A Study of Alirocumab in Participants With Autosomal Dominant Hypercholesterolemia (ADH) and Gain-of-Function Mutations (GOFm) of the Proprotein Convertase Subtilisin Kexin 9 (PCSK9) Gene or Loss-of-Function Mutations (LOFm) of the Apolipoprotein (Apo) B Gene
Completed · Phase 2 · Has a placebo group
Conditions studied: Hypercholesterolemia
In brief
The primary objective of the study is to assess the pharmacodynamic (PD) effect of alirocumab on serum low density lipoprotein cholesterol (LDL-C) during 14 weeks of subcutaneous (SC) administered alirocumab in patients with autosomal dominant hypercholesterolemia (ADH) and gain-of-function mutation (GOFm) in 1 or both alleles of the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or with loss-of-function mutation (LOFm) in 1 or more alleles of the apolipoprotein (ApoB) gene.
Key facts
- Study ID
- NCT01604824
- Run by
- Regeneron Pharmaceuticals
- People needed
- 23
- Starts
- 2012-02-22
- Expected to finish
- 2017-07-28
- Last updated by the study team
- 2020-06-17
Who can join
Age: 18 and older, up to 70. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Inclusion criteria include, but are not limited to the following:
- Between the ages of 18 and 70 years, inclusive
- A history of molecularly confirmed PCSK9 GOFm for cohort 1 and a history of molecularly confirmed PCSK9 GOFm or ApoB LOFm
- Plasma LDL-Cholesterol levels ≥70 mg/dL at the screening visit on a lipid-lowering therapy (LLT) regimen stable for at least 28 days
You may not qualify if…
- Exclusion criteria include, but are not limited to the following:
- Serum triglycerides >350 mg/dL at the screening visit
- Known to be positive for human immunodeficiency virus (HIV), hepatitis B virus, or hepatitis C virus
- Pregnant or breast-feeding women.
- Sexually active man or woman of childbearing potential who is unwilling to practice adequate contraception during the study
Where it is running
- Study site — Salt Lake City, Utah, United States
- Study site — Lille, Cedex, France
- Study site — Nantes, Cedex, France
- Study site — Paris, France
Full record on ClinicalTrials.gov
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