A Study Investigating Treatment Factor X in People With 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.

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Key information

Conditions

Factor X Deficiency Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Dr Gunter Auerswald, Bremen, Germany

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Human Coagulation FACTOR X

Biological

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 outcomes

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
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

Sponsors and collaborators

Lead sponsor

Bio Products Laboratory

Other

Registry information

Official study 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.

Important dates

Study start: 2010
Primary completion: 2013
Study completion: 2013
First posted: Jun 30, 2009
Registry last updated: Dec 12, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed