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    Clinical Trial Results:
    Effect of adipose tissue derived mesenchymal stromal cells on autism and leaky gut syndrome. A phase I pilot study.

    Summary
    EudraCT number
    2022-002940-27
    Trial protocol
    DK  
    Global end of trial date
    20 Sep 2024

    Results information
    Results version number
    v1(current)
    This version publication date
    09 Jul 2025
    First version publication date
    09 Jul 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    C2C1-1
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT05602116
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Cell2Cure ApS
    Sponsor organisation address
    Kajerødgård 9, Birkerød, Denmark, 3460
    Public contact
    Jens Kastrup, Cell2Cure ApS, 45 21202994, jk@cell2cure.com
    Scientific contact
    Jens Kastrup, Cell2Cure ApS, 45 21202994, jk@cell2cure.com
    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
    22 Apr 2025
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    20 Sep 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Sep 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The purpose of this dose titrating study was to evaluate the safety and efficacy of an allogeneic adipose tissue-derived mesenchymal stromal/stem cell product (C2C_ASC) in children with autism spectrum disorder (ASD) and gastrointestinal symptoms. Results from this study will be used to support the feasibility and safety of C2C_ASC treatment in children. And further to support the hypothesis of a connection between gastrointestinal symptoms, increased local gastrointestinal and systemic elevated immunological and inflammatory activity, bacterial toxins in the blood and symptoms of autism spectrum disorder that can be reduced or normalized by modulating the immunological activity and inflammation by treatment with mesenchymal stromal/stem cells.
    Protection of trial subjects
    Treatment visits was planned at least one week apart, to observe for serious adverse events related to the cell treatment and procedure, before treatment of a new trial subject. Data monitoring was done for all enrolled trial subjects.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    09 Jun 2023
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Denmark: 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)
    0
    Children (2-11 years)
    3
    Adolescents (12-17 years)
    7
    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
    The trial subjects was recruited through the Department of Child and Adolescent Psychiatry, Psychiatry Clinic South, Aalborg University Hospital, Denmark. A total number of 10 subjects was planned and enrolled. All 10 subjects received the study intervention and completed the study follow-up.

    Pre-assignment
    Screening details
    Date of enrolment (first participant’s first visit): 05 September 2023 Date of completion (last participant’s last visit): 20 September 2024 Key inclusion criteria were: • Children aged 6 – 14 years • Diagnosis of autism spectrum disorder (ASD) • Gastrointestinal symptoms or previous gastrointestinal symptoms

    Period 1
    Period 1 title
    Baseline (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    1 x 1 million ASCs/kg body weight
    Arm description
    Trial subject 1-5 was allocated to this arm with one treatment of 1 x 1 million ASCs/kg body weight.
    Arm type
    Experimental

    Investigational medicinal product name
    CSCC_ASC5010
    Investigational medicinal product code
    CSCC_ASC5010
    Other name
    Allogeneic adipose tissue-derived mesenchymal stromal/stem cells
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    The investigational product (IP) was an advanced therapy investigational medicinal product (ATIMP) manufactured from allogeneic adipose tissue-derived mesenchymal stromal/stem cells (ASCs). The active substance is in vitro expanded ASCs. The IP, CSCC_ASC5010, was manufactured as a cryopreserved suspension of 50 million ASCs per ml with a total volume of 1,3 ml per vial. In chronological order subjects was randomized to a low dose or a high dose where the first 5 enrolled subjects received 1 x 1 million ASCs/kg body weight (low dose), and the last 5 enrolled subjects received 2 x 1 million ASCs/kg body weight (high dose). The IP dose was calculated from the subject’s body weight. The calculated dose was extracted from one or more vials and was diluted in 50 ml isotonic saline and infused in a vein in the hand.

    Arm title
    2 x 1 million ASCs/kg body weight
    Arm description
    Trial subject 6-10 was allocated to this arm with one treatment of 2 x 1 million ASCs/kg body weight.
    Arm type
    Experimental

    Investigational medicinal product name
    CSCC_ASC5010
    Investigational medicinal product code
    CSCC_ASC5010
    Other name
    Allogeneic adipose tissue-derived mesenchymal stromal/stem cells
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    The investigational product (IP) was an advanced therapy investigational medicinal product (ATIMP) manufactured from allogeneic adipose tissue-derived mesenchymal stromal/stem cells (ASCs). The active substance is in vitro expanded ASCs. The IP, CSCC_ASC5010, was manufactured as a cryopreserved suspension of 50 million ASCs per ml with a total volume of 1,3 ml per vial. In chronological order subjects was randomized to a low dose or a high dose where the first 5 enrolled subjects received 1 x 1 million ASCs/kg body weight (low dose), and the last 5 enrolled subjects received 2 x 1 million ASCs/kg body weight (high dose). The IP dose was calculated from the subject’s body weight. The calculated dose was extracted from one or more vials and was diluted in 50 ml isotonic saline and infused in a vein in the hand.

    Number of subjects in period 1
    1 x 1 million ASCs/kg body weight 2 x 1 million ASCs/kg body weight
    Started
    5
    5
    Completed
    5
    5

    Baseline characteristics

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

    Reporting group values
    Baseline Total
    Number of subjects
    10 10
    Age categorical
    Units: Subjects
        Children (2-11 years)
    3 3
        Adolescents (12-17 years)
    7 7
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    12.6 ( 2.19 ) -
    Gender categorical
    Units: Subjects
        Female
    1 1
        Male
    9 9
    Reported gastrointestinal symptoms
    Reported gastrointestinal symptoms. Measurement: history of painful or hard stools.
    Units: Subjects
        History of painful or hard stools
    10 10
    Body weight
    Units: kilogram(s)
        arithmetic mean (standard deviation)
    47.06 ( 22.72 ) -
    Childrens communication checklist - Generel communication
    Questionnaire Childrens communication checklist. Measurement: generel communication.
    Units: unit(s)
        arithmetic mean (standard deviation)
    44.6 ( 20.58 ) -
    Childrens communication checklist - Social interaction
    Questionnaire Childrens communication checklist. Measurement: Social interaction.
    Units: unit(s)
        arithmetic mean (standard deviation)
    -9.9 ( 8.84 ) -
    IBS-QOL
    Questionnaire IBS-QOL. Measurement: Overall
    Units: unit(s)
        arithmetic mean (standard deviation)
    82.87 ( 13.51 ) -
    CRP
    Units: milligram(s)/litre
        arithmetic mean (standard deviation)
    1.4 ( 0.68 ) -
    Subject analysis sets

    Subject analysis set title
    Baseline data
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    Age

    Subject analysis sets values
    Baseline data
    Number of subjects
    10
    Age categorical
    Units: Subjects
        Children (2-11 years)
    3
        Adolescents (12-17 years)
    7
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    12.6 ( 2.19 )
    Gender categorical
    Units: Subjects
        Female
    1
        Male
    9
    Reported gastrointestinal symptoms
    Reported gastrointestinal symptoms. Measurement: history of painful or hard stools.
    Units: Subjects
        History of painful or hard stools
    9
    Body weight
    Units: kilogram(s)
        arithmetic mean (standard deviation)
    47.06 ( 22.72 )
    Childrens communication checklist - Generel communication
    Questionnaire Childrens communication checklist. Measurement: generel communication.
    Units: unit(s)
        arithmetic mean (standard deviation)
    44.6 ( 20.58 )
    Childrens communication checklist - Social interaction
    Questionnaire Childrens communication checklist. Measurement: Social interaction.
    Units: unit(s)
        arithmetic mean (standard deviation)
    -9.9 ( 8.84 )
    IBS-QOL
    Questionnaire IBS-QOL. Measurement: Overall
    Units: unit(s)
        arithmetic mean (standard deviation)
    82.87 ( 13.51 )
    CRP
    Units: milligram(s)/litre
        arithmetic mean (standard deviation)
    1.4 ( 0.68 )

    End points

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    End points reporting groups
    Reporting group title
    1 x 1 million ASCs/kg body weight
    Reporting group description
    Trial subject 1-5 was allocated to this arm with one treatment of 1 x 1 million ASCs/kg body weight.

    Reporting group title
    2 x 1 million ASCs/kg body weight
    Reporting group description
    Trial subject 6-10 was allocated to this arm with one treatment of 2 x 1 million ASCs/kg body weight.

