B0401016 - PF-04447943 Sickle Cell Disease Study
Research type
Research Study
Full title
A PHASE 1B, RANDOMIZED, DOUBLE-BLIND (SPONSOR OPEN), PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY,TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF PF-04447943, CO-ADMINISTERED WITH AND WITHOUT HYDROXYUREA, IN SUBJECTS WITH STABLE SICKLE CELL DISEASE
IRAS ID
164023
Contact name
Gemma Sheppard
Contact email
Sponsor organisation
Pfizer Inc.
Eudract number
2014-001677-13
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
119,467, IND number
Duration of Study in the UK
0 years, 11 months, 21 days
Research summary
Sickle Cell Disease is a hereditary blood disorder in which the red blood cells become an abnormal rigid sickle shape. This sickling occurs because of a mutation in the haemoglobin gene. These sickled red cells are unable to flow easily through the blood vessels so they are more likely to break down or to get stuck in the blood vessels. This causes a chronic anaemia and acute and chronic complications including acute pain, stroke, renal disease, cardiac disease and eye problems.
The purpose of this research study is to learn about the effects and safety of the study drug, PF-0447943, for the first time in patients with sickle cell disease. Hydroxyurea is a medication used in treated sickle cell anaemia and patients will be included whether or not they are taking this.
Up to sixty-four (64) male and female subjects, between 18 and 65 years of age with stable sickle cell disease will be enrolled in this study. The study is being conducted at approximately 20 different research sites in 3 to 5 countries.
Participants will be assigned randomly to receive either the study drug or placebo. The ratio of receiving the study drug to placebo is 3:1. Participants assigned to receive the study drug, will receive a twice-daily oral dose of either 25 mg or 40mg of the study drug. The dose may be amended depending on the results observed by the study doctor and study sponsor.
Participants will be in this study for about 12 weeks and will need to visit the research site at least 7 times during the study.
This is a Phase IB, Randomised, Double-Blind (Sponsor Open), Placebo-Controlled Study which means neither the participant nor the study doctor will know which treatment the participant is assigned to.
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
14/WM/1202
Date of REC Opinion
2 Dec 2014
REC opinion
Further Information Favourable Opinion