Five Familial Hypercholesterolemia Clinical Trials Currently Recruiting in the United States

It is essential for clinicians to remain informed about emerging clinical trial opportunities and resulting data that may inform therapeutic strategies, affect clinical guidelines, expand treatment options, and ultimately improve patient quality of life.

Today, we have a rundown of five, ongoing Phase 2, 3 clinical trials focusing on familial hypercholesterolemia, that are currently recruiting in the United States. While most trials focus on distinct subtypes of familial hypercholesterolemia, such as heterozygous and homozygous forms, one includes populations across the broader familial hypercholesterolemia spectrum. The ranges of anticipated primary completion dates range from June 2026 through December 2033. The trials are arranged in order of their start date.

Quick View Table of Familial Hypercholesterolemia Clinical Trials

Featured Phase 3 Clinical Trials

A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

• Sponsor: Arrowhead Pharmaceuticals
• Goal: To evaluate the efficacy and safety of zodasiran subcutaneous (SC) injection in subjects 12 years of age and older with genetically or clinically diagnosed Homozygous familial hypercholesterolemia (HoFH).
• Interventions: zodasiran Injection, Placebo
• Primary Outcome Measures: Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period).
• Time Frame: Baseline, Month 12
• Study Details
• Source

Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia (ORION-19)

• Sponsor: Novartis Pharmaceuticals
• Goal: To evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to <12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).
• Interventions: Inclisiran, Placebo
• Primary Outcome Measures: Percentage change in LDL-C from baseline to Day 330 (Year 1).
• Time Frame: Baseline and Day 330
• Study Details
• Source

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia (ORION-20)

• Sponsor: Novartis Pharmaceuticals
• Goal: To evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to <12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).
• Interventions: Inclisiran, Placebo
• Primary Outcome Measures: Percentage change in LDL-C from baseline to Day 330 (Year 1).
• Time Frame: Baseline and Day 330
• Study Details
• Source

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

• Sponsor: Merck Sharp & Dohme
• Goal: 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.
• Interventions: Enlicitide Decanoate, Placebo
• Primary Outcome Measures: Part A: Maximum Plasma Concentration (Cmax) of Enlicitide; Part A: Area Under the Concentration-Time Curve from 0 to 24 Hours (AUC0-24) of Enlicitide; Part B: Percent Change from Baseline in Low-Density Lipoprotein Cholesterol (LDL-C); Number of Participants Who Experience an Adverse Event (AE); Number of Participants Who Discontinue Study Treatment Due to an AE.
• Time Frame: At designated timepoints (up to 24 hours postdose on day 14); At designated timepoints (up to 24 hours postdose on day 14); Baseline and Week 24; Up to approximately 188 weeks; Up to approximately 180 weeks.
• Study Details
• Source

2-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia (FH)

• Sponsor: Vanderbilt University Medical Center
• Goal: To test the hypothesis that 2-HOBA will reduce modification of HDL and LDL and improve HDL function in humans with heterozygous FH.
• Interventions: 2-Hydroxybenzylamine, Placebo
• Primary Outcome Measures: 2-HOBA increases HDL cholesterol efflux capacity.
• Time Frame: Baseline to week 6.
• Study Details
• Source

Related Guidelines:

Familial Hypercholesterolemia

• National Lipid Association (NLA), April 2026

Homozygous Familial Hypercholesterolaemia

• European Atherosclerosis Society (EAS), February 2026

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