Safety and Efficacy of nonacog beta pegol (N9-GP) in Previously Untreated Patients with Haemophilia B

Phase 1

• Conditions: Haemophilia B; MedDRA version: 20.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2012-004867-38-DE
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-06-03
• Last Update Posted: 9 January 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 50

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 < 6 years of age at the time of signing informed consent
- Patients with the diagnosis of haemophilia B (FIX activity level = 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 exposure days to blood components are acceptable)
Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 patient’s safety or compliance with the protocol
- Patient’s parent(s)/LAR(s) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Secondary Objective: - To evaluate safety of N9-GP (nonacog beta pegol)<br>- To evaluate efficacy of N9-GP (nonacog beta pegol)<br> o in long-term prophylaxis treatment<br> o in the treatment of bleeding episodes<br> o through the surrogate marker: FIX activity<br> o through monitoring of number of doses and consumption of N9-GP;Main Objective: To evaluate immunogenicity of N9-GP (nonacog beta pegol);Primary end point(s): Incidence of inhibitory antibodies against FIX;Timepoint(s) of evaluation of this end point: All objectives/endpoints will be evaluated when minimum 20 PUPs have<br>reached at least 50 exposure days (EDs), when minimum 40 PUPs have<br>reached at least 100 EDs, and at end of trial.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Number and frequency of adverse events, serious adverse events, and Medical Events of Special interest<br>- Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) <br>- Haemostatic effect by 4-point haemostatic response scale (excellent”, good”, moderate” and poor”);Timepoint(s) of evaluation of this end point: All objectives/endpoints will be evaluated when minimum 20 PUPs have<br>reached at least 50 exposure days (EDs), when minimum 40 PUPs have<br>reached at least 100 EDs, and at end of trial.