Safety & Efficacy of BPL's High Purity FACTOR X in Treatment of Factor X Deficient Subjects Undergoing Surgery (Ten03)

Bio Products Laboratory

Status and phase

Terminated

Phase 3

Conditions

Factor X Deficiency

Treatments

Biological: FACTOR X

Study type

Interventional

Funder types

Other

Identifiers

NCT01086852

Ten03

Details and patient eligibility

About

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:

A subjective overall assessment by the investigator of FACTOR X in the control of bleeding during surgery.
The incidence of bleeding episodes during treatment with FACTOR X while the subject is at risk of post-operative bleeding, including location and duration.
Incremental recovery of FX:C and FX:Ag after the pre-surgery bolus infusion.
Assessment of FX:C and FX:Ag levels on each day post-surgery.
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.
Assessment of the cumulative doses of FACTOR X as measured by FX:C (IU) administered to each subject to maintain haemostasis.
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.

Full description

To investigate the safety and efficacy of FACTOR X administered by bolus infusion to prevent bleeding and achieve haemostasis in factor X deficient subjects undergoing surgery.

Enrollment

4 patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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.