Homozygous Familial Hypercholesterolemia Clinical Trials

5 recruiting

Frequently Asked Questions

Common questions about Homozygous 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 18 of 8 trials

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

The ORIGIN-FH Study

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

HoFH, the International Clinical Collaborators Registry

Homozygous Familial Hypercholesterolemia
University of Pennsylvania1,000 enrolled4 locationsNCT04815005
Recruiting

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

Homozygous Familial Hypercholesterolemia
The Rogosin Institute60 enrolled1 locationNCT01109368
Recruiting
Early Phase 1

NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia
First Affiliated Hospital Xi'an Jiaotong University12 enrolled1 locationNCT06125847
Recruiting

LOWER: Lomitapide Observational Worldwide Evaluation Registry

Homozygous Familial Hypercholesterolemia
Amryt Pharma300 enrolled75 locationsNCT02135705