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

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ClinicalTrials.gov Identifier: NCT01086852
Recruitment Status : Terminated (Closed early due to difficulties in enrolling the target number of 10 surgical procedures.)
First Posted : March 15, 2010
Results First Posted : July 3, 2015
Last Update Posted : February 5, 2019
Sponsor:
Information provided by (Responsible Party):
Bio Products Laboratory

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Factor X Deficiency
Intervention Biological: FACTOR X
Enrollment 4
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Active Treatment With FACTOR X
Hide Arm/Group Description Four subjects underwent 4 major surgeries with active treatment (FACTOR X)
Period Title: Overall Study
Started 4
Completed 4
Not Completed 0
Arm/Group Title Active Treatment With FACTOR X
Hide Arm/Group Description Four patients underwent 4 major surgeries with active treatment (FACTOR X)
Overall Number of Baseline Participants 4
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
<=18 years
0
   0.0%
Between 18 and 65 years
4
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 4 participants
57
(55 to 59)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Female
0
   0.0%
Male
4
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
4
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
  50.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
2
  50.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 4 participants
United States 2
United Kingdom 2
FX:C at diagnosis (IU/dL)  
Median (Full Range)
Unit of measure:  IU/dL
Number Analyzed 4 participants
7.5
(7 to 8)
1.Primary Outcome
Title Clinical Estimation of Volume of Blood Loss During Surgery
Hide Description

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:

  • Blood loss less than expected
  • Blood loss as expected
  • Blood loss more than expected
  • Blood loss excessive (defined as more than twice the pre defined amount that would be expected in a normal patient for this type of surgery)
Time Frame Blood loss is measured during and after surgery, the overall assessment is made after the last dose of FACTOR X.
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population will be defined as all surgical procedures in which subjects were treated with at least one dose of FACTOR X and have undergone surgery.
Arm/Group Title FACTOR X
Hide Arm/Group Description:

Human Coagulation Factor X

FACTOR X: 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.

Overall Number of Participants Analyzed 4
Geometric Mean (Standard Deviation)
Unit of Measure: ml
160.5  (168.49)
2.Primary Outcome
Title Clinical Assessment of Blood Loss During Surgery Against the Volume of Blood Loss Expected in Patients Without a Bleeding Disorder.
Hide Description 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.
Time Frame After wound closure
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects treated with FACTOR X
Arm/Group Title Active Treatment With FACTOR X
Hide Arm/Group Description:
Four patients underwent 4 major surgeries with active treatment (FACTOR X)
Overall Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: participants
Less Than 1
More Than 0
Equal to 3
3.Primary Outcome
Title Requirement for Blood Transfusion
Hide Description 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
Time Frame during and after surgery
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population will be defined as all surgical procedures in which subjects were treated with at least one dose of FACTOR X and have undergone surgery.
Arm/Group Title Active Treatment With FACTOR X
Hide Arm/Group Description:
Four patients underwent 4 major surgeries with active treatment (FACTOR X)
Overall Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: number of transfusions
0
4.Primary Outcome
Title Number of Post Operative Bleeding Episodes (See Table Below)
Hide Description 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
Time Frame End of surgery till end of study
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population will be defined as all surgical procedures in which subjects were treated with at least one dose of FACTOR X and have undergone surgery.
Arm/Group Title Active Treatment With FACTOR X
Hide Arm/Group Description:
Four patients underwent 4 major surgeries with active treatment (FACTOR X)
Overall Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: number of bleeds
0
5.Primary Outcome
Title Change of Haemoglobin From Pre-surgery Till End of Treatment
Hide Description 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
Time Frame 2 hrs pre-operatively till end of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population will be defined as all surgical procedures in which subjects were treated with at least one dose of FACTOR X and have undergone surgery.
Arm/Group Title Active Treatment With FACTOR X
Hide Arm/Group Description:
Four patients underwent 4 major surgeries with active treatment (FACTOR X)
Overall Number of Participants Analyzed 4
Geometric Mean (Standard Deviation)
Unit of Measure: g/L
-36.0  (26.17)
6.Primary Outcome
Title Number of Participants With Degree of Bleeding Control Rated as Excellent.
Hide Description

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:

  • Excellent -Parameters were similar to those in subjects without a bleeding disorder.
  • Good -Parameters were inferior to those in subjects without a bleeding disorder, but no other factor X containing agents were required to restore haemostasis.
  • Poor - Blood loss was excessive (defined as more than twice the pre defined amount that would be expected in a subject without a bleeding disorder for this type of surgery) and/or Haemostasis was not achieved and/or Additional factor X containing agents were required to restore haemostasis.
  • Unassessable -Efficacy was not possible to assess, or Additional factor X containing agents (excluding blood transfusions) were required before efficacy of FACTOR X could be assessed.
Time Frame During and till end of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population will be defined as all surgical procedures in which subjects were treated with at least one dose of FACTOR X and have undergone surgery.
Arm/Group Title Active Treatment With FACTOR X
Hide Arm/Group Description:
Four patients underwent 4 major surgeries with active treatment (FACTOR X)
Overall Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: Participants
4
7.Secondary Outcome
Title Incremental Recovery After Bolus Dose of FACTOR X
Hide Description

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)

Time Frame incremental recovery was assessed at approximately 30 minutes after the pre surgery bolus
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population will be defined as all surgical procedures in which subjects were treated with at least one dose of FACTOR X and have undergone surgery.
Arm/Group Title FACTOR X
Hide Arm/Group Description:

Human Coagulation Factor X

FACTOR X: 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.

Overall Number of Participants Analyzed 4
Geometric Mean (Standard Deviation)
Unit of Measure: IU/dL per IU/kg
2.14  (0.224)
8.Secondary Outcome
Title Dose Per Infusion (IU/kg)
Hide Description weight adjusted dose per infusion until a subject was no longer at risk of bleeding due to surgery
Time Frame before surgery, during the post operative period
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT population will be defined as all surgical procedures in which subjects were treated with at least one dose of FACTOR X and have undergone surgery.
Arm/Group Title Active Treatment With FACTOR X
Hide Arm/Group Description:
Four patients underwent 4 major surgeries with active treatment (FACTOR X)
Overall Number of Participants Analyzed 4
Median (Full Range)
Unit of Measure: IU/kg
16.14
(10.13 to 22.30)
Time Frame Adverse events were documented from the date the Informed Consent Form was signed until the end of the subject's participation in the study
Adverse Event Reporting Description Treatment-emergent AEs are defined as AEs occurring or worsening after the start (date and time) of the first study treatment until End of Treatment Assessment or follow-up safety assessment (28 days after the last dose of FACTOR X) if performed
 
Arm/Group Title FACTOR X
Hide Arm/Group Description

Human Coagulation Factor X

FACTOR X: 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.

All-Cause Mortality
FACTOR X
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
FACTOR X
Affected / at Risk (%) # Events
Total   0/4 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
FACTOR X
Affected / at Risk (%) # Events
Total   4/4 (100.00%)    
Blood and lymphatic system disorders   
Anaemia  1  1/4 (25.00%)  1
Gastrointestinal disorders   
Constipation  1  3/4 (75.00%)  3
Dyspepsia  1  3/4 (75.00%)  3
Nausea  1  1/4 (25.00%)  1
General disorders   
Procedural pain  1  2/4 (50.00%)  2
Oedema peripheral  1  2/4 (50.00%)  2
Infections and infestations   
Herpes zoster  1  1/4 (25.00%)  1
Injury, poisoning and procedural complications   
Post procedural discomfort  1  1/4 (25.00%)  2
Contusion  1  1/4 (25.00%)  1
Incision site complication  1  1/4 (25.00%)  1
Investigations   
Haemoglobin decreased  1  1/4 (25.00%)  1
Metabolism and nutrition disorders   
Hyperglycaemia  1  1/4 (25.00%)  1
Hypokalaemia  1  1/4 (25.00%)  1
Hypomagnesaemia  1  1/4 (25.00%)  1
Psychiatric disorders   
Insomnia  1  1/4 (25.00%)  1
Respiratory, thoracic and mediastinal disorders   
Oropharyngeal pain  1  1/4 (25.00%)  1
Skin and subcutaneous tissue disorders   
Ecchymosis  1  1/4 (25.00%)  1
Pruritus  1  1/4 (25.00%)  1
Vascular disorders   
Haematoma  1  1/4 (25.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (17.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Miranda Norton
Organization: Bio Products Laboratory
Phone: 020 8 957 2661
Responsible Party: Bio Products Laboratory
ClinicalTrials.gov Identifier: NCT01086852     History of Changes
Other Study ID Numbers: Ten03
First Submitted: November 10, 2009
First Posted: March 15, 2010
Results First Submitted: January 12, 2015
Results First Posted: July 3, 2015
Last Update Posted: February 5, 2019