Inherited lipid metabolism disorder
MONDO:0002525An inherited metabolic disorder caused by an enzyme deficiency, resulting in an inability to oxidize fatty acids for energy production.
Also known as: disorder of lipid metabolism, dyslipidaemia, dyslipidemia, lipid metabolism disorder, fatty acid metabolism disorder
585 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
Broader categories
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New oral drug shows promise for fabry disease in Long-Term trial
Disease control OngoingThis 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…
Phase: PHASE3 • Sponsor: Idorsia Pharmaceuticals Ltd. • Aim: Disease control
Last updated Jul 04, 2026 00:00 UTC
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Could a fatty acid drug stop dangerous sugar crashes in rare disease?
Disease control TerminatedThis 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 …
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Jul 04, 2026 00:00 UTC
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Experimental gene therapy aims to halt devastating brain disease in infants
Disease control TerminatedThis 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…
Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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One-Time gene therapy aims to halt rare, fatal brain disease in children
Disease control OngoingThis 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…
Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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New pill could protect hearts in rare genetic disease
Disease control OngoingThis 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…
Phase: PHASE3 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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New hope for babies with rare hormone disorder?
Disease control OngoingThis 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…
Phase: PHASE2 • Sponsor: Neurocrine Biosciences • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Could a pill replace infusions for gaucher brain symptoms?
Disease control OngoingThis 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…
Phase: PHASE2 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 13:08 UTC
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Experimental gene therapy aims to halt rare fatal brain disease in children
Disease control OngoingThis 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…
Phase: PHASE1, PHASE2 • Sponsor: Neela Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 13:06 UTC
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New program aims to tackle heart disease in pasifika populations
Disease control ENROLLING_BY_INVITATIONThis 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…
Phase: NA • Sponsor: University of Hawaii • Aim: Disease control
Last updated Jun 27, 2026 13:04 UTC
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New pill combo aims to crush bad cholesterol
Disease control OngoingThis 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…
Phase: PHASE2 • Sponsor: AstraZeneca • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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Gene therapy after stem cell transplant shows promise for rare brain disease
Disease control OngoingThis 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…
Phase: PHASE1, PHASE2 • Sponsor: Forge Biologics, Inc • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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New study tracks Long-Term safety of rare disease treatment
Disease control OngoingThis 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…
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Yerba mate extract tested for metabolic boost in overweight adults
Disease control ENROLLING_BY_INVITATIONThis 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 …
Phase: NA • Sponsor: Universidade Federal da Fronteira Sul • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Exercise as medicine: 10-week program targets diabetes and high blood pressure in seniors
Disease control OngoingThis 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 …
Phase: NA • Sponsor: Universidade do Porto • Aim: Disease control
Last updated Jun 27, 2026 12:37 UTC
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Can a lifestyle clinic help people with obesity? large trial launches
Disease control ENROLLING_BY_INVITATIONThis 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…
Phase: NA • Sponsor: University of Alabama at Birmingham • Aim: Disease control
Last updated Jun 27, 2026 12:36 UTC
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New study aims to boost cholesterol treatment in heart disease patients
Disease control ENROLLING_BY_INVITATIONThis 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 …
Phase: NA • Sponsor: Brigham and Women's Hospital • Aim: Disease control
Last updated Jun 27, 2026 12:35 UTC
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New hope for CAH: drug may slash steroid use
Disease control OngoingThis 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…
Phase: PHASE3 • Sponsor: Neurocrine Biosciences • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC
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Can a brazilian diet plus supplements tame inherited high cholesterol?
