Inborn errors of metabolism

Researchers test a one-time gene therapy infusion in children and adults with Sanfilippo syndrome (MPS IIIB), a rare inherited disease that damages the brain and body. The therapy delivers a working copy of the NAGLU gene to help the body make an enzyme that is missing or faulty.…

This trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare inherited disease that damages the nervous system. Researchers take a patient's own blood stem cells, add a corrected gene, and infuse them back. The goal is to see if this approach is safe and can slo…

This trial tests a medical device that has not been approved or cleared by the U.S. FDA. Researchers want to learn whether the device is safe and whether it can help people with a specific condition. Participants receive the device as part of the study, and the team monitors thei…

This study tests a 6-month program where patients work with a pharmacist and their primary care doctor to make shared decisions about medications and lifestyle. The goal is to see if this team-based approach improves heart and kidney health scores and medication adherence. The st…

This phase 3 trial tests a drug called tralesinidase alfa in 14 children aged 1-5 with Sanfilippo syndrome type B, a rare genetic disease that causes severe brain damage. The drug is given directly into the brain fluid to replace a missing enzyme. The goal is to see if it can slo…

This Phase 3 trial will test whether a combination of three cholesterol-lowering drugs—bempedoic acid, ezetimibe, and a high-intensity statin—can reduce and stabilize plaque buildup in the coronary arteries. The study enrolls 103 adults with coronary atherosclerosis who have neve…

This early-phase trial tests a single injection of a gene therapy called GS1168 in 9 adults with PKU whose condition is not well controlled by diet or other treatments. The therapy uses a harmless virus to deliver a working copy of the PAH gene, which is faulty in PKU, to help th…

This study will compare three approaches before gastric bypass surgery for severe obesity: taking the medication tirzepatide for 60 days, following a ketogenic diet for 4 weeks, or receiving standard care. The goal is to see which method leads to the most weight loss one year aft…

This study tests a community-based exercise program for people aged 50 and older who have at least two chronic conditions, such as heart disease, diabetes, arthritis, or obesity. The program is designed to improve muscle strength, heart and lung fitness, and overall well-being. R…

Researchers are testing an experimental oral drug called OMS1620 in 84 healthy adults. The drug blocks the receptor that adrenocorticotrophic hormone (ACTH) uses to signal the adrenal glands. The trial measures whether single and repeated doses are safe, how the drug moves throug…

This study is for adolescents and young adults (ages 12-39) newly diagnosed with aggressive brain tumors called high-grade glioma or DIPG. The goal is to see if a combination of two immunotherapy drugs (relatlimab and nivolumab, sold as Opdualag) is safe and tolerable when given …

This 52-week trial tests whether combining a structured lifestyle program with a mushroom-based supplement and the drug bupropion helps overweight adults lose weight and lower cardiometabolic risk. About 250 adults aged 18 to 55 with a BMI between 25 and 35 will be randomly assig…

This trial tests an experimental oral drug called DES-1357 in adults with metastatic colorectal cancer that has specific genetic features (dMMR or MSI-H). The study runs in three parts: first, to find a safe dose; second, to check tolerability and early signs of tumor shrinkage; …

This study tests whether a 12-week program of supervised exercise, Mediterranean diet education, and stress management workshops can improve health in 120 overweight or obese adults. Participants will wear a smart bracelet to track activity and sleep. Researchers will measure cha…

This trial tests whether EDP167, an experimental RNA drug, can safely lower triglycerides and other harmful fats in the blood of adults with mixed dyslipidemia. Participants receive one of three doses of EDP167 or a placebo as an injection every 12 weeks, alongside their usual st…

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

This trial tests a gene therapy for X-linked adrenoleukodystrophy (X-ALD), a rare genetic disease that damages the brain. The therapy uses a modified virus to deliver a working copy of the faulty gene directly into the spinal fluid and bloodstream. Up to 30 patients aged 1 year a…

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

This phase 2 trial tests whether teclistamab can treat Waldenstrom's macroglobulinemia, a rare blood cancer, in people whose disease has returned or not responded to prior therapy. Participants receive teclistamab as a subcutaneous injection for up to 9 cycles. Researchers measur…

This early-stage trial tests giving a gene therapy to unborn babies diagnosed with GM1 gangliosidosis, a severe genetic disorder that damages the brain and body. The therapy uses a harmless virus to deliver a working copy of the missing enzyme gene. The main goal is to see if the…

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

This early-stage trial is testing an experimental oral medication called S-606001 in adults with late-onset Pompe disease, a condition that causes progressive muscle weakness and breathing problems. The main goal is to see if the drug is safe and tolerable. Researchers will also …

This trial is testing whether a monthly injection of lerodalcibep (Lerochol) can safely and effectively lower LDL cholesterol over three years in children and adolescents aged 6 to 17 with heterozygous familial hypercholesterolemia (HeFH), an inherited condition causing very high…

This trial tests whether the drug lucerastat can reduce the buildup of a fatty substance called Gb3 in the kidneys of adult men with Fabry disease who have not received prior treatment. Participants will take lucerastat daily for 18 months and undergo kidney biopsies to measure c…

This early-stage trial is testing an experimental pill called HS-10582 to see if it is safe and how it affects the body, especially LDL cholesterol. The study includes healthy adults and those with mildly elevated LDL cholesterol. Participants will receive either the drug or a pl…

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

This trial tests whether adding semaglutide—a medication used for diabetes and weight management—to standard antipsychotic treatment can lower the 10-year risk of heart attack and stroke in people with schizophrenia who also have metabolic syndrome. The study will measure changes…

This phase 3 trial is testing whether a radioactive drug called iopofosine I 131 can keep Waldenstrom macroglobulinemia (a rare blood cancer) from getting worse for longer than the standard combination of rituximab, cyclophosphamide, and dexamethasone (R-CD). The study enrolls ab…

This phase 2 trial is testing whether combining two immunotherapy drugs—vilastobart and retifanlimab—can shrink tumors in people with metastatic pancreatic cancer that has a BRCA1, BRCA2, or PALB2 gene mutation. Participants receive both drugs by IV infusion over several months. …

This trial tests whether icosapent ethyl, a purified form of fish oil, can stabilize high-risk coronary plaques in people with heart disease. Participants have plaques that narrow a major heart artery by 30–70% and show at least one high-risk feature on a CT scan. Half will take …

This study tests whether a traditional Chinese herb called Polygonatum sibiricum, or Huangjing, can help reduce visceral fat—the deep belly fat around organs that raises risks for diabetes, fatty liver, and heart disease. Adults with visceral obesity will receive either the herb …

This study tests the long-term safety and tolerability of acoramidis, an oral medication that stabilizes the transthyretin protein, in people newly diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition that can lead to heart failure. Participants who complete…

This study tests whether eating a daily serving of pecans for 12 weeks can improve cholesterol levels and other markers of heart-disease risk. Adults aged 25–75 who are overweight or have borderline-high cholesterol will either add pecans to their diet or stick with their usual e…

