Familial Hypercholesterolemia Clinical Trials

16 recruiting

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 30 trials

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 3

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

Homozygous Familial Hypercholesterolemia
Arrowhead Pharmaceuticals60 enrolled22 locationsNCT07037771
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
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 enrolled21 locationsNCT07058077
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
Phase 3

Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

Familial Hypercholesterolemia - Homozygous
Novartis Pharmaceuticals9 enrolled18 locationsNCT06597006
Recruiting
Not Applicable

Familial Hypercholesterolemia Interpretive Comment - Nudging to Detection.

Familial Hypercholesterolemia
Odense University Hospital2,000 enrolled2 locationsNCT05614219
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 enrolled19 locationsNCT06958315
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

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 Coracao600 enrolled27 locationsNCT06546137
Recruiting
Not Applicable

An Adapted Brazilian Cardioprotective Diet, Phytosterols and Krill Oil in Familial Hypercholesterolemia (DICA-FH)

Familial Hypercholesterolemia
Hospital do Coracao300 enrolled24 locationsNCT06331195
Recruiting

HoFH, the International Clinical Collaborators Registry

Homozygous Familial Hypercholesterolemia
University of Pennsylvania1,000 enrolled4 locationsNCT04815005
Recruiting
Phase 3

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

Familial Hypercholesterolemia - Heterozygous
Novartis Pharmaceuticals51 enrolled66 locationsNCT06597019
Recruiting
Not Applicable

Atlantic Lipid Lowering Treatment Optimization Program

Familial HypercholesterolemiaLipoprotein Types--Lp System Lp(A) HyperlipoproteinemiaApolipoprotein B 100, Familial Defective+2 more
Atlantic Health System250 enrolled2 locationsNCT06439654
Recruiting

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

Homozygous Familial Hypercholesterolemia
The Rogosin Institute60 enrolled1 locationNCT01109368
Recruiting

Child-Parent Familial Hypercholesterolemia Screening

Familial Hypercholesterolemia
Children's Hospital of Fudan University15,000 enrolled6 locationsNCT04529967
Recruiting
Early Phase 1

Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)

Familial Hypercholesterolemia
RenJi Hospital20 enrolled1 locationNCT06458010