An Open-label Single-arm Multicentre Non-controlled Phase 3a Trial Investigating Safety and Efficacy of N8-GP in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Paediatric Patients With Severe Haemophilia A

Novo Nordisk A/S95 sites in 7 countries124 target enrollmentJune 26, 2014

ConditionsCongenital Bleeding Disorder Haemophilia A

Interventionsturoctocog alfa pegol

Drugsturoctocog alfa pegol

Overview

Phase: Phase 3
Intervention: turoctocog alfa pegol
Conditions: Congenital Bleeding Disorder
Sponsor: Novo Nordisk A/S
Enrollment: 124
Locations: 95
Primary Endpoint: Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII)
Status: Completed
Last Updated: 4 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.

Registry

clinicaltrials.gov

Start Date

June 26, 2014

End Date

June 7, 2023

Last Updated

4 months ago

Study Type

Interventional

Study Design

Single Group

Sex

Male

Investigators

Sponsor

Novo Nordisk A/S

Responsible Party

Sponsor

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 of age at the time of signing informed consent
•Diagnosis of severe haemophilia A (FVIII activity level 1%) based on medical records or central laboratory results
•No prior use of purified clotting factor products (5 previous exposures to blood components is acceptable)

Exclusion Criteria

•Any history of FVIII inhibitor (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 A
•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')/legally acceptable representative (LAR(s')) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation

Arms & Interventions

50 EDs (exposure days)

Intervention: turoctocog alfa pegol

Outcomes

Primary Outcomes

Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII)

Time Frame: From start of the treatment up to 7 years

Number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) was reported during the main and extension phase of the trial.

Secondary Outcomes

Number of Adverse Events Including Serious Adverse Events and Medical Events of Special Interest(From start of the treatment up to 8.9 years)
Number of Participants With Confirmed High Titre Inhibitors (Defined as Inhibitor Titre Above 5 Bethesda Units (BU)(From start of the treatment up to 8.9 years)
Number of Breakthrough Bleeding Episodes During Prophylaxis With N8-GP (Annualised Bleeding Rate)(From start of the treatment up to 8.9 years)
Haemostatic Effect of N8-GP in Treatment of Bleeding Episodes, Assessed by a Predefined 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "None")(From start of the treatment up to 8.9 years)
Consumption of N8-GP for Prophylaxis (International Unit Per Kilogram (IU/Kg))(From start of the treatment up to 8.9 years)
Consumption of N8-GP for Prophylaxis (Number of Injections)(From start of the treatment up to 8.9 years)
Consumption of N8-GP for Treatment of Bleeding Episodes (International Unit Per Kilogram Per Bleed (IU/kg/Bleed))(From start of the treatment up to 8.9 years)
Consumption of N8-GP for Treatment of Bleeding Episodes (Number of Injections)(From start of the treatment up to 8.9 years)
Total Consumption of N8-GP Per Patient (Prevention and Treatment of Bleeding Episodes) Annualised Value(From start of the treatment up to 8.9 years)
Outcome of ITI, Assessed by a Predefined 4-point ITI Outcome Scale ("Success", "Partial Success", "Failure", "Other")(From start of the treatment up to 8.9 years)