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ClinicalTrials.gov public records Last synced Oct 5, 2026, 1:10 AM EDT

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Showing 49–72 of 117 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Familial Hypercholesterolaemia Clear all
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 18, 2026 · Synced Oct 5, 2026, 1:10 AM EDT
Recruiting Phase 1 Interventional

Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia

NCT06451770
View directory record Official record
Conditions
Hypercholesterolemia, Hypertriglyceridemia, Refractory Hyperlipidemia
Interventions
VERVE-201
Drug
Lead sponsor
Verve Therapeutics, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
4
States / cities
Clearwater, Florida • Miami Lakes, Florida • Port Orange, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 5, 2026, 1:10 AM EDT
Recruiting Phase 1 Interventional

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

NCT06164730
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Interventions
VERVE-102
Drug
Lead sponsor
Verve Therapeutics, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Dothan, Alabama • Pomona, California • Boca Raton, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 5, 2026, 1:10 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 28, 2024 · Synced Oct 5, 2026, 1:10 AM EDT
Completed Phase 3 Interventional Results available

Atorvastatin Three Year Pediatric Study

NCT00827606
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
atorvastatin
Drug
Lead sponsor
Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Industry
Eligibility
6 Years to 15 Years
Enrollment
272 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
7
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Gainesville, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 5, 2026, 1:10 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 18, 2025 · Synced Oct 5, 2026, 1:10 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 19, 2025 · Synced Oct 5, 2026, 1:10 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 12, 2026 · Synced Oct 5, 2026, 1:10 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 15, 2026 · Synced Oct 5, 2026, 1:10 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 24, 2012 · Synced Oct 5, 2026, 1:10 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 9, 2016 · Synced Oct 5, 2026, 1:10 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 May 1, 2020 · Synced Oct 5, 2026, 1:10 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 29, 2021 · Synced Oct 5, 2026, 1:10 AM EDT
Completed Not applicable Interventional Results available

Evaluating the Efficacy of Pediatric Lipid Screening Alerts

NCT04118348
View directory record Official record
Conditions
Hypercholesterolemia, Hypercholesterolemia, Familial, Hypercholesteremia in Children, Hyperlipidemia in Children
Interventions
Best Practice Alert, Health Maintenance Topic
Behavioral
Lead sponsor
Geisinger Clinic
Other
Eligibility
9 Years to 11 Years
Enrollment
13,340 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Danville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 7, 2023 · Synced Oct 5, 2026, 1:10 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy

NCT01623115
View directory record Official record
Conditions
Hypercholesterolemia
Interventions
Alirocumab, Placebo (for alirocumab), Lipid Modifying Therapy (LMT)
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years and older
Enrollment
486 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
26
States / cities
Bell Gardens, California • Long Beach, California • Los Angeles, California + 21 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2016 · Synced Oct 5, 2026, 1:10 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 • Miami Lakes, Florida + 16 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Oct 5, 2026, 1:10 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 24, 2005 · Synced Oct 5, 2026, 1:10 AM EDT
Completed Phase 3 Interventional Results available

A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-013) CORALreef Lipids

NCT05952856
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
2,912 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
49
States / cities
Daphne, Alabama • Foley, Alabama • Phoenix, Arizona + 45 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 5, 2026, 1:10 AM EDT
Not listed No phase listed Observational

Study of Awareness and Detection of Familial Hypercholesterolemia

NCT01960244
View directory record Official record
Conditions
Hypercholesterolemia
Interventions
Not listed
Lead sponsor
The Familial Hypercholesterolemia Foundation
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2025
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Nov 27, 2023 · Synced Oct 5, 2026, 1:10 AM EDT
Completed Phase 2 Interventional Results available

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 Jul 17, 2026 · Synced Oct 5, 2026, 1:10 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 29, 2023 · Synced Oct 5, 2026, 1:10 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 9, 2023 · Synced Oct 5, 2026, 1:10 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 18, 2026 · Synced Oct 5, 2026, 1:10 AM EDT
Completed Phase 2 Interventional Results available

A Study of LY3015014 in Participants With High Cholesterol

NCT01890967
View directory record Official record
Conditions
Hypercholesterolemia
Interventions
LY3015014, Placebo, Statin, Ezetimibe
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
18 Years to 80 Years
Enrollment
527 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
15
States / cities
Encino, California • Lancaster, California • Spring Valley, California + 12 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2019 · Synced Oct 5, 2026, 1:10 AM EDT