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    Clinical Trial Results:
    A 24-Week Double-blind, Safety, Efficacy, and Pharmacodynamic Study Investigating Two Doses of Teduglutide in Pediatric Subjects Through 17 Years of Age with Short Bowel Syndrome who are Dependent on Parenteral Support

    Summary
    EudraCT number
    		 2015-002252-27
    Trial protocol
    		 GB   IT   FI   BE  
    Global end of trial date
    18 Aug 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Mar 2018
    First version publication date
    04 Mar 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    TED-C14-006
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02682381
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Shire
    Sponsor organisation address
    300 Shire Way, Lexington, MA, United States, 02421
    Public contact
    Study Physician, Shire, 1 866-842-5335,
    Scientific contact
    Study Physician, Shire, 1 866-842-5335,
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000482-PIP01-08
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    18 Aug 2017
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Aug 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of this study was to evaluate the safety, pharmacokinetics (PK), and efficacy/pharmacodynamics (PD) of teduglutide in pediatric subjects through 17 years of age with short bowel syndrome (SBS) and who are dependent on parenteral support.
    Protection of trial subjects
    This study was conducted in accordance with current applicable regulations, International Council for Harmonisation (ICH) of Good Clinical Practice, the principles of the Declaration of Helsinki, as well as other applicable local ethical and legal requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    03 Jun 2016
    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
    Belgium: 1
    Country: Number of subjects enrolled
    Canada: 5
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Finland: 3
    Country: Number of subjects enrolled
    United Kingdom: 8
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    United States: 39
    Worldwide total number of subjects
    59
    EEA total number of subjects
    15
    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)
    1
    Children (2-11 years)
    53
    Adolescents (12-17 years)
    5
    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
    		 Study was conducted at 27 study centers in the United States, Belgium, Canada, the United Kingdom, Finland, Germany and Italy between 03 June 2016 (first subject first visit) and 18 August 2017 (last subject last visit).

    Pre-assignment
    Screening details
    		 Overall, 59 subjects were enrolled; 50 in the teduglutide treatment arm (24 subjects in the 0.025 milligram per kilogram per day (mg/kg/day) dose group and 26 subjects in the 0.05 mg/kg/day dose group) and 9 in the standard of care arm.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 0.025 mg/kg/day Teduglutide
    Arm description
    		 Subjects received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Teduglutide
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects received teduglutide subcutaneously for 24 weeks along with standard medical therapy.

    Arm title
    		 0.05 mg/kg/day Teduglutide
    Arm description
    		 Subjects received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Teduglutide
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects received teduglutide subcutaneously for 24 weeks along with standard medical therapy.

    Arm title
    		 Standard of care
    Arm description
    		 Subjects received standard medical therapy.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Started
    24
    26
    9
    Completed
    24
    26
    9

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    0.025 mg/kg/day Teduglutide
    Reporting group description
    		 Subjects received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.

    Reporting group title
    0.05 mg/kg/day Teduglutide
    Reporting group description
    		 Subjects received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.

    Reporting group title
    Standard of care
    Reporting group description
    		 Subjects received standard medical therapy.

    Reporting group values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care Total
    Number of subjects
    		 24 26 9
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 6.6 ± 3.61 6.2 ± 3.67 5.7 ± 4.72 -
    Gender categorical
    Units: Subjects
        Female
    		 8 7 3 18
        Male
    		 16 19 6 41
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 5 5 4 14
        Not Hispanic or Latino
    		 16 20 2 38
        Unknown or Not Reported
    		 3 1 3 7
    Race
    Units: Subjects
        White
    		 16 21 2 39
        Black or African American
    		 3 3 1 7
        Asian
    		 1 1 1 3
        American Indian or Alaska Native
    		 0 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0
        Other
    		 1 0 2 3
        Not allowed based on local regulations
    		 3 1 3 7

    End points

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    End points reporting groups
    Reporting group title
    0.025 mg/kg/day Teduglutide
    Reporting group description
    		 Subjects received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.

    Reporting group title
    0.05 mg/kg/day Teduglutide
    Reporting group description
    		 Subjects received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.

