Metabolic disease

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

This phase 3 trial is testing whether adding the immunotherapy drug atezolizumab to standard chemotherapy can help prevent cancer from coming back in people with stage III colon cancer that has a specific DNA repair defect (dMMR). About 712 participants will receive either chemo …

This study is testing the Omnipod 5, a tubeless insulin pump that automatically adjusts insulin delivery, in real-world settings for people with type 1 diabetes. About 304 participants aged 2 and older will use the system for the first time. Researchers will track blood sugar con…

This study tests a system that combines a continuous glucose monitor (CGM) with daily insulin dose recommendations for people with type 2 diabetes who are starting basal insulin. Participants wear a Dexcom CGM sensor and receive suggested insulin doses through the DexBasal system…

This study tests an experimental drug called CAEL-101 in 125 people with a rare disease called AL amyloidosis, where abnormal proteins build up in organs like the heart and kidneys. The drug is designed to remove those protein deposits. The goal is to see if it helps people live …

This study tests the long-term safety and effectiveness of an experimental drug called BBP-418 (ribitol) in people with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle-weakening disease. Participants who completed a previous study will take BBP-418 orally twice d…

This study tests a drug called triheptanoin, already approved for similar conditions, to see if it can prevent dangerously low blood sugar in people with MCADD, a rare inherited disorder. About 24 participants aged 4 and older will take the medication and be monitored for safety …

This study tests a two-step treatment for people with relapsed or refractory B-cell Non-Hodgkin lymphoma. First, patients receive a bispecific antibody therapy (Obinutuzumab and Glofitamab) to help the immune system attack cancer cells. Then, they get an infusion of CD19-CAR T ce…

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

This study tests a combination of two drugs, ixazomib and rituximab, in people with slow-growing B-cell non-Hodgkin lymphoma. The goal is to see how well the drugs shrink or control the cancer. About 33 adults with different types of this lymphoma are taking part.

This large study compares two diabetes drugs—oral semaglutide and sitagliptin—in over 25,000 people with type 2 diabetes and heart failure. Researchers want to see which drug better prevents heart failure worsening or death. The study uses existing medical records to mimic a clin…

This pilot study tests whether giving people with type 2 diabetes personalized diet advice based on their continuous glucose monitor (CGM) readings can help them better control their blood sugar. Twenty adults with type 2 diabetes will receive either standard dietary care or a ta…

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

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

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

This study tests a new oral solution called NPI-001 in people with cystinosis, a rare disease that causes cystine buildup in cells. Researchers want to see if NPI-001 is safe and works better than the current drug cysteamine. About 12 participants aged 10 and older will stop thei…

This study tests a gene therapy called PBKR03 for babies with early infantile Krabbe disease, a severe genetic disorder that damages the brain and nerves. The treatment delivers a working copy of the GALC gene to the brain and body. Researchers will check safety and find the best…

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

This study looks at the safety and performance of the Endogenex System in 50 adults with type 2 diabetes. Participants previously received a sham (fake) procedure in an earlier study. The main goal is to track any serious side effects related to the device or procedure.

This early-phase trial is testing whether combining three drugs—tazemetostat, zanubrutinib, and an anti-CD20 antibody (rituximab or obinutuzumab)—is safe and effective for people with certain types of lymphoma that have come back or not responded to treatment. The study involves …

This study tests whether using a continuous glucose monitor (CGM) helps adults with type 2 diabetes who do not take insulin improve their blood sugar control. Participants are randomly assigned to either use a real-time CGM or receive routine care with occasional blinded CGM wear…

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

This phase 2b trial tests frexalimab, an antibody that calms the immune system, in 192 adults and teens newly diagnosed with type 1 diabetes. The goal is to see if it can preserve the body's ability to produce insulin, reducing reliance on injections. Participants receive frexali…

This study explores how different amounts of carbohydrates and a fiber supplement called inulin-FOS affect blood sugar control in adults with type 1 diabetes. Participants will try three two-week diets: one with 30% carbs, one with 50-55% carbs, and one with 50-55% carbs plus inu…

This study tests an experimental drug called ONO-2808 in people with multiple system atrophy (MSA), a rare and serious brain disease. About 92 participants will receive either the drug or a placebo daily for up to 80 weeks. The goal is to see if the drug is safe and might help co…

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

This phase 3 study tests Coagadex, a human clotting factor X, in 15 adults with acquired factor X deficiency caused by AL amyloidosis. The goal is to see if it safely stops active bleeding and helps manage bleeding during surgery. Participants receive the drug on demand for bleed…

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

This study is testing whether giving the BCG vaccine (normally used for tuberculosis) twice, four weeks apart, can improve blood sugar control and reduce insulin needs in children aged 8 to 17 who were recently diagnosed with type 1 diabetes. The trial involves 100 participants a…

This Phase 3 study tests plozasiran, an injectable drug, in 1456 adults with high triglycerides. Participants receive 4 doses over 12 months. The goal is to see if the drug safely lowers blood fat levels compared to a placebo.

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

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

This study tests whether giving the BCG vaccine (traditionally used for tuberculosis) twice, four weeks apart, can help children aged 8 to 18 with type 1 diabetes. The goal is to see if it improves blood sugar control, reduces insulin use, and lowers the risk of dangerously low b…

This early study tests a new drug called ABBV-101 in people with several types of non-Hodgkin lymphoma that have come back or not responded to other treatments. The drug is a pill that targets a protein (BTK) involved in cancer growth. The main goals are to find a safe dose and s…

This study tests a new drug called EYE103 (Restoret) against an existing treatment, ranibizumab (Lucentis), for people with diabetic macular edema, a common cause of vision loss in diabetes. About 1,054 participants receive injections into the eye every 4 weeks for a year, then l…

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

This study tests a new drug combination (AZD9550 and AZD6234) for weight loss in 166 adults who are overweight or obese, with or without type 2 diabetes. The main goals are to check safety, tolerability, and how the drug moves through the body. It is an early-stage trial, so it f…

This phase 3 trial tests the drug mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic disorder that causes red blood cells to break down too quickly. These children need regular blood transfusions. The study compares mitapivat to a placebo to see if…

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

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

This study looks at the long-term safety and tolerability of iptacopan in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have already completed earlier phase 2 or 3 studies with the drug. About 208 participants will continue taking iptacopan and be monitored for side e…

This phase 3 study tests a drug called PTC923 (sepiapterin) in 200 people with phenylketonuria (PKU), a genetic condition that requires a strict low-protein diet. The main goal is to see if the drug is safe over the long term and whether it allows people to eat more protein witho…

This study tests a new weekly injection called enicepatide (CT-388) in 447 overweight or obese adults with type 2 diabetes. Participants receive either the drug or a placebo for 48 weeks. The goal is to see if the drug helps with weight loss and improves blood sugar levels.

