Familial Hypercholesterolemia Clinical Trials

35 recruitingLast updated: May 21, 2026

There are 35 actively recruiting familial hypercholesterolemia clinical trials across 41 countries. Studies span Phase 3, Not Applicable, Early Phase 1, Phase 2, Phase 1. Top locations include Beijing, Beijing Municipality, China, London, United Kingdom, New York, New York, United States. Updated daily from ClinicalTrials.gov.


Familial Hypercholesterolemia Trials at a Glance

35 actively recruiting trials for familial hypercholesterolemia are listed on ClinicalTrialsFinder across 6 cities in 41 countries. The largest study group is Phase 3 with 8 trials, with the heaviest enrollment activity in Beijing, London, and New York. Lead sponsors running familial hypercholesterolemia studies include Novartis Pharmaceuticals, Hospital do Coracao, and Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi).

Browse familial hypercholesterolemia trials by phase

About Familial Hypercholesterolemia Clinical Trials

Looking for clinical trials for Familial Hypercholesterolemia? There are currently 16 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Familial Hypercholesterolemia trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Familial Hypercholesterolemia clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 35 trials

Recruiting

Child-Parent Familial Hypercholesterolemia Screening

Familial Hypercholesterolemia
Children's Hospital of Fudan University15,000 enrolled6 locationsNCT04529967
Recruiting
Phase 3

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

Familial Hypercholesterolemia - Heterozygous
Novartis Pharmaceuticals60 enrolled66 locationsNCT06597019
Recruiting
Phase 3

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies

Heterozygous or Homozygous Familial Hypercholesterolemia
Novartis Pharmaceuticals195 enrolled52 locationsNCT05682378
Recruiting

Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)

Homozygous Familial Hypercholesterolemia (HoFH)Coronary Computed Tomography Angiography
Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)52 enrolled13 locationsNCT07447648
Recruiting
Phase 2Phase 3

A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)

Heterozygous Familial Hypercholesterolemia (HeFH)
Merck Sharp & Dohme LLC153 enrolled26 locationsNCT07058077
Recruiting
Phase 3

A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

Homozygous Familial Hypercholesterolemia
Arrowhead Pharmaceuticals60 enrolled35 locationsNCT07037771
Recruiting

National Network for Cardiovascular Genomics: Advancing Cardiovascular Healthcare for Hereditary Diseases in Brazil's Unified Health System Through a Multicenter Registry

Sudden Cardiac DeathArrhythmogenic Right Ventricular DysplasiaCardiomyopathy, Dilated+11 more
Hospital do Coracao1,211 enrolled27 locationsNCT06546137
Recruiting
Phase 2

2-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia (FH)

Familial Hypercholesterolemia
Vanderbilt University Medical Center72 enrolled1 locationNCT04941599
Recruiting
Phase 3

IBI306 Monotherapy in Non-Familial Hypercholesterolemia and Mixed Hyperlipidemia

Non-familial Hypercholesterolemia and Mixed Hyperlipidemia
Innovent Biologics (Suzhou) Co. Ltd.198 enrolled1 locationNCT07473960
Recruiting

Inclisiran (Leqvio®) in Patients With Atherosclerotic Vascular Disease and/or Heterozygous Familial Hypercholesterolemia Treated in Spain: the INSPIRE Study

Heterozygous Familial HypercholesterolemiaAtherosclerotic Vascular Disease
Novartis Pharmaceuticals200 enrolled20 locationsNCT06958315
Recruiting
Not Applicable

Comparing Direct vs Indirect Methods for Cascade Screening

Genetic TestingLong QT SyndromeEthics+1 more
University of Maryland, Baltimore200 enrolled1 locationNCT05348564
Recruiting
Phase 3

A Study to Evaluate the Safety, Efficacy of SYH2053 as Monotherapy in Chinese Participants With Non-familial Hypercholesterolemia or Mixed Hyperlipidemia

Non-familial Hypercholesterolemia and Mixed Hyperlipidemia
CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.760 enrolled2 locationsNCT07421297
Recruiting

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

Homozygous Familial Hypercholesterolemia
The Rogosin Institute60 enrolled1 locationNCT01109368
Recruiting
Phase 1

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

Premature Coronary Heart DiseaseHeterozygous Familial Hypercholesterolemia
Verve Therapeutics, Inc.85 enrolled22 locationsNCT06164730
Recruiting
Phase 2

A Dose-exploration Study of EDP167 in HoFH

Homozygous Familial Hypercholesterolemia (HoFH)
Eddingpharm (Zhuhai) Co., Ltd.20 enrolled1 locationNCT07489209
Recruiting
Phase 1

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

HypercholesterolaemiaMetabolic DiseaseDyslipidemias+7 more
CRISPR Therapeutics AG90 enrolled18 locationsNCT07491172
Recruiting
Not Applicable

Study of the Determinants of Coronary Atherosclerosis in Familial Hypercholesterolemia (ATHERO-FH Study)

Atherosclerotic Cardiovascular Disease (ASCVD)Familial Hypercholesterolemias
Nantes University Hospital600 enrolled9 locationsNCT06960902
Recruiting
Not Applicable

The ORIGIN-FH Study

Homozygous Familial Hypercholesterolemia (HoFH)Familial HypercholesterolemiaHeterozygous Familial Hypercholesterolemia (HeFH)
University of Wisconsin, Madison70 enrolled1 locationNCT07470723
Recruiting

EAS Familial Hypercholesterolaemia Studies Collaboration

Familial Hypercholesterolemia
Imperial College London75,000 enrolled1 locationNCT04272697
Recruiting
Not Applicable

Familial Hypercholesterolemia Interpretive Comment - Nudging to Detection.

Familial Hypercholesterolemia
Odense University Hospital2,000 enrolled2 locationsNCT05614219