    Reporting group title
    Standard of care
    Reporting group description
    		 Subjects received standard medical therapy.

    Primary: Number of Subjects who Achieved at Least a 20 Percent (%) Reduction in Weight-Normalized Average Daily Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24

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    End point title
    		 Number of Subjects who Achieved at Least a 20 Percent (%) Reduction in Weight-Normalized Average Daily Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24 [1]
    End point description
    Reduction in weight-normalized PN/IV volume was performed using both subject diary and investigator prescribed data. Number of subjects who achieved at least a 20% reduction in weight-normalized PN/IV volume between the baseline and week 24/end of treatment (EOT) visit were reported. Intention to treat (ITT) population included all enrolled subjects.
    End point type
    Primary
    End point timeframe
    Week 24
    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 statistics were done, no inferential statistical analyses were performed.
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Subject
        Subject Diary
    13
    18
    1
        Investigator Prescribed
    13
    18
    2
    No statistical analyses for this end point

    Secondary: Number of Subjects With Treatment-emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Subjects With Treatment-emergent Adverse Events (TEAEs)
    End point description
    An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product and that did not necessarily have a causal relationship with this treatment. TEAEs were defined as AEs that started or worsened on or after the date of first dose for treatment groups and those that started or worsened on or after the baseline visit for standard of care group. Safety analysis population included all subjects who received at least 1 dose of teduglutide and had undergone at least 1 post-baseline safety assessment in teduglutide treatment group or subjects who had undergone at least 1 post-baseline safety assessment in standard of care treatment group.
    End point type
    Secondary
    End point timeframe
    From start of study treatment up to 28 weeks
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Subject
        Subject
    24
    25
    9
    No statistical analyses for this end point

    Secondary: Number of Subjects Who Were Completely Weaned off Parenteral Nutrition Intravenous (PN/IV) Support at Week 24

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    End point title
    		 Number of Subjects Who Were Completely Weaned off Parenteral Nutrition Intravenous (PN/IV) Support at Week 24
    End point description
    A subject was considered to have achieved independence from PN/IV support (completely weaned off PN/IV) if the investigator prescribed no PN/IV at EOT and there was no use of PN/IV recorded in the subject diary during the week prior to EOT. ITT population included all enrolled subjects.
    End point type
    Secondary
    End point timeframe
    Week 24
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Subject
        Subject
    2
    3
    0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24

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    End point title
    		 Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24
    End point description
    Change in PN/IV volume was reported based on the subject diary and the investigator prescribed data. ITT population included all enrolled subjects. In the below table, "n" signifies subjects who were evaluable for the specified category, respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Milliliter per kilogram per day
    arithmetic mean (standard deviation)
        Subject Diary (n=20,25,9)
    -16.16 ± 10.52
    -23.30 ± 17.50
    -6.03 ± 4.55
        Investigator Prescribed (n=24,26,9)
    -11.28 ± 15.51
    -22.13 ± 17.92
    -5.84 ± 9.80
    No statistical analyses for this end point

    Secondary: Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Calories at Week 24

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    End point title
    		 Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Calories at Week 24
    End point description
    Change in PN/IV calories was reported based on the subject diary and the investigator prescribed data. ITT population included all enrolled subjects. In the below table, "n" signifies subjects who were evaluable for the specified category, respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Kilocalorie per kilogram per day
    arithmetic mean (standard deviation)
        Subject Diary (n=20,25,9)
    -14.92 ± 8.29
    -18.99 ± 14.28
    -0.46 ± 4.95
        Investigator Prescribed (n=24,25,9)
    -11.76 ± 10.46
    -18.51 ± 13.22
    -0.27 ± 2.73
    No statistical analyses for this end point

    Secondary: Change From Baseline in Plasma Citrulline Levels at Week 24

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    End point title
    		 Change From Baseline in Plasma Citrulline Levels at Week 24
    End point description
    Plasma citrulline levels was assessed. ITT population included all enrolled subjects.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Micromole per liter (mcmol/L)
    arithmetic mean (standard deviation)
        Change at baseline (n=21,24,8)
    7.7 ± 8.50
    12.0 ± 12.0
    0.1 ± 7.79
    No statistical analyses for this end point

    Secondary: Change From Baseline in Enteral Nutritional Volume at Week 24

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    End point title
    		 Change From Baseline in Enteral Nutritional Volume at Week 24
    End point description
    Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Enteral Nutritional Volume was assessed. ITT population included all enrolled subjects. In the below table, "n" signifies subjects who were evaluable for the specified category, respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Milliliter per kilogram per day
    arithmetic mean (standard deviation)
        Subject Diary (n=18,25,9)
    7.69 ± 13.46
    10.96 ± 16.59
    0.74 ± 5.91
        Investigator Prescribed (n=18,21,5)
    7.67 ± 17.77
    8.17 ± 17.87
    0.33 ± 0.90
    No statistical analyses for this end point

    Secondary: Change From Baseline in Enteral Nutritional Calories at Week 24

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    End point title
    		 Change From Baseline in Enteral Nutritional Calories at Week 24
    End point description
    Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Enteral Nutritional Calories was assessed. ITT population included all enrolled subjects. In the below table, "n" signifies subjects who were evaluable for the specified category, respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Kilocalorie per kilogram per day
    arithmetic mean (standard deviation)
        Subject Diary (n=18,25,9)
    8.43 ± 14.39
    12.98 ± 18.93
    4.22 ± 13.75
        Investigator Prescribed (n=18,21,5)
    7.29 ± 15.88
    11.47 ± 21.13
    6.66 ± 14.77
    No statistical analyses for this end point

    Secondary: Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 28

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    End point title
    		 Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 28
    End point description
    Parenteral nutrition intravenous (PN/IV) volume was assessed. ITT population included all enrolled subjects. In the below table, "n" signifies subjects who were evaluable for the specified category, respectively.
    End point type
    Secondary
    End point timeframe
    Week 24, Week 28
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Milliliter per kilogram per day
    arithmetic mean (standard deviation)
        Subject Diary (n=24,26,8)
    2.63 ± 7.80
    1.52 ± 12.68
    -2.99 ± 4.94
        Investigator Prescribed (n=24,26,9)
    2.13 ± 6.95
    -0.35 ± 5.96
    -0.91 ± 2.18
    No statistical analyses for this end point

    Secondary: Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Calories at Week 28

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    End point title
    		 Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Calories at Week 28
    End point description
    Parenteral Nutrition Intravenous (PN/IV) Calories was assessed. ITT population included all enrolled subjects. In the below table, "n" signifies subjects who were evaluable for the specified category, respectively.
    End point type
    Secondary
    End point timeframe
    Week 24, Week 28
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Kilocalorie per kilogram per day
    arithmetic mean (standard deviation)
        Subject Diary (n=24,26,8)
    0.88 ± 2.94
    -0.91 ± 7.14
    -4.21 ± 10.30
        Investigator Prescribed (n=24,26,9)
    0.55 ± 3.42
    0.08 ± 6.13
    -4.34 ± 11.22
    No statistical analyses for this end point

    Secondary: Change From Week 24 in Plasma Citrulline Levels at Week 28

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    End point title
    		 Change From Week 24 in Plasma Citrulline Levels at Week 28
    End point description
    Plasma Citrulline Levels was assessed. ITT population included all enrolled subjects.
    End point type
    Secondary
    End point timeframe
    Week 24, Week 28
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: mcmol/L
    arithmetic mean (standard deviation)
        Change at Week 28 (n=24,25,9)
    -6.0 ± 9.26
    -9.5 ± 10.33
    1.8 ± 6.48
    No statistical analyses for this end point

    Secondary: Change From Week 24 in Enteral Nutritional Volume at Week 28

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    End point title
    		 Change From Week 24 in Enteral Nutritional Volume at Week 28
    End point description
    Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Enteral Nutritional Volume was assessed. ITT population included all enrolled subjects. In the below table, "n" signifies subjects who were evaluable for the specified category, respectively.
    End point type
    Secondary
    End point timeframe
    Week 24, Week 28
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Milliliter per kilogram per day
    arithmetic mean (standard deviation)
        Subject Diary (n=23,26,8)
    -0.68 ± 3.86
    1.38 ± 8.41
    -0.75 ± 6.78
        Investigator Prescribed (n=18,22,5)
    1.67 ± 5.73
    0.68 ± 6.65
    0.39 ± 0.57
    No statistical analyses for this end point

