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Showing 1–24 of 121 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hypercholesterolemia, Autosomal Dominant Clear all
Enrolling by invitation Phase 3 Interventional

Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children 6 to 17 Years, With Heterozygous FH

NCT07102511
View directory record Official record
Conditions
Familial Hypercholesterolemia - Heterozygous
Interventions
lerodalcibep 300 mg Monthly SC
Biological
Lead sponsor
LIB Therapeutics LLC
Industry
Eligibility
6 Years to 17 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Sep 23, 2026, 8:17 PM EDT
Terminated No phase listed Observational

A Study to Identify and Characterize LAL-D Patients in High-risk Populations

NCT02345421
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years and older
Enrollment
640 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 12 more
Source: ClinicalTrials.gov public record
Updated May 23, 2016 · Synced Sep 23, 2026, 8:17 PM EDT
Recruiting Not applicable Interventional

Atlantic Lipid Lowering Treatment Optimization Program

NCT06439654
View directory record Official record
Conditions
Familial Hypercholesterolemia, Lipoprotein Types--Lp System Lp(A) Hyperlipoproteinemia, Apolipoprotein B 100, Familial Defective, High Density Lipoprotein Deficiency, Low-Density-Lipoid-Type Hyperlipoproteinemia
Interventions
Supportive care
Other
Lead sponsor
Atlantic Health System
Other
Eligibility
18 Years to 120 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Clark, New Jersey • Springfield, New Jersey
Source: ClinicalTrials.gov public record
Updated Aug 2, 2025 · Synced Sep 23, 2026, 8:17 PM EDT
Completed Phase 3 Interventional Results available

Evolocumab Compared to LDL-C Apheresis in Patients Receiving LDL-C Apheresis Prior to Study Enrollment

NCT02585895
View directory record Official record
Conditions
Hypercholesterolemia
Interventions
Evolocumab, Low-density Lipoprotein Cholesterol (LDL-C) Apheresis
Biological · Procedure
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Boca Raton, Florida • Kansas City, Kansas • Grandville, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 29, 2022 · Synced Sep 23, 2026, 8:17 PM EDT
Completed Phase 2 Interventional Results available

A Study of Alirocumab in Participants With Autosomal Dominant Hypercholesterolemia (ADH) and Gain-of-Function Mutations (GOFm) of the Proprotein Convertase Subtilisin Kexin 9 (PCSK9) Gene or Loss-of-Function Mutations (LOFm) of the Apolipoprotein (Apo) B Gene

NCT01604824
View directory record Official record
Conditions
Hypercholesterolemia
Interventions
Alirocumab, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 70 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jun 16, 2020 · Synced Sep 23, 2026, 8:17 PM EDT
Recruiting Phase 2 Interventional

2-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia (FH)

NCT04941599
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
2-Hydroxybenzylamine, Placebo
Drug · Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years to 69 Years
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Sep 23, 2026, 8:17 PM 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 23, 2026, 8:17 PM EDT
Completed No phase listed Observational

Patterns of Inclisiran Use in the Real World: An Analysis of US Databases

NCT07023445
View directory record Official record
Conditions
Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
37,688 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 16, 2025 · Synced Sep 23, 2026, 8:17 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 Sep 23, 2026, 8:17 PM EDT
Completed Phase 4 Interventional Results available

Efficacy and Safety of Colesevelam in Pediatric Patients With Genetic High Cholesterol

NCT00145574
View directory record Official record
Conditions
Hypercholesterolemia
Interventions
colesevelam HCl, placebo
Drug
Lead sponsor
Daiichi Sankyo
Industry
Eligibility
10 Years to 17 Years
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
8
States / cities
Washington D.C., District of Columbia • St Louis, Missouri • New Hyde Park, New York + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2010 · Synced Sep 23, 2026, 8:17 PM EDT
Completed Phase 3 Interventional Accepts healthy volunteers

Improved Diagnosis of Familial Hypercholesterolemia Across the Northland (ID-FH)

NCT05238519
View directory record Official record
Conditions
Familial Hypercholesterolemia, Cholesterol, Elevated, Genetic Disease
Interventions
Motivational interview (MI)
Behavioral
Lead sponsor
Essentia Health
Other
Eligibility
2 Years to 75 Years
Enrollment
257 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
2
States / cities
Duluth, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 23, 2026, 8:17 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 Sep 23, 2026, 8:17 PM EDT
Completed Phase 2 Interventional Results available

Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects

NCT01375764
View directory record Official record
Conditions
Hyperlipidemia
Interventions
Evolocumab, Ezetimibe, Placebo to Evolocumab
Biological · Drug · Other
Lead sponsor
Amgen
Industry
Eligibility
18 Years to 75 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
16
States / cities
Anaheim, California • Mission Viejo, California • Westlake Village, California + 12 more
Source: ClinicalTrials.gov public record
Updated Nov 6, 2022 · Synced Sep 23, 2026, 8:17 PM EDT
Completed Phase 2 Interventional Results available

A Study of the Efficacy and Safety of Enclitide Chloride (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-008)

NCT05261126
View directory record Official record
Conditions
Hypercholesterolemia, Familial Hypercholesterolemia
Interventions
Enclitide Chloride, Placebo
Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years to 80 Years
Enrollment
381 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
20
States / cities
Beverly Hills, California • Sacramento, California • Santa Ana, California + 17 more
Source: ClinicalTrials.gov public record
Updated Dec 8, 2024 · Synced Sep 23, 2026, 8:17 PM EDT
Completed Phase 3 Interventional Results available

Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)

NCT03397121
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia, Elevated Cholesterol
Interventions
Inclisiran, Placebo
Drug
Lead sponsor
The Medicines Company
Industry
Eligibility
18 Years and older
Enrollment
482 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
10
States / cities
Mission Viejo, California • Newport Beach, California • Stanford, California + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2020 · Synced Sep 23, 2026, 8:17 PM EDT
Completed Phase 3 Interventional Results available

A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol

NCT01475825
View directory record Official record
Conditions
Hypercholesterolemia, Heterozygous Familial
Interventions
mipomersen sodium 200 mg, Placebo, mipomersen sodium 70 mg
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
309 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
20
States / cities
Mission Viejo, California • Aurora, Colorado • Cooper City, Florida + 17 more
Source: ClinicalTrials.gov public record
Updated Mar 25, 2019 · Synced Sep 23, 2026, 8:17 PM EDT
Completed Phase 3 Interventional Results available

Study of Alirocumab (REGN727/SAR236553) in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) Undergoing Low-density Lipoprotein (LDL) Apheresis Therapy

NCT02326220
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia
Interventions
Placebo, Alirocumab
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
7
States / cities
Aurora, Colorado • Hartford, Connecticut • Kansas City, Kansas + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2020 · Synced Sep 23, 2026, 8:17 PM EDT
Completed Phase 3 Interventional Results available

Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders

NCT02392559
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia
Interventions
Evolocumab, Placebo
Drug
Lead sponsor
Amgen
Industry
Eligibility
10 Years to 17 Years
Enrollment
158 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
12
States / cities
Farmington, Connecticut • Wilmington, Delaware • Iowa City, Iowa + 9 more
Source: ClinicalTrials.gov public record
Updated Nov 7, 2022 · Synced Sep 23, 2026, 8:17 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 Sep 23, 2026, 8:17 PM 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 23, 2026, 8:17 PM 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 23, 2026, 8:17 PM 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 23, 2026, 8:17 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 Sep 23, 2026, 8:17 PM 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 23, 2026, 8:17 PM EDT