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    Clinical Trial Results:
    Open label, group comparison, dose escalation trial to investigate safety, tolerability, pharmacokinetics and pharmacodynamics of macimorelin acetate after single oral dosing of 0.25 mg/kg, 0.5 mg/kg, and 1 mg/kg in pediatric patients with suspected growth hormone deficiency (GHD)

    Summary
    EudraCT number
    		 2018-001988-23
    Trial protocol
    		 HU   PL  
    Global end of trial date
    24 Jan 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    09 Aug 2020
    First version publication date
    09 Aug 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AEZS-130-P01
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 Study P01: Study P01
    Sponsors
    Sponsor organisation name
    Aeterna Zentaris GmbH
    Sponsor organisation address
    Weismuellerstr. 50, Frankfurt am Main, Germany, 60314
    Public contact
    Dr. med. Nicola Ammer, MSc PM, Aeterna Zentaris GmbH, 0049 69426023472, NAmmer@aezsinc.com
    Scientific contact
    Dr. med. Nicola Ammer, MSc PM, Aeterna Zentaris GmbH, 0049 69426023472, NAmmer@aezsinc.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001988-PIP01-16
    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
    17 Jun 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    24 Jan 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    24 Jan 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Primary: • To investigate the safety and tolerability of macimorelin acetate after ascending single oral doses of macimorelin in pediatric patients with suspected GHD. Secondary: • To investigate the PK of macimorelin acetate in pediatric patients with suspected GHD; • To investigate the PD of macimorelin acetate as measured by GH release in pediatric patients with suspected GHD; • To explore the PK/PD relationship following single oral dose administration of macimorelin acetate in pediatric patients with suspected GHD.
    Protection of trial subjects
    All patients were informed that they had the right to withdraw from the trial at any time, for any reason, without prejudice, and without having to justify their reasons or decisions. Additionally, the investigator might have discontinued patient’s participation at any time if he/she would have considered that to be in the patient’s best interest or if the investigator determined that continuing the participation would have resulted in a significant safety risk for that patient. Pediatic patients had to meet specific inclusion criterion to be eligible for admission to the trial, and they were ineligible to participate if meeting specific exclusion criteria. Once all the screening data became available, investigator performed the final evaluation of the patient’s eligibility in the eCRF and forwarded it to the Medical Monitor (MM) for a final review. The MM confirmed eligibility in the eCRF. The DRC comprised a panel of selected experts and a representative of the Sponsor. The dose titration program was reconsidered after completion of each dose step and would proceed only if the previous dose step reviewed and confirmed by the DRC had shown acceptable safety and tolerability. Sequential cohorts of trial participants received macimorelin at ascending single oral doses, The dose titration program was reconsidered after completion of each dose step and would proceed only if the previous dose step reviewed and confirmed by Data Review Committee (DRC) had shown acceptable safety and tolerability. This DRC comprised a panel of selected experts and a representative of the Sponsor. The investigational medicinal product (IMP) macimorelin was administered by trial personnel experience in pediatrics. A growth hormone stimulation test (GHST) Tolerability Questionniare was completed by patients or parents/legal guardians. It included specific questions regarding the acceptability of taste, any signs for an impact on sleep, appetite and gastrointestinal Symptoms.
    Background therapy
    		 The selection of the two different sGHSTs followed the respective trial site’s clinical standard practice and, therefore, the test agents used were considered as ‘background’ and not as ‘investigational’ medicinal products. The following pharmacological agents were accepted for sGHSTs performed as part of the standard procedures for diagnosing GHD in pediatric patients: Insulin tolerance test (ITT), Arginine, Arginine/Growth hormone releasing hormone (GHRH), Clonidine, Glucagon, L-dopa.
    Evidence for comparator
    		 This was a dose-escalation, PK/PD study. No comparators were used.
    Actual start date of recruitment
    01 Nov 2018
    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
    Poland: 2
    Country: Number of subjects enrolled
    Hungary: 8
    Country: Number of subjects enrolled
    Ukraine: 10
    Country: Number of subjects enrolled
    Russian Federation: 1
    Country: Number of subjects enrolled
    Belarus: 1
    Country: Number of subjects enrolled
    Serbia: 2
    Worldwide total number of subjects
    24
    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)
    13
    Adolescents (12-17 years)
    11
    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 trial started on 07-Feb-2019 with the first informed consent signed. The first sGHST was administered in Cohort 1 (C1) on 13-Feb-2019. In C1, the first macimorelin test was conducted on 28-Feb-2019, in Cohort 2 (C2) on 08-Jul-2019, and in Cohort 3 (C3) on 05-Dec-2019. The trial was completed with Last-Patient-Last-Visit on 24-Jan-2020.

    Pre-assignment
    Screening details
    		 Altogether 27 patients consented to trial participation in 11 trial centers in 6 countries. 24 patients had an evaluable first standard Growth Hormone Stimulation test and, thus, were considered as enrolled in the Trial.

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    The trial was conducted open-label as trial medication and procedures cannot be masked. Macimorelin was administered as oral solution. The sGHSTs were considered as ‘background’ (i.e., standard medication) and not as IMPs. Depending on the sGHSTs selected by the trial site, the test agents were administered i.v., i.m., s.c., or peroral. The Investigators were blinded towards GH values (test results) until both sGHSTs were performed and adjudicated evaluable by the DRC.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Macimorelin GHST: C1
    Arm description
    		 This is the first of three cohorts assessing ascending doses of macimorelin (one dose per pediatric patient). C1: macimorelin 0.25 mg/kg. Each cohort is comprising 8 pediatric patients.
    Arm type
    		 Experimental

    Investigational medicinal product name
    C1: macimorelin 0.25 mg/kg
    Investigational medicinal product code
    AEZS-130
    Other name
    macimorelin Growth Hormone Stimulation Test (GHST); macimorelin GHST
    Pharmaceutical forms
    Granules in sachet
    Routes of administration
    Oral use
    Dosage and administration details
    Macimorelin was supplied in single-use aluminum pouches (synonymous: sachets) each containing 63.6 mg macimorelin acetate, which provide 0.5 mg/mL of macimorelin when dissolved in 120 mL of water. Macimorelin had to be prepared and used according to specific directions for the three dosing groups. Applicable for C1: based on the macimorelin dose of 0.25 mg/kg, the required volume of the suspension was to be determined which corresponded to the patient body weight, i.e. required volume of suspension was 0.5 mL/kg (for example, a 30 kg patient requiring macimorelin dose of 0.25 mg/kg required 15 mL of the prepared suspension).

    Arm title
    		 Macimorelin GHST: C2
    Arm description
    		 This is the second of three cohorts assessing ascending doses of macimorelin (one dose per pediatric patient). C2: macimorelin 0.50 mg/kg. Each cohort is comprising 8 pediatric patients.
    Arm type
    		 Experimental

    Investigational medicinal product name
    C1: macimorelin 0.5 mg/kg
    Investigational medicinal product code
    AEZS-130
    Other name
    macimorelin Growth Hormone Stimulation Test (GHST); macimorelin GHST
    Pharmaceutical forms
    Granules in sachet
    Routes of administration
    Oral use
    Dosage and administration details
    Macimorelin was supplied in single-use aluminum pouches (synonymous: sachets) each containing 63.6 mg macimorelin acetate, which provide 0.5 mg/mL of macimorelin when dissolved in 120 mL of water. Macimorelin had to be prepared and used according to specific directions for the three dosing groups. Applicable for C2: based on the macimorelin dose of 0.5 mg/kg, the required volume of the suspension was to be determined which corresponded to the patient body weight, i.e. required volume of suspension was 1.0 mL/kg (for example, a 30 kg patient requiring macimorelin dose of 0.5 mg/kg required 30 mL of the prepared suspension).

    Arm title
    		 Macimorelin GHST: C3
    Arm description
    		 This is the third of three cohorts assessing ascending doses of macimorelin (one dose per pediatric patient). C3: macimorelin 1.0 mg/kg. Each cohort is comprising 8 pediatric patients.
    Arm type
    		 Experimental

    Investigational medicinal product name
    C1: macimorelin 1.0 mg/kg
    Investigational medicinal product code
    AEZS-130
    Other name
    macimorelin Growth Hormone Stimulation Test (GHST); macimorelin GHST
    Pharmaceutical forms
    Granules in sachet
    Routes of administration
    Oral use
    Dosage and administration details
    Macimorelin was supplied in single-use aluminum pouches (synonymous: sachets) each containing 63.6 mg macimorelin acetate, which provide 0.5 mg/mL of macimorelin when dissolved in 120 mL of water. Macimorelin had to be prepared and used according to specific directions for the three dosing groups. Applicable for C3: based on the macimorelin dose of 1.0 mg/kg, the required volume of the suspension was to be determined which corresponded to the patient body weight, i.e. required volume of suspension was 2.0 mL/kg (for example, a 30 kg patient requiring macimorelin dose of 1.0 mg/kg required 60 mL of the prepared suspension).

    Number of subjects in period 1
    		Macimorelin GHST: C1 Macimorelin GHST: C2 Macimorelin GHST: C3
    Started
    8
    8
    8
    Completed
    8
    8
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Macimorelin GHST: C1
    Reporting group description
    		 This is the first of three cohorts assessing ascending doses of macimorelin (one dose per pediatric patient). C1: macimorelin 0.25 mg/kg. Each cohort is comprising 8 pediatric patients.

    Reporting group title
    Macimorelin GHST: C2
    Reporting group description
    		 This is the second of three cohorts assessing ascending doses of macimorelin (one dose per pediatric patient). C2: macimorelin 0.50 mg/kg. Each cohort is comprising 8 pediatric patients.

    Reporting group title
    Macimorelin GHST: C3
    Reporting group description
    		 This is the third of three cohorts assessing ascending doses of macimorelin (one dose per pediatric patient). C3: macimorelin 1.0 mg/kg. Each cohort is comprising 8 pediatric patients.

    Reporting group values
    		 Macimorelin GHST: C1 Macimorelin GHST: C2 Macimorelin GHST: C3 Total
    Number of subjects
    		 8 8 8 24
    Age categorical
    Information on patient's current age was collected at screening.
    Units: Subjects
        Children (2-11 years)
    		 5 5 3 13
        Adolescents (12-17 years)
    		 3 3 5 11
    Age continuous
    Information on patient's current age was collected at screening.
    Units: years
        median (standard deviation)
    		 10.5 ± 3.5 8 ± 4.2 12.5 ± 3.9 -
    Gender categorical
    Information on gender was collected at screening.
    Units: Subjects
        Female
    		 3 3 1 7
        Male
    		 5 5 7 17
    Tanner Status
    Information was collected at screening. At least 3 patients per dose group were required to be pre-pubertal (Tanner Stage I) and pubertal (Tanner Stage II-IV), respectively. In this study, Tanner stage of the patients was either Tanner Stage I or Tanner Stage II. No Tanner Stage III or IV were observed.
    Units: Subjects
        Tanner Stage I
    		 4 5 4 13
        Tanner Stage II
    		 4 3 4 11
    Race
    Information was collected at Screening.
    Units: Subjects
        Asian
    		 0 0 0 0
        Black of African-American
    		 0 0 0 0
        White
    		 8 8 8 24
    Specification of the two sGHSTs
    The selection of the two different sGHSTs at V1 and V3 followed the trial site’s clinical standard practice and, therefore, the test agents used were considered as ‘background’ and not as ‘investigational’ medicinal products. The figures bleow present a summary of the two sGHST agents administered to a pediatric patient.
    Units: Subjects
        Arginine Test and Insulin Tolerance Test
    		 6 0 0 6
        Clonidine Test and Insulin Tolerance Test
    		 2 7 7 16
        Glucagon Test and Arginine Test
    		 0 1 1 2
    Height
    Actual height was measured at Screening.
    Units: cm
        median (standard deviation)
    		 114.8 ± 32.79 117.65 ± 20.95 137.60 ± 19.67 -
    Weight
    Body weight was recorded at Screening, at Visit 1 and Visit 3 (before the sGHST), and at Visit 2(before the macimorelin test). It was to be recorded in kg (rounded to closest integer). The values presented here were collected at Screening.
    Units: kg
        median (standard deviation)
    		 19.5 ± 10.1 27.5 ± 10.9 30.5 ± 10.0 -
    Height Standard Deviation Score (SDS)
    Auxology Parameters were collected at Screening.
    Units: SDS
        median (standard deviation)
    		 -2.50 ± 0.50 -2.20 ± 1.47 -2.45 ± 0.58 -
    Annualized Height Velocity SDS
    Auxology Parameters were collected at Screening.
    Units: SDS
        median (standard deviation)
    		 -1.90 ± 1.09 -1.50 ± 1.10 -0.65 ± 1.12 -

    End points

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    End points reporting groups
    Reporting group title
    Macimorelin GHST: C1
    Reporting group description
    		 This is the first of three cohorts assessing ascending doses of macimorelin (one dose per pediatric patient). C1: macimorelin 0.25 mg/kg. Each cohort is comprising 8 pediatric patients.

