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

Study Overview
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Brief Summary
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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.

Official Title
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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.

Conditions
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Factor X Deficiency

Intervention / Treatment
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* Biological: Human Coagulation FACTOR X


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Human Coagulation FACTOR X<br> | Biological: Human Coagulation FACTOR X<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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) | 
| 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 |