Factor X deficiency MedDRA version: 14.1 Level: PT Classification code 10052474 Term: Factor X deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Human factor X
Product Code: FACTOR X
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: Human coagulation Factor X
Current Sponsor code: FACTOR X
Other descript
Sponsors
Bio Products Laboratory Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Informed consent At least 12 years of age With hereditary severe or moderate factor X deficiency (=65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: History of inhibitor development to FX, or a positive inhibitor result at the Screening Visit Bleeding at the Baseline Visit Subjects with thrombocytopenia, clinically significant renal disease or clinically significant liver disease Subjects with other coagulopathy or thrombophilia Known or suspected hypersensitivity to the investigational medicinal product or its excipients Known to have abused chemicals or drugs within the past 12 months History of unreliability or non-cooperation Participation in another clinical trial within the past 30 days, with the exception of BPL FX surgery study (protocol No Ten03). In such cases, subjects should have completed their End-of-Study Visit either before or on the day of the Screening Visit for this study Female subjects who are pregnant or lactating. Subjects who are planning more than 4 weeks absence from the locality of the investigational site, between the Screening Visit and the Repeat PK Assessment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the pharmacokinetics of Factor X after a single dose of 25 IU/kg;Secondary Objective: To assess the efficacy of Factor X in the treatment of bleeding episodes over at least 6 months. To assess the safety of Factor X in the treatment of bleeding episodes over at least 6 months.;Primary end point(s): The PK parameters: incremental recovery (at 30 min post-dose), half-life (non-compartmental), AUC(0-144), AUC(0-8), AUC(0-t), clearance, MRT(0-8), volume of distribution, C0, Cmax(obs) Tmax and terminal elimination rate constant for FX:C at the Baseline Visit and the repeat PK assessment (usually at the 6 Month Visit).;Timepoint(s) of evaluation of this end point: Baseline Visit and repeat PK assessment (usually at the 6 Month Visit). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • PK endpoints: incremental recovery (at 30 min post-dose), half-life (non-compartmental), AUC(0-144h), AUC(0-8), AUC(0-t), clearance, MRT (0-8), volume of distribution, C0, Cmax(obs), Tmax and terminal elimination rate constant for FX:Ag • total dose of FACTOR X (IU and IU/kg FX:C), total number of infusions and average dose per infusion to treat a new bleed and ongoing bleeds, for any additional preventative use and overall use per subject. • total dose of FACTOR X to treat a bleed (IU/kg FX:C) (including initial dose for new bleeds and any repeated doses for ongoing bleeds), number of infusions and dose per infusion on a per bleed and a per subject basis. • dose of FACTOR X per infusion for all infusions, all infusions to treat bleeds, all first infusions to treat bleeds, all subsequent infusions to treat bleeds, and all infusions taken as a preventative measure. • average monthly and yearly dose of FACTOR X (IU/kg FX:C), and average monthly and yearly number of infusions to treat a bleed, for any additional preventative use and overall use, all per subject. • investigator’s overall assessment of efficacy • number of exposure days overall and per subject • average number of bleeds per month per subject • number of bleeds including severity, duration, location and cause • subject’s assessment of efficacy in treating a bleed (all bleeds) • investigator’s assessment of efficacy in treating a bleed (bleeds assessed at the hospital). • adverse events • haematology • serum biochemistry • PT and APTT • viral serology • FX inhibitor screens and Nijmegen-Bethesda assays • thrombogenicity markers • vital signs • physical examination • infusion site observations • genotype analysis (optional). ;Timepoint(s) of evaluation of this end point: PK endpoints are assessed at Baseline and repeat PK. Haematology, biochemistry, PT, APTT, viral serology and physical examination are assessed at Baseline, End of Study and (if applicable | — |
Countries
Germany, Spain, Turkey, United Kingdom, United States
Contacts
Public ContactMiranda Norton
Bio Products Laboratory Limited
Outcome results
None listed