This trial tests whether different intermittent fasting schedules—5:2, alternate-day, and time-restricted eating—can improve blood sugar, cholesterol, inflammation, and body composition in adults with prediabetes. Participants will be randomly assigned to one of three fasting pla…

This study tests NeoMayor, a smartphone app that gives personalized advice on exercise, diet, sleep, and mood to improve heart health. Researchers will enroll 240 adults aged 55 to 75 in Chile who are at risk for heart problems. Participants use the app for 4 months, and some als…

This study tests the long-term safety and effectiveness of an experimental oral drug called S-606001 in adults with late-onset Pompe disease, a genetic condition that causes progressive muscle weakness. Participants who completed a prior S-606001 study can join, and they will rec…

This trial tests whether taking coenzyme Q10 (CoQ10) for 12 weeks can reduce fat buildup in the liver of people with mitochondrial diabetes, a rare form of diabetes caused by mitochondrial dysfunction. Participants with the m.3243A>G mutation will take 300 mg of CoQ10 daily, and …

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

This phase 2 trial is testing an oral drug called PLX-200 (gemfibrozil) in children aged 2 to 15 with certain lysosomal storage disorders (LSDs), including CLN2, CLN3, Sandhoff disease, and Krabbe disease. The study aims to see if the drug is safe, tolerable, and may slow the pro…

This study looks at whether diazoxide, a drug that reduces insulin release, can help children with type 1 glycogenosis maintain steadier blood sugar levels. Type 1 glycogenosis is a rare inherited condition where the body cannot properly release glucose from stored glycogen, lead…

This Phase 2 trial tests lisaftoclax, an oral drug that blocks a protein helping cancer cells survive. It enrolls 75 adults with certain slow-growing lymphomas (CLL, WM, or MZL) who either cannot take standard BTK inhibitors due to health issues or have stopped them due to side e…

This trial explores whether starting transdermal estradiol (a skin gel or patch) soon after menopause can protect the heart and brain in women who have hot flashes and at least one cardiovascular risk factor. One hundred women will be randomly assigned to receive the hormone ther…

This trial tests an experimental oral drug, TTI-0102, in people aged 5 to 55 with Leigh syndrome spectrum, a rare genetic disorder that damages the brain and nerves. The study aims to find the right dose and check safety over 12 weeks of twice-daily treatment. Participants will h…

This study looks at whether taking macaúba oil supplements while doing outdoor exercise can improve how the body burns fat and handles mental stress. Researchers will enroll 60 physically active adults aged 35 to 59 who are overweight but not obese. Participants will take either …

This study tests an experimental drug called nizubaglustat (AZ-3102) in people aged 4 to 30 with Gaucher disease type 3, a rare genetic condition that can cause anemia, low platelets, an enlarged spleen, and neurological problems. Participants take the drug daily as a dissolvable…

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

This study tests whether a low-dose sugarcane-derived supplement called policosanol, combined with a structured exercise program, can improve physical fitness and blood lipid levels in adults with elevated cholesterol who do not yet need medication. Over 12 weeks, participants ar…

This study tests whether a modified version of the Crohn's Disease Exclusion Diet (CDED) can ease symptoms of non-constipation irritable bowel syndrome (IBS) better than the standard low FODMAP diet. About 150 adults with moderate-to-severe IBS will follow one of the two diets fo…

This study tests whether adding hands-on cooking classes to standard Mediterranean diet advice helps adults aged 30-60 with metabolic syndrome improve their health. Participants receive either diet counseling alone or counseling plus culinary training, including recipe booklets a…

This study tests whether verapamil, a common blood pressure medication, can help protect insulin-producing cells in people with cystic fibrosis who have early blood sugar problems or diabetes. Participants take verapamil for several weeks, and researchers measure changes in insul…

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

This trial tests whether a less intensive antiplatelet therapy is safer and as effective as standard dual therapy for people with spontaneous coronary artery dissection (SCAD), a rare cause of heart attack in women aged 30–55. Unlike typical heart attacks, SCAD involves a tear in…

This early-stage trial tests a single-dose gene therapy called LY-M003 in 18 adults with Wilson's disease, a genetic condition that causes copper buildup. The goal is to see if it's safe and can reduce or eliminate the need for standard daily medications. Participants receive one…

This trial tests whether using higher (less strict) bilirubin thresholds for starting light therapy in very preterm infants (born before 30 weeks) is as safe as the current Dutch guidelines. About 680 babies across Dutch NICUs will be randomly assigned to one of two threshold str…

This study tests whether a sugar called D-galactose (AVTX-801) can help people with SLC35A2-CDG, a rare inherited disorder that disrupts how the body adds sugar to proteins. The trial enrolls about 10 people of any age who have seizures or chronic digestive issues. Participants r…

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

This study tests a new microtablet protein substitute for people with phenylketonuria (PKU), a condition requiring a strict low-protein diet. The microtablets combine protein with vitamins and minerals, aiming to be easier to take than current liquid or powder options. About 20 a…

This study tests whether a cream containing simvastatin (a cholesterol-lowering drug) can safely shrink yellow cholesterol deposits on the eyelids, called xanthelasma. Thirty adults will apply either the active cream or a placebo nightly for 24 weeks, then everyone gets the activ…

This pilot study tests whether a connected blood pressure monitor, supervised daily by a healthcare assistant, helps patients with blood cancers like CLL or lymphoma stick to home monitoring. The goal is to catch and manage high blood pressure early, a common side effect of BTK i…

This study tests whether a low dose of colchicine, a common anti-inflammatory drug, can slow down blood vessel hardening and bone problems in people with stage 3 chronic kidney disease. About 60 adults will take colchicine or receive usual care for 12 months. The goal is to see i…

This study tests whether a once-weekly injectable drug called mazdutide can help adults with prediabetes achieve normal blood sugar levels. About 150 participants will receive either a low dose, a higher dose, or a placebo for 24 weeks, along with diet and exercise advice. The ma…

This early-phase trial tests whether giving healthy stem cells from the mother to a fetus diagnosed with Fanconi anemia can safely improve the baby's blood cell production. The treatment is a one-time injection into the fetus during pregnancy. The study will enroll 12 pregnant wo…

This early-stage trial tests a single injection of MWX205 in 37 adults with high cholesterol or blood fats (dyslipidemia). Researchers will check if the drug is safe, how the body processes it, and how it affects cholesterol levels compared to a placebo. The goal is to find the r…

This study follows 100 adults with congenital adrenal hyperplasia (CAH) who switch from standard steroids to a timed-release hydrocortisone (Efmody). The goal is to see if better mimicking the body's natural cortisol rhythm improves hormone levels, metabolic health, and quality o…

This study tests whether adding hyaluronic acid gel to standard deep cleaning (scaling and root planing) improves gum healing in people with both diabetes and advanced gum disease. Twenty-three participants will receive the standard cleaning on all teeth, and then two gum pockets…

