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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Bio Products Laboratory
ClinicalTrials.gov Identifier:
NCT00930176
First received: June 10, 2009
Last updated: December 9, 2013
Last verified: December 2013
  Purpose

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 Intervention Phase
Factor X Deficiency
Biological: Human Coagulation FACTOR X
Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: 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.

Resource links provided by NLM:


Further study details as provided by Bio Products Laboratory:

Primary Outcome Measures:
  • Pharmacokinetic parameters for FACTOR X:Concentrate at the Baseline Visit and the repeat Pharmacokinetic assessment (usually at the 6 month visit) [ Time Frame: maximum study duration of study treatment (excluding the screening period) for each subject is 2 years. ] [ Designated as safety issue: Yes ]

Enrollment: 16
Study Start Date: January 2010
Study Completion Date: November 2013
Primary Completion Date: November 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  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: NCT00930176

Locations
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
Germany
Dr Gunter Auerswald
Bremen, Germany
Spain
Dr. Bermejo
Caceres, Spain
Dr Maite Alvarez
Madrid, Spain
Turkey
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
Investigators
Principal Investigator: Amy Shapiro, Dr Co-Medical Director, Indiana Hemophilia and Thrombosis Center, 8402 Harcourt Road, Suite 420, Indianapolis, IN46260, USA
  More Information

No publications provided

Responsible Party: Bio Products Laboratory
ClinicalTrials.gov Identifier: NCT00930176     History of Changes
Other Study ID Numbers: Ten01
Study First Received: June 10, 2009
Last Updated: December 9, 2013
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

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 November 20, 2014