Phase 3 N=34 Treatment

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

Congenital Bleeding Disorder · Haemophilia B

Bottom Line

View on ClinicalTrials.gov: NCT02141074 ↗

Enrolled (actual)

Serious AEs

32.9%

Results posted

Dec 2023

Primary outcome: Primary: Number of Participants With Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) (50 Exposure Days) — 2; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: nonacog beta pegol (Drug)
Age: Pediatric · 0+ yrs
Sex: Male
Sponsor: Novo Nordisk A/S
Primary completion: Oct 2022

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) (50 Exposure Days)	2; 0	—
PRIMARY Number of Participants With Incidence of Inhibitory Antibodies Against FIX (100 ED)	2; 2	—
PRIMARY Number of Participants With Incidence of Inhibitory Antibodies Against FIX (At End of Trial)	2; 2	—
SECONDARY Number of Adverse Events	86; 291; 131; 610; 134; 794	—
SECONDARY Frequency of Adverse Events	5.59; 5.87; 5.92; 5.08; 5.52; 4.10	—
SECONDARY Number of Serious Adverse Events	9; 14; 13; 27; 14; 30	—
SECONDARY Frequency of Serious Adverse Events	0.59; 0.28; 0.59; 0.22; 0.58; 0.15	—
SECONDARY Number of Medical Events of Special Interest	5; 9; 6; 31; 6; 38	—
SECONDARY Frequency of Medical Events of Special Interest	0.33; 0.18; 0.27; 0.26; 0.25; 0.20	—
SECONDARY Number of Breakthrough Bleeding Episodes During Prophylaxis (Annualised Bleeding Rate)	0.00; 0.25; 0.33	—
SECONDARY Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")	23; 9; 8; 5; 2; 1	—
SECONDARY Incremental Recovery at 30 Minutes (IR30min)	0.012; 0.014	—
SECONDARY FIX Activity at 30 Minutes (C30min)	0.652; 0.824	—
SECONDARY FIX Trough Levels	0.150; 0.156; 0.167	—
SECONDARY Amount of Drug Administered to Treat a Bleeding Episode	55.0; 48.8; 47.7; 58.5; 47.5; 54.0	—
SECONDARY Number of Injections Needed to Treat a Bleeding Episode	1.3; 1.0; 1.1; 1.3; 1.1; 1.2	—

Summary

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.

Eligibility 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02141074). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.