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    Clinical Trial Results:
    Effects of intranasal administrations of oxytocin on beahvioural troubles, hyperphagia and social skills in children with Prader-Willi syndrome aged from 3 to 12 years.

    Summary
    EudraCT number
    2016-003273-18
    Trial protocol
    FR  
    Global end of trial date
    11 Jan 2019

    Results information
    Results version number
    v1(current)
    This version publication date
    14 Jun 2024
    First version publication date
    14 Jun 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    RC31-15-7837
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03114371
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    CHU de Toulouse
    Sponsor organisation address
    2 rue de Viguerie, Toulouse, France, 31059
    Public contact
    Nadège ALGANS, University Hospital of Toulouse, +33 0561777204, algans.n@chu-toulouse.fr
    Scientific contact
    Dr Sophie Cabal-Berthoumieu, University Hospital of Toulouse, +33 05 34 55 74 32 , cabal-berthoumieu.s@chu-toulouse.fr
    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
    11 Jan 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 Jan 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Jan 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Study the effect of the administration intranasale of oxytocin (OXT) during 12 weeks on the behavior disorders by comparison with a placebo
    Protection of trial subjects
    An Independent Monitoring Committee (IMC) was formed to review the study data and to assess the safety of the OXYJEUNE study based on Serious and Non-Serious Events, in particular their potential relationship to the investigational drug.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    28 Nov 2016
    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
    France: 40
    Worldwide total number of subjects
    40
    EEA total number of subjects
    40
    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)
    35
    Adolescents (12-17 years)
    5
    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
    Patients with Prader-Willi Syndrome will be recruited by the PWS Reference Center of the Toulouse University Hospital from the active file of patients followed.

    Pre-assignment
    Screening details
    Patients with PWS aged from 3 to 12 years

    Period 1
    Period 1 title
    Baseline
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    OXYTOCIN
    Arm description
    Daily intranasal administrations of oxytocin for 12 weeks. Oxytocin dose will be 8 International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.
    Arm type
    Experimental

    Investigational medicinal product name
    Syntocinon PR1
    Investigational medicinal product code
    H01BB02
    Other name
    Pharmaceutical forms
    Nasal spray
    Routes of administration
    Nasal use
    Dosage and administration details
    The dosage of OXT will be 8 IU for patients aged 3-6 years and 16 IU for patients aged 7-12 years at inclusion.

    Arm title
    PLACEBO
    Arm description
    Daily intranasal administrations of placebo for 12 weeks. Oxytocin dose will be International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.
    Arm type
    Placebo

    Investigational medicinal product name
    Syntocinon PR1
    Investigational medicinal product code
    H01BB02
    Other name
    Pharmaceutical forms
    Nasal spray
    Routes of administration
    Nasal use
    Dosage and administration details
    The dosage of OXT will be 8 IU for patients aged 3-6 years and 16 IU for patients aged 7-12 years at inclusion.

    Number of subjects in period 1
    OXYTOCIN PLACEBO
    Started
    20
    20
    Completed
    20
    20
    Period 2
    Period 2 title
    OT vs Placebo
    Is this the baseline period?
    No
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Monitor, Data analyst, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    OXYTOCIN
    Arm description
    Oxytocin treatment 8 unit for patients aged from 3 to 6 years and 16 units for patients aged from 7 to 12.
    Arm type
    Experimental

    Investigational medicinal product name
    Syntocinon PR1
    Investigational medicinal product code
    H01BB02
    Other name
    Pharmaceutical forms
    Nasal spray
    Routes of administration
    Nasal use
    Dosage and administration details
    The dosage of OXT will be 8 IU for patients aged 3-6 years and 16 IU for patients aged 7-12 years at inclusion.

    Arm title
    Placebo
    Arm description
    2 spray for patients aged from 3 to 6 years 4 spray for patients aged from 7 to 12 years
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nasal spray, solution
    Routes of administration
    Nasal use
    Dosage and administration details
    2 spray for patients aged from 3 to 6 years 4 spray for patients aged from 7 to 12 years

    Number of subjects in period 2
    OXYTOCIN Placebo
    Started
    20
    20
    Completed
    19
    20
    Not completed
    1
    0
         Consent withdrawn by subject
    1
    -
    Period 3
    Period 3 title
    open label period
    Is this the baseline period?
    No
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    Oxytocin
    Arm description
    8 UI (2 spray) for patients aged from 3 to 6 years 16 UI (4 spray) for patients aged from 7 to 12 years
    Arm type
    Experimental

    Investigational medicinal product name
    Syntocinon
    Investigational medicinal product code
    HB01BB02
    Other name
    Pharmaceutical forms
    Nasal spray, solution
    Routes of administration
    Nasal use
    Dosage and administration details
    8 UI (2 spray) for patients aged from 3 to 6 years 16 UI (4 spray) for patients aged from 3 to 6 years

    Number of subjects in period 3
    Oxytocin
    Started
    39
    Completed
    38
    Not completed
    1
         Consent withdrawn by subject
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    OXYTOCIN
    Reporting group description
    Daily intranasal administrations of oxytocin for 12 weeks. Oxytocin dose will be 8 International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.

