A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children <12 Years
|ClinicalTrials.gov Identifier: NCT01721681|
Recruitment Status : Completed
First Posted : November 6, 2012
Results First Posted : March 7, 2018
Last Update Posted : April 2, 2018
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).
|Condition or disease||Intervention/treatment||Phase|
|Factor X Deficiency||Biological: FACTOR X||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||April 2015|
|Actual Primary Completion Date :||October 2016|
|Actual Study Completion Date :||October 2016|
U.S. FDA Resources
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|
- The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months [ Time Frame: 6 months ]
The Investigator's assessment of the efficacy of FACTOR X in reduction/prevention of bleeding when given as routine prophylaxis over 6 months.
The efficacy was assessed according to tabulated criteria; Excellent, good, poor, unassessable.
- Safety of FACTOR X: Number of Participants Experiencing Adverse Events [ Time Frame: 6 months ]One of the secondary objectives was to assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks). The general strategy of the safety evaluation was to examine the summaries for any trends. No formal hypothesis was carried out. The number of participants who experienced Adverse Events is provided.
- Pharmacokinetics: FX:C Incremental Recovery [ Time Frame: Baseline Visit and End of Study Visit, 30 minutes post-dose ]One of the secondary objectives was to assess the pharmacokinetics (FX:C incremental recovery 30 minute post-dose at the Visit 1 (Baseline) and the End of Study Visit after a single dose of 50 IU/kg). The overall mean IR calculated for both visits is presented in the outcome measure table.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|