Disease control OngoingThis 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…
Phase: NA • Sponsor: Hospital do Coracao • Aim: Disease control
Last updated Jun 27, 2026 12:31 UTC
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New pill could help control high cholesterol – study underway
Disease control OngoingThis 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…
Phase: PHASE2, PHASE3 • Sponsor: AstraZeneca • Aim: Disease control
Last updated Jun 27, 2026 12:31 UTC
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New pill could tame inherited high cholesterol in major trial
Disease control OngoingThis 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…
Phase: PHASE3 • Sponsor: AstraZeneca • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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New drug aims to slash Sky-High cholesterol in rare genetic disease
Disease control OngoingThis 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 …
Phase: PHASE3 • Sponsor: Visirna Therapeutics HK Limited • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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New cholesterol drug enters early human testing
Disease control OngoingThis early-stage study tests a new drug called AZD1705 in adults with high cholesterol (dyslipidemia). The main goal is to check if the drug is safe and how the body processes it. About 98 participants will receive either the drug or a placebo. This research helps determine if th…
Phase: PHASE1 • Sponsor: AstraZeneca • Aim: Disease control
Last updated Jun 27, 2026 12:28 UTC
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New enzyme therapy for fabry disease tested in Real-World setting
Disease control OngoingThis study follows 60 adults with Fabry disease who are receiving pegunigalsidase-alfa, a newer enzyme replacement therapy. Researchers want to see how well it works in everyday medical practice, especially for kidney function. Participants will be treated for 2 years at speciali…
Sponsor: Universität Münster • Aim: Disease control
Last updated Jun 27, 2026 12:26 UTC
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Eye injection may save sight in kids with rare batten disease
Disease control OngoingThis study tests whether injecting a replacement enzyme directly into the eye can slow or stop vision loss in children with CLN2 Batten disease, a rare genetic disorder that causes blindness and brain damage. Five children aged 2 to 6 who already receive brain infusions of the en…
Phase: PHASE1, PHASE2 • Sponsor: David L Rogers, MD • Aim: Disease control
Last updated Jun 27, 2026 12:26 UTC
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New pill could slash heart risks for millions
Disease control OngoingThis Phase 3 trial is testing an oral drug called MK-0616 (enlicitide decanoate) in about 14,550 people at high risk for heart problems. The drug aims to lower cholesterol and reduce the chance of major events like heart attacks, strokes, and emergency artery procedures. Particip…
Phase: PHASE3 • Sponsor: Merck Sharp & Dohme LLC • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Diet or drugs? new study tests if fasting beats pills for fatty liver disease
Disease control ENROLLING_BY_INVITATIONThis study tests whether a Mediterranean diet combined with intermittent fasting can reduce liver scarring (fibrosis) better than the weight-loss medication naltrexone/bupropion. About 70 adults with overweight or obesity and heart or metabolic risks (like type 2 diabetes or high…
Phase: PHASE4 • Sponsor: Carmen Dietvorst • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Experimental spinal injection aims to keep kids with rare disease moving
Disease control OngoingThis study tests a drug called SHP611, given as a spinal injection, in 36 children with late infantile metachromatic leukodystrophy (MLD), a rare and severe brain disease. The main goal is to see if the treatment helps children keep their ability to walk and move for as long as p…
Phase: PHASE2 • Sponsor: Shire • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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Gene-Editing heart therapy under 15-Year watch
Disease control ENROLLING_BY_INVITATIONThis study follows 116 people who received an experimental gene-editing therapy for heart disease or high cholesterol. Researchers will monitor them for 15 years to track any side effects and how well the treatment controls cholesterol levels. The goal is to understand the long-t…
Sponsor: Verve Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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New oil therapy could slash Life-Threatening events in kids with rare metabolic disease
Disease control OngoingThis study tests whether triheptanoin, a special oil, can reduce major clinical events (like severe low blood sugar or heart problems) in children with long-chain fatty acid oxidation disorders (LC-FAOD), a rare condition where the body can't break down certain fats for energy. A…
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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New study tests video coaching to help older adults shed pounds and prevent disease
Disease control OngoingThis study tests a video-based healthy lifestyle program for adults aged 50 to 74 who are overweight and have conditions like prediabetes, type 2 diabetes, or high blood pressure. Participants use a tablet, scale, and activity tracker at home, and some get extra support from a co…
Phase: NA • Sponsor: University of Illinois at Chicago • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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Which airway method saves more kids? major trial aims to find out
Disease control TerminatedThis study tests three ways emergency responders help children breathe: a bag-mask, a throat tube, or a breathing tube. It includes 3,000 children under 18 with cardiac arrest, severe injury, or breathing failure. The goal is to see which method leads to more days alive and out o…
Phase: NA • Sponsor: Ohio State University • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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New hope for fabry patients with kidney failure: drug dosing study underway
Disease control OngoingThis study tests the safety and how the body processes migalastat in 14 adults with Fabry disease who have severe kidney impairment or are on dialysis. Participants take migalastat capsules, and researchers measure drug levels in blood and dialysate. The goal is to ensure proper …
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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Gene therapy injection aims to halt fatal brain disease in babies
Disease control OngoingThis early-phase trial is testing a single-dose gene therapy called LY3884961 in 7 infants with Type 2 Gaucher disease, a severe and rapidly fatal genetic disorder. The therapy is injected into the fluid around the brain and spinal cord to deliver a working copy of the faulty gen…
Phase: PHASE1, PHASE2 • Sponsor: Prevail Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:01 UTC
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Nurse-Led teams may cut hospital returns for patients with multiple illnesses