    Secondary: Change From Week 24 in Enteral Nutritional Calories at Week 28

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    End point title
    		 Change From Week 24 in Enteral Nutritional Calories at Week 28
    End point description
    Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Enteral Nutritional Calories was assessed. ITT population included all enrolled subjects. In the below table, "n" signifies subjects who were evaluable for the specified category, respectively.
    End point type
    Secondary
    End point timeframe
    Week 24, Week 28
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Kilocalorie per kilogram per day
    arithmetic mean (standard deviation)
        Subject Diary (n=23,26,8)
    -1.07 ± 4.36
    -0.14 ± 7.73
    -0.42 ± 6.13
        Investigator Prescribed (n=18,22,5)
    1.28 ± 4.30
    -0.11 ± 3.77
    0.45 ± 0.64
    No statistical analyses for this end point

    Secondary: Change From Baseline in Body Weight Z-score at Week 28

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    End point title
    		 Change From Baseline in Body Weight Z-score at Week 28
    End point description
    Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age greater than or equal to [>=] 2 years old) and World Health Organization (age less than [<] 2 years old) Z-score calculation charts were used for calculation. Safety analysis population included all subjects who received at least 1 dose of teduglutide and had undergone at least 1 post-baseline safety assessment in teduglutide treatment group or subjects who had undergone at least 1 post-baseline safety assessment in standard of care treatment group.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Z-score
    arithmetic mean (standard deviation)
        Z-score
    -0.12 ± 0.41
    -0.18 ± 0.59
    0.05 ± 0.37
    No statistical analyses for this end point

    Secondary: Change From Baseline in Body Height Z-score at Week 28

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    End point title
    		 Change From Baseline in Body Height Z-score at Week 28
    End point description
    Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts were used for calculation. Safety analysis population included all subjects who received at least 1 dose of teduglutide and had undergone at least 1 post-baseline safety assessment in teduglutide treatment group or subjects who had undergone at least 1 post-baseline safety assessment in standard of care treatment group.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    25
    9
    Units: Z-score
    arithmetic mean (standard deviation)
        Z-score
    0 ± 0.29
    0.05 ± 0.45
    0.16 ± 0.66
    No statistical analyses for this end point

    Secondary: Change From Baseline in Head Circumference Z-score at Week 28

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    End point title
    		 Change From Baseline in Head Circumference Z-score at Week 28
    End point description
    Head circumference was collected only for subjects of less than or equal to (<=) 36 months of age at the time of measurement. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts were used for calculation. Safety analysis population included all subjects who received at least 1 dose of teduglutide and had undergone at least 1 post-baseline safety assessment in teduglutide treatment group or subjects who had undergone at least 1 post-baseline safety assessment in standard of care treatment group. In the below table, "99999" signifies that the standard deviation was not calculated due to less number of subjects.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    0 [2]
    0 [3]
    1
    Units: Z-score
    arithmetic mean (standard deviation)
        Z-score
    ±
    ±
    -0.014 ± 99999
    Notes
    [2] - Head circumference was planned to be collected only for subjects <= 36 months of age.
    [3] - Head circumference was planned to be collected only for subjects <= 36 months of age.
    No statistical analyses for this end point

    Secondary: Change From Baseline in Body Mass Index (BMI) Z-score at Week 28

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    End point title
    		 Change From Baseline in Body Mass Index (BMI) Z-score at Week 28
    End point description
    Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention >= 2 years old) and World Health Organization (age < 2 years old) Z-score calculation charts were used for calculation. Safety analysis population included all subjects who received at least 1 dose of teduglutide and had undergone at least 1 post-baseline safety assessment in teduglutide treatment group or subjects who had undergone at least 1 post-baseline safety assessment in standard of care treatment group.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Z-score
    arithmetic mean (standard deviation)
        Z-score (n=24,25,9)
    -0.13 ± 0.57
    -0.22 ± 0.70
    -0.25 ± 1.42
    No statistical analyses for this end point

