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ClinicalTrials.gov public records Last synced May 22, 2026, 3:48 AM EDT

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Showing 1–24 of 47 matching trials from the live ClinicalTrials.gov search.

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Filters: Condition: Familial Hypercholesterolemia - Heterozygous Clear all

Completed Phase 3 Interventional Results available

Atorvastatin Three Year Pediatric Study

NCT00827606

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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 May 22, 2026, 3:48 AM EDT

Recruiting Phase 3 Interventional

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

NCT06597019

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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 May 22, 2026, 3:48 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

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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 May 22, 2026, 3:48 AM EDT

Completed Phase 2 Interventional Results available

An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

NCT02890992

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Conditions: Hypercholesterolaemia
Interventions: alirocumab SAR236553 (REGN727), statins, ezetimibe, cholestyramine, fenofibrate, omega-3 fatty acids, nicotinic acid; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 8 Years to 17 Years

Enrollment: 42 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 3
States / cities: St Louis, Missouri • Charlotte, North Carolina • Cincinnati, Ohio

Source: ClinicalTrials.gov public record

Updated Sep 5, 2019 · Synced May 22, 2026, 3:48 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

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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 May 22, 2026, 3:48 AM EDT

Completed Phase 3 Interventional Results available

Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.

NCT05425745

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Conditions: Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia, Lipid Metabolism Disorder, Metabolic Disease, Lipid Metabolism, Inborn Errors, Genetic Disease, Inborn, Hyperlipoproteinemias
Interventions: Obicetrapib, Placebo; Drug
Lead sponsor: NewAmsterdam Pharma; Industry
Eligibility: 18 Years and older

Enrollment: 354 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 20
States / cities: Jonesboro, Arkansas • Toluca Lake, California • Sarasota, Florida + 17 more

Source: ClinicalTrials.gov public record

Updated Jun 10, 2025 · Synced May 22, 2026, 3:48 AM EDT

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

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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 May 22, 2026, 3:48 AM 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

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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 May 22, 2026, 3:48 AM EDT

Completed Phase 2 Interventional

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

NCT00281008

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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 May 22, 2026, 3:48 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

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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 May 22, 2026, 3:48 AM EDT

Completed Phase 4 Interventional Results available

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

NCT00145574

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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 May 22, 2026, 3:48 AM EDT

Completed Phase 2 Interventional Results available

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

NCT00477594

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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 May 22, 2026, 3:48 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

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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 May 22, 2026, 3:48 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

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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 May 22, 2026, 3:48 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

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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 May 22, 2026, 3:48 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

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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 May 22, 2026, 3:48 AM EDT

Completed Phase 3 Interventional Results available

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

NCT03510884

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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 May 22, 2026, 3:48 AM EDT

Completed Phase 3 Interventional Results available

Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease

NCT00706849

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Conditions: Heterozygous Familial Hypercholesterolemia, Coronary Artery Disease
Interventions: mipomersen sodium, placebo; Drug
Lead sponsor: Kastle Therapeutics, LLC; Industry
Eligibility: 18 Years and older

Enrollment: 124 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 37
States / cities: La Jolla, California • Los Angeles, California • Mission Viejo, California + 31 more

Source: ClinicalTrials.gov public record

Updated Sep 8, 2016 · Synced May 22, 2026, 3:48 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

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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 May 22, 2026, 3:48 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

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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 May 22, 2026, 3:48 AM EDT

Completed Phase 3 Interventional Results available

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

NCT01763918

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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 May 22, 2026, 3:48 AM 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

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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 May 22, 2026, 3:48 AM EDT

Terminated Phase 4 Interventional Results available

Long Term Safety Study of PRALUENT

NCT03694197

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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 May 22, 2026, 3:48 AM EDT

Completed Phase 3 Interventional

Study To Evaluate The Safety And Efficacy Of Torcetrapib/Atorvastatin In Subjects With Familial Hypercholerolemia

NCT00134485

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Conditions: Hypercholesterolemia, Familial, Hyperlipidemia
Interventions: torcetrapib/atorvastatin, atorvastatin; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years and older

Enrollment: 400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 14
States / cities: San Diego, California • Washington D.C., District of Columbia • Lutz, Florida + 11 more

Source: ClinicalTrials.gov public record

Updated Oct 29, 2007 · Synced May 22, 2026, 3:48 AM EDT