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    Summary
    EudraCT Number:2016-001967-35
    Sponsor's Protocol Code Number:CALCIDEB2016
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-06-28
    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 ConcernedAustria - BASG
    A.2EudraCT number2016-001967-35
    A.3Full title of the trial
    A double-blind, placebo-controlled cross-over study to assess the efficacy of topical calcipotriol (Psorcutan®-ointment containing 0.05 µg/g calcipotriol) to improve wound healing in dystrophic epidermolysis bullosa (DEB)
    Doppel-blinde, Placebo-kontrollierte Crossover-Studie zur Beurteilung der Effizienz von topischem Calcipotriol (Psorcutan®-Salbe mit 0.05 µg/g Calcipotriol) in der Verbesserung der Wundheilung in dystropher Epidermolysis bullosa (DEB)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the efficacy of a vitamin D-containing ointment applied on the skin to improve wound healing in "butterfly children"
    Eine Studie zur Beurteilung der Effizienz einer auf die Haut aufgetragenen Vitamin D-hältigen Salbe in der Verbesserung der Wundheilung bei "Schmetterlingskindern"
    A.3.2Name or abbreviated title of the trial where available
    Topical calcipotriol in DEB
    Topisches Calcipotriol in DEB
    A.4.1Sponsor's protocol code numberCALCIDEB2016
    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 SponsorUniversitätsklinik für Dermatologie, Paracelsus medizinische Privatuniversität Salzburg
    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 supportEB-Haus Austria
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversitätslinik für Dermatologie
    B.5.2Functional name of contact pointEB-Haus Austria
    B.5.3 Address:
    B.5.3.1Street AddressMüllner-Hauptstrasse 48
    B.5.3.2Town/ citySalzburg
    B.5.3.3Post code5020
    B.5.3.4CountryAustria
    B.5.6E-mailinfo@eb-haus.org
    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 Yes
    D.2.1.1.1Trade name Psorcutan-Ointment
    D.2.1.1.2Name of the Marketing Authorisation holderLEO Pharma Gesellschaft m.b.H
    D.2.1.2Country which granted the Marketing AuthorisationAustria
    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 Ointment
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTopical use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCALCIPOTRIOL
    D.3.9.1CAS number 112828-00-9
    D.3.9.4EV Substance CodeSUB06046MIG
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.05
    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 placeboOintment
    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
    Subjects with dystrophic epidermolysis bullosa (DEB) with a known mutation in the type VII collagen gene.
    Patienten mit dystropher Epidermolysis bullosa (DEB) mit einer bekannten Mutation im Kollagen Typ-VII Gen
    E.1.1.1Medical condition in easily understood language
    "Butterfly children" with a known mutation in a certain structural protein of the skin
    "Schmetterlingskinder" mit einer bekannten Mutation in einem bestimmten Strukturprotein der Haut
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10014989
    E.1.2Term Epidermolysis bullosa
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To establish the superiority of topical calcipotriol (Psorcutan®-ointment containing 0.05 µg/g calcipotriol) therapy over placebo with respect to improved wound-healing (40% reduction of wound area in the treatment group compared to placebo after 4 weeks of treatment) for subjects with DEB.
    Erbringung des Nachweises der Überlegenheit der Therapie mit topischem Calcipotriol (Psorcutan®-Salbe mit 0.05 µg/g Calcipotriol) im Vergleich mit Placebo hinsichtlich Verbesserung der Wundheilung (40-prozentige Reduktion der Wundfläche im Vergleich mit Placebo nach 4-wöchiger Behandlungsdauer) in Patienten mit DEB.
    E.2.2Secondary objectives of the trial
    To evaluate and compare topical calcipotriol (Psorcutan®-ointment containing 0.05 µg/g calcipotriol) versus placebo with respect to bacterial wound colonization and with respect to pruritus/pain and to characterize the safety of topical calcipotriol (Psorcutan®-ointment containing 0.05 µg/g calcipotriol) therapy.
    Vergleich der Therapie mit topischem Calcipotriol (Psorcutan®-Salbe mit 0.05 µg/g Calcipotriol) mit Placebo hinsichtlich bakterieller Wundbesiedelung und Juckreiz/Schmerz sowie Charakterisierung der Sicherheit von topischem Calcipotriol (Psorcutan®-Salbe mit 0.05 µg/g Calcipotriol)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed Written Informed Consent
    a) Subjects or legal guardians for minor children must have signed and dated an IRB/IEC approved written informed consent form in accordance with regulatory and institutional guidelines. This must be obtained before the performance of any protocol related procedures that are not part of normal subject care.

    b) Subjects must be willing and able to comply with scheduled visits, treatment schedule, laboratory testing, and other requirements of the study.

