Condition

Homozygous Familial Hypercholesterolemia (HoFH) clinical trials and research

• 4Clinical trials
• 9Countries

Newest Homozygous Familial Hypercholesterolemia (HoFH) clinical trials

All recruiting trials (4)

• CTX310 for refractory high triglycerides or LDL cholesterol

  This trial will test whether a single IV dose of CTX310 is safe and tolerated in adults whose high triglycerides or LDL cholesterol have not responded to available treatments.

  Recruiting at 16 sites Phase 1 CRISPR Therapeutics · Hialeah, Florida and 16 other locations · NCT07491172

• EDP167 dose testing for homozygous familial hypercholesterolemia

  This trial tests whether two injections of an siRNA drug called EDP167 can lower ANGPTL3 and improve lipid levels in adults with homozygous familial hypercholesterolemia (HoFH).

  Recruiting Phase 2 Eddingpharm (Zhuhai) Co., Ltd. · Beijing · NCT07489209

• ORIGIN-FH: Newborn screening to detect familial high cholesterol

  This project will test a genetic cheek swab of parents plus blood screening of newborns to find familial high cholesterol in babies born to parents with familial hypercholesterolemia.

  Recruiting University of Wisconsin, Madison · Madison, Wisconsin · NCT07470723

• Intensifying lipid-lowering with evinacumab to reduce coronary plaque in homozygous familial hypercholesterolemia

  This project will test whether adding evinacumab to existing lipid-lowering therapy can shrink or slow coronary plaque buildup in people with homozygous familial hypercholesterolemia using CCTA imaging.

  Recruiting at 13 sites Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) · Paris and 12 other locations · NCT07447648

Related topics

Quick answers

How many Homozygous Familial Hypercholesterolemia (HoFH) 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 9 countries. The most sites are in United States (2 trials), Australia (1 trial), New Zealand (1 trial), United Kingdom (1 trial), France (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1: 1, Phase 2: 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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