Safety & Efficacy of BPL's High Purity FACTOR X in Treatment of Factor X Deficient Subjects Undergoing Surgery
Condition: Factor X Deficiency
MeSH terms: Factor X Deficiency
Intervention: FACTOR X
Intervention description: Presurgery loading dose- The FX level of 70%-90% should be achieved.This will be calculated based on the patients weight on day of surgery and the required rise. Initial dose should not exceed 60IU/kg.
Post surgery- FX trough levels of 50% should be achieved.
Intravenous infusion of factor X is given at a suggested rate of 10mL/min but not exceeding more than 20mL/min.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 4.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No
Keywords: Factor X deficiency; surgery; Factor X deficiency subjects requiring surgery

Summary:

      To primary efficacy variable is to assess the presence or absence of excessive blood loss
      during and after surgery.

      The secondary efficacy endpoints are as follows:

        1. A subjective overall assessment by the investigator of FACTOR X in the control of
           bleeding during surgery.

        2. The incidence of bleeding episodes during treatment with FACTOR X while the subject is
           at risk of post-operative bleeding, including location and duration.

        3. Incremental recovery of FX:C and FX:Ag after the pre-surgery bolus infusion.

        4. Assessment of FX:C and FX:Ag levels on each day post-surgery.

        5. Assessment of the cumulative weight-adjusted doses of FACTOR X as measured by FX:C
           (IU/kg body weight) administered to each subject to maintain haemostasis.

        6. Assessment of the cumulative doses of FACTOR X as measured by FX:C (IU) administered to
           each subject to maintain haemostasis.

        7. Amount of weight-adjusted FACTOR X as measured by FX:C (IU/kg body weight) administered
           daily (day of surgery and each post-operative day) to maintain haemostasis.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Subjects who are at least 12 years of age at date of written informed consent/assent.

          -  Subjects who have given written informed consent or, for subjects aged 12-17 years
             (inclusive), have given written assent and whose parent/guardian has given written
             informed consent.

          -  Subjects with hereditary mild to severe Factor X deficiency (<20% basal FX activity),
             including previously untreated subjects OR those currently treated with Fresh Frozen
             Plasma (FFP), Prothrombin Complex Concentrate (PCC) or factor IX/X concentrate by
             prophylaxis or on demand.

          -  Subjects who are to undergo surgery in which the investigator believes a factor X
             concentrate will be required due to a prior history of unusual bleeding either
             spontaneously or after surgery or trauma in the absence of treatment with a factor X
             containing product.

          -  Pregnant subjects undergoing obstetric delivery (including Caesarean surgery and
             vaginal delivery) may enter the study. Female subjects of child-bearing potential must
             have a negative result on a human chorionic gonadotropin-based pregnancy test. If a
             female subject is or becomes sexually active, she must practice contraception by using
             a method of proven reliability for the duration of the study.

        Exclusion Criteria:

          -  Subjects who are required or expected to take other factor X containing medications
             during or after surgery.

          -  Subjects with a history of inhibitor development to FX or a detectable inhibitor to FX
             (≥0.6 BU) on the Nijmegen-Bethesda assay at screening. Obtaining a FX inhibitor result
             at screening is not mandatory if the subject is to undergo emergency surgery and the
             local laboratory is unable to perform the analyses prior to the surgical procedure.

          -  Subjects with thrombocytopenia (platelets < 50 x 109/L).

          -  Subjects who have clinically significant renal disease (creatinine >200µmol/L).

          -  Subjects who have clinically significant liver disease (ALT levels greater than three
             times the upper limit of normal).

          -  Subjects known to have other coagulopathy or thrombophilia.

          -  Subjects who are currently participating or have participated in another trial within
             the last 30 days, with the exception of the BPL Factor X PK study (protocol number
             Ten01).

          -  Female subjects who are lactating.

          -  Subjects who have known or suspected hypersensitivity to the investigational medicinal
             product or its excipients.

          -  Subjects known to have abused chemicals or drugs within the past 12 months.

          -  Subjects with a history of unreliability or non-cooperation.