Inherited lipid metabolism disorder

This trial tests whether a single daily pill that combines treatments for high blood pressure, high cholesterol, and type 2 diabetes can lower the risk of heart disease and dementia. The study involves over 8,000 adults aged 50 to 75 in China who have all three conditions. Partic…

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

This study 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 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 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 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 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 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 adding ear acupressure (small pellets on specific ear points) to the standard cholesterol drug atorvastatin can lower blood fats and improve phlegm-dampness symptoms better than a sham ear treatment plus atorvastatin. Eighty adults with dyslipidemia and p…

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 new pill that combines two drugs (laroprovstat and rosuvastatin) to lower LDL or 'bad' cholesterol. About 600 adults with high cholesterol who have never taken cholesterol-lowering medication will receive either the combination, one of the drugs alone, or a pla…

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

This early-stage trial tests a new 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 Phase 2 trial tests an injection called SYH2070 in 18 Chinese adults with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol. Participants receive the drug for 48 weeks to see if it safely lowers their LDL cholesterol…

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 will test whether an artificial intelligence (AI) system can help community doctors better manage patients who have high blood pressure along with diabetes or high cholesterol. About 6,800 adults will be split into two groups: one where doctors use the AI tool to guide…

This phase 3 trial tests whether PLX-200 (gemfibrozil) can safely slow the progression of CLN3 disease, a rare genetic disorder that causes loss of motor skills, vision, and seizures in children. About 39 kids aged 6-18 will receive either the drug or a placebo for up to 96 weeks…

This study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare condition that causes muscle weakness and heart problems. Participants will receive either the drug or a placebo for 72 weeks. The main goal is to see if the drug…

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 early-stage study tests a single dose of BSA204, an experimental injection, in 40 Chinese adults with borderline high lipoprotein(a) – a fatty substance linked to heart disease. The main goal is to check safety and side effects, while also measuring changes in cholesterol le…

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 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 tests a drug called crinecerfont in 20 children aged 3 months to 4 years with classic congenital adrenal hyperplasia (CAH). The goal is to see if the drug is safe and tolerable. Participants must already be on stable hydrocortisone treatment. The study is not yet recru…

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 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 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 Phase 3 trial tests an injection called SYH2053 for people with heterozygous familial hypercholesterolemia (HeFH), a genetic condition causing very high LDL cholesterol. About 135 adults on stable statin therapy will receive either the drug or a placebo. The main goal is to …

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 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 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 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 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 study follows 75 adults with Fabry disease who are taking or starting pegunigalsidase alfa to see how well it works and how safe it is over time in real-world settings. Researchers will check heart structure and function, kidney health, and exercise capacity. The goal is to …

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 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…

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 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-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 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 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 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 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 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 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 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 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 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 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 study checks if a new version of a cholesterol-lowering drug (AJU-C715) works the same as the original (C715R) in 60 healthy adults aged 19-59. Participants take a single dose of each drug in random order and have blood tests to compare drug levels. The goal is to see if the…

This study will interview 35 adults in Sweden who have Fabry disease but are not yet receiving treatment. The goal is to learn how they experience their quality of life, daily activities, and healthcare support. By listening to patients' own stories, researchers hope to identify …

This study will look at how consistently nearly 6,000 adults in Italy take three newer cholesterol-lowering drugs (inclisiran, evolocumab, and alirocumab) over the long term. Researchers will analyze prescription records to measure adherence and persistence in everyday medical pr…

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 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 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 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 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 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 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 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…