Overview

This trial has been completed.

Condition heterozygous familial hypercholesterolemia
Treatments alirocumab, placebo
Phase phase 3
Sponsor Regeneron Pharmaceuticals
Collaborator Sanofi
Start date March 2015
End date January 2016
Trial size 62 participants
Trial identifier NCT02326220, R727-CL-1216

Summary

This is a randomized, double-blind, placebo-controlled, parallel-group study to evaluate the effect of alirocumab in comparison with placebo on the frequency of LDL apheresis treatments in patients with HeFH undergoing LDL apheresis therapy.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
Alirocumab subcutaneous (SC) dose regimen
alirocumab REGN727
(Placebo Comparator)
Placebo matching alirocumab subcutaneous (SC) dose regimen
placebo

Primary Outcomes

Measure
Rate of apheresis treatments during a 12-week period normalized by the number of planned apheresis treatments according to each patient's established schedule at screening
time frame: 12 weeks

Secondary Outcomes

Measure
Percent change from baseline in low-density lipoprotein cholesterol (LDL-C) (pre-apheresis)
time frame: Baseline to week 6
Standardized rate of apheresis treatments during a 4-week period
time frame: 4 weeks
Percent change from baseline in apolipoprotein B (ApoB) (pre-apheresis) (pre-apheresis) to week 6
time frame: Baseline to week 6
Percent change from baseline in non-high-density lipoprotein cholesterol (HDL-C) (pre-apheresis)
time frame: Baseline to week 6
Percent change from baseline in total cholesterol (pre-apheresis)
time frame: Baseline to week 6
Percent change from baseline in ApoA-1 (pre-apheresis)
time frame: Baseline to week 6
Proportion of patients with ≥30% reduction in LDL-C (pre-apheresis)
time frame: At week 6
Proportion of patients with ≥50% reduction in LDL-C (pre-apheresis)
time frame: At week 6
Percent change from baseline in LDL-C (pre-apheresis)
time frame: Baseline to week 18
Percent change from baseline in ApoB (pre-apheresis)
time frame: Baseline to week 18
Percent change from baseline in non-HDL-C (pre-apheresis) to week 18
time frame: baseline to week 18
Percent change from baseline in total cholesterol (pre-apheresis) to week 18
time frame: baseline to week 18
Percent change from baseline in ApoA-1(pre-apheresis) to week 18
time frame: baseline to week 18
Proportion of patients with ≥30% reduction in LDL-C (pre-apheresis) at week 18 (pre-apheresis)
time frame: At week 18
Proportion of patients with ≥50% reduction in LDL-C (pre-apheresis) at week 18
time frame: At week 18
Change of W-BQ22 (Well-Being Questionnaire 22-item version) from baseline to week 18
time frame: Baseline to week 18
Percent change from baseline in lipoprotein (a) [Lp(a)] (pre-apheresis)
time frame: Baseline to week 6
Percent change from baseline in HDL-C (pre-apheresis)
time frame: Baseline to week 6
Percent change from baseline in triglyceride (TG) levels (pre-apheresis)
time frame: Baseline to week 6
Percent change from baseline in Lp(a) (pre-apheresis) to week 18
time frame: Baseline to week 18
Percent change from baseline in HDL-C (pre-apheresis) to week 18
time frame: Baseline to week 18
Percent change from baseline in TG levels (pre-apheresis) to week 18
time frame: Baseline to week 18

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: 1. Men and women ≥18 years of age at the time of the screening visit 2. Diagnosis of HeFH (Heterozygous familial hypercholesterolemia) 3. Currently undergoing LDL (low-density lipoprotein) apheresis therapy QW (weekly) or Q2W (every 2 weeks) or at least 8 weeks prior to the screening visit Exclusion Criteria: 1. Homozygous FH (familial hypercholesterolemia) 2. Background medical LMT (lipid-modifying therapy) (if applicable) that has not been stable for at least 8 weeks prior to the screening visit 3. LDL apheresis schedule/ apheresis settings that have not been stable for at least 8 weeks prior to the screening visit 4. An LDL apheresis schedule other than QW to Q2W 5. Initiation of a new exercise program or exercise that has not remained stable within 8 weeks prior to the screening visit (week -2) 6. Initiation of a new diet or a diet that has not been stable within 8 weeks prior to the screening visit (week -2) 7. Use of nutraceuticals or over-the-counter therapies known to affect lipids, at a dose/amount that has not been stable for at least 8 weeks prior to the screening visit (week -2), or between the screening and randomization visit 8. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins 9. Known history of a positive test for human immunodeficiency virus 10. Use of any active investigational drugs within 1 month or 5 half-lives of screening, whichever is longer 11. Patients who have been treated with at least 1 dose of alirocumab or any other anti-PCSK9 monoclonal antibody in any other clinical studies 12. Pregnant or breastfeeding women

Additional Information

Official title A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Heterozygous Familial Hypercholesterolemia Undergoing Lipid Apheresis Therapy
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Regeneron Pharmaceuticals.