Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
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. 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
Timeline
- Start
- 2017-10-03
- Primary completion
- 2019-09-27
- Completion
- 2020-02-13
Outcome
Met primary endpoint
paper At week 12, the least squares mean difference in LDL-C percent change from baseline was -35.6% (alirocumab [-26.9%] vs. placebo [8.6%]; p < 0.0001). PMID 32646561 ↗
release “In March 2020, the Company announced that the Phase 3 trial in adult patients with homozygous familial hypercholesterolemia (HoFH) met its primary endpoint” regeneron.com ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Alirocumab | Monoclonal antibody | 150 mg | Subcutaneous |