Pfizer B1481021 - Phase 3 PF-04950615 LDL-C lowering in HeFH
Research type
Research Study
Full title
A 52 week, phase 3 double-blind, randomized, placebo-controlled, parallel-group study to assess the efficacy, safety and tolerability of PF-04950615 in subjects with heterozygous familial hypercholesterolemia
IRAS ID
142190
Contact name
Handrean Soran
Contact email
Sponsor organisation
Pfizer Inc
Eudract number
2013-002644-87
Research summary
Cardiovascular disease (CVD) due to atherosclerosis continues to be the leading single cause of death in industrialised countries. High serum lipid levels, and especially high low density lipoprotein cholesterol (LDL-C) levels, have been demonstrated to strongly and directly correlate with cardiovascular disease risks by numerous epidemiological studies. Moreover, large prospective clinical outcome trials have demonstrated that lowering LDL-C decreases cardiovascular morbidity and mortality. Despite the availability of lipid lowering therapies such as statins and ezetimibe, a significant percentage of subjects at high risk for CVD fail to reach or maintain their LDL-C treatment target.
Familial hypercholesterolemia (FH) is an inherited genetic disorder of lipoprotein metabolism. Heterozygous familial hypercholesterolemia (HeFH) has a prevalence of 1 in 500. Untreated, the majority of those affected will have symptomatic coronary disease by the age of 60 years and half of the mean and 15% of the women will have died.
PF-04950615 is a drug being developed for the treatment of primary hyperlipidemia and mixed dyslipidemia (elevated levels of lipids in the blood).
This is a Phase 3, double-blind, placebo-controlled, randomised, parallel group, multi-centre clinical trial designed to compare the efficacy, safety and tolerability of PF-04950615 150 mg administered subcutaneously (SC) (by injection) every two weeks to placebo for LDL-C lowering in subjects with HeFH. After consenting, participants will enter a 28 days screening period to verify eligibility for the trial. Only participants who meet all eligibility criteria will be randomised. Randomised participants will enter a 52-week treatment period, followed by an 8 week follow-up.
Participants will be randomised in a 1:1 ratio to receive PF-04950615 or placebo. Approximately 300 participants will be randomised at approximately 65 sites in 11 countries.
Throughout the study, participants will undergo physical examinations, vital signs and ECG; blood and urine will be taken for various tests; instruction on self-injection and diet and therapeutic lifestyle recommendations will be provided.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
13/YH/0389
Date of REC Opinion
29 Jan 2014
REC opinion
Further Information Favourable Opinion