    2. Target Population
    a) Subjects with dystrophic epidermolysis bullosa (DEB) with a known mutation in the type VII collagen gene.

    b) Subjects must have at least 2 wounds with a minimum size of 6 cm2 per wound.

    3. Age and Reproductive Status
    a) Children ≥ 6 years, men and women.

    b) Pregnant women are excluded from the study.

    c) Women of childbearing potential (not surgically sterilized and between menarche and 1 year postmenopause) must agree to use appropriate medically accepted highly effective methods of contraception (failure rate of less than 1% per year). Medically accepted highly effective methods of contraception are: 3-monthly contraceptive injection, combined oral hormonal contraceptive pills containing ethinyl estradiol and progestogens, oral hormonal contraception with Cerazette®, vaginal ring (e.g. NuvaRing®), contraceptive transdermal patch (e.g. Evra®), contraceptive implant (e.g. Implanon®) and gestagen-containing intrauterine device.
    Negative Serum ß-Human Chorionic Gonadotrophin (ß-HCG) pregnancy test at screening and crossover.

    d) Surgically sterilized female patients with documentation of prior
    hysterectomy, tubal ligation or complete bilateral oophorectomy and postmenopausal women do not require contraception.

    e) Women must not be breastfeeding.
    1. Unterzeichnete Einverständniserklärung
    Patienten oder gesetzliche Vertreter bei Minderjährigen müssen eine von der Ethikkommission genehmigte Einverständniserklärung vor der Durchführung von über die normale Patientenbetreuung hinausgehenden Protokoll-bezogenen Untersuchungen unterzeichnet und datiert haben.

    2.Zielpopulation
    a) Patienten mit dystropher Epidermolysis bullosa (DEB) mit einer bekannten Mutation im Kollagen Typ VII-Gen.

    b) Patienten müssen zumindest 2 Wunden mit einer Minimalgröße von 6 cm2 pro Wunde aufweisen.

    3. Alter und Reproduktionsstatus
    a) Kinder ≥ 6 Jahre, Männer und Frauen.

    b) Schwangere sind von der Teilnahme an der Studie ausgeschlossen.

    c) Gebärfähige Frauen (nicht operativ sterilisiert und zwischen Menarche und einem Jahr nach der Menopause) müssen der Verwendung einer geeigneten, medizinisch anerkannten, hochwirksamen Methode der Empfängnisverhütung (Fehlerrate < 1% pro Jahr) zustimmen. Medizinisch anerkannte, hochwirksame Methoden der Empfängnisverhütung sind: 3-Monatsspritze, orale hormonelle Kombinationspräparate mit Östrogen und Gestagen, orale hormonelle Verhütung mit Cerazette®, Vaginalring (z.B. NuvaRing®), Transdermalpflaster (z.B. Evra®), Hormonimplantat (z.B. Implanon®) und Gestagen-hältige Hormonspiralen.
    Negativer Serum ß-Human Chorionic Gonadotrophin (ß-HCG) Schwangerschaftstest bei der Screening- und Cross-over Visite.

    c) Operativ sterilisierte weibliche Patienten mit dokumentierter Hysterektomie, Tubenligatur, oder bilateraler Oophorektomie sowie Frauen in der Postmenopause benötigen keine Empfängnisverhütung.

    d) Stillende Mütter sind ausgeschlossen.
    E.4Principal exclusion criteria
    1. Medical History and Concurrent Diseases
    a) Simultaneous participation in another clinical trial.

    b) Any serious or uncontrolled medical disorder that, in the opinion of the investigator, may impair the ability of the subject to receive protocol therapy, or interfere with the interpretation of study results.

    c) Subjects with a condition requiring systemic treatment with either corticosteroids (>10 mg daily prednisone equivalents) or other immunosuppressive medications or antibiotics within14 days of study drug administration.

    d) Subjects with known severely impaired kidney-or liver function.

    e) Subjects with known disorders of calcium metabolism.

    2. Allergies and Adverse Drug Reactions
    a) History of a severe hypersensitivity reaction to calcipotriol or other study drug components.

    3. Sex and Reproductive Status
    a) Women of childbearing potential who are pregnant or breastfeeding.

    b) Women with a positive pregnancy test at screening or crossover.
    1. Medizinische Vorgeschichte und Begleiterkrankungen
    a) Gleichzeitige Teilnahme an anderen klinischen Studien.

    b) Schwerwiegende oder unkontrollierte medizinische Funktionsstörungen, die nach Ansicht des Studienarztes die Fähigkeit des Patienten beeinflussen könnten, Protokoll-gemäße Therapie zu erhalten, oder Auswirkungen auf die Interpretation der Studienergebnisse haben könnten.

    c) Patienten, welche einer systemischen Behandlung mit Kortikosteroiden ( >10 mg Prednisolon-Äquivalent pro Tag), anderer immunsuppressiver Medikation oder Antibiotika innerhalb von 14 Tagen vor Gabe der Studienmedikation bedürfen.

    d) Patienten mit bekannter stark eingeschränkter Nieren-oder Leberfunktion.

    e) Patienten mit bekannten Funktionsstörungen des Calciumstoffwechsels.

    2. Allergien und Arzneimittelnebenwirkungen
    a) Bekannte schwere Unverträglichkeitsreaktionen auf Calcipotriol oder andere Bestandteile der Studienmedikation.

    3. Geschlecht und Reproduktionsstatus
    a) Schwangere oder stillende gebärfähige Frauen.

    b) Frauen mit einem positiven Schwangerschaftstest bei der Screening- oder Crossover-Visite.
    E.5 End points
    E.5.1Primary end point(s)
    40% reduction of wound area in the treatment group compared to placebo after 4 weeks of treatment.
    Reduktion der Wundfläche in der Behandlungsgruppe um 40% im Vergleich mit Placebo nach 4-wöchiger Behandlung.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At screening (V1) and cross-over (V4) 2 target wounds (minimum size of 6 cm2 per wound) should be identified and photographically documented using a square grid.
    Target wounds should also be photographically documented using a square grid at visits V2 (2 weeks after initiation of treatment), V3 (4 weeks after initiation of treatment), V5 (2 weeks after initiation of treatment after cross-over) and V6 (4 weeks after initiation of treatment after cross-over) and evaluated for changes in wound area.
    Bei der Screening-Visite (V1) und Crossover-Visite (V4) sollen jeweils 2 Zielwunden (Minimalgröße von 6 cm2 pro Wunde) identifiziert und unter Verwendung eines Quadratgitters photographisch dokumentiert werden. Die Zielwunden sollen unter Verwendung eines Quadratgitters bei den Visiten V2 (2 Wochen nach Behandlungsstart), V3 (4 Wochen nach Behandlungsstart) , V5 (2 Wochen nach Behandlungsstart nach Crossover) und V6 (4 Wochen nach Behandlungsstart nach Crossover) photographisch dokumentiert und hinsichtlich Änderungen in der Wundfläche evaluiert werden.
    E.5.2Secondary end point(s)
    Evalution of bacterial wound colonization of the 2 target wound areas in the treatment group compared to placebo after 4 weeks of treatment.
    Evaluation of pruritus/pain of the 2 target wounds in the treatment group compared to placebo after 4 weeks of treatment.
    Characterization of the safety of topical calcipotriol therapy after 4 weeks of treatment.
    Evaluierung der bakteriellen Besiedlung der 2 Zielwunden in der Behandlungsgruppe im Vergleich mit Placebo nach 4-wöchiger Behandlung.
    Evaluierung von Pruritus/Schmerz der 2 Zielwunden in der Behandlungsgruppe im Vergleich mit Placebo nach 4-wöchiger Behandlung.
    Charakterisierung der Sicherheit einer 4-wöchigen topischen Therapie mit Calcipotriol.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Bacterial swabs will be taken from the 2 target wounds, itch and pain of the 2 target wounds will be evaluated by using a visual analogue scale ranging from 0 (no itch/pain) to 10 (maximum itch/pain) as well as the use of itch/pain medication including drug name, dose, frequency and route of administration will be recorded at each study visit.
    V1 (Screening visit)
    V2 (2 weeks after initiation of treatment)
    V3 (4 weeks after initiation of treatment)
    V4 (Cross-over visit)
    V5 (2 weeks after initiation of treatment after cross-over)
    V6 (4 weeks after initiation of treatment after cross-over)

    Adverse events will be documented at V2, V3 , V5 and V6.
    Es werden bei allen Studienvisiten sowohl bakterielle Abstriche von den 2 Zielwunden entnommen, als auch Pruritus und Schmerz anhand einer visuellen Analogskala von 0 (kein Pruritus/Schmerz) bis 10 (maximaler Pruritus/Schmerz) und der Verbrauch von Pruritus- bzw. Schmerzmedikation inklusive Name der Medikation, Dosis, Frequenz und Verabreichungsform dokumentiert.
    V1 (Screening Visite)
    V2 (2 Wochen nach Behandlungsstart)
    V3 (4 Wochen nach Behandlungsstart)
    V4 (Crossover-Visite)
    V5 (2 Wochen nach Behandlungsstart nach Crossover)
    V6 (4 Wochen nach Behandlungsstart nach Crossover).
    Nebenwirkungen werden bei den Visiten V2, V3, V5 und V6 dokumentiert.
    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) 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 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 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 study is defined as the last visit of the last patient.
    Das Studienende ist als letzte Visite des letzten Patienten definiert.
    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 months4
    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: 6
    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: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 9
    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 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)
    None
    Keine
    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 Decision2016-07-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-07-11
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2018-09-26
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