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.

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 Feb 2013

Longer than P75 for phase_3

Geographic Reach

16 countries

51 active sites

Status

completed

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

Trial Relationships

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Study Timeline

Key milestones and dates

5.6 years study duration

First Submitted

Initial submission to the registry

November 9, 2012

Completed

13 days until next milestone

First Posted

Study publicly available on registry

November 22, 2012

Completed

3 months until next milestone

Study Start

First participant enrolled

February 20, 2013

Completed

1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 15, 2014

Completed

4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 28, 2018

Completed

1.1 years until next milestone

Results Posted

Study results publicly available

October 30, 2019

Completed

Last Updated

November 23, 2020

Status Verified

November 1, 2020

Enrollment Period

1.6 years

First QC Date

November 9, 2012

Results QC Date

September 19, 2019

Last Update Submit

November 3, 2020

Conditions

Congenital Bleeding Disorder Haemophilia A

Outcome Measures

Primary Outcomes (1)

Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units
The number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) ≥0.6 Bethesda units was presented.
During the main phase of the trial (from 0-26 weeks of treatment)

Secondary Outcomes (16)

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)
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)
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)
+11 more secondary outcomes

Study Arms (1)

N8-GP

EXPERIMENTAL

Drug: turoctocog alfa pegol

Interventions

turoctocog alfa pegolDRUG

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.

Also known as: NNC 0129-0000-1003, N8-GP

N8-GP

Eligibility Criteria

Age0 Years - 11 Years

Sexmale

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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

You may not qualify if:

\- Any history of FVIII inhibitors

Contact the study team to confirm eligibility.