective. The trial will measure…

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

This study tests a new gene-editing treatment called BEAM-302 for adults with Alpha-1 antitrypsin deficiency, a genetic condition that can cause lung and liver disease. The therapy is given as an infusion and aims to correct the faulty gene. The trial will enroll 106 people to ch…

Researchers are testing a procedure called Stem Cell Educator therapy in people with type 1 diabetes. The treatment collects a patient's immune cells, briefly exposes them to cord blood stem cells, and returns them to the body. The goal is to calm the immune attack on insulin-mak…

This study tests a new drug called ABBV-383 in 38 people with Waldenström macroglobulinemia that has come back or not responded to treatment. The drug is a monoclonal antibody designed to block cancer growth. The trial has two parts: first finding a safe dose, then checking how w…

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

This study is testing a special soft cast for people with diabetic foot ulcers. The cast is a multi-layer wrap that cushions the foot and leg to help the wound heal. Researchers want to see if it works well, is comfortable, and safe. About 100 adults with diabetic foot ulcers wil…

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 tests whether using a mobile health app helps people with type 2 diabetes and obesity lose weight and control blood sugar better than standard programs. Thirty adults will track their weight daily and blood sugar before and after meals for six months. The goal is to se…

This study tests whether mobile health apps like Weight Watchers and Healthi can help low-income Hispanic adults with type 2 diabetes lose weight and improve blood sugar control. Forty-five participants will use one of the apps or a standard program for six months, tracking their…

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

This phase II trial at Mayo Clinic is testing whether longer maintenance therapy with daratumumab can improve survival for people with AL amyloidosis. About 96 adults who have already responded to initial daratumumab treatment will be randomly assigned to shorter or longer mainte…

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…

Researchers test whether adding pancreatin, a digestive enzyme, to standard blood sugar-lowering therapy helps adults with type 2 diabetes. The trial enrolls 100 people aged 35 to 75 whose diabetes is not fully controlled on oral drugs or insulin. Participants take pancreatin or …

This study is testing an education program for people with type 2 diabetes who also have chronic kidney disease. The program provides patients with information about their kidney health and suggests medications that could slow kidney damage. Researchers will see if this approach …

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

This study tests an experimental drug called mibavademab in 34 women with Functional Hypothalamic Amenorrhea (FHA), a condition where periods stop due to stress or intense exercise. The goal is to see if the drug can help the body produce the hormones needed for ovulation and imp…

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

This pilot study tests whether a daily probiotic supplement (Lactobacillus plantarum 299v) can reduce retinal swelling and improve vision in people with diabetic macular edema. Thirty-six participants will take the probiotic twice daily for four months. Researchers will measure c…

Researchers are testing how safely and comfortably people with type 2 diabetes can switch from semaglutide to CagriSema, a weekly injection that combines cagrilintide and semaglutide. About 210 adults who have taken a stable dose of branded semaglutide for at least three months w…

This study tests a traditional Chinese medicine called Jiedu Huayu oral prescription in 436 people who have had a brain bleed due to a condition called cerebral amyloid angiopathy. The goal is to see if the herbal treatment can lower the chance of having another stroke or blood v…

This phase 2 study is testing a time-limited combination of three drugs—pirtobrutinib, venetoclax, and rituximab—in 40 patients newly diagnosed with Waldenström's macroglobulinemia, a rare blood cancer. The goal is to see if this regimen can achieve a very good partial response o…

Researchers are observing how well a combination of three classes of oral diabetes drugs works in Korean adults with type 2 diabetes. The study follows people who already take metformin plus an SGLT2 inhibitor, a DPP-4 inhibitor, or a thiazolidinedione. It tracks safety through s…

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 looks at whether people with type 1 diabetes can safely start using an advanced hybrid closed-loop insulin pump system at home instead of in the hospital. Researchers will track blood sugar levels, quality of life, and costs over 12 months in 80 adults. Participants re…

Researchers are testing which combination of four family-focused components works best alongside a standard six-month behavioral weight loss program for Black adults. The components include communication training, cohesion-building content, the number of sessions, and whether ses…

This study tests a weekly injection of semaglutide (a GLP-1 drug) in 76 adults with 'double diabetes' — a mix of type 1 diabetes and inherited insulin resistance. The goal is to see if the drug improves blood sugar control and helps with weight loss. Participants must be over 18,…

This study tests whether wearing a continuous glucose monitor (CGM) for 3 months helps adults with prediabetes better control their blood sugar and stick to lifestyle changes compared to standard finger-prick testing. About 80 participants will be randomly assigned to use a CGM o…

Researchers at St. Louis University are testing whether short stair-climbing breaks throughout the day improve blood sugar and heart health in adults with prediabetes. Participants complete three separate visits in random order: one with uninterrupted sitting, one with 3-minute l…

This study tests a new medicine called PRX-115, given through a vein, to see if it can lower uric acid levels in adults with gout who haven't improved with standard pills. About 150 participants will receive either PRX-115 (with or without methotrexate) or a placebo over 24 weeks…

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 early-stage study tests a new drug called PATAS in healthy volunteers to see if it is safe and how the body processes it. The study involves 56 people aged 18 to 55 who are in good health. The goal is to find the right dose and check for side effects before testing in people…

This study tests a drug called ACP-204 for treating psychosis (hallucinations or delusions) in people with Lewy body dementia. It is a 52-week open-label extension, meaning all 126 participants will receive the drug. The main goal is to check for side effects over the long term. …

