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

Factor X Deficiency Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01721681
• Other study ID #: Ten 02
• Secondary ID: 2012-003093-98
• Status: Completed
• Phase: Phase 3
• First received: October 25, 2012
• Last updated: March 7, 2018
• Start date: April 2015
• Est. completion date: October 2016

Study information

• Verified date: March 2018
• Source: Bio Products Laboratory
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: October 2016
• Est. primary completion date: October 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 11 Years

Eligibility

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.

Related Conditions & MeSH terms

• Factor X Deficiency

Intervention

Biological:
• FACTOR X

Locations

Country	Name	City	State
United Kingdom	Addenbrookes Hospital	Cambridge
United Kingdom	Great Ormond Street Hospital	London
United Kingdom	Sheffield Children's Hospital	Sheffield

Sponsors (1)

Lead Sponsor	Collaborator
Bio Products Laboratory

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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