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    Summary
    EudraCT Number:2021-000094-91
    Sponsor's Protocol Code Number:RC31/20/0518
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-04-01
    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 ConcernedFrance - ANSM
    A.2EudraCT number2021-000094-91
    A.3Full title of the trial
    EFFECT OF INTRANASAL OXYTOCIN ON DYSPHAGIA RELATED TO OROPHARYNGO-OESOPHAGEAL DYSMOTILITY TRANSIT IN CHILDREN AND ADOLESCENTS WITH PRADER-WILLI SYNDROME: A PHASE 3 STUDY (DYSMOT)
    EFFETS DE L’ADMINISTRATION INTRANASALE D’OCYTOCINE SUR LA DYSPHAGIE LIÉE À UN TROUBLE DE LA MOTILITE OROPHARYNGO-ŒSOPHAGIENNE CHEZ DES ENFANTS ET DES ADOLESCENTS PORTEURS D’UN SYNDROME DE PRADER-WILLI : ÉTUDE DE PHASE 3 (DYSMOT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    EFFECT OF INTRANASAL OXYTOCIN ON DYSPHAGIA IN CHILDREN AND ADOLESCENTS WITH PRADER WILLI SYNDROME
    EFFETS DE L’ADMINISTRATION INTRANASALE D’OCYTOCINE SUR LA DYSPHAGIE CHEZ LES ENFANTS ET ADOLESCENTS ATTEINTS D’UN SYNDROME DE PRADER-WILLI
    A.3.2Name or abbreviated title of the trial where available
    DYSMOT
    A.4.1Sponsor's protocol code numberRC31/20/0518
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorToulouse University Hospital
    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 supportOT4B
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCentre de référence Prader-Willi
    B.5.2Functional name of contact pointTAUBER
    B.5.3 Address:
    B.5.3.1Street Address330 avenue de Grande-Bretagne
    B.5.3.2Town/ cityToulouse
    B.5.3.3Post code31059
    B.5.3.4CountryFrance
    B.5.4Telephone number+330534 55 85 51
    B.5.5Fax number+330534 55 85 58
    B.5.6E-mailtauber.mt@chu-toulouse.fr
    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.3.2Product code H01BB02
    D.3.4Pharmaceutical form Nasal spray, solution
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPNasal use
    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 Syndrome
    Syndrome de Prader-Willi
    E.1.1.1Medical condition in easily understood language
    Prader-Willi Syndrome
    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
    To compare the effect of a 12-week intranasal administration of OT versus (vs) placebo on dysphagia related to OPOD in children and adolescents with PWS.
    Comparer l’effet de l’administration intranasale d’ocytocine pendant 12 semaines versus placebo sur la dysphagie liée à un trouble de la motilité oropharyngo-œsophagienne chez des enfants et des adolescents présentant un SPW.
    E.2.2Secondary objectives of the trial
    To compare the effect of a 12-week intranasal administration of OT vs placebo on:
    • Swallowing symptoms;
    • Gastrointestinal disorders;
    • Feeding events
    • Behavioural problems such as:
    o Temper outbursts;
    o Skin picking;
    • Patient global improvement.
    To document the effect of 12-week OT treatment withdrawal on dysphagia related to OPOD and other efficacy parameters.
    To document the maintenance of efficacy over a 24 week-intranasal administration of OT.
    To document the long-term efficacy up to 48-weeks of intranasal administration of OT.
    Comparer l’effet de l’administration intranasale d’ocytocine pendant 12 semaines à un placebo selon les critères suivants :
    • Troubles de la déglutition ;
    • Troubles digestifs ;
    • Evènements particuliers au cours des repas et notamment l’étouffement ;
    • Troubles comportementaux, tels que:
    o Accès de colère ;
    o Grattage compulsif de la peau ;
    • Amélioration globale du patient.
    Documenter l’effet de l’arrêt du traitement par ocytocine sur la dysphagie reliée à l’OPOD et sur les autres paramètres d’efficacité.
    Documenter le maintien d’efficacité de l’administration intranasale d’ocytocine pendant 24 semaines.
    Documenter l’efficacité à long terme (jusqu’à 48 semaines) de l’administration intranasale d’ocytocine
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patients aged between 2 and 17,5 years.
    2. Genetically confirmed diagnosis of PWS.
    3. Parents (or legal representative) have signed the informed consent form and are willing to comply with all study procedures.
    1. Patients masculins et féminins âgés de 2 à 17ans et demi.
    2. Diagnostic de SPW confirmé génétiquement.
    3. Les parents (ou le représentant légal) ont signé le formulaire de consentement éclairé et acceptent de se conformer à toutes les procédures de l’étude.
    E.4Principal exclusion criteria
    1. A history of hypersensitivity to the study drug or drugs with similar chemical structures, to excipients of the product, or to latex;
    2. Intolerance of intranasal administrations (including when due to a major behavioural problem);
    3. Hyponatremia (clinically relevant at the discretion of the investigator);
    4. Hypokalaemia (clinically relevant at the discretion of the investigator);
    5. Prolongation of the QT interval and/or family history of prolongation of the QT interval;
    6. Concomitant treatment prolonging the QT interval;
    7. History of abnormal electrocardiogram (ECG) (validated by a cardiologist);
    8. Pregnant girls; (for girls aged 14 years of above who do not have contraception and are sexually active, a negative pregnancy test will be required)
    9. Patient with clinical signs in the context of contact with SARs-COV2 infected person
    10. Patient included in another study protocol on a medicinal product within the last 6 months
    11. Administrative problems:
    1. Antécédents d’hypersensibilité au médicament à l’étude ou à des médicaments avec des structures chimiques similaires, aux excipients du produit, ou au latex ;
    2. Intolérance aux administrations intranasales (y compris celles dues à un problème comportemental majeur) ;
    3. Hyponatrémie ;
    4. Hypokaliémie ;
    5. Allongement de l’intervalle QT et/ou antécédents familiaux d’allongement de l’intervalle QT ;
    6. Traitements concomitants allongeant l’intervalle QT ;
    7. Antécédents d’électrocardiogramme (ECG) anormal (validé par un cardiologue) ;
    8. Femmes enceintes (pour les filles âgées de 14 ans et plus sans contraception et sexuellement actives, a test de grossesse negatf sera requis)
    8. Patient inclus dans un autre protocole d’étude portant sur un médicament au cours des 6 derniers mois ;
    9. Problèmes administratifs
    E.5 End points
    E.5.1Primary end point(s)
    The percentage of patients showing normalisation of at least 1 of the following abnormal OPOD subscores after 12 weeks OT / placebo at V2 (groups 1 and 3 vs group 2):
    • Oral and pharyngeal propulsion;
    • Initiation and synchronisation of swallowing;
    • Oesophageal motility.
    Pourcentage de patients présentant une normalisation d’au moins un des 3 sous-scores relatifs à l’OPOD anormaux après 12 semaines de traitement par ocytocine vs placebo lors de la V2 (groupes 1 & 3 versus group 2) :
    • Propulsion buccale et pharyngée ;
    • Initiation et synchronisation de la déglutition ;
    • Motilité œsophagienne.
    E.5.1.1Timepoint(s) of evaluation of this end point
    visit 2 and (visit 3 for complementary analysis)
    visite 2 et (visite 3 pour analyse complémentaire)
    E.5.2Secondary end point(s)
    Secondary Endpoints Secondary Efficacy Endpoints:
    The following parameters will be assessed for differences between OT and placebo at V2 or change from baseline (groups 1 and 3 vs group 2):
    • Swallowing symptoms
    • Digestive questionnaire
    • Occurrence of feeding events –
    • Bristol stool scale
    • Temper outbursts questionnaire
    • Percentage of patients with improvement in CGI score
    •Percentage of patients with improvement in parent/caregiver’s global impression score (overall and by level of improvement);
    • Percentage of patients with improvement in CGI specific for skin picking (overall and by level of improvement).

