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    Clinical Trial Results:
    A Randomized, Open-label, 24-Week Safety, Efficacy, and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age with Short Bowel Syndrome Who are Dependent on Parenteral Support

    Summary
    EudraCT number
    		 2017-003606-40
    Trial protocol
    		 GB   FI   IT   FR  
    Global end of trial date
    24 Sep 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Mar 2021
    First version publication date
    28 Mar 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    SHP633-301
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03571516
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Shire
    Sponsor organisation address
    300 ShireWay, Lexington, United States, MA 02421
    Public contact
    Study Director, Shire, +1 866 842 5335, ClinicalTransparency@shire.com
    Scientific contact
    Study Director, Shire, +1 866 842 5335, ClinicalTransparency@shire.com
    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
    24 Sep 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    24 Sep 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study was to evaluate the safety, efficacy/pharmacodynamics(PD) and pharmacokinetics (PK) of teduglutide treatment in infants with short bowel syndrome (SBS) dependent on parenteral support.
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles that have their origins in the Declaration of Helsinki and in compliance with all applicable industry regulations, International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) Guideline E6 (1996), European Union (EU) Directive 2001/20/EC, as well as all applicable national and local laws and regulations.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    31 Aug 2018
    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
    Finland: 2
    Country: Number of subjects enrolled
    United Kingdom: 8
    Worldwide total number of subjects
    10
    EEA total number of subjects
    2
    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)
    10
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study was conducted at Finland and United Kingdom between 31 August 2018 (first subject first visit) and 24 September 2020 (last subject last visit).

    Pre-assignment
    Screening details
    		 A total of 10 subjects were enrolled into the study, of which 8 subjects completed the study.

    Period 1
    Period 1 title
    Overall study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Teduglutide (TED)
    Arm description
    		 Subjects (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection once daily (QD) in addition to standard medical therapy for 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Revestive
    Investigational medicinal product code
    A16AX08
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects (infants) received 0.05 mg/kg of teduglutide SC injection into abdomen or into either the thigh or arm.

    Arm title
    		 Standard of Care (SOC)
    Arm description
    		 Subjects (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		Teduglutide (TED) Standard of Care (SOC)
    Started
    5
    5
    Completed
    4
    4
    Not completed
    1
    1
         Consent withdrawn by subject
    -
    1
         Severe diarrhea
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Teduglutide (TED)
    Reporting group description
    		 Subjects (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection once daily (QD) in addition to standard medical therapy for 24 weeks.

    Reporting group title
    Standard of Care (SOC)
    Reporting group description
    		 Subjects (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.

    Reporting group values
    		 Teduglutide (TED) Standard of Care (SOC) Total
    Number of subjects
    		 5 5 10
    Age Categorical
    Units: Subjects
    Age Continuous
    Units: months
        arithmetic mean (standard deviation)
    		 8.5 ( 3.09 ) 8.3 ( 2.65 ) -
    Gender Categorical
    Units: Subjects
        Female
    		 1 2 3
        Male
    		 4 3 7
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0 0
        Not Hispanic or Latino
    		 5 5 10
    Race
    Units: Subjects
        White
    		 4 4 8
        Asian
    		 1 1 2

    End points

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    End points reporting groups
    Reporting group title
    Teduglutide (TED)
    Reporting group description
    		 Subjects (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection once daily (QD) in addition to standard medical therapy for 24 weeks.

    Reporting group title
    Standard of Care (SOC)
    Reporting group description
    		 Subjects (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.

    Primary: Number of Subjects Who Achieved At least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Number of Subjects Who Achieved At least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) [1]
    End point description
    Number of subjects who achieved at least 20% reduction from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. Intent-to-Treat (ITT) set consisted of all subjects randomized in the study.
    End point type
    Primary
    End point timeframe
    Baseline, EOT/ET (up to 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: No statistical and comparision analysis were performed for this endpoint.
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: Subjects
        Dairy Data: EOT/ET
    3
    1
        Prescribed Data: EOT/ET
    3
    3
    No statistical analyses for this end point

    Secondary: Plasma Concentration of Teduglutide at Nominal Time Points (Baseline at Pre-dose, and 1 Hour and 4 Hours Post-dose; 2 Hours Post-dose at Week 7)

