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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 ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01731600
First received: November 9, 2012
Last updated: June 26, 2014
Last verified: June 2014

November 9, 2012
June 26, 2014
February 2013
August 2014   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT01731600 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
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
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

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.

Not Provided
Interventional
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Congenital Bleeding Disorder
  • Haemophilia A
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.
Experimental: N8-GP
Intervention: Drug: NNC 0129-0000-1003
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
64
May 2018
August 2014   (final data collection date for primary outcome measure)

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
Male
up to 11 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Canada,   France,   Germany,   Greece,   Israel,   Italy,   Japan,   Lithuania,   Malaysia,   Portugal,   Puerto Rico,   Switzerland,   Turkey,   Ukraine,   United Kingdom
 
NCT01731600
NN7088-3885, U1111-1129-6009, 2012-001711-23, JapicCTI-132214
No
Novo Nordisk A/S
Novo Nordisk A/S
Not Provided
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
Novo Nordisk A/S
June 2014

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