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    Clinical Trial Results:
    Evaluate the efficacy of the cell therapy with NC1 medication in patients with post-traumatic syringomyelia

    Summary
    EudraCT number
    		 2015-002383-16
    Trial protocol
    		 ES  
    Global end of trial date
    07 Feb 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    03 Apr 2022
    First version publication date
    03 Apr 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CME-LEM4
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Fundación Investigación Biomédica Hospital Universitario Puerta de Hierro
    Sponsor organisation address
    C/ Joaquín Rodrigo, 2 Majadahonda, Madrid, Spain, 28222
    Public contact
    SIte contact point, Fundación Investigación Biomédica Hospital Universitario Puerta de Hierro, +34 91 1917760,
    Scientific contact
    SIte contact point, Fundación Investigación Biomédica Hospital Universitario Puerta de Hierro, +34 91 1917760,
    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
    07 Feb 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    07 Feb 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Feb 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To analyze the potential clinical efficacy of the administration in the intrathecal compartment, (intramedullary and in the subarachnoid space), of the medication NC1, to improve the neurological sequels of patients with established chronic spinal cord injury (LEM) and post-traumatic syringomyelia.
    Protection of trial subjects
    Previous to NC1 preparation, a sample of peripheral blood was retrieved from each patient for genomic studies in order to rule out chromosomal abnormalities that could discourage cell expansion.
    Background therapy
    		 From the start of treatment until the end of the trial (month 6 after CME administration) patients performed physical therapy exercises.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    07 Mar 2016
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 5 Years
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Spain: 6
    Worldwide total number of subjects
    6
    EEA total number of subjects
    6
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    6
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The planned duration of the clinical trial was 12 months. The duration of the recruitment phase was 6 months, and the duration of the follow-up period after treatment was 6 months.

    Pre-assignment
    Screening details
    		 After signing the Informed Consent Form, participants were tested to determine if they met all the inclusion criteria and none of the exclusion criteria.

    Pre-assignment period milestones
    Number of subjects started
    		 6
    Number of subjects completed
    		 6

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    Not applicable. All the participants received the same treatment.

    Arms
    Arm title
    		 Arm 1
    Arm description
    		 Treatment arm
    Arm type
    		 Experimental

    Investigational medicinal product name
    NC1
    Investigational medicinal product code
    Other name
    PEI number 12–141 (by the Spanish Agency of Medicament and Health Products)
    Pharmaceutical forms
    Suspension for injection
    Routes of administration
    Intrathecal use
    Dosage and administration details
    Administration inside of the syrinx of a single dose of 300 x 10^6 autologous expanded mesenchymal stromal cells, supported in autologous plasma, through a surgical approach to the spinal cord.

    Number of subjects in period 1
    		Arm 1
    Started
    6
    Completed
    6

    Baseline characteristics

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

    Reporting group values
    		 Overall trial Total
    Number of subjects
    		 6 6
    Age categorical
    Age ranged between 30 and 50 years (mean ± standard deviation [SD], 39 ± 7.6 years)
    Units: Subjects
        Adults (18-64 years)
    		 6 6
    Gender categorical
    Units: Subjects
        Female
    		 0 0
        Male
    		 6 6
    American Spinal Injury Association Impairment Scale (ASIA) grade
    Units: Subjects
        ASIA A
    		 3 3
        ASIA B
    		 2 2
        ASIA D
    		 1 1
    Spinal cord injury (SCI) vertebral level
    Units: Subjects
        D3
    		 1 1
        D4
    		 1 1
        D5
    		 2 2
        D8
    		 1 1
        L1
    		 1 1
    Time since Spinal cord injury (SCI)
    Units: Subjects
        5,75 years
    		 1 1
        6,16 years
    		 1 1
        8,07 years
    		 1 1
        17,01 years
    		 1 1
        17,72 years
    		 1 1
        27,68 years
    		 1 1

    End points

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    End points reporting groups
    Reporting group title
    Arm 1
    Reporting group description
    		 Treatment arm

    Subject analysis set title
    Before treatment
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Baseline characteristics of the subjects

