Anacetrapib in Patients with Homozygous Familial Hypercholesterolemia
Research type
Research Study
Full title
A Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-Week Study to Assess the Efficacy and Tolerability of Anacetrapib When Added to Ongoing Lipid-Lowering Therapy in Adult Patients with Homozygous Familial Hypercholesterolemia (HoFH) with a 52-Week Open-Label Extension
IRAS ID
117237
Contact name
Handrean Soran
Contact email
Sponsor organisation
Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc
Eudract number
2012-002434-37
Research summary
Homozygous Familial Hypercholesterolemia (HoFH) is a rare (1 in 1,000, 000 in the US and Europe) genetic disorder. HoFH causes extremely high levels of low density lipoproteins (LDL) cholesterol (LDL-C). Cholesterol is a type of fat found in the body. Total cholesterol is made up of LDL (low-density lipoprotein) and HDL (high-density lipoprotein) cholesterol as well as other components. LDL is called “bad” cholesterol because it can build up in the walls of arteries. This narrowing can slow or block blood flow and increase the risk of heart disease. High LDL (bad) cholesterol is a major cause of heart disease.
HoFH is caused by genetic mutations leading to poor or no functional ability to clear LDL-C via the liver.
A current therapy called LDL-C apheresis (a procedure in which blood is temporarily withdrawn, LDL-C selectively removed, and the rest of the blood is re-infused) lowers LDL-C by 40-70% but is invasive, costly, not widely available and must be regularly repeated (every 2 weeks).
This study aims to determine whether anacetrapib, an oral medication, can lower LDL-C effectively.
Approximately 45 patients will enter this study. Eligible patients will be randomized in a 1:1 ratio for 12 weeks to either anacetrapib 100 mg or placebo.
Patients will take one tablet of anacetrapib 100 mg or matching placebo once daily with a meal for the first 12 weeks of the study. Patients who complete the 12-week treatment period will be eligible to enter a 52-week open-label extension during which all patients will receive anacetrapib 100 mg. During the 52-week open-label extension, all patients will take one tablet of anacetrapib 100 mg once daily with a meal.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
13/NW/0001
Date of REC Opinion
23 May 2013
REC opinion
Further Information Favourable Opinion