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
Share blood samples to study cholesterol and hormone therapy effects
The goal of this study is to understand whether statin therapy, a common lipid-lowering therapy, may improve the coagulation profile of menopausal women with hypercholesterolemia undergoing hormone replacement therapy with oral estroprogestin. It will also learn about the liver safety of the combination. The main questions will be: 1. does hormone replacement therapy affect the coagulation profile? 2. when we add statin therapy due to hypercholesterolemia not improving with lifestyle, does it reverse eventual changes of the coagulation profile occurred after initiation of hormone replacement therapy? 3. do the two therapies have an effect on overall inflammation of the organism? Participants will be asked to participate to the study before starting any therapy, if they need hormone relacement therapy due to symptoms, if they have a new onset of dyslipidemia and if they do not have contraindications to the therapies object of the study. The study is observational, thus it will not affect in any way the clinical care, which will follow only guidelines. During routine blood sample collection, additional aliquotes will be taken for the study at the recruitment, after 3 months of hormone replacement therapy and lifestyle changes, at the end of the study (after one year from the recruitment).
Take a daily pill to lower cholesterol when statins don't work
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.
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