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    Clinical Trial Results:
    A Prospective Nonrandomized Study of Autologous Muscle Derived Cell (AMDC) Transplantation for Treatment of Fecal Incontinence

    Summary
    EudraCT number
    		 2013-004672-35
    Trial protocol
    		 GB  
    Global end of trial date
    27 Oct 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    11 Oct 2022
    First version publication date
    11 Oct 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    09-025
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01600755
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Cook MyoSite, Inc.
    Sponsor organisation address
    105 Delta Drive, Pittsburgh, United States, 15238
    Public contact
    Kelly Cardello, Cook MyoSite, Inc., +1 412-963-7380, Kelly.Cardello@CookMyoSite.com
    Scientific contact
    Ron Jankowski, PhD, Cook MyoSite, Inc., +1 412-963-7380, Ron.Jankowski@CookMyoSite.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
    27 Oct 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    27 Oct 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    27 Oct 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To investigate the safety and feasibility of Autologous Muscle Derived Cells (AMDC, generic name iltamiocel, preparation of a patient's own cells) injection into the anal sphincter for treatment of patients with fecal incontinence. Iltamiocel therapy seeks to allow remodeling of the external anal sphincter in patients with fecal incontinence from either defined structural defects or a generalized weakening of the external anal sphincter. The primary objective of the study was to assess the frequency and severity of adverse events related to the study product and study procedures through 12 months following treatment of fecal incontinence in adult male and female subjects.
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles that have their origins in the Declaration of Helsinki, including the International Council for Harmonization (ICH) Guideline for Good Clinical Practice and applicable regulations in Canada and the United Kingdom where the study took place.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    28 Mar 2013
    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
    United Kingdom: 15
    Country: Number of subjects enrolled
    Canada: 33
    Worldwide total number of subjects
    48
    EEA total number of subjects
    0
    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)
    24
    From 65 to 84 years
    23
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    		 Subjects were screened and enrolled at 2 sites globally; 1 site in Canada and 1 site in the United Kingdom.

    Pre-assignment
    Screening details
    		 53 subjects were enrolled (underwent biopsy procedure) and 48 subjects received study treatment (iltamiocel injection). The analysis population is based on 48 subjects that received study treatment (iltamiocel injection).

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Iltamiocel
    Arm description
    		 AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10^6 cells.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Iltamiocel
    Investigational medicinal product code
    Other name
    Autologous muscle-derived cells (AMDC)
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intramuscular use
    Dosage and administration details
    Single intrasphincteric injection of 250 x 10^6 cells.

    Number of subjects in period 1
    		Iltamiocel
    Started
    48
    Iltamiocel Injection
    48
    3-Month Follow-Up
    48
    6-Month Follow-Up
    48
    12-Month Follow-Up
    47
    Completed
    47
    Not completed
    1
         Consent withdrawn by subject
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Iltamiocel
    Reporting group description
    		 AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10^6 cells.