This study follows 16 infants aged 6 months or younger with infantile-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. All receive alglucosidase alfa (Myozyme) as part of their routine care. Researchers track how many survive without needing a brea…

This study offers continued access to the drug mitapivat for adults with pyruvate kinase deficiency who completed an earlier Agios-sponsored trial and cannot get the drug commercially. Only 6 participants are enrolled, and the main goal is to monitor side effects. The study does …

This phase 2 trial tests whether a low dose of everolimus, a drug used in cancer, can safely improve signs of aging in 106 adults aged 55-80 who have insulin resistance or prediabetes. Participants take the drug daily or weekly for 24 weeks, and researchers measure changes in met…

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

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

This study looks at the long-term safety of a weekly intravenous treatment for people with emphysema caused by alpha-1 antitrypsin deficiency. About 290 adults who completed a prior study will receive the drug for 2 more years. The main goal is to monitor side effects, not to cur…

This study tests a drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic condition that causes red blood cells to break down too quickly, leading to anemia. The trial compares mitapivat to a placebo to see if it can raise hemoglobin levels…

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

This trial tests a new drug called Cadonilimab in 28 people with advanced colorectal cancer that has not responded to standard immunotherapy. The drug is a bispecific antibody that targets two immune checkpoints (PD-1 and CTLA-4) to potentially reactivate the immune system agains…

This study monitors about 200 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already taking iptacopan (Fabhalta). Researchers will use registry data to count infections caused by certain bacteria. The goal is to better understand the real-world safety of this medic…

This phase I trial tests a new approach for patients with high-risk or recurrent non-Hodgkin lymphoma. After a stem cell transplant, participants receive a single infusion of their own genetically modified T-cells designed to recognize and attack cancer cells. The study aims to f…

This large study looks at real-world data from 60,000 people with type 2 diabetes and obesity to compare four diabetes drugs—dulaglutide, semaglutide, tirzepatide, and sitagliptin—in preventing heart attacks, strokes, and death. Researchers are analyzing insurance claims to see w…

This study tests whether acetyl L-carnitine, a dietary supplement, can help reduce pain, inflammation, and improve blood sugar control in obese, diabetic, postmenopausal women with osteoarthritis. One hundred women will take either the supplement or a placebo for 12 weeks, alongs…

This study tests the safety of a drug called ferric citrate in children aged 6 to 17 with chronic kidney disease who have high phosphorus levels. The drug is taken by mouth to help control phosphorus, which can cause bone and heart problems if too high. The study involves 45 chil…

This phase 2 trial tests whether the drug DFMO can protect the body's remaining insulin-producing cells in people recently diagnosed with type 1 diabetes. About 81 participants will take either DFMO or a placebo twice daily for six months. Researchers will measure insulin product…

This phase 3 trial tests an experimental drug called ALXN1850 in 124 adolescents and adults with hypophosphatasia, a rare genetic bone disease. Participants receive either the drug or a placebo by injection under the skin. The main goal is to see if the drug improves walking dist…

This early-phase study tests a new drug called BGB-11417 (sonrotoclax) in people with B-cell blood cancers that have come back or not responded to treatment. The goal is to find the safest dose and see how well it works alone or combined with other drugs. About 437 adults will ta…

This phase 2 trial tests an injectable drug called DII235 in 233 adults with high lipoprotein(a), a genetic condition that raises heart disease and stroke risk. Participants have heart disease or type 2 diabetes. The study compares different doses of DII235 against a placebo to s…

This study compares a combination diabetes drug (iGlarLixi) against insulin alone to see which better reduces liver fat in people with type 2 diabetes and a common fatty liver condition called MASLD. About 36 participants will take one of the two treatments for 12 weeks, and doct…

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

This study aimed to see if using an exercise phone app and a continuous glucose monitor could help teenagers with obesity, prediabetes, or type 2 diabetes lose weight and improve their health. Participants would have worn a glucose monitor and, if in the intervention group, used …

This phase 2 trial tests whether empagliflozin, a diabetes drug also known as Jardiance, can help people with glycogen storage disease type Ib (GSD-1b). GSD-1b causes low neutrophil counts, leading to frequent infections and bowel inflammation. The study gives empagliflozin orall…

This study tests a donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…

This early-stage trial tests whether a single infusion of donated mesenchymal stem cells is safe for children with cerebral adrenoleukodystrophy (cALD), a rare and life-threatening brain disease. The stem cells are given as a temporary bridge to buy time before a more definitive …

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

This study tests a stem cell transplant method for people with acquired or inherited bone marrow failure. Donor stem cells are specially processed to remove certain immune cells, which may lower the risk of graft rejection and graft-versus-host disease. The goal is to see if this…

This study tests a team-based approach to help African American and Latinx adults with uncontrolled type 2 diabetes lower their blood sugar levels. Participants will receive extra support from a multidisciplinary team, while a comparison group gets usual care plus educational mat…

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

This phase 3 trial tests whether CAEL-101, an antibody that removes abnormal protein deposits from organs, can help people with AL amyloidosis live longer and avoid heart-related hospital stays. About 281 participants who have not yet received treatment for their plasma cell diso…

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

This study aimed to see if an 8-week low-calorie diet (about 1,300 calories per day) could reverse isolated impaired fasting glucose, a form of prediabetes, in overweight adults aged 35 to 65. Participants would have received pre-prepared meals and been monitored for changes in b…

This early-stage trial is testing a new oral drug called F-02-2-Na in 69 adults with high uric acid levels, with or without gout. The main goal is to check the drug's safety and how the body processes it at different doses. Participants will receive either the drug or a placebo, …

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

This study tests an injected drug called DR10624 in 40 adults with high triglycerides and fatty buildup in their neck arteries. Participants receive either the drug or a placebo for 24 weeks, with doses gradually increased. The main goal is to see if the drug reduces artery wall …

This study compares two ways of using phototherapy (light treatment) for jaundice in extremely premature infants (under 750 grams or 27 weeks gestation). One group gets continuous light, the other gets timed cycles based on bilirubin levels. The main goal is to see if cycled phot…

This study follows about 80 people with paroxysmal nocturnal hemoglobinuria (PNH) who have already taken danicopan in a previous trial. They will continue taking danicopan as an add-on to their standard C5 inhibitor therapy. The goal is to see if the combination remains safe and …

This phase 3 trial is testing whether the drug retatrutide can lower the chance of serious heart problems (like heart attacks and strokes) and slow kidney disease in 10,000 overweight adults who already have heart or kidney disease. Participants receive either retatrutide or a pl…

This clinical trial is testing whether dimethyl fumarate, a drug already used for multiple sclerosis, can improve balance and walking in adults with adrenomyeloneuropathy (AMN), a rare nerve disease. Forty participants will take either the drug or a placebo daily for 36 months, w…

This study tests whether home visits by female community health volunteers plus regular SMS messages can help adults in Pokhara, Nepal, better control high blood pressure, type 2 diabetes, and smoking. About 2100 participants are split into two groups: one gets the home visits an…

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

This study looks at how the drug crinecerfont works in babies under 2 years old with congenital adrenal hyperplasia (CAH), a condition where the body can't make certain hormones properly. The goal is to measure drug levels in the blood and check for side effects. Only 7 infants a…

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

This study looks at how well the diabetes drug empagliflozin (Jardiance) works in everyday medical practice, outside of a controlled research setting. Researchers are comparing it to other diabetes medicines in over 230,000 adults with type 2 diabetes. The goal is to see if the h…

This early-stage study tests an investigational drug called LY4086940 in healthy people and those with overweight, obesity, or type 2 diabetes. The main goal is to check the drug's safety and how the body processes it. About 216 participants will receive single or multiple doses …

This study tests a one-time gene therapy called UX701 for adults with Wilson disease, a genetic condition that causes copper buildup. The goal is to see if it is safe and can help the body control copper levels, possibly allowing patients to take fewer or no daily medications. Ab…

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

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

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

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

This study was designed to test two immunotherapy drugs, nivolumab and ipilimumab, in children and young adults with cancers that returned or didn't respond to treatment and had many genetic mutations. The goal was to see if the drug combination was safe and could shrink tumors. …

This study looks at the long-term safety and effects of an experimental drug called DNL310 for people with Hunter syndrome (MPS II), a rare genetic disorder. About 99 participants who completed earlier studies will receive the drug for up to 5 years. Researchers will monitor side…

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

This study tests a combination of two drugs, avelumab and M6620, in people with advanced solid tumors that have spread or can't be removed by surgery. The tumors must have a specific DNA repair defect. The goal is to find the safest dose and see if the combination can help contro…

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

This study follows 64 Polish adults with paroxysmal nocturnal hemoglobinuria (PNH) who are taking ravulizumab as part of their routine care. Researchers will track blood markers like LDH and how many patients need blood transfusions over time. The goal is to see if the drug works…

