Metabolic disease

This study tests a new treatment called AZD0120 for people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. The therapy uses a patient's own immune cells, modified to target and destroy harmful cells. The study aims to see if it is safe and effectiv…

This study is testing whether limiting all food intake to a 10-hour window each day can help people with type 2 diabetes better manage their blood sugar. About 102 adults will follow this eating pattern for one year, tracking their first and last meals by text. Researchers will c…

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

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

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

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

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

This 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 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 early-phase study tests a new drug called NX-5948 in adults with advanced B-cell blood cancers (like certain leukemias and lymphomas) that have not responded to other treatments. The drug works by breaking down a protein (BTK) that helps these cancer cells grow. The main goa…

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

This 12-month pilot study tests whether a nurse-led smartphone app can help Medicaid patients with insulin-treated type 2 diabetes improve their blood sugar control. The program combines educational messages, phone-based problem-solving with a nurse, and remote monitoring via a d…

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

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

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

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

This study looks at whether eating avocados regularly can improve how the body handles fats and sugar in people with prediabetes and insulin resistance. About 82 adults aged 40-70 with high triglycerides and insulin resistance will eat avocados as part of their daily diet. Resear…

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

This 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 will follow 2,200 children and adults with type 1 diabetes who are using the Omnipod 5 system, a tubeless insulin pump that automatically adjusts insulin based on continuous glucose monitor readings. Over 12 months, researchers will track how often severe low or high b…

This Phase 3 trial will test a new once-daily insulin, GZR33, against the standard insulin degludec (Tresiba) in 350 adults with type 2 diabetes whose blood sugar is not well controlled by oral medications. The main goal is to see how well GZR33 lowers blood sugar over 26 weeks c…

This study offers a free 3-month diabetes education program at a local faith-based organization for adults with diabetes or prediabetes in Jefferson County, Alabama. Participants attend group sessions on diet, exercise, and coping, and are paired with a community health worker fo…

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

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

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

This study tests whether a weekly dose of alirocumab (Praluent) is safe and effective for adults with high cholesterol. Alirocumab is already approved for use every two weeks, but this trial explores if giving it weekly works better. About 420 participants will receive different …

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 program provides early access to the experimental drug venglustat for children and adults with Type 3 Gaucher disease who have brain-related symptoms. Participants must be at least 12 years old, weigh at least 15 kg, and be stable on standard enzyme replacement therapy. The …

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

This study tests whether 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…

This early-stage trial tests a single dose of TSHA-105, a gene therapy injected into the spinal fluid, in 8 people aged 2 to 20 with SLC13A5 citrate transporter disorder. The goal is to see if it is safe and whether it can improve motor and thinking skills. Because the trial is v…

This study tests a program for adults with type 2 diabetes who are leaving the hospital. Participants will work with a patient navigator to address unmet social needs like food, housing, or transportation. The goal is to see if this support reduces emergency visits and readmissio…

This study will give 800 pregnant women biweekly food kits with fruits, vegetables, and other healthy foods to see if it improves their diet and pregnancy outcomes. Participants will be followed from early pregnancy through birth. The goal is to learn whether providing healthy fo…

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

This study 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…

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

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

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

This study 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…

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

This study tests a new smartphone-based system that automatically delivers insulin for people with type 1 diabetes. Six adults who already use insulin pumps will try the system for a short time under close supervision. The goal is to see if the technology works safely and reliabl…

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

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

This study tests whether giving people access to healthy foods, personalized dietitian coaching, and a home blood pressure monitor can help Black and Hispanic adults with high blood pressure (and often diabetes) get their numbers down. About 100 participants will be randomly assi…

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

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

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

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

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

This study follows 630 people with type 1 diabetes in Japan who are already prescribed Awiqli, a once-weekly injection. Researchers will track side effects and blood sugar control over one year to see how the drug works in everyday life. The goal is to manage diabetes, not cure i…

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

This early-stage trial tests a new drug called MTX-439, an antibody that blocks a protein linked to kidney scarring. It will be given to healthy volunteers and people with diabetic kidney disease to check for safety and how the body processes it. The study is small (88 participan…

This study is testing the long-term safety and effectiveness of a daily pill called atumelnant for people with classic congenital adrenal hyperplasia (CAH). The drug works by blocking a hormone receptor to help control excess androgen production. Researchers will monitor 150 part…

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

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

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

This study tests a new care model for 80 young adults with type 1 diabetes who are moving from pediatric to adult healthcare. The program includes a nurse educator, psychologist, and transition navigator working together to provide support over six months. The goal is to see if t…

This study tests whether a daily probiotic called AKM Lab-01 can safely lower cholesterol levels in people with high cholesterol. About 60 adults aged 40-60 will take either the probiotic or a placebo for a period, and researchers will compare changes in their blood cholesterol a…

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

This early-stage study tests a new medicine called PYC-001, given as an injection into the eye, for people with a rare genetic eye disease (autosomal dominant optic atrophy) caused by a change in the OPA1 gene. The main goal is to check the safety of different doses and schedules…

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

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

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

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

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

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

This study is testing whether stem cells taken from a person's own bone marrow and then given back through the veins and nose can improve brain and nerve function. It includes 500 people with various conditions like stroke, Parkinson's, Alzheimer's, and nerve damage. The goal is …

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

This study tests 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 is monitoring the safety and effectiveness of the drug iptacopan (Fabhalta) in 21 Korean adults with paroxysmal nocturnal hemoglobinuria (PNH) or C3 glomerulopathy (C3G). Researchers will track side effects and lab results from routine medical records. The goal is to s…

This study tests whether providing money specifically for healthy foods helps pregnant women avoid gaining too much weight. About 1,440 women will either get standard nutrition advice or that plus up to $3,000 for healthy groceries. Researchers will compare weight gain between th…

This study tests whether taking three cholesterol-lowering drugs together works better than taking two in people with type 2 diabetes and high LDL cholesterol. 126 adults aged 35-60 will be split into three groups: one gets rosuvastatin plus ezetimibe, another gets bempedoic acid…

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 gene therapy given by IV for ADA-SCID, a rare and life-threatening immune disorder. The treatment uses a modified virus to deliver a working gene, aiming to restore the immune system. Ten participants will be monitored for safety and immune recovery over one ye…

This study is for adults with recurrent glioblastoma, an aggressive brain cancer. It tests whether adding a precise type of radiation (stereotactic radiosurgery guided by a special PET scan) to a device that delivers electric fields to the brain (TTFields) helps people live longe…

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

This study tests 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 Phase 2 trial tests a new drug called HDY015, which combines two medicines (QCZ484 and inclisiran) into a single injection. The goal is to see if it can safely lower both blood pressure and cholesterol in people who have both conditions. About 120 adults aged 18-75 with hype…

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

This phase 2 trial tests Kylo-11, an experimental drug, in 204 adults with heart disease and high levels of lipoprotein(a) – a genetic risk factor for heart attacks and strokes. Participants receive either Kylo-11 or a placebo injection for about a year. The main goal is to see i…

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 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 study tests whether a 12-week low-calorie meal replacement diet can improve heart function and exercise ability in people with heart failure with preserved ejection fraction (HFpEF) who are also obese. 63 adults will be randomly assigned to either the diet or standard care. …

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 phase 3 trial tests an experimental drug called sepofarsen in 32 people with Leber congenital amaurosis (LCA), a rare genetic condition that causes severe vision loss from birth. The drug is injected into one eye, while the other eye gets a placebo, to see if it safely impro…

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

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

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

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

This study looks at whether a second islet transplant can safely improve blood sugar control in adults with type 1 diabetes whose first transplant stopped working. Researchers will follow 20 patients for up to 5 years, checking graft function, insulin use, and severe low blood su…

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

This study follows 230 children with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC) who are taking Livmarli, a drug to reduce bile buildup and itching. Researchers will monitor side effects, liver function, and long-term outcomes like need for transplan…

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

This study tests a 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 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…

This study tests a device that uses radiofrequency energy to disrupt nerve signals in the kidney and liver arteries, aiming to lower blood pressure in people with uncontrolled hypertension. About 175 adults aged 18-80 with high blood pressure despite medication will be enrolled. …

This study compares two advanced heart procedures—stenting guided by imaging and blood flow tests (PCI) versus bypass surgery (CABG)—in 1,500 people with diabetes and three blocked coronary arteries. The goal is to see which approach better prevents major events like death, heart…

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

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

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

This study tests a new medicine called LY3938577 against a standard insulin (degludec) in 100 adults with type 2 diabetes who already use basal insulin. The goal is to see if the new drug helps keep blood sugar in a healthy range more safely and effectively over about 26 weeks. P…

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

This Phase 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 whether applying heat pads to the legs for 90 minutes a day can improve blood sugar control, muscle health, and physical function in older adults with prediabetes. Researchers will compare the effects to high-intensity interval training. The goal is to find a sim…

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

This early-stage trial tests a gene therapy for propionic acidemia, a rare genetic disorder that causes dangerous metabolic crises and organ damage. The study will enroll 9 children aged 6 months to 2 years with confirmed PCCA gene mutations. The therapy uses a harmless virus to …

This study compares two ways of tracking blood sugar in people with type 2 diabetes who use insulin after leaving the hospital. One group uses a continuous glucose monitor (CGM) that shows real-time levels and trends, while the other group uses traditional fingerstick tests. The …

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 an experimental oral drug called S-606001 in 45 adults with late-onset Pompe disease. Participants take the drug or a placebo on top of their standard enzyme replacement therapy. The main goal is to see if the drug improves lung function and walking ability over …

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

This study is testing a mobile app called Adhera Caring Digital Program for Obesity (ACDP-O) in 280 families. The app provides personalized education, mental health monitoring, and wearable device integration to support weight management and well-being in adolescents and young ad…

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-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 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 early-stage trial is testing the safety of a dietary supplement called NMN in 8 people with a rare genetic disorder known as DHDDS-CDG, which affects movement and development. Participants will take 250 mg of NMN daily for 6 months, with an optional 12-month follow-up. The m…