Disease control OngoingThis study tests whether a nurse practitioner-led team can improve care for adults with two or more chronic conditions like heart failure, diabetes, or kidney disease. Participants are randomly assigned to either the nurse-led team or usual care. The team reviews medications to e…
Phase: NA • Sponsor: National Taiwan University Hospital • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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New gene therapy targets Parkinson's in patients with specific gene flaw
Disease control OngoingThis study tests a new gene therapy called LY3884961 in 32 people with moderate to severe Parkinson's disease who have a specific genetic change (GBA1 mutation). The treatment is given as a single injection into the fluid around the brain and spinal cord. The main goal is to chec…
Phase: PHASE1, PHASE2 • Sponsor: Prevail Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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Digital health program could transform diabetes care for seniors with multiple illnesses
Disease control ENROLLING_BY_INVITATIONThis study tests a 6-month digital care program for adults aged 50+ with type 2 diabetes and at least one other chronic condition like high blood pressure or kidney disease. The program combines online education, phone support, a mobile health app, home visits, and personalized c…
Phase: NA • Sponsor: Chang Gung University of Science and Technology • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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Heart attack prevention hope for rare cholesterol disorder
Disease control OngoingThis study tracks 73 adults with homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder causing extremely high cholesterol and early heart disease. Researchers will compare major cardiovascular events (like heart attacks and strokes) during the first three years…
Sponsor: Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC
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New cholesterol drug inclisiran tested in everyday patients
Disease control OngoingThis study follows about 847 adults with high cholesterol or mixed dyslipidemia who are starting inclisiran, a newer cholesterol-lowering drug. Researchers want to see how much LDL cholesterol drops after 10 months and how well patients stick with the treatment in real-world sett…
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 09:02 UTC
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Gene therapy shot aims to fix gaucher disease in kids
Disease control OngoingThis early-phase trial tests a single intravenous dose of LY-M001 gene therapy in 9 children (ages 6 to 17) with type 1 Gaucher disease. The goal is to see if it is safe and can improve key symptoms like liver size and blood markers. Researchers will monitor participants for side…
Phase: EARLY_PHASE1 • Sponsor: Shanghai Jiao Tong University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 09:01 UTC
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Computer alert aims to boost cholesterol treatment in heart patients
Disease control OngoingThis study tests whether an electronic alert in the medical record can help doctors prescribe better cholesterol-lowering medications for heart disease patients with high LDL. About 400 patients from a single hospital will be randomly assigned to have their doctor receive the ale…
Phase: NA • Sponsor: Brigham and Women's Hospital • Aim: Disease control
Last updated Jun 27, 2026 08:12 UTC
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New shot aims to tame high cholesterol and triglycerides
Disease control OngoingThis study tests an investigational drug called BW-00112 in 145 adults with mixed dyslipidemia, a condition where levels of certain blood fats are too high. Participants receive injections of the drug or a placebo over several months while continuing their usual statin medication…
Phase: PHASE2 • Sponsor: Shanghai Argo Biopharmaceutical Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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New stem cell approach aims to tame rare genetic diseases
Disease control OngoingThis study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to get the donor cells to take hold while keeping side effects low. Participants receive chemotherapy drugs before the transplant to prepare their bo…
Phase: PHASE2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC
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Pharmacists take on heart disease: new study tests community-based risk reduction
Disease control OngoingThis study tests whether a pharmacist-led program can help people reduce their risk of heart disease. Over 1,000 adults with conditions like diabetes, high blood pressure, or obesity are taking part. Pharmacists use a step-by-step guide to assess risk and support lifestyle change…
Phase: NA • Sponsor: University of Alberta • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC
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One-Time gene injection aims to slow rare fatal brain disease in kids
Disease control OngoingThis early-stage study tests a one-time gene therapy injection into the spinal fluid of children aged 1-18 with CLN7 Batten disease, a rare and fatal genetic brain disorder. The main goal is to check safety, but researchers will also measure changes in movement, thinking, and vis…
Phase: PHASE1 • Sponsor: Benjamin Greenberg • Aim: Disease control
Last updated Jun 27, 2026 08:07 UTC
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Gene therapy aims to free patients from daily steroids for rare adrenal disorder
Disease control OngoingThis early-phase trial tests a gene therapy called BBP-631 for classic congenital adrenal hyperplasia (CAH), a genetic condition that forces patients to take daily steroids. The therapy uses a harmless virus to deliver a working copy of the faulty gene. Only 8 adults are enrolled…
Phase: PHASE1, PHASE2 • Sponsor: Adrenas Therapeutics Inc • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC
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Teen artery stiffness study tests carnitine supplement for heart health
Disease control OngoingThis study looks at whether a protein supplement called carnitine can reduce hardening of the arteries in teenagers at high risk for future heart disease. About 90 teens aged 11-21 with high triglycerides will take carnitine or a placebo. Researchers will measure artery stiffness…
Phase: PHASE2 • Sponsor: Baylor College of Medicine • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Diabetes snack break: golden rice cookies take on bad cholesterol
Disease control OngoingThis study tests whether a cookie made from Golden Rice and red betel leaf can improve cholesterol and triglyceride levels in adults with type 2 diabetes. Over 28 days, 102 participants will eat the cookies daily, and their blood fat levels will be measured before and after. The …
Phase: NA • Sponsor: Hasanuddin University • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC
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Heart stent patients: which statin combo keeps cholesterol lower longer?
