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:
- To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg.
- 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 ]
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 ]
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