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    Clinical Trial Results:
    A randomised, open-label, parallel-group, multi-centre trial comparing the efficacy and safety of 12 months treatment with one daily dose of ZOMACTON® to one daily dose of GENOTROPIN® in the treatment of children with idiopathic growth hormone deficiency

    Summary
    EudraCT number
    2008-004849-28
    Trial protocol
    HU  
    Global end of trial date
    25 Jun 2012

    Results information
    Results version number
    v1(current)
    This version publication date
    01 Mar 2016
    First version publication date
    26 Jul 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    FE 999905 CS07
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Ferring Pharmaceuticals A/S
    Sponsor organisation address
    Kay Fiskers Plads 11, Copenhagen S , Denmark, 2300
    Public contact
    Clinical Development Support, Ferring Pharmaceuticals, DK0-Disclosure@ferring.com
    Scientific contact
    Clinical Development Support, Ferring Pharmaceuticals, DK0-Disclosure@ferring.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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    04 Jul 2012
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Jun 2012
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To demonstrate that one daily dose of ZOMACTON (10 mg/mL) is equivalent to one daily dose of GENOTROPIN (12 mg/mL) in terms of growth measured as height velocity based on 12 months of treatment.
    Protection of trial subjects
    The target trial population was children aged 3-11 years old, who was unable to provide legally binding consent. Therefore, informed consent had to be sought from the parent(s)/legal representatives on the child’s behalf prior to enrolling the child in the trial. Oral information was given to the children and the parent(s)/legal representatives by an experienced investigator, or adequately trained delegate site staff. The information was also provided in writing. The process of the assent from each child was carried out slowly with an age-appropriate language. The assent process was conducted with sufficient time and at the same time as the consent was obtained from the parent(s)/legal representatives, so that the informed consent reflected the presumed will of the child. Besides the oral information given to the child, information sheet and assent form were provided in an age-appropriate language with wording that corresponded to the child’s psychological and intellectual maturity. Adequate time was given to the parent(s)/legal representative to discuss with their child with or without the presence of the Investigator, if required. The child’s assent was not sufficient to allow participation in research unless supplemented by informed consent of the parent(s)/legal representatives. The Investigator obtained freely given, written consent from each child’s parent(s)/legal representatives as well as a signed or indicated assent from each child after an appropriate explanation of the aims, methods, anticipated benefits, potential hazards, and any other aspects of the trial relevant to the decision of the child and parent(s)’/ legal representatives’ decision to participate.
    Background therapy
    -
    Evidence for comparator
    Growth hormone (GH) is essential for normal growth in children and acts by increasing growth, both via production of insulin-like growth factor (IGF), especially IGF-1, and via direct action on the growth plates. Lack of growth hormone in children leads to impairment of growth and eventually short stature. ZOMACTON is currently approved for treatment of children with growth hormone deficiency and for long-term treatment of growth retardation due to Turner’s syndrome (gonadal dysgenesis) confirmed by chromosomal analysis. Other indications for other somatropin-products (e.g., GENOTROPIN) include children born small for gestational age, Prader-Willi’s syndrome, patients with chronic renal insufficiency, and adults with growth hormone deficiency with adult or childhood onset. Biosimilarity would allow for expansion of the approved indications for ZOMACTON to include those approved for GENOTROPIN.
    Actual start date of recruitment
    22 Nov 2009
    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
    Ukraine: 41
    Country: Number of subjects enrolled
    Hungary: 11
    Country: Number of subjects enrolled
    India: 24
    Country: Number of subjects enrolled
    Israel: 30
    Country: Number of subjects enrolled
    Poland: 13
    Country: Number of subjects enrolled
    Romania: 11
    Country: Number of subjects enrolled
    Russian Federation: 35
    Worldwide total number of subjects
    165
    EEA total number of subjects
    35
    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)
    165
    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 259 subjects were screened in the trial of which 165 subjects were randomised: 82 in the ZOMACTON treatment group and 83 in the GENOTROPIN treatment group.

    Pre-assignment
    Screening details
    Trial was initiated with a pre-screening period during which time a confirmative standard GH stimulation test had to be performed. It took place up to 3 months (or 5 months in Israel) prior to the actual screening period. Pre-screening period was followed by a screening period which could be up to 21 days prior to actual 12-month treatment period.

    Period 1
    Period 1 title
    Visit 2 (Day 0)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Zomacton
    Arm description
    Zomacton 10 mg/mL: It was administered with ZomaJet® Vision X (subcutaneous transjection).
    Arm type
    Experimental

    Investigational medicinal product name
    Zomacton
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects randomised to ZOMACTON treatment received a daily dose of 0.03 mg/kg/day ZOMACTON (10 mg/mL) for 12 months. ZOMACTON was administered as a subcutaneous transjection using ZomaJet®Vision X.

    Arm title
    Genotropin
    Arm description
    Genotropin 12 mg/mL: It was administered with Genotropin Pen®12 (subcutaneous injection)
    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
    Subjects randomised to GENOTROPIN treatment received a daily dose of 0.03 mg/kg/day GENOTROPIN (12 mg/mL) for 12 months. GENOTROPIN was administered as a subcutaneous injection using Genotropin Pen®12.

    Number of subjects in period 1
    Zomacton Genotropin
    Started
    82
    83
    Completed
    82
    83
    Period 2
    Period 2 title
    Treatment period (Day 0 - 12 Month)
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Zomacton
    Arm description
    -
    Arm type
    Experimental

    Investigational medicinal product name
    Zomacton
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder and solvent for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Subjects randomised to ZOMACTON treatment received a daily dose of 0.03 mg/kg/day ZOMACTON (10 mg/mL) for 12 months. ZOMACTON was administered as a subcutaneous transjection using ZomaJet®Vision X.

    Arm title
    Genotropin
    Arm description
    -
    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
    Subjects randomised to GENOTROPIN treatment received a daily dose of 0.03 mg/kg/day GENOTROPIN (12 mg/mL) for 12 months. GENOTROPIN was administered as a subcutaneous injection using Genotropin Pen®12.

    Number of subjects in period 2
    Zomacton Genotropin
    Started
    82
    83
    Completed
    79
    82
    Not completed
    3
    1
         Pre-treatment adverse event
    1
    -
         Other
    -
    1
         Lack of efficacy
    1
    -
         Consent withdrawn by subject
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Zomacton
    Reporting group description
    Zomacton 10 mg/mL: It was administered with ZomaJet® Vision X (subcutaneous transjection).

    Reporting group title
    Genotropin
    Reporting group description
    Genotropin 12 mg/mL: It was administered with Genotropin Pen®12 (subcutaneous injection)

    Reporting group values
    Zomacton Genotropin Total
    Number of subjects
    82 83 165
    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)
    82 83 165
        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
    All the subjects were paediatric.
    Units: years
        arithmetic mean (standard deviation)
    7.09 ± 2.29 7.27 ± 2.2 -
    Gender categorical
    At baseline (at Visit 0)
    Units: Subjects
        Female
    27 28 55
        Male
    55 55 110
    Race
    At baseline (at Visit 0)
    Units: Subjects
        Asian
    12 13 25
        Black or African American
    1 0 1
        White
    69 70 139
    Bone age
    At baseline (at Visit 0)
    Units: Years
        arithmetic mean (standard deviation)
    4.29 ± 2.21 4.68 ± 2.09 -
    Baseline height
    At baseline (at Visit 0)
    Units: cm
        arithmetic mean (standard deviation)
    104 ± 12.8 106 ± 11.7 -
    Peak GH stimulation test
    At baseline (at Visit 0)
    Units: µg/L
        arithmetic mean (standard deviation)
    3.25 ± 2.65 2.95 ± 2.31 -
    Baseline Body Mass Index (BMI)
    Units: kg/m/m
        arithmetic mean (standard deviation)
    16 ± 2.27 16.3 ± 2.43 -
    Subject analysis sets

    Subject analysis set title
    Full analysis Set (FAS)
    Subject analysis set type
    Full analysis
    Subject analysis set description
    The FAS comprised data from all randomised and treated subjects. If a subject received incorrect treatment (i.e., the actual treatment was not as randomised), he or she was included in the group reflecting the actual treatment received.

    Subject analysis set title
    Per Protocol (PP) Analysis Set
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Subjects in the FAS analysis set were excluded from the PP analysis set if they met any of the pre-specified criteria of protocol deviation or otherwise excluded due to any serious unforeseen violations deemed to invalidate the data and affect the conclusions of the trial.

    Subject analysis set title
    Safety Analysis set
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    The definition of the safety analysis set was identical to the FAS.

    Subject analysis sets values
    Full analysis Set (FAS) Per Protocol (PP) Analysis Set Safety Analysis set
    Number of subjects
    165
    153
    165
    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)
    165
    153
    165
        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
    All the subjects were paediatric.
    Units: years
        arithmetic mean (standard deviation)
    7.18 ± 2.24
    7.15 ± 2.26
    7.18 ± 2.24
    Gender categorical
    At baseline (at Visit 0)
    Units: Subjects
        Female
    55
    51
    55
        Male
    110
    102
    110
    Race
    At baseline (at Visit 0)
    Units: Subjects
        Asian
    25
    22
    25
        Black or African American
    1
    1
    1
        White
    139
    130
    139
    Bone age
    At baseline (at Visit 0)
    Units: Years
        arithmetic mean (standard deviation)
    4.48 ± 2.16
    4.44 ± 2.17
    4.48 ± 2.16
    Baseline height
    At baseline (at Visit 0)
    Units: cm
        arithmetic mean (standard deviation)
    105 ± 12.3
    105 ± 12.4
    105 ± 12.3
    Peak GH stimulation test
    At baseline (at Visit 0)
    Units: µg/L
        arithmetic mean (standard deviation)
    3.1 ± 2.48
    3.07 ± 2.47
    3.1 ± 2.48
    Baseline Body Mass Index (BMI)
    Units: kg/m/m
        arithmetic mean (standard deviation)
    16.1 ± 2.35
    16.2 ± 2.35
    16.1 ± 2.35

    End points

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    End points reporting groups
    Reporting group title
    Zomacton
    Reporting group description
    Zomacton 10 mg/mL: It was administered with ZomaJet® Vision X (subcutaneous transjection).

    Reporting group title
    Genotropin
    Reporting group description
    Genotropin 12 mg/mL: It was administered with Genotropin Pen®12 (subcutaneous injection)
    Reporting group title
    Zomacton
    Reporting group description
    -

    Reporting group title
    Genotropin
    Reporting group description
    -

    Subject analysis set title
    Full analysis Set (FAS)
    Subject analysis set type
    Full analysis
    Subject analysis set description
    The FAS comprised data from all randomised and treated subjects. If a subject received incorrect treatment (i.e., the actual treatment was not as randomised), he or she was included in the group reflecting the actual treatment received.

    Subject analysis set title
    Per Protocol (PP) Analysis Set
    Subject analysis set type
    Per protocol
    Subject analysis set description
    Subjects in the FAS analysis set were excluded from the PP analysis set if they met any of the pre-specified criteria of protocol deviation or otherwise excluded due to any serious unforeseen violations deemed to invalidate the data and affect the conclusions of the trial.

    Subject analysis set title
    Safety Analysis set
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    The definition of the safety analysis set was identical to the FAS.

    Primary: Height velocity (HV) - Full analysis set

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    End point title
    Height velocity (HV) - Full analysis set
    End point description
    The HV was defined as: (height at visit – height at baseline) / actual length of time between the two measurements.
    End point type
    Primary
    End point timeframe
    12 months treatment
    End point values
    Zomacton Genotropin Full analysis Set (FAS)
    Number of subjects analysed
    80
    82
    162 [1]
    Units: cm/year
        arithmetic mean (standard deviation)
    10.7 ± 3.07
    10.9 ± 3.42
    10.8 ± 3.25
    Attachments
    HV Last Observation Carried Forward (LOCF)
    Notes
    [1] - Total no. of subjects at month 12 ' End of trial' Visit.
    Statistical analysis title
    ANCOVA of Height velocity at Month 12 (LOCF) - FAS
    Statistical analysis description
    Height velocity was analysed using an Analysis of Covariance (ANCOVA) model with baseline Chronological Age (CA), baseline HV, and log peak GH level after stimulation, as covariates and country, sex and treatment as factors. Equivalence was declared since the 95% confidence interval (CI) for the difference in HV was with [-2.0 ; 2.0] for both FAS and PP.
    Comparison groups
    Zomacton v Genotropin
    Number of subjects included in analysis
    162
    Analysis specification
    Pre-specified
    Analysis type
    equivalence
    Method
    Parameter type
    Mean difference (final values)
    Point estimate
    0.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.6
         upper limit
    1.1

    Primary: Height velocity - PP analysis set

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    End point title
    Height velocity - PP analysis set
    End point description
    The HV was defined as: (height at visit – height at baseline) / actual length of time between the two measurements.
    End point type
    Primary
    End point timeframe
    12 months treatment
    End point values
    Zomacton Genotropin Per Protocol (PP) Analysis Set
    Number of subjects analysed
    74
    74
    148 [2]
    Units: cm/year
        arithmetic mean (standard deviation)
    10.9 ± 3.07
    11.1 ± 3.23
    11 ± 3.15
    Notes
    [2] - Total no. of subjects at month 12 ' End of trial' Visit.
    Statistical analysis title
    ANCOVA of Height velocity at Month 12 (LOCF) - PP
    Statistical analysis description
    Height velocity was analysed using an ANCOVA model with baseline CA, baseline HV, and log peak GH level after stimulation, as covariates and country, sex and treatment as factors. Equivalence was declared since the 95% CI for the difference in HV was with [-2.0 ; 2.0] for both FAS and PP.
    Comparison groups
    Zomacton v Genotropin
    Number of subjects included in analysis
    148
    Analysis specification
    Pre-specified
    Analysis type
    equivalence
    Method
    Parameter type
    Mean difference (final values)
    Point estimate
    0.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.6
         upper limit
    1.1

