A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children <12 Years

Phase 3

Completed

• Conditions: Factor X Deficiency
• Interventions: Biological: FACTOR X

• Registration Number: NCT01721681
• Lead Sponsor: Bio Products Laboratory

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 Description

Not available

Study Timeline

• Study First Submitted: 2012-10-25
• Study First Submitted QC: 2012-11-02
• Study First Posted: 2012-11-06
• Study Start: 2015-04
• Primary Completion: 2016-10
• Study Completion: 2016-10
• Results First Submitted: 2017-10-11
• Results First Submitted QC: 2018-02-07
• Status Verified: 2018-03
• Results First Posted: 2018-03-07
• Last Update Submitted: 2018-03-07
• Last Update Posted: 2018-04-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
FACTOR X	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.

Primary Outcome Measures

Name	Time	Method
The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months	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.

Secondary Outcome Measures

Name	Time	Method
Safety of FACTOR X: Number of Participants Experiencing Adverse Events	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	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.

Trial Locations

Locations (3)

Great Ormond Street Hospital; 🇬🇧 London, United Kingdom

Addenbrookes Hospital; 🇬🇧 Cambridge, United Kingdom

Sheffield Children's Hospital; 🇬🇧 Sheffield, United Kingdom