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Showing 1–24 of 50 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Heterozygous Familial Clear all
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 Jul 22, 2026, 4:05 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 Jul 22, 2026, 4:05 PM 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 Jul 22, 2026, 4:05 PM 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 7, 2016 · Synced Jul 22, 2026, 4:05 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 Jul 22, 2026, 4:05 PM EDT
Completed Phase 2 Interventional Results available

Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia

NCT00477594
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
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
5
States / cities
Chicago, Illinois • Auburn, Maine • Biddeford, Maine + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2016 · Synced Jul 22, 2026, 4:05 PM 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 Jul 9, 2026 · Synced Jul 22, 2026, 4:05 PM 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 Jul 22, 2026, 4:05 PM EDT
Terminated No phase listed Observational

Inheritance of Osteosarcoma & Paget's Disease Through Chromosome 18:

NCT00615628
View directory record Official record
Conditions
Osteosarcoma, Paget's Disease
Interventions
Not listed
Lead sponsor
State University of New York - Upstate Medical University
Other
Eligibility
Not listed
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2009
U.S. locations
1
States / cities
Syracuse, New York
Source: ClinicalTrials.gov public record
Updated May 29, 2013 · Synced Jul 22, 2026, 4:05 PM EDT
Completed No phase listed Observational

Cancer Risk in Carriers of the Gene for Xeroderma Pigmentosum

NCT00046189
View directory record Official record
Conditions
Xeroderma Pigmentosum, Basal Cell Carcinoma, Squamous Cell Carcinoma, Melanoma, Skin Cancer
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month to 99 Years
Enrollment
301 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 7, 2024 · Synced Jul 22, 2026, 4:05 PM 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 20, 2021 · Synced Jul 22, 2026, 4:05 PM 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 Jul 22, 2026, 4:05 PM EDT
Completed Phase 3 Interventional Results available

PLUTO: Pediatric Lipid-redUction Trial of rOsuvastatin

NCT00355615
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
Rosuvastatin, Placebo
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
10 Years to 17 Years
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
4
States / cities
Los Angeles, California • Hyde Park, New York • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 30, 2011 · Synced Jul 22, 2026, 4:05 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 Jul 22, 2026, 4:05 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 Jul 22, 2026, 4:05 PM EDT
Completed Phase 3 Interventional

Carotid B-Mode Ultrasound Study to Compare Anti-Atherosclerotic Effect of Torcetrpib/Atorvastatin to Atorvastatin Alone.

NCT00136981
View directory record Official record
Conditions
Hypercholesterolemia, Familial, Hyperlipidemia
Interventions
torcetrapib/atorvastatin, atorvastatin
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 70 Years
Enrollment
800 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
7
States / cities
Hartford, Connecticut • Boston, Massachusetts • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2015 · Synced Jul 22, 2026, 4:05 PM 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 Jul 22, 2026, 4:05 PM 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 Jul 22, 2026, 4:05 PM 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 Jul 22, 2026, 4:05 PM 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 Jul 22, 2026, 4:05 PM 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 Jul 22, 2026, 4:05 PM EDT
Completed Phase 3 Interventional Results available

An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

NCT03510884
View directory record Official record
Conditions
Hypercholesterolaemia
Interventions
Alirocumab SAR236553 (REGN727), Rosuvastatin, Atorvastatin, Simvastatin, Pravastatin, Lovastatin, Fluvastatin, Ezetimibe, Cholestyramine, Nicotinic acid, Fenofibrate, Omega-3 fatty acids, Placebo
Drug
Lead sponsor
Sanofi
Industry
Eligibility
8 Years to 17 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
5
States / cities
Boca Raton, Florida • St Louis, Missouri • Charlotte, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated May 5, 2023 · Synced Jul 22, 2026, 4:05 PM 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 Jul 22, 2026, 4:05 PM EDT
Not yet recruiting Phase 3 Interventional

Randomized, Placebo-Controlled, Double-Blind, Phase 3b 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
2025 – 2027
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 2, 2025 · Synced Jul 22, 2026, 4:05 PM EDT