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    Summary
    EudraCT Number:2008-003662-25
    Sponsor's Protocol Code Number:12631A
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2009-08-21
    Trial results View 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 ConcernedGermany - BfArM
    A.2EudraCT number2008-003662-25
    A.3Full title of the trial
    Randomised, double blind, placebo controlled study of Lu AA24493 in patients with Friedreich's Ataxia to evaluate safety and tolerability and to explore efficacy
    A.4.1Sponsor's protocol code number12631A
    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 SponsorH. Lundbeck A/S
    B.1.3.4CountryDenmark
    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 nameLu AA24493
    D.3.2Product code Lu AA24493
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPIntravenous bolus use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeLu AA24493
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.25
    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) 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 placeboSolution for injection
    D.8.4Route of administration of the placeboIntravenous bolus use (Noncurrent)
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Friedreich's Ataxia (FRDA)
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 12.0
    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
    - To evaluate the safety and tolerability of 2 weeks treatment with Lu AA24493 in patients with FRDA
    E.2.2Secondary objectives of the trial
    - To explore biomarkers of efficacy (frataxin, 8-OHdG, peroxides, malondialdehyde)
    - To explore efficacy by neurological assessment (Scale for the Assessment and Rating of Ataxia (SARA), Friedreich’s Ataxia Rating Scale (FARS))
    - To explore efficacy by the Clinical Global Impression scales(CGI-I/S)
    - To explore population pharmacokinetic parameters of Lu AA24493
    - To evaluate the immunogenicity of Lu AA24493
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.The patient is able to read and understand the Patient Information Sheet.
    2.The patient or their representative has signed the Informed Consent Form
    3.The patient has FRDA, diagnosed according to ICD-10, and with genetic test demonstrating >400 GAA nucleotide triplet repeats on the shorter of the two frataxin alleles
    4.The patient has a SARA (Stance) sub-score of <=6
    5.The patient has a SARA (Gait) sub-score of <=6
    6.The patient is a man or woman, aged 18 years or over
    7.The patient, if female, must:
    -agree not to try to become pregnant during the study, AND
    -use adequate contraception (adequate contraception is defined as oral/systemic contraception, intrauterine device, diaphragm in combination with spermicide, or condom for male partner in combination with spermicide), OR
    -have had her last natural menstruation at least 24 months prior to screening, OR
    -have been surgically sterilised prior to screening, OR
    -have had a hysterectomy prior to screening, OR
    -not be sexually active
    E.4Principal exclusion criteria
    Co-morbidity:
    1.The patient has a clinically significant unstable illness, for example, hepatic or renal insufficiency, or a cardiovascular, pulmonary, gastrointestinal, endocrine, neurological (unrelated to the clinical manifestations of FRDA), infectious, neoplastic, or metabolic disturbance. Cardiac manifestations of FRDA (such as hypertrophic cardiomyopathy) are acceptable provided that the cardiac illness is considered stable and unlikely to compromise the patient's safety during the study.
    2.For patients previously exposed to EPO the patient has antibodies to EPO (established from assessment at screening, Visit 1)
    3.The patient has or has had polycythemia, venous thromboembolism or other recent vascular event.
    4.The patient has anaemia as determined by a haemoglobin concentration lower than 0.6mM (1g/dl) below local, gender adjusted, normal range.
    5.The patient has a disease or takes medication that, in the opinion of the investigator, could interfere with the assessments of safety, tolerability, or efficacy.
    Medication and Treatment:
    6.The patient used/uses disallowed recent or concomitant medication or it is anticipated that the patient will require treatment with at least one of the disallowed concomitant medications during the study.
    7.The patient has been treated with idebenone within 6 weeks prior to screening.
    8.The patient has been treated with erythropoietin or erythropoietin analogues within 16 weeks prior to screening.
    9.The patient has been treated with any investigational medicinal product within 30 days or 5 half lives (whichever is longer) prior to screening.
    Patient characteristics:
    10.The patient has a clinically significant abnormal ECG. Changes in ECG associated with cardiac manifestations of FRDA (such as hypertrophic cardiomyopathy) are acceptable provided that the cardiac illness is considered stable and unlikely to compromise the patient's safety during the study.
    11.The patient has clinically significant abnormal vital signs not relating to FRDA symptoms.
    12.The patient has one or more laboratory values outside the normal range, based on the blood or urine samples taken at the Screening Visit or Baseline Visit, that are considered by the investigator to be clinically significant.
    13.The patient has/has had a disorder related to alcohol or drug abuse, as defined in DSM IV TR, within 6 months prior to screening.
    14.The patient has a history of severe drug allergy or hypersensitivity, or known hypersensitivity to EPO.
    Patient status:
    15.The patient is pregnant or breast-feeding.
    16.The patient, in the opinion of the investigator, is unlikely to comply with the clinical study protocol or is unsuitable for any reason.
    17.The patient is a member of the site personnel or their immediate families.
    18.The patient has previously participated in this study.
    E.5 End points
    E.5.1Primary end point(s)
    As the efficacy analyses will be exploratory, no primary endpoint will be defined.
    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 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) 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 Information not present in EudraCT
    E.8.4 The trial involves multiple sites in the Member State concerned Information not present in EudraCT
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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 Information not present in EudraCT
    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
    The end of the study for an individual patient is defined as the last protocol-specified contact with that patient. The overall end of the study is defined as the last protocol-specified contact with the last patient ongoing in the study (study protocol section 7.5).
    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 months0
    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 months0
    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 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.1.3Elderly (>=65 years) Yes
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 36
    F.4.2.2In the whole clinical trial 36
    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)
    After withdrawal from the study or completion of the study, the patient should be treated according to normal clinical practice (study protocol section 7.5).
    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 Decision2010-01-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2011-03-11
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