Homozygous Familial Hypercholesterolemia Clinical Trials

10 recruitingLast updated: May 21, 2026

There are 10 actively recruiting homozygous familial hypercholesterolemia clinical trials across 34 countries. Studies span Phase 3, Early Phase 1, Not Applicable, Phase 1, Phase 2. Top locations include New York, New York, United States, Chicoutimi, Quebec, Canada, Boca Raton, Florida, United States. Updated daily from ClinicalTrials.gov.


Homozygous Familial Hypercholesterolemia Trials at a Glance

10 actively recruiting trials for homozygous familial hypercholesterolemia are listed on ClinicalTrialsFinder across 6 cities in 34 countries. The largest study group is Phase 3 with 2 trials, with the heaviest enrollment activity in New York, Chicoutimi, and Boca Raton. Lead sponsors running homozygous familial hypercholesterolemia studies include Arrowhead Pharmaceuticals, CRISPR Therapeutics AG, and Amryt Pharma.

Browse homozygous familial hypercholesterolemia trials by phase

About Homozygous Familial Hypercholesterolemia Clinical Trials

Looking for clinical trials for Homozygous Familial Hypercholesterolemia? There are currently 5 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 Homozygous 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 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 110 of 10 trials

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 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

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

Homozygous Familial Hypercholesterolemia
The Rogosin Institute60 enrolled1 locationNCT01109368
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

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
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