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

December 8, 2014 updated by: Bio Products Laboratory

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.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

16

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Bremen, Germany
        • Dr Gunter Auerswald
  • Spain
      • Caceres, Spain
        • Dr. Bermejo
      • Madrid, Spain
        • Dr Maite Alvarez
  • Turkey
      • Istanbul, Turkey
        • Istanbul University Cerrahpaşa School of Medicine
      • Istanbul, Turkey
        • Kanuni Sultan Suleyman Training and Research Hospital
      • Izmir, Turkey
        • Prof. Kavakli
      • Van, Turkey
        • Prof. Oner
    • Adana
      • Balcali, Adana, Turkey
        • Çukurova University Hospital
    • Istanbul
      • Goztepe, Istanbul, Turkey
        • Ministry of Health Istanbul Goztepe Training & Research Hospital
  • United Kingdom
      • Leicester, United Kingdom
        • Dr. Sue Pavord
      • London, United Kingdom
        • Dr. Steve Austin
  • United States
    • California
      • San Francisco, California, United States, 94117
        • UCSF School of Medicine
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Indiana Hemophilia & Thrombosis center
    • New York
      • New York, New York, United States, 10065
        • Dr. William Mitchell New York Blood Center, Weill Cornell Medical College

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose)
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
At Baseline and at 6 months post-Baseline

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2010

Primary Completion (ACTUAL)

November 1, 2013

Study Completion (ACTUAL)

November 1, 2013

Study Registration Dates

First Submitted

June 10, 2009

First Submitted That Met QC Criteria

June 29, 2009

First Posted (ESTIMATE)

June 30, 2009

Study Record Updates

Last Update Posted (ESTIMATE)

December 12, 2014

Last Update Submitted That Met QC Criteria

December 8, 2014

Last Verified

December 1, 2014

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Ten01

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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