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events (AEs) were monitored continuously throughout the study from the time of obtaining informed consent until the end of trial.
    Adverse event reporting additional description
    At each visit, AEs were elicited using a standard non-leading question. Adverse events could also be captured by symptoms spontaneously reported from the subject or as results of clinically significant changes and abnormalities observed by the Investigator. In addition, AEs were recorded in a booklet between the visits.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    12.1
    Reporting groups
    Reporting group title
    Zomacton
    Reporting group description
    Zomacton 10 mg/mL: It was administered with ZomaJet® Vision X (subcutaneous transjection).

    Reporting group title
    Genotropin
    Reporting group description
    Genotropin 12 mg/mL: It was administered with Genotropin Pen®12 (subcutaneous injection)

    Serious adverse events
    Zomacton Genotropin
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 82 (6.10%)
    0 / 83 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    1 / 82 (1.22%)
    0 / 83 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cough
         subjects affected / exposed
    1 / 82 (1.22%)
    0 / 83 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    2 / 82 (2.44%)
    0 / 83 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    abnormal behaviour
         subjects affected / exposed
    1 / 82 (1.22%)
    0 / 83 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Gastritis
         subjects affected / exposed
    1 / 82 (1.22%)
    0 / 83 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Viral infection
         subjects affected / exposed
    1 / 82 (1.22%)
    0 / 83 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Zomacton Genotropin
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    36 / 82 (43.90%)
    31 / 83 (37.35%)
    Investigations
    Insulin-like growth factor decreased
         subjects affected / exposed
    5 / 82 (6.10%)
    6 / 83 (7.23%)
         occurrences all number
    6
    9
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    3 / 82 (3.66%)
    5 / 83 (6.02%)
         occurrences all number
    3
    6
    Nervous system disorders
    Headache
         subjects affected / exposed
    6 / 82 (7.32%)
    3 / 83 (3.61%)
         occurrences all number
    6
    7
    General disorders and administration site conditions
    Injection site haematoma
         subjects affected / exposed
    9 / 82 (10.98%)
    4 / 83 (4.82%)
         occurrences all number
    10
    5
    Pyrexia
         subjects affected / exposed
    6 / 82 (7.32%)
    7 / 83 (8.43%)
         occurrences all number
    10
    9
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    7 / 82 (8.54%)
    7 / 83 (8.43%)
         occurrences all number
    8
    11
    Pharyngitis
         subjects affected / exposed
    7 / 82 (8.54%)
    1 / 83 (1.20%)
         occurrences all number
    9
    1
    Viral infection
         subjects affected / exposed
    5 / 82 (6.10%)
    3 / 83 (3.61%)
         occurrences all number
    5
    4

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    22 Dec 2009
    The original protocol required an X-ray for determination of bone age (BA) at baseline (performed at the screening). However, at some sites, an X-ray had already been taken prior to the pre-screening visit. Therefore, to avoid repeated X-rays, the period for performing this X-ray was extended. An X-ray taken within 3 months prior to visit 2 was acceptable. "The difference between CA-BA ≥1" was an inclusion criterion in original protocol whereas "BA above 9 years in girls and above 10 years in boys" was an exclusion criterion. This was to reassure that the subject was at pre-pubertal stage upon enrolment of the trial and able to complete the 12-month treatment period prior to entering the puberty. However, the restriction of "CA-BA ≥1" was not relevant for younger children. Therefore, the inclusion criterion was changed to "BA/CA ≤0.9". The baseline GH level for inclusion was changed from "9 ng/mL" to "10 ng/mL" as this was the cut-point value for initiation of GH treatment. Any use of corticoid steroids and medications that could interfere with GH treatment was prohibited in the original protocol. However, some children may suffer from adrenocorticotropic hormone deficiency and for these children glucocorticosteroid treatments are mandatory as physiological replacement. Hence, glucocorticosteroid treatment was allowed as long as it was at a stable dose level. As per exclusion criteria in the original protocol, children with any diagnosed or suspected severe chronic disease or clinical signs of dysmorphic features, malformations, or mental retardation were not allowed to be included in the trial. However, a child with minor dysmorphic features or malformation in stable condition could be enrolled into the trial as long as it doesn't place the child at excessive risk by participating in the trial, and therefore, the exclusion criteria were changed.

    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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