FOCUS FH
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study followed by an Open-Label continuation period to Assess the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients with Familial Hypercholesterolemia and Inadequately Controlled Low-Density-Lipoprotein Cholesterol
IRAS ID
92204
Contact name
Handrean Soran
Sponsor organisation
Genzyme Corporation and its Affiliates
Eudract number
2011-001480-42
Clinicaltrials.gov Identifier
Research summary
Purpose of this research study is to look at the safety, effectiveness and tolerability of two dosing schedules of an investigational drug called Mipomersen. Mipomersen is thought to be beneficial by interfering with steps in the body that produces Low-Density lipoprotein (LDL) cholesterol, the 'bad' cholesterol in blood. Mipomersen is being studied in this research study to better understand how it affects lipid (cholesterol) levels in patients with familial hypercholesterolemia (FH). FH is a genetically inherited condition which causes the LDL cholesterol levels to be higher than normal in patients suffering from this. These high levels lead to a range of associated heart diseases. Many of the current and most widely used treatment for FH is directed at lowering the amount of LDL cholesterol in the blood by orally taken medication like statins, bile-acid sequestrants, fibrates, niacin and cholesterol absorption inhibitors. However even aggressive combinations will often not be sufficient in patients with severe forms of FH. LDL apheresis has also only shown to briefly lower the cholesterol level in the blood for a short while. This study is to help determine whether Mipomersen is a safe and effective under the skin injectable alternative viable treatment option for people who have high levels of LDL cholesterol in their blood. The study will ask patients suffering from FH to take a similar dose of the investigational drug either once weekly or three times a week via injections for 14 months. In order to assess the safety and effectiveness of the new drug a placebo (dummy drug of inactive substance) will also be given to some patients however it will be unknown whether the study drug or the placebo is being taken. In total a patient??s involvement will last approximately 21 months and will involve 19 study visits for study related procedures.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
12/NW/0014
Date of REC Opinion
21 Feb 2012
REC opinion
Further Information Favourable Opinion