NCT01480180|CompletedPhase 3Results

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A

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A Multi-national Trial Evaluating Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Patients With Haemophilia A

Novo Nordisk A/S ClinicalTrials.gov

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4 other identifiers

NN7088-3859U1111-1119-74162011-001142-15JapicCTI-121749

Study Type

interventional

Target

186

Locations

24 countries

Sites

Timeline

RegisteredNov 2011

StartedJan 2012

CompletionDec 2018

Brief Summary

This trial is conducted globally. The aim of the trial is to evaluate the safety and efficacy, including pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in subjects with Haemophilia A.

Trial Health

On Track

Trial Health Score

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

Enrollment

186

participants targeted

Target at P25-P50 for phase_3

Timeline

Completed

Started Jan 2012

Longer than P75 for phase_3

Geographic Reach

24 countries

93 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

6.9 years study duration

First Submitted

Initial submission to the registry

November 23, 2011

Completed

5 days until next milestone

First Posted

Study publicly available on registry

November 28, 2011

Completed

2 months until next milestone

Study Start

First participant enrolled

January 30, 2012

Completed

6.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 10, 2018

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 10, 2018

Completed

1.4 years until next milestone

Results Posted

Study results publicly available

April 20, 2020

Completed

Last Updated

November 23, 2020

Status Verified

November 1, 2020

Enrollment Period

6.9 years

First QC Date

November 23, 2011

Results QC Date

December 10, 2019

Last Update Submit

November 3, 2020

Conditions

Congenital Bleeding Disorder Haemophilia A

Outcome Measures

Primary Outcomes (6)

The Incidence Rate of FVIII-inhibitors ≥0.6 BU: After Approximately 19 Months
All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as ≥0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors.
After approximately 19 months
Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 19 Months
Annualised bleeding rate (ABR) is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP.
After approximately 19 months
The Incidence Rate of FVIII-inhibitors ≥0.6 BU: After Approximately 25 Months
All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as ≥0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors.
After approximately 25 months
Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 25 Months
ABR is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP.
After approximately 25 months
Incidence Rate of FVIII-inhibitors ≥0.6 BU: At Approximately 80 Months
All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as ≥0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors.
At approximately 80 months
Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 80 Months
Annualised bleeding rate (ABR) is the number of bleeding episodes per year reported during the prophylactic treatment with N8-GP.
After approximately 80 months

Secondary Outcomes (69)

Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 19 Months
After approximately 19 months
Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 25 Months
After approximately 25 months
Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 80 Months
After approximately 80 months
Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 19 Months
After approximately 19 months
Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 25 Months
After approximately 25 months
+64 more secondary outcomes

Study Arms (2)

Prophylaxis

EXPERIMENTAL

Drug: turoctocog alfa pegol

On-demand

EXPERIMENTAL

Drug: turoctocog alfa pegol

Interventions

turoctocog alfa pegolDRUG

Administered i.v.

Also known as: NNC 0129-0000-1003

On-demandProphylaxis

Eligibility Criteria

Age12 Years+

Sexmale

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Contact the study team to confirm eligibility.