4031 N9-GP PASS (Paradigm 8)
Research type
Research Study
Full title
A Non-Interventional Post-Authorisation Safety Study (PASS) in male haemophilia B patients receiving Nonacog Beta Pegol (N9-GP) prophylaxis treatment
IRAS ID
264158
Contact name
Henry Watson
Contact email
Sponsor organisation
Novo Nordisk Ltd
Clinicaltrials.gov Identifier
U1111-1165-8657, UTN; EUPAS26592, EUPAS
Duration of Study in the UK
7 years, 5 months, 15 days
Research summary
The rationale for performing this study is to assess safety and effectiveness of Refixia® when given as long-term treatment (prophylaxis) to patients with haemophilia B. \nNovo Nordisk has received approval of N9-GP from the Food and Drug Administration(FDA),European Medicines Agency (EMA), Swissmedic, and Health Canada for the use in patients with haemophilia B either as prophylaxis or on-demand treatment for control of bleeding episodes as well as control or as prevention of bleeding in the perioperative setting.\nIn the EMA “Guideline on clinical investigation of recombinant and human plasma-derived factor IX (FIX) products”, EMA points to the low number of patients suffering from haemophilia B, and that data from pre-licensing studies are considered insufficient to assess all aspects of therapy with FIX products. Therefore, to collect additional clinical data and to ensure consistency between the\noutcome from the pre-authorisation clinical trials and those from post-approval local clinical practice use of commercially available product (Refixia®) a post-marketing investigation should be performed.\nThe key purpose of the N9-GP PASS is to gain additional knowledge of safety in prophylaxis patients and effectiveness of N9-GP when used as prophylactic treatment in haemophilia B patients.
REC name
South West - Central Bristol Research Ethics Committee
REC reference
20/SW/0061
Date of REC Opinion
18 Jun 2020
REC opinion
Further Information Favourable Opinion