A Study of Alirocumab (REGN727/SAR236553) in Patients With ADH and GOFm of the PCSK9 Gene or LOFm of the apoB Gene
| Status: | Unknown status |
|---|---|
| Conditions: | High Cholesterol |
| Therapuetic Areas: | Cardiology / Vascular Diseases |
| Healthy: | No |
| Age Range: | 18 - 70 |
| Updated: | 10/14/2017 |
| Start Date: | September 2012 |
| End Date: | September 2016 |
A Phase 2 Pilot Study With a Randomized Double-Blind Treatment Phase to Evaluate the Pharmacodynamics and Safety of Alirocumab in Patients With Autosomal Dominant Hypercholesterolemia and Gain-of-Function Mutations in 1 or Both Alleles of the PCSK9 Gene or Loss-of-Function Mutations in 1 or More Alleles of the Apolipoprotein B Gene
The purpose of this study is to assess the pharmacodynamic (PD) effect of alirocumab on serum
low-density lipoprotein cholesterol (LDL-C) in patients with autosomal dominant
hypercholesterolemia (ADH) and gain-of-function mutations (GOFm) in 1 or both alleles of the
proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or with a loss-of-function
mutation (LOFm) in 1 or more alleles of the apolipoprotein B-100 gene (apoB).
low-density lipoprotein cholesterol (LDL-C) in patients with autosomal dominant
hypercholesterolemia (ADH) and gain-of-function mutations (GOFm) in 1 or both alleles of the
proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or with a loss-of-function
mutation (LOFm) in 1 or more alleles of the apolipoprotein B-100 gene (apoB).
Inclusion Criteria:
Inclusion criteria include, but are not limited to the following:
1. Between the ages of 18 and 70 years, inclusive
2. A history of molecularly confirmed PCSK9 GOFm for cohort 1 and a history of
molecularly confirmed PCSK9 GOFm or ApoB LOFm
3. Plasma LDL-Cholesterol levels ≥70 mg/dL at the screening visit on a lipid lowering
therapy (LLT) regimen stable for at least 28 days
Exclusion Criteria:
Exclusion criteria include, but are not limited to the following:
1. Serum triglycerides >350 mg/dL at the screening visit
2. Known to be positive for human immunodeficiency virus (HIV), hepatitis B virus, or
hepatitis C virus
3. Pregnant or breast-feeding women.
4. Sexually active man or woman of childbearing potential who is unwilling to practice
adequate contraception during the study
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