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

This study looks at whether eating proteins and vegetables before carbohydrates (meal sequencing) can improve blood sugar control in 70 adults with diabetes or prediabetes who also experience food insecurity. Participants will receive free produce boxes and either standard nutrit…

This study tests whether a 12-week multimodal exercise program can improve physical performance and muscle function in adults aged 45-64 with type 2 diabetes. Participants are randomly assigned to either the exercise program or standard care. The goal is to find a practical way t…

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

This study tests a new drug called encaleret against usual treatments for people with a rare genetic condition called autosomal dominant hypocalcemia type 1 (ADH1), which causes low blood calcium and high urine calcium. About 67 participants will receive either encaleret or stand…

This study looks at whether the amount of effort you put into strength training can improve blood sugar control and mental health in people with type 2 diabetes. About 48 adults with type 2 diabetes will do resistance exercises at different effort levels. Researchers will track b…

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

This early-stage trial tests a one-time gene therapy called CLN-301 in 7 children aged 3 to 10 with CLN3 Batten disease, a rare genetic disorder that causes progressive loss of vision, movement, and thinking skills. The therapy delivers a working copy of the CLN3 gene directly in…

This Phase 2 trial tests whether a patient's own stem cells, injected into the spinal fluid, can slow the progression of multiple system atrophy (MSA), a rare and fatal brain disease. The study involves 71 adults aged 30-70 with early-stage MSA. Researchers will measure changes i…

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

This early-stage study tests a new drug, maridebart cafraglutide, to see if it helps people with type 2 diabetes use insulin better. About 56 adults on metformin will receive either the drug or a placebo. The main goal is to measure changes in insulin sensitivity after 25 weeks.

This study tests a daily oral drug called nemtabrutinib (ARQ 531) in 190 people with blood cancers like lymphoma or leukemia that have come back or not responded to prior treatment. The goal is to find a safe dose and see if the drug can shrink tumors. It is an early-phase trial,…

This early-stage trial tests a combination of three drugs—venetoclax, ixazomib, and dexamethasone—in 24 patients with light chain amyloidosis that has returned or not responded to prior treatment. The study focuses on finding the safest dose and checking side effects. All partici…

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

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

This large Phase 3 trial is testing whether a drug called inclisiran can reduce major heart problems like heart attacks and strokes in people who already have heart disease. About 17,000 participants are taking either inclisiran or a placebo, along with their usual cholesterol-lo…

This study looks at health records of 120,000 people with type 2 diabetes and overweight to compare two injectable drugs: semaglutide and dulaglutide. Researchers want to see which one better reduces the risk of death, heart attack, or stroke. The goal is to understand how these …

This study tests a new investigational medicine called CagriSema against an approved drug, tirzepatide, in people with type 2 diabetes whose blood sugar is not well controlled by current pills. About 1,023 adults will receive either CagriSema or tirzepatide once a week for up to …

This study tests a drug called ACER-001 (sodium phenylbutyrate) in a single patient with combined D,L-2-hydroxyglutaric aciduria, a rare genetic disorder that causes brain damage and seizures. The goal is to see if the drug is safe and can lower harmful acid levels in the body. R…

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

This study looks at whether allowing a slightly higher blood sugar range (70-110 mg/dL) during labor in mothers with diabetes can reduce the number of newborns with low blood sugar. Researchers will compare this approach to standard care in 169 pregnant women planning a vaginal d…

This early-phase trial tests whether adding immune cells—either the patient's own regulatory T cells or donor bone marrow cells—to a standard islet transplant can improve blood sugar control in people with brittle type 1 diabetes. About 24 adults aged 18 to 70 will receive one of…

This study tests a drug called eplontersen in Chinese adults with a heart condition caused by abnormal protein buildup. The goal is to see if the drug lowers that protein and is safe over time. Participants will be randomly assigned to receive the drug or a placebo for 24 weeks, …

This study looks at whether the diabetes drug metformin, combined with a low-calorie diet, helps overweight or obese adults with a large waistline lose weight. About 150 participants will take either metformin or a placebo for 24 weeks. The main goal is to see if at least 5% of b…

This study tests a 3-month lifestyle program called the PILI Pasifika Program for Native Hawaiian and Pacific Islander adults who are overweight or have conditions like high blood pressure or diabetes. The program includes group lessons on healthy eating and physical activity, pl…

This study tests a new medicine called SHR-3167 in adults with type 2 diabetes whose blood sugar is not well controlled by pills. About 100 participants will receive different dosing plans to see which works best at lowering blood sugar while avoiding dangerously low sugar levels…

This study looks at whether dulaglutide, a drug used for type 2 diabetes, can help regulate menstrual cycles in adolescent girls with type 1 diabetes who have poor blood sugar control. Researchers will give some participants dulaglutide plus insulin, while others get only insulin…

This Phase 3 study tests a new drug called JR-141 against the current standard treatment (idursulfase) in 86 people with Hunter syndrome (MPS II). The goal is to see if JR-141 can better reduce harmful substances in the brain and improve thinking skills. Participants can switch t…

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

This study tests a small, refillable implant placed in the eye that delivers medication every 24 weeks, compared to standard monthly eye injections. It includes about 634 adults with diabetic macular edema, a condition that causes vision loss. The goal is to see if the implant wo…

This study tests whether the drug tafamidis can safely stop the progression of ATTR amyloidosis in people who have already received a heart transplant. About 25 stable patients will take tafamidis daily for 12 months. Researchers will measure changes in blood protein levels, nerv…

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

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

This phase 2 trial tests a drug combination including daratumumab for people with AL amyloidosis, a rare disease where abnormal proteins damage organs, especially the heart. The study has two groups: one gets the full combo right away, the other starts some drugs later. Researche…

This Phase 3 trial tests an experimental drug called amlenetug in 401 people with multiple system atrophy (MSA), a rare and serious brain disorder. The goal is to see if the drug can slow the worsening of symptoms and improve survival. Participants receive either the drug or a pl…

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

This study looks at whether giving low-income people with diabetes or hypertension a monthly grocery card ($80 or $120) helps them control their disease and feel less stressed about food. About 4,450 adults in California are taking part. Researchers will check changes in blood su…

This study tests a gene therapy called RGX-121 in children aged 4 months to 5 years with Hunter syndrome, a rare genetic disease that affects the brain and body. The therapy delivers a working copy of the missing gene to the central nervous system. Researchers will measure improv…

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

This study tests two drugs—abiraterone and olaparib—alone or together in men with advanced prostate cancer that has spread and no longer responds to hormone therapy. Only men whose tumors have certain DNA repair defects (like BRCA or ATM mutations) are included. The goal is to se…

This phase 2 study tests a personalized immune cell therapy called tumor-infiltrating lymphocytes (TIL) in 20 people with advanced solid cancers (like stomach, colon, or pancreatic cancer) that have not responded to standard treatments. Patients first receive chemotherapy to prep…

This study tests a smartphone-based system called AIDANET that works with an insulin pump and continuous glucose monitor to automatically adjust insulin for children aged 6-13 with type 1 diabetes. The goal is to see if the system is safe and works as well as usual care in keepin…

This study tests a new insulin infusion set designed to last up to 7 days for people with type 1 diabetes. Twenty adults currently using a Tandem insulin pump and Dexcom CGM will try the SteadiSet at home. The goal is to see if the set can survive a full week without causing high…

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

This study tests a nurse-led program that uses home monitoring devices to help older adults with conditions like heart failure, diabetes, and depression manage their health after leaving the hospital. The goal is to improve care coordination and reduce hospital readmissions. Abou…

This study tests whether a church-based program led by community health workers can help Black adults in Louisiana lower their risk of heart disease, high blood pressure, diabetes, and high cholesterol. About 812 participants from local churches will receive either a health worke…

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

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

This study compares a new, less invasive shockwave device to standard surgery for treating severe calcium buildup in the main leg artery. The shockwave uses sound waves to break up the calcium, while surgery involves physically removing the blockage. Researchers want to see if th…

