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    Clinical Trial Results:
    Evaluation of tolerance, suckling and food intake after repeated nasals administrations of Oxytocin in PWS infants

    Summary
    EudraCT number
    		 2012-005325-67
    Trial protocol
    		 FR  
    Global end of trial date
    15 Jul 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Dec 2023
    First version publication date
    28 Dec 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    1239102
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02205034
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    CHu de Toulouse
    Sponsor organisation address
    2 rue viguerie, Toulouse, France, 31059
    Public contact
    Nadège ALGANS, CHU de Toulouse, +33 056177204, algans.n@chu-toulouse.fr
    Scientific contact
    Maithé TAUBER, CHU de Toulouse, +33 0534558551, tauber.m@chu-toulouse.fr
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-003148-PIP01-21
    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 Jun 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    15 Jul 2014
    Global end of trial reached?
    Yes
    Global end of trial date
    15 Jul 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To study tolerance of oxytocin repeated nasal administrations during 7 days (according to 3 plans of administration) to babies with Prader Willi Syndrom younger than 5 months.
    Protection of trial subjects
    A monitoring committee composed of the investigating physicians and a representative of the Research Department will meet every 6 months for the duration of the study. It will be in charge of analyzing the data, in particular the tolerance data, and of considering the course of action in case of repeated serious adverse events not related a priori to oxytocin.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    13 May 2013
    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: 18
    Worldwide total number of subjects
    18
    EEA total number of subjects
    18
    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)
    18
    Children (2-11 years)
    0
    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
    		 Children were recruited into the study as part of their disease caring. The study was proposed by the investigating physician to the representatives of the parental authority so that the children can be included in the study.

    Pre-assignment
    Screening details
    		 The investigating physician has given information about the study to the representatives of the child's parental authority within 10 days prior to inclusion and answered all their questions concerning the objective, the nature of the constraints, the foreseeable risks and the expected benefits of the research and verified the inclusion criteria

    Period 1
    Period 1 title
    overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 arm 1 : 4UI every 2 days
    Arm description
    		 children received 4UI of OT per day
    Arm type
    		 Experimental

    Investigational medicinal product name
    Oxytocin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nasal drops, solution
    Routes of administration
    Intranasal use
    Dosage and administration details
    4 UI per day

    Arm title
    		 arm2 : 4 UI/day
    Arm description
    		 children received 4UI/day of Oxytocin
    Arm type
    		 Experimental

    Investigational medicinal product name
    Oxytocin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nasal drops, solution
    Routes of administration
    Intranasal use
    Dosage and administration details
    4 UI per day

    Arm title
    		 arm 3 : 8 UI/day
    Arm description
    		 children received 4UI two times per day
    Arm type
    		 Experimental

    Investigational medicinal product name
    Oxytocin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nasal drops, solution
    Routes of administration
    Intranasal use
    Dosage and administration details
    4 UI per day

    Number of subjects in period 1
    		arm 1 : 4UI every 2 days arm2 : 4 UI/day arm 3 : 8 UI/day
    Started
    6
    6
    6
    Completed
    5
    6
    5
    Not completed
    1
    0
    1
         Adverse event, non-fatal
    1
    -
    -
         Lost to follow-up
    -
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    overall trial
    Reporting group description
    		 18 infants less than 3 months old

    Reporting group values
    		 overall trial Total
    Number of subjects
    		 18 18
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 18 18
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
        infants
    		 0 0
        infants < 6 months
    		 0 0
    Age continuous
    Age of infants recorded in months
    Units: months
        median (full range (min-max))
    		 3.8 (1.0 to 6.0) -
    Gender categorical
    Units: Subjects
        Female
    		 8 8
        Male
    		 10 10
    Subject analysis sets

    Subject analysis set title
    Safety analysis
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Safety was analysed by collecting adverse events

