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ClinicalTrials.gov public records Last synced Nov 7, 2026, 6:49 AM EST

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Showing 25–42 of 42 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: HoFH Clear all

Conditions: Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders
Interventions: mipomersen sodium; Drug
Lead sponsor: Kastle Therapeutics, LLC; Industry
Eligibility: 12 Years and older
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2011
U.S. locations: 5
States / cities: Chicago, Illinois • Auburn, Maine • Biddeford, Maine + 2 more

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Evinacumab; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 5 Years to 11 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 6
States / cities: Wilmington, Delaware • Boca Raton, Florida • Kansas City, Kansas + 3 more

Conditions: Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Familial Hypercholesterolemia
Interventions: Screening for FH; Diagnostic Test
Lead sponsor: University of Wisconsin, Madison; Other
Eligibility: 18 Years and older
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 1
States / cities: Madison, Wisconsin

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Zodasiran; Drug
Lead sponsor: Arrowhead Pharmaceuticals; Industry
Eligibility: 12 Years to 17 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: St Louis, Missouri

Conditions: Heterozygous or Homozygous Familial Hypercholesterolemia
Interventions: Inclisiran; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years to 100 Years
Enrollment: 195 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2032
U.S. locations: 5
States / cities: Boca Raton, Florida • New York, New York • Cincinnati, Ohio + 2 more

Conditions: Familial Hypercholesterolemia
Interventions: lomitapide; Drug
Lead sponsor: Aegerion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2014
U.S. locations: 2
States / cities: Los Angeles, California • Philadelphia, Pennsylvania

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Evolocumab, Placebo; Biological · Drug
Lead sponsor: Amgen; Industry
Eligibility: 12 Years to 80 Years
Enrollment: 58 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 2
States / cities: New York, New York • Cincinnati, Ohio

Conditions: Severe Familial Hypercholesterolemia
Interventions: Evolocumab; Biological
Lead sponsor: Amgen; Industry
Eligibility: 12 Years to 80 Years
Enrollment: 300 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 6
States / cities: Los Angeles, California • New York, New York • Cincinnati, Ohio + 1 more

Conditions: Familial Hypercholesterolemia - Homozygous
Interventions: Inclisiran, Placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years to 11 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2029
U.S. locations: 4
States / cities: San Francisco, California • Washington D.C., District of Columbia • St Louis, Missouri + 1 more

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: zodasiran Injection, Placebo; Drug
Lead sponsor: Arrowhead Pharmaceuticals; Industry
Eligibility: 12 Years and older
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 4
States / cities: Park Ridge, Illinois • New York, New York • Cincinnati, Ohio + 1 more

Conditions: Homozygous Familial Hypercholesterolemia, HoFH
Interventions: HDL Therapeutics PDS-2 System; Device
Lead sponsor: HDL Therapeutics; Industry
Eligibility: 12 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Familial Hypercholesterolemia - Homozygous
Interventions: Inclisiran, Placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 12 Years to 17 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 1
States / cities: Boca Raton, Florida

Conditions: Cardiovascular Disease With Mention of Arteriosclerosis, Elevated Cholesterol, Familial Hypercholesterolemia
Interventions: lerodalcibep; Drug
Lead sponsor: LIB Therapeutics LLC; Industry
Eligibility: 10 Years and older
Enrollment: 2,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 4
States / cities: Evanston, Illinois • Cincinnati, Ohio

Conditions: Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (sHTG), Mixed Hyperlipemia, Hypercholesterolaemia
Interventions: CTX310; Drug
Lead sponsor: CRISPR Therapeutics AG; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
U.S. locations: 8
States / cities: Hialeah, Florida • Jacksonville, Florida • Orlando, Florida + 5 more

Conditions: Familial Hypercholesterolemia
Interventions: Implitapide; Drug
Lead sponsor: Medical Research Laboratories International; Industry
Eligibility: 8 Years to 70 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2005
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Lomitapide; Drug
Lead sponsor: Aegerion Pharmaceuticals, Inc.; Industry
Eligibility: 13 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2004
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Familial Hypercholesterolemia
Interventions: Evolocumab; Biological
Lead sponsor: Amgen; Industry
Eligibility: 10 Years to 17 Years
Enrollment: 163 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 3
States / cities: The Bronx, New York • Cincinnati, Ohio • Nashville, Tennessee

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: CER-001; Drug
Lead sponsor: Cerenis Therapeutics, SA; Industry
Eligibility: 12 Years and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2014
U.S. locations: 2
States / cities: Hartford, Connecticut • North Massapequa, New York

Search by objective public record fields.

Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia

Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia

The ORIGIN-FH Study

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies

Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia

Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities

Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders

Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

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

HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia

Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia

Long-term Efficacy and Safety of OLE LIB003 in HoFH, HeFH, and High-risk CVD Patients Requiring Further LDL-C Reduction

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

Implitapide in Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering Therapy

Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor

Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)

Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects