BMN 270-901: Prospective Laboratory Study in Haemophilia A Patients
Research type
Research Study
Full title
Global Prospective Laboratory Study for Adenovirus- Associated Virus (AAV)Seroprevalence in Hemophilia A Patients with Residual Factor VIII Levels ≤2 IU/dL Who Were Previously Treated with Factor VIII Concentrates
IRAS ID
243200
Contact name
Syed Rashid Saeed Kazmi
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Duration of Study in the UK
0 years, 11 months, 28 days
Research summary
This study is being conducted by BioMarin Pharmaceutical Inc., to determine if adults with severe or moderately severe haemophilia A who have previously received Factor VIII concentrates carry immunity to a certain type of virus called adeno-virus associated virus (AAV). AAV is a virus that can infect people but does not cause any symptoms or illness. Many people are exposed to, and are infected with, a type of AAV as children, and a proportion of them naturally create antibodies to fight the virus, making them immune.
This study is a research study and there is no study medication or therapy. If a participant agrees to participate, he will be asked to have a blood sample taken in the clinic, and a follow-up telephone conversation with study staff approximately 24 hours later. The study doctor may also call the participant back into the clinic at 3 months and/or 6 months for re-testing (approximately 20% of patients enrolled will be called back for such re testing). Each re-testing cycle includes a clinic visit to draw blood, and a follow-up telephone conversation with study staff approximately 24 hours later for any study procedure adverse reactions (e.g., redness, warmth, pain, bruising, or bleeding at the site where had the blood taken). If there is an adverse reaction at the blood taken site, and if deemed appropriate by the Investigator, the patient will be called to the site for an in-person assessment of the adverse reaction.
A total of 600 patients are expected to participate in this study.
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
18/SC/0166
Date of REC Opinion
21 Mar 2018
REC opinion
Favourable Opinion