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    Clinical Trial Results:
    A multicenter, Phase 2, randomized, open label, active-controlled, parallel-group study investigating the safety, tolerability, and efficacy of different dose levels of ACP-001 administered once weekly versus standard daily rhGH replacement therapy in pre-pubertal children with Growth Hormone Deficiency (GHD)

    Summary
    EudraCT number
    2012-002787-27
    Trial protocol
    HU   DE   CZ   GR   BG   SI   FR  
    Global end of trial date
    22 Jul 2015

    Results information
    Results version number
    v1(current)
    This version publication date
    04 Feb 2017
    First version publication date
    04 Feb 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ACP-001_CT-004
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01947907
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Ascendis Pharma A/S
    Sponsor organisation address
    Tuborg Boulevard 5, Hellerup, Denmark, DK-2900
    Public contact
    Michael Beckert, VP Clinical Development , Ascendis Pharma A/S, +49 172 155 2596, mb@ascendispharma.com
    Scientific contact
    Michael Beckert, VP Clinical Development , Ascendis Pharma A/S, +49 172 155 2596, mb@ascendispharma.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
    28 Jan 2016
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    22 Jul 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    22 Jul 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To compare the safety and PK/PD profile of three different ACP-001 doses to that of a commercially available standard daily rhGH formulation in pre-pubertal children with growth failure due to GHD.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    16 Jul 2013
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Turkey: 3
    Country: Number of subjects enrolled
    Ukraine: 7
    Country: Number of subjects enrolled
    Belarus: 8
    Country: Number of subjects enrolled
    Bulgaria: 1
    Country: Number of subjects enrolled
    Egypt: 7
    Country: Number of subjects enrolled
    Greece: 3
    Country: Number of subjects enrolled
    Hungary: 1
    Country: Number of subjects enrolled
    Poland: 1
    Country: Number of subjects enrolled
    Romania: 3
    Country: Number of subjects enrolled
    Russian Federation: 21
    Worldwide total number of subjects
    55
    EEA total number of subjects
    9
    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)
    55
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    A total of 170 subjects from 38 initiated centers were screened for inclusion into the study of which 115 subjects were classified as screen failures. A total of 55 subjects were randomized at 20 centers in 10 countries located in Europe and North Africa (EEA and non-EEA).

    Pre-assignment
    Screening details
    A total of 170 subjects from 30 recruiting centers were screened for inclusion into the study of which 115 subjects were classified as screen failures.

    Pre-assignment period milestones
    Number of subjects started
    55
    Number of subjects completed
    53

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    Consent withdrawn by subject: 2
    Period 1
    Period 1 title
    Overall Trial (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Cohort 1
    Arm description
    ACP-001 once weekly in a dose equivalent to 0.14 mg rhGH/kg/week
    Arm type
    Experimental

    Investigational medicinal product name
    ACP-001
    Investigational medicinal product code
    Other name
    TransCon PEG hGH
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    ACP-001 was administered in a weekly dose strength equivalent to 0.14 mg rhGH/kg/week, for 26 weeks. Provided in glass vials, reconstituted with sterile water for injection, administered in the morning hours.

    Arm title
    Cohort 2
    Arm description
    ACP-001 once weekly in a dose equivalent to 0.21 mg rhGH/kg/week
    Arm type
    Experimental

    Investigational medicinal product name
    ACP-001
    Investigational medicinal product code
    Other name
    TransCon PEG hGH
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    ACP-001 was administered in a weekly dose strength equivalent to 0.21 mg rhGH/kg/week, for 26 weeks. Provided in glass vials, reconstituted with sterile water for injection, administered in the morning hours.

    Arm title
    Cohort 3
    Arm description
    ACP-001 once weekly in a dose equivalent to 0.30 mg rhGH/kg/week
    Arm type
    Experimental

    Investigational medicinal product name
    ACP-001
    Investigational medicinal product code
    Other name
    TransCon PEG hGH
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    ACP-001 was administered in a weekly dose strength equivalent to 0.30 mg rhGH/kg/week, for 26 weeks. Provided in glass vials, reconstituted with sterile water for injection, administered in the morning hours.

