Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43865   clinical trials with a EudraCT protocol, of which   7286   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    Efficacité et tolérance d’un traitement par inhibiteur de l’aromatase (Anastrozole) pour limiter l’avance de maturation osseuse liée à une adrénarche pathologique chez des enfants ayant un syndrome de Silver Russell ou de Prader Willi

    Summary
    EudraCT number
    		 2011-003309-12
    Trial protocol
    		 FR  
    Global end of trial date
    26 Jan 2016

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 Jun 2022
    First version publication date
    26 Jun 2022
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    P100129
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01520467
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS
    Sponsor organisation address
    4 Avenue Victoria, PARIS, France, 75004
    Public contact
    Pr Irene NETCHINE, ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS, 33 144736631, irene.netchine@aphp.fr
    Scientific contact
    Pr Irene NETCHINE, ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS, 33 144736631, irene.netchine@aphp.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
    20 May 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    26 Jan 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Jan 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Evaluer l'efficacité de l'Anastrozole comparée à celle de son placebo pour ralentir la maturation osseuse au cours de l'adrénarche pathologique chez des enfants ayant un Syndrome de Silver Russell ou un Syndrome de Prader Willi
    Protection of trial subjects
    Quelques essais cliniques menés chez l’enfant montrent que l’Anastrozole est généralement bien toléré. Ils ne décrivent pas d’effet indésirable pouvant être lié à ce médicament. Les effets indésirables de l’Anastrozole dont l’indication est le cancer du sein chez la femme ménopausée sont rares : - Bouffées de chaleur, sécheresse vaginale et raréfaction des cheveux. - Troubles digestifs (nausées, vomissements et diarrhée), fatigue, somnolence, douleurs articulaires, raideur articulaire, maux de tête ou éruptions cutanées - Perturbations du bilan hépatique - Hépatite - De légères augmentations du cholestérol ont été observées Les prélèvements sanguins réalisés peuvent occasionner des désagréments sans caractère de gravité. Il peut s’agir du malaise vagal. Pour limiter ce risque, le patient est positionné sur un fauteuil de prélèvement pour procéder à la prise de sang. En cas de malaise, on soulèvera les jambes du patient jusqu’à disparition des signes cliniques. Il peut également s’agir d’un hématome au point de prélèvement sanguin. Par ailleurs, afin de limiter le stress ou la douleur liée à la ponction veineuse, la ponction veineuse pourra être réalisée après apposition d’un patch EMLA ou l’utilisation d’un masque qui distille du Protoxyde d’azote, selon le souhait de l'enfant.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    23 May 2012
    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
    France: 27
    Worldwide total number of subjects
    27
    EEA total number of subjects
    27
    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)
    27
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 27 enfants ont été inclus dans l'étude entre le 23/05/2012 et le 30/07/2014 dans 5 hôpital en France (2 sites à Paris, Toulouse, Angers et Lyon)

    Pre-assignment
    Screening details
    		 Patient ayant un Syndrome de Silver Russell ou un Syndrome de Prader Willi suivi dans un des 5 centres participants à l'étude. Les patients devaient présenter un adrénarche associée à un âge osseux égal au moins à l’âge chronologique, être traité par GH dans le cadre habituel de sa pathologie et ne pas avoir débuter une puberté centrale

    Period 1
    Period 1 title
    Inclusion
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Inclusion
    Arm description
    		 -
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		Inclusion
    Started
    27
    Completed
    25
    Not completed
    2
         Protocol deviation
    2
    Period 2
    Period 2 title
    Randomisation
    Is this the baseline period?
    		 Yes [1]
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Actif
    Arm description
    		 Anastrozole
    Arm type
    		 Experimental

    Investigational medicinal product name
    Arimidex
    Investigational medicinal product code
    Other name
    Anastrozole
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 mg par jour pendant 18 mois

    Arm title
    		 Placebo
    Arm description
    		 Placebo
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo Arimidex
    Investigational medicinal product code
    Other name
    Placebo
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    1 mg par jour pendant 18 mois

    Notes
    [1] - Period 1 is not the baseline period. It is expected that period 1 will be the baseline period.
    Justification: L’analyse principale sera réalisée en intention de traiter (ITT). Seuls les patients ayant été randomisés ont été analysés
    Number of subjects in period 2 [2]
    		Actif Placebo
    Started
    12
    13
    Completed
    12
    13
    Notes
    [2] - 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: Deux patients inclus n’ont pas été randomisés, un début de puberté centrale ayant été détecté sur le test au GnRH.