This study looks at whether offering financial rewards, along with nurse coaching and home monitoring, helps adults with type 2 diabetes improve their blood sugar control. About 450 participants will be randomly assigned to receive either nurse coaching and home monitoring alone,…

This study tests an experimental injection called HRS-7249 in 224 adults with hyperlipidemia (high fat levels in the blood). The goal is to see if it safely lowers triglycerides, a type of blood fat linked to heart disease. Participants receive either the drug or a placebo, and r…

This study tests a stem cell transplant from a matched unrelated donor for people with advanced blood cancers or blood disorders. Patients get chemotherapy and radiation before the transplant to prepare their body, then take drugs to prevent the donor cells from attacking their o…

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

This study follows 11 adults with late-onset OTC deficiency who received a single dose of gene therapy (DTX301) in an earlier trial. Researchers are checking long-term safety and how well the body makes urea and controls ammonia levels. The goal is to see if the treatment remains…

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

This study tests whether the drug farcimab (Vabysmo), given as an injection before and after eye surgery, can reduce bleeding complications in people with diabetes who have bleeding inside the eye. About 100 adults will receive either farcimab or a sham injection. The goal is to …

This study follows 150 people with long-chain fatty acid oxidation disorders (LC-FAOD) to check the long-term safety of their treatment, including for pregnant women and their babies. Researchers track serious side effects and disease complications. The goal is to better understa…

This study compares two methods of tracking blood sugar in people with type 2 diabetes: traditional fingerstick testing and continuous glucose monitors (CGMs). About 360 adults aged 18 to 75 who use insulin will be followed for 12 months to see which method better lowers A1C leve…

This study looks at how well the Joslin Why WAIT? program helps people with type 2 diabetes and obesity lose weight and improve their health. About 110 participants who have a BMI over 35 are included. The main goal is to see if they can lose at least 7% of their body weight and …

This study tests whether a two-drug combination can help the immune system attack ovarian cancer that has returned after standard treatments. The drugs are E7777, which removes certain immune cells that block the body's defenses, and pembrolizumab, which reactivates cancer-fighti…

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

This study tests a new drug called CRB-913 for weight loss in people with obesity. It involves 252 participants and has two parts: first, measuring how the drug works in healthy adults, then comparing three doses against a placebo over 12 weeks. The goal is to check safety and ho…

This study looks at whether taking a yerba mate extract can help improve health markers in overweight adults. Eighty people aged 40-65 will take either the extract or a placebo for 90 days. Researchers will measure changes in weight, blood sugar, cholesterol, and inflammation to …

This study tests whether adding two extra medications (semaglutide and dapagliflozin) to insulin helps adults with type 1 diabetes control their blood sugar better than insulin alone or with just one extra drug. The 78 participants, aged 18-70 with a BMI of 25 or higher, will be …

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

This study tests a new drug called ALXN1850 in children aged 2 to 12 with hypophosphatasia, a rare genetic condition that weakens bones. The trial compares the drug to a placebo to see if it improves bone health and movement. About 30 children who have not received prior treatmen…

This pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…

This expanded access program offers the experimental drug zervimesine (CT1812) to people aged 50 to 86 with mild-to-moderate dementia with Lewy bodies. Participants take one 100 mg pill daily for up to a year. The goal is to provide access to the drug and collect long-term safety…

This study tests two oral drugs, pirtobrutinib and venetoclax, in 46 people with Waldenström macroglobulinemia who have already had at least one prior treatment. The goal is to see if the combination can shrink the cancer and improve response rates. Researchers are also tracking …

This early-stage study tests a new drug called EYE201, given as three eye injections over 12 weeks, to see if it is safe and can help reduce swelling in the back of the eye. It includes people with three different eye conditions that cause vision loss: branch retinal vein occlusi…

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

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

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

This study tests a health support program for 600 people with disabilities who also have chronic conditions like diabetes or obesity. The program includes weekly nutrition classes, three exercise sessions per week, and help managing chronic conditions. Researchers want to see if …

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

This study tests a drug called faricimab (VABYSMO) in 218 people with diabetic macular edema who have not been treated before. The participants are Black/African American, Hispanic/Latino American, Native American/Alaska Native/Native Hawaiian/Pacific Islander, or Asian Indian. T…

This study tests a daily pill called resmetirom in people with non-alcoholic fatty liver disease (NAFLD). The goal is to see if it safely reduces liver fat and cholesterol over 52 weeks. About 810 adults who completed earlier related studies are taking part. This is a long-term t…

This study tests a new eye injection called IBI302 for people with diabetic macular edema, a condition that causes vision loss due to diabetes. About 150 adults aged 18-80 with type 1 or 2 diabetes and swelling in the center of the retina will receive either IBI302 or an active c…

This study looks at whether 10 weeks of regular exercise (on land or in water) can lower blood sugar and blood pressure in people aged 60 and older who have several health conditions like diabetes, high blood pressure, and arthritis. 63 volunteers will be split into two exercise …

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

This study tests whether a daily injection of liraglutide (3.0 mg) can help children aged 6 to 12 with obesity lose weight. Over 56 weeks, 78 children will receive either liraglutide or a placebo, along with counseling on diet and exercise. The main goal is to see how much their …

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

This early-phase trial tests the drug phentermine/topiramate (Qsymia) in 13 adolescents aged 12-20 who have both obesity and type 2 diabetes. The drug is already approved for weight loss in adults. The study aims to see if it can safely lower body mass index (BMI) and improve blo…

This trial tests a 12-month lifestyle medicine program for 1000 adults with obesity and related conditions like diabetes or high blood pressure. Participants get regular visits with a dietitian and a doctor, plus optional behavioral health support. The goal is to see if the progr…

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

This study follows about 50 people with a rare immune disorder called ADA-SCID who have received a gene therapy treatment called Strimvelis. The goal is to track their health for many years to see if the treatment remains safe and effective. Researchers will monitor for side effe…

This study looks at whether switching to a plant-based diet can improve heart health and reduce inflammation in people who have had a kidney transplant. Twenty-five participants who are at least three months post-transplant will follow a plant-based diet for 16 weeks after a two-…

This study compares continuous glucose monitoring using the FreeStyle Libre 3 sensor to standard finger-stick testing in 470 adults with type 2 diabetes whose blood sugar is not well controlled. Participants continue their usual medications (basal insulin plus SGLT2 inhibitor and…

This study tests a special magnet system that creates a new connection between the stomach and intestine without traditional surgery. It is designed for adults with obesity (BMI 30-50) who also have type 2 diabetes or have regained weight after a previous sleeve gastrectomy. The …

This study tests whether a short course of two drugs, dasatinib and quercetin, can clear senescent (aging) cells in people with chronic kidney disease. The goal is to see if this reduces frailty and improves stem cell function. Thirty participants aged 40-80 with moderate-to-seve…

This study tests a new magnetic device (MagDI System) that creates a connection between the duodenum and ileum, bypassing part of the small intestine. The goal is to help with weight loss and blood sugar control in 60 adults with obesity and type 2 diabetes. Researchers are check…

This study tests a new magnetic system (MagDI) that creates a connection between two parts of the small intestine to help with weight loss and blood sugar control. It involves 25 adults with obesity and type 2 diabetes. The goal is to see if the device is safe, works as intended,…

This study tests a drug called loncastuximab tesirine in 21 people with Waldenström macroglobulinemia, a rare blood cancer, whose disease has not responded to at least two prior treatments. The goal is to see if the drug can shrink tumors and lower abnormal protein levels. Partic…

This study looks at different methods to help patients with high cholesterol and heart disease get the right medicines to lower their cholesterol. About 300 adults at high risk will take part. The goal is to see which approach helps more people reach their target LDL cholesterol …

This study tests a new medicine, crovalimab, for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease that destroys red blood cells. It involves healthy volunteers and people with PNH to check safety and how well the drug works. The goal is to control the disease, not …

This study tests whether eating dried Saskatoon berries daily for 10 weeks can improve blood sugar and cholesterol levels in people with untreated prediabetes or new type 2 diabetes. Researchers will compare the effects of Saskatoon berries to a control dried fruit in 92 adults a…

This study tests whether whole-body electromyostimulation (WB-EMS), a technology that stimulates muscles with mild electrical pulses, can lower resting blood pressure and improve other heart risk factors in overweight adults aged 50+ with mild hypertension. Over 12 weeks, 28 part…

This large study compares two popular diabetes drugs, tirzepatide and semaglutide, in 26,000 people who have both type 2 diabetes and a type of heart failure. Researchers will track who ends up in the hospital for heart failure or dies, to see if one drug works better. The goal i…

This early-stage trial is testing the safety and potential benefit of pembrolizumab, an immunotherapy drug, in 71 children whose brain tumors (like DIPG, high-grade gliomas, or medulloblastoma) have come back or not responded to standard treatments. The drug works by helping the …

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

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

This study tests whether a probiotic called Limosilactobacillus fermentum CRL 1446 can improve health in 80 adults with heart disease risk factors like obesity, high blood pressure, and high cholesterol. Participants take either the probiotic or a placebo daily, and researchers m…

This study tests a new drug called AR882, taken with a standard gout medication (XOI), in 15 people with severe, uncontrolled gout who have already failed uricase therapy. The goal is to see if the combination can lower uric acid levels and shrink painful uric acid crystal deposi…

This study tests whether adding an SGLT2 inhibitor to standard ARB therapy better reduces protein in the urine (a sign of kidney damage) in diabetic patients without high blood pressure. Sixty adults aged 30-60 with diabetes and microalbuminuria will receive either ARBs alone or …

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

This study tests whether adding culturally tailored heart-health education to medically tailored meal programs helps people with type 2 diabetes and high blood pressure eat better and improve their blood sugar and blood pressure. About 176 adults from low-income New York City nei…

This study looks at the best way to control blood sugar in pregnant women with diabetes who receive steroids to help their baby's lungs develop before a possible early birth. About 120 women will be randomly assigned to one of three insulin strategies. The goal is to find which a…

This study was designed to see if placing a stent in a blocked leg vein could help heal diabetic foot ulcers in people with diabetes and leg swelling. Patients would have received standard wound care plus either a stent or no stent. However, the trial was withdrawn before any par…

This early-stage trial is testing a new oral drug called ZE63-0302 in 60 adults with type 2 diabetes. The main goal is to check the drug's safety and how the body handles it, not yet whether it works. Participants will take either the drug or a placebo capsule twice a day for a s…

This phase 3 trial tests tirzepatide, a once-weekly injection, in 905 adults with type 1 diabetes who are also overweight or obese. Participants will receive either tirzepatide or a placebo for about 49 weeks. The study measures changes in blood sugar control (HbA1c) and body wei…

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

This phase 3 trial tests crovalimab in about 50 people with PNH who have not used similar drugs before. PNH causes red blood cells to break apart, leading to fatigue, pain, and need for transfusions. Crovalimab is given as an IV infusion and then as injections under the skin to b…

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

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

This study tests a combination of two targeted drugs, ibrutinib and venetoclax, in 45 adults with a rare blood cancer called Waldenström macroglobulinemia who have a specific gene mutation (MYD88). The goal is to see if the combo can shrink the cancer significantly. Participants …

This study looks at whether a special heart-friendly diet, with or without added plant sterols and krill oil, can improve cholesterol levels in people with familial hypercholesterolemia (a genetic condition causing very high cholesterol). About 300 participants will follow the di…

This study tests an experimental oral drug called AZD0780 in 432 adults with high LDL cholesterol (dyslipidaemia). Participants take the drug or a placebo daily for up to 52 weeks to see if it safely lowers cholesterol. The goal is to manage the condition, not cure it, as ongoing…

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

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

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

This study tests a new medicine called HM15136 for people aged 2 and older with congenital hyperinsulinism (CHI) who still have low blood sugar despite standard treatments. The medicine is given as a weekly shot for 8 weeks. The goal is to see if it is safe and helps control bloo…

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

This study tests whether a culturally tailored storytelling video, shown on a tablet, can help 200 gout patients—especially African Americans in the Southeast—better understand their condition and stick with long-term treatments like medication, diet, and exercise. The goal is to…

This study tests a 90-day fully virtual lifestyle program for adults aged 30-60 who are sedentary and at risk for heart disease. Participants exercise via Peloton, take daily supplements (Omega-3, Vitamin D3, Magnesium, fiber), and use wearable devices to track activity and sleep…

This study looks at whether remotely monitoring continuous glucose monitor (CGM) data can improve diabetes management in children within the first year after diagnosis. About 100 kids and teens newly diagnosed with type 1 diabetes will have their CGM data shared with their care t…

This Phase 2 trial tests whether tirzepatide, a drug already used for diabetes and weight loss, can improve kidney health in 140 people with obesity and chronic kidney disease (with or without type 2 diabetes). Researchers will use MRI scans to measure changes in kidney fat and b…

This study compares a new medicine, ALXN1850, to the current standard treatment (asfotase alfa) in 43 children aged 2 to 12 with hypophosphatasia, a rare genetic condition that weakens bones. All children have been on the standard treatment for at least 6 months before joining. T…

This study is for people with systemic amyloidosis, a disease where abnormal proteins build up in organs. It tests the long-term safety of an experimental drug called AT-02. About 120 adults will receive the drug and be monitored for side effects and how the drug behaves in the b…

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

This study tests an experimental drug called AZD0780 in 473 adults with a genetic condition that causes very high cholesterol (heterozygous familial hypercholesterolemia). The drug aims to lower LDL (bad) cholesterol more than a placebo over 12 weeks, with treatment lasting up to…

This study looks at whether eating almonds every day for 12 weeks can improve gut health and lower inflammation in people with metabolic syndrome. About 80 adults aged 35-60 with conditions like high blood pressure, high blood sugar, or unhealthy cholesterol levels will participa…

This study tests a 6-month home-based program called FIT Families, designed to help African American teens with obesity and their caregivers lose weight. The program includes coaching sessions, family counseling, and small prizes for completing tasks. Researchers will measure cha…

This study tests a device that delivers specific light and sound patterns to the brain, aiming to slow the progression of mild-to-moderate Alzheimer's disease. About 670 participants will use the device at home for an hour daily over a year, with half receiving a sham (inactive) …

This pilot study explores how a 6-month low-carbohydrate diet affects blood sugar control, body composition, and gut-brain interactions in 40 obese adults with or without glucose intolerance or diabetes. Participants follow a low-carb eating plan, and researchers measure changes …

This early-stage trial tests a single oral dose of BMN 349 in 6 adults with a genetic form of alpha-1 antitrypsin deficiency (PiZZ or PiMZ/MASH). The main goal is to check safety and how the drug moves through the body. Participants receive either BMN 349 or a placebo and are clo…

This study looks at how safe and effective Ozempic is for adults with type 2 diabetes when used in normal doctor visits. About 600 people in Korea will take Ozempic as their doctor prescribes, and the study lasts around 26 weeks. The main goal is to track any side effects and ser…

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

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

This study tests whether semaglutide, a drug used for diabetes, can reduce harmful belly fat and improve heart health in people with HIV who have excess fat around the midsection (lipohypertrophy). About 108 participants will receive either semaglutide or a placebo for 32 weeks. …

This study tests a one-time gene therapy called GS-100 in 10 children aged 2 to 18 with NGLY1 deficiency, a rare genetic disorder. The therapy is given directly into the brain fluid to deliver a working copy of the missing gene. The goal is to improve motor skills and development…

This study is testing the long-term safety of a daily oral drug called zagociguat in 44 adults with MELAS, a rare genetic disease that affects energy production in cells. All participants previously completed a lead-in study of the same drug. Researchers will monitor side effects…

This phase 3 trial tests VSA003, an RNAi drug that targets a protein called ANGPTL3, in 46 Chinese adolescents and adults with homozygous familial hypercholesterolemia (HoFH), a genetic condition causing extremely high cholesterol. Participants receive either VSA003 or a placebo …

This study tests whether a text-message based mobile health program can help people with type 2 diabetes in Nepal better manage their condition. 154 adults with type 2 diabetes will be randomly assigned to receive either the text-message program plus standard care, or standard ca…

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study tests an AI tool called AIDP that analyzes MRI scans to help diagnose Parkinson's disease and two similar conditions (MSA and PSP). Researchers at 21 sites will compare the AI's diagnosis to that of movement disorder specialists. The goal is to see if the tool can impr…

This study uses special brain scans (PET imaging) to measure amyloid buildup—a protein linked to Alzheimer's—in healthy older adults, people with mild memory problems, and those with dementia. Participants receive scans every 2-3 years for up to 20 years to see how amyloid change…

This study tests a new device called LabPatch that measures blood sugar from fluid just under the skin. About 60 adults with type 1 or type 2 diabetes who use insulin will wear the patch for 6 hours. Researchers will compare its readings to standard lab tests and two commercial g…

This study is testing whether a continuous glucose monitor (CGM) worn on the arm for 14 days can replace the standard oral glucose tolerance test (OGTT) for women who had gestational diabetes. Many women find the OGTT burdensome and skip it, missing a chance to catch lingering bl…

This study compares a new ultra-high resolution PET/CT scanner, the NeuroEXPLORER, with standard clinical PET/CT scanners for imaging the head and neck. Researchers aim to see if the new device provides more detailed images to improve diagnosis of conditions like dementia, Parkin…

This study follows 300 children aged 9 to 14 with obesity to see if ultrasound and MRI can detect early signs of liver disease. Researchers will compare imaging markers like liver fat and stiffness with standard tests. The goal is to improve diagnosis and monitoring without invas…

This study tests a drug called triheptanoin in 8 adults with MCADD, a rare condition that can cause dangerously low blood sugar. The goal is to see if the drug is safe and can prevent hypoglycemia during fasting. Participants will stay overnight at a hospital for monitoring and b…

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

This trial investigates whether a daily effervescent supplement containing calcium, magnesium, and citrate can prevent bone loss, magnesium deficiency, and kidney damage in adults who take proton pump inhibitors (PPIs) long-term for conditions like heartburn or GERD. Participants…

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

This study tests whether fenofibrate, a drug already used for high cholesterol, can prevent diabetic retinopathy from worsening in people with mild to moderate disease. About 560 adults with diabetes will take either fenofibrate or a placebo for up to 6 years. The goal is to see …

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

This study tests whether providing fresh meals, health coaching, and education to high-risk pregnant women can reduce the chance of developing gestational diabetes, high blood pressure, or preeclampsia. 69 women in Milwaukee are randomly assigned to either receive the program or …

This trial tests an online program to help women who had gestational diabetes get the recommended diabetes screening and lifestyle programs after giving birth. The program includes standard health information plus optional interactive components like values reflection and persona…

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

This study looks at whether pramipexole, a drug used for restless leg syndrome, can ease leg symptoms in women with X-linked adrenoleukodystrophy (ALD). About 24 women will take the drug or a placebo for two months. The goal is to see if it improves sleep, walking, and quality of…

This study tests whether a self-affirmation writing exercise—where people write about their core values—before routine diabetes checkups can reduce feelings of stigma and improve diabetes management. About 150 adults with type 2 diabetes who already use continuous glucose monitor…

This study tests a mobile health program called R2D2 designed to reduce diabetes distress in children aged 8-12 with type 1 diabetes and their parents. The program uses cognitive behavioral therapy, mindfulness, and behavioral strategies to help families cope. Researchers will me…

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

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

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

This study tests whether cognitive behavioral therapy (a type of talking therapy) delivered online, along with a continuous glucose monitor, can reduce diabetes-related distress in adults with type 1 diabetes. About 93 adults aged 18-64 will be randomly assigned to receive either…

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

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

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

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

This study tests a culturally tailored counseling and support program for Chinese and Korean American caregivers of relatives with dementia. The program includes family counseling sessions and online peer support groups. Researchers will measure whether it reduces stress, depress…

This study tests a new way to match dementia caregivers with each other for emotional support using a computer algorithm. Researchers want to see if matching based on personal preferences leads to better satisfaction and less stress than random matching. About 355 caregivers of p…

This early-stage study tests a single dose of a new antibody injection in 64 people. Part A includes healthy volunteers to check safety, and Part B includes people with an acute gout flare. The main goal is to see if the drug is safe and how the body processes it.

This study tests a smartphone app called PACTAID in 20 adults with type 1 diabetes. The app gives real-time advice during exercise to help keep blood sugar levels stable while using an automated insulin pump. Researchers will check if the app improves glucose control during and a…

This phase 2 trial tests a special hand and foot mask designed to ease nerve pain caused by chemotherapy or diabetes. Sixty adults will apply the mask daily for 5 minutes over one week. Researchers will measure changes in symptoms and quality of life using questionnaires.

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

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

This study looks at whether teenagers and young adults with Polycystic Ovary Syndrome (PCOS) have more fat in their liver than those without PCOS. Researchers will use advanced imaging and tests to measure liver fat, insulin resistance, and cholesterol problems in 80 participants…

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

This study looks at whether vitamin C can increase body heat produced by the nervous system and boost vitamin D levels in the blood. Researchers will measure temperature and blood samples in 20 overweight adults aged 18-40 during a stimulation test. The goal is to understand how …

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

This observational study follows 150,000 people with chronic kidney disease and type 2 diabetes to see how finerenone (Kerendia) works in everyday medical care. Researchers will compare those taking finerenone to those not taking it, looking at rates of heart attacks and heart fa…

This registry will follow 500 people with type 2 diabetes who have already been treated with the Endogenex System. The goal is to collect long-term safety data and see how the device performs in everyday use. Participants must agree to share their medical records and attend follo…

This study follows over 4,000 women who were tracked during their first pregnancy to see how pregnancy complications like high blood pressure or diabetes relate to future heart disease. Researchers will check for conditions like high blood pressure, diabetes, and obesity over tim…

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

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

This study follows 35 children with CLN2 disease (a rare, severe brain disorder) who are taking cerliponase alfa (Brineura). Researchers will monitor for side effects and serious reactions over time. The goal is to better understand the long-term safety of this enzyme replacement…

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

This observational study will follow 2,500 adults with vision loss from nAMD or DME who are already prescribed aflibercept 8 mg by their doctors. Researchers will collect data from routine visits over up to 24 months to see how vision changes. The goal is to understand how well t…

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

This study tests whether offering online educational videos and mailed saliva genetic testing kits helps more first-degree relatives of people with BRCA mutations get tested, compared to standard care with a family letter. About 820 participants will be enrolled across several me…

This study examines health data from about 800,000 Korean adults to see how often conditions like obesity, type 2 diabetes, heart disease, chronic kidney disease, and fatty liver occur together. Researchers will analyze national survey and insurance records from 2013 to 2023. No …

This study compares two ways of delivering a health and wellness program to people with chronic conditions like diabetes and high blood pressure. One group gets a kit with a tablet and home monitors for blood pressure and blood sugar; the other gets standard phone calls. The goal…

This observational study follows over 4,600 adults with chronic kidney disease and type 2 diabetes who are starting finerenone, a drug that helps slow kidney damage. Researchers will collect data from routine doctor visits over 12 months to understand why doctors prescribe it, ho…

