Clinical trial NCT00930176: A Study Investigating Treatment Factor X in People With Factor X Deficiency
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Factor X Deficiency
Lead sponsor: Bio Products Laboratory
Sponsor class: OTHER
First posted: 2009-06-30
Enrollment: 16
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: FX:C Incremental Recovery
timeFrame: At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose)
measure: FX:C Half-life
timeFrame: At Baseline and at 6 months post-Baseline
Secondary outcomes:
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: UCSF School of Medicine
state: California
city: Indianapolis
country: United States
facility: Indiana Hemophilia & Thrombosis Center
state: Indiana
city: New York
country: United States
facility: Dr. William Mitchell New York Blood Center, Weill Cornell Medical College
state: New York
city: Bremen
country: Germany
facility: Dr Gunter Auerswald
city: Caceres
country: Spain
facility: Dr. Bermejo
city: Madrid
country: Spain
facility: Dr Maite Alvarez
city: Balcali
country: Turkey
facility: Cukurova University Hospital
state: Adana
city: Goztepe
country: Turkey
facility: Ministry of Health Istanbul Goztepe Training & Research Hospital
state: Istanbul
city: Istanbul
country: Turkey
facility: Istanbul University Cerrahpasa School of Medicine
city: Istanbul
country: Turkey
facility: Kanuni Sultan Suleyman Training and Research Hospital
city: Izmir
country: Turkey
facility: Prof. Kavakli
city: Van
country: Turkey
facility: Prof. Oner
city: Leicester
country: United Kingdom
facility: Dr. Sue Pavord
city: London
country: United Kingdom
facility: Dr. Steve Austin
Study officials:
affiliation: Co-Medical Director, Indiana Hemophilia and Thrombosis Center, 8402 Harcourt Road, Suite 420, Indianapolis, IN46260, USA
name: Amy Shapiro, Dr
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.