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    Summary
    EudraCT Number:2012-002873-77
    Sponsor's Protocol Code Number:2013.2015
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2012-11-01
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2012-002873-77
    A.3Full title of the trial
    The effect of intranasal insulin on development and behaviour of children with Phelan-McDermid syndrome
    Het effect van intranasale insuline op ontwikkeling en gedrag van kinderen met het Phelan-McDermid syndroom
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The effect of insulin nasal spray on development and behaviour of children with Phelan-McDermid syndrome
    Het effect van insuline neusspray op ontwikkeling en gedrag van kinderen met het Phelan-McDermid syndroom
    A.3.2Name or abbreviated title of the trial where available
    Intranasal insulin in PMS
    Intranasale insuline bij PMS
    A.4.1Sponsor's protocol code number2013.2015
    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 Medical Center Groningen, Department of Genetics
    B.1.3.4CountryNetherlands
    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 supportZonMW
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity Medical Center Groningen, Department of Genetics
    B.5.2Functional name of contact pointUMCG, Department of Genetics
    B.5.3 Address:
    B.5.3.1Street AddressHanzeplein 1
    B.5.3.2Town/ cityGroningen
    B.5.3.3Post code9713 RB
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31503617229
    B.5.5Fax number+31503617231
    B.5.6E-mailc.m.a.van.ravenswaaij@umcg.nl
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameInsulin Intranasal 100IE/ml
    D.3.4Pharmaceutical form Nasal spray, solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntranasal use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNInsulin
    D.3.9.1CAS number 11061-68-0
    D.3.9.3Other descriptive nameRegular human insulin
    D.3.9.4EV Substance CodeSUB34009
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 placeboIntranasal use (Noncurrent)
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Phelan-McDermid syndrome
    Phelan-McDermid syndroom
    E.1.1.1Medical condition in easily understood language
    Phelan-McDermid syndrome
    Phelan-McDermid syndroom
    E.1.1.2Therapeutic area Psychiatry and Psychology [F] - Mental Disorders [F03]
    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 objective of this trial is to validate the hypothesis that intranasal insulin improves development in children with Phelan-McDermid syndrome.
    Het doel van dit onderzoek is het valideren van de hypothese dat intranasale insuline de ontwikkeling verbetert bij kinderen met het Phelan-McDermid syndroom.
    E.2.2Secondary objectives of the trial
    The secondary objective of this trial is to validate the hypothesis that intranasal insulin improves development in children with Phelan-McDermid syndrome.
    Het secundaire doel van dit onderzoek is het valideren van de hypothese dat intranasale insuline de ontwikkeling verbetert bij kinderen met het Phelan-McDermid syndroom.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Age between 12 months and 18 years 0 months old at 1-1-2013
    Proven SHANK3 deletion by array-comparative genomic hybridization (array-CGH)
    Parents need to speak and understand Dutch
    Leeftijd tussen 12 maanden en 18 jaar en 0 maanden op 1-1-2013
    Bewezen SHANK3 deletie met array-comparative genomic hybridization (array-CGH)
    Ouders dienen Nederlands te begrijpen en te spreken
    E.4Principal exclusion criteria
    A contra-indication for the use of intranasal application (e.g. anatomical obstruction)
    Severe perinatal brain damage (e.g. asphyxia, haemorrhage, infection)
    A metabolic or muscle disease responsible for neurological symptoms, independent of the 22q13 deletion
    Een contra-indicatie voor het gebruik van de intransale toedieningsvorm (Bijv. een anatomische obstructie)
    Ernsitge perinatale hersenschade (bijv. asphyxie, bloeding, infectie)
    Een metabole of spieraandoening die verantwoordelijk is voor de neurologische symptomen onafhankelijk van de 22q13 deletion
    E.5 End points
    E.5.1Primary end point(s)
    The primary end point is developmental pace. Development is assessed by the Bayley-III-NL (Dutch version of the Bayley-III, Bayley, 2006) or WPPSI-III-NL (Dutch version of the WPPSI-III, Wechsler, 2009) dependent on the developmental age of the children. Both the Bayley-III and the WPPSI are individually administered tests that provide subtests and composite scores that represent general functioning. Developmental pace is calculated as the difference in developmental age equivalent between two assessments divided by the difference in calendar age in months at the time of these assessments (typically 6 months), resulting in a value for developmental age increase / month.
    De primaire uitkomstmaat is ontwikkelingssnelheid. Ontwikkeling wordt getest met behulp van de Bayley-III-NL (Nederlandse versie van de Bayley-III) of WPPSI-III-NL (Nederlandse versie van de WPPSI-III) afhankelijk van de ontwikkelingsleeftijd van de kinderen. Zowel de Bayley-III als de WPPSI zijn individueel toegepaste testen die subtesten en composiet scores geven welke representatief zijn voor het algemeen functioneren. Ontwikkelingssnelheid wordt berekend als het verschil in ontwikkelingsleeftijd equivalent tussen twee testen gedeeld door het verschil in kalenderleeftijd op het moment van de testen (typisch 6 maanden), resulterend in een waarde voor de toename in ontwikkelingsleeftijd / maand.
    E.5.1.1Timepoint(s) of evaluation of this end point
    6, 12, 18 months after initiation of the clinical trial phase
    6, 12, 18 maanden na het begin van de clinical trial phase
    E.5.2Secondary end point(s)
    The secondary end point is behaviour. Behaviour is assessed by the following questionnaires: Vineland screener, ESSEON, CBCL1,5-5, and Brief-P. To evaluate behaviour in several domains, raw scores are determined.
    De secundaire uitkomstmaat is gedrag. De secundaire uitkomstmaat is gedrag. gedrag wordt getest met behulp van de volgende vragenlijsten: Vineland screener, ESSEON, CBCL1,5-5 en Brief-P. Om het gedrag te evalueren op verschillende gebieden zullen ruwe scores bepaald worden.
    E.5.2.1Timepoint(s) of evaluation of this end point
    6, 12, 18 months after initiation of the clinical trial phase
    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 No
    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 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
    Stepped wedge
    Stepped wedge
    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 trial5
    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 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
    The end of the trial is defined as the last assessment of the last subject undergoing the trial
    Het eind van de trial is gedefinieerd als de laatste test van de laatste deelnemer die deelneemt aan de trial.
    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
    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: 20
    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: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 17
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    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
    Mental disabled children
    Verstandelijk beperkte kinderen
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    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)
    At the end of the trial, medication will be ceased. There will be no standard follow-up, but if indicated parents will receive the results of the trial.
    Aan het eind van de trial zal de medicatie gestaakt worden. Er is geen standaard follow-up, maar als ouders dit hebben aangegeven zullen zij de resultaten van het onderzoek ontvangen.
    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 Decision2012-11-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-01-25
    P. End of Trial
    P.End of Trial StatusOngoing
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