Clinical Trials List
Protocol NumberNN7999-3895
NCT Number(ClinicalTrials.gov Identfier)NCT02141074
2014-04-30 - 2023-04-16
Phase III
Recruiting2
ICD-10D67
Hereditary factor IX deficiency
ICD-9286.1
Congenital factor IX disorder
Safety and Efficacy of nonacog beta pegol (N9-GP) in Previously Untreated Patients with Haemophilia B
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Trial Applicant
NOVO NORDISK PHARMA (TAIWAN) LTD.
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Sponsor
Novo Nordisk A/S
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
- Pei-Chin Lin Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- MENG-YAO LU Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Haemophilia B
Objectives
Primary objective:
To evaluate immunogenicity of N9-GP (nonacog beta pegol)
Secondary objectives:
To evaluate safety of N9-GP (nonacog beta pegol)
To evaluate efficacy of N9-GP (nonacog beta pegol)
-in long-term prophylaxis treatment
-in the treatment of bleeding episodes
-through the surrogate marker: FIX activity
-through monitoring of number of doses and consumption of N9-GP
Test Drug
N9-GP
Active Ingredient
Glycopegylated recombinant coagulation factor IX
Dosage Form
Injection
Dosage
2000U/vial, 500U/vial
Endpoints
1. Key Primary endpoint(s):
Incidence of inhibitory antibodies against FIX .
2. Key Secondary endpoints
Number and frequency of adverse events, serious adverse events, and Medical Events of
Special Interest
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate)
Haemostatic effect by 4-point haemostatic response scale (“excellent”, “good”, “moderate” and “poor”)
Incidence of inhibitory antibodies against FIX .
2. Key Secondary endpoints
Number and frequency of adverse events, serious adverse events, and Medical Events of
Special Interest
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate)
Haemostatic effect by 4-point haemostatic response scale (“excellent”, “good”, “moderate” and “poor”)
Inclution Criteria
Inclusion criteria
1. 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. (Note: By consenting to this trial, the parent(s)/LAR(s) accepts that genotype of
the patient’s haemophilia B mutation is analysed/recorded and used for trial purposes).
2. Male, < 6 years of age at the time of signing informed consent
3. Patients with the diagnosis of haemophilia B (FIX activity level ≤ 2%) based on medical
records or central laboratory results
4. 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)
1. 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. (Note: By consenting to this trial, the parent(s)/LAR(s) accepts that genotype of
the patient’s haemophilia B mutation is analysed/recorded and used for trial purposes).
2. Male, < 6 years of age at the time of signing informed consent
3. Patients with the diagnosis of haemophilia B (FIX activity level ≤ 2%) based on medical
records or central laboratory results
4. 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)
Exclusion Criteria
Exclusion criteria
1. Any history of FIX inhibitors (defined by medical records)
2. Known or suspected hypersensitivity to trial product or related products
3. Previous participation in this trial. Participation is defined as first dose administered of trial
product
4. Receipt of any investigational medicinal product within 30 days before screening
5. Congenital or acquired coagulation disorder other than haemophilia B
6. Any chronic disorder or severe disease which, in the opinion of the Investigator, might
jeopardise patient’s safety or compliance with the protocol
7. Patient’s parent(s)/LAR(s) mental incapacity, unwillingness to cooperate, or a language barrier
precluding adequate understanding and cooperation
1. Any history of FIX inhibitors (defined by medical records)
2. Known or suspected hypersensitivity to trial product or related products
3. Previous participation in this trial. Participation is defined as first dose administered of trial
product
4. Receipt of any investigational medicinal product within 30 days before screening
5. Congenital or acquired coagulation disorder other than haemophilia B
6. Any chronic disorder or severe disease which, in the opinion of the Investigator, might
jeopardise patient’s safety or compliance with the protocol
7. Patient’s parent(s)/LAR(s) mental incapacity, unwillingness to cooperate, or a language barrier
precluding adequate understanding and cooperation
The Estimated Number of Participants
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Taiwan
8 participants
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Global
60 participants