    Reporting group title
    PLACEBO
    Reporting group description
    Daily intranasal administrations of placebo for 12 weeks. Oxytocin dose will be International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.

    Reporting group values
    OXYTOCIN PLACEBO Total
    Number of subjects
    20 20 40
    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)
    18 17 35
        Adolescents (12-17 years)
    2 3 5
        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)
    6.7 ( 2.9 ) 7.2 ( 3.05 ) -
    Gender categorical
    Among all the children participating in the study, in the oxytocin (OTX) study arm there are 4 boys and 6 girls (3 and 6 years), and 5 boys and 5 girls (7/12 years). In the placebo arm there are 4 girls and 6 boys (3/6 years) and 2 boys and 8 girls (7/12 years).
    Units: Subjects
        Female
    11 12 23
        Male
    9 8 17
    age category
    Units: Subjects
        Age 3-6 years
    10 10 20
        Age 7-12 years
    10 10 20

    End points

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    End points reporting groups
    Reporting group title
    OXYTOCIN
    Reporting group description
    Daily intranasal administrations of oxytocin for 12 weeks. Oxytocin dose will be 8 International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.

    Reporting group title
    PLACEBO
    Reporting group description
    Daily intranasal administrations of placebo for 12 weeks. Oxytocin dose will be International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.
    Reporting group title
    OXYTOCIN
    Reporting group description
    Oxytocin treatment 8 unit for patients aged from 3 to 6 years and 16 units for patients aged from 7 to 12.

    Reporting group title
    Placebo
    Reporting group description
    2 spray for patients aged from 3 to 6 years 4 spray for patients aged from 7 to 12 years
    Reporting group title
    Oxytocin
    Reporting group description
    8 UI (2 spray) for patients aged from 3 to 6 years 16 UI (4 spray) for patients aged from 7 to 12 years

    Primary: Behavioural troubles (Child Behavior Check List Questionnaire)

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    End point title
    Behavioural troubles (Child Behavior Check List Questionnaire)
    End point description
    Change from baseline at week 12 of CBCL Total problems T score
    End point type
    Primary
    End point timeframe
    week 12
    End point values
    OXYTOCIN PLACEBO
    Number of subjects analysed
    19
    20
    Units: score
    19
    20
    Statistical analysis title
    Total problems score
    Statistical analysis description
    Change from baseline at Week 12 in Total Problems score
    Comparison groups
    OXYTOCIN v PLACEBO
    Number of subjects included in analysis
    39
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.05
    Method
    ANCOVA
    Confidence interval

    Secondary: Hyperphagia

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    End point title
    Hyperphagia
    End point description
    Responder analysis on the global score of Dykens Hyperphagia questionnaire: among the patients who had an abnormal score at baseline, number of patients with normal score at week 12
    End point type
    Secondary
    End point timeframe
    week 12
    End point values
    OXYTOCIN PLACEBO
    Number of subjects analysed
    19
    20
    Units: score
    19
    20
    No statistical analyses for this end point

    Secondary: Social skills

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    End point title
    Social skills
    End point description
    Responder analysis on social skills after 12 weeks of oxytocin/placebo treatment: the social skills were assessed by the PSA questionnaire in children aged from 3 to 6 years and by the SRS questionnaire for children aged from 7 to 12 years.Among the patients who had an abnormal score at baseline, number of patients with normal score at week 12
    End point type
    Secondary
    End point timeframe
    week 12
    End point values
    OXYTOCIN PLACEBO
    Number of subjects analysed
    19
    20
    Units: score
    19
    20
    No statistical analyses for this end point

    Secondary: Evaluation of auto- and hetero-aggressive behaviour

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    End point title
    Evaluation of auto- and hetero-aggressive behaviour
    End point description
    Responder analysis on ECAA questionnaire item 5 after 12 weeks of oxytocin/placebo treatment: among the patients who had an abnormal score at baseline, number of patients with normal score at week 12
    End point type
    Secondary
    End point timeframe
    week 12
    End point values
    OXYTOCIN PLACEBO
    Number of subjects analysed
    19
    20
    Units: score
    19
    20
    No statistical analyses for this end point

    Secondary: the effect of oxytocin (OXT) vs. placebo on psychopathology

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    End point title
    the effect of oxytocin (OXT) vs. placebo on psychopathology
    End point description
    Different scores are obtained: T scores from 50 to 64 are normal; T scores from 65 to 69 are borderline; beyond 69, we are in the pathological.
    End point type
    Secondary
    End point timeframe
    Evaluation based on the variation of each of the sub-scores obtained on the CBCL questionnaire between J0 and S12.
    End point values
    Number of subjects analysed
    Units: Score
    No statistical analyses for this end point

