Clinical Trial: A Study to Investigate BPL's Factor X in the Prophylaxis of Bleeding in Children <12 Years

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase III Open, Multicentre Study to Investigate the Safety, Pharmacokinetics and Efficacy of BPL's High Purity Factor X in the Prophylaxis of Bleeding in Factor X Deficient Children Under the Age o

Brief Summary:

The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months.

The secondary objectives of the study are:

  1. To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg.
  2. To assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks).

Detailed Summary:
Sponsor: Bio Products Laboratory

Current Primary Outcome: The investigator's assessment of the efficacy of FACTOR X in the reduction/prevention of bleeding when given as routine prophylaxis over 6 months. [ Time Frame: 6 months ]

This will take into account the child's risk of breakthrough bleeding (due to bleeding history, treatment history and genetic mutation) and other relevant factors eg compliance with the protocol, attainment of required factor X activity trough levels. Retrospective data will be included in the analysis as supportive data.


Original Primary Outcome: The primary efficacy variable is the investigator's assessment of the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months [ Time Frame: 1 year ]

The efficacy criteria will be 'excellent', 'good', 'poor' or 'unassessable' and will be based on the number of major and minor breakthrough bleeds, and the number of excessive bleeding episodes following injury.


Current Secondary Outcome:

  • Number of bleeds per month including severity, duration, location and cause [ Time Frame: 6 months ]
  • Investigator's and parent's/guardian's assessment of efficacy in treating a bleed for all bleeds (parent/guardian assessment) and for bleeds treated at hospital (Investigator's assessment). [ Time Frame: 6 months ]


Original Secondary Outcome: To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg [ Time Frame: 3 months after the baseline visit ]

For subjects in the intense PK assessment (> 1 year old) the following PK variables will be calculated: AUC(0-∞), AUC(0-72h), half-life (non-compartmental), MRT(0-∞), clearance and volume of distribution for FX:C.

For subjects in the sparse PK assessment (< 1 year old or > 1 year old whose body weight is less than 10.5kg)the following PK variables will be calculated, if possible, using combined data from all subjects: AUC(0-∞), AUC(0-72h), half-life (non-compartmental), MRT(0-∞), clearance and volume of distribution for FX:C.



Information By: Bio Products Laboratory

Dates:
Date Received: October 25, 2012
Date Started: April 2015
Date Completion:
Last Updated: November 7, 2016
Last Verified: November 2016