This study tests an oral drug called glycerol tributyrate in 24 adults with MELAS or LHON-Plus, two rare mitochondrial diseases that cause severe symptoms like strokes and vision loss. The trial is open-label (everyone gets the drug) and uses each person as their own control over…

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

This study tests whether a wearable goggle system that makes parathyroid glands glow can help surgeons find and protect them during thyroid removal. About 280 adults having thyroid surgery will take part. The goal is to reduce the risk of low calcium levels after surgery, a commo…

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

This early-stage study tests whether high-dose Vitamin C is safe for people with two rare inherited blood disorders—Pyruvate Kinase Deficiency (PKD) and a form of G6PD deficiency. Both conditions cause red blood cells to break down too quickly, leading to anemia. The study will e…

This early-stage trial tests a new gene therapy called VGN-R08b for children with type III Gaucher disease, a rare genetic disorder affecting the brain and body. The treatment is given as a single injection into the brain's fluid spaces. The study will monitor safety and measure …

This study will review medical records and call patients who received AGN1 LOEP, a device injected into the hip bone to treat osteoporosis. Researchers want to see how many patients later had hip fractures and whether any side effects occurred. The goal is to understand how well …

This early-phase trial tests a gene therapy for children with CLN6 Batten disease, a rare and fatal brain disorder. Twelve children will receive a single injection of the therapy into the fluid around the spinal cord. Researchers will check if it is safe and if it can slow the di…

This study tests whether a structured 12-week program focusing on nutrition, exercise, sleep, and stress management can improve fatigue, physical function, and quality of life in people with relapsing-remitting multiple sclerosis. Thirty participants will first be observed for 12…

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

This study tests whether a medication called chiglitazar sodium, combined with healthy lifestyle changes, can help people with prediabetes return to normal blood sugar levels. About 472 adults with prediabetes will take either the drug or a placebo for 52 weeks, along with lifest…

This study tests whether a dietary supplement made from halophyte plant extracts (like salicornia) and B vitamins can improve cholesterol levels in people recently diagnosed with dyslipidemia who do not yet need medication. About 250 participants will take the supplement or follo…

This phase II trial is testing the safety and effectiveness of combining two drugs, mosunetuzumab and pirtobrutinib, in 25 adults with relapsed or refractory Waldenstrom Macroglobulinemia, a rare blood cancer. Participants must have had at least one prior treatment. The study aim…

This study will give 150 children and teens with high blood pressure, high cholesterol, or obesity a free debit card to buy fruits and vegetables, plus virtual nutrition classes. The goal is to see if this program helps improve their heart health over 6 to 12 months. Participants…

This study looks at whether taking berberine, a natural supplement, can improve blood fat levels and blood pressure in people with cardiometabolic syndrome. Researchers will compare berberine to a placebo in 40 healthy adults aged 18-65. The goal is to see if berberine can help c…

This study tests a dietary supplement called Cardiol Forte in 50 adults with mildly high cholesterol but low overall heart risk. Participants will take either the supplement or a placebo for 8 weeks, then everyone gets the supplement for another 8 weeks. The goal is to see if it …

This study looks at whether metabolic surgery (like gastric bypass) can reduce the severity of atrial fibrillation (AF), a common heart rhythm problem, in people with obesity. About 100 adults with AF and a BMI between 35 and 65 will be randomly assigned to either surgery or medi…

This study tests whether adding exercise-focused lifestyle coaching to a personalized diet plan improves health in 435 overweight or obese adults with metabolic syndrome. Participants will either follow a diet plan alone or a diet plan plus an intelligent lifestyle program that t…

This early-stage trial tests a single dose of a gene therapy called DANAGALEX in 9 adults and children with aspartylglucosaminuria (AGU), a rare genetic disorder. The goal is to see if the treatment is safe and can reduce harmful substances in the body. Researchers will monitor s…

This study will test whether a low-calorie diet rich in medium-chain triglycerides (MCTs) from traditional Minangkabau foods can improve metabolic health in 40 obese adults. Participants will follow a diet based on coconut milk dishes for a period, and researchers will measure ch…

This study will follow 12,000 Korean adults with dyslipidemia (high cholesterol) who are prescribed Baroezet Tablet by their doctor. Researchers will measure changes in LDL ("bad") cholesterol and other fats in the blood over 24 weeks, and track any side effects. The goal is to s…

This early study tests a new injection called ART002g1 in 24 adults with a genetic form of high cholesterol (HeFH). The injection uses gene editing to lower a protein that raises cholesterol levels. The main goal is to check safety and how well the drug works at reducing choleste…

This trial is for adults with multiple myeloma or Waldenstrom macroglobulinaemia. It tests whether giving certain drugs less often can control the disease as well as the standard schedule. The goal is to find the best balance between effectiveness and quality of life. About 400 p…

This phase 2b trial tests AVTX-801, a D-galactose supplement, in 8 adults with PGM1-CDG, a rare genetic disorder affecting sugar metabolism. Participants currently on D-galactose will receive either the study drug or a placebo to see if it reduces disease-related events like low …

This study tests whether a person's own stem cells, processed and given by IV, can safely help with multiple system atrophy (MSA) — a rare, worsening brain disease that affects movement and automatic body functions like blood pressure. Fifty adults aged 35 to 65 will receive eith…

This study tests a combination of three drugs (nemtabrutinib, bortezomib, and rituximab) in people with Waldenstrom's macroglobulinemia, a rare blood cancer. The goal is to see how well the treatment shrinks tumors and controls the disease. About 19 adults who have not yet been t…

This study tests a new gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases. Researchers will take blood cells from 6 patients, fix the genetic defect in the lab, and then see if these cells can help brain cells work better. The goal is to show the …

This Phase III trial tests QLC7401, a new RNA-based drug, in 120 adults with heterozygous familial hypercholesterolemia (a genetic condition causing very high cholesterol). Participants already on cholesterol medication will receive either QLC7401 or a placebo injection. The stud…

This Phase 3 trial tests a new drug called QLC7401 in about 780 adults with high cholesterol that remains high despite standard therapy. Participants will receive either QLC7401 or a placebo, added to their current cholesterol medications. The main goal is to see if QLC7401 can s…

This study looks at whether extra education from a pharmacist can help people aged 18-60 who are starting a new antipsychotic medication for conditions like schizophrenia or bipolar disorder. The goal is to see if this support reduces weight gain and other metabolic side effects.…

This study tests whether a 9-month lifestyle coaching program (individual and group sessions with a dietician) helps obese male firefighters lose weight and improve metabolic health. Fifty participants will be randomly assigned to coaching or usual care. The main outcomes are cha…

This early study tests a drug called piclidenoson in 5 adult men with Lowe syndrome, a rare genetic disorder that harms the kidneys. The goal is to see if the drug can improve how the kidneys reabsorb important small proteins over 6 months. Researchers will also check safety and …

This study will test three types of intermittent fasting (5:2, alternate-day, and time-restricted eating) in 120 adults with prediabetes. Researchers will measure changes in blood sugar, cholesterol, body fat, and inflammation over 4-6 weeks. The goal is to see which fasting patt…

This study tests whether giving immunotherapy drugs (nivolumab with or without ipilimumab) or immunotherapy plus radiotherapy before surgery can improve outcomes for people with a specific genetic type of colorectal cancer called MSI-H/dMMR. About 114 participants will be randoml…

This study tests whether a non-invasive brain stimulation technique called rTMS can reduce liver fat in people with alcohol-related fatty liver disease (MetALD). 105 participants will receive either personalized rTMS, standard rTMS, or a sham (placebo) treatment over 20 sessions.…

This study tests whether a new omega-3 supplement called Ruby-O, made from phospholipids, raises omega-3 levels in the blood more effectively than standard omega-3 supplements. About 105 adults with mixed dyslipidemia (abnormal blood fats) who are already on statin therapy will t…

This study tests a new injectable drug called DNV001 in about 120 adults with high cholesterol that isn't well controlled by statins. Participants will receive different doses of the drug or a placebo as a shot under the skin, while continuing their usual statin pills. The main g…

This study tests whether a liquid carob extract can improve blood sugar levels in 70 adults with prediabetes. Participants take the extract or a placebo twice daily for 90 days. Researchers will measure blood glucose and insulin responses to see if the extract helps control blood…

This early-phase study tests whether combining focused ultrasound with the immunotherapy drug pembrolizumab is safe and feasible for people with recurrent glioblastoma, a severe brain cancer. Only 8 participants with a specific genetic feature (mismatch repair deficiency) will be…

This study tests two immunotherapy drugs (iparomlimab and tuvonralimab) in 30 women with a specific type of endometrial cancer that is mismatch repair-deficient. The goal is to see if the treatment can make the cancer disappear or shrink enough to avoid surgery. Participants must…

This phase 2 trial tests two different drug combinations given before surgery for people with a specific type of stomach cancer (dMMR/MSI-H). Participants will receive the immunotherapy drug adebrelimab plus either chemotherapy or another immunotherapy drug (SHR-8068). The goal i…

This phase 4 trial tests whether adding recaticimab to vunakizumab works better than vunakizumab alone for adults with moderate-to-severe plaque psoriasis and high cholesterol. Forty participants will be randomly assigned to receive either the drug combo or vunakizumab plus a pla…

This study compares a high-intensity loading dose of vitamin D2 to standard weekly dosing in 52 patients with newly diagnosed aggressive non-Hodgkin lymphoma who are also receiving standard chemotherapy. The goal is to see if the high-dose approach normalizes vitamin D levels fas…

This Phase 2 trial tests the drug luspatercept (Reblozyl) in 45 people with rare inherited anemias that affect red blood cells. The goal is to see if it can reduce the need for blood transfusions in those who depend on them, or raise hemoglobin levels in those who don't. Particip…

This study tests whether a lower dose of the chemotherapy drug treosulfan, given before a stem cell transplant, can help people with Nijmegen breakage syndrome. The condition causes immune problems and a high risk of cancer. The trial will enroll 24 patients, giving a lower dose …

This phase 2 trial will test a combination of immunotherapy drugs and chemotherapy given before surgery for people with a specific genetic subtype of stomach cancer (dMMR/MSI-H). The study aims to see how well this approach clears the tumor and to understand why some patients do …

This study tests whether Yangxin Dawayimixike Honey Paste can reduce carotid artery plaque and improve cholesterol in people with dyslipidemia. Over 400 participants will receive either the honey paste or a placebo for 12 months, with a one-year follow-up. The main goal is to see…

This study tests whether an AI-powered app called 'Smart family doctor' can help people who have had heart surgery (bypass or stent) better control their blood pressure, blood sugar, and cholesterol. About 951 participants from 10-20 hospitals will use the app for personalized he…

This early study tests whether high-dose vitamin B3 (nicotinamide) can help preserve or improve vision in people with Leber's hereditary optic neuropathy (LHON), a rare genetic disease that causes sudden vision loss. Researchers will give 13 participants 2 grams of vitamin B3 dai…

This study tests a combination of targeted therapy (zanubrutinib) with a reduced-dose chemotherapy and an antibody drug for people newly diagnosed with Waldenström macroglobulinemia, a rare blood cancer. About 43 participants will receive four cycles of the combo, then eight mont…

This early-stage trial will test a new drug formulation (AD-117) for mixed dyslipidemia, a condition involving high cholesterol and triglycerides. Sixty healthy adults will receive a single dose of either the new or a reference formulation to compare how the body processes each. …

This study tests a supplement called GlycoDual in 90 adults with prediabetes (higher-than-normal blood sugar). For 90 days, half will take GlycoDual and half a placebo. Researchers will check if GlycoDual lowers fasting and after-meal blood sugar and improves how the body uses in…

This study tests a combination of two oral drugs, zanubrutinib and BGB-11417, in adults with Waldenström macroglobulinemia who have already received at least one prior treatment. The goal is to see if the combination can shrink or control the cancer better than current options. P…

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

This study tests whether replacing half the wheat in rotis with barnyard millet, barley, or bengal gram flour can lower blood sugar and help reverse prediabetes. 140 adults with prediabetes will eat these special rotis for three months while researchers track their glucose levels…

This early-stage trial tests a gene therapy called GC310 for Wilson's disease, a genetic condition that causes copper buildup in the body. Researchers will give a single dose to 15 adults and monitor them for a year to check safety and whether it improves copper levels. The goal …

This early-phase trial tests a three-drug combination (ibrutinib, bendamustine, and rituximab) given for a fixed duration in people with Waldenström macroglobulinemia, a rare blood cancer. The study has two parts: first, finding the safest dose of bendamustine, and then testing t…

This study tests whether taking fenugreek seed powder daily for 12 weeks can improve key signs of metabolic syndrome, such as waist size, blood pressure, cholesterol, and blood sugar. Researchers will compare fenugreek to a placebo in 28 adults aged 30 to 60 who have metabolic sy…

This phase III trial tests a vitamin B12 injection (hydroxocobalamin chloride) in 20 children aged 6 months to 18 years with a specific type of methylmalonic acidemia (cobalamin C deficiency). The goal is to see if the injection can normalize levels of certain acids in the blood …

This study tests a comprehensive management service package for older adults (65+) who have two or more chronic conditions like high blood pressure, diabetes, or arthritis. The package includes digital tools, personalized lifestyle advice, and long-term support. Researchers will …

This study tests whether a special PET-CT scan using a drug called evuzamitide can detect signs of cardiac amyloidosis in people whose standard heart scans were unclear but who have high levels of a blood marker called TAD1. About 25 adults with heart failure or a genetic risk fo…

