New Treatments & Clinical Trials for Hypercholesterolemia

Hypercholesterolemia — high cholesterol — affects about 93 million U.S. adults and significantly increases cardiovascular risk. Treatment typically starts with statins, which can lower LDL cholesterol by 30-50%. Many people now combine statins with newer drugs like ezetimibe or PCSK9 inhibitors to reach aggressive LDL targets, especially after heart attacks or strokes.

What's actually going on in research

Trials are testing gene-silencing drugs that work for months after a single injection, new oral PCSK9 inhibitors that could replace twice-monthly shots, drugs targeting lipoprotein(a) — a genetic risk factor statins don't touch — and medications that address triglycerides and metabolic dysfunction. Researchers are also studying early treatment in younger adults with familial hypercholesterolemia to prevent lifelong cardiovascular damage.

What to know before you search

Eligibility typically depends on your LDL cholesterol level, whether you have cardiovascular disease, prior treatments tried, and genetic testing results if familial hypercholesterolemia is suspected.

What types of trials are currently open

• LDL-lowering trials — Testing new drugs or drug combinations to see how much they reduce LDL cholesterol and whether they prevent heart attacks and strokes.
• Lipoprotein(a) trials — Studies of drugs targeting lipoprotein(a), measuring whether lowering this genetic risk factor prevents cardiovascular events.
• Gene-silencing trials — Testing RNA-based drugs that reduce cholesterol production in the liver, typically requiring injections every few months.
• Familial hypercholesterolemia trials — Studies in people with inherited high cholesterol, often starting treatment earlier to prevent cardiovascular disease.
• Triglyceride trials — Testing drugs for people with high triglycerides, which often accompany high cholesterol and increase cardiovascular risk.