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    Summary
    EudraCT Number:2009-010865-22
    Sponsor's Protocol Code Number:LA29-EXT
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2009-07-29
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2009-010865-22
    A.3Full title of the trial
    An open-label, single treatment, safety and efficacy, long-term study of deferiprone in subjects with Friedreich's ataxia.
    A.3.2Name or abbreviated title of the trial where available
    Not applicable
    A.4.1Sponsor's protocol code numberLA29-EXT
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorApoPharma Incorporated
    B.1.3.4CountryCanada
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Yes
    D.2.1.1.1Trade name Ferriprox
    D.2.1.1.2Name of the Marketing Authorisation holderApotex Europe B.V.
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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.3.1Product nameDeferiprone
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDeferiprone
    D.3.9.1CAS number 30652-11-0
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    Friedreich's ataxia
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10017374
    E.1.2Term Friedreich's ataxia
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to evaluate the long-term safety and
    tolerability of deferiprone in subjects with Friedreich?s ataxia (FRDA).
    E.2.2Secondary objectives of the trial
    The secondary objective is to evaluate the long-term efficacy of
    deferiprone for the treatment of FRDA.

    The tertiary objectives are to evaluate the effect of deferiprone on:
    1) cardiac function,
    2) quality of life, and
    3) functional status.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects who completed the ApoPharma study LA29-0207.

    2. Female subjects of childbearing potential must have a negative
    pregnancy test. In addition, a female subject must confirm that during
    the study and for 30 days following the completion of the study or
    early termination she:
    - will use an effective method of contraception, OR
    - has had a tubal ligation (supporting evidence required), OR
    - has had a hysterectomy (supporting evidence required), OR
    - participates in a non-heterosexual lifestyle, OR
    - indicates her only male sexual partner has been sterilized
    (supporting evidence required).
    Effective methods of contraception include oral contraceptives,
    intrauterine devices (RIDs), diaphragm or condom, providing they are
    used with contraceptive foam or cream, or abstinence from sexual
    intercourse. Supporting evidence for sterilization consists of a surgical
    report or letter from the family physician.

    3. Male subjects must confirm that he and/or his female partner will use an effective method of contraception for the length of the trial and for
    30 days following completion of the study or early termination.
    Effective methods of contraception for males include condoms or
    sterilization or abstinence from sexual intercourse or participation in a
    non-heterosexual lifestyle.

    4. Signed and witnessed written informed consent/assent, obtained prior
    to the first study intervention, as well as the ability to adhere to study
    restrictions, appointments and evaluation schedules.
    E.4Principal exclusion criteria
    1. Serum Ferritin and Hemoglobin (Hb) levels are below the reference
    range for age and sex-matched controls.

    2. Unable to complete T25FW AND with a score > 5 minutes in the
    9HPT. (Subjects who can complete T25FW or with a score less than or equal to 5
    minutes in the 9HPT will be allowed to enrol).

    3. Doubling of score on 9HPT or T25FW compared to their study
    baseline results in LA29-0207.

    4. History or evidence of neutropenia/agranulocytosis defined by a
    confirmed absolute neutrophil count (ANC) < 1.5 x 10^9/L or
    thrombocytopenia defined by a platelet count <150 x 10^9/L.

    5. Occurrence of SAEs or any other AEs during the LA29-0207 study,
    which in the opinion of the investigator cause the patient's
    participation in the extension study to be inappropriate.

    6. Unable to comply with requirements of the protocol.

    7. Pregnant, breastfeeding or planning to become pregnant during the
    study period.

    8. QTc interval >450ms.

    9. Have been on antioxidants prior to start of study treatment.
    E.5 End points
    E.5.1Primary end point(s)
    Safety Assessments:

    1. Hematology assessments consisting of complete blood count (CBC) and differential, absolute neutrophil count (ANC): at End of Study assessment for LA29-0207, Weekly (every 7 ± 3 days from start of study) during the study, and at the End of the Study or early termination, whichever comes first.

