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ClinicalTrials.gov public records Last synced Jun 11, 2026, 5:32 AM EDT

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Showing 1–24 of 25 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Heterozygous Familial Hypercholesterolemia (HeFH) Clear all
Completed Phase 3 Interventional Results available

Evaluation of Long-Term Safety and Tolerability of ETC-1002 in High-Risk Patients With Hyperlipidemia and High CV Risk (CLEAR Harmony)

NCT02666664
View directory record Official record
Conditions
Hypercholesterolemia, Atherosclerotic Cardiovascular Diseases
Interventions
ETC-1002, Placebo
Drug
Lead sponsor
Esperion Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
2,230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
48
States / cities
Huntsville, Alabama • Cottonwood, Arizona • Phoenix, Arizona + 45 more
Source: ClinicalTrials.gov public record
Updated May 10, 2020 · Synced Jun 11, 2026, 5:32 AM EDT
Completed Phase 2 Interventional Results available

Study of the Safety and Efficacy of REGN727/SAR236553 in Patients With HeFH Hypercholesterolemia

NCT01266876
View directory record Official record
Conditions
Hypercholesterolemia
Interventions
Alirocumab, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 75 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
17
States / cities
Huntsville, Alabama • Mission Viejo, California • Newport Beach, California + 14 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2015 · Synced Jun 11, 2026, 5:32 AM EDT
Terminated Phase 2 Interventional

Implitapide in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) on Maximal Concurrent Lipid-Lowering Therapy

NCT00079859
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
Implitapide
Drug
Lead sponsor
Medical Research Laboratories International
Industry
Eligibility
18 Years to 70 Years
Enrollment
120 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 11, 2026, 5:32 AM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Safety and Efficacy of Bempedoic Acid Plus Ezetimibe Fixed-Dose Combination Compared to Bempedoic Acid, Ezetimibe, and Placebo in Patients Treated With Maximally Tolerated Statin Therapy

NCT03337308
View directory record Official record
Conditions
Hyperlipidemias
Interventions
Bempedoic Acid + Ezetimibe Fixed-Dose Combination, Bempedoic Acid, Ezetimibe, Placebos
Combination Product · Drug
Lead sponsor
Esperion Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
382 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
5
States / cities
Ames, Iowa • Nashua, New Hampshire • Statesville, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2020 · Synced Jun 11, 2026, 5:32 AM 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 Jun 11, 2026, 5:32 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 Jun 11, 2026, 5:32 AM EDT
Completed Phase 2 Interventional Results available

Open-Label Extension of Study R727-CL-1003 (NCT01266876) to Evaluate the Long-Term Safety and Efficacy of Alirocumab (REGN727) in Participants With Heterozygous Familial Hypercholesterolemia (HeFH)

NCT01576484
View directory record Official record
Conditions
Hypercholesterolemia, Heterozygous Familial Hypercholesterolemia
Interventions
Alirocumab, Placebo Matched to Alirocumab
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 75 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
11
States / cities
Mission Viejo, California • Newport Beach, California • Thousand Oaks, California + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2020 · Synced Jun 11, 2026, 5:32 AM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

NCT06597019
View directory record Official record
Conditions
Familial Hypercholesterolemia - Heterozygous
Interventions
Inclisiran, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
6 Years to 11 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
9
States / cities
San Francisco, California • Washington D.C., District of Columbia • Boca Raton, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Jun 11, 2026, 5:32 AM EDT
Completed Phase 3 Interventional

Study to Assess the Efficacy and Safety of LIB003 in HeFH Patients on Oral Lipid Therapy Needing Further LDL-C Reduction

NCT04797104
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia
Interventions
lerodalcibep
Drug
Lead sponsor
LIB Therapeutics LLC
Industry
Eligibility
18 Years and older
Enrollment
478 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 10, 2023 · Synced Jun 11, 2026, 5:32 AM EDT
Completed Phase 3 Interventional Results available

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

NCT04652726
View directory record Official record
Conditions
Familial Hypercholesterolemia - Heterozygous
Interventions
Inclisiran, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 17 Years
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
6
States / cities
Tucson, Arizona • New York, New York • Winston-Salem, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Jun 11, 2026, 5:32 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 Jun 11, 2026, 5:32 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 Jun 11, 2026, 5:32 AM EDT
Recruiting Phase 2Phase 3 Interventional

A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)

NCT07058077
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia (HeFH)
Interventions
Enlicitide Decanoate, Placebo
Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
6 Years to 17 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2037
U.S. locations
6
States / cities
Wilmington, Delaware • Washington D.C., District of Columbia • Boca Raton, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Jun 11, 2026, 5:32 AM EDT
Completed Phase 3 Interventional Results available

Randomized Study to Evaluate the Effect of Obicetrapib on Top of Maximum Tolerated Lipid-Modifying Therapies

NCT05142722
View directory record Official record
Conditions
Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Interventions
Placebo, Obicetrapib
Drug
Lead sponsor
NewAmsterdam Pharma
Industry
Eligibility
18 Years and older
Enrollment
2,530 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
69
States / cities
Anniston, Alabama • Birmingham, Alabama • Saraland, Alabama + 60 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2025 · Synced Jun 11, 2026, 5:32 AM EDT
Completed Phase 2 Interventional

A Clinical Study in Children With Heterozygous Familial Hypercholesterolemia (HeFH) Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing (CLEAR Path 1)

NCT05694260
View directory record Official record
Conditions
Hypercholesterolemia
Interventions
Bempedoic acid
Drug
Lead sponsor
Esperion Therapeutics, Inc.
Industry
Eligibility
6 Years to 17 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
8
States / cities
Los Angeles, California • West Covina, California • Boca Raton, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2025 · Synced Jun 11, 2026, 5:32 AM EDT
Completed Phase 3 Interventional Results available

Assessment of the Long-Term Safety and Efficacy of Bempedoic Acid (CLEAR Harmony OLE)

NCT03067441
View directory record Official record
Conditions
Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Interventions
bempedoic acid
Drug
Lead sponsor
Esperion Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
1,462 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
3
States / cities
Tampa, Florida • Louisville, Kentucky • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 28, 2021 · Synced Jun 11, 2026, 5:32 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 Jun 11, 2026, 5:32 AM 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 Jun 11, 2026, 5:32 AM 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 Jun 11, 2026, 5:32 AM EDT
Completed Phase 3 Interventional Results available

A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017/CORALreef HeFH)

NCT05952869
View directory record Official record
Conditions
Hypercholesterolemia, Familial Hypercholesterolemia
Interventions
Enlicitide Decanoate, Placebo
Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years and older
Enrollment
303 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
13
States / cities
Daphne, Alabama • Boca Raton, Florida • Cutler Bay, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Feb 23, 2026 · Synced Jun 11, 2026, 5:32 AM EDT
Active, not recruiting Phase 3 Interventional

A Phase III Study to Assess the Effect of AZD0780 on LDL-C in Patients With HeFH

NCT07000136
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolaemia
Interventions
AZD0780, Placebo
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years and older
Enrollment
473 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
19
States / cities
Garden Grove, California • San Diego, California • Hialeah, Florida + 16 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Jun 11, 2026, 5:32 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
7
States / cities
Jacksonville, Florida • Orlando, Florida • Port Orange, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Jun 11, 2026, 5:32 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
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 Jun 11, 2026, 5:32 AM EDT
Completed Phase 3 Interventional Results available

Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies

NCT06005597
View directory record Official record
Conditions
Dyslipidemias, Hypercholesterolemia, Familial Hypercholesterolemia, ASCVD, High Cholesterol
Interventions
Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe, Combination Therapy placebo, Obicetrapib Placebo, Ezetimibe Placebo
Combination Product · Drug · Other
Lead sponsor
NewAmsterdam Pharma
Industry
Eligibility
18 Years and older
Enrollment
407 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
45
States / cities
Gilbert, Arizona • Jacksonville, Florida • Port Orange, Florida + 37 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2025 · Synced Jun 11, 2026, 5:32 AM EDT