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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Heterozygous Familial Clear all
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 25, 2026, 1:29 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 1, 2026 · Synced Sep 25, 2026, 1:29 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 Sep 25, 2026, 1:29 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 Sep 25, 2026, 1:29 AM EDT
Terminated Phase 4 Interventional Results available

Long Term Safety Study of PRALUENT

NCT03694197
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia, Non-familial Hypercholesterolemia
Interventions
Praluent
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
40 Years to 85 Years
Enrollment
1,389 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
57
States / cities
Auburn, Alabama • Mobile, Alabama • Beverly Hills, California + 50 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2021 · Synced Sep 25, 2026, 1:29 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 Sep 25, 2026, 1:29 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 Sep 25, 2026, 1:29 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
8
States / cities
Hialeah, Florida • Jacksonville, Florida • Orlando, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Sep 25, 2026, 1:29 AM 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 Sep 25, 2026, 1:29 AM EDT
Completed Phase 3 Interventional Results available

Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study-2

NCT01763918
View directory record Official record
Conditions
Hyperlipidemia
Interventions
Evolocumab, Placebo
Biological · Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years to 80 Years
Enrollment
331 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013
U.S. locations
2
States / cities
Scottsdale, Arizona • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 16, 2019 · Synced Sep 25, 2026, 1:29 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 (ODYSSEY HIGH FH)

NCT01617655
View directory record Official record
Conditions
Hypercholesterolaemia
Interventions
Alirocumab, Placebo (for alirocumab), Lipid Modifying Therapy (LMT)
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years and older
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
14
States / cities
Bell Gardens, California • Newport Beach, California • Northridge, California + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 3, 2016 · Synced Sep 25, 2026, 1:29 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 Sep 25, 2026, 1:29 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 Sep 25, 2026, 1:29 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 Sep 25, 2026, 1:29 AM EDT
Completed Phase 3 Interventional Results available

A 52 Week Study To Assess The Use Of Bococizumab (PF-04950615; RN316) In Subjects With Heterozygous Familial Hypercholesterolemia

NCT01968980
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia
Interventions
Bococizumab (PF-04950615;RN316), Placebo
Drug · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
370 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
22
States / cities
Los Angeles, California • Hartford, Connecticut • Hialeah, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2017 · Synced Sep 25, 2026, 1:29 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 15, 2026 · Synced Sep 25, 2026, 1:29 AM 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 Sep 25, 2026, 1:29 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 Sep 25, 2026, 1:29 AM EDT
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 25, 2026, 1:29 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 Sep 25, 2026, 1:29 AM 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 Sep 25, 2026, 1:29 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 7, 2016 · Synced Sep 25, 2026, 1:29 AM EDT
Completed Phase 2 Interventional

Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

NCT00281008
View directory record Official record
Conditions
Hypercholesterolemia, Familial
Interventions
ISIS 301012 or Placebo
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
7
States / cities
Chicago, Illinois • Auburn, Maine • Scarborough, Maine + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 2, 2016 · Synced Sep 25, 2026, 1:29 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 17, 2019 · Synced Sep 25, 2026, 1:29 AM EDT