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    Summary
    EudraCT Number:2013-003240-23
    Sponsor's Protocol Code Number:967908487
    National Competent Authority:Ireland - HPRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2013-10-10
    Trial 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 ConcernedIreland - HPRA
    A.2EudraCT number2013-003240-23
    A.3Full title of the trial
    PERSONALising Factor VIII prophylaxis regimens: Efficacy of standard versus pharmacokinetically based regimens in adult patients with severe Haemophilia A (PERSONAL trial)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    To compare the effectiveness of standard weight based dosing of Factor VIII prophylaxis against regimens using an individual's own rate of Factor VIII clearance in adult patients with severe Factor VIII deficiency (Haemophilia A).
    A.3.2Name or abbreviated title of the trial where available
    PERSONAL trial
    A.4.1Sponsor's protocol code number967908487
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSt. James' Hospital
    B.1.3.4CountryIreland
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportBaxter Healthcare
    B.4.2CountryIreland
    B.4.1Name of organisation providing supportIrish Haemostasis Research Fund
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNational Centre for Hereditary Coagulation Disorders
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street AddressSt. James' Hospital
    B.5.3.2Town/ cityDublin 8
    B.5.3.3Post code0000
    B.5.3.4CountryIreland
    B.5.4Telephone number35314162141
    B.5.6E-mailmlavin@stjames.ie
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name ADVATE
    D.2.1.1.2Name of the Marketing Authorisation holderBaxter AG
    D.2.1.2Country which granted the Marketing AuthorisationIreland
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Concentrate and solvent for solution for 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 INNOCTOCOG ALFA
    D.3.9.1CAS number 139076-62-3
    D.3.9.4EV Substance CodeSUB16449MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU/mg international unit(s)/milligram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number4000 to 10000
    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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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
    Severe Haemophilia A
    E.1.1.1Medical condition in easily understood language
    Factor VIII level less than 1% of normal
    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 LLT
    E.1.2Classification code 10018937
    E.1.2Term Haemophilia A
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare efficacy and safety of standard weight based Factor VIII prophylaxis with individually tailored pharmacokinetically based FVIII prophylaxis
    E.2.2Secondary objectives of the trial
    To compare the number of clinically significant bleeds and

    factor VIII usage in patients who are taking:

    a. Prophylaxis prescribed according to routine clinical practice

    with

    b. Prophylaxis prescribed on alternate days to maintain

    predicted target trough of ≥1.5IU/dL based on sparse blood

    sampling and Bayesian pharmacokinetic estimation

    2) Compare the rate of all haemarthroses and soft

    tissue bleeds between the two regimens

    3) To compare the number of clinically evident joint

    bleeds with any MRI joint changes and Pettersson

    scores

    4) Establish the intra-patient variability of pharmacokinetic

    parameters over time by a population pharmacokinetic

    method

    5) Establish the accuracy of factor VIII levels predicted

    from studies using sparse (2-3 blood sample) data,

    versus measured factor VIII levels in patients on

    prophylaxis and to investigate patient-related variables

    that affect factor VIII pharmacokinetics
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria


    Inclusion criteria:

    • Severe Haemophilia A (baseline Factor VIII level of <1 IU/dL)

    • Age 18 years and above

    • Patients taking any regular prophylactic regimen (defined

    as regular factor VIII infusions, at least 5 times a fortnight, with the aim of minimising haemarthroses and other clinically significant bleeds).

    • Low titre inhibitors, past history of an inhibitor, abnormal liver function, drugs that interfere with haemostasis and low CD4 counts are allowed.
    E.4Principal exclusion criteria
    • Presence of a target joint on prophylaxis (defined as 3 bleeds into one joint, during a 6 month period, during the last year).

    • The occurrence of more than 3 haemarthroses in the last year which required more than 2 infusions to resolve.

    • Pregnant or breastfeeding women

    • Patients with a learning disability or dementia

    • Prisoners

    • Adults who are unconscious/unable to give informed consent

    • Participants with a pacemaker or implanted medical devices which are unsuitable to have a MRI will be excluded from the MRI scans during the trial but may proceed with other components.

    E.5 End points
    E.5.1Primary end point(s)
    Number of bleeding events experienced while on standard and Pk tailored prophylaxis
    E.5.1.1Timepoint(s) of evaluation of this end point
    6,18 months
    E.5.2Secondary end point(s)
    1. The factor VIII usage
    2. The total number of all haemarthroses and soft tissue bleeds
    3. Quality of life (EQ5D)
    4. Patients’ joint status (HJHS), MRI joint score (IPSG score ) and Pettersson score
    5. The accuracy of factor VIII levels predicted from studies using sparse (2-3 blood sample) data, versus measured factor VIII levels in patients on prophylaxis
    6. The intra-patient variability of pharmacokinetic parameters over time, as estimated by a population pharmacokinetic method
    E.5.2.1Timepoint(s) of evaluation of this end point
    6,18 month
    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 No
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    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 Yes
    E.8.2.3.1Comparator description
    Weight based prophylaxis
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    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 subject last visit
    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 months6
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 38
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state38
    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)
    Participants will continue on recombinant factor VIII prophylaxis post trial and data will be analysed to see if pharmacokinetically tailored is as efficacious and safe as weight based prophylaxis.
    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 Decision2013-12-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-09-16
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2015-09-01
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