Protect-NOW – Octapharma’s FVIII in PUPs and MTPs
Research type
Research Study
Full title
PRactical utilization of Octapharma FVIII Concentrates in Previously Untreated and Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment (with Nuwiq, Octanate or Wilate). A Real-World Efficacy and Safety Observational Study
IRAS ID
230615
Contact name
Mary Mathias
Contact email
Sponsor organisation
Octapharma Pharmazeutika Produktionsges m.b.H.
Duration of Study in the UK
4 years, 7 months, 30 days
Research summary
Haemophilia A is an inherited disorder that affects blood clotting, usually in males. Patients have a low level of coagulation factor VIII, which makes them vulnerable to recurrent bleeding, particularly in joints and muscles. This disease is usually managed by treating the patient with FVIII, which helps the blood to clot and limit uncontrolled bleeding. Treatment regimens are not always standardised with respect to dosage, frequency or optimal start age of FVIII prophylactic treatment.
This is a prospective and retrospective, observational, multicentre, international, non-interventional study to investigate the use of Octapharma FVIII concentrates in routine clinical practice. 140 previously untreated patients and minimally treated patients with severe haemophilia A in approximately 50 centres worldwide, who have recently been prescribed Octapharma’s FVIII concentrates will be invited to participate in this study. The purpose of this study is to evaluate product utilisation, effectiveness and safety, and gather inhibitor development information. The development of antibodies/inhibitors against factor VIII is the most important complication in haemophilia treatment, especially during the early treatment phase, but it is not fully understood, which factors may play a role whether a patient develops an inhibitor or not.
An optional sub-study will monitor patient therapy regarding inhibitor development or eradication. No extra blood samples will be taken, instead a small amount of blood used for standard clinical tests will be used for studying inhibitors and genetic analysis.
During this observational study, no additional study visits or assessments will be required. Patients will be assessed by their medical team according to standard clinical practice. Patients or their parents will be asked to document information regarding treatment, bleeding episodes, other medication taken and any adverse drug reactions in a treatment diary. The planned observational period per patient is 100 exposure days to FVIII, or a maximum time of 3 years.REC name
East of England - Cambridge South Research Ethics Committee
REC reference
17/EE/0467
Date of REC Opinion
17 Apr 2018
REC opinion
Further Information Favourable Opinion