Heterozygous Familial Hypercholesterolemia (HeFH)

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Review clinical trials related to Heterozygous Familial Hypercholesterolemia (HeFH). Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)

This study is designed to learn if enlicitide decanoate is safe and effective to treat children and adolescents with heterozygous familial hypercholesterolemia (HeFH) and high amounts of low-density lipoprotein cholesterol (LDL-C) in the blood. The goals of this study are to learn about the safety of enlicitide and if children tolerate it, what happens to enlicitide in a child's body over time, and if enlicitide works to lower cholesterol levels in children more than a placebo.

Participants needed: 153
Trial details
Phase: Phase 2, Phase 3Age: 6-17Biological sex: AllType: InterventionalSponsor: Merck Sharp & Dohme LLCUpdated: Aug 21, 2026Locations: 41
Eligibility criteria

Has possible or definite diagnosis of HeFH based on a locally accepted diagnosti... [+4]

Has a history of homozygous FH based on genetic or clinical criteria, or history... [+3]

Status: Not yet recruiting

Open Label Phase 3b Study to Evaluate the Long-Term Efficacy and Safety of Lerodalcibep in Children and Adolescents, 6 to 17 Years of Age, With Heterozygous Familial Hypercholesterolemia on Stable Diet and Oral Lipid-Lowering Therapy

The goal is to assess the long term efficacy (LDL cholesterol reduction) and safety over 3 years of lerodalcibep (Lerochol) SC 300 mg QM administered by auto-injector (AI)/pre-filled pen (PFP) in male and female pediatric patients 6 to 17 years of age, with inherited high cholesterol (HeFH) on a stable diet and maximally tolerated oral LDL C lowering drug therapy such as statins who completed the 24 week placebo controlled base trial. The main question\[s\] it aims to answer are: How effective is Lerochol in maintaining LDL cholesterol reductions over years? How well is it tolerated and are there any safety concerns? Participants will visit the clinic every month for 3 months and then home dosed with clinic visits every 3 months. They will undergo periodic physical exams, height and weight measurements, answer questions, have blood drawn from a vein in their arm, have blood pressure measurements, EKC heart tests, and receive monthly injections lasting about 5 seconds in their arms or abdomen with an autoinjector.

Participants needed: 150
Trial details
Phase: Phase 3Age: 6-17Biological sex: AllType: InterventionalSponsor: LIB Therapeutics LLCUpdated: Aug 20, 2026Locations: 5
Eligibility criteria

Not listed

Status: Recruiting

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.

Participants needed: 90
Trial details
Phase: Phase 1Age: 18-75Biological sex: AllType: InterventionalSponsor: CRISPR Therapeutics AGUpdated: Jul 23, 2026Locations: 19
Eligibility criteria

Age of ≥18 and ≤75 years at the time of signing the informed consent. [+5]

Participants with familial chylomicronemia syndrome (FCS). Some exceptions may a... [+6]

Status: Recruiting

A Study to Evaluate the SYH2053 Injection in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)

Heterozygous Familial Hypercholesterolemia (HeFH) is an autosomal dominant disorder characterized by markedly elevated low-density lipoprotein cholesterol (LDL-C) and increased risk of atherosclerotic cardiovascular disease (ASCVD). This trial aims to evaluate the SYH2053 Injection in patients with HeFH.

Participants needed: 135
Trial details
Phase: Phase 3Age: 18+Biological sex: AllType: InterventionalSponsor: CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.Updated: Jul 8, 2026Locations: 1
Eligibility criteria

Male or female participants ≥18 years of age. [+5]

HoFH or suspected HoFH. [+11]

Status: Recruiting

"Detection and Follow-up of Coronary Lesions in HeFH (DESTINY-FH Study)"

This multicenter, non-randomized interventional study aims to assess coronary artery disease progression over 5 years in patients with genetically confirmed heterozygous familial hypercholesterolemia (HeFH), using coronary computed tomography angiography (CCTA). The primary endpoint is the visual evaluation of coronary stenosis using CAD-RADS v2.0, identifying changes between baseline (2018-2022) and study inclusion. The study will enroll 300 patients (100 protected, 200 non-protected) from La Pitié-Salpêtrière hospital and Saint Antoine Hospital (Paris). Participation lasts up to one week. Total study duration is 2 years, with extended follow-up through routine care data over 10 years.

Participants needed: 300
Trial details
Age: 30-60Biological sex: AllType: InterventionalSponsor: Assistance Publique - Hôpitaux de ParisUpdated: Jun 24, 2026Locations: 3
Eligibility criteria

Patients with heterozygous familial hypercholesterolemia [+6]

Patient under guardianship, or unable to give consent [+10]

Status: Recruiting

The ORIGIN-FH Study

The goal of this clinical trial is to identify different types of Familial Hypercholesterolemia (FH) in infants and newborns. Participants will: * undergo a cheek swab for genetic testing (parents only) * have 5 blood samples collected Participants can expect to be in the trial for 2 years.

Participants needed: 70
Trial details
Age: 18+Biological sex: AllType: InterventionalSponsor: University of Wisconsin, MadisonUpdated: Mar 13, 2026Locations: 1
Eligibility criteria

Individuals in the expectant partnership providing informed consent are at least... [+5]

Expectant partnership where neither partner meets diagnostic criteria for HeFH o... [+3]

Status: Not yet recruiting

QLC7401 in Participants With Heterozygous Familial Hypercholesterolemia (HeFH)

This Phase III clinical trial aims to assess the efficacy and safety of QLC7401 Treatment in adults with Heterozygous Familial Hypercholesterolemia on a stable lipid-lowering therapy. Participants will be randomly assigned to receive subcutaneous injections of either QLC7401 or a placebo according to the study schedule.

Participants needed: 120
Trial details
Phase: Phase 3Age: 18-80Biological sex: AllType: InterventionalSponsor: Qilu Pharmaceutical Co., Ltd.Updated: Mar 4, 2026
Eligibility criteria

Aged 18-80 years (inclusive) [+7]

New York Heart Association (NYHA) class III or IV heart failure. [+7]