Researchers at Ankara Medipol University are testing an online, occupational therapy-led lifestyle program for adults with type 2 diabetes. The program runs over eight Zoom sessions and covers diabetes awareness, problem-solving, stress management, physical activity, and goal set…

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

This early-phase study tests an experimental drug called JNJ-79635322, a trispecific antibody designed to attack cancer cells in people with relapsed or refractory multiple myeloma or AL amyloidosis. About 180 participants will receive the drug to find a safe dose and check for s…

This study compares a continuous glucose monitor (CGM) to standard fingerstick testing in 270 adults with type 1 or type 2 diabetes who use insulin injections. Participants wear the Accu-Chek SmartGuide sensor to track blood sugar levels continuously. The goal is to see if the CG…

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 study tests whether people with severe diabetic macular oedema (swelling in the center of the retina) can switch from regular eye injections to a gentle laser once the swelling drops below a certain level. 264 adults with type 1 or type 2 diabetes will be randomly assigned t…

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

This early-stage trial is testing a new drug called AG-181 in 20 adults with phenylketonuria (PKU), a genetic condition that makes it hard to break down an amino acid called phenylalanine. The main goal is to check if the drug is safe and tolerable. Researchers will also measure …

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

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

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

This study tests an experimental drug called ARO-INHBE in 180 adults with obesity, some of whom also have type 2 diabetes. The drug is given as a shot under the skin, either alone or combined with the existing weight-loss drug tirzepatide. The main goal is to check safety and how…

This study tests whether the drug metyrapone can safely lower cortisol levels in people with mild autonomous cortisol secretion (MACS), a condition where the adrenal glands produce too much cortisol. Participants will receive either metyrapone or a placebo for a short period, and…

This pilot study tests a personalized lifestyle program called PRISM for adults with metabolic syndrome and insulin resistance. Participants follow a time-restricted eating schedule (eating all meals within an 8-hour window) and are later assigned extra lifestyle changes like phy…

This study aims to see if a hybrid closed-loop insulin pump (a smart insulin pump) can help adults with poorly controlled type 1 diabetes (A1c >8.5%) from diverse backgrounds. The study will enroll 40 participants from Los Angeles who do not currently use an insulin pump. The goa…

This early-stage trial is testing a new drug called JNJ-79635322 in combination with other myeloma treatments (daratumumab, lenalidomide, or pomalidomide) in 140 people with multiple myeloma. The main goal is to find a safe and effective dose and to see how well the body tolerate…

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 early-stage trial is testing a single-dose gene therapy called AMT-191 in 12 adult men with classic Fabry disease. The therapy uses a harmless virus to deliver a working copy of the GLA gene to the liver, so the body can produce the missing enzyme. The main goals are to chec…

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

This study will test whether a 4-month digital coaching program (Noom) helps adults who recently started GLP-1 medications improve their diet and physical activity. About 220 participants will use the app to track food and exercise, get coaching, and learn about managing challeng…

This phase 2 trial tests whether taking taurine, a natural amino acid found in energy drinks and supplements, can improve blood sugar, cholesterol, and inflammation in people with type 2 diabetes. About 94 adults with diabetes will take either taurine powder or a placebo twice da…

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 a new drug called R3R01 can reduce the amount of protein leaking into the urine in adults with type 2 diabetes and kidney disease. About 60 participants will take either R3R01 or a placebo for 3 months. Researchers will measure urine protein, kidney funct…

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

This study tests 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 an experimental drug called NNC6019-0001 in people with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart. About 1,280 adults will receive either the drug or a placebo by IV, alongside their usual heart trea…

This pilot study at Johns Hopkins University will enroll 40 Black women who recently had gestational diabetes and are overweight. Half will receive prepared low-carb, low-fat meals plus nutrition counseling, lactation support, and culturally tailored food bundles; the other half …

Researchers at National Taiwan University Hospital are following 40 people with Pompe disease who switched from one enzyme replacement therapy to avalglucosidase alfa. The study tracks urine and blood biomarkers, motor function, breathing, and safety over the short and long term.…

Researchers are testing whether a high-pressure cutting balloon can better prepare hardened coronary arteries before a stent is placed. The trial enrolls 280 adults with moderate-to-severe calcium buildup in their heart arteries who are scheduled for an imaging-guided drug-elutin…

This trial tests a one-time gene therapy called UX111 for children with Sanfilippo A, a rare genetic disorder that causes severe brain damage. The therapy delivers a working copy of the missing gene to cells. Researchers will measure whether it reduces harmful substances in the b…

Researchers are testing whether supervised exercise can improve fitness and slow artery disease in people with familial hypercholesterolemia, a genetic condition that causes very high cholesterol from birth. The trial compares two exercise programs: high-intensity interval traini…

This trial is testing whether an investigational drug called AZD6234, given as a weekly injection, can help adults with type 2 diabetes and excess weight lose more weight than a placebo. Participants will receive either AZD6234 or a placebo for 68 weeks, and researchers will meas…

Researchers are testing whether a low-calorie diet can improve metabolic syndrome in adolescents who also have familial Mediterranean fever. About 40 teens with both conditions will follow the diet for three months. The team will measure fasting blood sugar, body mass index, wais…

This early study tests a device that uses radiofrequency energy to create vapor and ablate (remove) a small part of the upper intestine. The idea is that this might improve how the body handles sugar. The study will enroll 20 adults with type 2 diabetes to see if the procedure is…

Researchers at Cairo University are testing whether a three-month walking program can improve metabolic syndrome and fatty liver in adolescent girls with celiac disease. Forty participants will walk on a treadmill for 40 minutes, three times a week. The study measures liver enzym…