    Reporting group values
    		 Iltamiocel Total
    Number of subjects
    		 48 48
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 24 24
        From 65-84 years
    		 24 24
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 61.7 ± 13.3 -
    Gender categorical
    Units: Subjects
        Female
    		 45 45
        Male
    		 3 3
    28-Day Incontinence Diary (Number of Episodes)
    Units: incontinence episodes
        median (full range (min-max))
    		 16 (0 to 92) -
    28-Day Incontinence Diary (Days with Episodes)
    Units: days with incontinence episodes
        median (full range (min-max))
    		 12 (0 to 28) -
    Cleveland Clinic Incontinence Score (CCIS)
    CCIS is a validated 5-questions tool assessing the frequency and the type of incontinence episodes (solid, liquid, gas), pad use, and lifestyle alteration. Scored from 0 to 20, with lower scores indicating less severe symptoms.
    Units: scores on scales
        arithmetic mean (standard deviation)
    		 13.6 ± 2.9 -
    Fecal Incontinence Quality of Life (FIQL) Questionnaire - Lifestyle Scale
    FIQL is a validated, 29-item tool assessing quality of life (QOL) of subjects with FI with four scales: Lifestyle (10 items), Coping/Behavior (9 items), Depression/Self-Perception (7 items), and Embarrassment (3 items). Scored 0 to 4, with higher scores indicating a better QOL.
    Units: scores on scales
        arithmetic mean (standard deviation)
    		 2.5 ± 0.9 -
    Fecal Incontinence Quality of Life (FIQL) Questionnaire - Coping/behavior Scale
    FIQL is a validated, 29-item tool assessing quality of life (QOL) of subjects with FI with four scales: Lifestyle (10 items), Coping/Behavior (9 items), Depression/Self-Perception (7 items), and Embarrassment (3 items). Scored 0 to 4, with higher scores indicating a better QOL.
    Units: scores on scales
        arithmetic mean (standard deviation)
    		 1.7 ± 0.6 -
    Fecal Incontinence Quality of Life (FIQL) Questionnaire - Depression/perception Scale
    FIQL is a validated, 29-item tool assessing quality of life (QOL) of subjects with FI with four scales: Lifestyle (10 items), Coping/Behavior (9 items), Depression/Self-Perception (7 items), and Embarrassment (3 items). Scored 0 to 4, with higher scores indicating a better QOL.
    Units: scores on scales
        arithmetic mean (standard deviation)
    		 2.5 ± 0.9 -
    Fecal Incontinence Quality of Life (FIQL) Questionnaire - Embarrassment Scale
    FIQL is a validated, 29-item tool assessing quality of life (QOL) of subjects with FI with four scales: Lifestyle (10 items), Coping/Behavior (9 items), Depression/Self-Perception (7 items), and Embarrassment (3 items). Scored 0 to 4, with higher scores indicating a better QOL.
    Units: scores on scales
        arithmetic mean (standard deviation)
    		 1.9 ± 0.8 -

    End points

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    End points reporting groups
    Reporting group title
    Iltamiocel
    Reporting group description
    		 AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10^6 cells.

    Primary: Incidence of Treatment-Related Adverse Events

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    End point title
    		 Incidence of Treatment-Related Adverse Events [1]
    End point description
    Assessment of frequency and severity of adverse events related to study product and study procedures through 12 months following treatment of FI in adult male and female subjects.
    End point type
    Primary
    End point timeframe
    12 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: This study was not a randomized controlled clinical trial. Therefore there was no comparator group. Frequency of serious adverse events and non-serious adverse events was calculated as the number of subjects with an event divided by the total number of subjects in the analysis population.
    End point values
    		 Iltamiocel
    Number of subjects analysed
    48
    Units: Events
        Study Product-related serious adverse events
    0
        Study Product-related adverse events
    1
        Injection procedure-related adverse events
    5
        Biopsy procedure-related adverse events
    27
    No statistical analyses for this end point

    Secondary: Number of Fecal Incontinence (FI) Episodes (Median 28-day Fecal Incontinence Diary)

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    End point title
    		 Number of Fecal Incontinence (FI) Episodes (Median 28-day Fecal Incontinence Diary)
    End point description
    Median number of fecal incontinence (FI) episodes as assessed by 28-day FI diary at 3, 6, and 12 months post-treatment.
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    		 Iltamiocel
    Number of subjects analysed
    48
    Units: incontinence episodes
    median (full range (min-max))
        Fecal incontinence diary at 3 months
    8 (0 to 100)
        Fecal incontinence diary at 6 months
    10 (0 to 206)
        Fecal incontinence diary at 12 months
    7 (0 to 161)
    No statistical analyses for this end point

    Secondary: Number of Days With FI Episodes (Median 28-day Fecal Incontinence Diary)

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    End point title
    		 Number of Days With FI Episodes (Median 28-day Fecal Incontinence Diary)
    End point description
    Median number of days with fecal incontinence (FI) episodes, as assessed by 28-day FI diary at 3, 6, and 12 months post-treatment.
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    		 Iltamiocel
    Number of subjects analysed
    48
    Units: days with incontinence episodes
    median (full range (min-max))
        Fecal incontinence diary at 3 months
    7 (0 to 28)
        Fecal incontinence diary at 6 months
    7 (0 to 28)
        Fecal incontinence diary at 12 months
    5 (0 to 28)
    No statistical analyses for this end point

    Secondary: Number of Fecal Incontinence (FI) Episodes (Categorical ≥50% Reduction in 28-day Fecal Incontinence Diary)

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    End point title
    		 Number of Fecal Incontinence (FI) Episodes (Categorical ≥50% Reduction in 28-day Fecal Incontinence Diary)
    End point description
    Number of fecal incontinence (FI) episodes (categorical ≥50% reduction in episodes) as assessed by 28-day FI diary at 3, 6, and 12 months post-treatment.
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    		 Iltamiocel
    Number of subjects analysed
    48
    Units: participants
        Fecal incontinence diary at 3 months
    17
        Fecal incontinence diary at 6 months
    19
        Fecal incontinence diary at 12 months
    22
    No statistical analyses for this end point

    Secondary: Number of Fecal Incontinence (FI) Episodes (Categorical ≥75% Reduction in 28-day Fecal Incontinence Diary)

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    End point title
    		 Number of Fecal Incontinence (FI) Episodes (Categorical ≥75% Reduction in 28-day Fecal Incontinence Diary)
    End point description
    Number of fecal incontinence (FI) episodes (categorical ≥75% reduction in episodes) as assessed by 28-day FI diary at 3, 6, and 12 months post-treatment.
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    		 Iltamiocel
    Number of subjects analysed
    48
    Units: participants
        Fecal incontinence diary at 3 months
    7
        Fecal incontinence diary at 6 months
    12
        Fecal incontinence diary at 12 months
    16
    No statistical analyses for this end point

    Secondary: Number of Fecal Incontinence (FI) Episodes (Categorical 100% Reduction in 28-day Fecal Incontinence Diary)

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    End point title
    		 Number of Fecal Incontinence (FI) Episodes (Categorical 100% Reduction in 28-day Fecal Incontinence Diary)
    End point description
    Number of fecal incontinence (FI) episodes (categorical 100% reduction in episodes) as assessed by 28-day FI diary at 3, 6, and 12 months post-treatment.
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    		 Iltamiocel
    Number of subjects analysed
    48
    Units: participants
        Fecal incontinence diary at 3 months
    1
        Fecal incontinence diary at 6 months
    4
        Fecal incontinence diary at 12 months
    10
    No statistical analyses for this end point

    Secondary: Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire)

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    End point title
    		 Change From Baseline in Patient-reported Quality of Life (Fecal Incontinence Quality of Life (FIQL) Questionnaire)
    End point description
    Mean change from baseline in patient-reported quality of life, as assessed by the Fecal Incontinence Quality of Life (FIQL) questionnaire at 3, 6, and 12 months post-treatment. The FIQL is a validated, 29-item tool assessing the quality of life (QOL) of subjects with FI with four scales: Lifestyle (10 items), Coping/Behavior (9 items), Depression/Self-Perception (7 items), and Embarrassment (3 items). Scored 0 to 4, with higher scores indicating a better QOL.
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    		 Iltamiocel
    Number of subjects analysed
    48
    Units: scores on scales
    arithmetic mean (standard deviation)
        Change in Lifestyle scale at 3 months
    0.4 ± 0.7
        Change in Lifestyle scale at 6 months
    0.4 ± 0.7
        Change in Lifestyle scale at 12 months
    0.6 ± 0.7
        Change in Coping/Behavior scale at 3 months
    0.4 ± 0.7
        Change in Coping/Behavior scale at 6 months
    0.5 ± 0.7
        Change in Coping/Behavior scale at 12 months
    0.5 ± 0.7
        Change in Depression/Perception scale at 3 months
    0.3 ± 0.7
        Change in Depression/Perception scale at 6 months
    0.3 ± 0.6
        Change in Depression/Perception scale at 12 months
    0.4 ± 0.6
        Change in Embarrassment scale at 3 months
    0.5 ± 0.8
        Change in Embarrassment scale at 6 months
    0.5 ± 0.8
        Change in Embarrassment scale at 12 months
    0.6 ± 0.9
    No statistical analyses for this end point

