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 12 months.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
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 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 ] [ Designated as safety issue: No ]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.
- Area Under Curve (AUC) [ Time Frame: predose, 30 mins, 4 hours, 24 hours, 48 hours and 72 hours ] [ Designated as safety issue: Yes ]For children equal to or greater than 1 year old.
- Area Under Curve (AUC) [ Time Frame: At two post-dose timepoints : 4 hours and 72 hours, or 30 mins and 48 hours, or 4 hours and 24 hours or 30 mins and 72 hours ] [ Designated as safety issue: Yes ]For children < 1 year old for for children equal to or > 1 year old whose bodyweight is less than 10.5kg
- Safety Outcomes [ Time Frame: Up to 28 days days after the final dose of FACTOR X. ] [ Designated as safety issue: Yes ]
Adverse events, haematology, serum biochemistry, PT and APTT, viral serology, FX inhibitor screens, vital signs, physical examination, infusion site observations, genotype analysis (optional) and number of exposure days.
Archive samples will be collected before first dose of FACTOR X and at the End-of-Study Visit.
|Study Start Date:||February 2013|
|Estimated Study Completion Date:||May 2015|
|Estimated Primary Completion Date:||May 2015 (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 12 months (52 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|
|Contact: Miranda Norton, Dr||+44 (0)208 957 firstname.lastname@example.org|
|Contact: Kate Gillanders||+44 (0)208 957 email@example.com|
|Great Ormond Street Hospital||Not yet recruiting|
|London, United Kingdom, WC1N 3JH|
|Principal Investigator:||Ri Liesner, Dr||Great Ormond Street Hospital|