    Subject analysis set title
    At 3 months follow-up
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Characteristics of the subjects at 3 months follow-up

    Subject analysis set title
    At 6 months follow-up
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Characteristics of the subjects at 6 months follow-up

    Primary: Change in the score in ASIA scale

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    End point title
    		 Change in the score in ASIA scale
    End point description
    ASIA scale was used for sensitivity and motor assessments. Efficacy was assessed by taking into account the variation in the scores in the different scales between the subject´s inclusion in the study and the scores obtained at the end of the follow-up period.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up (FU) period.
    End point values
    		 Before treatment At 3 months follow-up At 6 months follow-up
    Number of subjects analysed
    6
    6
    6
    Units: Score
    arithmetic mean (standard deviation)
        Total Score
    143.00 ( 16.20 )
    144.3 ( 16.69 )
    149.00 ( 17.18 )
        Pin Prick Score
    41.67 ( 5.27 )
    43.17 ( 6.36 )
    44.67 ( 7.03 )
        Light Touch Score
    50.33 ( 11.76 )
    50.17 ( 11.55 )
    53.33 ( 11.94 )
    Statistical analysis title
    		 Total Score: before treatment vs at 3 months FU
    Comparison groups
    Before treatment v At 3 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.37
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Total Score: before treatment vs at 6 months FU
    Comparison groups
    Before treatment v At 6 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.12
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 PinPrick Score: before treatment vs at 3 months FU
    Comparison groups
    Before treatment v At 3 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.25
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 PinPrick Score: before treatment vs at 6 months FU
    Comparison groups
    Before treatment v At 6 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.06
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Light Touch Score: before treatment vs at 3 months
    Comparison groups
    Before treatment v At 3 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 > 0.99
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Light Touch Score: before treatment vs at 6 months
    Comparison groups
    Before treatment v At 6 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.25
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Change in the score in IANR-SCIFRS scale

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    End point title
    		 Change in the score in IANR-SCIFRS scale
    End point description
    IANR-SCIFRS scale: the SCI Functional Rating Scale of the International Association of Neurorestoratology scale. This scale evaluates the global spinal cord function. Efficacy was assessed by taking into account the variation in the scores on the different scales between the subject´s inclusion in the study and the scores obtained at the end of the follow-up period.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    End point values
    		 Before treatment At 3 months follow-up At 6 months follow-up
    Number of subjects analysed
    6
    6
    6
    Units: score
        arithmetic mean (standard deviation)
    29.33 ( 7.86 )
    32.33 ( 6.31 )
    34.83 ( 3.86 )
    Statistical analysis title
    		 Before treatment vs at 3 months FU
    Comparison groups
    Before treatment v At 3 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.06
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Before treatment vs at 6 months FU
    Comparison groups
    Before treatment v At 6 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.06
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Change in the score in VAS scale

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    End point title
    		 Change in the score in VAS scale
    End point description
    VAS: Visual Analog Scale. This scale evaluates neuropathic pain. Efficacy was assessed by taking into account the variation in the scores on the different scales between the subject´s inclusion in the study and the scores obtained at the end of the follow-up period.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    End point values
    		 Before treatment At 3 months follow-up At 6 months follow-up
    Number of subjects analysed
    6
    6
    6
    Units: score
        arithmetic mean (standard deviation)
    2.00 ( 3.16 )
    1.33 ( 2.42 )
    1.00 ( 2.44 )
    Statistical analysis title
    		 Before treatment vs at 3 months FU
    Comparison groups
    At 3 months follow-up v Before treatment
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.25
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Before treatment vs at 6 months FU
    Comparison groups
    Before treatment v At 6 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.25
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Change in the score in PENN scale

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    End point title
    		 Change in the score in PENN scale
    End point description
    PENN scale measures the degree of spasms. Efficacy was assessed by taking into account the variation in the scores on the different scales between the subject´s inclusion in the study and the scores obtained at the end of the follow-up period.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    End point values
    		 Before treatment At 3 months follow-up At 6 months follow-up
    Number of subjects analysed
    6
    6
    6
    Units: score
        arithmetic mean (standard deviation)
    1.66 ( 1.21 )
    1.33 ( 1.03 )
    1.16 ( 0.75 )
    Statistical analysis title
    		 Before treatment vs at 3 months FU
    Comparison groups
    At 3 months follow-up v Before treatment
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 > 0.99
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Before treatment vs at 6 months FU
    Comparison groups
    Before treatment v At 6 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.5
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Change in the score in Ashworth scale

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    End point title
    		 Change in the score in Ashworth scale
    End point description
    Ashworth scale measures the degree of spasticity. Efficacy was assessed by taking into account the variation in the scores on the different scales between the subject´s inclusion in the study and the scores obtained at the end of the follow-up period.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    End point values
    		 Before treatment At 3 months follow-up At 6 months follow-up
    Number of subjects analysed
    6
    6
    6
    Units: score
        arithmetic mean (standard deviation)
    1.75 ( 1.17 )
    1.58 ( 1.35 )
    1.50 ( 0.83 )
    Statistical analysis title
    		 Before treatment vs at 3 months FU
    Comparison groups
    Before treatment v At 3 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 > 0.99
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Before treatment vs at 6 months FU
    Comparison groups
    Before treatment v At 6 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.5
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Change in the score in Geffner scale

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    End point title
    		 Change in the score in Geffner scale
    End point description
    Geffner scale was used for the study of bladder function. Efficacy was assessed by taking into account the variation in the scores on the different scales between the subject´s inclusion in the study and the scores obtained at the end of the follow-up period.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    End point values
    		 Before treatment At 3 months follow-up At 6 months follow-up
    Number of subjects analysed
    6
    6
    6
    Units: score
        arithmetic mean (standard deviation)
    1.83 ( 1.47 )
    2.50 ( 1.22 )
    2.83 ( 1.47 )
    Statistical analysis title
    		 Before treatment vs at 3 months FU
    Comparison groups
    Before treatment v At 3 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.5
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Before treatment vs at 6 months FU
    Comparison groups
    Before treatment v At 6 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.25
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Change in the score in NBD scale

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    End point title
    		 Change in the score in NBD scale
    End point description
    NBD scale was used for the study of of neurogenic bowel dysfunction (NBD). Efficacy was assessed by taking into account the variation in the scores on the different scales between the subject´s inclusion in the study and the scores obtained at the end of the follow-up period.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    End point values
    		 Before treatment At 3 months follow-up At 6 months follow-up
    Number of subjects analysed
    6
    6
    6
    Units: score
        arithmetic mean (standard deviation)
    14.67 ( 6.91 )
    9.83 ( 3.92 )
    8.50 ( 5.54 )
    Statistical analysis title
    		 Before treatment vs at 3 months FU
    Comparison groups
    At 3 months follow-up v Before treatment
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.12
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Before treatment vs at 6 months FU
    Comparison groups
    At 6 months follow-up v Before treatment
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.12
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Change in parameters of ano-rectal manometry: pressure of rectal sphincter at rest

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    End point title
    		 Change in parameters of ano-rectal manometry: pressure of rectal sphincter at rest [1]
    End point description
    Functional ano-rectal studies were performed using a High Resolution Manometry equipment (Solar GI HRM, MMS B.V., Enschede, NL) and a water perfused 8 channel catheter with a balloon at the tip. With the patient in a left lateral position, with hips and knees bent, and after a rest period of 5 to 10 minutes, the parameters were assessed. Descriptive analysis was performed for anorectal manomety parameters. Two subjects (2 and 5) showed improvement achieving a higher mean pressure of rectal sphincter.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    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: Descriptive analysis was performed for anorectal manomety parameters, comparing the results with the baseline values.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: mmHG
    number (not applicable)
        Subject 1
    50
    53
        Subject 2
    25
    83
        Subject 3
    31
    37
        Subject 4
    69
    36
        Subject 5
    60
    79
        Subject 6
    68
    54
    No statistical analyses for this end point

    Primary: Change in parameters of ano-rectal manometry: pressure of anal contraction

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    End point title
    		 Change in parameters of ano-rectal manometry: pressure of anal contraction [2]
    End point description
    Functional ano-rectal studies were performed using a High Resolution Manometry equipment (Solar GI HRM, MMS B.V., Enschede, NL) and a water perfused 8 channel catheter with a balloon at the tip. With the patient in a left lateral position, with hips and knees bent, and after a rest period of 5 to 10 minutes, the parameters were assessed. Descriptive analysis was performed for anorectal manomety parameters. Three patients (1, 3 and 5) showed improvement achieving a higher anal contraction pressure. 00 = non computable
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [2] - 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: Descriptive analysis was performed for anorectal manomety parameters, comparing the results with the baseline values.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: mmHg
    number (not applicable)
        Subject 1
    50
    70
        Subject 2
    35
    00
        Subject 3
    40
    61
        Subject 4
    82
    36
        Subject 5
    71
    164
        Subject 6
    102
    60
    No statistical analyses for this end point

    Primary: Change in parameters of ano-rectal manometry: first sensation of rectal filling

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    End point title
    		 Change in parameters of ano-rectal manometry: first sensation of rectal filling [3]
    End point description
    Functional ano-rectal studies were performed using a High Resolution Manometry equipment (Solar GI HRM, MMS B.V., Enschede, NL) and a water perfused 8 channel catheter with a balloon at the tip. With the patient in a left lateral position, with hips and knees bent, and after a rest period of 5 to 10 minutes, the parameters were assessed. Descriptive analysis was performed for anorectal manomety parameters. Three patients (2, 3 and 5) showed improvement of first rectal filling sensation. 00 = absent
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [3] - 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: Descriptive analysis was performed for anorectal manomety parameters, comparing the results with the baseline values.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: mmHg
    number (not applicable)
        Subject 1
    00
    00
        Subject 2
    00
    180
        Subject 3
    150
    80
        Subject 4
    100
    00
        Subject 5
    00
    60
        Subject 6
    00
    00
    No statistical analyses for this end point

    Primary: Change in urodynamic studies: first sensation at filling

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    End point title
    		 Change in urodynamic studies: first sensation at filling [4]
    End point description
    Urodynamic studies were performed using using a Solar Luna equipment (Medical Measurement Systems Inc., Dover, NH, USA). At the end of the study, all of the patients showed improvement in two or more of the parameters studied. Four patients (1, 3, 4 and 6) improved in first sensation at filling.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [4] - 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: Descriptive analysis was performed for urodinamic studies, comparing the results with the baseline values.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: cc
    number (not applicable)
        Subject 1
    454
    360
        Subject 2
    560
    610
        Subject 3
    307
    292
        Subject 4
    38
    33
        Subject 5
    140
    212
        Subject 6
    166
    134
    No statistical analyses for this end point

    Primary: Change in urodynamic studies: bladder capacity at filling

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    End point title
    		 Change in urodynamic studies: bladder capacity at filling [5]
    End point description
    Urodynamic studies were performed using using a Solar Luna equipment (Medical Measurement Systems Inc., Dover, NH, USA). At the end of the study, all of the patients showed improvement in two or more of the parameters studied. Four patients (2, 4, 5 and 6) improved in maximum cystometric capacity.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [5] - 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: Descriptive analysis was performed for urodinamic studies, comparing the results with the baseline values.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: cc
    number (not applicable)
        Subject 1
    456
    412
        Subject 2
    573
    611
        Subject 3
    346
    324
        Subject 4
    171
    209
        Subject 5
    147
    221
        Subject 6
    169
    431
    No statistical analyses for this end point

    Primary: Change in urodynamic studies: detrusor pressure at filling

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    End point title
    		 Change in urodynamic studies: detrusor pressure at filling [6]
    End point description
    Urodynamic studies were performed using using a Solar Luna equipment (Medical Measurement Systems Inc., Dover, NH, USA). At the end of the study, all of the patients showed improvement in two or more of the parameters studied. Three patients (2, 3 and 5) showed decrease in detrusor pressure at filling.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [6] - 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: Descriptive analysis was performed for urodinamic studies, comparing the results with the baseline values.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: cm H2O
    number (not applicable)
        Subject 1
    30
    51
        Subject 2
    19
    10
        Subject 3
    4
    3
        Subject 4
    10
    111
        Subject 5
    81
    61
        Subject 6
    8
    33
    No statistical analyses for this end point