    Secondary: Evaluation of Global Clinical Status After 12 Weeks of Oxytocin/Placebo Treatment

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    End point title
    Evaluation of Global Clinical Status After 12 Weeks of Oxytocin/Placebo Treatment
    End point description
    It is improvement of the patient's overall clinical condition after 12 weeks of treatment with oxytocin/placebo. It's assessed by the Clinical Global Impression Scale's score. responder analysis: responders were defined as patients with a score of +1, +2 or +3
    End point type
    Secondary
    End point timeframe
    week 12
    End point values
    OXYTOCIN PLACEBO
    Number of subjects analysed
    19
    20
    Units: Score
    19
    20
    No statistical analyses for this end point

    Secondary: Evaluation of acyl and desacyl ghrelin plasma levels after 12 weeks of oxytocin/placebo treatment

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    End point title
    Evaluation of acyl and desacyl ghrelin plasma levels after 12 weeks of oxytocin/placebo treatment
    End point description
    It is evolution of circulating levels of acylated and deacylated ghrelin will be the variations of these rates and the variation of the relationships between day 0 and week 12.
    End point type
    Secondary
    End point timeframe
    beetween day 0 and week 12
    End point values
    OXYTOCIN PLACEBO
    Number of subjects analysed
    19
    20
    Units: score
    1
    19
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    The adverse events (serious or not) are reported during all the study.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    20.0
    Reporting groups
    Reporting group title
    oxytocin group
    Reporting group description
    Daily intranasal administrations of oxytocin for 12 weeks, followed by an open-label period of 12 weeks of oxytocin. Oxytocin dose will be 8 International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.

    Reporting group title
    Placebo group
    Reporting group description
    Daily intranasal administrations of placebo for 12 weeks, followed by an open-label period of 12 weeks of oxytocin. Oxytocin dose will be International Unit for patients aged from 3 to 6 years and 16 International Unit for patients aged from 7 to 12 years.

    Serious adverse events
    oxytocin group Placebo group
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 20 (0.00%)
    0 / 20 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    oxytocin group Placebo group
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    19 / 20 (95.00%)
    18 / 20 (90.00%)
    Nervous system disorders
    Nervous system disorders
         subjects affected / exposed
    6 / 20 (30.00%)
    3 / 20 (15.00%)
         occurrences all number
    6
    3
    General disorders and administration site conditions
    pyrexia and site administration disorder
         subjects affected / exposed
    3 / 20 (15.00%)
    4 / 20 (20.00%)
         occurrences all number
    3
    4
    Gastrointestinal disorders
    Gastrointestinal disorder
         subjects affected / exposed
    4 / 20 (20.00%)
    5 / 20 (25.00%)
         occurrences all number
    4
    5
    Psychiatric disorders
    Psychiatric disorders
         subjects affected / exposed
    4 / 20 (20.00%)
    1 / 20 (5.00%)
         occurrences all number
    4
    1
    Musculoskeletal and connective tissue disorders
    Musculoskeletal and connective tissue disorders
         subjects affected / exposed
    3 / 20 (15.00%)
    3 / 20 (15.00%)
         occurrences all number
    3
    3
    Infections and infestations
    infections and infestations
         subjects affected / exposed
    9 / 20 (45.00%)
    11 / 20 (55.00%)
         occurrences all number
    9
    11

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Apr 2017
    The method of randomization has changed: - Patients now have a unique subject number that will correspond to the sequence number and then the first letter of the last and first names. - The pre-selection visit has been modified: the investigating physician is responsible for obtaining the signed informed consent form. - Change in sampling times and the timing of the ECG was requested in order to avoid duplication of blood sampling and to simplify patient management. - The addition of the appendix 11 corresponding to the grid the global evolution of the patient in the different times between D0 and S12 and between J0 and S24.
    23 Apr 2018
    Modification of a secondary endpoint for the analysis of data from the Dykens Questionnaire; modification of a secondary endpoint for the assessment of the evolution of the patient's clinical condition; extension of the duration of inclusions; modification of the dosage of oxytocin treatment
    26 Nov 2018
    Addition of an Independent Monitoring Committee (IMC).
    11 Jan 2019
    - Collaboration with CROs for statistical analysis design (Excelsus) and statistical analysis (Atlanstat). - Modification of secondary objectives: Circulating levels of OXT are measured to verify whether OXT accumulates or remains stable as a function of administered doses. - Modification of primary endpoint: Some children changed age groups during the study, so the questionnaire used is clarified.

    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.
    NA
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