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    Summary
    EudraCT Number:2013-002034-21
    Sponsor's Protocol Code Number:Dia2014
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-08-01
    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 ConcernedAustria - BASG
    A.2EudraCT number2013-002034-21
    A.3Full title of the trial
    Diacerein for the treatment of Epidermolysis bullosa simplex
    Diacerein für die Behandlung von Epidermolysis bullosa simplex
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Diacerein for butterfly children
    Diacerein für Schmetterlingskinder
    A.3.2Name or abbreviated title of the trial where available
    Diacerein for EBS
    Diacerein bei EBS
    A.4.1Sponsor's protocol code numberDia2014
    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 SponsorEB House Austria
    B.1.3.4CountryAustria
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportDEBRA Austria
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEB House Austria
    B.5.2Functional name of contact pointEB House Austria
    B.5.3 Address:
    B.5.3.1Street AddressMüllner Hauptstraße 48
    B.5.3.2Town/ citySalzburg
    B.5.3.4CountryAustria
    B.5.4Telephone number+4366244823110
    B.5.5Fax number+4366244823125
    B.5.6E-mailjo.bauer@salk.at
    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 nameSimpleCare
    D.3.4Pharmaceutical form Cream
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTopical use (Noncurrent)
    Cutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDIACEREIN
    D.3.9.1CAS number 13739-02-1
    D.3.9.4EV Substance CodeSUB07060MIG
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCream
    D.8.4Route of administration of the placeboTopical use (Noncurrent)
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Epidermolysis bullosa simplex - type Dowling-Meara
    Epdiermolysis bullosa simplex - Typ Dowling-Meara
    E.1.1.1Medical condition in easily understood language
    Butterfly children
    Schmetterlingskinder
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    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
    Reduction of blister numbers on the treated body surface by 40% in the diacerein group in comparison to placebo within 4 weeks.
    Reduktion der Blasenanzahl an der behandelten Körperfläche um 40% im Vergleich zu Placebo innerhalb von 4 Wochen.
    E.2.2Secondary objectives of the trial
    Time until the reaching of the initial blister number (+/-10%) during the follow-up phase.
    Zeitspanne bis zum Wiedererreichen der initialen Blasenzahl (+/-10%) während der Follow-up Phase.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Epidermolysis bullos simplex - type Dowling-Meara
    - Mutations in either the keratin 14 or the keratin 5 gene, leading to protein aggregation
    - Age: 6-19
    - Epidermolysis bullosa simplex - Typ Dowling-Meara
    - Mutationen im Keratin 14 oder Keratin 5 Gen, die zur Aggregatbildung führen.
    - Alter: 6-19 Jahre
    E.4Principal exclusion criteria
    - Other types of EB with mutations in the keratin 14 or teh keratin 5 gene.
    - participation in another clinical trial at the same time
    - known intolerance against diacerein, adjuvants (especially Tartrazine) or other substances with similar structures
    - pregnancy or lactation
    - relevant other illnesses (acute infections, ...)
    - patients with impaired liver functions
    - Andere Formen von EB mit Mutationen in Keratin 14 oder Keratin 5.
    - Gleichzeitige Teilnahme an einer anderen klinischen Studie
    -Bekannte oder vermutete Unverträglichkeiten gegen Diacerein, Hilfsstoffe des Prüfpräparats (insbesondere Tartrazin) oder Substanzen mit ähnlicher Struktur.
    - Schwangerschaft oder Stillzeit (siehe 7.2.2.1)
    - Relevante andere Erkrankungen (akute Infektionen, …)
    - PatientInnen mit schweren Leberfunktionsstörungen
    E.5 End points
    E.5.1Primary end point(s)
    Reduction of blister numbers in the treated area by 40% in comparison to placebo
    Reduktion der Blasenanzahl an den behandelten Körperarealen um 40% im vergleich zu Placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 4 weeks
    Nach 4 Wochen
    E.5.2Secondary end point(s)
    Time span until reaching the initial blister number (+/- 10%).
    Zeitspanne bis zum Erreichen der initialen Blasenzahl (+/- 10%).
    E.5.2.1Timepoint(s) of evaluation of this end point
    After 16 weeks.
    Nach 16 Wochen.
    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 Yes
    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 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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Yes
    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.8.2.4Number of treatment arms in the trial2
    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 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 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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 14
    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: 7
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 7
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 1
    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
    Children under 18 with parent's consent.
    Children from 8 on with additional personal consent.
    Kinder unter 18 mit dem Einverständnis der Eltern bzw. Erziehungsberechtigten.
    Kinder ab 8 zusätzlich mit dem eigenen Einverständnis.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    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)
    Usual care. Normal standard treatment.
    Usual care. Normale Standardbehandlung.
    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 Decision2013-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-05-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-09-20
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