This study tests whether wearing a continuous glucose monitor (CGM) helps pregnant women with gestational diabetes control their blood sugar and improve outcomes for both mother and baby. Researchers will also examine how socioeconomic status, diet, and culture affect results. Th…

This study compares two different strength training schedules—non-linear and linear periodization—in people with type 2 diabetes. Over 8 weeks, 48 participants will do supervised workouts three times a week or receive usual care. Researchers will measure changes in exercise capac…

This study tests whether a 20-day fasting schedule before hip replacement surgery can reduce complications like infections. 130 adults will either follow the fasting plan or receive standard care. Researchers will measure changes in gut bacteria and immune markers to see if fasti…

This study tests a device called ReCET that delivers gentle electric pulses to the first part of the small intestine. The goal is to trigger cell regeneration and improve blood sugar control in people with type 2 diabetes who are not on insulin. Participants are randomly assigned…

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

This early-stage trial is testing a new experimental medicine called NNC1679-0001 for type 2 diabetes. Researchers want to see if it is safe and how well the body tolerates it. The study includes both healthy volunteers and people with type 2 diabetes, who will receive either a s…

This study tests an experimental drug called linvoseltamab in 220 people with AL amyloidosis that has returned or not improved after prior therapy. The drug aims to reduce abnormal proteins that damage organs like the heart and kidneys. The trial first finds the safest dose, then…

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

This early-phase trial is testing a treatment where a patient's own immune cells are modified in a lab to better recognize and attack cancer cells. It is for people with certain types of Non-Hodgkin lymphoma that have come back or not responded to standard treatments. The main go…

This study tests whether sepiapterin, a powder taken daily, can preserve intelligence and quality of life in children with phenylketonuria (PKU) when started early in childhood. The trial will follow 56 children for years, measuring IQ and other outcomes. It is an open-label phas…

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 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…

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

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 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 an oral drug called nizubaglustat in children and teens with rare genetic disorders (GM1, GM2 gangliosidosis) that damage the brain and nerves. The goal is to see if the drug can slow disease progression and improve movement and coordination. About 75 participant…

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

This study tests the long-term safety and effectiveness of a daily medication called nizubaglustat in people with two rare genetic diseases that affect the brain and body. About 21 participants who were in a previous study or who have been on a similar drug (miglustat) will take …

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

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

This study tests a 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 whether computer alerts can help doctors prescribe cholesterol-lowering drugs to people with peripheral artery disease (PAD) who aren't taking them. About 400 adults with PAD will be randomly assigned to have their doctors receive an alert or not. The goal is to …

This study compares two digital approaches—live video classes and simple text messages—against traditional in-person classes to help 150 Hispanic/Latino adults with type 2 diabetes better manage their condition. The goal is to see if these digital tools can improve blood sugar co…

This study tests whether Enavogliflozin, a drug used for diabetes, can help people with amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart. About 68 adults with heart failure symptoms will take the drug or a placebo for 12 weeks, then switch. The ma…

This study tests a combination of two drugs, venetoclax and dexamethasone, in people with a rare blood disorder called AL amyloidosis that has come back or not improved after treatment. The trial includes 53 adults with a specific genetic marker (t(11;14)). The goal is to find th…

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

This study tests an experimental drug called ALN-CFB in people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cells despite standard treatment. The goal is to see if adding ALN-CFB is safe and can improve anemia. About 24 participants will receive eit…

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

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

This study tests whether a tart cherry citrate tablet can lower uric acid levels in people who have high uric acid but no gout symptoms. About 196 adults will take either the active tablet or a placebo twice daily for 24 weeks, along with lifestyle advice. The main goal is to see…

This study tests an experimental oral drug, TEV-56286, in 350 adults with multiple system atrophy (MSA), a rare and progressive brain disorder. Participants will receive either the drug or a placebo for 48 weeks to see if it slows disease worsening and is safe. The goal is to fin…

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

This study tests a 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 whether a special anti-inflammatory diet can lower inflammation and improve blood sugar control in people with type 2 diabetes. 88 adults will be randomly assigned to either the anti-inflammatory diet or standard diabetes dietary advice for 4 weeks. Researchers w…

This study tests whether peer coaching can help older adults (55+) with uncontrolled type 2 diabetes in rural Kentucky better manage their condition. Participants are paired with trained peer coaches who call them weekly or bi-weekly to discuss self-care and goals. The study will…

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

This study tests whether adding methotrexate (a drug that calms the immune system) can help adults with PKU tolerate and respond better to Palynziq, a protein-based treatment. About 12 adults who either haven't taken Palynziq before or have high blood Phe levels despite treatment…

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

This study tests if leg exercise (like pedaling) is better than arm exercise for controlling blood sugar, weight, and stress in pregnant women with gestational diabetes. 108 women in their second trimester will exercise three times a week for eight weeks. The goal is to find a sa…

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

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

This study tests a new drug called SHR-1918 for people with high cholesterol that remains high even when taking statins and another type of cholesterol-lowering drug (PCSK9 inhibitors). About 126 adults will receive either SHR-1918 or a placebo to see if it safely lowers their LD…

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

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

This study tests whether daily weight monitoring at home can reduce hospitalizations for heart failure in people with cardiac amyloidosis, a condition where protein buildup stiffens the heart. 320 adults with ATTR cardiomyopathy will either use a connected scale that alerts docto…

This study tests a new medicine called SHR-1918 for people with high triglycerides, a type of blood fat that can raise heart risk. About 360 adults will get either the drug or a placebo for 48 weeks to see if it safely lowers fat levels. The goal is to control the condition, not …

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

This study compares two cholesterol-lowering strategies in about 1,950 adults with chronic kidney disease and heart or blood vessel disease. One group gets a moderate-dose statin plus ezetimibe, the other gets a high-dose statin alone. The goal is to see if the combo is just as g…

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

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

This study tests a new daily pill, icovamenib, for people with type 2 diabetes who still have high blood sugar despite taking Ozempic. About 60 adults will receive either the pill or a placebo for 12 weeks. The main goal is to see if the pill lowers HbA1c (a measure of blood suga…

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 looks at whether THC, the active part of marijuana, can change how the body handles sugar and affects blood vessel health in people with type 2 diabetes. About 30 adults will take THC or a placebo for two weeks, then have their blood sugar and blood vessel function tes…

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

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 study tests a new drug called ADX-038 in healthy volunteers and people with PNH who still have low red blood cells despite treatment. The goal is to check safety and how the drug works in the body. It is an early-stage trial with 50 participants.

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

This early study tests a nasal spray called foralumab in 5 adults with multiple system atrophy (MSA), a rare brain disease that gets worse over time. The goal is to see if the drug can reduce harmful brain inflammation and slow down symptoms like movement problems. Researchers wi…

This study tests whether dipyridamole, a drug usually used for heart conditions, can raise low phosphate levels in kidney transplant recipients. Researchers will give the drug to 90 adult patients when their phosphate drops below normal. The goal is to see if it improves phosphat…

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

This 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 pilot study tests a 3-month program that combines online education, music-paced brisk walking, group discussions, and phone follow-ups for 40 adults with both metabolic syndrome and chronic kidney disease. The goal is to see if the program can reduce waist circumference and …

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

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

This study 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 a program called MiWeigh that helps people with obesity and related conditions like diabetes or high blood pressure lose weight. Participants get a personalized plan from a doctor, access to a website, and weekly check-ins. The goal is to see if this approach lea…

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

This 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…

This study tests a new medicine called zodasiran in people aged 12 and older who have a rare genetic condition that causes extremely high cholesterol (homozygous familial hypercholesterolemia). The goal is to see if the drug can safely lower bad cholesterol levels over 12 months.…

This study tests a new drug called TS-172 in 30 adults on peritoneal dialysis who have high phosphorus levels. Participants take the drug daily for several weeks to see if it can safely bring their phosphorus down to a target range. The goal is to find a better way to manage this…

This study will test if a 'produce prescription' program, called Eat Well, helps veterans with high blood pressure and food insecurity. About 1,500 veterans in North Carolina will either get money for fruits and vegetables for 6 or 12 months, or standard care. Researchers will tr…

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

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

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

This study 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…

This pilot study is testing whether combining cognitive behavioral therapy (CBT) with guided exercise can improve blood sugar control, reduce depression, and boost quality of life in Black men with type 2 diabetes. Eighty participants will receive 8 sessions of CBT, with half als…

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 compares two treatments for a condition called primary aldosteronism, where a small adrenal gland tumor causes high blood pressure. One treatment is laparoscopic surgery to remove the tumor; the other is radiofrequency ablation (RFA), which uses heat delivered through …

This study compares the iLet Bionic Pancreas, an automated insulin delivery system, to standard care for adults with type 1 or type 2 diabetes who use insulin. About 240 participants will use the device for 13 weeks or continue their usual routine. The goal is to see if the devic…

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 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 a digital tool that helps children under 12 with type 1 diabetes calculate their insulin doses. The tool considers factors like carbs, blood sugar levels, and trends. Researchers will compare blood sugar control (HbA1c) over 6 months between kids using the tool p…

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

This study tests whether a program called Sisters for Heart Health can help farmworker women ages 18-50 reduce stress, feel less isolated, and improve their heart health. Participants will join weekly peer support groups led by trained community health workers for 10 weeks. The s…

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

This early-phase trial is testing a new type of gene therapy called CAR 70-BCMA dual-target CAR-T for people with relapsed or refractory plasma cell neoplasms, including multiple myeloma. The therapy involves taking a patient's own immune cells, re-engineering them to recognize a…

This study tests whether sodium phenylbutyrate (ACER-001), a drug already approved for another condition, can help people with MCAD deficiency caused by a specific gene mutation. About 24 participants aged 4 and older will take the drug and be monitored for safety and how well it…

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

This early-phase study tests whether dichloroacetate (DCA), a drug used safely for decades in a rare metabolic disorder, can improve brain function during low blood sugar (hypoglycemia) in people with type 1 diabetes. Sixteen participants with a history of severe hypoglycemia or …

This early-stage study tests an experimental oral drug called ONO-3310 in healthy Japanese men and in people with type 2 diabetes who also have chronic kidney disease. The main goal is to check the drug's safety and how the body processes it. Researchers will also measure its eff…

This study investigates whether continuous glucose monitor (CGM) data can pinpoint the time of day when a person with prediabetes experiences the largest blood sugar spikes after eating. Participants wear a CGM and activity tracker for 10 days to identify their personal 'vulnerab…

This study explores whether MRI scans can safely and accurately detect cancerous or precancerous mouth lesions in people with Fanconi Anemia, a rare genetic condition that raises cancer risk. Researchers will compare MRI results to standard biopsies in 80 adult participants. If s…

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…

This study tests a new radioactive tracer called 18F-mFBG to see if it can detect nerve damage in the heart caused by Lewy body diseases like Parkinson's and Lewy body dementia. About 20 adults will get a PET scan after an injection of the tracer. The goal is to see if the scan c…

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

This study enrolls about 4,000 adults aged 60 and older with heart failure who are at high risk for ATTR amyloidosis, a rare disease that stiffens the heart. Doctors receive special training on how to spot and diagnose the condition using standard tests. The goal is to see if thi…

This study is testing a home-based eye-tracking system called AVIGA to see if it can detect when wet age-related macular degeneration (AMD) comes back. Sixty adults aged 50-99 who are being treated for wet AMD will use the AVIGA device at home, and its accuracy will be compared t…

This study is testing a cloud-based AI platform called Willem that analyzes electrocardiogram (ECG) data to detect Transthyretin cardiac amyloidosis (ATTR-CA), a rare and serious heart condition. Researchers will compare ECGs from 2000 adults—some with confirmed ATTR-CA and some …

LiverSeek is a study testing an automated screening program for advanced liver fibrosis in people aged 50-75 with risk factors like diabetes or obesity. When a routine blood test is done, the lab system automatically calculates a fibrosis score and, if needed, orders additional t…

This study is testing a new laser-based blood test that uses nanoparticles to detect pancreatic cancer early. Researchers will enroll 400 people, including those with diabetes, pancreatic cysts, or pancreatic cancer, to see if the test can accurately identify cancer. The goal is …

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

This early-phase study tests a new radioactive tracer called [¹⁸F]MODAG-009 to see if it can safely highlight abnormal protein clumps in the brains of people with Parkinson's disease or multiple system atrophy (MSA). About 13 participants, including healthy volunteers, will recei…

This study looks at whether taking a small piece of ligament during routine carpal tunnel surgery can help diagnose transthyretin amyloidosis, a serious condition that often goes unnoticed until it affects the heart. Researchers will compare this new biopsy method to the standard…

This study is testing a new, non-invasive liver scan called iLivTouch to see if it can accurately detect a serious liver condition called MASH in people who have both type 2 diabetes and fatty liver disease. The scan measures liver inflammation, fat, and stiffness without any nee…

This pilot study is testing whether eating fermented poi every day for four weeks can improve the gut health of pregnant women and lower their risk of developing gestational diabetes. Fifty pregnant women between 10 and 24 weeks will eat one pound of poi daily and receive nutriti…

This study is testing whether a home-based screening kit can identify hidden heart risks better than standard doctor visits. 45,000 adults in Sweden aged 50-75 will either receive a home blood pressure monitor, a finger-prick blood test, and a health questionnaire, or continue wi…

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

This study tests whether ursodeoxycholic acid (UDCA), a drug used for bile duct problems, can stop statins from raising blood sugar. 128 adults with high cholesterol but normal blood sugar will take either UDCA or a placebo alongside atorvastatin for 6 months. Researchers will tr…

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

This study tests whether screening women for type 2 diabetes while they are still in the hospital after giving birth can help catch the disease early and prevent it. About 116 women who had gestational diabetes will be randomly assigned to either early testing or usual care. The …

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

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

This 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 a 3-year lifestyle program for 1,305 Brazilian adults at high risk for type 2 diabetes. The program combines in-person and online counseling on diet, exercise, sleep, and stress. Researchers will track who develops diabetes and measure weight, blood sugar, and qu…

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

This 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 tests whether a natural bile acid called 7-Ketolithocholic Acid, taken as a supplement, can lower blood sugar levels in 100 adults with prediabetes. Participants will also follow a healthy diet and exercise plan. The goal is to see if this supplement can help prevent o…

This study tests whether taking part in racial justice activism can prevent depression and reduce physical stress in Black and Latinx young people aged 15-20. Researchers will measure mood, stress hormones, and health markers over two years. The goal is to see if activism can be …

This study looks at whether adding brewer's spent grain (a beer-making byproduct) to cookies can help control blood sugar after eating. Twenty healthy adults will eat regular cookies and special high-fiber cookies on different days, and their blood sugar will be checked over two …

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

This study will test whether giving women with gestational diabetes a home ultrasound device, guided remotely by a doctor, improves their satisfaction and health outcomes compared to standard care. About 90 participants will use the device for fetal checks from home. The goal is …

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

This study looks at whether the timing of a 9-hour eating window (early, mid, or late in the day) affects blood sugar control in people with obesity who have or are at risk for type 2 diabetes. Over 7 days, 120 participants will follow one of three time-restricted eating schedule…

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 …

This study tests whether ear acupuncture, led by village doctors and supported by telemedicine, can improve sleep in adults aged 65 and older who have both diabetes and high blood pressure. Participants will receive either real or sham ear acupuncture for 6 weeks. The goal is to …

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 tests whether a personalized form of transcranial magnetic stimulation (TMS) can improve motor and non-motor symptoms in people with Parkinson's disease, multiple system atrophy, or progressive supranuclear palsy. Fifty participants will receive 10 days of targeted TMS…

This Phase 2 trial tests whether KL1333 can reduce fatigue and improve leg strength in adults with primary mitochondrial disease, a genetic condition that affects energy production. About 180 participants will receive either KL1333 or a placebo twice daily for 48 weeks. The study…

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

This study tests whether a personalized mix of endurance and strength exercises, done at home with video check-ins, can improve mobility and muscle strength in people with mitochondrial myopathy. Fifteen adults with confirmed genetic mutations will follow the program for up to 12…

This study looks at whether walking in a natural setting (like a park) is better for reducing stress, anxiety, and improving mood compared to walking in a built-up commercial area. 216 adults with prediabetes will walk 150 minutes per week for six weeks in each environment, with …

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

This study tests whether the Thrive AI Health app can help adults with type 2 diabetes or overweight improve their daily habits like diet, exercise, and sleep. About 20 participants will either use the app or not, and researchers will compare changes over time. The app uses artif…

This study tests if shining a special light on the head (transcranial photobiomodulation) can improve memory and thinking in people with mild cognitive impairment. Forty participants will receive either real or fake light sessions for 12 weeks. Researchers will measure brain acti…

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

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

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

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

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

This study 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 looks for genetic changes linked to progressive familial intrahepatic cholestasis (PFIC) in adults with unexplained bile flow problems. Researchers will collect health information and blood samples from 150 participants across Spain during a single visit. The goal is t…

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

This study looks at whether older adults with prediabetes have lower brain energy use and slower thinking compared to those with normal blood sugar. It also tests if a single dose of a ketone supplement can improve brain energy and processing speed. Twenty participants will under…

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

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

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

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

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

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

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

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

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

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

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

This study looks at how improving sleep regularity might help control blood sugar and reduce heart disease risk in adults with type 1 diabetes. Researchers will monitor 100 participants' sleep, blood sugar, and heart health markers. The goal is to understand the body clock's role…

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

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

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

This 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…

This study explores the link between sleep duration and blood sugar control in adolescents aged 12-20 with type 2 diabetes. Researchers will first measure sleep and blood sugar in 67 teens, then test a program using Fitbits and text messages to encourage longer sleep in 15 teens …

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

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

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

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

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

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

This study looks at gum disease in people with rare kidney disorders like Alport syndrome, Fabry disease, and tuberous sclerosis, as well as lupus. Researchers will compare 100 participants to those with chronic kidney disease and healthy controls. They aim to understand how comm…

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

This study tests a new PET scan technique to better track heart damage in people with a condition called ATTR-CM, a progressive heart muscle disease. Researchers will scan 140 participants to see if the imaging can show how well treatments are working. The goal is to improve moni…

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

This pilot trial is testing whether amyloidosis patients can use the MyFitnessPal smartphone app to track their daily food and supplement intake for 8 weeks. The goal is to see if this low-cost method is feasible for studying malnutrition and weight loss in this condition. Forty …

This study follows 300 adults aged 30–60 with genetically confirmed familial hypercholesterolemia, a condition causing very high cholesterol. Researchers use CT scans to see how coronary artery plaques change over 5 years. The goal is to better understand disease progression and …

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 well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) in everyday medical practice. Researchers will track 50 Italian patients for 12 months to see if their anemia improves and if they need fewer blood transfusions. The go…

This study is looking for biological markers (biomarkers) in blood, saliva, and urine that signal damage to insulin-producing beta cells in the pancreas. Researchers will compare samples from people with type 1 diabetes, type 2 diabetes, prediabetes, and healthy volunteers. The g…

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

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

This study looks at whether CFTR modulators (ETI) help control blood sugar in people with cystic fibrosis-related diabetes (CFRD). Researchers will compare 30 adults with CFRD who take ETI to those who cannot take it. The goal is to see if the drug improves how the body handles s…

This study is testing a new device that combines an insulin pump with a continuous glucose monitor in one unit for adults with type 1 diabetes. The goal is to see how insulin delivery near the sensor affects glucose readings and to gather data to improve the design. About 40 part…

This study is observing 40 adults with multiple sclerosis to see how clinical tests of balance and walking relate to detailed motion-capture measurements. Participants will attend one session where they walk on force plates and perform balance tasks while cameras track their move…

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

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

This study tests a program called SPARK-CGM that helps primary care clinics prescribe continuous glucose monitors (CGM) to people with diabetes. CGM devices track blood sugar in real-time, but they are underused in primary care. The program provides training, workflow support, an…

This study looks at how liver fat changes the way the body processes rosuvastatin, a common cholesterol drug, in children aged 8 to 21. Researchers will give a single dose to 50 kids with high cholesterol and measure drug levels and response. The goal is to understand if fatty li…

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 looks at whether sulfasalazine, a drug for inflammatory arthritis, makes the standard diabetes blood test (HbA1c) falsely low. That could mean diabetes goes undiagnosed or is undertreated in these patients. Researchers will use continuous glucose monitors and fasting b…

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 global registry enrolls people with Pompe disease to track how the condition changes over time, whether they receive treatment or not. By collecting data from up to 2,000 participants, researchers hope to better understand the disease's progression and improve patient care. …

This study from Mayo Clinic looks at how one session of exercise changes breastmilk. Researchers will measure tiny particles called exosomes that carry messages between cells. They want to see if exercise boosts these particles in breastmilk, which might help prevent childhood ob…

This study looks at how parents and doctors handle the moment a newborn is diagnosed with phenylketonuria (PKU) through routine screening. Researchers will interview 80 parents and medical staff to understand the emotional impact and find ways to improve how the diagnosis is shar…

This study aims to better understand how Pompe disease affects the nerves and muscles involved in breathing. Researchers will analyze nerve conduction and diaphragm activity in 20 adults with Pompe disease or unexplained respiratory failure. The goal is to identify patterns that …

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

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

This study looks at what happens when adults with metabolic syndrome stop taking their usual metformin medication for 14 days. Researchers want to see if a short break affects insulin sensitivity, blood sugar levels, and other health markers. Participants will be randomly assigne…

This study follows up to 10 pregnant or breastfeeding individuals with transthyretin amyloidosis who have taken eplontersen, along with their infants. Researchers will track pregnancy complications, birth defects, and infant health during the first year of life. The goal is to de…

This study will analyze health records from 2009 to 2024 to find out how many people in Germany have three serious eye conditions: wet age-related macular degeneration, diabetic macular edema, and retinal vein occlusion. Researchers will look at data from two large German health …

This trial is studying a medical device that has not been approved by the U.S. FDA. The purpose and specific details are not publicly available. It likely involves a small group of participants to gather initial safety or effectiveness data.

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

This study will give 100 adults with diabetes a thermal camera that attaches to their smartphone to take daily pictures of their feet. The goal is to collect temperature data and see if hot spots can predict where foot ulcers might form. Participants will use the device at home f…

This study looks at how a 12-week lifestyle change affects inflammation and stress markers in 60 overweight adults with type 2 diabetes and moderate chronic kidney disease. Participants will follow a modified diet and exercise plan. The goal is to see if these changes can reduce …

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

This study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

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

This study 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 is testing how to safely perform skin allergy tests in people receiving biotherapies for cancers and blood disorders. Researchers will find the highest concentration of each biotherapy that does not cause a skin reaction in 9 out of 10 patients. The goal is to establis…

This study uses a special eye scan (OCT angiography) to take detailed pictures of blood vessels in the back of the eye. Researchers will follow about 1,050 people with retinal diseases like diabetic retinopathy and healthy volunteers to see how these images can help detect early …

This study looks at whether personality traits are linked to routine lab test results in 1,000 adults getting a health check-up. Participants fill out a personality questionnaire and provide blood samples. The goal is to see if certain personality types are more likely to have ab…

This study tracks 180 orthopedic patients in Cyprus to see if a single doctor visit changes how much they trust AI health information. Patients fill out surveys before and after their appointment, and doctors also report on the accuracy of any AI advice patients brought up. The g…

This study looks at tiny particles called extracellular vesicles (EVs) released from fat cells. Researchers want to understand how these particles carry signals that may affect heart and liver health in people with obesity. About 100 adults having weight-loss surgery will provide…

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 fat buildup in the pancreas contributes to type 2 diabetes and whether weight loss can reverse it. Researchers will follow 104 overweight adults, including those without diabetes, with prediabetes, and with short- or long-term diabetes. Participants will f…

This observational study will follow 50,000 people in Asia and the United States who have chronic kidney disease and type 2 diabetes and are already taking finerenone as part of their regular care. Researchers will collect data from electronic health records and insurance claims …

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 compares how natural sugar (sucrose) and the artificial sweetener sucralose affect blood sugar control and metabolism in 120 adults with normal weight, overweight, or obesity. Participants will consume low or high doses of each sweetener in solid or liquid form over 12…

This study aims to find a blood marker that can tell how healthy the blood vessels in the eye are for people with diabetic retinopathy. Researchers will collect blood samples from 192 participants with type 2 diabetes and analyze special cells in the blood. The goal is to discove…

This study reviews medical records of 400 adults aged 40-70 who were recently diagnosed with type 2 diabetes in five Southeast Asian countries. Researchers will look at health data from before and after diagnosis to understand how heart disease risk is distributed. No new treatme…

This early-stage trial in 16 healthy adults tests whether a new drug for high cholesterol, HRS-1301, changes how the body processes two common medicines: midazolam (a sedative) and atorvastatin (a cholesterol drug). Participants will take these drugs alone and together with HRS-1…

This study looks at how fat is stored in people with familial partial lipodystrophy (FPLD), a condition that can lead to diabetes and heart disease. Researchers will compare fat tissue from people with FPLD and healthy volunteers. Participants drink special water and get infusion…