Researchers compare low-dose rivaroxaban plus aspirin with aspirin alone in adults aged 40 to 70 who have chronic limb-threatening ischemia along with diabetes, hypertension, and high cholesterol. The trial tests whether the combination reduces major limb events like amputation a…

This study tests a new drug called EIK1005, alone or with another drug (pembrolizumab), in people with advanced solid tumors that have not responded to other treatments. The goal is to find a safe dose and see if the drug shrinks tumors. About 160 adults with certain cancer types…

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 early-stage trial tests a new drug called JR-446 in 12 children under 6 with MPS IIIB, a rare genetic disorder that damages the brain and body. The main goal is to see if the drug is safe and tolerable. Researchers will also measure changes in certain substances in the body …

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 is testing a new hip replacement system called the HIT Reverse Hip Replacement System. It is for people aged 50 to 75 who need a total hip replacement due to arthritis, joint damage, or other conditions. The goal is to see if the new system is as safe and effective as …

This study compares two drugs, sodium bicarbonate and THAM, for treating metabolic acidosis in patients after heart surgery. About 250 adults who develop acidosis after using a heart-lung machine will be randomly assigned to receive one of the two drugs. The main goal is to see i…

This study will test a new experimental automated insulin delivery system (called NMX8) in 116 people with type 1 or type 2 diabetes who need insulin. The goal is to see if the new system is safe and works as well as the current Medtronic 780G system at keeping blood sugar in a h…

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

This 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…

Researchers want to know whether some patients diagnosed with acquired aplastic anemia actually have an inherited bone marrow failure syndrome that was missed. The study enrolls children and adults with these conditions and collects clinical, laboratory, immune, and genetic data.…

AADC deficiency is a rare genetic disorder that disrupts brain chemicals and can cause severe neurological and developmental problems. Researchers in Liguria, Italy, are testing a newborn screening program that measures a marker called 3-O-methyldopa in dried blood spots from abo…

Researchers are comparing brain scans, sleep studies, genetic tests, and cognitive exams in people with Alzheimer's disease, Lewy body dementia, and Parkinson's disease. The goal is to find patterns that help doctors tell these conditions apart more accurately. The study includes…

Researchers are testing a blood-based screening approach to detect acid sphingomyelinase deficiency (ASMD), a rare genetic condition, in people who have interstitial lung disease (ILD) and other risk signs such as an enlarged spleen, low platelet count, or low HDL cholesterol. Th…

Researchers in Dijon, France, are comparing how the brain processes taste in three groups: healthy volunteers, people with early idiopathic Parkinson's disease, and people with early Lewy body disease. Participants undergo a taste-evoked potential test, which measures brain waves…

Researchers at the University of Bari are screening children aged 1 to 18 who have biopsy-confirmed celiac disease for four blood autoantibodies linked to type 1 diabetes. The study aims to enroll 600 children and measure how often these markers appear in this group. By identifyi…

This study is testing a small device called a nanosensor that measures oxygen levels in muscle tissue. The goal is to see if it can accurately assess mitochondrial function in people with mitochondrial myopathy compared to healthy volunteers. If it works, this sensor could become…

This pilot study is testing whether a rapid, point-of-care blood test can help identify early signs of infection and sepsis in preterm infants. Researchers will measure inflammatory markers like IL-6, PCT, CRP, and MxA in blood samples from about 40 to 80 premature babies, compar…

This completed early-phase study tested a new radioactive tracer called 18F-BL40 for PET/CT scans in 10 adults with certain blood cancers (like lymphoma and leukemia). The goal was to see if it safely and accurately detects tumors by targeting a receptor common in many cancers. R…

This study checks if a cheap thermal camera (about 200 euros) works as well as a professional one for spotting early signs of foot problems in people with diabetes. 130 adults with diabetes will have both cameras take pictures of the bottom of their feet. The goal is to see if th…

This study looks at whether doing a diabetes test right after giving birth (instead of waiting 4-12 weeks) helps more women get screened for type 2 diabetes after having gestational diabetes. It also asks women about their views on heart-health programs after pregnancy. About 104…

Researchers are testing an AI-based software that works with a fundus camera to detect diabetic retinopathy in adults with diabetes. The trial enrolls about 800 people aged 22 and older. The goal is to see whether the automated system is safe and accurate at identifying the eye c…

This study is testing a new blood test called Avantect to see if it can find pancreatic cancer early in people who were diagnosed with type 2 diabetes in the last 6 months. About 15,000 adults aged 50-84 will give blood samples and fill out anxiety questionnaires over a year. The…

Researchers at NYU Langone Health are testing whether a small virtual reality headset can measure two vision problems: metamorphopsia, where straight lines look wavy, and aniseikonia, where each eye sees images at different sizes. The study includes adults with normal vision and …

This study tests whether using an AI-driven retinal camera during regular diabetes checkups can increase the number of young people screened for diabetic retinopathy, a leading cause of blindness. Researchers will enroll 500 youth with type 1, type 2, or cystic fibrosis-related d…

Researchers are testing whether a non-invasive ultrasound technique can accurately measure muscle mass in people with metabolic syndrome, type 2 diabetes, and obesity. The study enrolls 320 participants, including healthy controls, and compares ultrasound measurements against DXA…

Researchers are testing whether a lifestyle program that includes a romantic partner works better than the standard diabetes prevention program for adults at high risk of type 2 diabetes. The trial enrolls 324 adults with prediabetes or other risk factors, along with their partne…

