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    Summary
    EudraCT Number:2014-004500-30
    Sponsor's Protocol Code Number:ADSTEM001
    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:2014-12-22
    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 number2014-004500-30
    A.3Full title of the trial
    A phase I/II study evaluating allogeneic mesenchymal stromal cells in adults 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 the use of mesenchymal stromal (stem) cells for the treatment of skin disease in adults with recessive dystrophic epidermolysis bullosa
    A.3.2Name or abbreviated title of the trial where available
    ADSTEM
    A.4.1Sponsor's protocol code numberADSTEM001
    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.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGuy's and St Thomas NHS Foundation Trust and King's College London
    B.5.2Functional name of contact pointProfessor John A. McGrath
    B.5.3 Address:
    B.5.3.1Street AddressSt John’s Institute of Dermatology, F9, Guy's Hospital
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeSE1 9RT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number44207188 6409
    B.5.5Fax number44207188 8050
    B.5.6E-mailjohn.mcgrath@kcl.ac.uk
    B.Sponsor: 2
    B.1.1Name of SponsorGuy's and St Thomas NHS Foundation Trust
    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 support Dystrophic Epidermolysis Bullosa Research Association
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGuy's and St Thomas NHS Foundation Trust
    B.5.2Functional name of contact pointProfessor John A. McGrath
    B.5.3 Address:
    B.5.3.1Street AddressSt John’s Institute of Dermatology, F9, Guy's Hospital
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeSE1 9RT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number44207188 6409
    B.5.5Fax number44207188 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 nameAllogeneic 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
    Severe inherited blistering disease
    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
    To evaluate the safety of allogeneic intravenously administered MSCs in adults with RDEB over a 8 or a 12-month period.
    E.2.2Secondary objectives of the trial
    1. Presence of new type VII collagen at the dermal-epidermal junction post treatment
    2. Changes in general markers of inflammation
    3.Changes in specific markers of inflammation using ELISA and LUMINEX platforms
    4.Changes in the clinical appearance of the skin
    5.Changes in BEBSS and EBDASI scores
    6.Change in Quality of Life Score using the QOLEB questionnaire.
    7.Change in pruritus score using the Leuven Itch Scale (LIS)
    8. Quantification of total blister numbers over the entire body surface area.
    9.Increase in the skin strength measured by time to blister formation after negative pressure skin suction test
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Individuals with a diagnosis of RDEB confirmed by DNA analysis .
    2) Individuals ≥ 18 years and ≤ 65 years of age, both male and female
    3) Individuals that have voluntarily signed and dated an informed consent form (ICF) prior to the first study intervention
    E.4Principal exclusion criteria
    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).
    3) Subjects with a known allergy to any of the constituents of the investigational product.
    4) Subjects with a medical history or evidence of malignancy, including cutaneous squamous cell carcinoma.
    5) 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.
    6) Subjects with both a) positive C7 ELISA and b) a positive indirect immunofluorescence (IIF) with binding to the base of salt split skin.
    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 8 or a 12 month period. SAEs are defined as any occurrence related to the administration of the IMP that results in death, or is life threatening or requires hospitalization or prolongation of existing hospitalization.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At every visit over a 12 month period for first 8 subjects and over 8 months for the final two subjects.
    E.5.2Secondary end point(s)
    1. Presence of new type VII collagen at the dermal-epidermal junction post treatment on Day 28, Day 60, and Month 6.
    2. Changes in general markers of inflammation at Day 14, Day 28, Day 60, Day 100, Month 6 (for all patients) and Month 12 (for the first eight eligible patients) or Month 8 (for the last two eligible patients) compared to baseline.
    3. Changes in specific markers of inflammation on Day 14, Day 28, Day 60 and Month 6 compared to baseline using ELISA and LUMINEX platforms. Specific inflammatory markers include: HMGB-1, TNF α, IFN γ, IL-17A, IL1 β, IL-10, MMP-2, MMP-9, MMP-11 and TIMP-1.
    4. Changes in the clinical appearance of the skin assessed with clinical photographs.
    5. Differences in quality of life data at Day 28, Day 60, Day 100, Month 6 (for all patients) and Month 12 (for the first eight eligible patients) or Month 8 (for the last two eligible patients) compared to baseline.
    6. Changes in BEBSS and EBDASI scores at Day 28, Day 60, Day 100, Month 6 (for all patients) and Month 12 (for the first eight eligible patients) or Month 8 (for the last two eligible patients) compared to baseline. 7. Change in pruritus score using the Leuven Itch Scale (LIS) at Day 28, Day 60, Day 100, Month 6 (for all patients) and Month 12 (for the first eight eligible patients) or Month 8 (for the last two eligible patients) compared to baseline.
    8. Quantification of total blister numbers over the entire body surface area at Day 28, Day 60, Day 100, Month 6 (for all patients) and Month 12 (for the first eight eligible patients) or Month 8 (for the last two eligible patients) compared to baseline.
    9. Increase in the skin strength measured by time to blister formation after negative pressure skin suction test at Day 28, Day 60, Day 100, Month 6 (for all patients) and Month 12 (for the first eight eligible patients) or Month 8 (for the last two eligible patients) compared to baseline.
    E.5.2.1Timepoint(s) of evaluation of this end point
    As above
    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
    Evidence of potential benefit to trial subjects has been demonstrated.
    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.8.5The 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
    Last study subject‟s last scheduled follow-up visit according to the protocol, which will be the 12 month follow-up after the first MSC infusion of the first eight eligible subjects entering the trial,or the 8 month follow-up of the night or tenth eligible patient, whichever comes last.
    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 months3
    E.8.9.1In the Member State concerned days1
    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.1.2.1Number of subjects for this age range: 10
    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 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)
    If the administration of MSCs proves to be safe in adults, we would like to offer this treatment as a "special" on
    compassionate grounds
    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 Decision2015-02-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-01-14
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-07-12
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