    Subject analysis set title
    Baseline data
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    Age

    Primary: Safety

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    End point title
    Safety [1]
    End point description
    To assess the safety and tolerability of intravenous administration of a single dose 1 x 1 million ASCs per kg body weight and 2 x 1 million ASCs per kg body weight in children with autism spectrum disorder and gastrointestinal symptoms. Endpoint defined by adverse events (AEs), serious adverse events (SARs), and suspected unexpected serious adverse events (SUSARs).
    End point type
    Primary
    End point timeframe
    From treatment to the 12-week follow-up.
    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: The primary end point is safety registred as the number of safety events, thus no statistical analyses are needed.
    End point values
    1 x 1 million ASCs/kg body weight 2 x 1 million ASCs/kg body weight
    Number of subjects analysed
    5
    5
    Units: Totals
        Adverse events (AEs)
    8
    1
        Adverse reactions (ARs)
    2
    0
        Serious adverse events (SAEs)
    0
    0
        suspected unexpected serious adverse events (SUSAR
    0
    0
    No statistical analyses for this end point

    Secondary: Efficacy - changes in symptoms of autism spectrum disorder

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    End point title
    Efficacy - changes in symptoms of autism spectrum disorder
    End point description
    To characterize changes in symptoms of autism spectrum disorder. Assessed by questionnaires: • Children’s Communication Checklist (second edition, Pearson Clinical)
    End point type
    Secondary
    End point timeframe
    From baseline to the 12-week follow-up.
    End point values
    1 x 1 million ASCs/kg body weight 2 x 1 million ASCs/kg body weight
    Number of subjects analysed
    5
    5
    Units: unit(s)
    arithmetic mean (standard deviation)
        Generel communication
    48.6 ( 21.31 )
    47.2 ( 24.51 )
        Social interaction
    -8.6 ( 6.11 )
    -4.2 ( 12.99 )
    No statistical analyses for this end point

    Secondary: Efficacy - changes in gastrointestinal symptoms

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    End point title
    Efficacy - changes in gastrointestinal symptoms
    End point description
    To characterize changes in gastrointestinal symptoms. Assessed by questionnaire: • IBS-QOL (irritable bowel syndromequality of life) (ePROVIDE)
    End point type
    Secondary
    End point timeframe
    From baseline to the 12-week follow-up.
    End point values
    1 x 1 million ASCs/kg body weight 2 x 1 million ASCs/kg body weight
    Number of subjects analysed
    5
    5
    Units: unit(s)
    arithmetic mean (standard deviation)
        Overall
    90.44 ( 8.76 )
    75.15 ( 27.1 )
    No statistical analyses for this end point

    Secondary: Efficacy - changes in inflammatory markers

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    End point title
    Efficacy - changes in inflammatory markers
    End point description
    To characterize changes in inflammatory markers. Assessed by plasma analysis of: • CRP
    End point type
    Secondary
    End point timeframe
    From baseline to the 12-week follow-up.
    End point values
    1 x 1 million ASCs/kg body weight 2 x 1 million ASCs/kg body weight
    Number of subjects analysed
    5
    5
    Units: milligram(s)/litre
    arithmetic mean (standard deviation)
        CRP
    1.52 ( 1.05 )
    1.74 ( 1.19 )
    No statistical analyses for this end point

    Secondary: Efficacy - changes in gastrointestinal symptoms

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    End point title
    Efficacy - changes in gastrointestinal symptoms
    End point description
    End point type
    Secondary
    End point timeframe
    Reported gastrointestinal symptoms. Measurement: history of painful and hard stools.
    End point values
    1 x 1 million ASCs/kg body weight 2 x 1 million ASCs/kg body weight
    Number of subjects analysed
    5
    5
    Units: Subjects
        History of painful or hard stools
    1
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From treatment to the 12-week follow-up.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    18
    Reporting groups
    Reporting group title
    1 x 1 million ASCs/kg body weight
    Reporting group description
    -

    Reporting group title
    2 x 1 million ASCs/kg body weight
    Reporting group description
    -

    Serious adverse events
    1 x 1 million ASCs/kg body weight 2 x 1 million ASCs/kg body weight
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 5 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    1 x 1 million ASCs/kg body weight 2 x 1 million ASCs/kg body weight
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 5 (60.00%)
    2 / 5 (40.00%)
    Gastrointestinal disorders
    Diarrhea
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Vomitting
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Increased fecal calprotectin
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Atopic eczema
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Pain in testicles
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Headache
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Fever
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Increased C-reactive protein
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Common cold
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 5 (40.00%)
         occurrences all number
    0
    2

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? No

    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
    For support, Contact us.
    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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