This trial tests whether measuring glucose in umbilical cord blood, already collected at birth, can screen newborns for congenital hyperinsulinism, a condition that causes dangerously low blood sugar and can lead to brain damage if missed. Researchers will check the test's accura…

This study tests whether a simple calculation from a standard blood test—the creatinine-to-body weight ratio—can predict fatty liver disease in people with metabolic syndrome. Metabolic syndrome is a cluster of conditions like obesity, high blood pressure, and high blood sugar th…

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

This trial tests whether a new ultra-rapid genetic test, using third-generation sequencing, can diagnose familial lymphohistiocytosis (FHL) in children within 5 days instead of the usual 6-8 weeks. FHL is a rare, life-threatening genetic condition where the immune system attacks …

This study tests whether annual liquid biopsies (from blood, urine, stool, and vaginal swabs) can safely replace colonoscopies every 18 months for people with Lynch syndrome. 2,000 participants will be split into two groups: one gets standard care (colonoscopy every 18 months), t…

This study tests whether a combination of blood markers, ultrasound, and genetic information can accurately diagnose metabolic dysfunction-associated steatotic liver disease (MASLD) in children aged 8 to 18 with obesity. Each child provides a blood sample, undergoes a liver ultra…

This trial tests whether a finger-prick HbA1c test can detect type 2 diabetes better than the standard random blood glucose test in at-risk adults living in rural Dali, China. Researchers will compare detection rates and costs among 720 permanent rural residents with no known dia…

This study aims to see if a new radioactive dye can detect early signs of a heart condition called amyloidosis in people who carry a TTR gene mutation but have no heart failure symptoms. About 80 adults aged 30-80 will receive an injection of the dye and then have a PET/CT scan t…

This study tests whether a computer model can find signs of a heart condition called cardiac amyloidosis in bone scans that were done for other reasons. Researchers will look back at 57 patients' scans to see if the model can correctly identify those with the disease. If it works…

This study tests a new, contactless camera technology that measures heart rate, blood pressure, and heart disease risk from a short video of your face. Researchers will compare these readings to standard medical tests in 300 adults aged 30 and older in Jakarta. If accurate, this …

This study will enroll 5000 adults to see if a one-minute, single-lead ECG, analyzed by machine learning, can detect heart conditions like high blood pressure, heart failure, and diabetes. Participants will have their ECG recorded twice and compared with full medical exams. The g…

This pilot study aims to develop a new digital PCR technique to more accurately diagnose mitochondrial diseases. Researchers will test the method on blood, urine, saliva, and muscle fiber samples from 4 patients. If validated, the technique could be faster and cheaper than curren…

This trial will test an artificial intelligence system designed to interpret routine newborn screening tests for inherited metabolic disorders. Researchers will compare the AI's accuracy to standard manual review by trained staff. The study plans to include 200,000 newborns in Ch…

This study looks at whether the triglyceride-glucose (TyG) index, a simple measure from a blood test, can predict insulin resistance in people with pre-diabetes and chronic kidney disease. Researchers will enroll 60 adults with and without kidney problems to see how well the TyG …

People with Fanconi anaemia have a high risk of developing oral cancer, but standard biopsies are painful and risky due to their genetic sensitivity. This study tests a non-invasive oral brushing technique to detect early cancer cells in 115 patients aged 15 and older. If accurat…

This study tests whether the BIND score, a quick neurological check, can predict brain risk in newborns with severe jaundice. Researchers will use the score to guide phototherapy treatment in 80 infants under 28 days old. The goal is to catch and treat dangerous jaundice early, p…

Researchers are testing an AI chatbot that gives personalized, culturally tailored advice on nutrition, physical activity, and goal setting to adults at risk for type 2 diabetes. About 30 adults aged 18 to 55 will use the chatbot for 6 weeks. The study will check whether the chat…

This trial tests whether giving semaglutide (Wegovy) to women shortly after a pregnancy affected by gestational diabetes can reduce their risk of developing metabolic syndrome, a cluster of conditions that raise the chance of heart disease and diabetes. About 226 postpartum women…

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

This trial tests whether taking coenzyme Q10 (CoQ10) for 10 weeks can improve biological resilience in adults aged 65 and older who show early signs of frailty. Participants will be randomly assigned to receive either 200 mg of CoQ10 daily or a placebo. The study will measure cha…

This pilot study tests whether a simple program of brief, frequent movement throughout the day can help reduce cardiometabolic risk in rural women. Participants wear a Fitbit that reminds them to move each hour and complete health assessments before and after the 12-week program.…

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

This study tests a community-based program to help Black and Hispanic teens aged 15-20 cope with chronic stress and prevent depression. Participants will join peer groups to learn skills and will be tracked for 2 years. The goal is to reduce depressive symptoms and improve physic…

This study tests whether combining continuous glucose monitors (CGMs) with group lifestyle sessions can help people with prediabetes lower their blood sugar and prevent type 2 diabetes. Twenty adults with prediabetes will attend regular group meetings on diet, exercise, and stres…

This study tests a 12-session virtual program to help adults with prediabetes in the UAE prevent type 2 diabetes. The program covers diet, physical activity, sleep, and stress management, and is led by a health coach from the same cultural background. Thirty participants will att…

Researchers are testing whether mazdutide, a weekly injected drug, reduces the number of gout flares in adults who have both obesity and gout. The study enrolls 30 people aged 18 to 65 with a BMI of at least 30 and at least two gout flares in the past six months. Everyone receive…

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

This trial tests whether XPhe Piccos, a slow-release protein substitute in tiny tablets without phenylalanine, is acceptable and well-tolerated in children aged 3 and older with phenylketonuria (PKU) or hyperphenylalaninemia. Participants replace some or all of their usual protei…

This trial tests whether giving patients a prescription for haloperidol to use as needed at home can help control symptoms of cannabinoid hyperemesis syndrome — a condition marked by severe nausea, vomiting, and abdominal pain from heavy cannabis use. The goal is to see if having…

This trial is testing whether a combination of Bao Yuan Tang, a traditional Chinese medicine extract, and galactooligosaccharides, a type of prebiotic, can improve sleep quality, reduce fatigue, and support better metabolism in female nurses who work night shifts. The study will …

This study compares two ways to break up kidney stones using a laser and a suction device. One method turns stones into dust, the other into small fragments. The goal is to see which leaves fewer stone pieces behind after one month. About 86 adults with small kidney stones will t…

This study tests whether light physical activity after meals can improve blood sugar levels in adults with metabolic syndrome and prediabetes. Twenty-five participants will try three routines in random order: sitting, a 15-minute walk, or brief resistance exercises like squats ev…

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

This early-stage trial tests a new robotic surgical method for esophageal cancer that avoids cuts between the ribs, which may cause less chest pain and faster recovery. About 31 adults with stage I to III esophageal cancer will be randomly assigned to either the new approach or s…

This study tests whether maple syrup, mixed with water and electrolytes, can improve cycling performance in trained male cyclists. Thirty-two participants will drink different amounts of maple syrup or a placebo during long rides. Researchers will measure how fast they finish a 2…

This study investigates how eating three high-fat meals in a row changes vitamin E levels in the blood. Researchers will compare people with normal blood fats to those with high blood fats (hyperlipidemia). The goal is to understand whether fats in the blood can trap vitamin E, p…

This study looks at how eating a high-fat or high-carb breakfast changes fat levels in the blood over 8 hours. Researchers will compare results among people with conditions like diabetes, fatty liver, or kidney disease, as well as healthy volunteers. Participants will have two ov…

This study screens up to 1,000 people with personal or family histories of certain cancers to see if they qualify for ongoing genetics research at the National Cancer Institute. Participants fill out a 15-20 minute online survey about their health and family history. No treatment…

This study compares two approaches to notify relatives of people diagnosed with familial hypercholesterolemia (FH), a genetic condition causing very high cholesterol. One method lets patients share information themselves; the other has the study team contact relatives directly. R…

This observational study gathers information from routine care on how localized rectal cancer is treated across Austrian hospitals. It aims to document the variety of treatment approaches, including total neoadjuvant therapy and immunotherapy, and their outcomes. By analyzing thi…

This study follows 250 people with cystinosis, a rare genetic disorder that causes a buildup of the amino acid cystine, leading to damage in the kidneys, eyes, and other organs. Researchers will track how the disease progresses over time, including kidney function, eye problems, …

Researchers at Assiut University are studying whether ionized calcium levels, measured from a routine blood sample when a patient enters the ICU, can signal how severe sepsis or septic shock is. The study follows 160 adults admitted to intensive care within 24 hours of presentati…

Researchers at Hacettepe University are studying how aerobic fitness and meal timing affect the body's use of fat and carbohydrates during exercise, and how immune cells called peripheral blood mononuclear cells (PBMCs) respond. Thirty healthy young men, split into high and low f…

This study looks at whether mindfulness-based stress reduction can help Black teenage girls who have been involved with the justice system and their parents or caregivers. About 90 participants will report on stress, PTSD symptoms, and other outcomes before and after the program.…

This study asks whether women with lipodystrophy syndromes or Turner syndrome experience lower sexual quality of life than women in the general population. Researchers will compare survey responses from affected women with those from a large French cohort. The goal is to understa…

This study looks at whether a blood protein called asprosin is linked to metabolic syndrome and heart risk in people with rheumatoid arthritis. Researchers will compare asprosin levels in three groups: rheumatoid arthritis patients with metabolic syndrome, those without it, and h…

This study will compare how vegan, omnivorous, and carnivore diets affect the way LDL cholesterol particles clump together in the blood, which can lead to artery plaque. Researchers will measure blood lipids and other health markers in 90 adults aged 25-60 who have followed their…

This early-phase study looks at whether metabolic syndrome reduces brain blood flow more in women than in men. Researchers will use MRI scans and a drug called indomethacin to test this in 72 healthy adults and those with metabolic syndrome. The goal is to understand sex differen…

This study looks at anonymized health records from about 100,000 people in a Turkish hospital network to see how common cardiometabolic conditions—like obesity, type 2 diabetes, heart disease, fatty liver, and kidney disease—are and how often they occur together. By tracking thes…

This study tests a structured program to help 40 adolescents and young adults with congenital adrenal hyperplasia (CAH) move from pediatric to adult healthcare. Participants will learn about their condition, emergency preparedness, and self-management. The goal is to see if the p…

This study will follow about 2,500 adults in Italy who have high cholesterol and are at high risk for heart problems. Researchers want to see if their current treatments match official guidelines and how well their cholesterol is controlled over one year. The goal is to find gaps…

This study asks whether swapping a beef burger for a plant-based pea burger changes how the body handles fats and sugar after a meal. Ten adults with metabolic syndrome (and a BMI of 25 or higher) will eat both types of burgers on separate occasions in a crossover design. Researc…

This study is a registry that will collect health information from 1,000 people who carry the gene for hereditary amyloidosis, including those with and without symptoms. Researchers will track who develops the disease and how it progresses, including the need for heart transplant…

This study measures specific substances (metabolites) in the breath and blood of people with inherited metabolic disorders, those on a special ketogenic diet for epilepsy, and healthy volunteers. The goal is to see if a simple breath test can reliably track these metabolites, pot…

This study looks at how beta-cells (which make insulin) respond to growth hormone in people with prediabetes. Researchers will measure insulin release in 10 adults with impaired fasting glucose or impaired glucose tolerance. The goal is to better understand what raises the risk o…

This study explores whether polycystic ovary syndrome (PCOS) independently contributes to fatty liver disease in adolescent girls, beyond the effects of obesity. Researchers will compare three groups of girls aged 10–18: those with PCOS and obesity, those with obesity alone, and …

This observational study is investigating whether vitamin D levels are linked to cholesterol and other blood fats in obese adults with type 2 diabetes. Researchers will measure vitamin D status and lipid profiles in 150 participants from a diabetes clinic in Cairo. No treatments …

This study investigates how pomegranate dietary supplements influence gut bacteria and inflammation in people with alcohol use disorder and alcohol-related liver disease. Researchers will measure levels of beneficial gut metabolites called urolithins and inflammatory markers in b…

This study looks at whether brown adipose tissue (brown fat), which burns energy as heat, can predict how well people with obesity respond to the weight loss drug semaglutide (Wegovy). Eighty adults starting semaglutide will have PET/CT scans before and after 24 weeks of treatmen…

This study explores whether at-home saliva samples can reliably measure key steroid hormones in people with congenital adrenal hyperplasia (CAH), a condition requiring lifelong hormone therapy. Currently, monitoring involves morning blood tests that miss daily hormone fluctuation…

This study tracks how Sanfilippo syndrome type C, a rare genetic disorder that affects the brain, progresses over time. Caregivers of children and young adults aged 1 to 25 will record videos of daily activities and answer questionnaires using a smartphone app every six months fo…

This study investigates how often atrial fibrillation (AF), a type of irregular heartbeat, occurs in people with certain blood cancers who are starting a targeted cancer drug called a Bruton's tyrosine kinase inhibitor (BTKI). Researchers will follow 500 participants—400 starting…

This study looks at how atrial cardiomyopathy—a condition where the heart's upper chambers are damaged—relates to cardiovascular-kidney-metabolic (CKM) syndrome, a cluster of heart, kidney, and metabolic problems. Researchers will use non-invasive tests like ECG, echocardiography…

This study follows 300 newborns treated with light therapy for jaundice to find clues that predict whether jaundice will come back. Researchers will check blood bilirubin levels, feeding patterns, and other factors. The goal is to help doctors decide when it's safe to stop treatm…

