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    Clinical Trial Results:
    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.

    Summary
    EudraCT number
    		 2012-001394-87
    Trial protocol
    		 GB  
    Global end of trial date
    11 Dec 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Oct 2018
    First version publication date
    28 Oct 2018
    Other versions
    Summary report(s)
    		 EBSTEM Final Clinical Study Report

    Trial information

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    Trial identification
    Sponsor protocol code
    EBSTEM001
    Additional study identifiers
    ISRCTN number
    		 ISRCTN46615946
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    King's College London
    Sponsor organisation address
    The Strand, London, United Kingdom, WC2R 2LS
    Public contact
    Dept of Genetics and Molecular Med., Prof. John A. McGrath, 0044 0207188 6409, john.mcgrath@kcl.ac.uk
    Scientific contact
    Dept of Genetics and Molecular Med., Prof. John A. McGrath, 0044 0207188 6409, john.mcgrath@kcl.ac.uk
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    17 Dec 2014
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 Dec 2014
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Dec 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety of allogeneic intravenously administered MSCs in children with RDEB over a 24-month period.
    Protection of trial subjects
    The study subjects can continue to receive their regular medication(s). All IMP administration is completed in a specialist hospital environment.
    Background therapy
    		 N/A
    Evidence for comparator
    		 N/A
    Actual start date of recruitment
    04 Jul 2013
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 10
    Worldwide total number of subjects
    10
    EEA total number of subjects
    10
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    4
    Children (2-11 years)
    6
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Ten children were enrolled at Great Ormond Street Hospital (London, UK).

    Pre-assignment
    Screening details
    		 Eleven children with RDEB were screened for inclusion into the trial. One child was excluded because of both positive ELISA for C7 antibodies and positive indirect immunofluorescence microscopy (IIF) with binding of the antibodies to the dermal-epidermal junction (DEJ) within the base of salt-split skin.

    Period 1
    Period 1 title
    Whole Group (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    n/a

    Arms
    Arm title
    		 Full study
    Arm description
    		 Single Arm Study. All study participants will receive three intravenous MSC infusions at baseline Day 0, D7 and D28 and will be followed up for a 24-month period following the last infusion
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mesenchymal stromal cells
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for solution for infusion
    Routes of administration
    Intravenous drip use
    Dosage and administration details
    Third-party bone marrow-derived mesenchymal stromal cells administered by intravenous infusion on 3 occasions.

    Number of subjects in period 1
    		Full study
    Started
    10
    Completed
    10

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Whole Group
    Reporting group description
    		 -

    Reporting group values
    		 Whole Group Total
    Number of subjects
    		 10 10
    Age categorical
    Units: Subjects
        12months to 3 years
    		 3 3
        4 to 6 years
    		 3 3
        7 to 10years
    		 2 2
        11 to 14 years
    		 2 2
    Gender categorical
    Units: Subjects
        Female
    		 5 5
        Male
    		 5 5

    End points

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    End points reporting groups
    Reporting group title
    Full study
    Reporting group description
    		 Single Arm Study. All study participants will receive three intravenous MSC infusions at baseline Day 0, D7 and D28 and will be followed up for a 24-month period following the last infusion

    Primary: Primary safety endpoint

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    End point title
    		 Primary safety endpoint [1]
    End point description
    To evaluate the safety of allogeneic intravenously administered MSCs in children with RDEB over a 24-month period
    End point type
    Primary
    End point timeframe
    0 to 24 months
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Please see attached documents for results
    End point values
    		 Full study
    Number of subjects analysed
    10
    Units: whole
    10
    No statistical analyses for this end point

    Secondary: Secondary Efficacy Parameters

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    End point title
    		 Secondary Efficacy Parameters
    End point description
    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.
    End point type
    Secondary
    End point timeframe
    0 to day 180
    End point values
    		 Full study
    Number of subjects analysed
    10
    Units: whole
    10
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline to day 180
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Whole Trial
    Reporting group description
    		 -

    Serious adverse events
    		 Whole Trial
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 10 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Whole Trial
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    10 / 10 (100.00%)
    Injury, poisoning and procedural complications
    Accidental injuries
         subjects affected / exposed
    5 / 10 (50.00%)
         occurrences all number
    18
    Cardiac disorders
    Bradycardia
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Surgical and medical procedures
    Oesophageal dilatation
         subjects affected / exposed
    4 / 10 (40.00%)
         occurrences all number
    4
    Routine surgical procedure related to complications of EB
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Dental procedure
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Epistaxis
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Sore Throat
         subjects affected / exposed
    3 / 10 (30.00%)
         occurrences all number
    3
    Eye disorders
    Conjunctivitis
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Corneal abraision
         subjects affected / exposed
    4 / 10 (40.00%)
         occurrences all number
    20
    Sore eyes
         subjects affected / exposed
    3 / 10 (30.00%)
         occurrences all number
    3
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Reflux
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Constipation
         subjects affected / exposed
    2 / 10 (20.00%)
         occurrences all number
    2
    Diarrhea
         subjects affected / exposed
    5 / 10 (50.00%)
         occurrences all number
    9
    Increased appetite
         subjects affected / exposed
    2 / 10 (20.00%)
         occurrences all number
    2
    Nausea
         subjects affected / exposed
    2 / 10 (20.00%)
         occurrences all number
    3
    Vomiting
         subjects affected / exposed
    5 / 10 (50.00%)
         occurrences all number
    6
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 10 (30.00%)
         occurrences all number
    4
    Respiratory tract infection
         subjects affected / exposed
    5 / 10 (50.00%)
         occurrences all number
    10
    Skin infections
         subjects affected / exposed
    5 / 10 (50.00%)
         occurrences all number
    7
    Skin and subcutaneous tissue disorders
    Skin/mucosal blisters/wounds
         subjects affected / exposed
    9 / 10 (90.00%)
         occurrences all number
    16
    Dry skin
         subjects affected / exposed
    2 / 10 (20.00%)
         occurrences all number
    2
    Fine hair growth
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Milia
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Pruritus
         subjects affected / exposed
    4 / 10 (40.00%)
         occurrences all number
    4
    Rash
         subjects affected / exposed
    2 / 10 (20.00%)
         occurrences all number
    4
    Renal and urinary disorders
    Oliguria
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Joint pain
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1
    Infections and infestations
    Fever
         subjects affected / exposed
    2 / 10 (20.00%)
         occurrences all number
    2
    Urinary tract infections
         subjects affected / exposed
    1 / 10 (10.00%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    04 Feb 2014
    Reduction of Data Monitoring Committee meetings detailed in the protocol
    25 Sep 2014
    To change primary end point and reduce follow up period from 24 months to 12 months.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/25905587
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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