Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

Conditions

• Homozygous Familial Hypercholesterolemia

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Alirocumab — DRUG
  Alirocumab SC Q2W
• Placebo — DRUG
  Matching placebo SC Q2W

Study Details

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. The secondary objectives of the study are: * To evaluate the effect of alirocumab Q2W on other lipid parameters (ie, apolipoprotein \[Apo\] A-1 and B, non-high-density lipoprotein cholesterol \[non-HDL-C\], total-cholesterol \[TC\], proportion of participants with 15%, 30%, and 50% LDL-C reductions, Lp(a), HDL-C, triglycerides \[TG\]) in participants with HoFH * To evaluate the safety and tolerability of alirocumab SC Q2W in participants with HoFH * To assess the pharmacokinetics of alirocumab SC Q2W in participants with HoFH * To assess the potential development of anti-drug (alirocumab) antibodies

Key Dates

Start date

Oct 3, 2017

Status verified

Jun 2021

Primary completion

Sep 27, 2019

Completion

Feb 13, 2020

Study Design

Enrollment

69 participants (actual)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Alirocumab SC Q2W
  Alirocumab SC every 2 weeks (Q2W) from baseline (day 1) through week 10 during the double-blind treatment period Starting at week 12, and continuing through week 22, participants will receive open-label alirocumab SC Q2W
• Experimental: Placebo SC Q2W
  Matching placebo SC Q2W from baseline through week 10 during the double-blind treatment period Starting at week 12, and continuing through week 22, participants will receive open-label alirocumab SC Q2W

Primary Outcome Measure

Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 12 (Intent-to-Treat [ITT] Estimand) [ Time Frame: Baseline to Week 12 ]

Locations (4)

Find similar trials in Boca Raton, FL

Related Studies

• The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository
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• A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
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• HoFH, the International Clinical Collaborators Registry
  Recruiting · University of Pennsylvania · Philadelphia, Pennsylvania