Hypercholesterolemia means having too much cholesterol in the blood, which increases the risk of heart attack and stroke. Statins have been the backbone of treatment for decades, but newer drugs now offer additional ways to lower cholesterol for people who don't reach their goals on statins alone or can't tolerate them.
What's actually going on in research
Trials are testing PCSK9 inhibitors given as shots or pills, inclisiran (a twice-yearly injection that silences cholesterol production), bempedoic acid (a non-statin pill), and gene therapy approaches that aim to lower cholesterol permanently with a single treatment. Research also focuses on people with familial hypercholesterolemia, who inherit very high cholesterol and need aggressive treatment from childhood.
Gene editing and silencing
CRISPR-based gene editing and RNA therapies are being tested to permanently lower cholesterol with one or a few treatments. Early results show drops of 50% or more lasting months to years.
Oral PCSK9 inhibitors
Pills that block PCSK9 are in trials as an alternative to the current PCSK9 shots. They could make powerful cholesterol lowering more convenient for people who need it.
Familial hypercholesterolemia in children
Trials are testing earlier and more aggressive treatment for children who inherit very high cholesterol. The goal is to prevent artery damage before it starts.
What to know before you search
Eligibility typically depends on cholesterol levels, whether you have cardiovascular disease, family history of heart disease, and response to or tolerance of statins.
What types of trials are currently open
- Drug comparison trials — Testing whether newer drugs like PCSK9 inhibitors or bempedoic acid work better than statins or add benefit when combined with them.
- Gene therapy trials — Testing single treatments that use gene editing or RNA silencing to lower cholesterol long-term without daily pills.
- Cardiovascular outcome trials — Following people for years to see if a drug prevents heart attacks and strokes, not just lowers cholesterol numbers.
- Familial hypercholesterolemia trials — Testing treatments specifically for people with inherited high cholesterol, who often need multiple drugs.
- Statin intolerance trials — Testing non-statin treatments for people who can't tolerate statins due to muscle pain or other side effects.
Recently added Hypercholesterolemia trials
Take a blood thinner with aspirin to prevent leg damage
The goal of this \*\*clinical trial\*\* is to compare the effectiveness of \*\*low-dose rivaroxaban plus aspirin versus aspirin alone\*\* in patients aged 40-70 years with \*\*chronic limb-threatening ischemia, diabetes mellitus, hypertension, and hypercholesterolemia\*\*. The main questions it aims to answer are: * Does low-dose rivaroxaban plus aspirin reduce \*\*major adverse limb events (MALE)\*\*, such as acute limb ischemia and amputation? * Does the combination therapy reduce \*\*major adverse cardiovascular events (MACE)\*\*, such as myocardial infarction and stroke, and improve limb-related outcomes? Researchers will compare \*\*low-dose rivaroxaban (2.5 mg twice daily) plus aspirin (75-100 mg daily)\*\* with \*\*aspirin alone\*\* to see if combination therapy provides better outcomes over a period of \*\*6 months\*\*. Participants will: * Undergo baseline clinical assessment, blood tests, ABPI/TBI, and Doppler/duplex assessment. * Be randomly assigned to either the rivaroxaban plus aspirin group or the aspirin-alone group. * Attend follow-up visits at specified intervals for assessment of \*\*WIFI score, ABPI, wound healing, MALE, MACE, and quality of life\*\*. * Be assessed for treatment compliance, side effects, and bleeding during follow-up.
Work with a community health worker to address your daily health needs
The goal of the Arizona Community Engagement Alliance (AZ-CEAL) will be to combine social care with healthcare programs. The study will be connecting selected patients from clinics with trained community health workers (CHW) who will assist participants in getting the help that is needed for their health such as transportation needs, accessing healthy food, health information, accessing good health care, job opportunities, and housing. The study will collect information from both the patients and the health care systems to test how effectively the care plan works over time.
Find Hypercholesterolemia trials matched specifically to you
Answer 3 quick questions and we'll show you trials that fit your situation.