    Subject analysis sets values
    		 Safety analysis
    Number of subjects
    18
    Age categorical
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
        Newborns (0-27 days)
    0
        Infants and toddlers (28 days-23 months)
    18
        Children (2-11 years)
    0
        Adolescents (12-17 years)
    0
        Adults (18-64 years)
    0
        From 65-84 years
    0
        85 years and over
    0
        infants
    0
        infants < 6 months
    0
    Age continuous
    Age of infants recorded in months
    Units: months
        median (full range (min-max))
    3.8 (1.0 to 6.0)
    Gender categorical
    Units: Subjects
        Female
    8
        Male
    10

    End points

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    End points reporting groups
    Reporting group title
    arm 1 : 4UI every 2 days
    Reporting group description
    		 children received 4UI of OT per day

    Reporting group title
    arm2 : 4 UI/day
    Reporting group description
    		 children received 4UI/day of Oxytocin

    Reporting group title
    arm 3 : 8 UI/day
    Reporting group description
    		 children received 4UI two times per day

    Subject analysis set title
    Safety analysis
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Safety was analysed by collecting adverse events

    Primary: systemic adverse effects

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    End point title
    		 systemic adverse effects [1]
    End point description
    End point type
    Primary
    End point timeframe
    overall trial
    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: There was no systemic effects observed so no statistical analyses have been conducted.
    End point values
    		 arm 1 : 4UI every 2 days arm2 : 4 UI/day arm 3 : 8 UI/day
    Number of subjects analysed
    6
    6
    6
    Units: numbers
    10
    9
    6
    No statistical analyses for this end point

    Secondary: NOMAS score

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    End point title
    		 NOMAS score
    End point description
    NOMAS score evaluate sucking/swallowing abilitites of infants during feeding; endpoint is the % of infants who reached a NOMAS score <= 10 (normal score)
    End point type
    Secondary
    End point timeframe
    Before and after 7 days of treatment
    End point values
    		 arm 1 : 4UI every 2 days arm2 : 4 UI/day arm 3 : 8 UI/day
    Number of subjects analysed
    6
    6
    5
    Units: percentage
    67
    100
    100
    No statistical analyses for this end point

    Secondary: Videofluoroscopy of swallowing score (VFSS score)

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    End point title
    		 Videofluoroscopy of swallowing score (VFSS score)
    End point description
    change from baseline (before treatment) of VFSS score
    End point type
    Secondary
    End point timeframe
    before and after 7 days of treatment
    End point values
    		 arm 1 : 4UI every 2 days arm2 : 4 UI/day arm 3 : 8 UI/day
    Number of subjects analysed
    5
    5
    6
    Units: number
        arithmetic mean (standard deviation)
    -5.2 ( 4.1 )
    -5.2 ( 2.7 )
    -4.8 ( 2.2 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    The investigator shall report to the sponsor, without delay from the date of knowledge, any serious adverse event. The sponsor promptly reports unexpected AEs and developments during the course of the research.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    NA
    Reporting groups
    Reporting group title
    adverse event of study participants
    Reporting group description
    		 -

    Serious adverse events
    		 adverse event of study participants
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 18 (5.56%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Infections and infestations
    pneumonia
    Additional description: Pneumonia with infectious focus on the left.
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 adverse event of study participants
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    18 / 18 (100.00%)
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    3 / 18 (16.67%)
         occurrences all number
    3
    Gastrointestinal disorders
    Teething
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Constipation
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Gastroesophageal reflux disease
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Diarrhoea
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Infantile colic
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Hepatobiliary disorders
    Hepatobiliary injury
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Rash maculo-papular
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Hyperhidrosis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Lung infection
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Conjunctivis bacterial
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Urinary tract infection
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Oral candidiasis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    25 Sep 2013
    Primary and secondary endpoints evaluation report following the 1st dose level. In order to be able to start the second step corresponding to 4 IU of oxytocin every day in one dose (i.e. 7 administrations) following the good progress of step 1 equivalent to 4 IU every 2 days in one dose (i.e. 4 administrations), we transmit you the results obtained on the 6 babies who participated in the first step. The research protocol, the information leaflet and the observation booklet are not modified.
    03 Mar 2014
    As for the 2nd level, the ANSM authorizaition is requested to start the third level by presentation of vigilance results of the second level.

    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

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/28100688
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