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 ]
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