This study tests two lifestyle programs in 400 older African American and non-Hispanic white adults at risk for dementia and diabetes. One group does aerobic exercise three times a week; the other learns to follow the MIND diet, which emphasizes berries, greens, and whole grains.…

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

Severe low blood sugar is a serious risk for people with diabetes, causing falls, heart problems, and hospital stays. Researchers at Kaiser Permanente developed a simple guide called 'Hypoglycemia on a Page' to help prevent it. In this study, they compare two groups of high-risk …

Older adults having lower limb orthopedic surgery fast before anesthesia so stomach contents cannot enter the lungs. Researchers at Prof. Ngoerah General Hospital in Denpasar, Indonesia, randomly assign 62 patients aged 60 and older to drink either a maltodextrin carbohydrate bev…

Researchers at Laval University are studying a mobile healthcare unit that travels to Indigenous and remote communities in Canada to offer cardiovascular risk screening. About 500 adults with a body mass index above 27 can take part. The team measures blood pressure, cholesterol,…

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 phase 3 trial tests whether orforglipron, a daily oral medication, can help overweight adults reduce their risk of developing obesity-related complications such as type 2 diabetes, high blood pressure, and high cholesterol, as well as progression to class 1 obesity. Particip…

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 two types of fish oil, EPA and DHA, can improve metabolic health in people at high risk for type 2 diabetes. Over 12 weeks, 84 participants will take daily capsules of EPA, DHA, or a placebo (corn oil) and undergo specialized tests to measure insulin func…

Researchers in Pakistan are testing whether giving school children trout fish, cow milk with egg, vitamin D supplements, or diet education can improve their vitamin D levels, nutrition, and thinking skills. The trial enrolls 304 children aged 6 to 12 in Aga Khan schools. Each chi…

Researchers in South Savo, Finland, invite residents turning 45, 50 or 55 to take a blood test that measures many small molecules at once. The test estimates each person's 10-year risk of type 2 diabetes, heart attack, stroke, chronic kidney disease and fatty liver disease. Peopl…

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 tests whether the drug ORC-13661 can prevent hearing loss in people receiving intravenous amikacin for a lung infection called NTM. About 105 adults will take either a high dose, low dose, or placebo daily for up to 90 days. Researchers will compare hearing changes amo…

This study tests a new way to screen for heart failure risk in people with type 2 diabetes. Doctors use a blood test (NT-proBNP) and a risk score to identify high-risk patients, then offer early prevention treatments. About 300 patients are taking part. The goal is to see if this…

This study is testing whether a special software that analyzes CT scans of the heart's arteries can better prevent heart attacks, strokes, and other cardiovascular events compared to usual care based on risk factors like cholesterol and blood pressure. The trial will enroll 7,500…

This study investigates whether drinking kefir, a fermented dairy drink rich in live bacteria and yeasts, from around 14 weeks of pregnancy until routine diabetes screening can improve blood sugar control and reduce the risk of gestational diabetes. Pregnant individuals are rando…

Researchers are testing whether a low-cost anti-inflammatory drug called colchicine can slow early damage to the arteries in adults with type 2 diabetes. Diabetes speeds up hardening of the arteries partly through ongoing low-grade inflammation. About 300 adults with type 2 diabe…

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

People with prediabetes have blood sugar levels that are higher than normal but not yet diabetic. Researchers at the University of Texas Health Science Center at San Antonio are testing four diabetes drugs, dapagliflozin, saxagliptin, pioglitazone, and metformin, to see whether a…

Researchers are testing whether a supplement combining alpha-lipoic acid, vitamins B1, B6, B12, and vitamin E improves nerve symptoms more than a vitamin B-complex alone in people with type 2 diabetes and diabetic symmetrical polyneuropathy. About 100 participants at one hospital…

This study tests whether butylphthalide, a drug used for stroke, can improve symptoms of Multiple System Atrophy (MSA), a rare and serious brain disorder. About 150 adults with MSA will take either the drug or a placebo three times a day for six months. Researchers will track sym…

This Phase 2 trial tests punarlimab, a new IV drug, against a standard steroid injection for quick relief of acute gouty arthritis pain. About 170 adults with frequent gout flares will receive a single dose and report their pain levels over 72 hours. The goal is to see if punarli…

This study tests a virtual health coaching program for adults with type 2 diabetes who also face mental health challenges like anxiety or diabetes distress. Participants receive weekly coaching calls, support from a care team, and online tools to help manage both conditions. The …

Researchers are testing whether a carbohydrate-rich clear drink given two hours before bariatric surgery helps patients with metabolic syndrome recover normal gastrointestinal function faster. About 208 adults scheduled for gastric bypass or sleeve gastrectomy will be randomly as…

This study tests whether the drug sonlicromanol can reduce fatigue and improve physical abilities like balance and leg strength in adults with a specific genetic form of mitochondrial disease. About 220 participants will take either the drug or a placebo twice daily for 52 weeks.…

This study compares two steroid injections for knee osteoarthritis in people with type 2 diabetes. It aims to see which one controls pain better while causing fewer blood sugar spikes. About 120 adults with diabetes and knee arthritis will receive either a long-acting or short-ac…

This study tests whether a program that combines a smartphone app with face-to-face therapy sessions can improve psychological well-being in children and adolescents with type 1 diabetes. The goal is to reduce diabetes-related distress and help young patients better manage their …