    Primary: Change in urodynamic studies: bladder compliance at filling

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    End point title
    		 Change in urodynamic studies: bladder compliance at filling [7]
    End point description
    Urodynamic studies were performed using using a Solar Luna equipment (Medical Measurement Systems Inc., Dover, NH, USA). At the end of the study, all of the patients showed improvement in two or more of the parameters studied. Three patients (2, 3 and 5) improved in bladder compliance at filling.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [7] - 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: Descriptive analysis was performed for urodinamic studies, comparing the results with the baseline values.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: cc
    number (not applicable)
        Subject 1
    15
    8
        Subject 2
    30
    61
        Subject 3
    86
    108
        Subject 4
    17
    2
        Subject 5
    2
    4
        Subject 6
    21
    13
    No statistical analyses for this end point

    Primary: Change in urodynamic studies: postmictional residue

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    End point title
    		 Change in urodynamic studies: postmictional residue [8]
    End point description
    Urodynamic studies were performed using using a Solar Luna equipment (Medical Measurement Systems Inc., Dover, NH, USA). At the end of the study, all of the patients showed improvement in two or more of the parameters studied. Four patients (1, 3, 4 and 5) improved in postmictional residue.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [8] - 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: Descriptive analysis was performed for urodinamic studies, comparing the results with the baseline values.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: cc
    number (not applicable)
        Subject 1
    268
    106
        Subject 2
    573
    611
        Subject 3
    346
    324
        Subject 4
    171
    87
        Subject 5
    127
    50
        Subject 6
    75
    431
    No statistical analyses for this end point

    Primary: Change in spinal cord morphology, after neuroimaging study: length of syrinx

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    End point title
    		 Change in spinal cord morphology, after neuroimaging study: length of syrinx
    End point description
    Before treatment, in magnetic resonance, all of the patients had a large syringomyelic cavity on either side of the SCI area. The extension of the syrinx ranged between 87 and 300 mm (mean ± SD, 210.3 ± 90.94 mm). The measurements were taken by means of software associated with MR- 3T equipment (Philips Intera Achieva XR, v 263.9; Philips Healthcare) on sagittal T2-weighted images and MR-myelography images achieved with sequences of “turbo spin-echo” The measurements must be considered as approximate due to the difficulty of obtaining strictly superimposable images in sequential studies.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: mm
    number (not applicable)
        Subject 1
    120
    113
        Subject 2
    87
    47
        Subject 3
    280
    280
        Subject 4
    195
    195
        Subject 5
    300
    142
        Subject 6
    280
    100
    Statistical analysis title
    		 Before treatment vs at 6 months FU
    Comparison groups
    Before treatment v At 6 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.12
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Change in spinal cord morphology, after neuroimaging study: width of syrinx

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    End point title
    		 Change in spinal cord morphology, after neuroimaging study: width of syrinx
    End point description
    Before treatment, in magnetic resonance, all of the patients had a large syringomyelic cavity on either side of the SCI area. In the medium sagittal plain, the width of the syrinx ranged between 10 and 20 mm (mean ± SD, 12.05 ± 3.97 mm). The measurements were taken by means of software associated with MR- 3T equipment (Philips Intera Achieva XR, v 263.9; Philips Healthcare) on sagittal T2-weighted images and MR-myelography images achieved with sequences of “turbo spin-echo” The measurements must be considered as approximate due to the difficulty of obtaining strictly superimposable images in sequential studies.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: mm
    number (not applicable)
        Subject 1
    10.3
    6.3
        Subject 2
    10.0
    1.2
        Subject 3
    10.0
    8.0
        Subject 4
    10.0
    4.6
        Subject 5
    20.0
    14.2
        Subject 6
    12.0
    4.0
    Statistical analysis title
    		 Before treatment vs at 6 months FU
    Comparison groups
    At 6 months follow-up v Before treatment
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.03
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Change in spinal cord morphology, after neuroimaging study: syrinx/canal index

