Human-cl rhFVIII treatment patients with Severe Haemophillia A Ext 01

  • Research type

    Research Study

  • Full title

    Extension Study for Patients who completed GENA-05 (NuProtect) – to Investigate Immunogenicity, Efficacy and Safety of Treatment with Human-cl rhFVIII

  • IRAS ID

    141253

  • Contact name

    Raina J Liesner

  • Contact email

    ri.liesner@gosh.nhs.uk

  • Sponsor organisation

    Octapharma Pharmazeutika Produktionsges m.B.H

  • Eudract number

    2013-003997-28

  • ISRCTN Number

    N/A

  • Research summary

    This study is designed as a prospective, multicentre, multinational, (approximately 20-30 centres worldwide) open label, non controlled phase IIIb study. This study is only designed for patients who completed GENA-05 in accordance with the study protocol.

    For each patient the exposure and the effect (efficacy) of Human cl rhFVIII in the prevention and treatment of bleeds, the frequency of break through bleeds in the case of prophylactic treatment and the tolerability and safety of Human cl rhFVIII will be thoroughly assessed.

    Screening Visit,6 Monthly Follow up Visits, Surgical Visits and Completion Visit will be performed. In the course of the follow up visits patients’ body weight will be checked, and blood samples will be obtained for FVIII inhibitor screen (after a wash-out period of at least 2, preferably 3 days after the previous IMP administration) and for the safety panel. Vital signs will be checked and a physical examination will be performed.

    The occurrence of AEs and changes in concomitant medication will be checked and documented. For each patient, the study will be completed once the IMP is registered and launched in the respective country, but no later than 2 years from the screening visit.

    At each visit, the patient diary will be reviewed and data transferred into the CRF.

    The patient is planned to stay in the study until the IMP is registered and launched in the individual country or for a maximum of 2 years.

    The study is planned to start in the 1st quarter of 2014 and continues until the 1st quarter of 2016.

    Screening Visits, 6-monthly Follow up Visits, and Completion Visit will be performed.

  • REC name

    Yorkshire & The Humber - Leeds East Research Ethics Committee

  • REC reference

    13/YH/0406

  • Date of REC Opinion

    10 Dec 2013

  • REC opinion

    Favourable Opinion