    Secondary: Change From baseline in Subjects' Stool Consistency at Week 28

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    End point title
    		 Change From baseline in Subjects' Stool Consistency at Week 28
    End point description
    Stool consistency was assessed by typical stool form based on Bristol Stool Form Scale: 1 - Separate hard lumps, hard to pass, 2 - Sausage-shaped, but lumpy, 3 - Like a sausage but with cracks on the surface, 4 - Like a sausage or snake, smooth and soft, 5 - Soft blobs with clear-cut edges, 6 - Fluffy pieces with ragged edges, a mushy stool, 7 - Watery, no solid pieces, entirely liquid. Safety analysis population included all subjects who received at least 1 dose of teduglutide and had undergone at least 1 post-baseline safety assessment in teduglutide treatment group or subjects who had undergone at least 1 post-baseline safety assessment in standard of care treatment group.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 28
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Average Bristol Stool Form Score
    arithmetic mean (standard deviation)
        Change at baseline (n=8,16,2)
    -1.3 ± 1.77
    -1.4 ± 1.38
    -3.3 ± 0.35
    No statistical analyses for this end point

    Secondary: Change From Baseline in Hours Per Day of Parenteral Nutrition Intravenous (PN/IV) Support

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    End point title
    		 Change From Baseline in Hours Per Day of Parenteral Nutrition Intravenous (PN/IV) Support
    End point description
    The mean duration of the PN/IV infusions in hours, on the days when PN/IV was administered was assessed. ITT population included all enrolled subjects. In the below table, "n" signifies subjects who were evaluable for the specified category, respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, week 24
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Hours per day (hours/day)
    arithmetic mean (standard deviation)
        Subject Diary (n=22,26,9)
    -2.47 ± 2.73
    -3.03 ± 3.84
    -0.21 ± 0.69
        Investigator Prescribed (n=24,26,9)
    -1.48 ± 3.59
    -1.79 ± 3.52
    0.11 ± 0.33
    No statistical analyses for this end point

    Secondary: Change From Baseline in Days Per Week of Parenteral Nutrition Intravenous (PN/IV) Support at Week 24

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    End point title
    		 Change From Baseline in Days Per Week of Parenteral Nutrition Intravenous (PN/IV) Support at Week 24
    End point description
    The number of days per week of PN/IV infusions was assessed. ITT population included all enrolled subjects. In the below table, "n" signifies subjects who were evaluable for the specified category, respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Number of subjects analysed
    24
    26
    9
    Units: Days per week (Days/week)
    arithmetic mean (standard deviation)
        Subject Diary (n=22,26,9)
    -0.88 ± 1.78
    -1.34 ± 2.24
    0.0 ± 0.0
        Investigator Prescribed (n=24,26,9)
    -0.79 ± 1.62
    -1.42 ± 2.32
    0 ± 0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From start of study treatment up to 28 weeks
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.1
    Reporting groups
    Reporting group title
    0.025 mg/kg/day Teduglutide
    Reporting group description
    		 Subjects received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.

    Reporting group title
    0.05 mg/kg/day Teduglutide
    Reporting group description
    		 Subjects received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.

    Reporting group title
    Standard of care
    Reporting group description
    		 Subjects received standard medical therapy.