This study surveys 1,000 adults with Restless Legs Syndrome (RLS) to find out how often pain is part of their symptoms and how it affects their daily lives. Participants fill out an online questionnaire about their medical history, sleep, pain, and quality of life. The goal is to…

This study screens adults aged 18 and older in suburban and peri-urban areas of Faisalabad, Pakistan, for four major noncommunicable diseases: diabetes, hypertension, obesity, and high cholesterol. Participants who are found to have these conditions are referred to doctors for st…

This study will take 3D scans of the inside of the mouth from 240 people with rare bone or cartilage diseases and from healthy volunteers. Researchers will use shape analysis and artificial intelligence to see if these scans can help tell different diseases apart. The goal is to …

This study will send different types of WhatsApp messages to 235,000 people in Chile who are due for cancer or chronic disease screenings. Researchers want to see if the way a message is worded (basic, risk-focused, or peace-of-mind) and whether it mentions one test or both tests…

This study looks at whether a tumor's DNA repair status (MMR) changes how well the presence of cancer cells in lymph vessels predicts spread to lymph nodes in stomach cancer. Researchers will review medical records of 3,000 patients who had surgery. The goal is to improve risk as…

This study will test whether smelling a pleasant food odor (madeleine scent) before drinking a sugary solution changes how the body handles blood sugar. Twenty adults, some with obesity and mild insulin resistance and some without, will each undergo two sessions: one with the sce…

This study follows 45 adults with alpha-1 antitrypsin deficiency (a genetic condition that can cause liver damage) to learn how the disease progresses. Researchers will analyze stored blood and liver tissue samples to find biomarkers and understand the molecular pathways involved…

This study follows 40 adults who recently had laparoscopic sleeve gastrectomy for obesity. Over 6 months, researchers will measure changes in blood sugar, cholesterol, gut barrier markers, and digestive symptoms. The goal is to understand how the surgery affects gut health and me…

This study uses a wearable glucose sensor and artificial intelligence to understand how healthy adults process sugar. Researchers will track blood sugar patterns for 14 days and link them to diet, activity, and health measures. The goal is to develop personalized nutrition tools …

This study uses the FreeStyle Libre 2, a small sensor worn on the arm, to track blood sugar changes in 120 people with rare forms of diabetes, including monogenic diabetes and hyperinsulinemia. Researchers will compare these patterns with data from healthy people and those with t…

This study will ask 75 people with diabetes and their healthcare providers about their experience using the Instara-1 continuous glucose monitor. Participants will wear the device and answer questionnaires about comfort, ease of use, and how it helps manage their diabetes. The go…

This study looks at how metabolic health (measured by a simple blood test) relates to artery stiffness and daily blood pressure changes in 800 adults. Researchers will use routine tests and non-invasive measurements to find better ways to identify people at higher risk for heart …

This study looks at how aging changes tiny parts of our cells called mitochondria, which produce energy. Researchers will take small skin samples and blood from 90 healthy adults aged 18-90 to measure inflammation and cell aging markers. The goal is to better understand why we ag…

This 5-year study will follow 2,800 overweight or obese Chinese adults aged 18-60 to see how their health changes over time. Participants will use a smartphone app to track their data and get lifestyle support. The goal is to understand who develops diabetes or complications, and…

This early-stage study will test a new experimental drug called AD-231 in 60 healthy adults. The goal is to see how safe the drug is and how the body processes it, not to treat any disease. Participants will receive a single dose of the drug and have their blood levels measured o…

This observational study in Mexico will follow 30 adults already taking tirzepatide for conditions like diabetes or obesity. Researchers will use MRI scans of the knees, CT scans of the heart, and body composition tests to see how the drug affects cartilage, heart fat, and overal…

This study looks for genetic differences that might explain a rare form of diabetes that is not caused by the immune system. Researchers will study 118 adults of African or Indian ancestry in the French West Indies who have this atypical diabetes. The goal is to find gene changes…

This study aims to validate a blood test for measuring oxalate levels, which is currently not available in Israel. Researchers will compare results from a local lab with an external lab using 50 blood samples. They will also explore how vitamin C levels relate to oxalate in patie…

This study will check if a routine blood measure called mean platelet volume (MPV) can predict acute coronary syndrome (heart attack or unstable angina) in people with metabolic dysfunction-associated steatotic liver disease (MASLD). Researchers will compare MPV levels between 60…

This study looks at whether wearing a continuous glucose monitor (CGM) and receiving education can help teenagers with prediabetes improve their blood sugar levels and adopt healthier behaviors. Twenty-five teens aged 13-17 will take part in a 20-week program, first receiving sta…

This study looks at how abnormal proteins, like alpha-synuclein and tau, build up and affect brain function in people with Parkinson's disease. Researchers will use brain scans, blood tests, and skin biopsies to track these changes. The goal is to find better ways to diagnose and…

This study will give fecal transplants to 90 people with recurrent C. difficile infection, ulcerative colitis, or metabolic syndrome. Researchers will use advanced tests on stool and tissue samples to track how donor bacteria settle in the gut and interact with the host. The goal…

This study will look at health records of 10,000 patients in Qatar who were hospitalized for heart problems and have diabetes or prediabetes. Researchers will use artificial intelligence to find patterns that predict future heart attacks, strokes, or death. The goal is to create …

This study looks at whether metabolic syndrome (a cluster of conditions like high blood pressure and excess belly fat) makes it harder to recover from a rotator cuff injury. Forty adults with rotator cuff tears will be split into two groups—those with and without metabolic syndro…

This observational study will test whether a 1-3 minute AI-powered facial scan can accurately measure vital signs like heart rate and blood pressure, as well as estimate blood sugar, cholesterol, and mental health status. Researchers will compare the scan results with standard me…

This study looks at how the amount and type of carbohydrates in your diet affect blood sugar, fats, and gut health. Twenty adults with or at risk for metabolic syndrome will eat four different carb diets in a random order. The goal is to understand which carb patterns are best fo…

The SIRENA study will follow 300 children and adults who have tested positive for islet autoantibodies, which are early markers of type 1 diabetes. Over up to 10 years, researchers will track how their blood sugar, immune system, and metabolism change. The goal is to better predi…

This survey will ask about 60 people—patients with Gaucher disease, their caregivers, and home infusion nurses—how well they understand and use educational materials for at-home VPRIV infusions. The materials include an infusion diary, a guide, and an emergency plan. The goal is …

This study works with 35 primary care clinics to help them screen patients for physical inactivity and refer them to community-based exercise programs. The goal is to see if this approach can be smoothly integrated into routine care and whether it leads to better health outcomes …

This study will survey 200 adults with Fabry disease to understand how men and women experience different paths to diagnosis. Researchers want to see if symptoms or family screening lead to diagnosis more often in one sex, and how long diagnosis takes. The goal is to identify pat…

This study will look at medical records from 3,600 people in Japan to see how well they follow their prescribed cholesterol-lowering medication. It compares two drugs, inclisiran and evolocumab, over 12 months. The goal is to understand real-world adherence, not to test new treat…

This study looks at whether eating a breakfast with two types of carbohydrates (glucose and fructose) can increase glycogen stored in the liver. Twelve healthy male athletes will be tested after different breakfasts using MRI scans and blood samples. The goal is to learn the best…

This study will check if a specific cholesterol ratio (non-HDL to HDL) is linked to kidney disease in people with diabetes. Researchers will measure this ratio and kidney function in 84 adults. The goal is to better understand risk factors, not to test a new treatment.

