NCT00930176

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jan 2010

Typical duration for phase_3

Geographic Reach
5 countries

14 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 10, 2009

Completed
20 days until next milestone

First Posted

Study publicly available on registry

June 30, 2009

Completed
6 months until next milestone

Study Start

First participant enrolled

January 1, 2010

Completed
3.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2013

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

December 12, 2014

Completed
Last Updated

December 12, 2014

Status Verified

December 1, 2014

Enrollment Period

3.8 years

First QC Date

June 10, 2009

Results QC Date

November 25, 2014

Last Update Submit

December 8, 2014

Conditions

Factor X Deficiency

Outcome Measures

Primary Outcomes (2)

  • FX:C Incremental Recovery

    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

    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

Study Arms (1)

Human Coagulation FACTOR X

EXPERIMENTAL
Biological: Human Coagulation FACTOR X

Interventions

Human Coagulation FACTOR XBIOLOGICAL

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.

Human Coagulation FACTOR X

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

UCSF School of Medicine

San Francisco, California, 94117, United States

Location

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, 46260, United States

Location

Dr. William Mitchell New York Blood Center, Weill Cornell Medical College

New York, New York, 10065, United States

Location

Dr Gunter Auerswald

Bremen, Germany

Location

Dr. Bermejo

Cáceres, Spain

Location

Dr Maite Alvarez

Madrid, Spain

Location

Cukurova University Hospital

Balcali, Adana, Turkey (Türkiye)

Location

Ministry of Health Istanbul Goztepe Training & Research Hospital

Göztepe, Istanbul, Turkey (Türkiye)

Location

Istanbul University Cerrahpasa School of Medicine

Istanbul, Turkey (Türkiye)

Location

Kanuni Sultan Suleyman Training and Research Hospital

Istanbul, Turkey (Türkiye)

Location

Prof. Kavakli

Izmir, Turkey (Türkiye)

Location

Prof. Oner

Van, Turkey (Türkiye)

Location

Dr. Sue Pavord

Leicester, United Kingdom

Location

Dr. Steve Austin

London, United Kingdom

Location

Related Publications (1)

  • Oner AF, Celkan T, Timur C, Norton M, Kavakli K. Use of a High-Purity Factor X Concentrate in Turkish Subjects with Hereditary Factor X Deficiency: Post Hoc Cohort Subanalysis of a Phase 3 Study. Turk J Haematol. 2018 May 25;35(2):129-133. doi: 10.4274/tjh.2017.0446. Epub 2018 Mar 16.

    PMID: 29545231DERIVED

MeSH Terms

Conditions

Factor X Deficiency

Interventions

Factor X

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Enzyme PrecursorsEnzymes and CoenzymesBlood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Results Point of Contact

Title
Head of Medical Affairs
Organization
Bio Products Laboratory
medinfo@bpl.co.uk
+44 20 8957 2200

Study Officials

  • Amy Shapiro, Dr

    Co-Medical Director, Indiana Hemophilia and Thrombosis Center, 8402 Harcourt Road, Suite 420, Indianapolis, IN46260, USA

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
GT60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 10, 2009

First Posted

June 30, 2009

Study Start

January 1, 2010

Primary Completion

November 1, 2013

Study Completion

November 1, 2013

Last Updated

December 12, 2014

Results First Posted

December 12, 2014

Record last verified: 2014-12

Locations

GB(2)US(3)ES(2)TU(6)DE(1)