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Showing 1–24 of 40 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Homozygous Familial Hypercholesterolemia Clear all
Completed Phase 2 Interventional Results available

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

NCT02265952
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 8, 2019 · Synced Jun 10, 2026, 11:37 PM EDT
Terminated Phase 2 Interventional Results available

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

NCT05217667
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 2 Interventional Results available

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

NCT01556906
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 9, 2013 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 2 Interventional

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

NCT00280995
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 2, 2016 · Synced Jun 10, 2026, 11:37 PM EDT
Recruiting Phase 3 Interventional

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

NCT07037771
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
zodasiran Injection, Placebo
Drug
Lead sponsor
Arrowhead Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
60 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
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 3 Interventional Results available

Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

NCT03156621
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 28, 2021 · Synced Jun 10, 2026, 11:37 PM EDT
Completed No phase listed Observational

Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients

NCT04148001
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 1, 2021 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 2 Interventional Results available

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

NCT00477594
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 8, 2016 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 3 Interventional Results available

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

NCT04233918
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 15, 2024 · Synced Jun 10, 2026, 11:37 PM EDT
Not listed Not applicable Interventional

HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia

NCT03135184
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 22, 2019 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 3 Interventional

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

NCT00134511
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 6, 2007 · Synced Jun 10, 2026, 11:37 PM EDT
Recruiting Not applicable Interventional

The ORIGIN-FH Study

NCT07470723
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Jun 10, 2026, 11:37 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

NCT02651675
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 12, 2023 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 2Phase 3 Interventional Results available

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

NCT01624142
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 27, 2024 · Synced Jun 10, 2026, 11:37 PM EDT
Not listed No phase listed Observational

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

NCT04080050
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 8, 2023 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 2 Interventional Results available

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

NCT02963311
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 17, 2020 · Synced Jun 10, 2026, 11:37 PM EDT
Recruiting No phase listed Observational

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

NCT01109368
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 3 Interventional Results available

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

NCT03409744
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 7, 2025 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 2 Interventional

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

NCT01412034
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 17, 2015 · Synced Jun 10, 2026, 11:37 PM EDT
Completed No phase listed Observational

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

NCT03018678
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 5, 2020 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 3 Interventional Results available

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

NCT03814187
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 5, 2024 · Synced Jun 10, 2026, 11:37 PM EDT
Terminated Phase 2 Interventional

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

NCT00079846
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jun 10, 2026, 11:37 PM EDT
Completed Phase 3 Interventional Results available

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

NCT02624869
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 28, 2024 · Synced Jun 10, 2026, 11:37 PM EDT
Recruiting No phase listed Observational

HoFH, the International Clinical Collaborators Registry

NCT04815005
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Jun 10, 2026, 11:37 PM EDT