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    Summary
    EudraCT Number:2019-002385-12
    Sponsor's Protocol Code Number:RC31/15/7825
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    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 ConcernedGermany - BfArM
    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)
    OXYTOCIN-BEHANDLUNG VON NEUGEBORENEN UND SÄUGLINGEN MIT PRADER-WILLI-SYNDROM IM ALTER VON 0 BIS 3 MONATEN: EINE STUDIE ZUR SICHERHEIT UND WIRKSAMKEIT VON INTRANASALEN GABEN VON OXYTOCIN IM VERGLEICH ZU PLACEBO AUF DIE ORALEN UND SOZIALEN FÄHIGKEITEN UND DAS FÜTTERUNGSVERHALTEN (KLINISCHE PRÜFUNG DER 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
    Behandlung mit Oxytocin bei Neugeborenen und Säuglingen im Alter von 0-3 Monaten mit Prader-Willi-Syndrom
    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 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 number+330561777204
    B.5.5Fax number+330561778411
    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.3.4Pharmaceutical 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 Syndrome
    Prader-Willi Syndrom
    E.1.1.1Medical condition in easily understood language
    Prader-Willi Syndrome
    Prader-Willi Syndrom
    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 Syndrome (PWS) aged less than or equal to 3 months at inclusion
    Das primäre Ziel ist der Nachweis der Überlegenheit einer 4-wöchigen intranasalen OT-Gabe im Vergleich zum Placebo in Bezug auf orale Fähigkeiten, bewertet anhand der Neonatal Oral-Motor Assessment Scale (NOMAS), bei Säuglingen mit PWS im Alter von höchstens 3 Monaten bei Aufnahme.
    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.
    Die sekundären Ziele bestehen darin, die Auswirkungen der 1-wöchigen und 4-wöchigen intranasalen OT-Gabe verglichen mit dem Placebo auf folgende Faktoren zu dokumentieren:
    - Saug-/Schluckschwierigkeiten, bewertet anhand von Videofluoroskopie
    - sozialer Rückzug, bewertet anhand der Alarm Distress Baby Scale (ADBB)
    - Verfassung des Kindes, soziale Teilhabe und Mutter-Kind-Interaktionen, bewertet anhand von Coding Interactive Behaviour (CIB)-Subscores
    - Nahrungsaufnahme, bewertet anhand des Proficiency-Scores
    - Werte zirkulierender Formen von Ghrelin
    - Bewertung der Fütterung durch die Eltern
    - OT-Werte
    - Dokumentation der Sicherheit der wiederholten OT-Gabe über 4 Wochen oder 8 Wochen mit einer Nachbeobachtung von insgesamt 26 Wochen
    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.
    - männliche oder weibliche Neugeborene oder Säuglinge mit genetisch bestätigtem PWS
    - Alter < 92 Tage (zuzüglich einer Toleranz von höchstens 8 Tagen) (bei Frühgeborenen, die vor 37 Wochen nach Amenorrhoe geboren werden, wird das korrigierte Alter verwendet)
    - Einholung der unterschriebenen Einwilligungserklärung von den Eltern/Erziehungsberechtigten
    - Fähigkeit und Bereitschaft der Eltern zur Einhaltung aller Studienverfahren
    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.
    - derzeitige Aufnahme des Neugeborenen oder Säuglings auf die Notfallstation aufgrund von anhaltenden Komorbiditäten wie schweren respiratorischen, kardiovaskulären oder neurologischen Anomalien
    - Neugeborene oder Säuglinge mit verlängertem QT-Intervall
    - Neugeborene oder Säuglinge ohne Krankenversicherung
    - Neugeborene oder Säuglinge mit Überempfindlichkeit gegen Oxytocin oder sonstige Bestandteile des Präparats
    - Neugeborene oder Säuglinge mit Begleitbehandlung, die das QT-Intervall verlängert (siehe Anhang 16)
    - Neugeborene oder Säuglinge mit genetischer Pathologie in der Familienanamnese, die eine Verlängerung des QT-Intervalls bewirkt
    - Neugeborene oder Säuglinge mit Hypokaliämie (klinisch relevant nach Ermessen Ihres Arztes)
    - Neugeborene oder Säuglinge, die gleichzeitig an einer anderen interventionellen Studie teilnehmen
    - Neugeborene oder Säuglinge, bei denen die Situation der Eltern die Interpretation der Ergebnisse gefährden kann
    - Neugeborene oder Säuglinge, deren Eltern Videoaufnahmen ablehnen, die im Hinblick auf das primäre Ziel der Studie erforderlich sind
    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.
    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 PWS aged less than or equal to 3 months at inclusion.
    Der primäre Endpunkt ist der Anteil der Neugeborenen/Säuglinge, die nach 4 Wochen (V4) intranasaler OT-/Placebo-Behandlung einen Score von ≤ 10, d. h. einen normalen oder subnormalen Score (definiert als Responder) beim Saugen/Schlucken erzielen, bewertet anhand der Neonatal Oral-Motor Assessment Scale (NOMAS) durch zentrale Auswertung auf Videos.
    E.5.1.1Timepoint(s) of evaluation of this end point
    4 weeks
    4 Wochen
    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.
    • Anteil der Säuglinge mit abnormem Videofluoroskopie-Score bei Baseline (für mindestens eines der zwei Items), die nach 4 Behandlungswochen einen normalen Score für alle 2 Items erreichten, und zwar i) pharyngeale Propulsion ii) Schutz der Atemwege nach 4 Wochen (V4).
    • Veränderung des Alarm Distress Baby Scale (ADBB)-Scores gegenüber Baseline nach 4 Wochen (V4)
    • Veränderung des Coding Interacting Behaviour (CIB)-Subscores gegenüber Baseline nach 4 Wochen (V4)
    • Veränderung des Proficiency-Scores, der dem in den ersten fünf Minuten der Fütterung aufgenommenen Milchvolumen entspricht, gegenüber Baseline nach 1 Woche (V3) und 4 Wochen (V4)
    • Veränderung der Konzentration von Ghrelin (nicht acyliert [UAG] und acyliert [AG]) gegenüber Baseline nach 1 Woche (V3) und 4 Wochen (V4)
    • Veränderung der Beurteilung der Fütterung durch die Eltern (Schwere) gegenüber Baseline (V1) nach 4 Wochen (V4)
    • Die Beurteilung der Fütterung durch die Eltern (Verbesserung) nach 4 Wochen (V4)
    • Veränderung der OT-Konzentration im Plasma gegenüber Baseline nach 4 Wochen (V4)
    • Biologische Sicherheitsparameter (Natriämie, plasmatische Osmolalität, kapillarer Blutzuckerspiegel, Gesamtbilirubinspiegel, Harndichte, Kaliämie), Vitalzeichen, EKG und auftretende unerwünschte Ereignisse in allen Patientengruppen.
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 1 and week 4 of treatment
    1. Woche und 4. Woche der Behandlung
    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
    Mischung aus Parallelgruppen- und Cross-Over-Methodik
    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.8.5The 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
    Letzte Visite des letzten Patienten
    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) Yes
    F.1.1.2.1Number of subjects for this age range: 48
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 48
    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 Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects are neonates or infants, signed informed consent will be obtained from the parents/holders of parental authority
    Die Prüfungsteilnehmer sind Neugeborene oder Kleinkinder, eine unterschriebene Einwilligungserklärung der Eltern/Erziehungsberechtigten wird eingeholt.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    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
    keine
    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 Decision2021-07-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-24
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-03-14
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