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

• Register: ClinicalTrials.gov
• Last refreshed on: 8 April 2024
• Main ID: NCT02141074
• Date of registration: 28/03/2014
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B paradigm™6
• Scientific title: 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)
• Date of first enrolment: July 2, 2014
• Target sample size: 54
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02141074
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Algeria, Argentina, Australia, Austria, Canada, France, Germany, Israel, Italy, Japan, Malaysia, Netherlands, Spain, Taiwan, Thailand, United Kingdom, United States

Contacts

• Name: Global Clinical Registry (GCR, 1452)
• Affiliation: Novo Nordisk A/S

Key inclusion & exclusion 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



Age minimum: 0 Years
Age maximum: 6 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Congenital Bleeding Disorder

Haemophilia B

Intervention(s)

Drug: nonacog beta pegol

Primary Outcome(s)

Number of Participants With Incidence of Inhibitory Antibodies Against FIX (100 ED) [Time Frame: When minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)]

Number of Participants With Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) (50 Exposure Days) [Time Frame: 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 (At End of Trial) [Time Frame: At end of trial (up to 434 weeks)]

Secondary Outcome(s)

Incremental Recovery at 30 Minutes (IR30min) [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)]

Amount of Drug Administered to Treat a Bleeding Episode [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

FIX Activity at 30 Minutes (C30min) [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)]

Number of Injections Needed to Treat a Bleeding Episode [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

Frequency of Serious Adverse Events [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

FIX Trough Levels [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

Number of Medical Events of Special Interest [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

Number of Serious Adverse Events [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

Frequency of Adverse Events [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor") [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

Number of Adverse Events [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

Frequency of Medical Events of Special Interest [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

Number of Breakthrough Bleeding Episodes During Prophylaxis (Annualised Bleeding Rate) [Time Frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)]

Secondary ID(s)

JapicCTI-142611

NN7999-3895

U1111-1135-9557

NL53683.091.15

2012-004867-38

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 29/12/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02141074