Disease control OngoingThis study compares two high-dose statin/ezetimibe combinations (rosuvastatin/ezetimibe vs. atorvastatin/ezetimibe) in 200 adults who have received a drug-eluting stent for coronary artery disease. The goal is to see which combo better maintains LDL cholesterol below 55 mg/dL ove…
Phase: PHASE4 • Sponsor: Yonsei University • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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Gene therapy boost for krabbe patients after transplant
Disease control OngoingThis early-phase trial tests a single infusion of a gene therapy called FBX-101 in 9 people with Krabbe disease who have already received a stem cell transplant. The therapy uses a harmless virus to deliver a working copy of the GALC gene, aiming to improve motor function and saf…
Phase: PHASE1, PHASE2 • Sponsor: Forge Biologics, Inc • Aim: Disease control
Last updated Jun 27, 2026 07:59 UTC
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Gene therapy for gaucher disease: 5-year safety check begins
Disease control OngoingThis study follows 75 people with Gaucher disease type 1 who previously received FLT201 gene therapy. Researchers will monitor them for 5 years to see if the treatment remains safe and if its effects last. The goal is to understand whether a single dose can provide long-term dise…
Phase: PHASE1, PHASE2 • Sponsor: Spur Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Last-resort drug offered to kids with deadly cholesterol condition
Disease control APPROVED_FOR_MARKETINGThis program provides evinacumab to children with homozygous familial hypercholesterolemia (HoFH) or severe high triglycerides when standard treatments don't work. The drug helps lower dangerous fat levels in the blood, but patients must keep taking it long-term to control their …
Sponsor: Regeneron Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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Could a daily pill replace IV infusions for gaucher type 3?
Disease control OngoingThis phase 3 trial tests venglustat, a daily oral drug, against the standard IV treatment Cerezyme in 43 people with Gaucher disease type 3. Participants must have been on enzyme replacement therapy for at least 3 years. The goal is to see if venglustat can stabilize or improve n…
Phase: PHASE3 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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Gene therapy for rare brain diseases: 5-Year safety check begins
Disease control OngoingThis study follows 7 people who previously received a gene therapy called AXO-AAV-GM2 for Tay-Sachs or Sandhoff disease — rare, fatal genetic disorders that destroy nerve cells. Researchers will track them for up to 5 years to see if the treatment remains safe and whether it help…
Sponsor: Terence Flotte • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC
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Can a statin and diet tweak protect hearts of epilepsy patients on High-Fat diets?
Disease control ENROLLING_BY_INVITATIONThis study looks at whether a statin drug (atorvastatin) and swapping some saturated fats for healthier ones can safely lower cholesterol in adults with epilepsy who have been on a modified Atkins diet for at least a year. The diet helps control seizures but can raise cholesterol…
Phase: PHASE1, PHASE2 • Sponsor: Johns Hopkins University • Aim: Disease control
Last updated Jun 27, 2026 07:54 UTC
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Mango a day keeps prediabetes at bay?
Disease control OngoingThis study tests whether eating 1.5 cups of fresh mango daily for 12 weeks can improve blood sugar, cholesterol, and inflammation in people with prediabetes. Thirty-one participants will eat mango for 12 weeks, then take a break, and then avoid mango for another 12 weeks. Researc…
Phase: NA • Sponsor: Loma Linda University • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC
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Gene therapy for rare brain disease tracked over years
Disease control OngoingThis study follows 10 people with CLN6 Batten disease who received a single dose of gene therapy (AT-GTX-501) in an earlier trial. Researchers are checking long-term safety and how the disease progresses over time. No new treatment is given in this follow-up.
Sponsor: Emily de los Reyes • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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Could a urea cycle drug help MCAD patients fast longer?