    Serious adverse events
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Total subjects affected by serious adverse events
         subjects affected / exposed
    15 / 24 (62.50%)
    20 / 26 (76.92%)
    4 / 9 (44.44%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    Blood urea increased
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Transaminases increased
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Superior vena cava syndrome
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    4 / 24 (16.67%)
    7 / 26 (26.92%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 6
    0 / 8
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Faecaloma
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Haematemesis
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ileus
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholestasis
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hypocapnia
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Bacteraemia
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Catheter site infection
         subjects affected / exposed
    0 / 24 (0.00%)
    3 / 26 (11.54%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Corona virus infection
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Device related infection
         subjects affected / exposed
    1 / 24 (4.17%)
    3 / 26 (11.54%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Device related sepsis
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Fungaemia
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastritis viral
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    2 / 24 (8.33%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metapneumovirus infection
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Orchitis
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Otitis media
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Otitis media acute
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Parainfluenzae virus infection
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory syncytial virus infection
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Roseola
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Rotavirus infection
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Subcutaneous abscess
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 24 (8.33%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Viral infection
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 26 (7.69%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Product issues
    Device breakage
    Additional description: TEAEs coded to Product issues were related to central line complications, and not due to complications of the investigational product and ancillary supplies.
         subjects affected / exposed
    2 / 24 (8.33%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Device dislocation
    Additional description: TEAEs coded to Product issues were related to central line complications, and not due to complications of the investigational product and ancillary supplies.
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Device issue
    Additional description: TEAEs coded to Product issues were related to central line complications, and not due to complications of the investigational product and ancillary supplies.
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Device occlusion
    Additional description: TEAEs coded to Product issues were related to central line complications, and not due to complications of the investigational product and ancillary supplies.
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Acidosis
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Dehydration
         subjects affected / exposed
    4 / 24 (16.67%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hypokalaemia
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolic acidosis
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 0.025 mg/kg/day Teduglutide 0.05 mg/kg/day Teduglutide Standard of care
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    24 / 24 (100.00%)
    23 / 26 (88.46%)
    9 / 9 (100.00%)
    General disorders and administration site conditions
    Catheter site erythema
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    2 / 9 (22.22%)
         occurrences all number
    0
    1
    2
    Catheter site related reaction
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Injection site bruising
         subjects affected / exposed
    3 / 24 (12.50%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences all number
    4
    1
    0
    Medical device site pain
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 26 (7.69%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    1
    Pain
         subjects affected / exposed
    2 / 24 (8.33%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences all number
    2
    1
    0
    Pyrexia
         subjects affected / exposed
    5 / 24 (20.83%)
    6 / 26 (23.08%)
    3 / 9 (33.33%)
         occurrences all number
    6
    9
    4
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    1 / 24 (4.17%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences all number
    1
    2
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 24 (8.33%)
    10 / 26 (38.46%)
    3 / 9 (33.33%)
         occurrences all number
    2
    11
    4
    Hyperventilation
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Nasal congestion
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    2
    Oropharyngeal pain
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Rhinorrhoea
         subjects affected / exposed
    3 / 24 (12.50%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    3
    0
    1
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Product issues
    Device breakage
         subjects affected / exposed
    2 / 24 (8.33%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences all number
    2
    2
    0
    Device occlusion
         subjects affected / exposed
    3 / 24 (12.50%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    3
    0
    0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    7 / 24 (29.17%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences all number
    7
    2
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    5 / 24 (20.83%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    5
    0
    0
    Blood bicarbonate decreased
         subjects affected / exposed
    4 / 24 (16.67%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    5
    0
    0
    Blood triglycerides increased
         subjects affected / exposed
    2 / 24 (8.33%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences all number
    2
    1
    0
