Overview

This trial is active, not recruiting.

Conditions congenital bleeding disorder, haemophilia a
Treatment turoctocog alfa
Phase phase 3
Sponsor Novo Nordisk A/S
Start date September 2012
End date June 2018
Trial size 60 participants
Trial identifier NCT01493778, 2011-001033-16, CTR20150455, JapicCTI-142544, NN7008-3809, P/50/2010, U1111-1119-6116

Summary

This trial is conducted in Asia, Europe and North America. The purpose of the trial is to evaluate the safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with haemophilia A.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
turoctocog alfa
Patients will be scheduled to receive treatment with turoctocog alfa for at least 100 exposure days. In most cases, treatment will be given at home with intravenous (i.v., into the vein) self-injection by the parent/caregiver/support person.

Primary Outcomes

Measure
Incidence rate of Factor VIII inhibitors (above or equal to 0.6 BU (Bethesda Units)/mL)
time frame: From Visit 2 to Visit 5, main phase of the trial (exposure day 50-55, expected to occur between 3 and 24 months of trial participation)

Secondary Outcomes

Measure
Haemostatic effect of turoctocog alfa on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None
time frame: From Visit 2 to Visit 5, main phase of the trial (exposure day 50-55, expected to occur between 3 and 24 months of trial participation)
Haemostatic effect of turoctocog alfa on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None
time frame: From Visit 6 to end of trial, extension phase of the trial (exposure day 100, expected to occur between 6 and 48 months of trial participation)
Haemostatic effect of turoctocog alfa on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None
time frame: From Visit 2 to end of trial, the combined main and extension phases of the trial (exposure day 100, expected to occur between 6 and 48 months)
Annualized bleeding rate
time frame: From Visit 2 to Visit 5, main phase of the trial (exposure day 50-55, expected to occur between 3 and 24 months of trial participation)
Annualized bleeding rate
time frame: From Visit 6 to end of trial, the extension phase of the trial (exposure day 100, expected to occur between 6 and 48 months of trial participation)
Annualized bleeding rate
time frame: From Visit 2 to end of trial, the combined main and extension phases of the trial (exposure day 100, expected to occur between 6 and 48 months of trial participation

Eligibility Criteria

Male participants up to 6 years old.

Inclusion Criteria: - Age below 6 years - Informed consent obtained before any trial-related activities (trial-related activities are any procedure that would not have been performed during normal management of the patient) - Male patients diagnosed with congenital severe haemophilia A (FVIII level equal to or below 1%) - No prior use of purified clotting factor products (previous exposure, equal to or less than 5 ED to blood components, e.g. cryoprecipitate, fresh frozen plasma, is accepted) including commercially available NovoEight® /Novoeight® Exclusion Criteria: - Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products - Previous participation in this trial defined as withdrawal after administration of trial product - Congenital or acquired coagulation disorders other than haemophilia A - Any history of Factor VIII inhibitor - Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG), routine systemic corticosteroids)

Additional Information

Official title Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients With Haemophilia A
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Novo Nordisk A/S.