Disease control TerminatedThis study tests whether sodium phenylbutyrate, a drug already approved for another condition, can help people with MCAD deficiency (a genetic disorder that affects fat breakdown). About 24 participants aged 10 and older will take the drug for 4 weeks. Researchers will check for …
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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Can a drug tame excess cortisol? new trial tests metyrapone
Disease control OngoingThis phase 2 trial tests whether metyrapone, a drug that lowers cortisol production, is safe and effective for people with mild autonomous cortisol secretion (MACS). About 30 participants will take the drug for a period, and researchers will track side effects and cortisol levels…
Phase: PHASE2 • Sponsor: Mayo Clinic • Aim: Disease control
Last updated Jun 27, 2026 07:51 UTC
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Gene therapy hope for rare brain disease in children
Disease control OngoingThis study tests a single infusion of OTL-200 gene therapy in 6 people with late juvenile metachromatic leukodystrophy (MLD), a rare genetic disease that damages the brain and nerves. The therapy uses the patient's own blood stem cells, modified to produce a missing enzyme. Resea…
Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Jun 26, 2026 13:33 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis 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…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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Brain scan breakthrough could save babies from rare disease
Diagnosis OngoingThis study looks at whether a special brain scan called diffusion tensor imaging (DTI) can help doctors find Krabbe disease in newborns and decide who needs urgent treatment. About 100 babies with a positive newborn screen or family history of Krabbe disease will be scanned and f…
Sponsor: University of Pittsburgh • Aim: Diagnosis
Last updated Jun 27, 2026 09:11 UTC
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AI and nudge tactics aim to catch hidden high cholesterol
Diagnosis ENROLLING_BY_INVITATIONThis study uses a machine-learning tool to scan electronic health records and flag patients likely to have familial hypercholesterolemia (FH), a genetic condition causing very high cholesterol. Researchers then test a simple referral system that automatically schedules a visit wi…
Phase: NA • Sponsor: University of Pennsylvania • Aim: Diagnosis
Last updated Jun 27, 2026 09:10 UTC
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New DNA reader cracks tough genetic mysteries
Diagnosis OngoingThis study tests a new DNA-reading technology called ultra-long read sequencing to find hidden genetic changes that standard tests miss. Researchers will analyze blood samples from 15 patients with known or suspected genetic variants in difficult-to-read DNA regions. The goal is …
Phase: NA • Sponsor: IRCCS Azienda Ospedaliero-Universitaria di Bologna • Aim: Diagnosis
Last updated Jun 27, 2026 08:09 UTC
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New york program offers extra screening for 100,000 newborns
Diagnosis ENROLLING_BY_INVITATIONScreenPlus is a large pilot program that offers families the option to have their newborn screened for a panel of rare genetic disorders, in addition to standard newborn screening. The study aims to screen 100,000 infants born at eight hospitals in New York. Researchers will eval…
Sponsor: Albert Einstein College of Medicine • Aim: Diagnosis
Last updated Jun 26, 2026 16:15 UTC
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Could a fatty acid drug stop dangerous sugar crashes in rare disease?
Prevention TerminatedThis 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…
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Prevention
Last updated Jul 04, 2026 00:00 UTC
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Can a digital coach help you avoid heart disease? new study aims to find out
Prevention ENROLLING_BY_INVITATIONThis study is testing a digital health platform called STELA that creates personalized lifestyle plans to reduce risk factors for heart disease, like obesity, high cholesterol, and insulin resistance. About 100 adults without existing heart disease will use the platform for a yea…
Phase: NA • Sponsor: Nemocnice AGEL Trinec-Podlesi a.s. • Aim: Prevention
Last updated Jun 27, 2026 12:25 UTC
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Ancient chinese herb berberine tested to stop heart disease and diabetes in its tracks
Prevention OngoingThis large, phase 4 trial is testing whether berberine, a natural compound, can prevent heart disease and diabetes in over 2,000 high-risk adults in China. Participants take berberine or a placebo twice daily, plus receive lifestyle advice. The study tracks blood sugar and heart …
Phase: PHASE4 • Sponsor: China National Center for Cardiovascular Diseases • Aim: Prevention
Last updated Jun 27, 2026 08:07 UTC
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Dairy workers test special shoes to stop dangerous slips
Prevention OngoingThis study tests whether special slip-resistant shoes can help dairy workers avoid slipping on wet, slippery floors. About 22 full-time employees at a Danish dairy will wear different shoes and rate how slippery they feel each day. The goal is to find footwear that reduces fall r…
Phase: NA • Sponsor: Aalborg University • Aim: Prevention
Last updated Jun 27, 2026 08:04 UTC
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New vitamin supplements aim to make life easier for kids with rare metabolic disorders
Symptom relief OngoingThis 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…
Phase: NA • Sponsor: NeoteriQ Ltd. • Aim: Symptom relief
Last updated Jun 27, 2026 13:00 UTC
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Exercise program aims to boost fitness in fabry patients
Symptom relief ENROLLING_BY_INVITATIONThis study is testing whether a 12-week physiotherapy and exercise program can improve physical fitness and quality of life in adults with Fabry disease. Fifteen participants will do regular exercise sessions using body weight, resistance bands, and increased daily activity. The …
Phase: NA • Sponsor: General University Hospital, Prague • Aim: Symptom relief
Last updated Jun 27, 2026 12:01 UTC
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Hope for fabry pain: new drug trial targets nerve and belly discomfort
Symptom relief OngoingThis study tests whether venglustat can reduce nerve pain in the arms and legs, and belly pain, in people with Fabry disease. Participants are 16 or older and have not had Fabry treatment for at least 6 months. The trial lasts 12 months, with visits every 3 months, and compares v…
Phase: PHASE3 • Sponsor: Sanofi • Aim: Symptom relief
Last updated Jun 27, 2026 09:01 UTC
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Hospital patients get a game plan to beat bedrest
Symptom relief TerminatedThis study aimed to see if making physical activity into a game could help older hospital patients move more. Participants would have received daily text messages, set step goals, earned points, and worn a Fitbit to track their steps. The study was withdrawn before any patients w…