    Secondary: Change From Baseline in Patient-reported Fecal Incontinence Symptom Severity (Cleveland Clinic Incontinence Score (CCIS))

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    End point title
    		 Change From Baseline in Patient-reported Fecal Incontinence Symptom Severity (Cleveland Clinic Incontinence Score (CCIS))
    End point description
    Mean change from baseline in patient-reported fecal incontinence symptom severity, as assessed by the Cleveland Clinic Incontinence Score (CCIS) questionnaire at 3, 6, and 12 months post-treatment. CCIS is a validated 5-questions tool assessing the frequency and the type of incontinence episodes (solid, liquid, gas), pad use, and lifestyle alteration. Scored from 0 to 20, with lower scores indicating less severe symptoms.
    End point type
    Secondary
    End point timeframe
    12 months
    End point values
    		 Iltamiocel
    Number of subjects analysed
    48
    Units: scores on scales
    arithmetic mean (standard deviation)
        Change in CCIS score at 3 months
    -2.2 ± 3.0
        Change in CCIS score at 6 months
    -2.0 ± 2.8
        Change in CCIS score at 12 months
    -2.9 ± 2.8
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    12 months
    Adverse event reporting additional description
    Collection at baseline, 1 month, 3 months, 6 months and 12 months post-treatment
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    Iltamiocel
    Reporting group description
    		 AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intrasphincteric injection of 250 x 10^6 cells.

    Serious adverse events
    		 Iltamiocel
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 48 (8.33%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Surgical and medical procedures
    Sinus operation
         subjects affected / exposed
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Small intestine obstruction
         subjects affected / exposed
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Nephrotic syndrome
         subjects affected / exposed
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    2 / 48 (4.17%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Urosepsis
         subjects affected / exposed
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Iltamiocel
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    30 / 48 (62.50%)
    Injury, poisoning and procedural complications
    Post procedural contusion
         subjects affected / exposed
    5 / 48 (10.42%)
         occurrences all number
    5
    Procedural pain
         subjects affected / exposed
    13 / 48 (27.08%)
         occurrences all number
    13
    Nervous system disorders
    Hypoesthesia
         subjects affected / exposed
    5 / 48 (10.42%)
         occurrences all number
    5
    Gastrointestinal disorders
    Diarrhea
         subjects affected / exposed
    8 / 48 (16.67%)
         occurrences all number
    9
    Hemorrhoids
         subjects affected / exposed
    3 / 48 (6.25%)
         occurrences all number
    3
    Proctalgia
         subjects affected / exposed
    3 / 48 (6.25%)
         occurrences all number
    4
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 48 (6.25%)
         occurrences all number
    3
    Infections and infestations
    Cystitis
         subjects affected / exposed
    3 / 48 (6.25%)
         occurrences all number
    4
    Lower respiratory tract infection
         subjects affected / exposed
    6 / 48 (12.50%)
         occurrences all number
    6
    Nasopharyngitis
         subjects affected / exposed
    4 / 48 (8.33%)
         occurrences all number
    4
    Urinary tract infection
         subjects affected / exposed
    5 / 48 (10.42%)
         occurrences all number
    7

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 Jun 2013
    Substantial amendment 01 to protocol for use in Canada.
    04 May 2015
    Substantial amendment 03 to protocol for use in Canada.
    30 Sep 2015
    Substantial amendment 03 to protocol for use in United Kingdom.
    26 May 2016
    Substantial amendment 04 to protocol for use in Canada.
    06 Mar 2017
    Substantial amendment 08 to protocol for use in United Kingdom.

    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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