    Reporting group title
    Macimorelin GHST: C2
    Reporting group description
    		 This is the second of three cohorts assessing ascending doses of macimorelin (one dose per pediatric patient). C2: macimorelin 0.50 mg/kg. Each cohort is comprising 8 pediatric patients.

    Reporting group title
    Macimorelin GHST: C3
    Reporting group description
    		 This is the third of three cohorts assessing ascending doses of macimorelin (one dose per pediatric patient). C3: macimorelin 1.0 mg/kg. Each cohort is comprising 8 pediatric patients.

    Primary: Macimorelin PK

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    End point title
    		 Macimorelin PK [1]
    End point description
    Plasma concentrations of macimorelin were analyzed in a central laboratory.
    End point type
    Primary
    End point timeframe
    Blood samples were collected on V2 at the following time-points: pre-dose (sampling time window: +/- 15 minutes), then 15, 30, 45, 60, 90, 120 minutes (+/- 5 min window) and 360 minutes (+/- 10 minutes window) after administration of macimorelin.
    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: The trial had an explorative, descriptive aim and did not intend to prove or disprove any statistical hypothesis. Furthermore, the statistical analyses sections in this electronic reporting template do not fit with the way the explorative data analyses were conducted in this Trial (e.g., ROC, sensitivity, specificity analyses). Related data are presented in the 'Endpoints'. Therefore, the statistical analyses sections are empty.
    End point values
    		 Macimorelin GHST: C1 Macimorelin GHST: C2 Macimorelin GHST: C3
    Number of subjects analysed
    8
    8
    8
    Units: Macimorelin concentration
    arithmetic mean (standard deviation)
        AUC 0-6 (h*ng/mL)
    6.685 ± 3.093
    18.015 ± 9.800
    30.920 ± 11.510
        Cmax (ng/mL)
    3.460 ± 1.783
    8.126 ± 4.176
    12.868 ± 3.011
        Tmax (min)
    45.5 ± 32.8
    40.6 ± 22.3
    31.9 ± 5.3
        T1/2 (min)
    73.183 ± 29.437
    96.307 ± 41.031
    102.851 ± 19.938
    No statistical analyses for this end point

    Primary: Macimorelin PD

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    End point title
    		 Macimorelin PD [2]
    End point description
    Serum concentrations of growth hormone (GH) were analyzed in a central laboratory.
    End point type
    Primary
    End point timeframe
    Blood samples were collected on V2 at the following time-points: pre-dose (sampling time window: +/- 15 minutes), then 15, 30, 45, 60, 90, 120 minutes (+/- 5 min window) and 360 minutes (+/- 10 minutes window) after administration of macimorelin.
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The trial had an explorative, descriptive aim and did not intend to prove or disprove any statistical hypothesis. Furthermore, the statistical analyses sections in this electronic reporting template do not fit with the way the explorative data analyses were conducted in this Trial (e.g., ROC, sensitivity, specificity analyses). Related data are presented in the 'Endpoints'. Therefore, the statistical analyses sections are empty.
    End point values
    		 Macimorelin GHST: C1 Macimorelin GHST: C2 Macimorelin GHST: C3
    Number of subjects analysed
    8
    8
    8
    Units: growth hormone concentration
    arithmetic mean (standard deviation)
        Cmax (ng/mL)
    9.791 ± 6.226
    14.590 ± 8.046
    29.533 ± 18.829
        Tmax (min)
    52.5 ± 11.3
    37.5 ± 13.9
    37.5 ± 8.0
    No statistical analyses for this end point

