Familial hypercholesterolemia

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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…