Phase: NA • Sponsor: University of Pennsylvania • Aim: Symptom relief
Last updated Jun 27, 2026 08:07 UTC
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NIH launches major study to unravel mysteries of rare genetic diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis 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…
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Jul 04, 2026 00:00 UTC
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Rare disease drug safety tracked in new observational study
Knowledge-focused OngoingThis 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…
Sponsor: BioMarin Pharmaceutical • Aim: Knowledge-focused
Last updated Jul 02, 2026 00:00 UTC
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Massive korean study maps the overlap of heart, kidney, and metabolic diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis 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 …
Sponsor: Novo Nordisk A/S • Aim: Knowledge-focused
Last updated Jul 01, 2026 00:00 UTC
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New cholesterol drug candidate tested in healthy volunteers
Knowledge-focused OngoingThis 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…
Phase: PHASE1 • Sponsor: AJU Pharm Co., Ltd. • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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Leqvio safety under the microscope: Real-World data on 585 patients
Knowledge-focused OngoingThis 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…
Sponsor: Novartis Pharmaceuticals • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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Scientists create a 'Lipid Library' to unlock secrets of rare cholesterol conditions
Knowledge-focused OngoingThis 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…
Sponsor: University of Texas Southwestern Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:06 UTC
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Scientists track rare liver diseases in kids to unlock clues
Knowledge-focused TerminatedThis 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…
Sponsor: Arbor Research Collaborative for Health • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:03 UTC
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Major study on rare childhood liver diseases halted
Knowledge-focused TerminatedThis 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 …
Sponsor: Arbor Research Collaborative for Health • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:02 UTC
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Real-World study tracks cholesterol drug inclisiran in heart patients
Knowledge-focused OngoingThis 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 …
Sponsor: Novartis Pharmaceuticals • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:01 UTC
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Massive genetic study aims to unlock secrets of rare metabolic diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis 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 …
Sponsor: Region Stockholm • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:38 UTC
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Study on COVID-19 and metabolic disorders pulled before it began
Knowledge-focused TerminatedThis 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…
Sponsor: University Hospital, Lille • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:37 UTC
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STREAM trial: should seniors ditch statins?
Knowledge-focused OngoingThis 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 …
Phase: NA • Sponsor: Insel Gruppe AG, University Hospital Bern • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:35 UTC
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Taiwan study sheds light on rare fabry mutation and treatment effects
Knowledge-focused OngoingThis 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…
Sponsor: Sanofi • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:32 UTC
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450 babies help scientists unlock secrets of healthy eating
Knowledge-focused OngoingThis 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…
Sponsor: University of Illinois at Urbana-Champaign • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:28 UTC
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Scientists hunt for hidden genes behind Ultra-Low cholesterol
Knowledge-focused OngoingThis study aims to find new genes that cause very low cholesterol levels (a condition called FHBL). Researchers are recruiting 435 people with low cholesterol and their family members to analyze their DNA. The goal is to discover new targets for understanding and potentially mana…
Phase: NA • Sponsor: Nantes University Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:24 UTC
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Rare brain disease study seeks to unlock mysteries of atypical TPP1 deficiency
Knowledge-focused OngoingThis study follows 5 people with a rare, late-onset form of TPP1 deficiency (a brain disease) to track how their symptoms change over time. Researchers will use tests like brain scans, eye exams, and movement assessments to better understand the condition. The goal is to gather i…
Sponsor: Children's Hospital of Orange County • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC
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New study tracks bempedoic acid combo in asian and latin american patients
Knowledge-focused OngoingThis study is observing over 2,500 adults with high cholesterol who are taking a combination of bempedoic acid and ezetimibe in routine medical care. Researchers are collecting data on side effects and heart risk to better understand how the treatment works in Asian and Latin Ame…
Sponsor: Daiichi Sankyo • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:02 UTC
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Heart damage in fabry disease: new study tracks silent progression
Knowledge-focused OngoingThis study follows 31 Chinese adults with a specific genetic mutation (IVS4+919G>A) that causes Fabry disease, a condition where harmful substances build up and damage organs, especially the heart. Researchers use advanced heart scans and blood tests to track how the disease prog…
Sponsor: Chinese University of Hong Kong • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:02 UTC
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Scientists hunt for Muscle's secret Fat-Burning signals
Knowledge-focused OngoingThis study looks at how exercise changes the signals muscles send to the rest of the body, especially those that help burn fat. Researchers will compare obese, lean, and athletic participants to find proteins or genetic codes released from muscle during exercise. The goal is to b…
Phase: NA • Sponsor: AdventHealth Translational Research Institute • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:02 UTC
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New cholesterol drug AD-117: does food change how it works?