    2. Serum ferritin assessment: at End of Study for LA29-0207, Monthly (every 30 ± 7 days from start of study), and at the End of Study or early termination, whichever comes first.

    3. Blood chemistry assessments consisting of zinc, fasting glucose, alanine aminotransferase (ALT), aspartate aminotransferase (AST) and creatinine: at End of Study for LA29-0207, Quarterly (13, 26, 39 weeks ± 7 days) and at the end of the study or early termination, whichever comes first.

    4. Physical examination including head, eyes, ears, nose, throat, cardiovascular, respiratory, musculoskeletal, dermatological, neurological, lymph nodes, endocrine/metabolic, gastrointestinal and genitourinary and reproductive: at End of Study for LA29-0207, and at the end of the study or early termination, whichever comes first.

    5. Vital signs (height, weight, pulse, blood pressure and temperature): at End of Study for LA29-0207, Quarterly (13, 26, 39 weeks ± 7 days), and at the end of the study or early termination, whichever comes first.

    6. 9HPT and T25FW: at End of Study for LA29-0207, Quarterly (13, 26, 39 weeks ± 7 days), and at the End of Study or early termination, whichever comes first. In addition, 9HPT and T25FW may be repeated at any visit during the study for safety reasons as deemed necessary by the investigator.

    7. Electrocardiogram: at End of Study for LA29-0207, Semi-Annually (26 weeks ± 7 days from start of study) and at the End of Study or early termination, whichever comes first.

    8. Adverse Events (AEs) and medication(s): subjects will be questioned about the occurrence of any AEs and the use of any medication(s) at End of Study for LA29-0207, Weekly (every 7 ± 3 days from start of study), at the End of Study or early termination.

    9. Pregnancy tests: at End of Study for LA29-0207, Monthly (every 30 ± 7 days from start of study) and at the End of Study or early termination for all females of childbearing potential. Monthly (every 30 ± 7 days from start of study) contraceptive counselling will also be provided for all sexually active males and females.

    Efficacy Assessments:

    1. 9HPT: at End of Study for LA29-0207, Quarterly (13, 26, 39 weeks ± 7 days) and at the End of Study or early termination, whichever comes first.

    2. T25FW: at End of Study for LA29-0207, Quarterly (13, 26, 39 weeks ± 7 days), and at the End of Study or early termination, whichever comes first.

    3. LCLA: at End of Study for LA29-0207, Quarterly (13, 26, 39 weeks ± 7 days), and at the End of Study or early termination, whichever comes first.

    4. ICARS: at End of Study for LA29-0207, Quarterly (13, 26, 39 weeks ± 7 days), and at the End of Study or early termination, whichever comes first.

    5. FARS: at End of Study for LA29-0207, Quarterly (13, 26, 39 weeks ± 7 days), and at the End of Study or early termination, whichever comes first.

    6. Quality of life survey (SF-10 for minors/SF-36 for adults): at End of Study for LA29-0207, Semi-Annually (26 weeks ± 7 days from start of study), and at the End of Study or early termination, whichever comes first.

    7. ECHO: at End of Study for LA29-0207, Semi-Annually (26 weeks ± 7 days from start of study), and at the End of Study or early termination, whichever comes first.

    8. Activities of Daily Living (ADL): at End of Study for LA29-0207, Semi-Annually (26 weeks ± 7 days from start of study), and at the End of Study or early termination, whichever comes first.
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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 Information not present in EudraCT
    E.7.1.2Bioequivalence study Information not present in EudraCT
    E.7.1.3Other Information not present in EudraCT
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    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 months3
    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 months6
    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.1.1.1In 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) Yes
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minors will require legal representative's assent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 47
    F.4.2.2In the whole clinical trial 48
    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)
    Specified in the protocol.
    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 Decision2009-10-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-07-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2011-03-02
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