A Phase III Study to Assess the Effect of AZD0780 on LDL-C in Patients With HeFH
This is a study to evaluate the efficacy and safety of AZD0780 in adults with HeFH and elevated LDL-C, either with clinical ASCVD and LDL-C levels of 55 mg/dL or higher or without clinical ASCVD and L...
Improved Diagnosis of Familial Hypercholesterolemia Across the Northland (ID-FH)
The overall goal of this study is to promote awareness of Familial Hypercholesterolemia (FH). The investigators aim to enroll patients with suspected FH into the study and will randomize them to recei...
A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)
This study is designed to learn if enlicitide decanoate is safe and effective to treat children and adolescents with heterozygous familial hypercholesterolemia (HeFH) and high amounts of low-density l...
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and el...
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elev...
Top Cities for Familial Hypercholesterolemia Clinical Trials
Familial Hypercholesterolemia clinical trials are recruiting across 27 cities. Here are the cities with the most active studies:
About Familial Hypercholesterolemia
Familial hypercholesterolemia is a genetic condition causing very high LDL cholesterol levels from birth, dramatically increasing the risk of early heart disease. It affects about 1 in 250 people. Treatment includes high-intensity statins, PCSK9 inhibitors, and lifestyle modifications.
Clinical trials are advancing new treatments for familial hypercholesterolemia. Currently, 5 studies are recruiting a combined 918 participants across the United States. Research is being conducted by 4 organizations including AstraZeneca, Essentia Health, Merck Sharp & Dohme LLC and 1 others.
2026 Familial Hypercholesterolemia Research Landscape
As of March 2026, the familial hypercholesterolemia clinical trial landscape includes 5 actively recruiting studies across 27 cities in the United States. These studies are collectively seeking 918 participants, with an average enrollment target of 184 per study.
Research is being led by 4 different organizations, including AstraZeneca, Essentia Health, Merck Sharp & Dohme LLC, Novartis Pharmaceuticals.
Geographically, familial hypercholesterolemia trials are most concentrated in San Francisco, California (3 trials); Washington D.C., District of Columbia (3 trials); Duluth, Minnesota (2 trials); Boca Raton, Florida (2 trials); Vienna, (2 trials) and 7 other cities.
Featured Familial Hypercholesterolemia Studies
Highlighted recruiting studies for familial hypercholesterolemia, selected by enrollment size and research scope.
A Phase III Study to Assess the Effect of AZD0780 on LDL-C in Patients With HeFH
This is a study to evaluate the efficacy and safety of AZD0780 in adults with HeFH and elevated LDL-C, either with clinical ASCVD and LDL-C levels of 55 mg/dL or higher or without clinical ASCVD and LDL-C levels of 70 mg/dL or higher. AZD0780 is a small molecule that reduces the amount of LDL-C in the blood. Placebo will be used for comparison, and neither the participants nor the Investigators wi...
Improved Diagnosis of Familial Hypercholesterolemia Across the Northland (ID-FH)
The overall goal of this study is to promote awareness of Familial Hypercholesterolemia (FH). The investigators aim to enroll patients with suspected FH into the study and will randomize them to receive usual care or motivational interview. Primary study outcomes include knowledge of FH, as well as clinical and patient-reported outcomes. This study aims to promote optimal disease management and im...
A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)
This study is designed to learn if enlicitide decanoate is safe and effective to treat children and adolescents with heterozygous familial hypercholesterolemia (HeFH) and high amounts of low-density lipoprotein cholesterol (LDL-C) in the blood. The goals of this study are to learn about the safety of enlicitide and if children tolerate it, what happens to enlicitide in a child's body over time, a...
Frequently Asked Questions About Familial Hypercholesterolemia Clinical Trials
Are there familial hypercholesterolemia clinical trials near me?
Yes, there are 5 familial hypercholesterolemia clinical trials currently recruiting across 27+ cities in the United States, including San Francisco, California; Washington D.C., District of Columbia; Duluth, Minnesota. Browse the studies above to find one at a location convenient for you.
How do I join a familial hypercholesterolemia clinical trial?
To join a familial hypercholesterolemia clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are familial hypercholesterolemia clinical trials free?
Yes, participation in familial hypercholesterolemia clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of familial hypercholesterolemia treatments are being studied?
Current familial hypercholesterolemia clinical trials are testing a range of approaches. These include new drugs, combination therapies, medical devices, and other interventions sponsored by 4 research organizations.
Is it safe to participate in familial hypercholesterolemia clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated March 2, 2026 from ClinicalTrials.gov
About This Data
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated March 2, 2026 · Data from ClinicalTrials.gov