Condition

Homozygous Familial Hypercholesterolemia clinical trials and research

• 4Clinical trials
• 6Countries

Newest Homozygous Familial Hypercholesterolemia clinical trials

All recruiting trials (4)

• Zodasiran for teens with homozygous familial hypercholesterolemia

  This study tests whether subcutaneous Zodasiran can help adolescents with HoFH who have very high LDL-C despite lipid-lowering therapy.

  Recruiting at 3 sites Phase 3 Arrowhead Pharmaceuticals · St Louis, Missouri and 2 other locations · NCT07473843

• Gene therapy for treating a rare cholesterol disorder

  This study is testing a new gene therapy to see if it can help people with a rare cholesterol disorder called homozygous familial hypercholesterolemia lower their cholesterol levels.

  Recruiting Early Phase 1 First Affiliated Hospital Xi'an Jiaotong University · Xi'an, Shaanxi · NCT06125847

• Registry for patients with homozygous familial hypercholesterolemia

  This study is creating a registry to collect health information from people with homozygous familial hypercholesterolemia to better understand this rare condition and improve future treatments.

  Recruiting at 4 sites University of Pennsylvania · Philadelphia, Pennsylvania and 3 other locations · NCT04815005

• A repository for monitoring health in patients with homozygous familial hypercholesterolemia

  This study is trying to gather health information from people with homozygous familial hypercholesterolemia to better understand their condition and find ways to improve their treatment.

  Recruiting The Rogosin Institute · New York, New York · NCT01109368

Quick answers

How many Homozygous Familial Hypercholesterolemia clinical trials are recruiting right now?

4, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 6 countries. The most sites are in United States (3 trials), China (1 trial), Denmark (1 trial), Poland (1 trial), Netherlands (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Early Phase 1: 1, Phase 3: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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