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    Summary
    EudraCT Number:2012-001394-87
    Sponsor's Protocol Code Number:EBSTEM001
    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:2012-08-22
    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 ConcernedUK - MHRA
    A.2EudraCT number2012-001394-87
    A.3Full title of the trial
    A prospective phase I/II study to evaluate allogeneic mesenchymal stromal cells for the treatment of skin disease in children with recessive dystrophic epidermolysis bullosa.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A prospective phase I/II study to evaluate allogeneic mesenchymal stromal cells for the treatment of skin disease in children with recessive dystrophic epidermolysis bullosa.
    A.3.2Name or abbreviated title of the trial where available
    EBSTEM
    A.4.1Sponsor's protocol code numberEBSTEM001
    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 SponsorKing's College London
    B.1.3.4CountryUnited Kingdom
    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 supportDystrophic Epidermolysis Bullosa Research Association
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationProf. John A. McGrath
    B.5.2Functional name of contact pointDept of Genetics and Molecular Med.
    B.5.3 Address:
    B.5.3.1Street Address9th Floor Tower Wing, Guy’s Hospital, Great Maze Pond
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeSE1 9RT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number00440207188 6409
    B.5.5Fax number00440207188 8050
    B.5.6E-mailjohn.mcgrath@kcl.ac.uk
    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 nameAllogenic mesenchymal stromal cells
    D.3.2Product code TC-MSC
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    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
    Recessive Dystrophic Epidermolysis Bullosa
    E.1.1.1Medical condition in easily understood language
    Recessive Dystrophic Epidermolysis Bullosa
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    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 evaluate the safety of allogeneic intravenously administered MSCs in children with RDEB over a 24-month period.
    E.2.2Secondary objectives of the trial
    • Incidence of infusional toxicity.
    • Increase in C7 deposition at the DEJ post treatment at D0 and D60.
    • Quantitative analysis of the donor cells chimerism at D60.
    • Improvement of haematological and serological markers of generalised inflammation at D0, D7, D28, D60 and D180 compared to baseline.
    • Improvement in the clinical appearances of the skin.
    • Improved quality of life according to validated paediatric QoL scoring systems at screening, D60, D100 and D180.
    • Pain scoring at screening, D0, D7, D28, D60, D100 and D180.
    • Reduction in blister occurrence over entire body surface at D0, D7, D28, D60, D100 and D180 as compared to baseline.
    • Increase in skin strength measured by time to blister formation after skin suction at screening and D100.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Subjects who have a diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) characterised by partial or complete C7 deficiency.
    2) Subjects who are ≥ 12 months and ≤ 17 years of age at the time of enrolment.
    3) Subjects whose responsible parent/guardian has voluntarily signed and dated an Informed Consent Form (ICF) prior to the first study intervention. Whenever the minor child is able to give consent, the minor’s assent will be obtained in addition to the signed consent of the minor’s legal guardian.
    E.4Principal exclusion criteria
    Subjects will be excluded from the study if ANY of the following conditions exist:
    1) Subjects who have had other investigational medicinal products within 90 days prior to screening or during the treatment phase.
    2) Subjects who have received immunotherapy including oral corticosteroids for more than 1 week (intranasal and topical preparations are permitted) or chemotherapy within 60 days of enrolment into this study.
    3) Subjects with a known allergy to any of the constituents of the investigational product.
    4) Subjects with signs of active infection.
    5) Subjects with a medical history or evidence of malignancy, including cutaneous squamous cell carcinoma.
    6) Subjects with both a) positive C7 ELISA and b) a positive indirect immunofluorescence (IIF) with binding to the base of salt split skin.
    7) Subjects who are pregnant or of child-bearing potential who are not abstinent or practicing an acceptable means of contraception, as determined by the Investigator, for the duration of the treatment phase.
    E.5 End points
    E.5.1Primary end point(s)
    Lack of serious and severe adverse events (SAEs) related to the administration of the investigational medicinal product over a 12-month follow-up period. SAEs are defined as any occurrences related to the administration of the IMP that result in death, or is life-threatening or requires hospitalisation or prolongation of existing hospitalisation.
    E.5.1.1Timepoint(s) of evaluation of this end point
    at every visit up to 12-months after last IMP infusion
    E.5.2Secondary end point(s)
    •Incidence of infusional toxicity.
    •Increase in collagen VII deposition at the DEJ post treatment.
    •Quantitative analysis of the donor cells dermal chimerism.
    •Improvement of haematological and serological markers of generalised inflammation.
    •General clinical appearance of the skin based on medical photographs, generalised severity score and BEBSS score.
    •Improved quality of life.
    •Pain scoring.
    •Reduction in blister numbers.
    •Increase in skin strength measured by increased time to blister formation after skin suction
    E.5.2.1Timepoint(s) of evaluation of this end point
    •toxicity 1 hour post infusion
    •Increase in collagen VII deposition at the DEJ post treatment at Day 0 and D60
    •Quantitative analysis of the donor cells dermal chimerism at Day 60
    •Improvement of markers of generalised inflammation at D0, D7, D28, D60 and D180
    •General clinical appearance of the skin based on medical photographs on each visit
    •Improved quality of life according to validated paediatric QoL scoring systems at screening, D60, D100 and D180
    •Pain scoring at screening, D0, D7, D28, D60, D100 and D180
    •Reduction in blister occurrence over entire body surface at D0, D7, D28, D60, D100 and D180 as compared to baseline
    •Increase in skin strength measured by time to blister formation after skin suction at screening and D100
    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) 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
    There is evidence of potential benefit to proposed participants
    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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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
    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 months8
    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: 10
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 10
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    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 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
    minor subjects
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    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
    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 Decision2012-10-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-11-16
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-12-11
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