Knowledge-focused ENROLLING_BY_INVITATIONThis early-stage trial is testing a new drug called AD-117, which is being developed for mixed dyslipidemia (abnormal cholesterol levels). The study will give a single dose to 60 healthy adults, either with or without food, to see how food changes the drug's levels in the blood a…
Phase: PHASE1 • Sponsor: Addpharma Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:01 UTC
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Big data takes on heart disease: can a toolbox of info boost statin use?
Knowledge-focused OngoingThis study tests whether a 'toolbox' of educational materials—including report cards for doctors, patient handouts, and an online decision aid—can increase the use of cholesterol-lowering statins in communities with high rates of heart disease. Researchers will track prescription…
Phase: NA • Sponsor: Institute for Clinical Evaluative Sciences • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:01 UTC
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Home infusions may help patients stick to treatment
Knowledge-focused OngoingThis study looks at whether people with Fabry, Gaucher, or Hunter disease are more likely to continue their IV treatment when it's given at home versus at a hospital. Researchers will review existing data from 222 patients in Mexico. No new treatments are given; the goal is to un…
Sponsor: Takeda • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC
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Thousands tracked: does bempedoic acid work in real life?
Knowledge-focused OngoingThis study is observing 5000 adults with high cholesterol who are taking bempedoic acid (alone or with ezetimibe) as part of their normal medical care. Researchers will collect data on cholesterol levels, heart events, side effects, and quality of life over one year. The goal is …
Sponsor: Daiichi Sankyo Europe, GmbH, a Daiichi Sankyo Company • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:03 UTC
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Hospital staff study reveals hidden heart risk?
Knowledge-focused OngoingThis study checks how common high levels of lipoprotein(a) — a genetic risk factor for heart disease — are among hospital staff and their families. About 2800 participants will give blood samples and be followed over time to see if high levels lead to more heart problems. The goa…
Sponsor: Changi General Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:02 UTC
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New registry aims to unravel mysteries of statin side effects
Knowledge-focused OngoingThis registry is following 1,000 people who have trouble tolerating statins, a common cholesterol-lowering drug. Researchers will track how often treatments are changed, cholesterol levels, and quality of life over time. The goal is to better understand this condition and improve…
Sponsor: University of Leipzig • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:06 UTC
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Soy showdown: does tofu lower cholesterol as well as soy protein powder?
Knowledge-focused OngoingThis study reviews past clinical trials to see if different soy foods (like tofu, tempeh, and soy drinks) lower cholesterol as effectively as isolated soy protein. The goal is to help update dietary guidelines and health claims. No new participants are being enrolled; the researc…
Sponsor: John Sievenpiper • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC
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3,000 haitians join Long-Term heart health study
Knowledge-focused OngoingThis study follows about 3,000 adults in Port-au-Prince, Haiti, to learn how common heart disease risk factors like high blood pressure, diabetes, and smoking are, and how they lead to heart attacks, strokes, and other heart problems. Researchers will track participants for about…
Sponsor: Weill Medical College of Cornell University • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:04 UTC
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New study aims to find hidden heart disease in families
Knowledge-focused ENROLLING_BY_INVITATIONThis study tests two new ways to help people with familial hypercholesterolemia (a genetic high cholesterol condition) get their family members screened. About 800 adults from Penn Medicine will be assigned to receive automated reminders, a patient navigator, or usual care. The g…
Phase: NA • Sponsor: Northwestern University • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:03 UTC
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Breastfeeding's hidden heart impact on moms with high cholesterol
Knowledge-focused OngoingThis study looks at how breastfeeding changes cholesterol and heart disease risk in women with familial hypercholesterolemia (FH), a genetic condition causing high cholesterol. Researchers will follow 50 pregnant women with and without FH from late pregnancy to one year after bir…
Sponsor: Oslo University Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:02 UTC
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Talking it out: study tests if a phone call can tame inherited high cholesterol
Knowledge-focused OngoingThis study looks at whether a motivational interview—a 20-40 minute phone or video chat—can help people with familial hypercholesterolemia (FH) get their cholesterol checked and talk to family about screening. About 300 people with suspected or confirmed FH will be randomly assig…
Phase: PHASE3 • Sponsor: Essentia Health • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC
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Silent tumors may still harm the heart, new study warns
Knowledge-focused ENROLLING_BY_INVITATIONThis study looks at people with rare adrenal tumors (pheochromocytomas and paragangliomas) that do not produce excess hormones. Researchers want to see if these 'silent' tumors still cause heart problems, blood pressure changes, or metabolic issues like diabetes and high choleste…
