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

This study has been terminated.
(This study was closed early due to difficulties in enrolling the target number of 10 surgical procedures within a reasonable timeframe.)
Sponsor:
Information provided by (Responsible Party):
Bio Products Laboratory
ClinicalTrials.gov Identifier:
NCT01086852
First received: November 10, 2009
Last updated: January 17, 2014
Last verified: January 2014
  Purpose

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.

Condition Intervention Phase
Factor X Deficiency
Biological: FACTOR X
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Bio Products Laboratory:

Primary Outcome Measures:
  • The primary efficacy variable is the presence or absence of excessive blood loss during surgery. [ Time Frame: After surgery for each subject. ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • A subjective overall assessment by the investigator of FACTOR X in the control of bleeding due to surgery throughout the whole study. [ Time Frame: This will assessed througout the study until discharge of the patient. ] [ Designated as safety issue: Yes ]

Enrollment: 7
Study Start Date: March 2011
Study Completion Date: January 2014
Primary Completion Date: January 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: FACTOR X
Human Coagulation Factor X
Biological: 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.

Other Name: FACTOR X

Detailed 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.

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01086852

Locations
United States, Texas
University Of Texas Health Science Center, Gulf States Hemophilia and Thrombophilia Center 6655 Travis St
Houston, Texas, United States, 77030
Spain
Unidad Coagulopatías, Congenitas, Edificio Dotacional, 1ra Planta Hospital Universito La Paz
Madrid, Spain, 28046
Turkey
Ege University School of Medicine, Departmant of Pediatric Hematology
Bornova, Izmir, Turkey, 35100
Istanbul University Cerrahpasa Medicine Faculty Department of Pediatric Hematology
Istanbul, Turkey, 34098
United Kingdom
Department of Hematology, Royal Cornwall Hospital,
Truro, Cornwall, United Kingdom, TR1 3LJ
The Katherine Dormandy Haemophilia Centre and Thrombosis Unit, The Royal Free Hospital,Pond Street
Hampstead, London, United Kingdom, NW3 2QG
Hammersmith Hospital
London, United Kingdom, W12 0NN
Sponsors and Collaborators
Bio Products Laboratory
Investigators
Principal Investigator: Tim Aldwinckle Bio Products Laboratory
  More Information

No publications provided

Responsible Party: Bio Products Laboratory
ClinicalTrials.gov Identifier: NCT01086852     History of Changes
Other Study ID Numbers: Ten03
Study First Received: November 10, 2009
Last Updated: January 17, 2014
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Bio Products Laboratory:
Factor X deficiency
surgery
Factor X deficiency subjects requiring surgery

Additional relevant MeSH terms:
Factor X Deficiency
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on October 20, 2014