A Study Investigating Treatment Factor X in People With Factor X Deficiency

Factor X Deficiency Clinical Trial

Official title:

A Phase III Open, Multicentre Study to Investigate the Pharmacokinetics, Safety and Efficacy of BPL's High Purity Factor X in the Treatment of Severe and Moderate Factor X Deficiency.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00930176
• Other study ID #: Ten01
• Status: Completed
• Phase: Phase 3
• First received: June 10, 2009
• Last updated: December 8, 2014
• Start date: January 2010
• Est. completion date: November 2013

Study information

• Verified date: December 2014
• Source: Bio Products Laboratory
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial Summary

The main objective of the study is to assess the pharmacokinetics of FACTOR X after a single dose of 25IU/kg.

The secondary objectives of the study are to assess efficacy and safety of FACTOR X in the treatment of bleeding episodes over at least 6 months.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: November 2013
• Est. primary completion date: November 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Written informed consent given, or for subjects aged 12-17 years, have given written assent and whose parent/guardian has given written informed consent

• At least 12 years of age at dtae of written informed consent

• Have hereditary severe or moderate FX deficiency

• Currently treated with Fresh Frozen Plasma FFP, Prothrombin Complex Concentrate PCC or factor IX/X concentrate

• Must have a minimum of one spontaneous or menorrhagic bleed in the last 12 months which required treatment of FFP, PCC or factor IX/X concentrate. Newly diagnosed subjects who present at the hospital with a bleed may be included

• Must have had at least 7 days, and ideally 10-14 days, since an infusion of either FFP, PCC or factor IX/X concentrate at Baseline Visit

• Females of child bearing potential must have a negative result on a HCG based pregnancy test. If they are or become sexually active, they must practise contraception by using a method of proven reliability for the duration of the study

Exclusion Criteria:

• Have a history of inhibitor development to FX or a positive result at the Screening Visit

• Bleeding at the appointment for the PK assessment

• Subjects who have thrombocytopenia

• Have clinically significant liver disease

• Known to have other coagulopathy or thrombophilia

• Have known or suspected hypersensitivity to the investigational medicinal product or its excipients

• Have abused chemicals or drugs within the past 12 months

• Have a history of unreliability or non-cooperation

• Participating or have taken part in another trial within the last 30 days, with the exception of BPL FX surgery study - Protocol Ten03. In such cases, subjects should have completed their End of Study Visit either before or on the day of Screening Visit for this study

• Female subjects who are pregnant or lactating

• Subjects planning greater than 4 weeks absence from the locality of the Investigational site, between the screening visit and the repeat PK assessment

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Factor X Deficiency

Intervention

Biological:
• Human Coagulation FACTOR X
Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.

Locations

Country	Name	City	State
Germany	Dr Gunter Auerswald	Bremen
Spain	Dr. Bermejo	Caceres
Spain	Dr Maite Alvarez	Madrid
Turkey	Cukurova University Hospital	Balcali	Adana
Turkey	Ministry of Health Istanbul Goztepe Training & Research Hospital	Goztepe	Istanbul
Turkey	Istanbul University Cerrahpasa School of Medicine	Istanbul
Turkey	Kanuni Sultan Suleyman Training and Research Hospital	Istanbul
Turkey	Prof. Kavakli	Izmir
Turkey	Prof. Oner	Van
United Kingdom	Dr. Sue Pavord	Leicester
United Kingdom	Dr. Steve Austin	London
United States	Indiana Hemophilia & Thrombosis Center	Indianapolis	Indiana
United States	Dr. William Mitchell New York Blood Center, Weill Cornell Medical College	New York	New York
United States	UCSF School of Medicine	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Bio Products Laboratory

Countries where clinical trial is conducted

United States,  Germany,  Spain,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	FX:C Incremental Recovery	Incremental recovery is defined as the peak rise in plasma FX levels (IU/dL), as measured at 15, 30 and 60 minutes post-dose, divided by the dose (IU/kg).; Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment	At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose)	No
Primary	FX:C Half-life	Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment	At Baseline and at 6 months post-Baseline	No