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    The EU Clinical Trials Register currently displays   43839   clinical trials with a EudraCT protocol, of which   7280   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).

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    Summary
    EudraCT Number:2019-002385-12
    Sponsor's Protocol Code Number:RC31/15/7825
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-03-06
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2019-002385-12
    A.3Full title of the trial
    OXYTOCIN TREATMENT IN NEONATES AND INFANTS AGED FROM 0 TO 3 MONTHS WITH PRADER-WILLI SYNDROME: A STUDY OF THE SAFETY AND EFFICACY ON ORAL AND SOCIAL SKILLS AND, FEEDING BEHAVIOR OF INTRANASAL ADMINISTRATIONS OF OXYTOCIN VS. PLACEBO (PHASE III CLINICAL TRIAL)
    Traitement par ocytocine chez les nouveau-nés et les nourrissons âgés de 0 à 3 mois présentant un syndrome de Prader-Willi : étude de la tolérance et de l’efficacité sur les troubles de l’oralité, les interactions sociales et le comportement alimentaire d’administrations intra-nasales d’ocytocine versus placebo (Étude clinique de phase III)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    OXYTOCIN TREATMENT IN NEONATES AND INFANTS AGED FROM 0 TO 3 MONTHS WITH PRADER-WILLI SYNDROME
    Traitement par ocytocine chez les nouveau-nés et les nourrissons âgés de 0 à 3 mois présentant un syndrome de Prader-Willi
    A.3.2Name or abbreviated title of the trial where available
    OTBB3
    OTBB3
    A.4.1Sponsor's protocol code numberRC31/15/7825
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorUniversity Hospital of Toulouse
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportFrench Ministry of Health
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportPaediatric Clinical Research Infrastructure Network
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportOT4B
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportPrader Willi Association
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLuxembourg Institute of Health, Clinical & Epidemiological Investigation Center
    B.5.2Functional name of contact pointLucile Pernot
    B.5.3 Address:
    B.5.3.1Street Address1A, rue Thomas EDISON
    B.5.3.2Town/ cityStrassen
    B.5.3.3Post codeL-1445
    B.5.3.4CountryLuxembourg
    B.5.4Telephone number+35226970 811
    B.5.5Fax number+35226970 717
    B.5.6E-mailecrin@lih.lu
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/141302
    D.3 Description of the IMP
    D.3.1Product nameOxytocin
    D.3.4Pharmaceutical form Nasal spray, solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPNasal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNOXYTOCIN
    D.3.9.1CAS number 50-56-6
    D.3.9.3Other descriptive nameOXYTOCIN
    D.3.9.4EV Substance CodeSUB09580MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number44,44
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboNasal spray
    D.8.4Route of administration of the placeboNasal use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Prader-Willi Syndrom
    Syndrome de Prader-Willi
    E.1.1.1Medical condition in easily understood language
    Prader-Willi Syndrom
    Syndrome de Prader-Willi
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to demonstrate the superiority versus placebo of a 4 weeks intranasal OT administration on oral skills assessed by the Neonatal Oral-Motor Assessment Scale (NOMAS) in infants with Prader Willi Syndrom (PWS) aged less than or equal to 3 months at inclusion
    L'objectif principal est de démontrer la supériorité d'une administration intranasale d'ocytocine pendant 4 semaines, versus placebo, sur les compétences orales évaluées par l'échelle NOMAS (Neonatal Oral-Motor Assessment Scale) sur des enfants avec un Syndrome de Prader Willi âgés de 0 à 3 mois à l'inclusions.
    E.2.2Secondary objectives of the trial
    The secondary objectives are to document the effects of 1 week and 4 weeks intranasal OT administration versus Placebo on:
    - Sucking/swallowing troubles
    - Social Withdrawal score ADBB
    - CIB subscores
    - Food intake (medical assesment and parental assesment)
    - Circulating forms of ghrelin
    - OT levels

    To document the safety of repeated OT administration for 4 weeks or 8 weeks with total follow-up over 26 weeks.



    Les objectifs secondaires sont de documenter les effets de l'administration intranasale d'ocytocine versus placebo, pendant une semaine et 4 semaines de traitements sur :
    - les problèmes de succion/ingestion
    - ADBB,
    - Sous-scores CIB
    - Apport alimentaire évalué par le médecin et les parents
    - Formes circulantes de ghréline
    - Niveaux d’OT

