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    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   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:2022-000254-28
    Sponsor's Protocol Code Number:RC31/19/0500
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-01-21
    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 number2022-000254-28
    A.3Full title of the trial
    Interest of oxytocin as an adjuvant treatment of psycho-educational measures in challenging behaviors in children with autism spectrum disorders and moderate to severe intellectual disability: feasibility and safety study
    Intérêt de l’ocytocine comme traitement adjuvant des mesures psycho-éducatives dans les comportements-défis chez les enfants avec troubles du spectre de l’autisme et handicap intellectuel moyen à profond : l’étude de faisabilité et de sécurité
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Interest of oxytocin as an adjuvant treatment of psycho-educational measures in challenging behaviors in children with autism spectrum disorders and moderate to severe intellectual disability: feasibility and safety study
    ntérêt de l’ocytocine comme traitement adjuvant des mesures psycho-éducatives dans les comportements-défis chez les enfants avec troubles du spectre de l’autisme et handicap intellectuel moyen à profond : l’étude de faisabilité et de sécurité
    A.3.2Name or abbreviated title of the trial where available
    OT-DEFI
    OT-DEFI
    A.4.1Sponsor's protocol code numberRC31/19/0500
    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.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity Hospital of Toulouse
    B.5.2Functional name of contact pointFlorine LEGAY
    B.5.3 Address:
    B.5.3.1Street Address2 rue Viguerie
    B.5.3.2Town/ cityToulouse
    B.5.3.3Post code31059
    B.5.3.4CountryFrance
    B.5.4Telephone number+330561778204
    B.5.5Fax number+330561778411
    B.5.6E-maillegay.f@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 Yes
    D.2.1.1.1Trade name Syntocinon
    D.2.1.1.2Name of the Marketing Authorisation holderMylan phrma
    D.2.1.2Country which granted the Marketing AuthorisationSwitzerland
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSyntocinon (Oxytocin)
    D.3.4Pharmaceutical form Nasal spray, solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPNasal use
    D.3.11 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, solution
    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
    autism
    autisme
    E.1.1.1Medical condition in easily understood language
    autism
    autisme
    E.1.1.2Therapeutic area Psychiatry and Psychology [F] - Behaviours [F01]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10003805
    E.1.2Term Autism
    E.1.2System Organ Class 100000004873
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The main objective of this study is to assess the feasibility of a protocol for administering OT intranasally as an adjuvant drug treatment to standard psycho-educational interventions in children aged 6 to 12 years with ASD of intensity severe and moderate to profound HI and presenting DC, at three levels:
    1. The feasibility of setting up the pre-therapeutic and monitoring protocol for OT treatment: regular pediatric examination, taking blood samples, carrying out an electrocardiogram;
    2. The feasibility of administering a daily OT intranasal spray treatment at the point of care and at home;
    3. The safety profile of the medicinal product in this specific population at the two doses (4 IU then 8 IU).
    L’objectif principal de cette étude vise à évaluer la faisabilité d’un protocole d’administration d’OT par voie intra-nasale comme traitement médicamenteux adjuvant des interventions psycho-éducatives standards chez des enfants de 6 à 12 ans avec TSA d’intensité sévère et HI moyen à profond et présentant des CD, à trois niveaux :
    1. La faisabilité de la mise en place du protocole pré-thérapeutique et de surveillance du traitement par OT : examen pédiatrique régulier, réalisation des prises de sang, réalisation d’un électrocardiogramme ;
    2. La faisabilité de l’administration d’un traitement journalier d’OT en spray intranasal sur le lieu de soin et à domicile ;
    3. Le profil de sécurité du médicament dans cette population spécifique aux deux doses (4 UI puis 8 UI).
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study include the evaluation of secondary feasibility criteria, the description of the population on certain genetic and hormonal parameters, the evaluation of the safety profile of the OT treatment as well as the description of the efficacy of the treatment. on a certain number of parameters, at the end of each of the administration periods.
    Les objectifs secondaires de l’étude incluent l’évaluation de critères de faisabilité secondaires, la description de la population sur certains paramètres génétiques et hormonaux, l’évaluation du profil de sécurité du traitement par OT ainsi que la description de l'efficacité du traitement sur un certain nombre de paramètres, à la fin de chacune des périodes d'administration.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Minor affiliated or beneficiary of a social security scheme.
    • Age: 6 – 12 years old (> 5 years old and < 13 years old)
