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    Summary
    EudraCT Number:2011-000460-10
    Sponsor's Protocol Code Number:EMR200104_10PREDICTVALIDATION
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-02-27
    Trial results View 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 number2011-000460-10
    A.3Full title of the trial
    First year growth response associated genetic markers validation Phase IV open-label study in Growth Hormone Deficient and Turner Syndrome pre-pubertal children : the PREDICT Pharmacogenetics Validation Study
    Studio in aperto di fase IV per la validazione dei marcatori genetici associati alla risposta in termini di crescita durante il primo anno di trattamento nei bambini in eta' prepuberale affetti da deficit di ormone della crescita o da sindrome di Turner: studio di validazione farmacogenetica PREDICT.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Validation study for genetic markers for growth hormone treatment in growth hormone deficient and turner syndrome patients
    Studio per la validazione dei marcatori genetici associati al trattamento con ormone della crescita in pazienti affetti da deficit dell'ormone della crescita o da Sindrome di Turner
    A.3.2Name or abbreviated title of the trial where available
    PREDICT PGx Validation Study
    PREDICT PGx Studio di Validazione
    A.4.1Sponsor's protocol code numberEMR200104_10PREDICTVALIDATION
    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 SponsorMERCK SERONO SA
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportMerck Serono SA
    B.4.2CountrySwaziland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMerck KgaA
    B.5.2Functional name of contact pointCommunication Center Merck KGaA
    B.5.3 Address:
    B.5.3.1Street AddressFrankfurter Strasse 250
    B.5.3.2Town/ cityDarmstadt
    B.5.3.3Post code64293
    B.5.3.4CountryGermany
    B.5.4Telephone number49 6151 725200
    B.5.5Fax number49 6151 722000
    B.5.6E-mailservice@merck.de
    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.2.1.2Country which granted the Marketing AuthorisationItaly
    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.3.4Pharmaceutical form Powder and solvent for solution 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 INNNA
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration numberNA
    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
    Idiopathic growth hormone deficienty Turner Syndrome
    Deficit dell'ormone idiopatico della crescita Sindrome di Turner
    E.1.1.1Medical condition in easily understood language
    Patients having a growth hormone deficiency or Turner Syndrome
    Pazienti affetti da deficit dell'ormone della crescita o da sindrome di Turner
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level SOC
    E.1.2Classification code 10014698
    E.1.2Term Endocrine disorders
    E.1.2System Organ Class 10014698 - Endocrine disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10056438
    E.1.2Term Growth hormone deficiency
    E.1.2System Organ Class 10014698 - Endocrine disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10045181
    E.1.2Term Turner's syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To confirm that at least one of the genetic markers associated to the amplitude of first year growth response to recombinant human growth hormone (r-hGH) treatment identified in PREDICT Long- Term Follow-Up (LTFU) Study (28614) is replicated in an independent population of this study of prepubertal children with either idiopathic growth hormone deficiency (IGHD) or Turner Syndrome (TS).
    Confermare che almeno uno dei marcatori genetici associati all'ampiezza della risposta in termini di crescita durante il primo anno di trattamento con l'ormone della crescita umano ricombinante (r-hGH) identificati nello Studio di follow-up a lungo termine (LTFU, Long-Term Follow-Up) (28614) risulta replicato in una popolazione indipendente di questo studio costituita da bambini (entrambi i sessi) in età prepuberale affetti o da deficit idiopatico di ormone della crescita (IGHD) o da sindrome di Turner (TS).
    E.2.2Secondary objectives of the trial
    1) To evaluate the contribution of validated genetic markers related to the amplitude of first year growth in IGHD children in response to r-hGH therapy using a model derived from the growth hormone deficiency (GHD) Kabi-Pharmacia International Growth Study (KIGS) predictive model of the first year growth response to r-hGH. 2) To evaluate the contribution of validated genetic markers related to the amplitude of first year growth in TS girls in response to rhGH therapy in TS girls using a model derived from the TS KIGS predictive model of the first year growth response to r-hGH.
    1) Valutare il contributo dei marcatori genetici convalidati correlati all'ampiezza della risposta in termini di crescita durante il primo anno di terapia con r-hGH nei bambini (entrambi i sessi) affetti da IGHD usando un modello derivato dal modello predittivo dello studio KIGS (Kabi-Pharmacia International Growth Study) nel deficit di ormone della crescita (GHD) relativo alla risposta in termini di crescita durante il primo anno di terapia con rhGH. 2) Valutare il contributo dei marcatori genetici convalidati correlati all'ampiezza della risposta in termini di crescita durante il primo anno di terapia con r-hGH nelle bambine affette da TS usando un modello derivato dal modello predittivo dello studio KIGS nella TS relativo alla risposta in termini di crescita durante il primo anno di terapia con rhGH.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Pre-established diagnosis of IGHD or TS based on classical criteria with at least 1 year of r-hGH therapy and with Tanner stage 1 at treatment start 2) Retrospective availability of a complete set of clinical, auxological and biological parameters necessary for building the predictive model
    1) Diagnosi precedentemente accertata di IGHD o TS basata sui criteri classici, con almeno 1 anno di terapia con r-hGH e con stadio 1 di Tanner all'inizio del trattamento. 2) Disponibilità retrospettiva di una documentazione completa dei parametri clinici, auxologici e biologici necessari per costruire il modello predittivo.
    E.4Principal exclusion criteria
    1) Acquired GHD 2) Any drug or disease that could affect growth
    1)GHD acquisito. 2) Qualsiasi farmaco o patologia che possa influenzare l'accrescimento.
    E.5 End points
    E.5.1Primary end point(s)
    The list of genetic markers associated to the first year growth response to r-hGH measured by the 3 following growth parameters: Change from baseline to year 1 in Height (cm) Change from baseline to year 1 in Height standard deviation score (SDS) Height Velocity (HV) (cm/year) SDS at 1 year
    L'elenco dei marcatori genetici (vedi Appendice I) associati alla risposta in termini di crescita durante il primo anno di terapia con r-hGH misurata mediante i seguenti 3 parametri di crescita: variazione in altezza (H) (cm) dal basale all'anno 1; variazione del punteggio della deviazione standard (SDS) relativa all'altezza (H) (cm) dal basale all'anno 1; SDS della velocità di accrescimento (HV) (cm/anno) a 1 anno.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Al the time of final report
    Al momento della relazione finale
    E.5.2Secondary end point(s)
    The first year growth predictive value of the models including the validated genetic markers on HV (cm/year) at 1 year in IGHD children and in TS girls treated with r-hGH combined to wellestablished clinical, auxological and biological markers derived from the KIGS GHD predictive model of the first year growth response to r-hGH therapy
    Il valore predittivo della crescita durante il primo anno fornito dai modelli costituiti dalla combinazione data dai marcatori genetici convalidati relativi alla HV (cm/anno) a 1 anno di trattamento con r-hGH nei bambini (entrambi i sessi) affetti da IGHD e nelle bambine affette da TS e dai parametri clinici, auxologici e biologici accertati; modelli derivanti dal modello predittivo dello studio KIGS nel GHD sulla risposta in termini di crescita alla terapia con r-hGH.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Al the time of final report
    Al momento della relazione finale
    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 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 Yes
    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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    Russian Federation
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months13
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months13
    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.1.1Number of subjects for this age range: 350
    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: 175
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 175
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 45
    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
    Paediatric patiets
    Pazienti pediatrici
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state60
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 335
    F.4.2.2In the whole clinical trial 395
    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)
    No IMP Trial
    Studio Clinico che non prevede IMP
    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 UKCRN
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-11-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-07-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2012-10-03
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