This early-stage trial in 48 healthy volunteers checks whether a new drug called elecoglipron changes how the body processes two common statins (atorvastatin and rosuvastatin). Participants take the statin alone and later with elecoglipron, and researchers measure drug levels in …

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

This study aims to improve how doctors test for autonomic failure, a condition where the body's automatic functions like blood pressure control don't work properly. Researchers will use advanced imaging, sweat tests, and skin biopsies in up to 89 adults with Parkinson's disease, …

This study is a global registry that will follow about 500 people with pyruvate kinase (PK) deficiency, a rare inherited anemia, for up to 9 years. Researchers will collect medical data to better understand the disease's natural history, treatments, and complications. No new drug…

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

This study uses healthcare claims data from nearly 40,000 people to see if the results of a major clinical trial (EMPEROR-Preserved) can be reproduced in a real-world setting. Researchers are comparing two diabetes drugs, empagliflozin and sitagliptin, in patients with heart fail…

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

This study aimed to see if a personalized exercise app (Gingko Active) could improve physical function in adults aged 45 and older better than a standard exercise booklet. The trial was withdrawn before any participants enrolled, so no results are available. The app was designed …

This early-stage trial tests a new drug called AJU-C716 against a reference drug (C716R) in 41 healthy adults aged 19 to 55. The goal is to see how safe the drug is and how the body processes it. This study does not test if the drug works for high cholesterol—it only looks at saf…

This study looks at American Indians with type 2 diabetes who had early kidney disease and took a drug called losartan years ago. Researchers want to see if the benefits of that treatment still help protect their kidneys today. About 141 people who were in the earlier study or th…

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

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

This study surveys 500 doctors in Egypt to see how well they understand prediabetes—a condition where blood sugar is high but not yet diabetic. Doctors answer questions about risk factors, diagnostic tests, and lifestyle recommendations. The goal is to find gaps in knowledge that…

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

This study tracks 585 patients with familial or regular high cholesterol who are already prescribed Leqvio. Researchers will monitor side effects and cholesterol levels over time to see how safe and effective the drug is in everyday use. No new treatment is given—it's purely obse…

This study looks at whether taking short walks throughout the day is better for kids at risk for type 2 diabetes than one longer exercise session. Researchers will measure blood sugar, insulin, and thinking skills in 188 children aged 8-11 with overweight or obesity. The goal is …

This study reviews medical records of 1200 Indian adults with chronic kidney disease and type 2 diabetes who started finerenone between August 2022 and April 2024. Researchers will analyze how doctors prescribed the drug, dose changes, and lab results like kidney function and pot…

This study looks at whether two common artificial sweeteners, saccharin and acesulfame potassium, affect blood sugar control in older adults with prediabetes. Researchers will monitor 30 participants over 8 weeks using continuous glucose monitors and oral glucose tolerance tests.…

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

This study looks at health insurance claims from about 30,000 people with type 2 diabetes to see if real-world data can produce similar results to a large clinical trial called REWIND. The goal is to learn when and how to trust real-world data for answering medical questions. No …

This study looks at how gastric bypass surgery changes the way the small intestine processes sugar and other fuels, which may explain why the surgery improves obesity and type 2 diabetes. Researchers will collect intestinal tissue and blood samples from 32 people before and up to…

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

This study uses healthcare claims data from nearly 24,000 people to see if it can reproduce the results of the EMPEROR-Reduced trial, which tested the diabetes drug empagliflozin against sitagliptin in heart failure patients. The goal is not to test a new treatment, but to learn …

This study is creating a collection of blood samples and health information from 250 adults with very high or very low cholesterol or related lipid problems. Researchers will analyze these samples to learn more about the genetic and biological causes of these extreme lipid disord…

This pilot study looks at how environmental stress affects daily blood sugar changes in 30 Hispanic adults whose parent has type 2 diabetes. Researchers use continuous glucose monitors to track glucose and aim to design a future program to prevent diabetes. The study does not tes…

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

This study looks at whether the adrenaline (epinephrine) commonly added to local anesthesia for tooth extraction raises blood sugar levels. Researchers will measure blood glucose before, during, and after the procedure in 90 adults—half with type 2 diabetes and half without. The …

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

This study follows up to 90 children and young adults with mitochondrial liver diseases to learn how these conditions progress over time. Researchers will collect medical data and samples to better understand the diseases and find markers that predict outcomes. The goal is to imp…

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

This study tests different ways to invite adults with diabetes who struggle to take their medications as prescribed. Researchers will compare letters and phone call strategies to see which approach gets more people to schedule and attend a pharmacist appointment. The goal is to f…

This pilot study tests whether an enhanced telemedicine program is practical for adults with complex type 2 diabetes who use insulin and have other health conditions. Fifty participants will receive either standard telemedicine care or an enhanced version with extra pre-visit pre…

This study looks at how the liver burns fat and makes sugar in people with NASH (a serious liver disease linked to obesity) who are planning to have weight-loss surgery. Researchers will use special scans to measure liver function before and after surgery in 30 adults. The goal i…

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

This study followed children and young adults with genetic liver diseases that cause bile buildup. The goal was to track how these diseases progress over time, including the need for liver transplants or other complications. No treatments were tested; the aim was simply to learn …

This observational study follows about 198 patients in Spain who are already taking inclisiran (Leqvio) for high cholesterol linked to heart disease or a genetic condition. Researchers will check if patients reach their LDL cholesterol goals after 6 and 12 months. The goal is to …

This study follows 600 people with Albright hereditary osteodystrophy, a rare genetic condition affecting bones and hormones. Researchers are looking at how growth hormone treatment affects height and weight in those with a related hormone problem, and also studying thinking and …

This study looks at how the menstrual cycle changes blood sugar and energy use in women with type 1 diabetes. About 40 women will use a closed-loop insulin pump and continuous glucose monitor to track these changes. The goal is to understand how hormone shifts affect insulin need…

This study looks at how canagliflozin (Invokana), a diabetes drug, protects the kidneys in people with type 2 diabetes. Researchers will use advanced imaging, kidney biopsies, and function tests to uncover the mechanisms. The trial involves 45 adults and aims to explain the drug'…

Citrin deficiency is a rare, underdiagnosed condition that affects the liver and metabolism. There is no cure, and patients manage symptoms with lifelong diet changes. This study analyzes blood samples from 100 patients to find biomarkers that could help track disease severity an…

This study tests a web-based tool called gp-multitool.de that helps general practitioners follow guidelines for older patients with multiple chronic conditions. About 659 patients from GP practices in Germany are taking part. The tool lets patients fill out questionnaires online …

This study looks at ways to help more adults with type 2 diabetes get screened for colorectal cancer. Researchers are testing a program that works with patients, their care teams, and clinics to make screening easier. The goal is to see if this approach increases the number of pe…

This study examines how common cardiac autonomic neuropathy (nerve damage affecting the heart) is in children and teens with type 1 diabetes. Researchers will monitor 75 participants using heart tests and check if longer diabetes duration or poor blood sugar control raises the ri…

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

This study looks at how often people with type 1 diabetes have fatty liver disease and serious liver scarring. Researchers used a special ultrasound and blood tests to check 533 adults. The goal is to find high-risk patients who need a liver specialist.

