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 (pathfinder™5)

This study is currently recruiting participants.

Verified May 2013 by Novo Nordisk

Sponsor:

Information provided by (Responsible Party):

Novo Nordisk

ClinicalTrials.gov Identifier:

NCT01731600

First received: November 9, 2012

Last updated: May 17, 2013

Last verified: May 2013

History of Changes

Tracking Information

First Received Date ^ICMJE

November 9, 2012

Last Updated Date

May 17, 2013

Start Date ^ICMJE

February 2013

Estimated Primary Completion Date

August 2014 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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) ] [ Designated as safety issue: No ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT01731600 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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) ] [ Designated as safety issue: No ]
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) ] [ Designated as safety issue: No ]
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) ] [ Designated as safety issue: No ]
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) ] [ Designated as safety issue: No ]
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) ] [ Designated as safety issue: No ]
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) ] [ Designated as safety issue: No ]
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) ] [ Designated as safety issue: No ]
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. ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
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. ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
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. ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
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. ] [ Designated as safety issue: No ]
Clearance evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

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

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.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 3

Study Design ^ICMJE

Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Congenital Bleeding Disorder
Haemophilia A

Intervention ^ICMJE

Drug: NNC 0129-0000-1003

Fixed dose of N8-GP for intravenous injections (i.v.) twice weekly for prophylaxis. In addition, N8-GP 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.

Study Arm (s)

Experimental: N8-GP

Intervention: Drug: NNC 0129-0000-1003

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

May 2018

Estimated Primary Completion Date

August 2014 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Gender

Male

Ages

up to 11 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Novo Nordisk

clinicaltrials@novonordisk.com

Location Countries ^ICMJE

United States, Brazil, France, Germany, Greece, Israel, Italy, Japan, Lithuania, Malaysia, Switzerland, Turkey, Ukraine, United Kingdom

Administrative Information

NCT Number ^ICMJE

NCT01731600

Other Study ID Numbers ^ICMJE

NN7088-3885, U1111-1129-6009, 2012-001711-23

Has Data Monitoring Committee

Responsible Party

Novo Nordisk

Study Sponsor ^ICMJE

Novo Nordisk

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Study Director:

Marianne Fridberg

Novo Nordisk

Information Provided By

Novo Nordisk

Verification Date

May 2013

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

To Top