A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A

Novo Nordisk A/S1 site in 1 country68 target enrollmentFebruary 20, 2013

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: 68
Locations: 1
Primary Endpoint: Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units
Status: Completed
Last Updated: 5 years 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 safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products.

Registry

clinicaltrials.gov

Start Date

February 20, 2013

End Date

September 28, 2018

Last Updated

5 years ago

Study Type

Interventional

Study Design

Single Group

Sex

Male

Investigators

Sponsor

Novo Nordisk A/S

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Male patients with severe congenital haemophilia A (FVIII activity level below 1%)
•Weight above or equal to 10 kg - Documented history of 150 exposure days (ED) to FVIII products for patients aged 6-11 years and above 50 ED to FVIII products for patients aged 0-5 years

Exclusion Criteria

•Any history of FVIII inhibitors

Arms & Interventions

N8-GP

Intervention: turoctocog alfa pegol

Outcomes

Primary Outcomes

Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units

Time Frame: During the main phase of the trial (from 0-26 weeks of treatment)

The number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) ≥0.6 Bethesda units was presented.

Secondary Outcomes

Consumption of N8-GP During Prophylaxis (Number of Injections)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for N8-GP(From 1 hour prior to and up to 96 hours after initial administration of N8-GP)
Clearance Evaluated for N8-GP(From 1 hour prior to and up to 96 hours after initial administration of N8-GP.)
Number of Bleeding Episodes During Prophylactic Treatment With N8-GP (Annualised Bleeding Rate)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
Consumption of N8-GP Per Bleeding Episode (Number of Injections)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
Area Under the Curve Evaluated for N8-GP(From 1 hour prior to and up to 96 hours after initial administration of N8-GP)
Terminal Half-life Evaluated for N8-GP(From 1 hour prior to and up to 96 hours after initial administration of N8-GP)
Frequency of Adverse Events Including Serious Adverse Events Reported During the Trial Period(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes and Assessed as: Excellent, Good, Moderate, or None(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
Consumption of N8-GP Per Bleeding Episode (U/kg)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
Terminal Half-life Evaluated for Previous FVIII Product(2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product)
Consumption of N8-GP During Prophylaxis (U/kg Per Month)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
Consumption of N8-GP During Prophylaxis (U/kg Per Year)(Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years))
Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for Previous FVIII Product(2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product)
Area Under the Curve Evaluated for Previous FVIII Product(2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product)
Clearance Evaluated for Previous FVIII Product(2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product)