Extension study to investigate CSL627 in Subjects with Haemophilia A
Research type
Research Study
Full title
A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII-SingleChain, CSL627) in Subjects with Severe Hemophilia A
IRAS ID
157260
Contact name
Pratima Chowdary
Contact email
Sponsor organisation
CSL Behring GmbH
Eudract number
2013-003262-13
Research summary
Hemophilia A is a serious bleeding disorder that affects males and is caused because of a deficiency in clotting factor VIII. The severity of Hemophilia A is based on the level of clotting FVIII that a patient has and patients with less than 1% of the normal level of FVIII are considered severe and often bleed spontaneously without any cause.
The treatment for patients with severe Hemophilia A is replacement of the missing clotting factor (FVIII) however 33% of patients develop a resistance to this treatment, CSL Behring have developed CSL627 ( A Recombinant Factor VIII (rFVIII)) and the purpose of this study is to evaluate the safety and efficacy of the study drug CSL627 in patients with Hemophilia A. This submission is an application to conduct an extension study within the UK.
Approximately 200 patients ages birth = 65 years of age will be enrolled. The study includes a day 1 visit to start this study, and an active treatment period with the goal of you receiving a minimum of 100 exposure days to the study medication which would include previous exposures from other rVIII-SingleChain studies. An exposure day is defined as any day that the patient receives an infusion of the study medication. The length of time that a patient will be in the study will vary but may be up to 3 years or when the study is stopped, the product becomes commercially available in your country or patient withdraws from the study.REC name
London - Bloomsbury Research Ethics Committee
REC reference
14/LO/1035
Date of REC Opinion
1 Sep 2014
REC opinion
Further Information Favourable Opinion