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    Summary
    EudraCT Number:2012-000432-26
    Sponsor's Protocol Code Number:versie1,dd01-02-2012
    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:2012-03-27
    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 number2012-000432-26
    A.3Full title of the trial
    Prospective study on the metabolic and linear growth effects of growh hormone treatment in children with Kabuki Syndrome
    Prospectieve studie naar de metabole en lineaire groei-effecten van groeihormoon behandeling bij kinderen met Kabuki syndroom
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Prospective study on the metabolic and linear growth effects of growh hormone treatment in children with Kabuki Syndrome
    Verkennende studie naar de stofwisseling en groei-effecten van groeihormoon behandeling bij kinderen met Kabuki syndroom
    A.3.2Name or abbreviated title of the trial where available
    Metabolic effects of Growth Hormone
    Metabole effecten van groeihormoon
    A.4.1Sponsor's protocol code numberversie1,dd01-02-2012
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorMaastricht University Medical Centre
    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 supportPfizer
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMaastricht University Medical Centre
    B.5.2Functional name of contact pointD.A. Schott
    B.5.3 Address:
    B.5.3.1Street Address P. Debyelaan 25
    B.5.3.2Town/ cityMaastricht
    B.5.3.3Post code6229 HX
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31433875239
    B.5.5Fax number+31433875246
    B.5.6E-mailda.schott@mumc.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 Yes
    D.2.1.1.1Trade name Genotropin
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Limited
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameGenotropin
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSOMATROPIN
    D.3.9.1CAS number 12629-01-5
    D.3.9.3Other descriptive namerecombinant DNA-derived human growth hormone
    D.3.9.4EV Substance CodeSUB10584MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number12
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Kabuki Syndrome (KS, OMIM 147920) with a mutation in MLL2 gene.
    Kabuki Syndroom (KS, OMIM 147920) met een mutatie in MLL2 gen.
    E.1.1.1Medical condition in easily understood language
    Kabuki syndrome children. Some of the clinical signs and symptoms in KS children are obesity, hypertension, hypotonia and short stature.
    Kabuki Syndroom kinderen. Een deel van de klinische tekenen en symptomen bij KS kinderen zijn overgewicht, hoge bloeddruk, hypotonie en kleine gestalte.
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to assess the relation between the short term metabolic changes after start of rhGH therapy and the long term change in height SDS after one year of treatment.
    Het primaire doel van deze studie is het beoordelen van de relatie tussen de korte termijn metabole veranderingen na start van rhGH therapie en de lange termijn verandering in de lengte SDS na een jaar behandeling.
    E.2.2Secondary objectives of the trial
    Secondly, we want to assess the effects of GH on metabolic risk parameters which are typical parameters for the metabolic syndrome in adults.
    Ten tweede willen we de effecten van GH te beoordelen op metabole risicofactoren parameters die kenmerkend zijn voor het metabool syndroom bij volwassenen.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Children with the MML2 mutation.
    -Children who meet at least four out of five KS characteristics:
    #Facial features: long palpebral fissures with eversion of outer third, arched eyebrows with sparse outer half, prominent and/or misshapen ears, and depressed nasal tip.
    #Skeletal abnormalities.
    #Intellectual disability (mild to moderate).
    #Postnatal short stature.
    #Abnormalities of dermal ridges.
    -Informed consent.
    -Age ≥ four years.
    -Kinderen met de MML2 mutatie.
    -Kinderen die aan ten minste vier van de vijf KS kenmerken:
    # Facial eigenschappen: lange oogspleten met eversie van de buitenste derde, gebogen wenkbrauwen met spaarzame buitenste helft, opvallende en / of misvormde oren, neus en naar beneden hangende tip.
    # Skeletafwijkingen.
    # Verstandelijke handicap (licht tot matig).
    # Postnatale klein gestalte.
    #Abnormale dermatoglyfen.
    -Informed consent.
    -Leeftijd ≥ vier jaar.
    E.4Principal exclusion criteria
    -Children with a chronological or bone age greater than 8 years for girls and 10 years for boys, because of the influence of puberty.
    -Extremely low dietary intake (less than minimal required intake for age according to WHO criteria).
    -Use of medication that might interfere with growth during GH therapy, such as corticosteroids and sex steroids.
    -Kinderen met een chronologische of bot leeftijd hoger dan 8 jaar voor meisjes en 10 jaar voor jongens, vanwege de invloed van de puberteit.
    -Extreem lage inname via de voeding (minder dan minimaal benodigde inname voor leeftijd volgens WHO-criteria).
    -Gebruik van medicatie die kunnen interfereren met de groei tijdens de GH therapie, zoals corticosteroïden en geslachtshormonen.
    E.5 End points
    E.5.1Primary end point(s)
    Is there an increase in TEE during 6 weeks treatment with rhGH in children with Kabuki Syndrome?
    What is the relation between the short-term (6 weeks) change in TEE as measured with the DLW technique and the long term change in height SDS during treatment with rhGH?
    What is the effect of rhGH treatment on metabolic risk parameters typical for the metabolic syndrome in adults?
    Is er een toename van de TEE gedurende 6 weken behandeling met groeihormoon bij kinderen met Kabuki syndroom?
    Wat is de relatie tussen de korte termijn (6 weken) verandering in de TEE gemeten met de DLW techniek en de lange termijn verandering in de lengte SDS tijdens de behandeling met rhGH?
    E.5.1.1Timepoint(s) of evaluation of this end point
    Increase in TEE during 6 weeks treatment and the long term change in height SDS during treatment with rhGH after one year.
    Een toename van de TEE gedurende 6 weken behandeling met groeihormoon en de lange termijn verandering in de lengte SDS na een jaar.
    E.5.2Secondary end point(s)
    To assess the long (1 year) term safety of growth hormone therapy on metabolic risk parameters and body composition.
    Wat is het effect van de groeihormoon behandeling op metabole risicofactoren parameters typisch zijn voor het metabool syndroom bij volwassenen?
    E.5.2.1Timepoint(s) of evaluation of this end point
    After one year of treament with growth hormone.
    Na een jaar groeihormoon behandeling.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 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 study period for each individual is 1 year, which will be for 1 year were included. The study ends when the last visit of the last subject is done.
    De studieduur voor ieder individu is 1 jaar, waarbij gedurende 1 jaar geincludeerd zal worden. De studie eindigd wanneer bij de laatste proefpersoon de laatste controle plaats vindt.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    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
    The metabolic effects of growth hormone treatment will be related to increase in length, so the subject must still be growing (so automatically minors).
    Het metabole effect van groeihormoon behandeling zal gerelateerd worden aan de lengte groeiwinst, dus de proefpersoon moet nog in de groei zijn (dus automatisch minderjarig).
    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)
    If the study results are positive for the subjects, a hormone therapy for this indication group requested
    Indien de studie resultaten positief zijn voor de proefpersonen zal een groeihormoon therapie indicatie voor deze groep aangevraagd worden.
    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 Decision2012-03-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-09-26
    P. End of Trial
    P.End of Trial StatusOngoing
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