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    End point title
    		 Plasma Concentration of Teduglutide at Nominal Time Points (Baseline at Pre-dose, and 1 Hour and 4 Hours Post-dose; 2 Hours Post-dose at Week 7) [2]
    End point description
    Mean plasma concentration of teduglutide at nominal time points was reported. Pharmacokinetic (PK) set included all subjects who received at least one dose of teduglutide and have at least one evaluable and interpretable post-dose PK concentration value. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint and n=number of subjects analysed refer to the subjects evaluable for this specific time points. Data was not planned to be collected and analysed for SOC arm.
    End point type
    Secondary
    End point timeframe
    Baseline: Pre-dose, 1, 4 hours post-dose, and 2 hours post-dose on Week 7
    Notes
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” arm.
    End point values
    		 Teduglutide (TED)
    Number of subjects analysed
    4
    Units: Nanogram per milliliter (ng/mL)
    median (full range (min-max))
        Baseline: At Pre-dose (n=4)
    0.00 (0.000 to 0.00)
        Baseline: At 1 hour (n=3)
    16.300 (7.25 to 25.70)
        Baseline: At 4 hour (n=4)
    8.385 (3.86 to 22.5)
        Week 7: At 2 hours (n=2)
    16.950 (14.80 to 19.10)
    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 any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. TEAEs are defined as AEs that start or deteriorate on or after the date of the first dose of investigational product. Safety set consisted of all subjects who met the following criteria: Teduglutide treatment arm: subjects who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: subjects who had undergone at least 1 post baseline safety assessment.
    End point type
    Secondary
    End point timeframe
    From start of study treatment up to end of study (EOS) (up to Week 28)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: Subjects
    5
    5
    No statistical analyses for this end point

    Secondary: Change From Baseline in Body Weight Z-score at Week 24

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    End point title
    		 Change From Baseline in Body Weight Z-score at Week 24
    End point description
    Body weight was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in body weight Z-score at Week 24 was reported. Safety set consisted of all subjects who met the following criteria: Teduglutide treatment arm: subjects who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: subjects who had undergone at least 1 post baseline safety assessment. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    4
    3
    Units: Z-score
        arithmetic mean (standard deviation)
    -0.408 ( 0.377 )
    -0.289 ( 0.278 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Length Z-Score at Week 24

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    End point title
    		 Change From Baseline in Length Z-Score at Week 24
    End point description
    Length was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in length Z-score at Week 24 was reported. Safety set consisted of all subjects who met the following criteria: Teduglutide treatment arm: subjects who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: subjects who had undergone at least 1 post baseline safety assessment. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    4
    2
    Units: Z-Score
        arithmetic mean (standard deviation)
    -0.274 ( 1.258 )
    -0.422 ( 0.384 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Head Circumference Z-Score at Week 24

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    End point title
    		 Change From Baseline in Head Circumference Z-Score at Week 24
    End point description
    Head circumference was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in head circumference Z-score at Week 24 was reported. Safety set consisted of all subjects who met the following criteria: Teduglutide treatment arm: subjects who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: subjects who had undergone at least 1 post baseline safety assessment. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    4
    2
    Units: Z-Score
        arithmetic mean (standard deviation)
    -0.544 ( 0.446 )
    -0.167 ( 0.690 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Weight-for-Length Z-Score at Week 24

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    End point title
    		 Change From Baseline in Weight-for-Length Z-Score at Week 24
    End point description
    Weight-for-length was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in weight-for-length Z-score at Week 24 was reported. Safety set consisted of all subjects who met the following criteria: Teduglutide treatment arm: subjects who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: subjects who had undergone at least 1 post baseline safety assessment. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    4
    3
    Units: Z-Score
        arithmetic mean (standard deviation)
    -0.447 ( 1.042 )
    -0.058 ( 0.992 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Average Total Urine Output at Week 24

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    End point title
    		 Change From Baseline in Average Total Urine Output at Week 24
    End point description
    Average total urine output was recorded over a 48-hour period of nutritional stability at Week 24 was reported. Safety set consisted of all subjects who met the following criteria: Teduglutide treatment arm: subjects who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: subjects who had undergone at least 1 post baseline safety assessment. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint. Here, milliliter per kilogram per day is abbreviated as mL/kg/day.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    3
    3
    Units: mL/kg/day
        arithmetic mean (standard deviation)
    -0.61 ( 14.548 )
    10.25 ( 17.383 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Fecal Output at Week 24

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    End point title
    		 Change From Baseline in Fecal Output at Week 24
    End point description
    Change from baseline in the fecal output (Average number of stools per day) at Week 24 was reported. Safety set consisted of all subjects who met the following criteria: Teduglutide treatment arm: subjects who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: subjects who had undergone at least 1 post baseline safety assessment. Here, the number of subjects analysed refer to the subjects evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    3
    3
    Units: Average number of stools per day
        arithmetic mean (standard deviation)
    -3.33 ( 3.547 )
    1.67 ( 1.756 )
    No statistical analyses for this end point

    Secondary: Number of Subjects With Positive Specific Antibodies to Teduglutide

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    End point title
    		 Number of Subjects With Positive Specific Antibodies to Teduglutide [3]
    End point description
    Number of subjects with positive specific antibodies to teduglutide were used to summarize the presence of antibodies. Safety set consisted of all subjects who met the following criteria: Teduglutide treatment arm: subjects who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: subjects who had undergone at least 1 post baseline safety assessment. Here, the number of subjects analysed signifies subjects who were evaluable for this endpoint. Data for this endpoint was not planned to be collected and analyzed for SOC group.
    End point type
    Secondary
    End point timeframe
    Baseline, EOS (up to week 28)
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: This endpoint was only planned to be analysed for the reporting group of “Teduglutide” arm.
    End point values
    		 Teduglutide (TED)
    Number of subjects analysed
    3
    Units: Subjects
    0
    No statistical analyses for this end point

    Secondary: Number of Subjects Who Achieved At least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Number of Subjects Who Achieved At least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET)
    End point description
    Number of subjects who achieved at least 20% reduction from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. ITT set consisted of all subjects randomized in the study.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: Subjects
        Dairy Data: EOT/ET
    3
    1
        Prescribed Data: EOT/ET
    3
    3
    No statistical analyses for this end point

    Secondary: Number of Subjects Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at Week 24

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    End point title
    		 Number of Subjects Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at Week 24
    End point description
    Number of subjects who achieved 100% reduction in complete weaning off (enteral autonomy) PS volume at Week 24 were reported. ITT set consisted of all subjects randomized in the study. Here, the number of subjects analysed signifies subjects who were evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Week 24
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    4
    4
    Units: Subjects
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Subjects Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at End of Study (EOS)

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    End point title
    		 Number of Subjects Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at End of Study (EOS)
    End point description
    Number of subjects who achieved 100% reduction in complete weaning off (enteral autonomy) PS volume at EOS (up to Week 28) were reported. ITT set consisted of all subjects randomized in the study. Here, the number of subjects analysed signifies subjects who were evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    EOS (up to Week 28)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    4
    5
    Units: Subjects
    0
    0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)
    End point description
    Change from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. ITT set consisted of all subjects randomized in the study. Here, n=number of subjects analysed refer to the subjects evaluable for this specific time points.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: mL/kg/day
    arithmetic mean (standard deviation)
        Dairy Data: EOT/ET (n=5, 3)
    -21.54 ( 28.909 )
    -9.51 ( 7.497 )
        Prescribed Data: EOT/ET (n=5, 5)
    -22.90 ( 26.940 )
    -14.90 ( 12.323 )
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)
    End point description
    Percent change from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. ITT set consisted of all subjects randomized in the study. Here, n=number of subjects analysed refer to the subjects evaluable for this specific time points.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: Percent change
    arithmetic mean (standard deviation)
        Dairy Data: EOT/ET (n=5, 3)
    -24.77 ( 34.723 )
    -16.75 ( 16.392 )
        Prescribed Data: EOT/ET (n=5, 5)
    -27.28 ( 33.518 )
    -22.39 ( 17.198 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET)
    End point description
    Change from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day). ITT set consisted of all subjects randomized in the study. Here, n=number of subjects analysed refer to the subjects evaluable for this specific time points.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: kcal/kg/day
    arithmetic mean (standard deviation)
        Dairy Data: EOT/ET (n=5, 3)
    -16.14 ( 17.547 )
    -6.10 ( 10.386 )
        Prescribed Data: EOT/ET (n=5, 5)
    -15.31 ( 17.839 )
    -20.40 ( 21.024 )
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET)
    End point description
    Percent change from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. ITT set consisted of all subjects randomized in the study. Here, n=number of subjects analysed refer to the subjects evaluable for this specific time points.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: Percent change
    arithmetic mean (standard deviation)
        Dairy Data: EOT/ET (n=5, 3)
    -27.00 ( 29.473 )
    -13.68 ( 21.873 )
        Prescribed Data: EOT/ET (n=5, 5)
    -27.81 ( 30.777 )
    -38.86 ( 39.893 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET)
    End point description
    Change from baseline in weight-normalized EN volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. ITT set consisted of all subjects randomized in the study. Here, n=number of subjects analysed refer to the subjects evaluable for this specific time points.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: mL/kg/day
    arithmetic mean (standard deviation)
        Dairy Data: EOT/ET (n=4, 3)
    16.14 ( 18.683 )
    -15.25 ( 31.496 )
        Prescribed Data: EOT/ET (n=4, 5)
    -1.28 ( 2.563 )
    2.27 ( 22.232 )
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) volume at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) volume at End of Treatment/Early Termination (EOT/ET)
    End point description
    Percent change from baseline in weight-normalized EN volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. ITT set consisted of all subjects randomized in the study. Here, '99999' indicates that standard deviation was not analysed for this category as there were only one subjects available at specified time points. Here, n=number of subjects analysed refer to the subjects evaluable for this specific time points.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: Percent change
    arithmetic mean (standard deviation)
        Dairy Data: EOT/ET (n=2, 2)
    273.20 ( 246.784 )
    -44.25 ( 78.847 )
        Prescribed Data: EOT/ET (n=1, 4)
    -16.40 ( 99999 )
    14.80 ( 69.834 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET)
    End point description
    Change from baseline in weight-normalized EN caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. ITT set consisted of all subjects randomized in the study. Here, n=number of subjects analysed refer to the subjects evaluable for this specific time points.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: kcal/kg/day
    arithmetic mean (standard deviation)
        Dairy Data: EOT/ET (n=4, 3)
    9.08 ( 10.662 )
    -9.38 ( 21.402 )
        Prescribed Data: EOT/ET (n=4, 5)
    -1.15 ( 2.306 )
    3.11 ( 16.285 )
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET)
    End point description
    Percent change from baseline in weight-normalized EN caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. ITT set consisted of all subjects randomized in the study. Here, '99999' indicates that standard deviation was not analysed for this category as there were only one subjects available at specified time points. Here, n=number of subjects analysed refer to the subjects evaluable for this specific time points.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: Percent change
    arithmetic mean (standard deviation)
        Dairy Data: EOT/ET (n=2, 2)
    207.06 ( 153.159 )
    -44.25 ( 78.847 )
        Prescribed Data: EOT/ET (n=1, 4)
    -16.40 ( 99999 )
    24.18 ( 78.115 )
    No statistical analyses for this end point

    Secondary: Number of Subjects Who Achieved At least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Number of Subjects Who Achieved At least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET)
    End point description
    Number of subjects who achieved at least 20% increase from baseline in weight-normalized EN volume at EOT/ET was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. ITT set consisted of all subjects randomized in the study.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: Subjects
        Dairy Data: EOT/ET
    2
    0
        Prescribed Data: EOT/ET
    0
    2
    No statistical analyses for this end point

    Secondary: Number of Subjects Who Achieved At least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

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    End point title
    		 Number of Subjects Who Achieved At least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET)
    End point description
    Number of subjects who achieved at least 20% increase from baseline in weight-normalized EN caloric intake at EOT/ET was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. ITT set consisted of all subjects randomized in the study.
    End point type
    Secondary
    End point timeframe
    Baseline, EOT/ET (up to Week 24)
    End point values
    		 Teduglutide (TED) Standard of Care (SOC)
    Number of subjects analysed
    5
    5
    Units: Subjects
        Dairy Data: EOT/ET
    2
    0
        Prescribed Data: EOT/ET
    0
    2
    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 end of study (EOS) (up to Week 28)
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    21.0
    Reporting groups
    Reporting group title
    Teduglutide
    Reporting group description
    		 Subjects (infants) 4 to 12 months of corrected gestational aged received 0.05 mg/kg of teduglutide SC injection once daily for 24 weeks.

    Reporting group title
    Standard of Care (SOC)
    Reporting group description
    		 Subjects (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.

    Serious adverse events
    		 Teduglutide Standard of Care (SOC)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 5 (80.00%)
    3 / 5 (60.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Investigations
    Transaminases increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Ataxia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune system disorders
    Immunisation reaction
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Device related infection
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 5 (40.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Product issues
    Device breakage
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Device leakage
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Device occlusion
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Metabolic acidosis
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Teduglutide Standard of Care (SOC)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 5 (100.00%)
    5 / 5 (100.00%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Blood iron decreased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Faecal volume increased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Respiratory rate increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Serum ferritin decreased
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Transaminases increased
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    2
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Lip injury
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Skin abrasion
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    1
    Secretion discharge
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Abdominal distension
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    3
    0
    Abnormal faeces
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Constipation
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Diarrhoea
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 5 (0.00%)
         occurrences all number
    3
    0
    Faeces discoloured
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Flatulence
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Frequent bowel movements
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Gastrointestinal sounds abnormal
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Mucous stools
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Retching
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Teething
         subjects affected / exposed
    0 / 5 (0.00%)
    2 / 5 (40.00%)
         occurrences all number
    0
    3
    Vomiting
         subjects affected / exposed
    3 / 5 (60.00%)
    1 / 5 (20.00%)
         occurrences all number
    8
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    1
    Rhinorrhoea
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    1
    Skin and subcutaneous tissue disorders
    Dermatitis diaper
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Eczema
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Rash papular
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Psychiatric disorders
    Irritability
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Sleep disorder
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Gastroenteritis norovirus
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Hand-foot-and-mouth disease
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Medical device site infection
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    2 / 5 (40.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Respiratory tract infection viral
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 5 (20.00%)
    2 / 5 (40.00%)
         occurrences all number
    1
    2
    Viral infection
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    1
    Product issues
    Device breakage
         subjects affected / exposed
    1 / 5 (20.00%)
    1 / 5 (20.00%)
         occurrences all number
    1
    1
    Device occlusion
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Hypoglycaemia
         subjects affected / exposed
    0 / 5 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Hypophagia
         subjects affected / exposed
    1 / 5 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    18 Jan 2018
    Protocol Amendment 1: - Clarification that subjects must have been 4 to 12 months corrected gestational age at screening. - Pharmacokinetic sampling on Week 6 was moved to Week 7 so that samples could be collected without exceeding the weekly/monthly total blood volume restrictions. Clarification that blood for post-dose PK samples might be taken within ±10 minutes of the time pre-specified. - Teduglutide dose adjustments were changed to Week 12 rather than at every clinic visit to reduce the site burden. - Assessment of the 5-level EuroQol five dimensions questionnaire was removed to reduce the caregiver burden.
    04 Dec 2018
    Protocol Amendment 2: - The dose selection rationale was updated with results from a simulation work using the previous population PK model. Based on the totality of clinical data, 0.05 mg/kg once daily was expected to provide comparable Cmax concentrations in infants as compared to pediatric subjects with SBS and was recommended as an evaluation dosing regimen in Study SHP633-301.
    24 May 2019
    Amendment 3: - The significant change in this amendment included the deletion of inclusion criterion 6: “Lack of terminal ileum and ileocecal valve.” due to difficulties in enrollment.
    17 Dec 2019
    Protocol Amendment 4: - Specification that the pre-dose PK sample was not to be collected from subjects who weighed lesser than (<) 7 kilograms (kg). The optional PK measurement at Week 12 was removed; postbaseline PK samples were to be performed at Week 7. The information to be collected for PK assessments was clarified. - Clarification that subjects might enroll in an extension study at EOS if that study was open to enrollment at the time of the SHP633-301 EOS.

    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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