This study tests whether raising the heart rate with a pacemaker can improve heart failure symptoms and exercise ability in 10 people with cardiac amyloidosis who already have a pacemaker. Participants will get a personalized pacing protocol based on their height and heart functi…

Diabetic foot ulcers are a common and serious complication of diabetes that can lead to amputation. Researchers at the University of Lahore are testing whether adding custom polyethene foam insoles with a Poron top layer, and transcutaneous electrical nerve stimulation (TENS), to…

This study tests whether acupressure, a technique that applies gentle pressure to specific points on the body, can lower blood sugar and reduce anxiety in people aged 65 and older with diabetes. Forty-eight participants will receive either real acupressure or a sham version for 6…

Pasireotide, a treatment for acromegaly, often causes high blood sugar. Researchers are testing whether a very-low-carbohydrate ketogenic diet can manage this side effect better than standard diabetes medications. The trial enrolls 30 adults with acromegaly who develop high blood…

This study tests whether an activity book called 'Think-Find-Solve' can improve insulin self-management in children aged 8-12 with type 1 diabetes. Forty children and their parents will take part, with one group using the book for three months. The goal is to see if the book help…

This study tests whether a structured 5-week cardiac rehabilitation program can improve exercise ability and quality of life in 40 adults with cardiac amyloidosis. Participants will undergo supervised aerobic and strength training five days a week, with tests before and after the…

Researchers are studying a dietary supplement called tributyrin in people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement, balance, and eye control. The trial enrolls about 15 adults aged 45 and older who have PSP-Richardson syndrome, access…

This study tests the safety of a new drug called IGT-303 in healthy volunteers and people with chronic kidney disease (CKD). About 94 participants will receive either the drug or a placebo, given as a shot under the skin or into a vein. Researchers will monitor for side effects t…

This study tests whether cognitive behavioral therapy for insomnia (CBTi) can improve sleep, blood sugar levels, and quality of life in adults with type 1 diabetes. Fifteen participants will either receive 8 virtual CBTi sessions or health education emails and calls over 12 weeks…

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 whether Northera (droxidopa) is safe and can improve symptoms like dizziness, fainting, and low blood pressure in children aged 7 to 17 with Menkes disease. Six to ten participants will receive either the drug or a placebo in two four-week periods, with careful d…

This study tests whether a 16-week hydration program can reduce side effects like nausea and dehydration in people starting GLP-1 medications (such as Ozempic or Wegovy). Researchers will track 30 adults aged 18-60 who recently started the drug, measuring hydration levels and sid…

This study tests whether a gentle, non-invasive brain stimulation technique called tACS can improve memory and thinking in people with Alzheimer's disease, frontotemporal dementia, Lewy body dementia, and related conditions. Two hundred participants will receive either real or sh…

This study tests whether applying an ice pack or a warm gel pad to the drain site before removal can lower anxiety, improve comfort, and stabilize vital signs in patients recovering from open urological surgery. 120 adults will be randomly assigned to ice, warm gel, or standard c…

Researchers at Mayo Clinic are running a pilot study to see whether clearing a blocked pancreatic duct improves how the pancreas makes insulin in people with chronic pancreatitis. Participants are adults with minimally symptomatic chronic pancreatitis and a duct blockage. The stu…

Gout causes sudden, intense joint pain and swelling. Researchers are testing whether metformin, a common diabetes drug, can reduce gout symptoms. In this Phase 2 trial, 70 people with gout receive either metformin or a placebo for a period of time. Doctors measure changes in dise…

This study compares giving cancer treatment at home versus in a clinic for 27 adults with various cancers in the Florida Panhandle. The goal is to see if home treatment is preferred and improves satisfaction, while also checking safety. Participants will receive their usual cance…

This study tests whether eating all meals within an early 8-hour window (and cutting calories by 25%) can improve blood sugar and heart health in people who are overweight and have prediabetes, type 2 diabetes, or normal blood sugar. Thirty participants will try either early or l…

This early-stage study tests a new drug called LY4057996 in 124 healthy volunteers and people with type 1 or type 2 diabetes. The main goal is to see if the drug is safe and what side effects it may cause. Participants receive the drug as a shot under the skin or into a vein, and…

This study tests whether the experimental drug ACP-204 can reduce psychosis symptoms like hallucinations and delusions in people with Lewy body dementia. About 180 adults aged 55 to 85 will take either ACP-204 or a placebo daily for 6 weeks. The goal is to see if the drug safely …

Researchers at Vanderbilt University Medical Center are studying whether inflammation proteins in the eye drive diabetic macular edema, a swelling of the retina that can blur vision in people with type 2 diabetes. The trial enrolls about 20 adults with diabetic retinopathy and ma…

This early-phase study tests whether a weight-loss drug called tirzepatide can help overweight or obese men with intermediate-risk prostate cancer lose weight before their prostate removal surgery. The study will enroll 30 participants and focus on safety and how well the program…

This study looks at whether foot reflexology and body massage can help newborns with jaundice who are already receiving light therapy (phototherapy). Researchers will measure bilirubin levels, heart rate, breathing, and stress in 105 babies. The goal is to see if these gentle, dr…

Researchers are studying whether pioglitazone, a diabetes medication, can improve heart function in obese adults with type 2 diabetes and heart failure with preserved ejection fraction. The trial enrolls 78 participants aged 30 to 70 who take pioglitazone or a placebo for 24 week…

This study tests whether a safe, non-invasive brain stimulation technique called tACS can improve cognitive abilities in people with Lewy body dementia. Forty participants will receive either real or sham stimulation over two weeks, and their thinking, memory, and daily function …

