A study for people with severe and moderate coagulation factor X deficiency, to assess the effectiveness and safety of a high purity factor X concentrate, and how it is handled by the body.

• Register: EUCTR
• Last refreshed on: 7 April 2014
• Main ID: EUCTR2009-011145-18-GB
• Date of registration: 24/09/2009
• Prospective Registration: Yes
• Primary sponsor: Bio Products Laboratory Ltd
• Public title: A study for people with severe and moderate coagulation factor X deficiency, to assess the effectiveness and safety of a high purity factor X concentrate, and how it is handled by the body.
• Scientific 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 - A PK study of BPL's FX in patients with FX deficiency
• Date of first enrolment: 02/11/2009
• Target sample size: 16
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-011145-18
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other:

Countries of recruitment

Germany, Spain, Turkey, United Kingdom, United States

Contacts

• Name: Miranda Norton
• Affiliation: Bio Products Laboratory Limited

• Name: Miranda Norton
• Affiliation: Bio Products Laboratory Limited

Key inclusion & exclusion criteria

Inclusion criteria:
Informed consent
At least 12 years of age
With hereditary severe or moderate factor X deficiency (<5% basal activity at diagnosis)
Currently treated with fresh frozen plasma (FFP), prothrombin complex concentrates (PCC) or factor IX/X concentrate
Have had a minimum of one spontaneous or menorrhagic bleed in the last 12 months which required treatment with either FFP, PCC or factor IX/X concentrate
At least 7 days, and ideally 10-14 days, since an infusion of FFP, PCC or factor IX/X concentrate at the Baseline Visit
Female subjects of childbearing potential must not be pregnant at entry to the study and must practice contraception for the duration of the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 4
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 11
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

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 descriptive name: Human factor X
Concentration unit: IU international unit(s)
Concentration type: range
Concentration number: 400-625

Primary Outcome(s)

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).

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.

Secondary Outcome(s)

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) the 9 Month Visit.
FX inhibitor screens and Nijmegen-Bethesda assays are assessed at 3-monthly intervals.
Vitals and infusion site observations are performed before sampling at Baseline and repeat PK. Vitals are also performed at screening, End of Study and at 3-month intervals.
Thrombogenicity is measured pre-dose and at intervals up to 72 h post-dose at Baseline and repeat PK.
Efficacy in treating bleeds is assessed after bleeds have stopped.
Genotyping performed at screening.
AEs are assessed at all study visits.
All other endpoints will be evaluated on study completion.

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).

Secondary ID(s)

TEN01

Source(s) of Monetary Support

Bio Products Laboratory Limited