    Other pre-specified: Macimorelin GHST versus PI Assessment and versus sGHST

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    End point title
    		 Macimorelin GHST versus PI Assessment and versus sGHST
    End point description
    The diagnostic outcome of the sGHST was considered as ‘confirmed’ if both sGHSTs were available and both resulted in a peak GH ≤ 7 ng/mL or ‘not confirmed’ of at least one of the peaks was above 7 ng/mL. The investigator’s assessment was based on local diagnostic standard practice. The macimorelin GHST was tested against a cut-off point calculated from the individual peak GH values.
    End point type
    Other pre-specified
    End point timeframe
    Macimorelin GHST (MAC) was performed on V2, and sGHSTs on V1 and V3. Blood sampling for MAC at pre-dose, 15, 30, 45, 60, 90, 120 min. (+/- 5 min) , 360 min. (+/- 10 min.), for sGHSTs at time points according to local Standards.
    End point values
    		 Macimorelin GHST: C1 Macimorelin GHST: C2 Macimorelin GHST: C3
    Number of subjects analysed
    8 [3]
    8 [4]
    8 [5]
    Units: pediatric patients
        PI's Assessment GHD - MAC confirmed
    3
    4
    3
        PI's Assessment GHD - MAC not confirmed
    0
    1
    0
        PI's Assessment Non GHD - MAC confirmed
    3
    0
    1
        PI's Assessment Non GHD - MAC not confirmed
    2
    3
    4
        sGHST confirmed - MAC confirmed
    1
    3
    3
        sGHST confirmed - MAC not confirmed
    0
    1
    0
        sGHST not confirmed - MAC confirmed
    5
    1
    1
        sGHST not confirmed - MAC not confirmed
    2
    3
    4
    Notes
    [3] - Exploratory GH cut-off point for the macimorelin GHST in C1: 10.030 ng/mL
    [4] - Exploratory GH cut-off point for the macimorelin GHST in C2: 10.430 ng/mL
    [5] - Exploratory GH cut-off point for the macimorelin GHST in C3: 17.130 ng/mL
    No statistical analyses for this end point

    Other pre-specified: Exploration of a suitable GH cut-off point for the macimorelin GHST

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    End point title
    		 Exploration of a suitable GH cut-off point for the macimorelin GHST
    End point description
    ROC curves were created to plot the sensitivity and specificity of all possible peak GH values after macimorelin GHST at the categorization of subjects with GHD vs. non GHD based on investigator’s assessment. Cut-off point with the maximum Youden index value was selected by cohort. In case of equivalence, cut-off point with higher sensitivity was chosen. Weighted Youden index was calculated with having more weight on sensitivity. The weight for the Youden index was 0.6 and 0.7. The following formula was used for the calculation: Weighted Youden index= 2w SEN + 2 (1-w) SPEC - 1. Specificity, sensitivity, negative predictive value (NPV), positive predictive value (PPV) and weighted Youden index are presented by cohort. As a sensitivity analysis, three different GH cut-off points were selected based on the categorization of subjects with standard GHST considering ‘confirmed’ vs. ‘not confirmed’ cases.
    End point type
    Other pre-specified
    End point timeframe
    Macimorelin GHST (MAC) was performed on V2, and sGHSTs on V1 and V3. Blood sampling for MAC at pre-dose, 15, 30, 45, 60, 90, 120 min. (+/- 5 min) , 360 min. (+/- 10 min.), for sGHSTs at time points according to local Standards.
    End point values
    		 Macimorelin GHST: C1 Macimorelin GHST: C2 Macimorelin GHST: C3
    Number of subjects analysed
    8
    8
    8
    Units: GH peak values
    number (not applicable)
        peak GH cut-off point (ng/mL)
    10.030
    10.430
    17.130
        Specificity
    0.40
    1.00
    0.80
        Sensitivity
    1.00
    0.80
    1.00
        Youden-Index
    0.40
    0.80
    0.80
        Weighted Youden-Index (w=0.6)
    0.52
    0.76
    0.84
        Weighted Youden-Index (w=0.7)
    0.64
    0.72
    0.88
        Negative Predictive Value
    1.00
    0.75
    1.00
        Positive Predictive Value
    0.50
    1.00
    0.75
        ROC AUC
    0.60
    0.80
    0.93
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events (AEs) were recorded from after the moment of signing ICF and up to the end of the trial (V4), i.e. from Day -28 to D-1 (Screening) up to D19+ (V4).
    Adverse event reporting additional description
    No AE was reported in relationship to macimorelin. No serious AE (SAE) was reported in this study. Majority of AEs was related to the Insulin tolerance test (ITT). AEs were mostly of mild to moderate intensity. Known side effects were reported for sGHST. ITT related AES comprised symptoms of hypoglycemia, which is a clinical endpoint of this sGHST.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    21.0
    Reporting groups
    Reporting group title
    C1 macimorelin GHST
    Reporting group description
    		 This group includes all pediatric patients observed in C1.

    Reporting group title
    C2 macimorelin GHST
    Reporting group description
    		 This group includes all pediatric patients observed in C2.

    Reporting group title
    C3 macimorelin GHST
    Reporting group description
    		 This group includes all pediatric patients observed in C3.

    Serious adverse events
    		 C1 macimorelin GHST C2 macimorelin GHST C3 macimorelin GHST
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 C1 macimorelin GHST C2 macimorelin GHST C3 macimorelin GHST
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 8 (100.00%)
    8 / 8 (100.00%)
    7 / 8 (87.50%)
    Investigations
    Blood pressure diastolic decreased
    Additional description: Blood pressure diastolic decreased was reported in relationship to the clonidine GHST.
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 8 (25.00%)
         occurrences all number
    0
    0
    2
    Blood pressure systolic decreased
    Additional description: Blood pressure systolic decreased was reported in relationship to the clonidine GHST.
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    3 / 8 (37.50%)
         occurrences all number
    0
    0
    3
    Vascular disorders
    Pallor
    Additional description: Pallor was reported in relationship to the ITT.
         subjects affected / exposed
    0 / 8 (0.00%)
    2 / 8 (25.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    2
    1
    Cardiac disorders
    Palpitations
    Additional description: Palpitations were reported in relationship to the hypoglycemia, which is an expected endpoint of the ITT.
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    1
    1
    0
    Tachycardia
    Additional description: Tachycardia was reported in relationship to the hypoglycemia, which is an expected endpoint of the ITT.
         subjects affected / exposed
    1 / 8 (12.50%)
    4 / 8 (50.00%)
    1 / 8 (12.50%)
         occurrences all number
    1
    4
    1
    Nervous system disorders
    Somnolence
    Additional description: Somnolence was reported in relationship to the ITT as well as the clonidine GHST, and it is a known side effect of these sGHSTs.
         subjects affected / exposed
    3 / 8 (37.50%)
    2 / 8 (25.00%)
    3 / 8 (37.50%)
         occurrences all number
    4
    2
    3
    Tremor
    Additional description: Tremor was reported in relationship to the hypoglycemia, which is an expected endpoint of the ITT.
         subjects affected / exposed
    2 / 8 (25.00%)
    1 / 8 (12.50%)
    3 / 8 (37.50%)
         occurrences all number
    2
    1
    3
    Dizziness
    Additional description: Dizziness was reported in relationship to the hypoglycemia, which is an expected endpoint of the ITT.
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    4 / 8 (50.00%)
         occurrences all number
    0
    0
    4
    Gastrointestinal disorders
    Hunger
    Additional description: Hunger was reported in relationship to the ITT.
         subjects affected / exposed
    6 / 8 (75.00%)
    3 / 8 (37.50%)
    1 / 8 (12.50%)
         occurrences all number
    6
    3
    2
    Skin and subcutaneous tissue disorders
    Hyperhidrosis
    Additional description: Hyperhidrosis was reported in relationship to the hypoglycemia, which is an expected endpoint of the ITT.
         subjects affected / exposed
    4 / 8 (50.00%)
    5 / 8 (62.50%)
    3 / 8 (37.50%)
         occurrences all number
    4
    5
    3
    Infections and infestations
    Bronchitis
    Additional description: AE reported as not being related to an sGHST and/or the macimorelin GHST, respectively.
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    Oral herpes
    Additional description: AE reported as not being related to an sGHST and/or the macimorelin GHST, respectively.
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory tract infection
    Additional description: AE reported as not being related to an sGHST and/or the macimorelin GHST, respectively.
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    Viral upper respiratory tract infection
    Additional description: AE reported as not being related to an sGHST and/or the macimorelin GHST, respectively.
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    2 / 8 (25.00%)
         occurrences all number
    0
    0
    2

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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