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    Summary
    EudraCT Number:2009-011145-18
    Sponsor's Protocol Code Number:TEN01
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2009-09-24
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2009-011145-18
    A.3Full title of the trial
    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.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    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.
    A.3.2Name or abbreviated title of the trial where available
    A PK study of BPL's FX in patients with FX deficiency
    A.4.1Sponsor's protocol code numberTEN01
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBio Products Laboratory Ltd
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportBio Products Laboratory Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBio Products Laboratory Limited
    B.5.2Functional name of contact pointMiranda Norton
    B.5.3 Address:
    B.5.3.1Street AddressDagger Lane
    B.5.3.2Town/ cityElstree
    B.5.3.3Post codeWD6 3BX
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number0208957 2661
    B.5.5Fax number0208957 2611
    B.5.6E-mailmiranda.norton@bpl.co.uk
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/07/471
    D.3 Description of the IMP
    D.3.1Product nameHuman factor X
    D.3.2Product code FACTOR X
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHuman coagulation Factor X
    D.3.9.2Current sponsor codeFACTOR X
    D.3.9.3Other descriptive nameHuman factor X
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number400 to 625
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Factor X deficiency
    E.1.1.1Medical condition in easily understood language
    Factor X deficiency is lack of human coagulation factor X, one of the proteins in blood which enables blood to clot. This deficiency can cause patients to bleed spontaneously or following trauma.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10052474
    E.1.2Term Factor X deficiency
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the pharmacokinetics of Factor X after a single dose of 25 IU/kg
    E.2.2Secondary objectives of the trial
    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.
    E.2.3Trial contains a sub-study No
    E.3Principal 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.
    E.4Principal 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.
    E.5 End points
    E.5.1Primary end point(s)
    The PK parameters: incremental recovery (at 30 min post-dose), half-life (non-compartmental), AUC(0-144), AUC(0-∞), AUC(0-t), clearance, MRT(0-∞), 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).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline Visit and repeat PK assessment (usually at the 6 Month Visit).
    E.5.2Secondary end point(s)
    • PK endpoints: incremental recovery (at 30 min post-dose), half-life (non-compartmental), AUC(0-144h), AUC(0-∞), AUC(0-t), clearance, MRT (0-∞), 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).
    E.5.2.1Timepoint(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.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Turkey
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    See protocol Section 4.4.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 4
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 11
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 16
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    See protocol Section A-6.6.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2009-07-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-11-02
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2013-10-30
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