Vanderbilt Clinical Trials

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)		Drug: 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.

More Details

Status: Completed
Sponsor: Novo Nordisk A/S

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.