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

Phase 1

• Conditions: Haemophilia B; MedDRA version: 19.0Level: 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-NL
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-07-23
• Last Update Posted: 5 January 2021

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, < 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
Main Objective: To evaluate immunogenicity of N9-GP (nonacog beta pegol);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;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 the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial. End of trial will be up to 4 years after the patient has reached 100 EDs.

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 the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial. End of trial will be up to 4 years after the patient has reached 100 EDs.