    Arm title
    Cohort 4
    Arm description
    Genotropin® once daily in a dose equivalent to 0.21 mg rhGH/kg/week
    Arm type
    Active comparator

    Investigational medicinal product name
    Genotropin®
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Genotropin® was administered daily in a standard daily dose of 0.03 mg rhGH/kg/day (equivalent to 0.21 mg rhGH/kg/week), for 26 weeks. Provided as sterile white lyophilized powder dispensed in a two-chamber cartridge, administered with an injection device in the evening hours.

    Number of subjects in period 1 [1]
    Cohort 1 Cohort 2 Cohort 3 Cohort 4
    Started
    12
    14
    14
    13
    Completed
    12
    14
    14
    13
    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: During the pre-assignment period 2 subjects withdrew the consent.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    ACP-001 once weekly in a dose equivalent to 0.14 mg rhGH/kg/week

    Reporting group title
    Cohort 2
    Reporting group description
    ACP-001 once weekly in a dose equivalent to 0.21 mg rhGH/kg/week

    Reporting group title
    Cohort 3
    Reporting group description
    ACP-001 once weekly in a dose equivalent to 0.30 mg rhGH/kg/week

    Reporting group title
    Cohort 4
    Reporting group description
    Genotropin® once daily in a dose equivalent to 0.21 mg rhGH/kg/week

    Reporting group values
    Cohort 1 Cohort 2 Cohort 3 Cohort 4 Total
    Number of subjects
    12 14 14 13 53
    Age categorical
    Units: Subjects
        In utero
    0 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0 0 0
        Newborns (0-27 days)
    0 0 0 0 0
        Infants and toddlers (28 days-23 months)
    0 0 0 0 0
        Children (2-11 years)
    12 14 14 13 53
        Adolescents (12-17 years)
    0 0 0 0 0
        Adults (18-64 years)
    0 0 0 0 0
        From 65-84 years
    0 0 0 0 0
        85 years and over
    0 0 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    7.98 ± 2.889 8.24 ± 2.13 7.32 ± 2.784 7.53 ± 2.483 -
    Gender categorical
    Units: Subjects
        Female
    3 4 5 3 15
        Male
    9 10 9 10 38
    Race
    Units: Subjects
        White
    12 14 14 13 53
    Baseline IGF-I SDS
    Units: --
        arithmetic mean (standard deviation)
    -2.034 ± 0.7429 -2.017 ± 0.7713 -2.19 ± 0.7169 -2.502 ± 0.896 -
    Baseline Height SDS
    Units: --
        arithmetic mean (standard deviation)
    -3.05 ± 1.127 -2.75 ± 0.383 -3.17 ± 1.04 -3.27 ± 1.077 -
    Screening Peak GH Levels
    The highest GH peak serum level of the two GH stimulation tests at Screening was used for calculation.
    Units: ng/mL
        arithmetic mean (standard deviation)
    5.09 ± 3.169 5.16 ± 2.598 4.44 ± 2.77 5.15 ± 3.068 -
    Subject analysis sets

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    The Safety Analysis Set (SAS) includes all patients who receive at least one dose of planned study medication.

    Subject analysis set title
    Full Analysis Set
    Subject analysis set type
    Full analysis
    Subject analysis set description
    The Full Analysis Set (FAS) includes all patients who are randomized, receive at least one dose of planned study medication and provide a baseline and at least one post-baseline height measurement value.

    Subject analysis set title
    Per Protocol Analysis Set
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Patients with major protocol deviations or premature termination of the treatment due to reasons that were definitely not related to study medication will be excluded from the Per-Protocol (PP) analysis. Major protocol deviations will be detailed in the Protocol Deviation Plan and will include aspects of: o availability of measurements, o non-compliance with respect to assigned study medication, assigned dose level and / or dose regimen, o non-compliance to visit schedule (flexibility defined by visit windows), o failure to satisfy inclusion or exclusion criteria, o taking any not permitted concomitant medication during the study, o other parameters.

