A Study to Investigate BPL's Factor X in the Prophylaxis of Bleeding in Children <12 Years
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).
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
|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 of 12 Years|
- 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.
- 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 ]
- Factor X activity trough levels at all scheduled study visits and at all Bleed Assessment and Trough Measurement unscheduled visits. [ Time Frame: 6 months ]
- Factor X activity 30 minute post-dose incremental recovery at the Baseline Visit and the End of Study Visit [ Time Frame: 6 months ]
- Factor X activity incremental recovery and trough levels following any change in dose regimen required for clinical reasons/insufficient trough levels. [ Time Frame: 6 months ]
- Dose to treat a bleed (IU/kg factor X activity) (including initial dose for new bleeds and any repeated doses for ongoing bleeds), number of infusions to treat a bleed and dose per infusion; all analysed on a per-bleed and a per-subject basis. [ Time Frame: 6 months ]
- Total dose in IU/kg of factor X (factor X activity), total number of infusions and average dose per infusion for: prophylactic use, to treat a bleed, any additional preventative use, any surgical use and overall use; all analysed on a per subject basis. [ Time Frame: 6 months ]
- Average monthly dose in IU/kg of factor X (factor X activity), and average monthly number of infusions for: prophylactic use, to treat a bleed, any additional preventative use, any surgical use and overall use; all analysed on a per subject basis. [ Time Frame: 6 months ]
|Study Start Date:||April 2015|
|Study Completion Date:||October 2016|
|Primary Completion Date:||October 2016 (Final data collection date for primary outcome measure)|
Experimental: FACTOR X
At the Baseline Visit, eligible children will receive a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children will be treated with FACTOR X prophylactically for a period of 6 months (26 weeks).
A dosing regimen of 40-50 IU/kg twice a week is recommended, but is not mandatory. Each dose of FACTOR X must not exceed 60 IU/kg.
|Biological: FACTOR X|
Please refer to this study by its ClinicalTrials.gov identifier: NCT01721681
|Cambridge, United Kingdom, CB2 0QQ|
|Great Ormond Street Hospital|
|London, United Kingdom, WC1N 3JH|
|Sheffield Children's Hospital|
|Sheffield, United Kingdom, S10 2TH|
|Principal Investigator:||Ri Liesner, Dr||Great Ormond Street Hospital|