Condition
Heterozygous Familial Hypercholesterolemia (HeFH) clinical trials and research
As of November 12, 2026, Find a Trial lists 5 Heterozygous Familial Hypercholesterolemia (HeFH) clinical trials that are recruiting, at sites in 20 countries (most in United States (3), Australia (2), China (2)). By phase: Phase 2/3 (1), Phase 1 (1), Phase 3 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 5Clinical trials
- 20Countries
Newest Heterozygous Familial Hypercholesterolemia (HeFH) clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| CTX310 for refractory high triglycerides or LDL cholesterol | Phase 1 | Hialeah, Florida and 16 other locations | Recruiting | Mar 24, 2026 |
| ORIGIN-FH: Newborn screening to detect familial high cholesterol | — | Madison, Wisconsin | Recruiting | Mar 13, 2026 |
| SYH2053 Injection for adults with heterozygous familial hypercholesterolemia | Phase 3 | Beijing, Beijing Municipality | Recruiting | Mar 11, 2026 |
| Coronary Plaque Progression in HeFH: DESTINY-FH | — | Paris and 2 other locations | Recruiting | Feb 23, 2026 |
| Enlicitide decanoate (MK-0616), an oral PCSK9 inhibitor, for children and adolescents with heterozygous familial hypercholesterolemia | Phase 2/3 | Wilmington, Delaware and 47 other locations | Recruiting | Jul 10, 2025 |
All recruiting trials (5)
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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.
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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.
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SYH2053 Injection for adults with heterozygous familial hypercholesterolemia
A phase 3 trial tests whether SYH2053 injection, added to standard diet and statin therapy, can lower LDL cholesterol in adults with HeFH compared with placebo.
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Coronary Plaque Progression in HeFH: DESTINY-FH
This study tests how heart arteries change over 5 years in people with heterozygous familial hypercholesterolemia using coronary CT angiography.
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Enlicitide decanoate (MK-0616), an oral PCSK9 inhibitor, for children and adolescents with heterozygous familial hypercholesterolemia
This trial tests whether a daily oral medicine called enlicitide decanoate can safely lower LDL cholesterol in children and teens with heterozygous familial hypercholesterolemia.
Related topics
Quick answers
How many Heterozygous Familial Hypercholesterolemia (HeFH) clinical trials are recruiting right now?
5, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 20 countries. The most sites are in United States (3 trials), Australia (2 trials), China (2 trials), France (2 trials), New Zealand (2 trials). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 2/3: 1, 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.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.