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    Summary
    EudraCT Number:2009-010865-22
    Sponsor's Protocol Code Number:LA29-EXT
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2009-04-16
    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 ConcernedItaly - Italian Medicines Agency
    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
    ND
    A.4.1Sponsor's protocol code numberLA29-EXT
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberND
    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 INC., TORONTO, ONTARIO
    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.3.4Pharmaceutical 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.3.10 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
    patients affected by Friedreich Ataxia
    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
    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.
    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 at the End of Study LA29-0207. 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 (IUDs), 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. Iron deficiency defined as serum ferritin levels below the reference range for age-and sex- matched controls at the End of Study assessment (End of Study) for LA29-0207. 2. Unable to complete T25FW AND with a score < 5 minutes in the 9HPT at the End of Study assessment for LA29-0207. (Subjects who can complete T25FW or with a score &amp;#8804; 5 minutes in the 9HPT will be allowed to enrol). 3. Doubling of score on 9HPT or T25FW at end of LA29-0207 compared to their study baseline results. 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 at the End of Study assessment for LA29-0207. 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 at the End of Study assessment for LA29-0207. 9. Have been on antioxidants prior to start of study treatment.
    E.5 End points
    E.5.1Primary end point(s)
    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 &amp;#61617; 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 &amp;#61617; 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 &amp;#61617; 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 Yes
    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) 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 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 No
    E.8.1.7Other No
    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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    ultima visita dell`ultimo paziente arruolato
    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 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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 74
    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 Decision2009-05-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-04-01
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2011-03-02
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