This study tests whether colchicine, a drug used for gout, can lower inflammation and pain in people with CPPD disease. About 150 adults will take either colchicine or a placebo daily for 6 months. Researchers will measure blood markers and joint symptoms to see if the drug helps…

Researchers are testing whether a five-day program of drawing and reflective writing can reduce anxiety and improve mood in adolescents with type 1 diabetes. Fifty teens aged 9 to 18 who use an insulin pump take part, with half doing the art activities online and half receiving s…

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

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

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

This study 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 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 looks at whether a continuous glucose monitor (CGM) — a small sensor worn under the skin — is an acceptable and useful tool for checking diabetes risk in women who recently had gestational diabetes. Twenty women will wear the CGM for 10 days at 6-8 weeks after giving b…

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

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

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

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

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

This study 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 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…

Researchers in France are following adults and children with acid sphingomyelinase deficiency (ASMD), a rare inherited disorder also known as Niemann-Pick disease. The study aims to describe how symptoms, organ problems, and complications develop over time, and how the disease af…

This observational study follows people with Glycogen Storage Disease Type Ia who have already received the gene therapy DTX401. The goal is to track their health and safety for at least 10 years after treatment. No new drugs or treatments are given in this study. It will help re…

This study is a worldwide registry that collects health information from people with Gaucher disease over many years. It does not test any new treatments—instead, it observes patients as they receive their usual care. The goal is to better understand how the disease progresses, i…

This study tests whether a drink made from broccoli sprouts can improve how well endurance athletes perform and recover after cycling. Thirty healthy adults who exercise regularly will drink either the broccoli sprout drink or a placebo at different times before a cycling test. R…

This study will follow 500 adults with chronic kidney disease and type 2 diabetes who are taking finerenone, a drug that helps protect the kidneys and heart. Researchers want to see if a tool called the Klinrisk score, which estimates the risk of kidney disease getting worse, imp…

This study is a long-term registry that collects health information from people with Fabry disease, a rare genetic disorder. It does not test any new treatments; instead, it follows up to 9,000 patients worldwide to better understand how the disease progresses and how current tre…

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

This study follows people with peroxisome biogenesis disorders (PBD) to learn more about how the disease progresses. Researchers will collect medical records, test results, and images over time from up to 244 participants. No new treatments are being tested; the goal is to better…

This study aims to identify different types of familial hypercholesterolemia (FH) in infants and newborns. Expectant parents with FH will provide a cheek swab for genetic testing, and their babies will have 5 blood samples collected over 2 years. The goal is to improve early dete…

This observational study looks at factors that influence bicarbonate levels in people on maintenance hemodialysis for end-stage renal disease. Researchers will collect data on demographics, medical history, diet, and dialysis details, and measure bicarbonate levels before and aft…

This study aims to find out if people who get calcium oxalate kidney stones absorb more oxalate from food than healthy people. Researchers will compare stone formers and healthy volunteers by having them eat special diets and measuring oxalate in urine, blood, and stool. The goal…

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 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 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 follows 500 American Indian adults with early kidney disease to learn how their condition progresses and what factors affect heart health. Researchers will measure kidney function and track heart events like heart attacks and strokes. The goal is to fill knowledge gaps…

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

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

This study is building a large database of health information from 250 people with Parkinson's disease and related conditions. Researchers will collect data on symptoms, treatments, genetics, and brain scans over time. The goal is to better understand how the disease progresses a…

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 …

Researchers are following children with rhizomelic chondrodysplasia punctata (RCDP), a rare genetic condition that affects bone growth and development, to understand how it changes over time. About 75 children aged 6 months to 21 years will take part. They will visit the study te…

Researchers at Yale are studying how high blood sugar affects the brain in people with poorly controlled type 2 diabetes. The trial tests whether intensive diabetes management, including nutrition counseling and medication adjustments, can improve the brain's ability to transport…

Researchers at Miami University are studying how a person's fitness level and body fat affect their body's ability to control blood sugar and blood vessel function after a week of reduced physical activity. They will recruit 48 healthy adults aged 18 to 40, who normally walk more…

Mitochondrial leukoencephalopathies are rare inherited disorders that damage the brain's white matter by disrupting cellular energy production. Researchers at international reference centers are collecting past clinical, imaging, biochemical, and genetic records from about 100 pe…

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

This pilot study tests a program that provides education, motivation, and social support to adults with diabetes and early-stage chronic kidney disease (CKD). The goal is to see if the program is practical and helps patients stay motivated, learn about kidney disease, and use sup…

Researchers at the German Institute of Human Nutrition are studying why blood sugar responses to food differ so much from person to person. The LIME study follows 300 adults who are overweight or obese, including people with prediabetes, people with type 2 diabetes, and people wi…

This study looks at whether newer automated insulin delivery systems help people with type 1 diabetes have healthier eating behaviors. Researchers will survey over 100 youth and adults across Canada about their eating habits and blood sugar control. The goal is to understand if t…

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

This study creates a Canadian registry for children under 18 with Alagille syndrome or genetic intrahepatic cholestasis, rare liver conditions that cause bile buildup. Researchers will collect health data from medical records over time to understand how these diseases progress an…

Researchers at Nanyang Technological University are studying how pressure shifts across the bottom of the foot when people with type 2 diabetes walk after losing part of the forefoot, such as the fifth toe. The study enrolls 70 adults with a healed lateral forefoot amputation who…

This study explores whether resting metabolic rate (the calories your body burns at rest) runs in families. Researchers will measure metabolism and body composition in 30 biological parent-adult child pairs. The goal is to understand how much of our metabolism is inherited, which…

This study will test whether combining continuous glucose monitor (CGM) data with information from a smartwatch and a phone app (like meals and activity) can better predict blood sugar levels in people with type 2 diabetes. Thirty-six adults will wear a CGM and smartwatch and log…

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 aims to find out how many adults with type 2 diabetes experience severe diabetes-related distress—a feeling of being overwhelmed by the demands of managing the disease. Researchers at a French hospital will ask 246 participants to fill out questionnaires about their em…

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

This study looks at how semaglutide, a drug used for diabetes and weight loss, changes fat tissue around the kidneys, heart, and liver in people with type 2 diabetes and chronic kidney disease. Researchers will compare patients taking semaglutide to those not taking it. The goal …

This study looks at how insulin and glucagon work together to control blood sugar levels when people are not eating. Researchers will give insulin and a fat emulsion to 60 volunteers with normal or impaired fasting glucose to see how their bodies respond. The goal is to better un…

This study examines whether using advanced automated insulin pumps soon after diagnosis can protect brain growth and thinking skills in children aged 5-7 with type 1 diabetes. Researchers will compare brain scans and cognitive tests over 18 months between children using standard …

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

This study will follow 20,000 adults in rural China for three years to understand how chronic diseases like high blood pressure, diabetes, and frailty develop. Participants will provide health data and samples to build a comprehensive database. The goal is to create better diseas…

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…

Researchers are testing whether a continuous glucose monitor, a small sensor worn on the skin, can estimate how well the body responds to insulin. The study enrolls 270 adults aged 21 to 70 who do not have diabetes and have no history of heart attack, stroke, or heart failure. Pa…

This study uses advanced MRI and spectroscopy to track how semaglutide therapy changes muscle strength, volume, and quality over six months. Researchers will measure muscle function and fat infiltration in people with type 2 diabetes and obesity. The goal is to understand whether…

This study is tracking 850 adults with normal cognition or early memory problems, some of whom have prediabetes. Researchers will monitor memory, brain scans, and spinal fluid over time to see how blood sugar issues might affect Alzheimer's risk. The goal is to better understand …

This study looks at dental health and oral quality of life in people with Wilson's disease, comparing those with liver versus neurological symptoms to healthy controls. Researchers will collect saliva samples and use questionnaires and dental exams to see if copper levels in sali…

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

This study is collecting information from children and adults with pyruvate dehydrogenase complex deficiency (PDCD), a rare genetic disorder that affects energy production in cells. Researchers will review medical records, ask participants about their health history, and perform …

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

This study tests a digital tool called CGM ASSIST that combines a continuous glucose monitor with easy-to-read displays. The goal is to help people with Alzheimer's or dementia and diabetes, along with their caregivers, better understand blood sugar levels and make informed decis…

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…

Researchers at Texas A&M are testing whether a wearable device can measure insulin levels from sweat and skin fluid instead of blood. 32 healthy adults aged 18-30 or 50-90 will eat a standard meal and have their insulin tracked over time. The goal is to see if this noninvasive me…

Hypoparathyroidism is a rare condition where the parathyroid glands make too little parathyroid hormone, leading to low calcium and high phosphate in the blood. Symptoms can include tingling, muscle cramps, and weakness, and most cases follow neck surgery such as thyroid removal.…

This study looks at how a heart hormone called atrial natriuretic peptide (ANP) affects metabolism and heart health. Researchers will test 200 adults with different genetic versions of ANP to see how their bodies respond to exercise and sugar. The goal is to understand why some p…

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

This study compares two ways of inviting people to screen for type 1 diabetes at home: a simple email or letter versus a personal phone call. Researchers want to see which method leads more people to complete and return a home screening kit. Participants are adults and children a…

This study looks at how whole milk and skim milk affect blood sugar and cholesterol in 100 adults who drink at least one cup of dairy milk daily. Participants will switch between drinking only whole milk and only skim milk for set periods, while researchers track changes in their…

This early-phase study looks at how alpha cells in the pancreas communicate with beta cells to control blood sugar. Researchers will give healthy volunteers exendin-9 (a GLP-1 blocker) and dexamethasone to see how insulin secretion changes. The goal is to better understand diabet…

Researchers compare lumbopelvic stability in pregnant women who have gestational diabetes with those who do not. The study includes 170 women in their second trimester, all pregnant with one baby for the first time. Participants complete pain ratings and two physical tests: the a…

This study looks at whether sending home test kits or ordering standard lab tests can help more people with type 2 diabetes get screened for chronic kidney disease (CKD). About 750 adults who are missing key kidney function tests will be enrolled. The goal is to see if these appr…

Researchers are studying the nutritional status, body composition, and dietary intake of Filipino children with inborn errors of intermediary protein metabolism, a group of rare genetic conditions. The study enrolls 176 children from metabolic clinics across the Philippines. By m…

This study looks at how often kidney problems occur in people with Wilson's disease, a rare genetic condition that causes copper buildup. Researchers will check 150 patients aged 7 and older for signs of kidney damage, including chronic kidney disease, tubule issues, and calcium …

This study looks at copper levels in breast milk from women with Wilson's disease who are on treatment. Wilson's disease causes copper buildup, and treatment can lower copper, which might affect breast milk. The goal is to find out if breastfeeding is safe for their babies. About…

This study looks at whether Wilson's disease, a condition where copper builds up in the body, also damages the heart. Researchers will monitor 150 adults with Wilson's disease using heart scans, blood tests, and long-term heart rhythm recorders. The goal is to find out how often …

