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    Summary
    EudraCT Number:2009-010865-22
    Sponsor's Protocol Code Number:LA29-EXT
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2009-06-12
    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 ConcernedBelgium - FPS Health-DGM
    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.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberNot applicable
    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 Information not present in EudraCT
    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 109/L or
    thrombocytopenia defined by a platelet count <150 x 109/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)
    Evaluation of long-term safety and tolerability of deferiprone in subjects with Friedreich's ataxia.

    9-Hole Peg Test
    The 9HPT is a quantitative test of the functionality of the upper extremities. The test will be administered by a trained examiner (not necessarily
    the Investigator) in accordance with the Schedule of Events. The subject is
    instructed to place pegs into set holes as quickly as possible and then remove all
    the pegs. The test is performed two consecutive times with the dominant hand,
    followed by two consecutive times with the non-dominant hand. The maximum
    time allotted for each test (dominant or non-dominant hand) will be 5 minutes.
    The total time in seconds it takes the subject to complete the task is recorded. The
    average time for the four tests will be evaluated as a measure of treatment
    efficacy.

    Timed 25-Foot Walk
    The T25FW is a quantitative mobility and leg function performance test. The test will be administered by a trained examiner (not necessarily
    the Investigator) in accordance with the Schedule of Events. The subject is
    instructed to walk 25 feet along a pre-defined straight course as quickly as
    possible. The task will be immediately repeated by having the subject walk back
    to the starting point. Subjects will be allowed to use assistive devices when
    completing this task. The total time in seconds it takes the subject to walk 25 feet
    will be recorded for each test. The average time for the two tests will be evaluated
    as a measure of treatment efficacy.

    Low Contrast Letter Acuity
    LCLA is a quantitative Clinical measure to determine the level of visual
    dysfunction. Low-Contrast Sloan Letter Charts (LCSLC, Precision Vision,
    LaSalle, IL), which provide a quantitative and standardized method of visual
    function assessment, will be used for this study. These charts have gray letters of
    progressively smaller size on a white background. Two charts for low contrast
    (2.5% and 1.25%) as well as one chart for high contrast (100%; to determine
    visual acuity) will be used in this study. Subjects are requested to identify single
    letters on each chart at 3.2 meters distance for the 100% contrast and at 2 meters
    distance for the 2.5 and 1.25% contrast and scored based on number of letters
    correctly identified on each chart. The combined letter score from four charts will
    be recorded as a summary measure and will be evaluated as a measure of
    treatment efficacy. Subjects will be allowed to use their habitual distance
    correction glasses or contact lenses. The test will be administered by a trained
    individual.

    International Cooperative Ataxia Rating Scale
    ICARS is a semi-quantitative scale based upon 19 test parameters that assess
    sensory and motor skills. ICARS allows measurement of
    postural and gait ataxia (seven tasks, 0-34 points), limb ataxia (six tasks on sides,
    and one writing task, 0-52 points), dysarthria of speech (two items, 0-8 points),
    and oculomotor signs (three items, 0-6 points). The overall score ranges from 0 to
    100, with a score of 0 representing normal sensory and motor skills and 100 the
    worst. Subjects who are unable to perform a task receive the worst score,
    whatever the cause may be. ICARS testing will be performed by the same
    examiner (where possible) at various scheduled time points in the study as
    outlined in the Schedule of Events.

    Friedreich’s Ataxia Rating Scale
    FARS is a neurological exam with 25 maneuvers and three quantitative
    performance measures, encompassing bulbar function,
    coordination of upper and lower limbs, peripheral nervous system function, deep
    tendon reflexes, stability and gait. FARS will be conducted by a trained examiner
    (not necessarily the Investigator) in accordance with the Schedule of Events.
    Whenever possible, FARS testing will be performed by the same examiner at each
    of the scheduled time points outlined in the Schedule of Events. Since there is
    some overlap between ICARS and FARS, the exams will be merged into one
    complete exam consisting of a total of 34 maneuvers.

    Quality of Life
    The SF-b and SF-36 are questionnaires that assess a range of health concepts.
    The SF-b will be completed by the parent(s) or guardian(s) of subjects (aged 7 to
    <18 years), and the SF-36 will be completed by the adult subjects (aged 18 to 35
    years), in accordance with the Schedule of Events.

    Echocardiogram
    Echocardiogram (ECHO) to assess LVSF, LVEF and LV mass will be performed
    at visits outlined in the Schedule of Events.

    Activities of Daily Living
    Activities of Daily Living (ADL) is a quantitative method to assess the functional
    status of the subject. Subjects are given a score between 0 and 36, with a score of
    o representing normal functional status, and 36 the worst. Whenever possible, the
    ADL will be performed by the same examiner at each of the scheduled time points
    outlined in the Schedule of Events.
    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 months4
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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 state3
    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 Decision2009-06-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-07-16
    P. End of Trial
    P.End of Trial StatusOngoing
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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