Gena-99: Human-cl rhFVIII treatment for Haemophilia-A patients
Research type
Research Study
Full title
Prospective, multinational, non-interventional post-authorisation study to document the long-term immunogenicity, safety, and efficacy of Human-cl rhFVIII (simoctocog alfa) in patients with haemophilia A treated in routine clinical practice
IRAS ID
180245
Contact name
Johann Bichler
Contact email
Sponsor organisation
Octapharma AG
Duration of Study in the UK
3 years, 11 months, 31 days
Research summary
This is a prospective, multinational, non-interventional post-authorisation study designed to assess the long-term immunogenicity, safety, and efficacy of Human-cl rhFVIII in patients with haemophilia A treated in routine clinical practice. In brief, 200 male patients of any age with haemophilia (FVIII:C ≤ 2%) will be treated and followed for at least 100 exposure days (EDs). Of the 200 enrolled patients, at least 100 patients should have severe haemophilia A (FVIII:C < 1%). The overall duration of this study will be approximately 4 years.
The objectives of this study are:
• To assess the long-term immunogenicity and safety of Human-cl rhFVIII in treating or preventing bleeding episodes (BEs) in patients with Haemophilia A.
• To assess the long-term efficacy of Human-cl rhFVIII in treating or preventing BEs in patients with Haemophilia A.
The study will be performed in countries from across the EU and possibly other countries where Human-cl rhFVIII is intended to be marketed.REC name
London - Westminster Research Ethics Committee
REC reference
15/LO/1575
Date of REC Opinion
23 Nov 2015
REC opinion
Further Information Favourable Opinion