    The percentage of normalised OPOD subscores at V2 which became abnormal at V3, in Group 1. All above parameters presented, as percentage of patients at V3 and as change from baseline to V3 vs change from V2 to V3 in Group 1 (withdrawal effect).

    The percentage of normalised OPOD subscores at V2 which remains
    normal at V3, in Group 3; all above parameters presented, as percentage of patients and as change from baseline to V3 vs change from V2 to V3 in Group 3. (Maintenance of efficacy)

    All parameters presented, as percentage of patients and as change from baseline to V4 in all groups (48 weeks for group 3, 36 weeks for group 2 and 24 weeks for group 1) (long-term efficacy).

    Other Endpoints:
    Plasma OT at V1, V2 and V3.
    Acylated ghrelin (AG), unacylated ghrelin (UAG), AG/UAG at V1, V2 and V3.
    Safety Endpoints:
    • Adverse events;
    • Laboratory safety parameters at V1, V2, V3 and V4;
    • Vital signs (SBP, DBP and HR) at V1, V2, V3 and V4;
    • ECG at V1, V2, and V3.
    Critères d’efficacité secondaires :
    Les paramètres suivants seront évalués afin de déterminer les différences entre l’ocytocine et le placebo lors de la V2 ou changement par rapport aux valeurs initiales (groupes 1&3 vs groupe 2) :
    • Troubles de la déglutition
    • Questionnaire digestif
    • Evènements particuliers au cours des repas
    • Échelle des selles de Bristol
    • Questionnaire sur les accès de colère
    • CGI
    • score des impressions globales
    • CGI spécifiques du grattage

    Pourcentage des sous-scores, relatifs à l’OPOD, lors de la V2 devenant anormaux lors de la V3 dans le groupe 1. Tous les paramètres ci-dessus présentés en pourcentage de patients lors de la V3 et en changement par rapport aux valeurs initiales lors de la V3 versus changement entre la V2 et la V3 dans le groupe 1 (effet de l’interruption du traitement).




    Tous les paramètres ci-dessus présentés en pourcentage de patients et en changement par rapport aux valeurs initiales lors de la V4 dans tous les groupes (48 semaines pour le groupe 3, 36 semaines pour le groupe 2 et 24 semaines pour le groupe 1) (efficacité à long terme).

    Autres critères d’évaluation :
    Taux d’ocytocine plasmatique lors des visites V1, V2 et V3.
    Ghréline acétylée (AG), ghréline non acétylée (UAG), AG/UAG lors des visites V1, V2 et V3.

    Critères de sécurité d’emploi :
    • Événements indésirables ;
    • Paramètres biologiques de sécurité d’emploi lors des visites V1, V2, V3 et V4 ;
    • Signes vitaux (PAS, PAD et FC) lors des visites V1, V2, V3 et V4 ;
    • ECG lors des visites V1, V2 et V3.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Visit 2, Visit 3 and Visit 4
    Visite 2 Visite 3 et Visite 4
    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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    a first period with double blind versus placebo and a second period open label for all patients
    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 trial3
    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 No
    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 No
    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 months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days0
    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: 36
    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) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 18
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 18
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Most patients SPW who will become adults during the trial won't be able to give their consent personnally
    La plupart des patients SPW qui deviendront adultes durant l'étude ne pourront pas donner leur consentement eux-mêmes.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state36
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 36
    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)
    There will be one-month clinical trial exclusion period for the patients enrolled in this study.
    Un mois de periode d'exclusion à l'issue de la dernière visite.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-01-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-27
    P. End of Trial
    P.End of Trial StatusCompleted
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