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

Bio Products Laboratory

Status and phase

Completed

Phase 3

Conditions

Factor X Deficiency

Treatments

Biological: Human Coagulation FACTOR X

Study type

Interventional

Funder types

Other

Identifiers

Ten01

Details and patient eligibility

About

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.

Enrollment

16 patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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