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 follows 100 obese patients starting dialysis to understand why they are less likely to receive a kidney transplant. Researchers will track health changes over two years and compare them to non-obese patients. The goal is to identify barriers and improve access to trans…

This study is testing whether mixed reality (using a Meta Quest 3 headset) can reliably measure walking ability, balance, and leg strength in people with type 2 diabetes. Researchers will compare the digital versions of three common physical tests (6-minute walk, timed up and go,…

This study tracks eye pressure in 100 people receiving a larger-volume injection of Aflibercept (0.07mL) for macular diseases like age-related macular degeneration. Researchers measure pressure right after the injection and at several time points, then check for any nerve damage …

This study looks at women with lipodystrophy, a condition causing abnormal fat tissue, who have been pregnant. Researchers want to see if taking the drug metreleptin during pregnancy affects the mother's or child's health. About 90 women and their children will share medical hist…

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

This study 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…

This early-stage trial is testing whether a single tablet containing both laroprovstat and ezetimibe works as well as taking the two drugs separately. The study involves 18 healthy adults who will receive different pill combinations in random order. Researchers will measure how t…

This study uses leftover tissue from heart and orthopedic surgeries to learn more about how calcification (hardening) happens in bones and blood vessels. Researchers will grow cells from these tissues in the lab to study the process. The goal is to better understand the disease, …

This observational study is gathering detailed metabolic data from 25 adults with insulin-dependent type 2 diabetes. After drinking a standard liquid meal, participants will have frequent blood tests to measure glucose, insulin, and other markers. They will also wear continuous g…

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

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 watches 500 adults in South Korea who have chronic kidney disease and type 2 diabetes and are taking finerenone as part of their normal care. Researchers will collect information from medical records and doctor visits over up to 12 months to see why doctors prescribe i…

This study looks at how menstrual cycles and birth control pills impact blood sugar levels and exercise in women with type 1 diabetes. Researchers will monitor 150 women for three months using continuous glucose monitors and offer personalized insulin and diet adjustments. The go…

This study looks at how blood sugar changes day-to-day in people with ketosis-prone diabetes as they go into remission. Researchers will use this data to build a math model that describes the remission process. The study involves 12 African American adults with a BMI of 28 or hig…

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

This study is collecting health data from 1,500 adults with diabetes in Switzerland to see how well current treatments work in everyday life. Researchers will track complications, quality of life, and survival over time. The goal is to find out what's working and what needs impro…

This study looks at how an FDA-approved diabetes drug, Canagliflozin (Invokana), might protect the kidneys in people with type 2 diabetes. Twenty adults aged 18-80 with type 2 diabetes for at least 3 years will take the drug daily for 6 months. Researchers will use kidney biopsie…

This study follows 214 people in China with multiple system atrophy, a rare and fast-worsening brain disease that affects movement and automatic body functions. Researchers will track symptoms over time to learn how the disease progresses and what factors influence it. The goal i…

This study monitors up to 50 people with MPS VII (Sly Syndrome) to understand how the disease changes over time. It also checks the long-term safety and effectiveness of the drug vestronidase alfa. Participants may or may not be taking the drug, and the study involves regular che…

This study looks at whether people with type 2 diabetes, COPD, or heart disease who join a health program in Halsnaes Municipality improve their physical function and well-being. About 194 adults will be tracked before and after the program using tests and questionnaires. The goa…

This study gives 200 athletes either high-dose vitamin D3 (4,000 IU daily) or a placebo for 4 to 8 weeks. Researchers measure changes in vitamin D levels, hormones, muscle function, cognitive performance, and body composition. The goal is to see if extra vitamin D improves athlet…

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

This observational study looks at whether taking raw cornstarch with meals helps control blood sugar in adults with glycogen storage disease type I (GSD I). Eight participants will wear continuous glucose monitors and keep food diaries for two weeks. Researchers will compare bloo…

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

This study follows 800 adults with synucleinopathies—diseases like Parkinson's, Lewy body dementia, and multiple system atrophy—to learn how they develop and change over time. Researchers will track symptoms, biological markers, and early signs like dizziness on standing or dream…

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

This study looks at how accurate continuous glucose monitors (CGMs) are in children and young adults with type 1 diabetes when they have surgery. Researchers will compare CGM readings to standard blood glucose tests during the procedure. The goal is to see if these devices can be…

This study looks at whether a special nursing care model helps people with long-term health conditions like diabetes, heart disease, or Parkinson's work better with their advanced practice nurse. About 420 adults will take part. The goal is to see if this approach improves how pa…

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

This study looks at how lean pork loin and plant-based protein affect muscle building and blood sugar control in adults with prediabetes. Twenty participants aged 40-65 will eat prepared meals for three days. Researchers will measure muscle protein synthesis and glucose levels to…

This study aims to collect DNA samples and detailed eye exam data from 1,000 people with rare inherited eye diseases like aniridia, Best disease, and albinism. Participants provide a saliva or blood sample and share their eye health records. The goal is to expand a research repos…

This study collects blood, bone marrow, and other samples along with health information from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze the participants' genes to find new genetic causes of these conditions and understa…

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

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