A Study Investigating Treatment Factor X in People With Factor X Deficiency
Condition: Factor X Deficiency
MeSH terms: Factor X Deficiency
Intervention: Human Coagulation FACTOR X
Intervention description: 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.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 16.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No

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.
    

Eligibility criteria:

        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