    Subject analysis sets values
    Safety Analysis Set Full Analysis Set Per Protocol Analysis Set
    Number of subjects
    53
    53
    51
    Age categorical
    Units: Subjects
        In utero
    0
    0
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
    0
    0
        Newborns (0-27 days)
    0
    0
    0
        Infants and toddlers (28 days-23 months)
    0
    0
    0
        Children (2-11 years)
    53
    53
    51
        Adolescents (12-17 years)
    0
    0
    0
        Adults (18-64 years)
    0
    0
    0
        From 65-84 years
    0
    0
    0
        85 years and over
    0
    0
    0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    7.76 ± 2.53
    7.76 ± 2.53
    7.76 ± 2.542
    Gender categorical
    Units: Subjects
        Female
    15
    15
    15
        Male
    38
    38
    36
    Race
    Units: Subjects
        White
    53
    53
    51
    Baseline IGF-I SDS
    Units: --
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    Baseline Height SDS
    Units: --
        arithmetic mean (standard deviation)
    ±
    ±
    ±
    Screening Peak GH Levels
    The highest GH peak serum level of the two GH stimulation tests at Screening was used for calculation.
    Units: ng/mL
        arithmetic mean (standard deviation)
    ±
    ±
    ±

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    ACP-001 once weekly in a dose equivalent to 0.14 mg rhGH/kg/week

    Reporting group title
    Cohort 2
    Reporting group description
    ACP-001 once weekly in a dose equivalent to 0.21 mg rhGH/kg/week

    Reporting group title
    Cohort 3
    Reporting group description
    ACP-001 once weekly in a dose equivalent to 0.30 mg rhGH/kg/week

    Reporting group title
    Cohort 4
    Reporting group description
    Genotropin® once daily in a dose equivalent to 0.21 mg rhGH/kg/week

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    The Safety Analysis Set (SAS) includes all patients who receive at least one dose of planned study medication.

    Subject analysis set title
    Full Analysis Set
    Subject analysis set type
    Full analysis
    Subject analysis set description
    The Full Analysis Set (FAS) includes all patients who are randomized, receive at least one dose of planned study medication and provide a baseline and at least one post-baseline height measurement value.

    Subject analysis set title
    Per Protocol Analysis Set
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Patients with major protocol deviations or premature termination of the treatment due to reasons that were definitely not related to study medication will be excluded from the Per-Protocol (PP) analysis. Major protocol deviations will be detailed in the Protocol Deviation Plan and will include aspects of: o availability of measurements, o non-compliance with respect to assigned study medication, assigned dose level and / or dose regimen, o non-compliance to visit schedule (flexibility defined by visit windows), o failure to satisfy inclusion or exclusion criteria, o taking any not permitted concomitant medication during the study, o other parameters.

    Primary: Incidence of anti-hGH binding antibody formation

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    End point title
    Incidence of anti-hGH binding antibody formation [1]
    End point description
    Number of subjects with positive results for Anti-hGH binding antibodies at two consecutive post-dose visits
    End point type
    Primary
    End point timeframe
    Visit 2 - Visit 5
    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: As this is an exploratory study, no hypothesis tests were performed.
    End point values
    Cohort 1 Cohort 2 Cohort 3 Cohort 4
    Number of subjects analysed
    12
    14
    14
    13
    Units: Number of subjects with positive results
    1
    0
    0
    0
    No statistical analyses for this end point

    Primary: Incidence of anti-hGH neutralizing antibody formation

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    End point title
    Incidence of anti-hGH neutralizing antibody formation [2]
    End point description
    Number of subjects with positive results for anti-hGH neutralizing antibodies at two consecutive post-dose visits
    End point type
    Primary
    End point timeframe
    Visit 2 - Visit 5
    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: As this is an exploratory study, no hypothesis tests were performed.
    End point values
    Cohort 1 Cohort 2 Cohort 3 Cohort 4
    Number of subjects analysed
    12
    14
    14
    13
    Units: Number of subjects with positive results
    0
    0
    0
    0
    No statistical analyses for this end point

    Primary: Local tolerability (assessed by the patient and the investigator)

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    End point title
    Local tolerability (assessed by the patient and the investigator) [3]
    End point description
    Overview of subject number of local tolerability assessment results
    End point type
    Primary
    End point timeframe
    From start of study treatment and continues until the end of the patient’s participation in the study
    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: As this is an exploratory study, no hypothesis tests were performed.
    End point values
    Cohort 1 Cohort 2 Cohort 3 Cohort 4
    Number of subjects analysed
    12
    14
    14
    13
    Units: Number of Subjects with ISRs
    7
    6
    6
    6
    No statistical analyses for this end point