    Gamma-Glutamyltransferase increased
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    0
    Lymph node palpable
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    2
    0
    1
    Transaminases increased
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Injury, poisoning and procedural complications
    Anaesthetic complication neurological
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Stoma site erythema
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences all number
    0
    3
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    2
    Headache
         subjects affected / exposed
    3 / 24 (12.50%)
    5 / 26 (19.23%)
    1 / 9 (11.11%)
         occurrences all number
    4
    7
    3
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    1
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences all number
    0
    2
    0
    Abdominal pain
         subjects affected / exposed
    4 / 24 (16.67%)
    6 / 26 (23.08%)
    0 / 9 (0.00%)
         occurrences all number
    5
    7
    0
    Abdominal pain lower
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    0
    Abdominal pain upper
         subjects affected / exposed
    3 / 24 (12.50%)
    3 / 26 (11.54%)
    1 / 9 (11.11%)
         occurrences all number
    8
    3
    1
    Diarrhoea
         subjects affected / exposed
    8 / 24 (33.33%)
    2 / 26 (7.69%)
    1 / 9 (11.11%)
         occurrences all number
    9
    3
    1
    Nausea
         subjects affected / exposed
    3 / 24 (12.50%)
    3 / 26 (11.54%)
    1 / 9 (11.11%)
         occurrences all number
    3
    3
    1
    Perianal erythema
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    2
    Proctalgia
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    2
    Vomiting
         subjects affected / exposed
    10 / 24 (41.67%)
    8 / 26 (30.77%)
    5 / 9 (55.56%)
         occurrences all number
    23
    17
    7
    Hepatobiliary disorders
    Drug-Induced liver injury
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Dermatitis diaper
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    20
    0
    0
    Eczema
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    1
    Excessive granulation tissue
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Rash
         subjects affected / exposed
    1 / 24 (4.17%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    1
    4
    1
    Red man syndrome
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Renal and urinary disorders
    Nephrolithiasis
         subjects affected / exposed
    0 / 24 (0.00%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    1 / 24 (4.17%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    1
    0
    1
    Pain in extremity
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    3
    0
    0
    Conjunctivitis
         subjects affected / exposed
    3 / 24 (12.50%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences all number
    3
    1
    0
    Device related infection
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences all number
    0
    2
    0
    Ear infection
         subjects affected / exposed
    1 / 24 (4.17%)
    3 / 26 (11.54%)
    1 / 9 (11.11%)
         occurrences all number
    1
    3
    1
    Gastroenteritis viral
         subjects affected / exposed
    3 / 24 (12.50%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    3
    0
    0
    Gastrointestinal bacterial overgrowth
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    6
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    4 / 24 (16.67%)
    6 / 26 (23.08%)
    2 / 9 (22.22%)
         occurrences all number
    4
    9
    2
    Otitis media
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Pharyngitis
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences all number
    0
    2
    0
    Respiratory tract infection
         subjects affected / exposed
    0 / 24 (0.00%)
    2 / 26 (7.69%)
    0 / 9 (0.00%)
         occurrences all number
    0
    3
    0
    Respiratory tract infection viral
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    2
    Rhinitis
         subjects affected / exposed
    1 / 24 (4.17%)
    5 / 26 (19.23%)
    0 / 9 (0.00%)
         occurrences all number
    1
    6
    0
    Upper respiratory tract infection
         subjects affected / exposed
    5 / 24 (20.83%)
    6 / 26 (23.08%)
    4 / 9 (44.44%)
         occurrences all number
    7
    8
    5
    Urinary tract infection
         subjects affected / exposed
    2 / 24 (8.33%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences all number
    4
    1
    0
    Urinary tract infection bacterial
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Viral infection
         subjects affected / exposed
    3 / 24 (12.50%)
    1 / 26 (3.85%)
    1 / 9 (11.11%)
         occurrences all number
    4
    2
    1
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 24 (0.00%)
    0 / 26 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    1
    Metabolism and nutrition disorders
    Acidosis
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    0
    Dehydration
         subjects affected / exposed
    5 / 24 (20.83%)
    1 / 26 (3.85%)
    0 / 9 (0.00%)
         occurrences all number
    9
    1
    0
    Metabolic acidosis
         subjects affected / exposed
    2 / 24 (8.33%)
    0 / 26 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    4
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    22 Jun 2015
    - Plasma citrulline to be collected from all study subjects, regardless of treatment group. - Introduced z-scores for vital signs. - Information about the subject diary was expanded to clarify the requirements for diary completion. - The lower limit of the age range for this protocol was eliminated to allow for potential enrollment of children under 1 year of age. - Weaning algorithms for toilet trained and untrained children were revised and a dehydration assessment was added.
    06 Oct 2015
    - Administration of investigational product was modified to allow for a dose adjustment at Week 12, if warranted based on subjects’ weight gain/loss over the initial 12-week treatment period. - Urine and stool collection will be done for 48 hours prior to all scheduled visits (phone and clinic) rather than for 72 hours. - Specifications for the management of nutritional support were updated based on expert opinion.
    25 Feb 2016
    - Capture of output diary data was limited to 48 hours prior to every clinic visit and phone visit.

    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
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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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