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   43854   clinical trials with a EudraCT protocol, of which   7284   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

    Print Download

    Summary
    EudraCT Number:2019-002385-12
    Sponsor's Protocol Code Number:RC31/15/7825
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-06-04
    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 ConcernedItaly - Italian Medicines Agency
    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)
    Trattamento con ossitocina nei neonati e nei bambini affetti dalla sindrome di Prader-Willi di età compresa tra 0 e 3 mesi: Studio riguardante la sicurezza e l'efficacia sulle capacità orali e sociali e sul comportamento alimentare delle somministrazioni intranasali di ossitocina rispetto al placebo (sperimentazione clinica di fase III) - OTBB3
    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
    Trattamento con Ossitocina in neonati e bambini di età compresa tra 0 e 3 mesi affetti dalla sindrome di 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.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN12345678
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT12345678
    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 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 organisationUniversity Hospital of Toulouse
    B.5.2Functional name of contact pointNadège ALGANS
    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 number61777204
    B.5.5Fax number61778411
    B.5.6E-mailalgans.n@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
    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 INNOSSITOCINA
    D.3.9.1CAS number 50-56-6
    D.3.9.2Current sponsor codeH01BB02
    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
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Malattia di Prader-Willi
    E.1.1.1Medical condition in easily understood language
    Prader-Willi Syndrom
    Malattia di Prader Willy
    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’obiettivo primario dello studio è di dimostrare la superiorità verso placebo del trattamento di OT per via nasale per 4 settimane sulla funzionalità orale valutata mediante la Scala di Valutazione Motoria Orale Neonatale (NOMAS) in lattanti con PWS di età inferiore o pari a 3 mesi al momento dell’inclusione nello studio.
    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.
    Gli obiettivi secondari sono di documentare l’effetto di 1 e 4 settimane di OT intranasale rispetto al placebo su:
    - Problematiche di suzione e deglutizione valutate mediante videofluoroscopia
    - Valutazione del ritiro sociale valutato mediante l’Alarm Distress Baby Scale (ADBB).
    - Stato infantile, impegno sociale e interazioni madre-bambino valutate dai sottoscores Coding Interactive Behaviour (CIB)
    - Valutazione dell’assunzione di cibo mediante punteggio di competenza
    - Livelli di grelina circolante
    - Valutazione dei genitori sull'alimentazione
    - Livelli di OT
    - Documentare la sicurezza di somministrazioni ripetute di 4 o 8 settimane di OT con un follow-up totale di 26 settimane.
    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.
    - Maschi o femmine neonati o lattanti con PWS confermata geneticamente
    - Età inferiore a 92 giorni ( con una tolleranza massima di 8 gg) ( per i lattanti pretermine , nati prima della 37a settimana di gestazione, si applicherà l’età corretta)
    - Sottoscrizione del consenso informato da parte dei genitori o tutore legale
    - Genitori convinti e in grado di aderire a tutte le procedure previste dallo studio.
    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.
    - Neonato/lattante ricoverato terapia intensiva neonatale perché affetto da comorbidità a rischio della vita come gravi distress respiratori, neuropatie e/o cardiopatie.
    - Neonato/lattante con prolungamento del QTc
    - Neonato/lattante senza copertura assicurativa medica
    - Neonato/lattante con ipersensibilità all’ossitocina o eccipienti
    - Neonato/lattante in trattamento con farmci che prolungano l’intervallo QTc
    - Neonato/lattante con ipokaliemia (clinicamente rilevante a discrezione del medico curante)
    - Neonato/lattante che partecipa simultaneamente al un altro studio interventistico
    - Neonato/lattante con genitori non affidabili
    Neonato/lattante i cui genitori rifiutano la videoregistrazione, essenziale per lo scopo primario dello studio.
    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 (= 10) on sucking/swallowing as assessed by the neonatal oral motor rating scale (NOMAS) centrally assessed on video recording, after 4 weeks (V4) of intranasal OT / placebo treatment.
    L'endpoint primario è la proporzione di neonati/lattanti che raggiungono un punteggio =10, cioè un punteggio normale o sub-normale (definito come responder) su suzione/deglutizione, come valutato dalla scala di valutazione orale motoria neonatale (NOMAS) valutata centralmente su registrazione video, dopo 4 settimane (V4) di trattamento intranasale di OT/placebo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    4 weeks
    4 settimane
    E.5.2Secondary end point(s)
    • Proportion of infants with abnormal score of videofluoroscopy at baseline (on at least one of the two items) who reached a normal score after 4 weeks treatment for all of the 2 items, namely i) pharyngeal propulsion ii) airway protection at 4 weeks (V4).
    • The change from baseline of the Alarm Distress Baby Scale (ADBB) score at 4 weeks (V4).
    • The change from baseline of the Coding Interacting Behaviour (CIB) subscores at 4 weeks (V4).
    • The change from baseline of proficiency score which is the volume of milk taken in the first five minutes of feeding at 1 week (V3) and at 4 weeks (V4).
    • The change from baseline of ghrelin (unacylated/UAG and acylated/AG) concentration at 1 week (V3) and at 4 weeks (V4).
    • The change from baseline (V1) of the parent assessment of feeding (Severity) at 4 weeks (V4)
    • The Parent assessment of feeding (Improvement) at 4 weeks (V4)
    • The change from baseline of plasma OT concentration at 4 weeks (V4).
    • Biological safety parameters (natremia, plasmatic osmolality, capillary blood glucose, total bilirubin level, urinary density, kalemia), vital signs, ECG and emergent adverse events in all groups of patients.
    - Proporzione di neonati con punteggio anormale di videofluoroscopia basale (su almeno uno dei due elementi) che hanno raggiunto un punteggio normale dopo 4 settimane di trattamento (V4) per tutti e 2 gli elementi, vale a dire i) propulsione faringea ii) protezione delle vie aeree.
    - La variazione rispetto al basale a 4 settimane (V4) del punteggio della Alarm Distress Baby Scale (ADBB).
    - La modifica rispetto al basale a 1 settimana (V3) e a 4 settimane (V4) dei punteggi di codifica Interacting Behavior (CIB).
    - La variazione rispetto al basale a 4 settimane (V4) del punteggio di competenza, ovvero il volume di latte assunto nei primi cinque minuti di alimentazione.
    - La variazione dal basale della concentrazione di grelina (non acilata / UAG e acilata / AG) a 1 settimana (V3) e a 4 settimane (V4).
    - La variazione dal basale (V1) della valutazione genitoriale dell'alimentazione (Gravità) a 4 settimane (V4)
    - La valutazione del genitore dell'alimentazione (Miglioramento) a 4 settimane (V4)
    - La variazione dal basale della concentrazione plasmatica di OT a 4 settimane (V4).
    - Parametri di sicurezza biologica (natremia, osmolalità plasmatica, glicemia capillare, livello totale di bilirubina, densità urinaria, kalemia), segni vitali, ECG ed eventi avversi emergenti in tutti i gruppi di pazienti.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1 week and 4 week
    1 settimana e 4 settimane
    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
    Disegno misto tra gruppi paralleli e 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    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 Information not present in EudraCT
    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
    LVLS
    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 days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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.1.1.1In 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: 2
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    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 state4
    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
    Nessuno
    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 Decision2021-05-14
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
    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-Tue Apr 23 09:41:08 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA