Clinical Trial: Study in Participants ≥12 Years of Age With Homozygous Familial Hypercholesterolemia (hoFH)

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia

Brief Summary: The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) with alirocumab subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment.

Detailed Summary:
Sponsor: Regeneron Pharmaceuticals

Current Primary Outcome: Percent change in LDL-C from baseline to Week 12 (Intent-to-Treat (ITT) population) [ Time Frame: Baseline to Week 12 ]

ITT population: All randomized participants who had an evaluable primary endpoint


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Percent change in apolipoprotein (Apo) B from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population
  • Percent change in non-high-density lipoprotein cholesterol (non-HDL-C) from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population
  • Percent change in total cholesterol (TC) from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population
  • Proportion of participants with ≥15% reduction in LDL-C at week 12 [ Time Frame: At Week 12 ]
    ITT population
  • Proportion of participants with ≥30% reduction in LDL-C at week 12 [ Time Frame: At Week 12 ]
    ITT population
  • Percent change in lipoprotein(a) [Lp(a)] from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population
  • Proportion of participants with ≥50% reduction in LDL-C at week 12 [ Time Frame: At Week 12 ]
    ITT population
  • Percent change in HDL-C from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population
  • Percent change in fasting triglycerides (TG) from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population
  • Percent change in Apo A-1 from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population
  • Percent change in LDL-C from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population
  • Percent change in LDL-C from baseline to week 12 (Modified Intent-to-Treat (m)ITT population) [ Time Frame: Baseline to Week 12 ]
    Modified Intent-to-Treat (m)ITT population: All randomized participants who took at least 1 dose or part of a dose of double-blind investigational study drug and has an evaluable primary endpoint
  • Percent change in Apo B from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT) population
  • Percent change in non-HDL-C from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT) population
  • Percent change in TC from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT population)
  • Percent change in Lp(a) from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT population)
  • Percent change in HDL-C from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT) population
  • Percent change in fasting TG from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT) population
  • Percent change in Apo A-1 from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    m(ITT) population
  • Proportion of patients with ≥15% reduction, ≥30% reduction, and ≥50% reduction in LDL-C at week 12 [ Time Frame: At Week 12 ]
    m(ITT) population
  • Absolute change in the ratio of Apo B/Apo A-1 from baseline to week 12 [ Time Frame: Baseline to Week 12 ]
    ITT population


Original Secondary Outcome: Same as current

Information By: Regeneron Pharmaceuticals

Dates:
Date Received: May 15, 2017
Date Started: August 2017
Date Completion: April 2019
Last Updated: May 16, 2017
Last Verified: May 2017