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    Clinical Trial Results:
    Multicenter, open label trial to investigate the efficacy and safety of a single oral dose of 1.0 mg/kg macimorelin acetate as growth hormone stimulation test (GHST) in pediatric patients with suspected growth hormone deficiency (GHD)

    Summary
    EudraCT number
    		 2018-001989-42
    Trial protocol
    		 SI   DE   PL   IT   SK  
    Global end of trial date
    13 Jun 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    01 Jan 2025
    First version publication date
    01 Jan 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AEZS-130-P02
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04786873
    WHO universal trial number (UTN)
    		 U1111-1248-5075
    Other trial identifiers
    		 IND Number: 154015
    Sponsors
    Sponsor organisation name
    Aeterna Zentaris GmbH
    Sponsor organisation address
    Weismuellerstraße 50, Frankfurt am Main, Germany, D-60314
    Public contact
    Clinical trial information desk, Aeterna Zentaris GmbH, 0049 69426023472, clinical.trials@aezsinc.com
    Scientific contact
    Clinical trial information desk, Aeterna Zentaris GmbH, 0049 69426023472, clinical.trials@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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    09 Aug 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    13 Jun 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Jun 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To determine the diagnostic efficacy of macimorelin in diagnosing suspected growth hormone deficiency (GHD) in pediatric subjects assuming the outcome of GHD status adjudication by the external adjudication committee as the "true" GHD status.
    Protection of trial subjects
    Participants were closely monitored for adverse events, with safety assessments integrated into the protocol to promptly identify any issues requiring investigator intervention. At the same time, the frequency of assessments and visits was limited to essential procedures, balancing thorough safety oversight with minimising the burden on participants. Enrollment criteria and concomitant medications were defined to exclude participants with a high risk of events during the trial. To ensure adequate treatment also in emergency situations (e.g., allergic reaction), the investigator ensured test administration and participant observation in an adequate facility, with the required standards of treatment being available if required. Additionally, several efforts were made to minimise the inconvenience to the participants during the trial. The fasting period was reduced to the extent possible; the investigators were encouraged to use numbing cream according to local practice to reduce the pain during peripheral venous access.
    Background therapy
    		 Prepubertal boys >11 years and prepubertal girls >10 years underwent sex steroid priming before each GHST12. Both boys and girls should have received 2 mg (1 mg for body weight <20 kg) of β-estradiol orally on each of the two evenings preceding the test.
    Evidence for comparator
    		 Besides the macimorelin GHST, arginine and clonidine standard GHSTs (sGHSTs) were performed. R-Gene 10 for the arginine GHST, and CATAPRESAN 75 for the clonidine GHST were administered as labelled IMP. No direct comparison was performed between macimorelin GHST and the sGHSTs, but the results of the sGHSTs were considered to determine the diagnosis of growth hormone deficiency used to determine the diagnostic efficacy of the macimorelin GHST.
    Actual start date of recruitment
    22 Mar 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
    Poland: 9
    Country: Number of subjects enrolled
    Romania: 10
    Country: Number of subjects enrolled
    Slovakia: 13
    Country: Number of subjects enrolled
    Germany: 1
    Country: Number of subjects enrolled
    Italy: 3
    Country: Number of subjects enrolled
    Armenia: 9
    Country: Number of subjects enrolled
    Georgia: 52
    Country: Number of subjects enrolled
    Serbia: 4
    Country: Number of subjects enrolled
    Türkiye: 4
    Country: Number of subjects enrolled
    United States: 20
    Worldwide total number of subjects
    125
    EEA total number of subjects
    36
    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)
    73
    Adolescents (12-17 years)
    52
    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 16-Nov-2021 with the first informed consent signed. The first IMP (macimorelin GHST, V2) was administered on 14-Dec-2024. The trial was completed with Last-Subject-Last-Visit on 13-Jun-2024. Participants from Europe, the US, and the Caucasus and Anatolia region were enrolled.

    Pre-assignment
    Screening details
    		 Subjects were eligible if they met these criteria: informed consent from the subject, parent(s), or legally authorised representative (and assent if applicable); aged 2 to <18 years; required growth hormone stimulation testing; and had a height measurement 6–18 months before screening.

    Pre-assignment period milestones
    Number of subjects started
    		 125
    Number of subjects completed
    		 102

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 Inclusion/Exclusion Criteria not met: 15
    Reason: Number of subjects
    		 Subject withdrawal by parent or guardian: 3
    Reason: Number of subjects
    		 Withdrawal by subject: 5
    Period 1
    Period 1 title
    Period of GHSTs (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 GHST sequence 1
    Arm description
    		 Besides two macimorelin GHSTs, participants underwent sGHSTs in the randomized order of arginine and clonidine.
    Arm type
    		 GHST order

    Investigational medicinal product name
    Arginine
    Investigational medicinal product code
    Other name
    R-Gene® 10
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Infusion , Intravenous use
    Dosage and administration details
    After an overnight fast soluble arginine hydrochloride (0.5 g/kg) was given i.v. as an infusion with an infusion duration of 30 min. Blood was collected for GH measurement, at altogether 4 sampling time points (pre-dose, and 30, 60, and 90 minutes after the end of the infusion).

