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    Clinical Trial Results:
    A Multicenter, Open-label, Metabolic Balance Study to Evaluate the Effects of Apraglutide on Intestinal Absorption in Adult Subjects with Short Bowel Syndrome, Intestinal Failure (SBS-IF), and Colon-in-continuity (CIC)

    Summary
    EudraCT number
    		 2020-005129-99
    Trial protocol
    		 FR   BE   DK  
    Global end of trial date
    06 Jun 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    15 Aug 2024
    First version publication date
    15 Aug 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    TA799-013
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04964986
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    VectivBio AG
    Sponsor organisation address
    Aeschenvorstadt 36, Basel, Switzerland, 4051
    Public contact
    Clinical Trial Information Desk, VectivBio AG, clinicaltrial.enquiries@vectivbio.com
    Scientific contact
    Clinical Trial Information Desk, VectivBio AG, clinicaltrial.enquiries@vectivbio.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    06 Jun 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Jun 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objectives of this trial were to evaluate the safety and tolerability of apraglutide and the efficacy of apraglutide in increasing intestinal energy absorption assessed by bomb calorimetry in relation to metabolic balance assessments.
    Protection of trial subjects
    This trial was performed in compliance with: • Declaration of Helsinki (revised version of Fortaleza, Brazil, 2013). • The ICH-GCP guidelines (ICH E6 (R2), November 2016). • European Union Clinical Trials Regulation No. 536/2014. • Any amendments to these regulations. • Local laws and regulations.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    14 Jun 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belgium: 8
    Country: Number of subjects enrolled
    France: 2
    Worldwide total number of subjects
    10
    EEA total number of subjects
    10
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    8
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 10 participants with SBS-IF and CIC were enrolled as planned, eight participants at the Belgium site and two participants at the France site. A total of nine participants were treated and analyzed.

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 10
    Number of subjects completed
    		 9

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 Enrolled but not treated: 1
    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Apraglutide
    Arm description
    		 All participants received, based on their body weight, either 2.5 mg (<50 kg) or 5 mg (≥50 kg) apraglutide once weekly, for 52 weeks. The investigational medicinal product (IMP) was administered subcutaneously (SC).
    Arm type
    		 Experimental

    Investigational medicinal product name
    Apraglutide
    Investigational medicinal product code
    TA799
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    The IMP was administered SC.

    Number of subjects in period 1 [1]
    		Apraglutide
    Started
    9
    Completed
    9
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: One participant was enrolled but not treated.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Apraglutide
    Reporting group description
    		 All participants received, based on their body weight, either 2.5 mg (<50 kg) or 5 mg (≥50 kg) apraglutide once weekly, for 52 weeks. The investigational medicinal product (IMP) was administered subcutaneously (SC).

    Reporting group values
    		 Apraglutide Total
    Number of subjects
    		 9 9
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 8 8
        From 65-84 years
    		 1 1
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 46.8 ( 17.46 ) -
    Gender categorical
    Units: Subjects
        Female
    		 7 7
        Male
    		 2 2
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 1 1
        Not Hispanic or Latino
    		 8 8
    Race
    Units: Subjects
        White
    		 9 9

    End points

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    End points reporting groups
    Reporting group title
    Apraglutide
    Reporting group description
    		 All participants received, based on their body weight, either 2.5 mg (<50 kg) or 5 mg (≥50 kg) apraglutide once weekly, for 52 weeks. The investigational medicinal product (IMP) was administered subcutaneously (SC).

    Primary: Number of Participants who Experienced a Treatment-Emergent Adverse Event (TEAE)

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    End point title
    		 Number of Participants who Experienced a Treatment-Emergent Adverse Event (TEAE) [1]
    End point description
    Applicable to Protocol V4.0 implemented in Belgium (classed as secondary endpoint in Protocol V3.0 [implemented in France]). A TEAE was any unfavorable and unintended sign, symptom, or disease temporally associated with apraglutide, whether or not related, that occurred or worsened after the dose of apraglutide. A serious TEAE was defined as any TEAE that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. Clinically significant changes from baseline in clinical chemistry, hematology, hemostasis, anti-drug antibodies (ADAs), and urine analysis were reported as adverse events. Adverse events of special interest (AESI): • Injection site reaction • Gastrointestinal obstruction • Gallbladder, biliary, and pancreatic disease • Fluid overload • Colorectal polyps • Malignancies
    End point type
    Primary
    End point timeframe
    Day 1 up to approximately 55 weeks
    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 additional statistical analysis was pre-specified for this endpoint.
    End point values
    		 Apraglutide
    Number of subjects analysed
    9 [2]
    Units: participants
        At least one TEAE
    9
        At least one treatment-related TEAE
    5
        At least one TESAE
    3
        At least one treatment-related TESAE
    1
        At least one treatment-emergent AESI
    3
        At least one TEAE leading to dose interruption
    1
        At least one TEAE leading to dose discontinuation
    0
    Notes
    [2] - The safety analysis set included all participants exposed to trial medication.
    No statistical analyses for this end point

    Primary: Absolute Change in Absorption of Energy Over Metabolic Balance (MB) Periods From Baseline at Week 48

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    End point title
    		 Absolute Change in Absorption of Energy Over Metabolic Balance (MB) Periods From Baseline at Week 48 [3]
    End point description
    Applicable to Protocol V3.0 implemented in France (classed as secondary endpoint in Protocol V4.0 [implemented in Belgium]). The absorption was defined as dietary intake minus output from fecal excretion over a 72-hour MB period at a given analysis time point. Since dietary intake and fecal excretion were measured daily, i.e., up to three measurements may contribute to absorption calculations, the average over all available daily absorption measurements over the 72-hour period were used for analysis. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. The increase from baseline at Week 48 was statistically significant (p-value=0.041). The p-value was computed based on the paired t-test.
    End point type
    Primary
    End point timeframe
    Baseline and Week 48
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Additional statistical analysis presented as free-text due to inability to add this for single arm trials within EudraCT.
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: kJ/day
    arithmetic mean (standard deviation)
        Week 48
    1133.963 ( 1399.8793 )
    No statistical analyses for this end point

    Secondary: Relative Change From Baseline in Actual Weekly Parenteral Support (PS) Volume at Weeks 4, 24, and 52

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    End point title
    		 Relative Change From Baseline in Actual Weekly Parenteral Support (PS) Volume at Weeks 4, 24, and 52
    End point description
    The sum of daily PS volume (including extra fluids) from weekly PS diary data recorded for the corresponding analysis timepoint was used for analysis. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 4, Week 24, and Week 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: percentage change in PS volume
    arithmetic mean (standard deviation)
        Week 4
    -0.73 ( 2.709 )
        Week 24
    -39.99 ( 22.588 )
        Week 52
    -52.44 ( 29.192 )
    No statistical analyses for this end point

    Secondary: Absolute Change From Baseline in Actual Weekly PS Volume at Weeks 24 and 52

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    End point title
    		 Absolute Change From Baseline in Actual Weekly PS Volume at Weeks 24 and 52
    End point description
    The sum of daily PS volume (including extra fluids) from weekly PS diary data recorded for the corresponding analysis timepoint was used for analysis. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. At Week 24 and Week 52, significant decreases (p-values=<0.001 for both) in mean PS volume were reported. The p-values were computed based on the paired t-test.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24 and Week 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: mL
    arithmetic mean (standard deviation)
        Week 24
    -3510.000 ( 1893.7859 )
        Week 52
    -4701.778 ( 2389.9652 )
    No statistical analyses for this end point

    Secondary: Number of Participants Considered Clinical Responders at Weeks 24 and 52

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    End point title
    		 Number of Participants Considered Clinical Responders at Weeks 24 and 52
    End point description
    Clinical response was defined as a 20% reduction of PS volume from Baseline. The sum of daily PS volume (including extra fluids) from weekly PS diary data recorded for the corresponding analysis timepoint was used for analysis. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24 and 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: participants
        Week 24
    7
        Week 52
    9
    No statistical analyses for this end point

    Secondary: Number of Participants Who Achieved Enteral Autonomy at Weeks 24 and 52

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    End point title
    		 Number of Participants Who Achieved Enteral Autonomy at Weeks 24 and 52
    End point description
    Enteral autonomy was defined as a participant not receiving PS for hydration or parenteral nutrition (PN) for calories. Participants may have still received minimal fluid to maintain patency of the central line or for specific elemental/micro-nutrient needs (e.g., <100 mL fluid for administration of magnesium). The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
    End point type
    Secondary
    End point timeframe
    Week 24 and Week 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: participants
        Week 24
    0
        Week 52
    2
    No statistical analyses for this end point

    Secondary: Absolute Change in Total Energy in PN From Baseline at Weeks 24 and 52

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    End point title
    		 Absolute Change in Total Energy in PN From Baseline at Weeks 24 and 52
    End point description
    PN was defined as PS that includes protein, carbohydrate, fat, vitamins, and/or trace elements. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. At Week 24 and Week 52, there was a significant decrease (p-value=0.002 and p-value=<0.001, respectively) in mean total energy from baseline. The p-values were computed based on the paired t-test.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24 and Week 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: kcal
    arithmetic mean (standard deviation)
        Week 24
    -2763.889 ( 1830.8065 )
        Week 52
    -3510.111 ( 1927.2066 )
    No statistical analyses for this end point

    Secondary: Number of Participants Who Achieved a Reduction of at Least 1 Day Per Week of PS From Baseline at Weeks 24 and 52

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    End point title
    		 Number of Participants Who Achieved a Reduction of at Least 1 Day Per Week of PS From Baseline at Weeks 24 and 52
    End point description
    Participants were considered to have a reduction of at least one day per week of PS from Baseline (incl. extra fluids) if the number of days with PS from weekly PS diary data recorded for the corresponding analysis timepoint was smaller compared to the number of days with PS from weekly PS diary data for Baseline. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24 and Week 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: participants
        Week 24
    5
        Week 52
    7
    No statistical analyses for this end point

    Secondary: Absolute Change in Urine Volume over MB Periods From Baseline at Week 4 and Week 48

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    End point title
    		 Absolute Change in Urine Volume over MB Periods From Baseline at Week 4 and Week 48
    End point description
    Based on average daily urine volume data derived as per balance period calculations. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. At Week 4, there was a non-significant increase from baseline (p-value=0.063). At Week 48, there was a non-significant decrease from baseline (p-value=0.112). The p-values were computed based on the paired t-test.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 4 and Week 48
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: mL/day
    arithmetic mean (standard deviation)
        Week 4
    246.889 ( 343.3609 )
        Week 48
    -178.556 ( 299.5613 )
    No statistical analyses for this end point

    Secondary: Absolute Change in Absorption of Energy Over MB Periods From Baseline at Week 4

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    End point title
    		 Absolute Change in Absorption of Energy Over MB Periods From Baseline at Week 4
    End point description
    The absorption was defined as dietary intake minus output from fecal excretion over a 72-hour MB period at a given analysis time point. Since dietary intake and fecal excretion were measured daily, i.e., up to three measurements may contribute to absorption calculations, the average over all available daily absorption measurements over the 72-hour period were used for analysis. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. The increase from baseline at Week 4 was not statistically significant (p-value= 0.306). The p-value was computed based on the paired t-test.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 4
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: kJ/day
        arithmetic mean (standard deviation)
    494.259 ( 1355.5857 )
    No statistical analyses for this end point

    Secondary: Absolute Change in Absorption of Macronutrients Over MB Periods From Baseline at Weeks 4 and 48

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    End point title
    		 Absolute Change in Absorption of Macronutrients Over MB Periods From Baseline at Weeks 4 and 48
    End point description
    The absorption was defined as dietary intake minus output from fecal excretion over a 72-hour MB period at a given analysis time point. Since dietary intake and fecal excretion were measured daily, i.e., up to three measurements may contribute to absorption calculations, the average over all available daily absorption measurements over the 72-hour period were used for analysis. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. Fat absorption increase at Weeks 4 and 48 was not statistically significant (p-value=0.294 and 0.155, respectively). Carbohydrate absorption at Week 4 was not statistically significant (p-value=0.260); however, the increase at Week 48 was statistically significant (p-value=0.024). Protein aborption increase at Weeks 4 and 48 was not statistically significant (p-value=0.096 and 0.075, respectively). P-values were computed based on paired t-test.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 4 and Week 48
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: kJ/day
    arithmetic mean (standard deviation)
        Fat - Week 4
    247.281 ( 661.1866 )
        Fat - Week 48
    406.741 ( 776.4802 )
        Carbohydrate - Week 4
    272.881 ( 675.1125 )
        Carbohydrate - Week 48
    877.011 ( 945.2279 )
        Protein - Week 4
    176.178 ( 280.0788 )
        Protein - Week 48
    194.400 ( 285.2597 )
    No statistical analyses for this end point

    Secondary: Relative Change in Absorption of Energy Over MB Periods From Baseline at Week 48

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    End point title
    		 Relative Change in Absorption of Energy Over MB Periods From Baseline at Week 48
    End point description
    The absorption was defined as dietary intake minus output from fecal excretion over a 72-hour MB period at a given analysis time point. Since dietary intake and fecal excretion were measured daily, i.e., up to three measurements may contribute to absorption calculations, the average over all available daily absorption measurements over the 72-hour period were used for analysis. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
    End point type
    Secondary
    End point timeframe
    Baseline and Week 48
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: percentage energy absorption
    arithmetic mean (standard deviation)
        Week 48
    29.23 ( 36.898 )
    No statistical analyses for this end point

    Secondary: Absolute Change in Urinary Electrolytes over MB Periods From Baseline at Week 4 and Week 48

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    End point title
    		 Absolute Change in Urinary Electrolytes over MB Periods From Baseline at Week 4 and Week 48
    End point description
    Since urinary electrolytes data were measured over the 72-hour MB period, the average of all available results was used for analyses for each MB parameter at a given analysis time point. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. No significant changes from baseline were reported at Week 4 or Week 48 for calcium, magnesium, potassium, or creatinine. A significant change from baseline was reported for sodium at Week 4 (p-value=0.004) but not Week 48. A significant change from baseline in urea was reported at Week 48 (p-value-0.009) but not Week 4.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 4 and Week 48
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: cmol/L
    arithmetic mean (standard deviation)
        Calcium - Week 4
    0.1444 ( 1.09818 )
        Calcium - Week 48
    -0.5532 ( 2.28264 )
        Magnesium - Week 4
    -0.258 ( 0.8370 )
        Magnesium - Week 48
    -2.306 ( 3.1400 )
        Sodium - Week 4
    31.668 ( 24.1946 )
        Sodium - Week 48
    17.097 ( 50.1676 )
        Potassium - Week 4
    2.047 ( 16.8154 )
        Potassium - Week 48
    -8.012 ( 13.5798 )
        Urea - Week 4
    7.693 ( 59.2590 )
        Urea - Week 48
    -89.649 ( 77.9786 )
        Creatinine - Week 4
    0.198 ( 1.0071 )
        Creatinine - Week 48
    -0.011 ( 1.3283 )
    No statistical analyses for this end point

    Secondary: Change from Baseline in Pittsburgh Sleep Quality Inventory (PSQI) Total Score at Week 24 and Week 52

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    End point title
    		 Change from Baseline in Pittsburgh Sleep Quality Inventory (PSQI) Total Score at Week 24 and Week 52
    End point description
    The PSQI is a patient-reported questionnaire used to measure the quality and patterns of sleep, over the past month. The PSQI has seven components: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleep medication, and daytime dysfunction over the last month. Minimum Score = 0 (better); Maximum Score = 21 (worse). A negative change from baseline represents a reduction in symptoms. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point. The decrease in mean PSQI total score at Week 24 was not statistically significant (p-value=0.066); however, the decrease at Week 52 was statistically significant (p-value=0.015). The p-values were computed based on the paired t-test.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24 and Week 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: score on a scale
    arithmetic mean (standard deviation)
        Week 24
    -1.89 ( 2.667 )
        Week 52
    -1.56 ( 1.509 )
    No statistical analyses for this end point

    Secondary: Patient Global Impression of Treatment Satisfaction (PGI-TS) at Week 24 and Week 52

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    End point title
    		 Patient Global Impression of Treatment Satisfaction (PGI-TS) at Week 24 and Week 52
    End point description
    This form is a single-item questionnaire assessing the participant’s satisfaction with the trial medication over the preceding 7 days. Response options range from -2 to 2, very dissatisfied to very satisfied. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
    End point type
    Secondary
    End point timeframe
    Week 24 and Week 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9 [4]
    Units: participants
        Week 24: 2 Very Satisfied
    5
        Week 24: 1 Satisfied
    3
        Week 52: 2 Very Satisfied
    3
        Week 52: 1 Satisfied
    6
    Notes
    [4] - Week 24 n = 8.
    No statistical analyses for this end point

    Secondary: Change from Baseline in Patient Global Impression of Change (PGIC) at Week 24 and Week 52

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    End point title
    		 Change from Baseline in Patient Global Impression of Change (PGIC) at Week 24 and Week 52
    End point description
    PGIC v2.0 is a single-item questionnaire using a 5-point verbal rating scale, to assess overall change in the participants status after taking the IMP. Response options range from 2= very much better to -2= very much worse. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24 and Week 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9 [5]
    Units: participants
        Week 24: 2 Much Better
    6
        Week 24: 1 A Little Better
    2
        Week 52: 2 Much Better
    8
        Week 52: 1 A Little Better
    1
    Notes
    [5] - Week 24 n = 8.
    No statistical analyses for this end point

    Secondary: Change from Baseline in Patient Global Impression of Satisfaction with Parenteral Support (PGI-SPS) at Week 24 and Week 52

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    End point title
    		 Change from Baseline in Patient Global Impression of Satisfaction with Parenteral Support (PGI-SPS) at Week 24 and Week 52
    End point description
    This is a single-item questionnaire assessing the participant's satisfaction with PS over the preceding 7 days. Response options range from -2 to 2, very dissatisfied to very satisfied. A reduction from baseline represents a decrease in satisfaction. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
    End point type
    Secondary
    End point timeframe
    Week 24 and Week 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    5
    Units: score on a scale
    arithmetic mean (standard deviation)
        Week 24 (n = 5)
    -0.20 ( 0.447 )
        Week 52 (n = 4)
    0.00 ( 0.000 )
    No statistical analyses for this end point

    Secondary: Change from Baseline in Patient Global Impression of Parenteral Support Impact (PGI-PSI) at Week 24 and Week 52

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    End point title
    		 Change from Baseline in Patient Global Impression of Parenteral Support Impact (PGI-PSI) at Week 24 and Week 52
    End point description
    This is a three-item questionnaire assessing the impact of PS on the participant’s sleep, daily activities, and quality of life (QoL) over the past 7 days. All questions have response options ranging from 0 to 4, not at all to extremely. A reduction from baseline represents a decrease in symptoms. The full analysis set included all participants who received at least one dose of trial treatment and contributed at least one valid post-baseline efficacy point.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24 and Week 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    5 [6]
    Units: score on a scale
    arithmetic mean (standard deviation)
        Sleep Impact: Week 24
    -0.40 ( 1.517 )
        Sleep Impact: Week 52
    -0.50 ( 1.291 )
        Daily Activities Impact: Week 24
    -0.80 ( 1.643 )
        Daily Activities Impact: Week 52
    -1.00 ( 1.414 )
        QoL Impact: Week 24
    -1.40 ( 1.140 )
        QoL Impact: Week 52
    -1.25 ( 0.957 )
    Notes
    [6] - Week 52 n = 4.
    No statistical analyses for this end point

    Secondary: Trough Apraglutide Plasma Concentration (Ctrough)

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    End point title
    		 Trough Apraglutide Plasma Concentration (Ctrough)
    End point description
    The safety analysis set included all participants exposed to trial medication.
    End point type
    Secondary
    End point timeframe
    Pre-dose on Weeks 2, 4, 12, 24, 32, 40, 48, and 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9
    Units: ng/mL
    arithmetic mean (standard deviation)
        Week 2 (n = 4)
    1.928 ( 0.7058 )
        Week 4 (n = 3)
    1.217 ( 0.2902 )
        Week 12 (n = 5)
    1.934 ( 0.6665 )
        Week 24 (n = 4)
    2.863 ( 1.0668 )
        Week 32 (n = 7)
    1.739 ( 0.6805 )
        Week 40 (n = 5)
    2.386 ( 0.9587 )
        Week 48 (n = 3)
    3.810 ( 1.1031 )
        Week 52 (n = 9)
    2.678 ( 1.4039 )
    No statistical analyses for this end point

    Secondary: Mean Plasma Citrulline Level

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    End point title
    		 Mean Plasma Citrulline Level
    End point description
    The safety analysis set included all participants exposed to trial medication.
    End point type
    Secondary
    End point timeframe
    Baseline and Weeks 2, 4, 12, 24, 32, 40, 48, and 52
    End point values
    		 Apraglutide
    Number of subjects analysed
    9 [7]
    Units: μmol/L
    arithmetic mean (standard deviation)
        Baseline
    11.571 ( 9.7091 )
        Week 2
    13.516 ( 9.8136 )
        Week 4
    15.531 ( 12.4055 )
        Week 12
    14.860 ( 12.2165 )
        Week 24
    14.810 ( 11.8877 )
        Week 32
    14.994 ( 12.6880 )
        Week 40
    17.860 ( 15.8020 )
        Week 48
    16.638 ( 14.9346 )
        Week 52
    14.714 ( 11.7896 )
    Notes
    [7] - Week 4 n = 8.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 up to approximately 55 weeks
    Adverse event reporting additional description
    The safety analysis set included all participants exposed to trial medication.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Apraglutide
    Reporting group description
    		 All participants received, based on their body weight, either 2.5 mg (<50 kg) or 5 mg (≥50 kg) apraglutide once weekly, for 52 weeks. The IMP was administered SC.

    Serious adverse events
    		 Apraglutide
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 9 (33.33%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pancreatitis acute
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Cholangitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Campylobacter gastroenteritis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Septic shock
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Product issues
    Device dislocation
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Apraglutide
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 9 (100.00%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Complication associated with device
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Catheter site irritation
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Injection site erythema
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    7
    Injection site pruritus
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    6
    Fatigue
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Injection site haematoma
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Injection site pain
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Dysphonia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Investigations
    Weight decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Procedural vomiting
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    3
    Procedural pain
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Bone contusion
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Post procedural complication
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Post procedural haematoma
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Procedural nausea
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Radius fracture
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Vascular access site pain
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Dizziness
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Metabolic encephalopathy
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gastrointestinal disorders
    Stomatitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    5 / 9 (55.56%)
         occurrences all number
    42
    Diarrhoea
         subjects affected / exposed
    3 / 9 (33.33%)
         occurrences all number
    3
    Abdominal pain
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    3
    Anal fissure
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Anorectal discomfort
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Regurgitation
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Hepatobiliary disorders
    Portal vein thrombosis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Nephrolithiasis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Musculoskeletal chest pain
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Musculoskeletal stiffness
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Tenosynovitis stenosans
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Infections and infestations
    Influenza
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Oral herpes
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Sinusitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    COVID-19
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Vascular device infection
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    3
    Nasopharyngitis
         subjects affected / exposed
    5 / 9 (55.56%)
         occurrences all number
    5
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Asymptomatic COVID-19
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Hypokalaemia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Iron deficiency
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Hypocalcaemia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Hypomagnesaemia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Magnesium deficiency
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Feb 2020
    • Corrected inconsistencies and typos • Corrected the wording of the primary endpoint • Updates to inclusion and exclusion criteria • Updated planned trial dates • Added glutamine to the list of prior medications to be specifically recorded • Added fecal sample testing for H. pylori at baseline and added vital signs assessment at Visit 3 (Day 0); reduced the time window for the final visit of the treatment period (Visit 17, Week 52) to ±2 days • Specified that body temperature is to be taken in the axilla for all vital signs assessments • Additional clinical chemistry parameters to be tested • Included microbiota questionnaires and measurement of hip and waist circumference • Updated the number of biopsies required for the experimental trials and clarified the distinction between standard and single-cell ribonucleic acid sequencing • Specified the parameters to be analyzed for microbiota, and the duration of sample storage and results archiving • Added details of the considerations on IMP dose reduction and temporary discontinuation of IMP • Specified that in the case that a vulnerable participant enters the trial, the Principal Investigator is encouraged to consult their local independent ethics committee/institutional review board for guidance
    03 Sep 2021
    • Corrected inconsistencies and typos • Minor wording changes and clarifications • Sponsor representative changed • Abbreviations list updated • Synopsis: clarified definition of short bowel syndrome and deleted PS definition • Secondary endpoints and Section 5.1.9 PROs updated • Updated inclusion and exclusion criteria • Updates to Tables 1 and 2 • Section 2.2: wording added to include definitions of PS, fluids, and other parameters during the trial • Section 2.2.1: inclusion of wording regarding history of vomiting • Section 4.5: Added further details on the PS reduction procedure • Section 4.7 and exclusion criteria: use of somatostatin analogs removed • Section 4.7.1 & 4.7.2: Definition of use of antibiotics updated. Added routine vaccinations allowed • Section 5.1.11: ADA follow up procedures post EOT clarified • Section 5.2.9: Total HCO3- added to blood lab tests at safety evaluation post PS reduction • Section 6.6.2: Updated the stopping rules • Section 8: Added section regarding DMC • Section 10.2.1: Updated that biomarker samples will be stored for 15 years • Section 16.5: Electrolytes all now analyzed by Atomic absorptiometry
    07 Feb 2022
    • General changes to improve clarity and minor formatting issues • International Coordinating Investigator changed • Primary objective changed to evaluate the safety and tolerability of apraglutide • Evaluation of calories added to secondary objectives • Primary endpoints updated to reflect the change in primary objective by including AEs, AESIs, clinical laboratory assessment and ADA • Addition of the Week 4 assessment in relative change from baseline in actual weekly PS volume • Changes to secondary endpoints • Section 1.5.2.4: Definition of enteral autonomy added • Inclusion criterion 2 clarified • Table 1: Addition of definition of MB period • Table 2: Addition of Bristol Stool Form Scale on Days 2 and 3 • Section 2.1: Additional background added • Section 2.2: Confirmation that infusion of a small amount of fluid to maintain catheter patency is not considered PS • Section 2.2.1: Added that investigator can use clinical judgment to demonstrate that the small intestine is <200 cm • Section 2.2.2: Additional instructions relating to drinking menu and enteral nutrition • Section 4.5: Clarifications on PS volume reduction criteria and process and clarification on method of calculation baseline urine average

    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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