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    End point title
    		 Change in spinal cord morphology, after neuroimaging study: syrinx/canal index
    End point description
    The syrinx/canal index was studied as an index that indirectly values intramedullary tension.
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    End point values
    		 Before treatment At 6 months follow-up
    Number of subjects analysed
    6
    6
    Units: S/C Index
        arithmetic mean (standard deviation)
    0.84 ( 0.24 )
    0.33 ( 0.19 )
    Statistical analysis title
    		 Before treatment vs at 6 months FU
    Comparison groups
    Before treatment v At 6 months follow-up
    Number of subjects included in analysis
    12
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 = 0.03
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Change in neurophysiological parameters: improvement in Somatosensory Evoked Potentials (SSEP)

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    End point title
    		 Change in neurophysiological parameters: improvement in Somatosensory Evoked Potentials (SSEP) [9]
    End point description
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [9] - 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: A qualitative evaluation was performed, comparing the results with the baseline values.
    End point values
    		 Arm 1
    Number of subjects analysed
    6
    Units: Subjects
        Improvement in SSEP
    2
        No improvement in SSEP
    4
    No statistical analyses for this end point

    Primary: Change in neurophysiological parameters: improvement in Motor Evoked Potentials (MEP)

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    End point title
    		 Change in neurophysiological parameters: improvement in Motor Evoked Potentials (MEP) [10]
    End point description
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [10] - 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: A qualitative evaluation was performed, comparing the results with the baseline values.
    End point values
    		 Arm 1
    Number of subjects analysed
    6
    Units: Subjects
        Improvement in MEP
    1
        No improvement in MEP
    5
    No statistical analyses for this end point

    Primary: Change in neurophysiological parameters: improvement in sensitivity conduction

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    End point title
    		 Change in neurophysiological parameters: improvement in sensitivity conduction [11]
    End point description
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [11] - 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: A qualitative evaluation was performed, comparing the results with the baseline values.
    End point values
    		 Arm 1
    Number of subjects analysed
    6
    Units: Subjects
        Improvement
    2
        No improvement
    4
    No statistical analyses for this end point

    Primary: Change in neurophysiological parameters: improvement in motor conduction

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    End point title
    		 Change in neurophysiological parameters: improvement in motor conduction [12]
    End point description
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [12] - 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: A qualitative evaluation was performed, comparing the results with the baseline values.
    End point values
    		 Arm 1
    Number of subjects analysed
    6
    Units: Subjects
        Improvement
    1
        No improvement
    5
    No statistical analyses for this end point

    Primary: Change in neurophysiological parameters: improvement in voluntary muscle contraction

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    End point title
    		 Change in neurophysiological parameters: improvement in voluntary muscle contraction [13]
    End point description
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [13] - 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: A qualitative evaluation was performed, comparing the results with the baseline values.
    End point values
    		 Arm 1
    Number of subjects analysed
    6
    Units: Subjects
        Improvement
    1
        No improvement
    5
    No statistical analyses for this end point

    Primary: Change in neurophysiological parameters: presence of infralesional activity muscle reinnervation

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    End point title
    		 Change in neurophysiological parameters: presence of infralesional activity muscle reinnervation [14]
    End point description
    End point type
    Primary
    End point timeframe
    Between the subject´s inclusion in the study and the end of the follow-up period.
    Notes
    [14] - 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: A qualitative evaluation was performed, comparing the results with the baseline values.
    End point values
    		 Arm 1
    Number of subjects analysed
    6
    Units: Subjects
        Improvement
    2
        No improvement
    4
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    During the entire clinical trial
    Adverse event reporting additional description
    Adverse events were collected asking questions to the participants and performing general clinical examinations and neurological examinations.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    Arm 1
    Reporting group description
    		 Treatment arm

    Serious adverse events
    		 Arm 1
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 6 (16.67%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    		 Arm 1
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 6 (50.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    General disorders and administration site conditions
    Temperature regulation disorder
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Thermal burn
         subjects affected / exposed
    1 / 6 (16.67%)
         occurrences all number
    1

    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.
    New studies with a greater number of cases are required.

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/29784434
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