    Investigational medicinal product name
    Clonidine
    Investigational medicinal product code
    Other name
    CATAPRESAN® 75
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    After an overnight fast clonidine (0.15 mg/m2 body surface) was given orally. Blood was collected for GH measurement, at altogether 4 sampling time points (pre-dose, and 30, 60, and 90 minutes post-dose).

    Investigational medicinal product name
    Macimorelin
    Investigational medicinal product code
    Other name
    AEZS-130
    Pharmaceutical forms
    Oral powder in sachet
    Routes of administration
    Oral use
    Dosage and administration details
    Macimorelin was supplied in single-use aluminum pouches, each containing 63.6 mg macimorelin as acetate, which provided 0.5 mg/mL of macimorelin when dissolved in 120 mL of water. Macimorelin oral solution/suspension was prepared by trial personnel by dissolving the entire contents of each pouch in 120 mL of water. The dose of macimorelin was 1.0 mg/kg body weight. Macimorelin GHST was performed twice, before and after the sGHSTs, on separated visit days. Subjects were fasting for 8 hours prior to the start and throughout the sampling period of the macimorelin GHST. Blood was collected for GH measurement, at altogether 5 sampling time points (pre-dose, and 30, 45, 60, and 90 minutes post-dose).

    Arm title
    		 GHST sequence 2
    Arm description
    		 Besides two macimorelin GHSTs, participants underwent sGHSTs in the randomized order of clonidine and arginine.
    Arm type
    		 GHST sequence 2

    Investigational medicinal product name
    Clonidine
    Investigational medicinal product code
    Other name
    CATAPRESAN® 75
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    After an overnight fast clonidine (0.15 mg/m2 body surface) was given orally. Blood was collected for GH measurement, at altogether 4 sampling time points (pre-dose, and 30, 60, and 90 minutes post-dose).

    Investigational medicinal product name
    Arginine
    Investigational medicinal product code
    Other name
    R-Gene® 10
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Infusion , Intravenous use
    Dosage and administration details
    After an overnight fast soluble arginine hydrochloride (0.5 g/kg) was given i.v. as an infusion with an infusion duration of 30 min. Blood was collected for GH measurement, at altogether 4 sampling time points (pre-dose, and 30, 60, and 90 minutes after the end of the infusion).

    Investigational medicinal product name
    Macimorelin
    Investigational medicinal product code
    Other name
    AEZS-130
    Pharmaceutical forms
    Oral powder in sachet
    Routes of administration
    Oral use
    Dosage and administration details
    Macimorelin was supplied in single-use aluminum pouches, each containing 63.6 mg macimorelin as acetate, which provided 0.5 mg/mL of macimorelin when dissolved in 120 mL of water. Macimorelin oral solution/suspension was prepared by trial personnel by dissolving the entire contents of each pouch in 120 mL of water. The dose of macimorelin was 1.0 mg/kg body weight. Macimorelin GHST was performed twice, before and after the sGHSTs, on separated visit days. Subjects were fasting for 8 hours prior to the start and throughout the sampling period of the macimorelin GHST. Blood was collected for GH measurement, at altogether 5 sampling time points (pre-dose, and 30, 45, 60, and 90 minutes post-dose).

    Number of subjects in period 1 [1]
    		GHST sequence 1 GHST sequence 2
    Started
    51
    51
    Completed
    47
    44
    Not completed
    4
    7
         Adverse event, non-fatal
    1
    2
         Study Subject Withdrawal by Parent or Guardian
    1
    2
         Non-Compliance With Study Drug
    -
    2
         Not defined
    2
    1
    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: 23 Subjects were not randomized, only screened.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    GHST sequence 1
    Reporting group description
    		 Besides two macimorelin GHSTs, participants underwent sGHSTs in the randomized order of arginine and clonidine.

    Reporting group title
    GHST sequence 2
    Reporting group description
    		 Besides two macimorelin GHSTs, participants underwent sGHSTs in the randomized order of clonidine and arginine.

    Reporting group values
    		 GHST sequence 1 GHST sequence 2 Total
    Number of subjects
    		 51 51 102
    Age categorical
    Units: Subjects
        Children (2-11 years)
    		 31 30 61
        Adolescents (12-17 years)
    		 20 21 41
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 10.1 ( 3.32 ) 10.3 ( 3.49 ) -
    Gender categorical
    Units: Subjects
        Female
    		 12 12 24
        Male
    		 39 39 78
    Pubertal status
    Units: Subjects
        Tanner I
    		 33 33 66
        Tanner II
    		 14 11 25
        Tanner III
    		 3 5 8
        Tanner IV
    		 1 2 3
    X-ray bone assessment age
    Units: year
        arithmetic mean (standard deviation)
    		 8.57 ( 3.963 ) 8.92 ( 3.592 ) -
    Subject analysis sets

    Subject analysis set title
    FAS
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All subjects randomized.

    Subject analysis sets values
    		 FAS
    Number of subjects
    102
    Age categorical
    Units: Subjects
        Children (2-11 years)
    61
        Adolescents (12-17 years)
    41
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    10.2 ( 3.39 )
    Gender categorical
    Units: Subjects
        Female
    24
        Male
    78
    Pubertal status
    Units: Subjects
        Tanner I
    66
        Tanner II
    25
        Tanner III
    8
        Tanner IV
    3
    X-ray bone assessment age
    Units: year
        arithmetic mean (standard deviation)
    8.74 ( 3.767 )

    End points

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    End points reporting groups
    Reporting group title
    GHST sequence 1
    Reporting group description
    		 Besides two macimorelin GHSTs, participants underwent sGHSTs in the randomized order of arginine and clonidine.

    Reporting group title
    GHST sequence 2
    Reporting group description
    		 Besides two macimorelin GHSTs, participants underwent sGHSTs in the randomized order of clonidine and arginine.

    Subject analysis set title
    FAS
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    All subjects randomized.

    Primary: Macimorelin ROC

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    End point title
    		 Macimorelin ROC [1]
    End point description
    Assuming the outcome of GHD status adjudication final clinical diagnosis as the “true” GHD status, the diagnostic efficacy (estimated sensitivity, specificity, misclassification) of the macimorelin GHST was based on the area under the receiver operating characteristic curve (ROC AUC). ROC AUC based on peak GH levels (Cmax GH) after stimulation with macimorelin was estimated non-parametrically using the trapezoidal area under the empirical ROC plot. Based on the observed PK and PD data for macimorelin, which are in-line with previous data in children (trial AEZS-130-P01) and adults, the outcome of the efficacy analysis is surprising for the sponsor. Considering that macimorelin has been validated successfully as diagnostic test in the adult population, post-hoc analyses activities have been started to understand the reasons for the efficacy outcome. The final post-hoc analyses are not available yet at the time of finalization of this CTR.
    End point type
    Primary
    End point timeframe
    Macimorelin GHST on visit 2.
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: This is a single-arm study with no statistical comparison of treatment arms.
    End point values
    		 FAS
    Number of subjects analysed
    95
    Units: area
        number (confidence interval 95%)
    0.762 (0.663 to 0.861)
    Attachments
    		 Untitled (Filename: Figure 14.2.1.1.pdf)
    Untitled (Filename: Figure 14.2.1.2.pdf)
    Untitled (Filename: Figure 14.2.5.1.pdf)
    Untitled (Filename: Figure 14.2.6.1.pdf)
    No statistical analyses for this end point

    Secondary: Sensitivity for the macimorelin GHST

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    End point title
    		 Sensitivity for the macimorelin GHST
    End point description
    End point type
    Secondary
    End point timeframe
    Macimorelin GHST on visit 2.
    End point values
    		 FAS
    Number of subjects analysed
    95
    Units: %
        number (confidence interval 95%)
    78.6 (63.2 to 89.7)
    No statistical analyses for this end point

    Secondary: Specificity of macimorelin GHST

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    End point title
    		 Specificity of macimorelin GHST
    End point description
    End point type
    Secondary
    End point timeframe
    Macimorelin GHST on visit 2.
    End point values
    		 FAS
    Number of subjects analysed
    95
    Units: %
        number (confidence interval 95%)
    67.9 (53.7 to 80.1)
    No statistical analyses for this end point

    Secondary: Overall agreement between the outcome of the macimorelin GHST and the combined outcome from the 2 sGHSTs

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    End point title
    		 Overall agreement between the outcome of the macimorelin GHST and the combined outcome from the 2 sGHSTs
    End point description
    An overall agreement between the outcome of the macimorelin GHST and the combined outcome from the two sGHST was calculated.
    End point type
    Secondary
    End point timeframe
    Macimorelin GHST on visit 2.
    End point values
    		 FAS
    Number of subjects analysed
    95
    Units: %
        number (confidence interval 95%)
    65.3 (54.8 to 74.7)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the signature of ICF up to the end of the study (visit 6).
    Adverse event reporting additional description
    Macimorelin, Arginine, and Clonidine reporting groups include AEs starting after the IMP drug administration up to the next GHST, last trial contact, or 7 days after the administration (whichever comes first). In reporting group Total, all AEs reported during the study are reported.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27
    Reporting groups
    Reporting group title
    Macimorelin
    Reporting group description
    		 -

