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A Study Investigating Treatment Factor X in People With Factor X Deficiency

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00930176
Recruitment Status : Completed
First Posted : June 30, 2009
Results First Posted : December 12, 2014
Last Update Posted : December 12, 2014
Information provided by (Responsible Party):
Bio Products Laboratory

Brief Summary:

The main objective of the study is to assess the pharmacokinetics of FACTOR X after a single dose of 25IU/kg.

The secondary objectives of the study are to assess efficacy and safety of FACTOR X in the treatment of bleeding episodes over at least 6 months.

Condition or disease Intervention/treatment Phase
Factor X Deficiency Biological: Human Coagulation FACTOR X Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase III Open, Multicentre Study to Investigate the Pharmacokinetics, Safety and Efficacy of BPL's High Purity Factor X in the Treatment of Severe and Moderate Factor X Deficiency.
Study Start Date : January 2010
Actual Primary Completion Date : November 2013
Actual Study Completion Date : November 2013

Arm Intervention/treatment
Experimental: Human Coagulation FACTOR X Biological: Human Coagulation FACTOR X
Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.

Primary Outcome Measures :
  1. FX:C Incremental Recovery [ Time Frame: At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose) ]

    Incremental recovery is defined as the peak rise in plasma FX levels (IU/dL), as measured at 15, 30 and 60 minutes post-dose, divided by the dose (IU/kg).

    Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment

  2. FX:C Half-life [ Time Frame: At Baseline and at 6 months post-Baseline ]
    Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Written informed consent given, or for subjects aged 12-17 years, have given written assent and whose parent/guardian has given written informed consent
  • At least 12 years of age at dtae of written informed consent
  • Have hereditary severe or moderate FX deficiency
  • Currently treated with Fresh Frozen Plasma FFP, Prothrombin Complex Concentrate PCC or factor IX/X concentrate
  • Must have a minimum of one spontaneous or menorrhagic bleed in the last 12 months which required treatment of FFP, PCC or factor IX/X concentrate. Newly diagnosed subjects who present at the hospital with a bleed may be included
  • Must have had at least 7 days, and ideally 10-14 days, since an infusion of either FFP, PCC or factor IX/X concentrate at Baseline Visit
  • Females of child bearing potential must have a negative result on a HCG based pregnancy test. If they are or become sexually active, they must practise contraception by using a method of proven reliability for the duration of the study

Exclusion Criteria:

  • Have a history of inhibitor development to FX or a positive result at the Screening Visit
  • Bleeding at the appointment for the PK assessment
  • Subjects who have thrombocytopenia
  • Have clinically significant liver disease
  • Known to have other coagulopathy or thrombophilia
  • Have known or suspected hypersensitivity to the investigational medicinal product or its excipients
  • Have abused chemicals or drugs within the past 12 months
  • Have a history of unreliability or non-cooperation
  • Participating or have taken part in another trial within the last 30 days, with the exception of BPL FX surgery study - Protocol Ten03. In such cases, subjects should have completed their End of Study Visit either before or on the day of Screening Visit for this study
  • Female subjects who are pregnant or lactating
  • Subjects planning greater than 4 weeks absence from the locality of the Investigational site, between the screening visit and the repeat PK assessment

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00930176

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United States, California
UCSF School of Medicine
San Francisco, California, United States, 94117
United States, Indiana
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, United States, 46260
United States, New York
Dr. William Mitchell New York Blood Center, Weill Cornell Medical College
New York, New York, United States, 10065
Dr Gunter Auerswald
Bremen, Germany
Dr. Bermejo
Caceres, Spain
Dr Maite Alvarez
Madrid, Spain
Cukurova University Hospital
Balcali, Adana, Turkey
Ministry of Health Istanbul Goztepe Training & Research Hospital
Goztepe, Istanbul, Turkey
Istanbul University Cerrahpasa School of Medicine
Istanbul, Turkey
Kanuni Sultan Suleyman Training and Research Hospital
Istanbul, Turkey
Prof. Kavakli
Izmir, Turkey
Prof. Oner
Van, Turkey
United Kingdom
Dr. Sue Pavord
Leicester, United Kingdom
Dr. Steve Austin
London, United Kingdom
Sponsors and Collaborators
Bio Products Laboratory
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Principal Investigator: Amy Shapiro, Dr Co-Medical Director, Indiana Hemophilia and Thrombosis Center, 8402 Harcourt Road, Suite 420, Indianapolis, IN46260, USA

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Bio Products Laboratory Identifier: NCT00930176     History of Changes
Other Study ID Numbers: Ten01
First Posted: June 30, 2009    Key Record Dates
Results First Posted: December 12, 2014
Last Update Posted: December 12, 2014
Last Verified: December 2014
Additional relevant MeSH terms:
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Factor X Deficiency
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn