- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01086852
Safety & Efficacy of BPL's High Purity FACTOR X in Treatment of Factor X Deficient Subjects Undergoing Surgery (Ten03)
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:
- 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.
Study Overview
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
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Madrid, Spain, 28046
- Unidad Coagulopatías, Congenitas, Edificio Dotacional, 1ra Planta Hospital Universito La Paz
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Istanbul, Turkey, 34098
- Istanbul University Cerrahpasa Medicine Faculty Department of Pediatric Hematology
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Izmir
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Bornova, Izmir, Turkey, 35100
- Ege University School of Medicine, Departmant of Pediatric Hematology
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London, United Kingdom, W12 0NN
- Hammersmith Hospital
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Cornwall
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Truro, Cornwall, United Kingdom, TR1 3LJ
- Department of Hematology, Royal Cornwall Hospital,
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London
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Hampstead, London, United Kingdom, NW3 2QG
- The Katherine Dormandy Haemophilia Centre and Thrombosis Unit, The Royal Free Hospital,Pond Street
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Texas
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Houston, Texas, United States, 77030
- University Of Texas Health Science Center, Gulf States Hemophilia and Thrombophilia Center 6655 Travis St
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: FACTOR X
Human Coagulation Factor X
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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. |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Clinical Estimation of Volume of Blood Loss During Surgery
Time Frame: Blood loss is measured during and after surgery, the overall assessment is made after the last dose of FACTOR X.
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As soon as possible after wound closure, the investigator estimated the volume of blood loss during surgery and made a clinical assessment against the volume of blood loss typically expected in a normal patient (i.e. one without a bleeding disorder and undergoing the same surgical procedure). The assessment may have been supported by a swab and pad count. The clinical assessment was rated as follows:
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Blood loss is measured during and after surgery, the overall assessment is made after the last dose of FACTOR X.
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Clinical Assessment of Blood Loss During Surgery Against the Volume of Blood Loss Expected in Patients Without a Bleeding Disorder.
Time Frame: After wound closure
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The investigator's estimation of the volume of blood loss during surgery compared to the volume of blood loss expected in patients without a bleeding disorder undergoing the same surgical procedure and reported as greater than, equal to or less than.
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After wound closure
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Requirement for Blood Transfusion
Time Frame: during and after surgery
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Number of blood transfusions required (units of packed red blood cells or units of whole blood) or infusion of autologous red cells during and after surgery
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during and after surgery
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Number of Post Operative Bleeding Episodes (See Table Below)
Time Frame: End of surgery till end of study
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Bleeding was assessed at least once each day by the investigator, more frequently if indicated by the severity of the operation or the subject's response.
This included all bleeding episodes from the end of the surgical procedure until the subject was no longer at risk of bleeding due to surgery
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End of surgery till end of study
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Change of Haemoglobin From Pre-surgery Till End of Treatment
Time Frame: 2 hrs pre-operatively till end of treatment
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The subject's haemoglobin was measured pre operatively, within 2 hours post operatively and at the End of Treatment Assessment.
Changes in the subject's haemoglobin from pre to post operatively and from post operatively to the End of Treatment Assessment were assessed, taking into account the volume of fluid infused into the subject during the intervening periods, any blood transfusions in the intervening periods, the subject's haematocrit at the same time points and the subject's pre dose serum ferritin
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2 hrs pre-operatively till end of treatment
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Number of Participants With Degree of Bleeding Control Rated as Excellent.
Time Frame: During and till end of treatment
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Investigators made an overall assessment of FACTOR X in controlling bleeding at the End of Treatment Assessment. The degree of bleeding control was rated as excellent, good, poor or unassessable, in accordance with the following criteria listed below:
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During and till end of treatment
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Incremental Recovery After Bolus Dose of FACTOR X
Time Frame: incremental recovery was assessed at approximately 30 minutes after the pre surgery bolus
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Incremental Recovery of FX:C after the Pre surgery Bolus Infusion The factor X increment is calculated by subtracting the pre-infusion factor X level from the post-dose value. Incremental recovery is calculated by FX increment (IU/dL)/ FX dose (IU/kg) |
incremental recovery was assessed at approximately 30 minutes after the pre surgery bolus
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Dose Per Infusion (IU/kg)
Time Frame: before surgery, during the post operative period
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weight adjusted dose per infusion until a subject was no longer at risk of bleeding due to surgery
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before surgery, during the post operative period
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Tim Aldwinckle, Bio Products Laboratory
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- Ten03
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