|Principal Investigator: HassanYaish|
|Keywords: Novo Nordisk , Nonacog Beta Pegol , Paradigm , Haemophilia , Phase III||Department: Pediatric Administration|
|IRB Number: 00072297||Co Investigator:|
|Specialty: Pediatric Hematology and Oncology|
|Sub Specialties: Hemophilia|
The rationale for performing this trial is to investigate safety and efficacy of N9-GP in the treatment of previously untreated patients (PUP) with haemophilia B, hereby supporting anticipated marketing authorisation of N9-GP and line extension in the PUP population. EMA requires separate investigation of PUPs as part of the development programme initiated before market authorisation (MA) gets obtained. A final guideline on the clinical investigation of recombinant and human plasma-derived factor IX products5 from the Committee for Medicinal Products for human use Committee for Medical Producdt for Human Use(CHMP), describes the mandatory components for trials in PUP. In some countries outside the EU, PUP Paediatric investigation is necessary to achieve labelled indication for all children.
• To evaluate immunogenicity of N9-GP (nonacog beta pegol)
• To evaluate safety of N9-GP (nonacog beta pegol)
• To evaluate efficacy of N9-GP (nonacog beta pegol)
o in long-term prophylaxis treatment
o in the treatment of bleeding episodes
o through the surrogate marker: FIX activity
o through monitoring of number of doses and consumption of N9-GP
1. Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
2. Male, < 6 years of at the time of signing informed consent.
3. Patients with the diagnosis of haemophilia B (FIX activity level ≤ 2%) based on medical records or central laboratory results.
4. Previously untreated or exposed to FIX containing products less than or equal to 3 EDs (5 previous exposure days to blood components are acceptable).
1. Known or suspected hypersensitivity to trial product or related products.
2. Previous participation in this trial. Participation is defined as first dose administered of trial product.
3. Receipt of any investigational medicinal product within 30 days before screening.
4. Congenital or acquired coagulation disorder other than haemophilia B.
5. Any chronic disorder or severe disease which, in the opinion of the investigator, might jeopardise patient’s safety or compliance with the protocol.
6. Patient’s parent(s)/LAR(s) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.
7. Any history of FIX inhibitors (defined by medical records)