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ClinicalTrials.gov public records Last synced May 22, 2026, 3:53 AM EDT

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Showing 1–3 of 3 matching trials from the live ClinicalTrials.gov search.

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Filters: Condition: Heterozygous or Homozygous Familial Hypercholesterolemia Clear all

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, 3:53 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, 3:53 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

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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 May 22, 2026, 3:53 AM EDT