Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B (paradigm™6)

Novo Nordisk

Status and phase: Completed; Phase 3

Conditions: Haemophilia B; Congenital Bleeding Disorder

Treatments: Drug: nonacog beta pegol

Study type: Interventional

Funder types: Industry

Identifiers

NCT02141074

2012-004867-38 (EudraCT Number)

NN7999-3895

NL53683.091.15 (Other Identifier)

JapicCTI-142611 (Registry Identifier)

U1111-1135-9557 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 54 patients

Sex: Male

Ages: Under 6 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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54 participants in 1 patient group

50 EDs (exposure days)

Experimental group

Treatment:

Drug: nonacog beta pegol