Brief 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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_3

Timeline

Completed

Started Sep 2012

Longer than P75 for phase_3

Geographic Reach

18 countries

69 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

6.2 years study duration

First Submitted

Initial submission to the registry

December 15, 2011

Completed

1 day until next milestone

First Posted

Study publicly available on registry

December 16, 2011

Completed

9 months until next milestone

Study Start

First participant enrolled

September 17, 2012

Completed

4.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 16, 2017

Completed

1.2 years until next milestone

Results Posted

Study results publicly available

October 30, 2018

Completed

1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

December 5, 2018

Completed

Last Updated

November 30, 2020

Status Verified

November 1, 2020

Enrollment Period

4.9 years

First QC Date

December 15, 2011

Results QC Date

August 14, 2018

Last Update Submit

November 26, 2020

Conditions

Congenital Bleeding Disorder Haemophilia A

Outcome Measures

Primary Outcomes (1)

Incidence Rate of Factor VIII Inhibitors (Above or Equal to 0.6 BU (Bethesda Units)/mL) for the Main Phase of the Trial
The incidence rate (percentage of participants with inhibitors) of inhibitors defined as inhibitor titres ≥0.6 BU for main phase of the trial.
From Visit 2 (21 days after screening) to Visit 5 (50-55 exposure day)

Secondary Outcomes (13)

Haemostatic Effect of Turoctocog Alfa on Treatment of Bleeds Assessed on a Predefined Four Point Scale: Excellent, Good, Moderate and None
From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Annualised Bleeding Rate (ABR)
From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Number of Turoctocog Alfa (N8) Injections Required Per Bleed
From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Month
From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Year
From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
+8 more secondary outcomes

Study Arms (1)

turoctocog alfa

EXPERIMENTAL

Drug: turoctocog alfa

Interventions

turoctocog alfaDRUG

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.

turoctocog alfa

Eligibility Criteria

Age0 Years - 6 Years

Sexmale

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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®

You may not qualify if:

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)

Contact the study team to confirm eligibility.