Sponsor: Laikο General Hospital, Athens • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC
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120,000-Person study hopes to crack the code of chronic illness
Knowledge-focused TerminatedThis observational study plans to enroll 120,000 people with various chronic diseases like pancreatitis, diabetes, and arthritis. Researchers will collect blood samples and health data to study how genetics and lifestyle affect disease progression. The goal is to find common dise…
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:08 UTC
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Fabry patients monitored in new enzyme therapy study
Knowledge-focused ENROLLING_BY_INVITATIONThis study watches 100 people with Fabry disease who are already taking enzyme replacement therapy as part of their regular care. Researchers want to see how the treatment affects their health over time by checking things like kidney function and pain levels. The study does not t…
Sponsor: NPO Petrovax • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:07 UTC
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New program aims to catch hidden cholesterol disorder before heart attacks strike
Knowledge-focused ENROLLING_BY_INVITATIONThis study tests new tools to help primary care doctors diagnose familial hypercholesterolemia (FH), a genetic condition that causes very high cholesterol and early heart attacks. About 532 clinicians will use special checklists and reminders during patient visits. The goal is to…
Phase: NA • Sponsor: Geisinger Clinic • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:06 UTC
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Online video aims to ease tough transition for teens with rare hormone disorder
Knowledge-focused TerminatedThis pilot study aimed to see if a self-paced virtual education video could help adolescents and young adults (ages 16-22) with congenital adrenal hyperplasia (CAH) become more independent in managing their health. Participants would have been split into two groups: one watching …
Phase: NA • Sponsor: National Institutes of Health Clinical Center (CC) • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:03 UTC
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Rare disease diagnosis boost: new study eyes key clues in ASMD patients
Knowledge-focused OngoingThis study looks at symptoms and lab tests to help doctors better diagnose acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease, a rare genetic disorder. Researchers will track changes in spleen and liver size using ultrasound over 12 months in 7 patients. …
Sponsor: Sohag University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:03 UTC
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Scientists investigate link between blood fats and sickle cell crises
Knowledge-focused OngoingThis study looks at how different types of fats in the blood (like cholesterol) are linked to complications in adults with sickle cell disease. Researchers will follow 116 participants from the French Caribbean islands to track their fat levels over time and during acute events l…
Phase: NA • Sponsor: Centre Hospitalier Universitaire de la Guadeloupe • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:03 UTC
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New study aims to measure how CLN2 disease affects Children's development
Knowledge-focused ENROLLING_BY_INVITATIONThis study looks at how children with CLN2 Batten disease develop over time, including their thinking, language, and motor skills. Researchers will compare children who receive treatment (cerliponase alfa) with those who do not. The goal is to find better ways to measure the dise…
Sponsor: Jessica Scherr • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC
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Plant protein swap may cut heart & diabetes risk – but how much?
Knowledge-focused OngoingThis study is a review of many smaller trials that looked at what happens when people replace animal protein (like meat, dairy, eggs) with plant protein (like beans, nuts, seeds). The goal is to see if this simple swap can improve blood sugar, cholesterol, blood pressure, weight,…
Sponsor: John Sievenpiper • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:53 UTC
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Amino acid drink tested for liver fat in obese kids – study never started
Knowledge-focused TerminatedThis study planned to test whether an 8-week course of a flavored drink containing essential amino acids plus arginine could reduce liver fat and improve metabolism in obese children aged 7-10. The researchers intended to measure changes in liver fat, blood fats, insulin sensitiv…
Phase: NA • Sponsor: Arkansas Children's Hospital Research Institute • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:53 UTC
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Can better education ease statin side effect worries?
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to develop and test educational content that explains statin side effects, including placebo and nocebo effects. Researchers will gather feedback from 30 adults with cardiovascular risk factors through surveys and interviews. The goal is to see if this content is …
Phase: NA • Sponsor: Weill Medical College of Cornell University • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:45 UTC
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Fish oil and exercise combo study for obesity inflammation pulled before start
Knowledge-focused TerminatedThis pilot study aimed to see if taking fish oil supplements could improve gut bacteria and make exercise more effective at reducing inflammation in people with obesity. The study was withdrawn before any participants were enrolled, so no results or conclusions are available.
Phase: NA • Sponsor: Texas Tech University • Aim: Knowledge-focused
Last updated Jun 26, 2026 16:45 UTC