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ClinicalTrials.gov public records Last synced May 22, 2026, 12:04 AM EDT

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Showing 25–40 of 16 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Familial Hypercholesterolemia - Homozygous Clear all
Completed Phase 3 Interventional Results available
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 May 22, 2026, 12:04 AM EDT
Recruiting Phase 3 Interventional
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 May 5, 2026 · Synced May 22, 2026, 12:04 AM EDT
Not listed Not applicable Interventional
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 May 22, 2026, 12:04 AM EDT
Not listed No phase listed Observational
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 May 22, 2026, 12:04 AM EDT
Recruiting Not applicable Interventional
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 May 22, 2026, 12:04 AM EDT
Completed No phase listed Observational
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 May 22, 2026, 12:04 AM EDT
Active, not recruiting Phase 2 Interventional
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 Oct 9, 2025 · Synced May 22, 2026, 12:04 AM EDT
Recruiting Phase 3 Interventional
View directory record Official record
Conditions
Heterozygous or Homozygous Familial Hypercholesterolemia
Interventions
Inclisiran
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 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 May 14, 2026 · Synced May 22, 2026, 12:04 AM EDT
Completed Phase 3 Interventional Results available
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 May 22, 2026, 12:04 AM EDT
Completed Phase 3 Interventional Results available
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 May 22, 2026, 12:04 AM EDT
Completed Phase 2 Interventional Results available
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 May 22, 2026, 12:04 AM EDT
Completed Phase 2 Interventional
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 May 22, 2026, 12:04 AM EDT
Recruiting No phase listed Observational
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 May 22, 2026, 12:04 AM EDT
Completed Phase 2 Interventional Results available
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 May 22, 2026, 12:04 AM EDT
Recruiting Phase 1 Interventional
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
7
States / cities
Jacksonville, Florida • Orlando, Florida • Port Orange, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced May 22, 2026, 12:04 AM EDT
Completed Phase 3 Interventional Results available
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 May 22, 2026, 12:04 AM EDT