Phase 3
N=16
A Study Investigating Treatment Factor X in People With Factor X Deficiency
Factor X Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT00930176 ↗Enrolled (actual)
16
Serious AEs
37.5%
Results posted
Dec 2014
Primary outcome: Primary: FX:C Incremental Recovery — 2.07 IU/dL per IU/kg
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Human Coagulation FACTOR X (Biological)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Bio Products Laboratory
- Primary completion
- Nov 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY FX:C Incremental Recovery |
2.07 | — |
| PRIMARY FX:C Half-life |
29.36 | — |
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
Data sourced from ClinicalTrials.gov (NCT00930176). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.