Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B
Purpose
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.
Conditions
- Congenital Bleeding Disorder
- Haemophilia B
Eligibility
- Eligible Ages
- Between 0 Years and 6 Years
- Eligible Genders
- Male
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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 - Male, age below 6 years at the time of signing informed consent - Patients with the diagnosis of haemophilia B (FIX (coagulation factor IX) activity level below or equal to 2%) based on medical records or central laboratory results - Previously untreated or exposed to FIX containing products less than or equal to 3 exposure days (5 previous exposures to blood components is acceptable)
Exclusion Criteria
- Any history of FIX inhibitors (defined by medical records) - Known or suspected hypersensitivity to trial product or related products - Previous participation in this trial. Participation is defined as first dose administered of trial product - Receipt of any investigational medicinal product within 30 days before screening - Congenital or acquired coagulation disorder other than haemophilia B - Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise the patient's safety or compliance with the protocol - Patient's parent(s)/LAR(s) (legally acceptable representative) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental 50 EDs (exposure days) |
|
More Details
- Status
- Completed
- Sponsor
- Novo Nordisk A/S