This study will find the correct dose of sugammadex, a drug that reverses muscle relaxants, for infants under 2 years old. About 99 babies having non-emergency surgery will receive small, increasing doses until their muscle function returns. The goal is to ensure safe and complet…

This study will compare a carnivore diet (only animal foods) with a Mediterranean diet (fruits, veggies, grains, legumes) in 30 adults aged 30-60 with high LDL cholesterol. Over 3 weeks, researchers will measure how each diet affects cholesterol clumping, blood fats, and blood ve…

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

This observational study looks at whether eating a diet that causes inflammation can make gum disease worse in people with metabolic syndrome. About 100 adults with metabolic syndrome will have their gum health checked and their diet analyzed using a special inflammation score. T…

This study will look at 3000 young Asians in Singapore who do not have diabetes yet. Researchers will use blood tests, glucose monitors, food diaries, and body measurements to find better ways to detect diabetes early. The goal is to create new risk tools to identify young people…

This study will test a new computer program designed to help doctors make better decisions for patients with high cholesterol and heart disease. Researchers will review the medical records of 500 adults and compare the program's recommendations to what doctors actually prescribed…

This study looks at how blood phenylalanine (Phe) levels affect thinking, emotions, and daily functioning in adults with PKU. Researchers will ask 30 adults with PKU and their relatives to complete questionnaires. The goal is to understand why some people with high Phe levels hav…

This study will look at how getting only 3 hours of sleep (instead of 8) affects blood sugar, blood vessel stiffness, and thinking skills in healthy adults aged 20-45. Researchers will also test if regular exercise can help protect against these negative effects. The goal is to b…

This study tests a new DNA sequencing method that reads long stretches of DNA to find hidden genetic changes causing rare diseases like albinism and intellectual disability. Researchers will analyze stored blood or DNA from 150 patients who haven't gotten a clear diagnosis yet. T…

This study looks at how the cholesterol-lowering drug rosuvastatin affects fitness and muscle symptoms in people who run long distances at least three times a week. Researchers will compare runners taking rosuvastatin to those not taking it over three months, measuring changes in…

This study looks at how artificial sweeteners (saccharin, sucralose) and a natural one (erythritol) affect blood sugar control, blood clotting, and heart disease risk in 80 adults with prediabetes. Participants will be randomly assigned to take one sweetener or water for two week…

This study looks at a condition called 'metabolically obese normal weight' (MONW), where people have a healthy BMI but too much body fat, raising their risk for heart problems and diabetes. Researchers will study 176 young women with normal weight, measuring body fat, blood marke…

This study will follow 500 Beijing residents aged 60 and older to see how urinary function (like bladder and kidney health) relates to the development of multiple chronic conditions such as diabetes, heart disease, and Alzheimer's. Participants will have regular health checks, bl…

This study will test a special PET scan tracer and blood tests to better understand and predict heart damage in people with Fabry disease. Researchers will look at heart tissue samples and blood markers from 20 adult patients. The goal is to find new ways to diagnose and forecast…

This study looks at how metabolic syndrome (a cluster of conditions like high blood pressure and blood sugar) might make gum disease worse. Researchers will measure gum inflammation in 100 adults and compare it to their metabolic health. The goal is to see if a simple gum measure…

This study will screen up to 10,000 adults aged 18-75 who are overweight or have metabolic issues like high blood sugar or cholesterol. Researchers will use questionnaires, blood tests, and liver ultrasounds to find out how common fatty liver disease is and what factors increase …

This study follows 30 people with achalasia (a swallowing disorder) for one year after their treatment. Researchers want to see if the weight gain that often happens after treatment leads to worse metabolic health, like fatty liver or higher blood sugar. Participants will have bl…

This study aims to find out how often a rare genetic condition called alpha-1 antitrypsin deficiency (AATD) occurs in people who develop liver cancer (hepatocellular carcinoma) but do not have cirrhosis or other known causes like hepatitis or heavy drinking. Researchers will look…

This study will test 70 people with IBS-related diarrhea to see how many actually have lactose intolerance. Researchers will use a PCR test and a symptom questionnaire to find links with age and gender. The goal is to better understand if lactose intolerance is a common, overlook…

This study will check if a standard jaundice test (a light meter on the skin) works equally well for newborns of all skin colors. About 510 babies will have their skin color classified and their jaundice levels measured both with the meter and a blood test. The goal is to see if …

This study will examine how metabolic syndrome—a cluster of conditions like high blood pressure, high blood sugar, and excess belly fat—relates to gastroesophageal reflux disease (GERD). Researchers will enroll 50 adults with metabolic syndrome and use an endoscopy to check for s…

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

This study looks at whether not getting enough sleep affects blood sugar control differently in healthy men and women. Researchers will ask 32 adults to sleep only 4 hours a night for three nights and then measure their blood sugar and hormone responses after a meal. The goal is …

This study will look at 225 adults with diabetic retinopathy to find out how many also develop an epiretinal membrane — a thin, scar-like layer on the retina that can blur vision. Researchers will use eye scans to detect the membrane and check for related factors like age, diabet…

This study looks at the real-life challenges faced by people with Wilson's disease in Upper Egypt, a rare condition where copper builds up in the body. Researchers will survey 66 patients and their caregivers about diet, knowledge, attitudes, and barriers to care. The goal is to …

This study will check for hormone problems in 30 children with Wilson's disease, aged 3 to 18. Researchers will look at how the pituitary, thyroid, adrenal, and sex glands are working. The goal is to find out how common these issues are and what types occur, which could help impr…

This 10-year observational study will follow 10,000 adults with diabetes, metabolic disorders, and related conditions like high blood pressure and fatty liver disease. Researchers will collect routine medical data to identify markers of disease severity and activity. The goal is …

This study will look at 35 children with joint problems that are not caused by inflammation, such as stiffness or deformity. Researchers will use exams, lab tests, and imaging to find the true cause, which could be rare genetic conditions like mucopolysaccharidoses or osteogenesi…

This study compares levels of oxidized LDL (a type of 'bad' cholesterol linked to heart disease) in the gum fluid of people with advanced gum disease versus those with healthy gums. Researchers aim to better understand the connection between gum inflammation and heart health. The…

This study will follow 175 children and young adults who had a bone marrow transplant before age 18. Researchers will test their thinking, memory, and attention skills, and check for links to physical health issues like metabolism and fitness. The goal is to find early warning si…