This study will collect and analyze genetic data from 1000 people with suspected inherited metabolic diseases, including conditions like epilepsy and mitochondrial disorders. Researchers at Karolinska University Hospital aim to improve diagnosis by using advanced genetic testing …

This study looks at how a measure called the hemoglobin glycation index (HGI) changes over time in 750 adults with diabetes who use continuous glucose monitors. HGI compares lab-measured blood sugar levels with estimated levels from a glucose monitor. Researchers will collect dat…

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

This study looks at whether removing overactive parathyroid glands (parathyroidectomy) can improve blood sugar control in people who also have type 2 diabetes. Researchers will compare 80 patients who have surgery with those who do not, measuring changes in blood sugar and bone h…

This observational study follows 782 people with X-linked hypophosphatemia (XLH), a rare genetic bone disease, to understand how the condition changes over time. It also monitors the long-term safety and effectiveness of the drug burosumab. No new treatment is given; participants…

This study looks at how aging changes the way older adults respond to exercise. Researchers will have 26 people who previously completed a structured exercise program do a 6-month aerobic workout. They will measure changes in insulin sensitivity and heart fitness to see if age af…

This study uses special PET/CT scans to measure inflammation in the brains of people with Alzheimer's disease or Lewy body dementia. Researchers want to see if brain inflammation is linked to memory decline and other dementia symptoms. The study also tests a new radioactive trace…

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

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

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

This study looks at whether drinking a beverage with artificial sweeteners (erythritol or xylitol) changes how blood platelets clump together, which could affect heart attack or stroke risk. Researchers will measure platelet activity and blood levels of these sweeteners in 50 hea…

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

This study uses anonymous health insurance records from Germany to learn more about a rare heart condition called ATTR-CM, where abnormal protein builds up in the heart. Researchers will look at survival rates, hospital visits, and how common the disease is. No new treatments are…

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

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

This observational study is looking at a protein called neurofilament light chain (NfL) in the blood of people who carry a gene variant for hereditary ATTR amyloidosis, both those without symptoms and those with nerve damage. Researchers want to see if NfL levels can help detect …

This study looks at how long you need to fast and how stopping the diabetes/weight-loss drug tirzepatide changes the amount of food left in your stomach after a meal. Researchers will use ultrasound to check stomach contents in 40 people with type 2 diabetes or obesity. The goal …

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

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

This study is testing whether people with cancer or diabetes can use a Fitbit and a continuous glucose monitor (CGM) to track their daily exercise and glucose levels. The goal is to see if it's practical and if people will stick with it. Thirty-five participants will wear both de…

This study looks at whether adults aged 70 and older with multiple chronic conditions (but no heart disease) can safely stop taking statins. Researchers are comparing those who continue statins to those who stop, tracking deaths and major heart events over 2-4 years. The goal is …

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

This study looks at whether a program called I-SITE can help rural health clinics use teleophthalmology (remote eye exams) to screen more diabetic patients for eye problems. Up to 10 health systems and 10,000 patients will take part. The goal is to see if screening rates improve …

This study looks at health records from over 780,000 people with type 2 diabetes to compare four common second-line medications: SGLT2 inhibitors, GLP-1 receptor agonists, DPP-4 inhibitors, and sulfonylureas. The goal is to see which drug class is most effective at preventing hea…

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

This Phase 1 trial tests two experimental drugs (L03RD1 and L03RD2) in 48 healthy adults to see how safe they are and how the body processes them. Participants take the drugs alone or together in a single dose. The study does not aim to treat diabetes, but to gather information f…

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

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

This early-stage study in 32 healthy adults looks at how a new drug called AZD5004 changes the way the body processes two diabetes medications, mitiglinide and pioglitazone. The goal is to understand potential drug interactions, not to treat any disease. Participants take the dru…

This study follows 78 adults in Taiwan who have Fabry disease and carry a specific genetic mutation called GLA IVS4. Researchers are looking at how the disease affects the heart and other organs over time, both in people who have never been treated and those who have received aga…

This study looks at how skin temperature and blood flow change in people with a leg amputation, especially those with diabetes. Researchers will test two types of prosthetic legs—rigid and shock-absorbing—to see which one keeps the skin cooler and healthier. Sixty participants wi…

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

This study tests whether a tablet-based decision support system helps frontline health workers in India and Nepal provide better antenatal care. About 1,320 pregnant women will take part. The tool guides workers to check blood pressure, blood sugar, urine protein, and hemoglobin,…

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

This study follows 1,000 patients in China who are already receiving faricimab for diabetic macular edema, retinal vein occlusion, or wet age-related macular degeneration. Researchers will collect data from routine clinic visits over one year to see how well the drug works and ho…

This study aims to find new biological markers (biomarkers) that can show how active acute porphyria is in the body and how well treatments are working. Researchers will study 50 people with confirmed acute porphyria to measure gene activity related to heme production and stress …

This study follows 225 pregnant women—some who had gastric bypass or sleeve gastrectomy, and some without surgery—to see how blood sugar changes affect fetal growth. Participants wear a continuous glucose monitor and activity tracker for 10 days each trimester. The goal is to lea…

This observational study examines how aflibercept 8 mg, an injection into the eye, is used in routine care for patients in Japan with age-related macular degeneration or diabetic macular edema. Researchers will analyze claims data from about 3000 patients to understand patient ch…

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

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

This study surveys 30 adults with Wilson disease to learn about their daily management, treatment adherence, and interest in gene editing clinical trials. By gathering this information, researchers hope to design better future studies for potential gene therapies targeting specif…

This study looks at whether routine blood sugar testing during dental checkups can help find hidden high blood sugar in people with advanced gum disease. About 300 adults with severe gum disease will get a simple finger-prick blood test during their dental visit. The goal is to s…

This study analyzes de-identified lipid test results from over 5 million patients to better understand cholesterol and related blood fats. Researchers aim to identify rare lipid disorders and improve how doctors personalize treatment for abnormal blood lipids. The data comes from…

This early-stage study looks at how healthy postmenopausal women absorb, break down, and eliminate the experimental drug GS1-144. Six participants will take a single oral dose, and researchers will measure the drug and its byproducts in blood, urine, and stool. The goal is purely…

This study explores whether artificial intelligence can detect stress in people with type 1 diabetes by analyzing their voice recordings. Participants record their voice four times a day for a period. The goal is to develop a tool that helps patients and doctors better understand…

This study uses artificial intelligence to predict when patients will fully recover from muscle relaxants given during surgery. The goal is to help doctors give the right dose at the right time, reducing the risk of breathing problems after surgery. The study will look at data fr…

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

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

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

This study tests a 10-week educational program called HomeStyles-China, designed to help Chinese American adults improve their diet, increase physical activity, and reduce risk factors for heart disease and diabetes. Researchers will enroll 400 participants and measure changes in…

This study looks at whether giving doctors a patient's genetic information can help them personalize chemotherapy doses and reduce serious side effects. About 860 adults with certain cancers (like breast, stomach, or head and neck) will be randomly assigned to have their doctors …

This study looks at how everyday stress impacts blood sugar levels in people with type 1 diabetes. Researchers will ask 125 participants to rate their stress four times a day using a smartphone app, while a continuous glucose monitor tracks their blood sugar. The goal is to see i…

This study looks at whether advanced monitoring devices can accurately measure blood thickness (hematocrit) and oxygen levels during continuous dialysis in critically ill patients with acute kidney injury or end-stage kidney failure. About 30 adults in the intensive care unit wil…

This study looks back at medical records of over 46,000 diabetic patients who had surgery in Alberta, Canada. Researchers want to see how often patients had a recent HbA1C test (a measure of long-term blood sugar control) before their operation. They also want to find out if poor…

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

This study tests whether a two-week plant-based Mediterranean diet can change gut bacteria and reduce inflammation in people who have had colon polyps removed. Thirty participants will eat high-fiber meals delivered to their homes, and researchers will track changes in blood and …

This study is testing a new type of clinic that screens people with prediabetes and type 2 diabetes for several hidden health problems at the same time, including fatty liver disease, sleep apnea, and heart failure. The goal is to see how common these undiagnosed conditions are a…

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

This study collects bone marrow and blood samples, along with medical history, from people with multiple myeloma, Waldenstrom macroglobulinemia, and related disorders. The samples are stored in a tissue bank for future research to understand what causes these cancers, how they pr…

This study looks at over 10,000 adults in Ecuador to understand why some people with diabetes develop kidney disease. Researchers will collect health surveys, blood and urine samples, and kidney tissue to study social, environmental, and biological factors. The goal is to find ne…

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

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