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ClinicalTrials.gov public records Last synced Nov 7, 2026, 6:37 AM EST

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Showing 1–24 of 42 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: HoFH Clear all

Conditions: Homozygous Familial Hypercholesterolemia (HoFH)
Interventions: AAV directed hLDLR gene therapy; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2020
U.S. locations: 5
States / cities: Boca Raton, Florida • Kansas City, Kansas • Portland, Oregon + 2 more

Conditions: Hypercholesterolemia, Familial
Interventions: ISIS 301012; Drug
Lead sponsor: Kastle Therapeutics, LLC; Industry
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2007
U.S. locations: 5
States / cities: Chicago, Illinois • New York, New York • Winston-Salem, North Carolina + 2 more

Conditions: Homozygous Familial Hypercholesterolemia (HoFH)
Interventions: Not listed
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 1
States / cities: Boca Raton, Florida

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Alirocumab, Placebo; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 69 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 4
States / cities: Boca Raton, Florida • New York, New York • Cincinnati, Ohio + 1 more

Conditions: Homozygous Familial Hypercholesterolemia (HoFH)
Interventions: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy; Drug
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2025
U.S. locations: 4
States / cities: Columbus, Ohio • Portland, Oregon • Philadelphia, Pennsylvania + 1 more

Conditions: Hypercholesteremia
Interventions: Gemcabene; Drug
Lead sponsor: NeuroBo Pharmaceuticals Inc.; Industry
Eligibility: 17 Years and older
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 1
States / cities: Beverly Hills, California

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: evinacumab; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 12 Years and older
Enrollment: 116 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 9
States / cities: Los Angeles, California • Boca Raton, Florida • Boston, Massachusetts + 5 more

Conditions: Hypercholesterolemia, Familial
Interventions: Kinetics test; Other
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: ALN-PCSSC, Standard of Care; Drug
Lead sponsor: The Medicines Company; Industry
Eligibility: 12 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2018
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: REGN1500 250 mg SC/15 mg/kg IV/450 mg SC; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 3
States / cities: Los Angeles, California • Philadelphia, Pennsylvania • Dallas, Texas

Conditions: Hypercholesterolemia, Familial
Interventions: Not listed
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years and older
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Not listed
Lead sponsor: The Rogosin Institute; Other
Eligibility: Not listed
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2030
U.S. locations: 1
States / cities: New York, New York

Conditions: Hypercholesterolemia
Interventions: Lapaquistat acetate and current lipid-lowering treatment, Current lipid-lowering treatment; Drug
Lead sponsor: Takeda; Industry
Eligibility: 8 Years and older
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2007
U.S. locations: 2
States / cities: Cincinnati, Ohio • Columbus, Ohio

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Lomitapide; Drug
Lead sponsor: Amryt Pharma; Industry
Eligibility: Not listed
Enrollment: 260 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2026
U.S. locations: 46
States / cities: Birmingham, Alabama • Mobile, Alabama • Cottonwood, Arizona + 37 more

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: ARO-ANG 3 Injection; Drug
Lead sponsor: Arrowhead Pharmaceuticals; Industry
Eligibility: 16 Years and older
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 2
States / cities: Mount Sinai, New York • Cincinnati, Ohio

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: AEGR-733; Drug
Lead sponsor: Aegerion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2011
U.S. locations: 2
States / cities: Los Angeles, California • Philadelphia, Pennsylvania

Conditions: ASCVD, Elevated Cholesterol, Heterozygous Familial Hypercholesterolemia, Homozygous Familial Hypercholesterolemia
Interventions: Inclisiran Sodium; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 3,275 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 141
States / cities: Birmingham, Alabama • Foley, Alabama • Huntsville, Alabama + 109 more

Conditions: Homozygous Familial Hypercholesterolemic
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 2 Years to 70 Years
Enrollment: 73 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1992
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hypercholesterolemia, Familial
Interventions: Torcetrapib/atorvastatin; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005
U.S. locations: 1
States / cities: Boston, Massachusetts

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: 144 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2014
U.S. locations: 20
States / cities: Mission Viejo, California • Newport Beach, California • Bridgeport, Connecticut + 17 more

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Demographics, diagnosis type, genotype, lipid profile, treatment allocation, country of residence.; Other
Lead sponsor: University of Pennsylvania; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2026
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: lerodalcibep, evolocumab; Drug
Lead sponsor: LIB Therapeutics LLC; Industry
Eligibility: 10 Years and older
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 2
States / cities: Evanston, Illinois • Cincinnati, Ohio

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, Placebo; Drug
Lead sponsor: Kastle Therapeutics, LLC; Industry
Eligibility: 12 Years and older
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 2
States / cities: Charlotte, North Carolina • Cincinnati, Ohio

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: evinacumab, Placebo; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 12 Years and older
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 6
States / cities: Boca Raton, Florida • Boston, Massachusetts • New York, New York + 3 more

Search by objective public record fields.

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

Efficacy and Safety of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid-Lowering Therapy (COBALT-1)

Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Regeneron 1331 Kinetics Sub-Study HoFH

A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

Screening Protocol for a Gene Therapy Trial in Subjects With Homozygous Familial Hypercholesterolemia

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

Efficacy of Lapaquistat Acetate Co-Administered With Current Lipid-Lowering Treatment on Blood Cholesterol Levels in Subjects With Homozygous Familial Hypercholesterolemia

LOWER: Lomitapide Observational Worldwide Evaluation Registry

Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)

A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)

Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C

Cardiovascular Evaluation of Patients With High Cholesterol and Normal Volunteers

Study To Evaluate The Effect Of Torcetrapib/Atorvastatin In Patients With Genetic High Cholesterol Disorder

An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia

HoFH, the International Clinical Collaborators Registry

Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH

Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia

Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia