Overview

This trial is active, not recruiting.

Conditions congenital bleeding disorder, haemophilia a
Treatment turoctocog alfa pegol
Phase phase 3
Sponsor Novo Nordisk A/S
Start date February 2013
End date May 2018
Trial size 68 participants
Trial identifier NCT01731600, 2012-001711-23, JapicCTI-132214, NN7088-3885, U1111-1129-6009

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
turoctocog alfa pegol NNC 0129-0000-1003
Fixed dose of turoctocog alfa pegol for intravenous injections (i.v.) twice weekly for prophylaxis. In addition, turoctocog alfa pegol will be administered to treat bleeding episodes during the trial period. Bleeding episodes will be treated with doses of 20-75 U/kg body weight.

Primary Outcomes

Measure
Incidence of inhibitory antibodies against coagulation factor VIII (FVIII) equal to or above 0.6 Bethesda units
time frame: During the main phase of the trial (from 0-26 weeks of treatment)

Secondary Outcomes

Measure
Frequency of adverse events including serious adverse events reported during the trial period
time frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial)
Haemostatic effect of N8-GP when used for treatment of bleeding episodes and assessed as: Excellent, Good, Moderate, or None
time frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial)
Number of bleeding episodes during prophylactic treatment with N8-GP (annualised bleeding rate)
time frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial)
Consumption of N8-GP per bleeding episode (number of injections)
time frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial)
Consumption of N8-GP per bleeding episode (U/kg)
time frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial)
Consumption of N8-GP during prophylaxis (number of injections)
time frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial)
Consumption of N8-GP during prophylaxis ( U/kg per month and year)
time frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial)
Incremental recovery (defined as the peak level recorded 60 min after end of injection) evaluated for FVIII product
time frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product.
Incremental recovery (defined as the peak level recorded 60 min after end of injection) evaluated for N8-GP
time frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
Area under the curve evaluated for FVIII product
time frame: 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 N8-GP
time frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
Terminal half-life evaluated for FVIII product
time frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product.
Terminal half-life evaluated for N8-GP
time frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
Clearance evaluated for FVIII product
time frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product.
Clearance evaluated for N8-GP
time frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP

Eligibility Criteria

Male participants up to 11 years old.

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

Additional Information

Official title 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
Trial information was received from ClinicalTrials.gov and was last updated in July 2016.
Information provided to ClinicalTrials.gov by Novo Nordisk A/S.