    • Diagnosis: ASD according to the DSM-5 of severe intensity; validated by at least one of the two reference diagnostic tools (ADOS or ADI-R)
    • Comorbidities:
    -HI medium to severe
    -Presence of CD objectified using the ABC and ABC-I scales
    • Psycho-educational and re-educational care within one of the 4 investigation centers
    • If psychotropic treatments prescribed, the dosages must be stable for 3 months*
    • One of the two parents at least speaking French and able to understand in particular the instructions for administering the product
    • Informed consent signed by the holders of parental authority
    • Mineur affilié ou bénéficiaire d’un régime de sécurité sociale.
    • Âge : 6 – 12 ans (> 5 ans et < 13 ans)
    • Diagnostic : TSA selon le DSM-5 d’intensité sévère ; validé par au moins un des deux outils diagnostics de référence (ADOS ou ADI-R)
    • Comorbidités :
    -HI moyen à sévère
    -Présence de CD objectivée au moyen des échelles ABC et ABC-I
    • Prises en charge psycho-éducatives et rééducatives au sein de l’un des 4 centres investigateurs
    • Si traitements psychotropes prescrits, les posologies doivent être stables depuis 3 mois*
    • Un des deux parents au moins parlant français et capable de comprendre notamment les consignes d’administration du produit
    • Consentement éclairé signé par les titulaires de l’autorité parentale
    E.4Principal exclusion criteria
    • The refusal of holders of parental authority
    • The absence of a recent general pediatric examination
    • The absence of a basic paraclinical assessment
    • The lack of balance of any other treatments taken by the child (stable dosages for at least 3 months (12 weeks)
    • Pregnant girls, determined by a positive baseline blood pregnancy test
    • Criteria respecting the Syntocinon SPC:
    - Hypersensitivity to Syntocinon
    - Hyponatremia
    - Hypokalemia
    hypertension or hypotension
    • Behavioral intolerance to the intranasal route
    • Hepatic impairment (ALAT>ASAT > 3N)
    • Kidney failure (creatinine > 3 N)
    • History of abnormal ECG (validated by a cardiologist)
    • Type 1 or 2 diabetes
    • Prolongation of the QT interval and/or family history of QT prolongation
    • Le refus des titulaires de l’autorité parentale
    • L’absence d’examen pédiatrique général récent
    • L’absence de bilan paraclinique de base
    • L’absence d’équilibre des éventuels autres traitements pris par l’enfant (posologies stables depuis au moins 3 mois (12 semaines)
    • Filles enceintes, déterminé par un test de grossesse sanguin positif à l’inclusion
    • Critères respectant le RCP du Syntocinon :
    - Hypersensibilité au Syntocinon
    - Hyponatrémie
    - Hypokaliémie
    HTA ou hypotension
    • Intolérance comportementale à la voie intranasale
    • Insuffisance hépatique (ALAT>ASAT > 3N)
    • Insuffisance rénale (créatinine > 3 N)
    • Antécédent d’ECG anormal (validé par un cardiologue)
    • Diabète de type 1 ou 2
    • Allongement de l’intervalle QT et/ou antécédent familial d’allongement du QT
    E.5 End points
    E.5.1Primary end point(s)
    The results will be considered positive if this percentage is greater than or equal to 80%, with a lower limit of the confidence interval of this estimate beyond 61.4%.
    Les résultats seront considérés comme positifs si ce pourcentage est supérieur ou égal à 80%, avec une borne inférieure de l’intervalle de confiance de cette estimation au-delà de 61,4%.
    E.5.1.1Timepoint(s) of evaluation of this end point
    14 Weeks
    14 semaines
    E.5.2Secondary end point(s)
    - Precise statement of the reasons for a possible interruption of the protocol;

    - Qualitative assessment by caregivers and parents of the heaviness of implementing the protocol through semi-directive qualitative and quantitative interviews

    -ii. Constitution of a biological collection for the determination of ghrelin, acetylated ghrelin and circulating OT before and after treatment. These assays will be carried out later.

    - Results of the questionnaires
    - Relevé précis des raisons d’une éventuelle interruption du protocole ;

    - Évaluation qualitative par les soignants et les parents de la lourdeur de la mise en place du protocole par des entretiens qualitatifs et quantitatifs semi-directifs

    - Constitution d’une collection biologique en vue du dosage de la ghréline, de la ghréline acétylé et de l’OT circulante avant et après traitement. Ces dosages seront réalisés ultérieurement.

    - Résultats des questionnaires
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 18
    semaine 18
    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 No
    E.6.6Pharmacokinetic No
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned4
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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: 30
    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: 30
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 30
    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
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-05-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-03-30
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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