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    The EU Clinical Trials Register currently displays   43851   clinical trials with a EudraCT protocol, of which   7283   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2012-003093-98
    Sponsor's Protocol Code Number:Ten02
    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:2013-01-02
    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 number2012-003093-98
    A.3Full title of the trial
    A Phase III Open, Multicentre Study to Confirm the Safety, Pharmacokinetics and Efficacy of BPL’s High Purity Factor X in the Prophylaxis of Bleeding in Factor X Deficient Children Under the Age of 12 Years
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The purpose of this study is to look at how well FACTOR X works in preventing bleeding when taken regularly in children aged under 12 years old, over a period of 6 months. The study will also look at how safe FACTOR X is by closely following the subjects progress during the study.
    A.4.1Sponsor's protocol code numberTen02
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/57/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBio Products Laboratory Limited
    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 pointHead of Clinical Research
    B.5.3 Address:
    B.5.3.1Street AddressDagger Lane
    B.5.3.2Town/ cityElstree, Hertfordshire
    B.5.3.3Post codeWD6 3BX
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number4402089572200
    B.5.5Fax number4402089572611
    B.5.6E-mailmedinfo@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 concentrate for solution for injection/infusion
    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.3.9.1CAS number 9001-29-0
    D.3.9.3Other descriptive nameFACTOR X
    D.3.9.4EV Substance CodeSUB13816MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 a 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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 6 months
    E.2.2Secondary objectives of the trial
    (1) to assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg

    (2) to assess the safety of FACTOR X when given as routine prophylaxis over 6 months
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Children with hereditary severe or moderate FX deficiency (<5 IU/dL FX:C, based on their lowest reliable FX:C recorded)
    Children under 12 years old, whose parent/guardian has given informed consent.
    Children with a history of severe bleeding (a minimum of one bleed with a bleed score of 3 or 4, Appendix XI), or a mutation in the F10 gene causing a documented severe bleeding phenotype.
    E.4Principal exclusion criteria
    Children must not suffer from clinically significant liver disease, renal disease, or other coagulopathy or thrombophilia, and must have no history of inhibitors to FX.
    Children who have known or suspected hypersensitivity to the investigational medicinal product or its excipients.
    Children with a history of unreliability or non-cooperation.
    Children who are participating or have taken part in another trial within the last 30 days.
    Children planning more than 4 weeks’ continuous absence from the locality of the investigational site, between the Screening Visit and the End of Study Visit.
    E.5 End points
    E.5.1Primary end point(s)
    The investigator’s assessment of the efficacy of FACTOR X in the reduction/prevention of bleeding when given as routine prophylaxis over 6 months.
    This will take into account the child’s risk of breakthrough bleeding (due to bleeding history, treatment history and genetic mutation) and other relevant factors eg compliance with the protocol, attainment of required FX:C trough levels.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At the end of the study
    E.5.2Secondary end point(s)
    Number of bleeds per month including severity, duration, location and cause.

    Investigator’s and parent's/guardian's assessment of efficacy in treating a bleed for all bleeds (parent/guardian assessment) and for bleeds treated at hospital (investigator's assessment).

    Factor X activity trough levels at all scheduled study visits and at all Bleed Assessment and Trough Measurement unscheduled Visits.

    FX:C 30 minute post-dose incremental recovery at the Baseline Visit and the End of Study Visit.

    Factor X activity incremental recovery and trough levels following any change in dose regimen required for clinical reasons/insufficient trough levels.

    Dose to treat a bleed (IU/kg FX:C) (including initial dose for new bleeds and any repeated doses for ongoing bleeds), number of infusions to treat a bleed and dose per infusion; all analysed on a per bleed and a per subject basis.

    Total dose in IU/kg of factor X (factor X activity), total number of infusions and average dose per infusion for: prophylactic use, to treat a bleed, any additional preventative use, any surgical use and overall use; all analysed on a per subject basis.

    Average monthly dose in IU/kg of factor X (factor X activity), and average monthly number of infusions for: prophylactic use, to treat a bleed, any additional preventative use, any surgical use and overall use; all analysed on a per subject basis.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At the end of the study
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 concerned3
    E.8.5The trial involves multiple Member States No
    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 Kingdom
    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
    Last child's follow up safety assessment.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months5
    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: 8
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 1
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects under age incapable of giving consent personally.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 8
    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)
    Following the study, children will revert to their pre-study treatment, or they may continue to receive FACTOR X supplied commercially on a named-patient basis according to product availability, if permitted by local regulations, until the product receives regulatory approval or the development programme is terminated.
    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 Decision2013-02-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-02-24
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-08-19
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