    Primary: Cmax of hGH

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    End point title
    Cmax of hGH [4]
    End point description
    As part of the following endpoint: PK profile of serum hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the PK profile of hGH from the daily rhGH group during V1 and V3 Uncorrected Cmax (maximum value of concentration) values at Week 13
    End point type
    Primary
    End point timeframe
    0 hours to 168 hours at Visit 3 (Week 13)
    Notes
    [4] - 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: As this is an exploratory study, no hypothesis tests were performed.
    End point values
    Cohort 1 Cohort 2 Cohort 3 Cohort 4
    Number of subjects analysed
    12
    14
    14
    13
    Units: ng/mL
        arithmetic mean (standard deviation)
    12.558 ± 8.678
    13.418 ± 9.428
    31.8 ± 17.499
    16.612 ± 12.777
    No statistical analyses for this end point

    Primary: AUC0-168h of hGH

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    End point title
    AUC0-168h of hGH [5]
    End point description
    As part of the following endpoint: PK profile of serum hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the PK profile of hGH from the daily rhGH group during V1 and V3. Uncorrected AUC0-168h (area under the curve from 0h to 168h) values at Week 13 For Cohort 4 AUC0-24h*7 has been presented
    End point type
    Primary
    End point timeframe
    0 hours to 168 hours at Visit 3 (Week 13)
    Notes
    [5] - 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: As this is an exploratory study, no hypothesis tests were performed.
    End point values
    Cohort 1 Cohort 2 Cohort 3 Cohort 4
    Number of subjects analysed
    12
    14
    14
    13
    Units: h*ng/mL
        arithmetic mean (standard deviation)
    696.34 ± 410.096
    787.41 ± 483.169
    2167.43 ± 1064.729
    556.88 ± 412.618
    No statistical analyses for this end point

    Primary: Etrough of IGF-I

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    End point title
    Etrough of IGF-I [6] [7]
    End point description
    As part of the following endpoint: PD profile of serum IGF-I during V1 and V3 compared between the ACP-001 dose groups and to the daily rhGH group. Uncorrected Etrough (the pre-dose efficacy response) values at Week 13
    End point type
    Primary
    End point timeframe
    0 hours to 168 hours at Visit 3 (Week 13)
    Notes
    [6] - 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: As this is an exploratory study, no hypothesis tests were performed.
    [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The difference in the dosing regimen of investigational product and comparator does not enable to consistently compare PD parameters across treatments.
    End point values
    Cohort 1 Cohort 2 Cohort 3
    Number of subjects analysed
    12
    14
    14
    Units: ng/mL
        arithmetic mean (standard deviation)
    97.17 ± 50.53
    156 ± 76.235
    167.83 ± 74.01
    No statistical analyses for this end point

    Primary: Emax of IGF-I

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    End point title
    Emax of IGF-I [8] [9]
    End point description
    As part of the following endpoint: PD profile of serum IGF-I during V1 and V3 compared between the ACP-001 dose groups and to the daily rhGH group. Uncorrected Emax (the maximum observed efficacy response) values at Week 13
    End point type
    Primary
    End point timeframe
    0 hours to 168 hours at Visit 3 (Week 13)
    Notes
    [8] - 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: As this is an exploratory study, no hypothesis tests were performed.
    [9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The difference in the dosing regimen of investigational product and comparator does not enable to consistently compare PD parameters across treatments.
    End point values
    Cohort 1 Cohort 2 Cohort 3
    Number of subjects analysed
    12
    14
    14
    Units: ng/mL
        arithmetic mean (standard deviation)
    209.5 ± 120.189
    276 ± 126.632
    289.92 ± 129.469
    No statistical analyses for this end point

