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    Summary
    EudraCT Number:2011-004667-76
    Sponsor's Protocol Code Number:Cardoz-004
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-10-21
    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 ConcernedSweden - MPA
    A.2EudraCT number2011-004667-76
    A.3Full title of the trial
    An open-label, un-controlled, single-centre trial investigating the efficacy and safety of CRD007 tablets administered twice daily for 12 weeks in children with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or children being symptomatic carriers for DMD or BMD.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An open single center study investigating the effects and side-effects of CRD007 in children with Duchenne Muscular Dystrophy (DMD -an inherited disorder which results in muscle degeneration) or Becker Muscular Dystrophy (BMD- an inherited disorder characterized by slowly progressive muscle weakness of the legs and pelvis) or children being symptomatic carriers for DMD or BMD.
    A.3.2Name or abbreviated title of the trial where available
    CRD007 for the treatment of DMD, BMD and symptomatic carriers
    A.4.1Sponsor's protocol code numberCardoz-004
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCardoz AB
    B.1.3.4CountrySweden
    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 supportCardoz AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCardoz AB
    B.5.2Functional name of contact pointClinical trial information desk
    B.5.3 Address:
    B.5.3.1Street AddressKornhamnstorg 53
    B.5.3.2Town/ cityStockholm
    B.5.3.3Post codeSE-11127
    B.5.3.4CountrySweden
    B.5.4Telephone number+46 70 975 98 63
    B.5.5Fax number+46 8566 300 13
    B.5.6E-mailcarl-johan.dalsgaard@ofco.se
    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 namepemirolast
    D.3.2Product code CRD007
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    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 INNPEMIROLAST
    D.3.9.1CAS number 69372-19-6
    D.3.9.2Current sponsor codeCRD007
    D.3.9.4EV Substance CodeSUB09656MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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) No
    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 No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    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 No
    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
    Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers for DMD or BMD
    E.1.1.1Medical condition in easily understood language
    muscular dystrophy
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10059117
    E.1.2Term Becker's muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10052655
    E.1.2Term Duchenne muscular dystrophy gene carrier
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the ability of CRD007 to decrease creatine kinase activity levels in subjects with DMD or BMD and in subjects being carriers for DMD or BMD
    E.2.2Secondary objectives of the trial
    -To assess the ability of CRD007 to decrease the level of a range of other disease related biomarkers

    -To assess the ability of CRD007 to improve the outcome of functional tests

    -To assess the safety of CRD007

    - To assess the safety margins of CRD007 in relation to plasma concentrations of pemirolast observed in clinical and toxicological studies
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Documented diagnosis of dystrophinopathy in the form of mutation analysis in the dystrophin gene

    2.Clinically documented delayed motor skills and muscle weakness

    3.Prior documented elevated creatine kinase (>5 x ULN)

    4.Age <12 years (females in the pre-pubertal stage as defined by Tanner criterion I)

    5.Body weight ≥14 kg

    6.Informed assent from subject before any trial related activity is carried out

    7.Written informed consent obtained from parent(s) / legal guardian(s) before any trial related activity is carried out


    E.4Principal exclusion criteria
    1.Severe functional impairment (e.g. non-walkers) resulting in inability to perform meaningful repeated assessment of functional capabilities, according to the investigator’s judgment
    2.Moderate or severe hepatic impairment or severe renal impairment, according to the investigator’s judgment
    3.Prior or ongoing medical condition or laboratory abnormality that in the investigator’s opinion could adversely affect the safety of the subject.
    4.Subject or parent(s) / legal guardian(s) not willing and able to comply with the requirements of the study
    5.Subject not able to follow trial procedures, according to the investigator’s judgement
    6.Known individual hypersensitivity to any of the ingredients/excipients of the study medication (see Section 7.1.1)
    7. Applicable for part B, only: Systemic glucocorticoid therapy within 30 days prior to start of IMP treatment
    8.Planned systemic glucocorticoid therapy during the trial period
    9.Previous exposure to idebenone
    10.Participation in any other therapeutic trial and/or intake of any investigational drug within 90 days prior to Visit 1
    E.5 End points
    E.5.1Primary end point(s)
    The relative change in creatine kinase from enrolment (Visit 1) to 12 weeks of treatment
    (Visit 5)

    E.5.1.1Timepoint(s) of evaluation of this end point
    From enrolment (Visit 1) to 12 weeks of treatment (Visit 5)

    E.5.2Secondary end point(s)
    •The relative change in other disease related biomarkers between baseline and 12 weeks of treatment
    a. Myoglobin
    b. miR-1, miR-133, miR-206
    •The relative change in the total North Star Ambulatory Assessment score
    •The relative change in the time needed to perform "Rise from floor", "Run (10 m)", "Climb 4 steps"
    Change in safety related assessments between baseline and 12 weeks
    a. Blood pressure (diastolic and systolic) and pulse
    b. Electrocardiogram
    c. Biochemistry laboratory values
    d. Hematology laboratory values
    •Incidence of adverse events by type and severity
    •Cpl(CRD007) and derived pharmacokinetic parameters
    E.5.2.1Timepoint(s) of evaluation of this end point
    •Myoglobin: at baseline and 12 weeks of treatment

    •miR-1, miR-133, miR-206:at baseline and 12 weeks of treatment

    •The relative change in the total North Star Ambulatory Assessment score:at baseline and 12 weeks of treatment
    •The relative change in the time needed to perform "Rise from floor", "Run (10 m)", "Climb 4 steps": at baseline and 12 weeks of treatment
    •Change in safety related assessments : at baseline, after 6 and 12 weeks of treatment
    •Incidence of adverse events: at baseline and once every week and 2 weeks after end of treatment
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    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 No
    E.8.7Trial has a data monitoring committee No
    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 trial is defined as the date of last subject last visit.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 21
    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.1.1.5.1Number of subjects for this age range: 21
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    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 No
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    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)
    IMP will not be available to the subject after the completion of Visit 2 (PartA) or Visit 5 (Part B). The investigator will notify the subject's treating physician in Sweden, Denmark or Norway about time of treatment completion for the individual subject and advice the treating physician to ensure other treatment according to local clinical practice.
    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 Decision2011-12-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-11-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2012-09-28
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