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Showing 1–24 of 41 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: HoFH Clear all
Completed Phase 3 Interventional Results available

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

NCT00730236
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 19, 2018 · Synced Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM EDT
Completed Phase 3 Interventional Results available

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

NCT00607373
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, 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
Source: ClinicalTrials.gov public record
Updated Sep 8, 2016 · Synced Sep 21, 2026, 9:59 AM 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 Jun 11, 2026 · Synced Sep 21, 2026, 9:59 AM EDT
Completed Phase 3 Interventional Results available

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

NCT00943306
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 12, 2018 · Synced Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM EDT
Terminated Phase 3 Interventional

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

NCT00263081
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 23, 2012 · Synced Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities

NCT01588496
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 28, 2018 · Synced Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM EDT
Completed Phase 3 Interventional Results available

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

NCT00694109
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
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
Source: ClinicalTrials.gov public record
Updated Sep 8, 2016 · Synced Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM EDT
Recruiting Phase 3 Interventional

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

NCT06597006
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM EDT
Completed No phase listed Observational

Cardiovascular Evaluation of Patients With High Cholesterol and Normal Volunteers

NCT00001204
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 30, 2026 · Synced Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM 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 Sep 21, 2026, 9:59 AM EDT