    Reporting group title
    Arginine
    Reporting group description
    		 -

    Reporting group title
    Clonidine
    Reporting group description
    		 -

    Reporting group title
    Total
    Reporting group description
    		 -

    Serious adverse events
    		 Macimorelin Arginine Clonidine Total
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Infections and infestations
    Pharyngitis streptococcal
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Macimorelin Arginine Clonidine Total
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 102 (11.76%)
    6 / 97 (6.19%)
    18 / 96 (18.75%)
    40 / 102 (39.22%)
    Investigations
    Electrocardiogram QT prolonged
         subjects affected / exposed
    3 / 102 (2.94%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    5 / 102 (4.90%)
         occurrences all number
    3
    0
    0
    6
    Amylase increased
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    10 / 96 (10.42%)
    11 / 102 (10.78%)
         occurrences all number
    0
    0
    10
    11
    Diastolic hypotension
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    1
    0
    0
    1
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 97 (1.03%)
    0 / 96 (0.00%)
    3 / 102 (2.94%)
         occurrences all number
    0
    1
    0
    3
    Sinus bradycardia
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Nervous system disorders
    Somnolence
         subjects affected / exposed
    1 / 102 (0.98%)
    1 / 97 (1.03%)
    12 / 96 (12.50%)
    14 / 102 (13.73%)
         occurrences all number
    1
    1
    12
    14
    Headache
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 97 (0.00%)
    1 / 96 (1.04%)
    2 / 102 (1.96%)
         occurrences all number
    1
    0
    1
    2
    Brain fog
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    1 / 96 (1.04%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    1
    1
    Syncope
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 97 (1.03%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    1
    0
    1
    Hypothermia
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    1
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    3 / 102 (2.94%)
         occurrences all number
    0
    0
    0
    3
    Vaccination site reaction
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    3 / 102 (2.94%)
    1 / 97 (1.03%)
    0 / 96 (0.00%)
    4 / 102 (3.92%)
         occurrences all number
    3
    1
    0
    4
    Abdominal pain upper
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    1
    0
    0
    1
    Nausea
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 97 (1.03%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    1
    0
    1
    Vomiting
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 97 (1.03%)
    0 / 96 (0.00%)
    2 / 102 (1.96%)
         occurrences all number
    0
    1
    0
    2
    Anorectal disorder
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 102 (1.96%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    2 / 102 (1.96%)
         occurrences all number
    2
    0
    0
    2
    Rhinitis allergic
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Dermatitis contact
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 97 (1.03%)
    1 / 96 (1.04%)
    1 / 102 (0.98%)
         occurrences all number
    0
    1
    1
    2
    Dermatitis diaper
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Infections and infestations
    Beta haemolytic streptococcal infection
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    1 / 96 (1.04%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    1
    1
    COVID-19
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    2 / 102 (1.96%)
         occurrences all number
    1
    0
    0
    2
    Conjunctivitis
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    1
    0
    0
    1
    Enterocolitis viral
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    1 / 96 (1.04%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    1
    1
    Nasopharyngitis
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    1 / 96 (1.04%)
    2 / 102 (1.96%)
         occurrences all number
    0
    0
    1
    2
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 97 (1.03%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    1
    0
    1
    Blood alkaline phosphatase increased
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Blood bicarbonate decreased
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Ear infection
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Gastroenteritis Escherichia coli
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Influenza
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Laryngitis
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Pharyngotonsillitis
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Respiratory tract infection
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    2 / 102 (1.96%)
         occurrences all number
    0
    0
    0
    2
    Rhinitis
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1
    Staphylococcal skin infection
         subjects affected / exposed
    0 / 102 (0.00%)
    0 / 97 (0.00%)
    0 / 96 (0.00%)
    1 / 102 (0.98%)
         occurrences all number
    0
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    23 Sep 2021
    The overall rationale for the changes implemented in the amended protocol was to overcome inconsistencies between the flowchart, synopsis and the full text body, respectively. Furthermore, β-estradiol was being presented as NIMP supplied centrally to Non-USA based sites.
    28 Feb 2024
    The overall rationale for the changes implemented in the amended protocol was to update the number of sites and list of countries involved in this trial, to update the contact information for service providers, and to introduce the collection of certified redacted paper ECG copies.

    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.
    Considering that macimorelin has been validated successfully as diagnostic test in the adult population, post-hoc analyses activities have been started to understand the reasons for the efficacy outcome.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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