    Documenter la sécurité de l’administration répétée de l’OT pendant 4 semaines ou 8 semaines avec un suivi total de plus de 26 semaines.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male or female neonate or infant, with PWS genetically confirmed
    - Age <92 days (plus a tolerance of up to 8 days maximum) (for preterm infants, born before 37 weeks amenorrhea, corrected age will be applied)
    - Signed informed consent obtained from the parents/holders of parental authority
    - Parents willing and able to comply with all study procedures.
    -Nouveau-né/Nourrisson Fille ou Garçon, avec PWS génétiquement confirmée
    -Age < 92 jours (avec une tolérance jusqu’à 8 jours maximum supplémentaire) (pour les nourrissons prématurés, nés avant 37 semaines d’aménorrhée, l’âge corrigé sera appliqué)
    -Signature du consentement éclairé obtenu auprès des parents/détenteurs de l’autorité parentale
    -Parents désireux et capables de se conformer à toutes les procédures d’étude.
    E.4Principal exclusion criteria
    - Neonate or infant admitted to the emergency care unit for ongoing life-threatening comorbidities like severe respiratory, cardiovascular or neurological abnormalities
    - Neonate or infant with prolongation of the QT interval
    - Neonate or infant with hypokaliema
    - Neonate or infant without medical insurance
    - Neonates or infants whose parents’ situations may jeopardize the interpretation of the results
    - Neonate or infant with known hypersensitivity to the excipients of the product
    - Neonate or infant participating simultaneously in another interventional study.
    -Nouveau-né/Nourrisson admis à l’unité de soins d’urgence pour les comorbidités en cours de vie, comme des anomalies respiratoires graves, cardiovasculaires ou neurologiques
    -Nouveau-né/Nourrisson avec prolongation de l’intervalle QT
    -Nouveau-né/Nourrisson présentant une hypokaliémie
    -Nouveau-né/Nourrisson sans assurance médicale
    -Nouveau-né/Nourrisson présentant une hypersensibilité connue aux excipients du produit
    -Nouveau-né/Nourrisson participant simultanément à une autre étude interventionnelle.
    -Nouveau-né dans une situation compromettant l’analyse des résultats.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the proportion of neonates/infants who achieve a quasi-normal score on sucking/swallowing after 4 weeks (V4) OT/ Placebo intranasal treatment.
    Le critère d’évaluation principal est la proportion de nouveau-nés/nourrissons qui atteignent un score quasi-normal (≤ 10) (défini comme répondeurs) sur la succion/la déglutition, tel qu’évalué par l’échelle de calcul du taux oral-moteur néonatal (NOMAS), marqué au centre sur les vidéos, après 4 semaines (v4) de traitement intranasal de placebo ou d'ocytocine
    E.5.1.1Timepoint(s) of evaluation of this end point
    4 weeks
    4ème semaine
    E.5.2Secondary end point(s)
    - Change from baseline global score of swallowing at 4 weeks.
    - Proportion of infants with abnormal score at baseline who reached a normal score after 4 weeks treatment for all of the 3 items, namely i) velopharyngeal continence ii) pharyngeal propulsion iii) swallowing troubles at 4 weeks.
    - The change from baseline of the Alarm Distress Baby Scale (ADBB) score at 4 weeks.
    - The change from baseline of the Coding Interacting Behaviour (CIB) subscores at 4 weeks.
    - The change from baseline of volume of milk taken in the first five minutes of feeding at 1 week (V3) and at 4 weeks.
    - The change from baseline of ghrelin (unacylated/UAG and acylated/AG) concentration at 1 week (V3) and at 4 weeks.
    - The change from baseline of blood OT concentration at 4 weeks.
    - Biological safety parameters vital signs, ECG and emergent adverse events at all study time points in all groups of patients.

    -Changement de la note globale de base de la déglutition à 4 semaines.
    -Proportion de nourrissons ayant un score anormal à l’inclusion qui ont atteint un score normal après 4 semaines de traitement pour tous les 3 éléments, à savoir i) l’incontinence du développement du développement II) la propulsion pharyngée III) les troubles de la déglutition à 4 semaines.
    -Le changement de la ligne de base de l’alarme de détresse Baby Scale (ADBB) Score à 4 semaines.
    -Le changement par rapport au niveau de référence du comportement en interaction de codage (CIB) subsiste à 4 semaines.
    -Le changement de la valeur de base du score de compétence (PRO) qui est le volume de lait pris dans les cinq premières minutes de l’alimentation à 1 semaine et à 4 semaines.
    -Le changement de la concentration de ghréline (unacylated/UAG et acylated/AG) à une semaine et à 4 semaines.
    -Le changement de la base de la concentration de sang OT à 4 semaines.
    -Paramètres de sécurité biologique, signes vitaux, ECG et événements indésirables émergents à tous les points de temps d’étude dans tous les groupes de patients.


    E.5.2.1Timepoint(s) of evaluation of this end point
    1 week and 4 week
    1ère semaine et 4ème semaine de traitement
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    mélange entre une méthodologie en groupes parallèles et en cross over
    Parallel group and cross over mixed
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    Dernière visite du dernier patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 48
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 48
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 48
    F.4.2.2In the whole clinical trial 48
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    Aucun
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Paediatric Clinical Research Infrastructure Network
    G.4.3.4Network Country France
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-03-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-03-14
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