Hypercholesterolemia means having high cholesterol in the blood, increasing the risk of heart attack and stroke. About 95 million U.S. adults have total cholesterol above 200 mg/dL. Statins remain the backbone of treatment, but newer drugs like PCSK9 inhibitors and bempedoic acid now offer additional options for people who can't tolerate statins or need further LDL reduction.
What's actually going on in research
Trials are testing new ways to lower LDL cholesterol beyond current drugs, including RNA-based therapies that work for months after a single dose, oral PCSK9 inhibitors, and drugs targeting lipoprotein(a), an inherited risk factor that statins don't address. Researchers are also studying whether lowering cholesterol earlier in life prevents more heart disease than waiting until problems appear.
RNA therapies
Inclisiran is an RNA drug given twice yearly by injection that silences the gene for PCSK9, keeping LDL low between doses. Trials are testing whether similar RNA approaches can target other cholesterol pathways with even less frequent dosing.
Lipoprotein(a) lowering
About one in five people has high lipoprotein(a), which raises heart disease risk independently of LDL. New drugs in trials can lower lipoprotein(a) by 80-90 percent, and studies will show whether that prevents heart attacks.
Oral PCSK9 inhibitors
Current PCSK9 inhibitors require injections. Pills that block PCSK9 are now in trials, which could make this powerful LDL-lowering approach more accessible.
What to know before you search
Eligibility typically depends on baseline LDL or total cholesterol levels, history of heart disease, prior cholesterol medications tried, and sometimes genetic testing for familial forms.
What types of trials are currently open
- LDL-lowering trials — Testing new drugs or drug combinations to see how much they lower LDL cholesterol compared to placebo or current treatments.
- Cardiovascular outcome trials — Following people for years to see if a cholesterol drug actually prevents heart attacks, strokes, and death, not just lowers lab numbers.
- Lipoprotein(a) trials — Testing drugs specifically designed to lower lipoprotein(a), which standard cholesterol drugs don't address effectively.
- Familial hypercholesterolemia trials — Studies in people with inherited extremely high cholesterol, who often need aggressive treatment from childhood.
- Statin intolerance trials — Testing alternative treatments or strategies for people who develop muscle pain or other side effects from statins.
Recently added Hypercholesterolemia trials
A Post-Marketing Clinical Trial of Pemafibrate in Patients ｗith Hypercholesterolemia and Inadequate Response to Statins
To investigate the efficacy and safety of Pemafibrate 0.2 mg/day or 0.4 mg/day for 12 weeks in patients with hypercholesterolemia and inadequate response to statins, using placebo as a control.
Take a new cholesterol-lowering injection for 12 weeks
This Phase 3, randomized, double-blind, active-controlled study evaluates the efficacy and safety of obicetrapib (OBI) compared to bempedoic acid (BPA) in participants with dyslipidemia at high or very high cardiovascular risk. Participants must have elevated low density lipoprotein cholesterol (LDL-C) levels despite receiving maximally tolerated lipid lowering therapy. The primary objective is to compare the LDL-C lowering effect of OBI (a CETP inhibitor) against BPA at 12 weeks
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