BMN 270-902: Non-Interventional Study in Severe Hemophilia A Patients
Research type
Research Study
Full title
A Prospective Non-Interventional Study of Bleeding Episodes, Factor VIII Infusions, and Patient-Reported Outcomes in Individuals with Severe Hemophilia A
IRAS ID
236410
Contact name
John Pasi
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Duration of Study in the UK
2 years, 0 months, 31 days
Research summary
This study is being conducted by BioMarin Pharmaceutical Inc. to prospectively collect bleeding episodes, Factor VIII (FVIII) utilisation, and quality of life information from patients with severe Haemophilia A across multiple countries.
Haemophilia A (HA) is an X-linked recessive bleeding disorder that affects approximately 1 in 5,000 males. It is caused by deficiency in the activity of coagulation factor VIII (FVIII), which is essential to blood clotting. Severe HA is
classified as FVIII activity less than 1%. Clinical manifestations of severe HA are frequent spontaneous bleeding episodes, predominantly in joints and soft tissues, with a substantially increased risk of death from haemorrhage when the brain is involved.This study is a research study and there is no study medication or therapy that will be given to the participants. Approximately 100 participants will be recruited to participate in the study for a minimum of 6 months and up to 1 year which will include a Screening and Enrollment period, followed by monthly visits which can either be conducted during the participants normal standard of care visits to the clinic or done via telephone or email.
REC name
South Central - Hampshire B Research Ethics Committee
REC reference
17/SC/0604
Date of REC Opinion
27 Dec 2017
REC opinion
Further Information Favourable Opinion