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

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 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).

Study Overview

• Status: Completed
• Conditions: Factor X Deficiency
• Intervention / Treatment: Biological: FACTOR X

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 3

Contacts and Locations

Study Locations

• United Kingdom
  • Cambridge, United Kingdom, CB2 0QQ
    • Addenbrookes Hospital
  • London, United Kingdom, WC1N 3JH
    • Great Ormond Street Hospital
  • Sheffield, United Kingdom, S10 2TH
    • Sheffield Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 11 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria

1. Children with hereditary severe or moderate FX deficiency (FX:C <5 IU/dL), based on their lowest reliable FX:C recorded.
2. Children under 12 years old, whose parent/guardian has given informed consent.
3. Children with a history of severe bleeding e.g.: intracranial haemorrhage, before starting prophylactic therapy, OR a mutation in the F10 gene causing a documented severe bleeding phenotype.

Exclusion Criteria

1. Children must not suffer from clinically significant liver disease, renal disease, or other coagulopathy or thrombophilia
2. Children must have no history or suspicion of inhibitors to factor X.
3. Children who have known or suspected hypersensitivity to the investigational medicinal product or its excipients.
4. Children with a history of unreliability or non-cooperation.
5. Children who are participating or have taken part in another trial within the last 30 days.
6. Children planning more than 4 weeks' continuous absence from the locality of the investigational site, between the Screening Visit and the End of Study Visit at approximately 6 months (26 weeks) post-Baseline.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 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.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of FACTOR X: Number of Participants Experiencing Adverse Events	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.	6 months
Pharmacokinetics: FX:C Incremental Recovery	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.	Baseline Visit and End of Study Visit, 30 minutes post-dose

Collaborators and Investigators

• Sponsor: Bio Products Laboratory
• Investigators: Principal Investigator: Ri Liesner, Dr, Great Ormond Street Hospital

Study record dates

Study Major Dates

• Study Start: April 1, 2015
• Primary Completion (Actual): October 1, 2016
• Study Completion (Actual): October 1, 2016

Study Registration Dates

• First Submitted: October 25, 2012
• First Submitted That Met QC Criteria: November 2, 2012
• First Posted (Estimate): November 6, 2012

Study Record Updates

• Last Update Posted (Actual): April 2, 2018
• Last Update Submitted That Met QC Criteria: March 7, 2018
• Last Verified: March 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Factor X Deficiency
• Other Study ID Numbers: Ten 02; 2012-003093-98 (EudraCT Number)