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

Researchers at NYU Langone Health are studying how the brain produces visual hallucinations in people with Lewy body diseases, including Parkinson's disease and Lewy body dementias. The study enrolls up to 234 adults, both patients and healthy volunteers, who complete behavioral …

This study aims to learn how the binding of RNA with DNA (called R-loops) is linked to amyotrophic lateral sclerosis type 4 (ALS4) and other inherited neurological disorders. Researchers will observe up to 330 people aged 5 and older, including those with ALS4, related conditions…

This study is testing whether people with diabetes can collect their own blood at home (using a finger prick or a device on the upper arm) to check blood sugar, cholesterol, and thyroid levels. The results will be compared to standard blood draws done by a nurse in the clinic. Th…

This study explores whether taking a dietary fibre supplement during a 10-day fast can improve gut health and metabolism more than fasting alone. Overweight adults (BMI 25 or higher) will fast at a clinic and then either receive a resistant starch fibre or a placebo. Researchers …

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

This study will follow 5,500 first-time pregnant women to see if blood tests and eye scans can predict complications like preeclampsia, fetal growth restriction, and gestational diabetes. Researchers will collect samples and images at different points during pregnancy and compare…

Researchers at Mayo Clinic are studying whether daily text-message check-ins can track symptoms and quality of life in adults starting treatment for multiple myeloma or AL amyloidosis. About 150 participants will receive short daily prompts about how they feel, and the study will…

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 tests whether a computer alert in the electronic health record can help doctors identify patients with familial hypercholesterolemia, a genetic condition that causes very high cholesterol. About 450 adults at Brigham and Women's Hospital will be included. The alert cal…

This study is observing 55 children and adults in Russia who have hypophosphatasia (HPP), a rare genetic bone disease. Researchers will track symptoms, lab results, and quality of life to learn how the disease naturally progresses. No treatments are being tested—the goal is to ga…

This study looks at how things in your environment—like pollution, chemicals, diet, and sleep—might affect your hormones and reproductive health. Researchers will follow 300 people aged 8 and older who have conditions like low or high hormone levels, early or late puberty, or inf…

This study looks at how a cholesterol-regulating protein called LXR might protect the eyes in people with diabetic retinopathy. Researchers will take blood samples from 104 adults with or without diabetes to test how certain blood cells function. The goal is to understand the bio…

Researchers at Mayo Clinic are studying 600 Somali-American adults to understand how common obstructive sleep apnea is and how it affects heart health. Participants will have sleep tests, blood pressure monitoring, and glucose testing. The goal is to learn about unique risk facto…

This study is creating a biobank and data registry for people with amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will collect blood, urine, tissue samples, and medical information from 505 participants over time. The goal is to store these ma…

Researchers are observing adults with obesity or type 2 diabetes who start taking a GLP-1 receptor agonist medication as part of their regular care. The study uses DXA scans, strength tests, and walking speed checks to measure how muscle and fat change over about a year. About 16…

Researchers at Aristotle University of Thessaloniki are asking physicians in Greece how often they follow American Diabetes Association guidelines when treating patients with type 2 diabetes and obesity. About 100 doctors who care for these patients will complete an 18-question s…

This study is testing whether artificial intelligence can predict if someone will develop one of five common diseases: diabetes, heart disease, breast cancer, back pain, or arthritis. Researchers will collect health data from 1,000 adults aged 30 to 60, including medical records,…

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…

Researchers in China are studying how environmental factors may contribute to type 1 diabetes. The study enrolls people recently diagnosed with type 1 diabetes, their first-degree relatives, and healthy controls. Over one year, researchers measure insulin-producing capacity and d…

This phase 1 trial asks whether a single dose of tirzepatide changes how quickly the stomach empties in adults with type 1 diabetes who are overweight or have obesity. Researchers will give participants either tirzepatide or a placebo injection, then measure stomach emptying usin…

This study will follow about 1,850 people with ATTR amyloidosis, a rare disease that affects the heart and nerves. Researchers will collect information on patients' health, treatments, and outcomes over time to better understand the disease and how current therapies work in every…

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

Researchers at UT Southwestern are studying people with dystonia and healthy volunteers to understand how known gene mutations affect brain structure and function. Participants complete clinical assessments, give family and medical history, undergo MRI scans, and provide blood fo…

Researchers want to know whether a liver protein called TMPRSS6 relates to blood sugar control, lipid levels, body fat, and liver health in people with leptin receptor deficiency or genetic lipodystrophy. These rare conditions cause severe obesity, extreme hunger, or loss of body…

This study aims to see if short videos featuring Latinx patients and doctors can encourage more Latinx people with diabetes to get regular eye exams. Researchers will survey about 1,500 people and hold small group discussions to measure changes in eye health knowledge and trust i…

This study reviews 10 years of medical records from 600 adults who had thyroid surgery at Minia University Hospital. The goal is to find out which patients are most likely to develop low calcium levels after surgery, a common complication. By identifying key risk factors, doctors…

Researchers are observing adults having elective upper-extremity surgery to see how often sudden, severe rebound pain occurs after an ultrasound-guided brachial plexus block. The study compares patients with type 2 diabetes to those without diabetes. Participants receive the stan…

ALS is a rapidly progressive nerve disease with no cure, and extending breathing support into later stages raises hard questions about quality of life. Researchers in Norway are following 200 ALS patients, their partners and their children with questionnaires before and after lon…