Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    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).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2007-003331-23
    Sponsor's Protocol Code Number:LA29-0207
    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:2007-08-02
    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 ConcernedUK - MHRA
    A.2EudraCT number2007-003331-23
    A.3Full title of the trial
    A six-month double-blind, randomized, placebo-controlled study investigating the safety and tolerability of deferiprone in participants with Friedreich's ataxia
    A.3.2Name or abbreviated title of the trial where available
    Not applicable
    A.4.1Sponsor's protocol code numberLA29-0207
    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 Inc.
    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 No
    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 100 mg/mL oral solution
    D.3.2Product code L1, APO-066, CP20
    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.3.9.2Current sponsor codeL1, APO-066, CP20
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral solution
    D.8.4Route of administration of the placeboOral use
    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 demonstrate the 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 efficacy of deferiprone for the treatment of FRDA, as assessed by a 9-Hole Peg Test (9HPT), Timed 25-Food Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich’s Ataxia Rating Scale (FARS).

    The tertiary objects are to evaluate the effect of deferiprone on:
    1) cardiac function as measured by changes in Left Ventricular Shortening Fraction (LVSF), Left Ventricular Ejection Fraction (LVEF) and Left Ventricular (LV) mass using echocardiogram (ECHO),
    2) quality of life using quality-of-life surveys, and
    3) functional status using Activities of Daily Living (ADL).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosis of FRDA, with confirmed mutation (excludes point mutation) in the frataxin (FXN) gene and GAA repeats ≥ 400 on the shorter allele.
    2. Males or females aged 7 to 35 years
    3. No exposure to idebenone, coenzyme Q10, vitamin C, vitamin E or other antioxidants as a supplement or as a drug therapy for a period of at least one month prior to start of treatment and during the study.
    4. Neurological testing: A FARS score >20 and <85 at Screening and Baseline.
    5. Female subjects of childbearing potential must have a negative pregnancy test at Baseline. In addition, a female subject must confirm that during the study and within 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) (in place for at least 3 months prior to first dose), 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.
    6. If the subject is a heterosexual, sexually-active male, he confirms 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.
    7. 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 schedule.
    E.4Principal exclusion criteria
    1. Iron deficiency defined as ferritin levels below the reference range for age- and sex-matched controls
    2. Unable to complete T25FW AND with score > 5 minutes in the 9HPT. (Subjects who can complete T25FW or with a score ≤ 5 minutes in the 9HPT will be allowed to enrol if the score has not doubled compared to screening).
    3. Abnormal ALT, greater than 2.0 times the upper limit of normal on two consecutive assessments.
    4. Serum creatinine outside the normal reference range.
    5. History or evidence of neutropenia/agranulocytosis defined by an absolute neutrophil count (ANC) < 1.5 x 10E9/L or thrombocytopenia defined by a platelet count <150 x 10E9/L.
    6. Refusal to participate in screening procedures or unable to participate in screening procedures or unable to comply with the requirements of the protocol.
    7. Receiving any investigational drug products or having received any investigational product within 30 days prior to enrolment into this study.
    8. Subjects who have previously taken deferiprone.
    9. Subjects who, in the opinion of the Investigator, represent poor medical, psychological or psychiatric risks, and for whom participation in an investigational trial would be unwise.
    10. Pregnant, breastfeeding or planning to become pregnant during the study period.
    11. History of malignancy.
    12. History of alcohol or drug abuse.
    13. Investigators, site personnel directly affiliated with this study and their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biological or legally adopted.
    14. Hypersensitivity to the active substance (deferiprone) or any of the excipients in the oral solution.
    15. QT Interval Greater than 450 ms at Baseline.
    E.5 End points
    E.5.1Primary end point(s)
    9-Hole Peg Test:
    The 9HPT is a quantitative test of the functionality of the upper extremities (see Appendix 4). 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 (see Appendix 5). 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. Three charts for low contrast (5%, 1.25% and 0.6%) 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 2 meters distance 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 (see Appendix 6). ICARS allows measurement of postural and gait ataxia (seven tasks, 0-34 points), limb ataxia (six tasks on both 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 (see Appendix 7), 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-10 and SF-36 are questionnaires that assess a range of health concepts. The SF-10 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:
    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 0 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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    New indication of deferiprone for the treatment of Friedreich's ataxia.
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    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 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
    Specified in the protocol.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months10
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 80
    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 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 Decision2007-08-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2008-11-20
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2009-02-03
    For support, Contact us.
    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
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat Apr 20 10:03:00 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA