Safety & Efficacy of BPL's High Purity FACTOR X in Treatment of Factor X Deficient Subjects Undergoing Surgery
| Status: | Terminated |
|---|---|
| Conditions: | Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | 12 - Any |
| Updated: | 2/7/2019 |
| Start Date: | March 2011 |
| End Date: | January 2014 |
A Phase III Open, Multicentre Study to Investigate the Safety and Efficacy of BPL's High Purity FACTOR X in the Treatment of Factor X Deficient Subjects Undergoing Surgery
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.
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.
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.
bleeding and achieve haemostasis in factor X deficient subjects undergoing surgery.
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.
We found this trial at
2
sites
Madrid, 28046
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Houston, Texas 77030
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