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Showing 25–41 of 17 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: HoFH Clear all
Completed Not applicable Interventional

Regeneron 1331 Kinetics Sub-Study HoFH

NCT04722068
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 16, 2021 · Synced Sep 22, 2026, 1:32 AM EDT
Recruiting Phase 3 Interventional

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

NCT05682378
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Sep 22, 2026, 1:32 AM EDT
Completed No phase listed Observational

LOWER: Lomitapide Observational Worldwide Evaluation Registry

NCT02135705
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Sep 22, 2026, 1:32 AM EDT
Active, not 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
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
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Sep 22, 2026, 1:32 AM EDT
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 Sep 22, 2026, 1:32 AM 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 Sep 22, 2026, 1:32 AM EDT
Completed Phase 3 Interventional

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

NCT04034485
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 28, 2023 · Synced Sep 22, 2026, 1:32 AM EDT
Enrolling by invitation Phase 3 Interventional

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

NCT04798430
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 21, 2024 · Synced Sep 22, 2026, 1:32 AM EDT
Recruiting Phase 1 Interventional

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

NCT07491172
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Sep 22, 2026, 1:32 AM EDT
Completed Phase 2 Interventional Results available

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

NCT02722408
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 24, 2020 · Synced Sep 22, 2026, 1:32 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

NCT03399786
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 17, 2021 · Synced Sep 22, 2026, 1:32 AM 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 Sep 22, 2026, 1:32 AM 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 Sep 22, 2026, 1:32 AM 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 Sep 22, 2026, 1:32 AM 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 Sep 22, 2026, 1:32 AM EDT
Completed Phase 3 Interventional Results available

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

NCT04659863
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Sep 22, 2026, 1:32 AM 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 Sep 22, 2026, 1:32 AM EDT