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

Phase 3

Completed

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.

Recruitment & Eligibility

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 Outcome Measures

Name	Time	Method
FX:C Incremental Recovery	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	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

Secondary Outcome Measures

Name	Time	Method

Locations (14): UCSF School of Medicine
🇺🇸
San Francisco, California, United States
Indiana Hemophilia & Thrombosis Center
🇺🇸
Indianapolis, Indiana, United States
Dr. William Mitchell New York Blood Center, Weill Cornell Medical College
🇺🇸
New York, New York, United States
Dr Gunter Auerswald
🇩🇪
Bremen, Germany
Dr. Bermejo
🇪🇸
Caceres, Spain
Dr Maite Alvarez
🇪🇸
Madrid, Spain
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
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UCSF School of Medicine
🇺🇸San Francisco, California, United States