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Randomisation
    Reporting group description
    		 -

    Reporting group values
    		 Randomisation Total
    Number of subjects
    		 25 25
    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)
    		 0 0
        Children (2-11 years)
    		 25 25
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        median (full range (min-max))
    		 8.8 (5.8 to 11.6) -
    Gender categorical
    Units: Subjects
        Female
    		 11 11
        Male
    		 14 14
    Pathologie
    Units: Subjects
        Syndrome de Prader Willi
    		 21 21
        Syndrome de Silver Russel
    		 4 4

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Inclusion
    Reporting group description
    		 -
    Reporting group title
    Actif
    Reporting group description
    		 Anastrozole

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

    Primary: différence de vitesse de progression de la maturation osseuse d’au moins 9 mois après 18 mois de traitement

    		 Close Top of page
    End point title
    		 différence de vitesse de progression de la maturation osseuse d’au moins 9 mois après 18 mois de traitement
    End point description
    End point type
    Primary
    End point timeframe
    après 18 mois de traitement
    End point values
    		 Actif Placebo
    Number of subjects analysed
    12
    13
    Units: Nombre de patients en succès ou échec
        Succès
    6
    4
        Echec
    6
    9
    Statistical analysis title
    		 Analyse du critère principal
    Comparison groups
    Actif v Placebo
    Number of subjects included in analysis
    25
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.4283
    Method
    		 Fisher exact
    Confidence interval

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    18 mois
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19
    Reporting groups
    Reporting group title
    Anastrozole
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Serious adverse events
    		 Anastrozole Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 12 (0.00%)
    2 / 13 (15.38%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Psychiatric disorders
    INTENTIONAL DRUG MISUSE
    alternative dictionary used: MedDRA 16
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    ACUTE GASTROENTERITIS
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 13 (7.69%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Anastrozole Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 12 (100.00%)
    11 / 13 (84.62%)
    Gastrointestinal disorders
    transit disorder
    alternative dictionary used: NA NA
         subjects affected / exposed
    8 / 12 (66.67%)
    4 / 13 (30.77%)
         occurrences all number
    13
    9
    Abdominal pain
    alternative dictionary used: NA NA
         subjects affected / exposed
    1 / 12 (8.33%)
    2 / 13 (15.38%)
         occurrences all number
    1
    2
    Respiratory, thoracic and mediastinal disorders
    Asthmatic crisis
    alternative dictionary used: NA NA
         subjects affected / exposed
    2 / 12 (16.67%)
    1 / 13 (7.69%)
         occurrences all number
    3
    1
    Skin and subcutaneous tissue disorders
    Itch
    alternative dictionary used: NA NA
         subjects affected / exposed
    3 / 12 (25.00%)
    1 / 13 (7.69%)
         occurrences all number
    3
    1
    Hyperhidrosis
    alternative dictionary used: NA NA
         subjects affected / exposed
    0 / 12 (0.00%)
    2 / 13 (15.38%)
         occurrences all number
    0
    2
    Musculoskeletal and connective tissue disorders
    limb pain
    alternative dictionary used: NA NA
         subjects affected / exposed
    4 / 12 (33.33%)
    6 / 13 (46.15%)
         occurrences all number
    7
    7
    Asthenia
    alternative dictionary used: NA NA
         subjects affected / exposed
    5 / 12 (41.67%)
    2 / 13 (15.38%)
         occurrences all number
    6
    4
    Back pain
    alternative dictionary used: NA NA
         subjects affected / exposed
    2 / 12 (16.67%)
    0 / 13 (0.00%)
         occurrences all number
    2
    0
    Infections and infestations
    Fever, Headache
    alternative dictionary used: NA NA
         subjects affected / exposed
    3 / 12 (25.00%)
    3 / 13 (23.08%)
         occurrences all number
    3
    5
    Otorhinolaryngology Infection
    alternative dictionary used: NA NA
         subjects affected / exposed
    5 / 12 (41.67%)
    3 / 13 (23.08%)
         occurrences all number
    5
    5
    Allergy
    alternative dictionary used: NA NA
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 13 (7.69%)
         occurrences all number
    1
    2
    Metabolism and nutrition disorders
    Metabolic disorder
    alternative dictionary used: NA NA
         subjects affected / exposed
    2 / 12 (16.67%)
    1 / 13 (7.69%)
         occurrences all number
    3
    1

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    16 Nov 2012
    - Modification des critères d'inclusion - Modification du calendrier de l'étude au niveau des bilans biologiques et /ou examens radiologiques prévus à la visite V1. - Actualisation de la grille de classification des EIG suite à la mise à jour du RCP.
    16 Apr 2013
    Modification d'un critère d'inclusion : les patients présentant une adrénarche associé à un âge osseux égal au moins à l'âge chronologique (et plus uniquement ceux présentant une adrénarche associé à une avance d'âge osseux au moins supérieure à 6 mois sur l'âge chronologique) Ajout de 3 nouveaux centres investigateurs (Lyon, Marseille et Angers) Modification de la composition du Comité Indépendant de Surveillance
    03 Oct 2013
    Prolongation de l'étude de 11 mois jusqu'en janvier 2016

    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
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sun Apr 28 12:47:51 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA