Clinical trial

An Open-label Single-arm Multicentre Non-controlled Phase 3 a Trial Investigating Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Patients With Haemophilia B (FIX Activity Below or Equal to 2 Percent)

Name

NN7999-3895

Description

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.

Trial arms

Trial start

2014-07-02

Estimated PCD

2022-10-27

Trial end

2022-10-27

Status

Completed

Phase

Early phase I

Treatment

nonacog beta pegol

For intravenous (i.v.) injection. A single dose of 40 U/kg, unless the bleeding episode is severe in which case it should be treated with 80 U/kg.

Arms:

50 EDs (exposure days)

Size

Primary endpoint

Number of Participants With Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) (50 Exposure Days)

When minimum 20 previously untreated patients (PUPs) have reached at least 50 exposure days (ED) (up to 156 weeks)

Number of Participants With Incidence of Inhibitory Antibodies Against FIX (100 ED)

When minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)

Number of Participants With Incidence of Inhibitory Antibodies Against FIX (At End of Trial)

At end of trial (up to 434 weeks)

Eligibility criteria

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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 54, 'type': 'ACTUAL'}}

Updated at

2024-03-26

1 organization

2 products

2 indications

Organization

Novo Nordisk

Product

Nonacog beta pegol

Indication

Congenital Bleeding Disorder

Indication

Hemophilia B

Product

Nonacog Beta Pegol