    Primary: AUEC0-168h of IGF-I

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    End point title
    AUEC0-168h of IGF-I [10] [11]
    End point description
    As part of the following endpoint: PD profile of serum IGF-I during V1 and V3 compared between the ACP-001 dose groups and to the daily rhGH group. Uncorrected AUEC0-168 (Area Under the Efficacy Curve (AUC) from 0h to 168h) values at Week 13
    End point type
    Primary
    End point timeframe
    0 hours to 168 hours at Visit 3 (Week 13)
    Notes
    [10] - 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: As this is an exploratory study, no hypothesis tests were performed.
    [11] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: The difference in the dosing regimen of investigational product and comparator does not enable to consistently compare PD parameters across treatments.
    End point values
    Cohort 1 Cohort 2 Cohort 3
    Number of subjects analysed
    12
    14
    14
    Units: h*ng/mL
        arithmetic mean (standard deviation)
    28526.19 ± 15756.44
    35591.94 ± 17068.59
    36066.01 ± 17379.44
    No statistical analyses for this end point

    Secondary: Annualized HV during treatment with ACP-001 or daily rhGH at the end of 6 months, for each ACP-001 dose group and for the daily rhGH dose group

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    End point title
    Annualized HV during treatment with ACP-001 or daily rhGH at the end of 6 months, for each ACP-001 dose group and for the daily rhGH dose group
    End point description
    Descriptive Statistics of Annualized Height Velocity
    End point type
    Secondary
    End point timeframe
    Baseline to 6 Months (Visit 5)
    End point values
    Cohort 1 Cohort 2 Cohort 3 Cohort 4
    Number of subjects analysed
    12
    14
    14
    13
    Units: cm/year
        arithmetic mean (standard deviation)
    11.93 ± 4.066
    12.89 ± 3.464
    13.85 ± 4.009
    11.64 ± 3.592
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From start of study treatment and continues until the end of the patient’s participation in the study (until 4 weeks after stop of patient’s study participation for serious adverse events).
    Adverse event reporting additional description
    Treatment-emergent adverse events are summarized.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    15.1
    Reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    ACP-001 once weekly in a dose equivalent to 0.14 mg rhGH/kg/week

    Reporting group title
    Cohort 2
    Reporting group description
    ACP-001 once weekly in a dose equivalent to 0.21 mg rhGH/kg/week

    Reporting group title
    Cohort 3
    Reporting group description
    ACP-001 once weekly in a dose equivalent to 0.30 mg rhGH/kg/week

    Reporting group title
    Cohort 4
    Reporting group description
    Genotropin® once daily in a dose equivalent to 0.21 mg rhGH/kg/week

    Serious adverse events
    Cohort 1 Cohort 2 Cohort 3 Cohort 4
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Gastrointestinal disorders
    Inguinal hernia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         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
    Cohort 1 Cohort 2 Cohort 3 Cohort 4
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 12 (58.33%)
    6 / 14 (42.86%)
    8 / 14 (57.14%)
    8 / 13 (61.54%)
    Immune system disorders
    Food allergy
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hyperthermia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Pyrexia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    3 / 13 (23.08%)
         occurrences all number
    0
    0
    1
    3
    Injury, poisoning and procedural complications
    Heat exhaustion
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Open wound
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Procedural dizziness
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Investigations
    Blood triglycerides
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Congenital, familial and genetic disorders
    Thalassaemia beta
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    2 / 14 (14.29%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    0
    2
    1
    0
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    2 / 14 (14.29%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    2
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    1 / 13 (7.69%)
         occurrences all number
    2
    0
    1
    1
    Eye disorders
    Strabismus
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Middle ear inflammation
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    0
    0
    1
    Gastrointestinal disorders
    Anal pruritus
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Diarrhoea
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Inguinal hernia
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Nausea
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Vomiting
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    1
    0
    0
    1
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 14 (7.14%)
    1 / 14 (7.14%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    1
    1
    Secondary hyperthyroidism
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 14 (7.14%)
    2 / 14 (14.29%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    2
    1
    Nasopharyngitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    0 / 14 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    1
    0
    0
    1
    Respiratory tract infection
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Rhinitis
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Tonsillitis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Tooth abscess
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 14 (7.14%)
    0 / 14 (0.00%)
    1 / 13 (7.69%)
         occurrences all number
    0
    1
    0
    1
    Tracheitis
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Varicella
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 14 (0.00%)
    1 / 14 (7.14%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0
    1
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Apr 2013
    The Protocol was amended: To address changes in Exclusion criteria To clarify and specify study procedures To add new references To address a change of address To clarify abbreviations and address typographical and grammar mistakes

    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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