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    The EU Clinical Trials Register currently displays   43974   clinical trials with a EudraCT protocol, of which   7311   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:2020-006161-11
    Sponsor's Protocol Code Number:RC31/20/0421
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-04-21
    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 number2020-006161-11
    A.3Full title of the trial
    LONG -TERM INTERVENTIONAL FOLLOW-UP STUDY UP TO 4 YEARS OF AGE OF CHILDREN WITH PRADER-WILLI SYNDROME INCLUDED IN THE OTBB3 CLINICAL TRIAL AND COMPARISON WITH AN UNTREATED COHORT OF CHILDREN WITH PRADER-WILLI SYNDROME
    Étude interventionnelle de suivi à long terme jusqu’à l’âge de 4 ans des enfants avec un syndrome de Prader-Willi inclus dans l’étude clinique OTBB3 et comparaison avec une cohorte non traitée d’enfants avec un syndrome de Prader-Willi
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    FOLLOW-UP STUDY UP TO 4 YEARS OF AGE OF CHILDREN WITH PRADER-WILLI SYNDROME INCLUDED IN THE OTBB3 CLINICAL TRIAL
    A.3.2Name or abbreviated title of the trial where available
    OTBB3 Follow-Up
    A.4.1Sponsor's protocol code numberRC31/20/0421
    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 Sponsor Toulouse 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+330534558551
    B.5.5Fax number+330534558558
    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.3.4Pharmaceutical 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
    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 confirm the long term safety profile including the associated comorbidities in all children with PWS who have been treated in the OTBB3 study.
    confirmer le profil de sécurité d’emploi à long terme, notamment les principales comorbidités associées, chez tous les enfants présentant un SPW ayant été traités au cours de l’étude OTBB3.
    E.2.2Secondary objectives of the trial
    • To complete the safety assessment by describing the development of children and severity of the disease in all children included in the OTBB3 study;
    • To compare the long-term safety and efficacy of early OT treatment in children included in OTBB3 with an untreated cohort of French children with PWS. This comparison will be performed on the subset of patients who have been treated in France in the OTBB3 study in a comparable context.
    Compléter l’évaluation de la sécurité d’emploi en décrivant le développement des enfants et la sévérité de la maladie chez tous les enfants inclus dans l’étude OTBB3 ;
    Comparer la sécurité d’emploi et l’efficacité à long terme d’un traitement précoce par l’ocytocine chez les enfants inclus dans l’étude OTBB3, avec une cohorte non traitée d’enfants français présentant un SPW. Cette comparaison sera réalisée sur le sous-ensemble de patients ayant été traités en France dans l’étude OTBB3 dans un contexte comparable.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Male or female child with a genetically confirmed diagnosis of PWS
    The parents (or legal representative) must have signed the consent form;
    Treated cohort: the child participated in the OTBB3 study
    Untreated cohort: the child has never received OT, is aged 30±6 months at inclusion and is followed in France.
    1. Enfant de sexe masculin ou féminin présentant un diagnostic génétiquement confirmé de SPW
    2. Les parents (ou le représentant légal) doivent avoir signé le formulaire de consentement ;
    3. Cohorte traitée : l’enfant a participé à l’étude OTBB3 ;
    4. Cohorte non traitée : l’enfant n’a jamais reçu d’ocytocine, est âgé de 30±6 mois à l’inclusion et est suivi en France.
    E.4Principal exclusion criteria
    1. Administrative problems:
    a. Inability for the parents (or legal representative) to understand/fulfil study requirements;
    b. No coverage by a social security regime;
    2. Refusal of parents (or legal representative) to sign the consent form;

    1. Problèmes administratifs :
    a. Incapacité des parents (ou du représentant légal) à comprendre/respecter les exigences de l’étude ;
    b. Absence de couverture par un régime de sécurité sociale ;
    2. Refus des parents (ou du représentant légal) de signer le formulaire de consentement ;
    E.5 End points
    E.5.1Primary end point(s)
    • The number and percentage of patients with adverse events (AEs) and serious adverse events (SAEs);
    • The occurrence of the following main comorbidities:
    o Digestive disorders
    o Scoliosis;
    o Sleep disorders;
    - Obstructive and central sleep apnoea
    - Narcolepsy;
    - Excessive daytime sleepiness
    o Endocrine disorders
    o Metabolic disorders
    o Other comorbidities assessed by a checklist.
    • The occurrence of medications, surgery and rehabilitations
    • Nombre et pourcentage de patients présentant des événements indésirables (EI) et des événements indésirables graves (EIG) ;
    • Survenue des principales comorbidités suivantes :
    o Troubles digestifs
    o Scoliose ;
    o Troubles du sommeil ;
    - Apnée obstructive et apnée centrale du sommeil
    - Narcolepsie ;
    - Somnolence diurne excessive
    o Troubles endocriniens
    o Troubles métaboliques
    o Autres comorbidités évalués par une liste de contrôle.
    • Prise de médicaments, chirurgie et rééducation
    E.5.1.1Timepoint(s) of evaluation of this end point
    every year at V1, V2 and V3 between 36 to 48 months (42+/-6 months)



    chaque année en V1, V2 et V3 entre 36 et 48 mois (42 +/- 6 mois)
    E.5.2Secondary end point(s)
    • The number and percentage of patients with AEs and SAEs;
    • The occurrence of the following main comorbidities:
    o Digestive disorders
    o Scoliosis;
    o Sleep disorders
    o Endocrine disorders
    o Metabolic disorders
    o Other comorbidities
    • The occurrence of medications, surgery and rehabilitations
    • The severity of the comorbidities
    • Auxology, anthropometry and psychomotor development
    • Severity of the disease in terms of:
    o Miller nutritional phases;
    o Eating behaviour
    o Adaptive behaviour
    o Behavioural and psychiatric disorders
    • Caregiver burden
    • Nombre et pourcentage de patients présentant des EI et des EIG ;
    • Survenue des principales comorbidités suivantes :
    o Troubles digestifs
    o Scoliose ;
    o Troubles du sommeil
    o Troubles endocriniens
    o Troubles métaboliques
    o Autres comorbidités
    • Prise de médicaments, chirurgie et rééducation
    • Sévérité des comorbidités
    • Auxologie, anthropométrie et développement psychomoteur
    • Sévérité de la maladie en utilisant les critères suivants :
    o Phases nutritionnelles de Miller ;
    o Comportement alimentaire
    o Comportement adaptatif
    o Troubles comportementaux et psychiatriques
    • Charge pesant sur les aidants
    E.5.2.1Timepoint(s) of evaluation of this end point
    every year at V1, V2 and V3 between 36 to 48 months (42+/-6 months)
    chaque année en V1, V2 et V3 entre 36 et 48 mois (42 +/- 6 mois)
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    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) 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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3.1Comparator description
    children untreated by oxytocin
    E.8.2.4Number of treatment arms in the trial2
    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 concerned14
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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 years4
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    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: 80
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 48
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 32
    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 Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    young children who can't give their consent
    F.4 Planned number of subjects to be included
    F.4.1In the member state70
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 80
    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
    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 Decision2021-06-23
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-09
    P. End of Trial
    P.End of Trial Status
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