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    Summary
    EudraCT Number:2016-003874-42
    Sponsor's Protocol Code Number:CP-4-006
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-09-06
    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 number2016-003874-42
    A.3Full title of the trial
    A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency
    Studio multicentrico, randomizzato, in aperto, di fase 3, a 12 mesi, per valutare la sicurezza ed efficacia della terapia con MOD-4023 a somministrazione settimanale rispetto a quella con Genotropin a somministrazione giornaliera per il trattamento di bambini/e affetti da deficienza di ormone della crescita in età prepuberale
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency
    Studio di efficacia e sicurezza sulla terapia con somministrazione settimanale di MOD-4023 in confronto con la somministrazione giornaliera di genotropin® in bambini/e affetti da deficienza di ormone della crescita.
    A.3.2Name or abbreviated title of the trial where available
    N/A
    N/A
    A.4.1Sponsor's protocol code numberCP-4-006
    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 SponsorPRM|21097
    B.1.3.4CountryUnited States
    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 supportOPKO Biologics Ltd.
    B.4.2CountryIsrael
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationClinical Professionals Inc (CPI Global CRO)
    B.5.2Functional name of contact pointProject Manager
    B.5.3 Address:
    B.5.3.1Street AddressVia Paolo Sarpi 56
    B.5.3.2Town/ cityMilano
    B.5.3.3Post code20154
    B.5.3.4CountryItaly
    B.5.4Telephone number+39 02 33104372
    B.5.5Fax number+39 040 996699
    B.5.6E-mailpgraziani@cpiglobalcro.com
    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/3/12/1087
    D.3 Description of the IMP
    D.3.2Product code MOD-4023
    D.3.4Pharmaceutical form 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 INNSomatrogon
    D.3.9.1CAS number 1663481-09-1
    D.3.9.2Current sponsor codeMOD-4023
    D.3.9.4EV Substance CodeSUB31414
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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 Yes
    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.IMP: 2
    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/3/12/1087
    D.3 Description of the IMP
    D.3.2Product code MOD-4023
    D.3.4Pharmaceutical form 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 INNSomatrogon
    D.3.9.1CAS number 1663481-09-1
    D.3.9.2Current sponsor codeMOD-4023
    D.3.9.4EV Substance CodeSUB31414
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 Yes
    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.IMP: 3
    D.1.2 and D.1.3IMP RoleComparator
    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 AuthorisationUnited Kingdom
    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 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 INNSomatropin
    D.3.9.2Current sponsor codeGenotropin
    D.3.9.4EV Substance CodeSUB12322MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 Yes
    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.IMP: 4
    D.1.2 and D.1.3IMP RoleComparator
    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 AuthorisationUnited Kingdom
    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 Powder and solvent for solution for infusion
    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.3.9.2Current sponsor codeGenotropin
    D.3.9.4EV Substance CodeSUB12322MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 Yes
    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
    Growth hormone deficiency in pre-pubertal children
    Deficit di ormone della crescita in bambini/e prepuberali
    E.1.1.1Medical condition in easily understood language
    Treatment of children with growth failure due to growth hormone deficiency
    Trattamento di bambini/e con ritardo della crescita a causa di deficit di ormone della crescita
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10056438
    E.1.2Term Growth hormone deficiency
    E.1.2System Organ Class 10014698 - Endocrine disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate that weekly MOD-4023 administration is non-inferior to daily Genotropin administration in terms of safety and efficacy outcomes
    Dimostrare che la somministrazione settimanale di MOD-4023 è non-inferiore alla somministrazione giornaliera di Genotropin in termini di sicurezza ed efficacia
    E.2.2Secondary objectives of the trial
    To evaluate the effect of weekly MOD-4023 and daily Genotropin administration on quality of life, as measured by the QoLISSY (Quality of Life in Short Stature Youth) at specific number of countries.
    Valutare l'effetto della somministrazione settimanale di MOD-4023 e giornaliera di Genotropin sulla qualità della vita, quando misurata con QoLISSY (Quality of Life in Short Stature Youth) in uno specifico numero di nazioni.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Pre-pubertal children aged =3 years , and not yet 11 years for girls (10 years and 364 days) or not yet 12 years (11 years and 364 days) for boys, (on the date of ICF signature), with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiency.
    2. Confirmed diagnosis of GHD by two different GH provocation tests defined as a peak plasma GH level of =10 ng/mL, determined by local or central laboratory using a validated assay . Global Medical Monitor may accept prior local laboratory results; subject to pre-approval and if the tests were conducted as recommended in the protocol
    3. Bone age (BA) is not older than chronological age and should be less than 10 for girls and less than 11 for boys.
    4. Without prior exposure to any rhGH therapy (naïve patients).
    5. Impaired height velocity defined as:
    • Annualized height velocity (HV) below the 25th percentile for CA (HV < -0.7 SDS) and gender according to Prader HV standard tables, Tanner HV curves, or local primary care provider standard.
    • The interval between two height measurements should be at least 6 months, but should not exceed 18 months prior to inclusion.
    6. BMI must be within ±2 SDS of mean BMI for the chronological age and sex.
    7. Baseline IGF-I level of at least 1 SD below the mean IGF-I level standardized for age and sex (IGF-I SDS =-1) according to the central laboratory reference values. A single re-test will be allowed (subject to discussion with medical monitor) if all other criteria are met.
    8. Normal calculated GFR based on updated “bedside” Schwartz formula for pediatric patients (calculation is recommended below):
    CrCL (mL/min/1.73 m2) =0.413 * Ht / Scr
    Ht: height in cm;
    Scr: serum creatinine in mg/dL;
    9. Children with multiple hormonal deficiencies must be on stable replacement therapies (no change in dose) for other hypothalamo-pituitary organ axes for at least 3 months prior to ICF signing
    10. Normal 46XX karyotype for girls.
    Willing and able to provide written informed consent of the parent or legal guardian of the patient and written assent from pediatric patients (where applicable based on age and country regulation).
    1. Bambini/e prepuberi di =3 anni di età, che non abbiano ancora compiuto (alla data della firma dell’ICF) 11 anni (10 anni e 364 giorni), per le bambine, o 12 anni (11 anni e 364 giorni) per i bambini, con GHD isolato o con insufficienza GH come parte di un deficit ormonale ipofisario multiplo.
    2. Diagnosi di GHD confermata con due test di provocazione GH, definiti come livello di picco plasmatico di GH =10 ng/mL tramite analisi condotta dal laboratorio locale o da quello centralizzato con test convalidatoa. Se condotti come da protocollo e previa pre-approvazione, il medical monitor globale potrà accettare i risultati di precedenti test effettuati nei laboratorio locali Allegato B.
    3. Età ossea (EO) non superiore all’età cronologica e inferiore a 10 anni per le bambine e a 11 anni per i bambini.
    4. Nessuna precedente esposizione a qualsiasi terapia rhGH (pazienti naïve).
    5. Compromissione della velocità di crescita definita come segue:
    • Velocità di crescita (HV) annualizzata inferiore al 25esimo percentile per EC (SDS di HV < -0,7) e sesso, secondo le tabelle standard HV Prader, le curve HV di Tanner, o lo standard locale del medico di base.
    • L’intervallo tra due misurazioni della statura deve essere di almeno 6 mesi, ma non superiore a 18 mesi prima dell’inclusione.
    6. Il BMI deve cadere entro ±2 SDS del BMI medio per età cronologica e sesso.
    7. Livello di IGF-I al basale di almeno 1 SD al di sotto del livello medio di IGF-I standardizzato per età e sesso (IGF-I SDS =-1)a secondo i valori di riferimento del laboratorio centralizzato. Se tutti gli altri criteri sono soddisfatti, sarà consentita una singola ripetizione del test (subordinata alla discussione con il medical monitor).
    8. GFR normale calcolato con il metodo “bedside” di Schwartz per soggetti pediatrici (formula di calcolo raccomandata riportata sotto):
    CrCL (mL/min/1,73 m2) =0,413 * Ht/Scr
    Ht: statura in cm;
    Scr: creatinina sierica in mg/dL;
    9. I bambini con deficit ormonali multipli devono trovarsi in regime terapeutico sostitutivo stabile (nessuna variazione posologica) per gli altri organi dell’asse ipotalamo-ipofisario da almeno 3 mesi prima della firma
    ICF.
    10. Cariotipo 46 XX normale per le bambine.
    11. Disponibilità e capacità di fornire il consenso informato scritto del genitore o del tutore legale del paziente e assenso scritto dei pazienti pediatrici (ove applicabile in base all’età e alla normativa nazionale)
    E.4Principal exclusion criteria
    1. Children with prior history of leukemia, lymphoma, sarcoma or any other forms of cancer.
    2. History of radiation therapy or chemotherapy
    3. Malnourished children defined as BMI < -2 SDS for age and sex
    4. Children with psychosocial dwarfism
    5. Children born small for gestational age (SGA – birth weight and/or birth length <-2 SDS for gestational age)
    6. Presence of anti-hGH antibodies at screening
    7. Any clinically significant abnormality likely to affect growth or the ability to evaluate growth, such as, but not limited to, chronic diseases like renal insufficiency, spinal cord irradiation, etc.
    8. Type 1 and type 2 diabetic patients who, in the opinion of the investigator, are not receiving standard of care treatment, or are non-compliant with their prescribed treatment or who are in poor metabolic control
    9. Chromosomal abnormalities including Turner’s syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasias.
    10. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids, or sex steroids, with the exception of ADHD drugs or hormone replacement therapies (thyroxin, hydrocortisone, desmopressin [DDAVP])
    11. Children requiring glucocorticoid therapy (e.g. for asthma) that are taking chronically a dose greater than 400 µg/d of inhaled budesonide or equivalent
    12. Major medical conditions and/or presence of contraindication to r-hGH treatment.
    13. Closed epiphyses
    14. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
    15. Drug, substance, or alcohol abuse.
    16. Known hypersensitivity to the components of study medication.
    17. Other causes of short stature such as celiac disease, uncontrolled primary hypothyroidism and rickets.
    18. The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct.
    Participation in any other trial of an investigational agent within 30 days prior to ICF signature (including administration of investigational agent).
    1. Bambini/e con pregressa storia clinica di leucemia, linfoma, sarcoma o altre forme di cancro.
    2. Storia clinica di terapia radiante o chemioterapia.
    3. Bambini/e con malnutrizione definita come BMI < -2 SDS per età e sesso.
    4. Bambini/e con nanismo psicosociale.
    5. Bambini/e nati piccoli per età gestazionale (SGA – peso alla nascita e/o lunghezza alla nascita <-2 SDS per età gestazionale)
    6. Presenza di anticorpi anti-hGH allo screening
    7. Qualsiasi anomalia clinicamente significativa capace di influenzare la crescita o la capacità di valutazione della crescita, come, ad esempio, ma non limitatamente, malattie croniche come l’insufficienza renale, l’irradiazione del midollo spinale, ecc.
    8. Pazienti diabetici di tipo 1 e tipo 2 che a giudizio dello sperimentatore non abbiano ricevuto una terapia standard, o che siano inadempienti rispetto al trattamento prescritto, o con scarso controllo del metabolismo (i criteri di controllo del diabete sono definiti in Allegato F).
    9. Anomalie cromosomiche, tra cui sindrome di Turner, sindrome di Laron, sindrome di Noonan, sindrome di Prader-Willi, sindrome di Russell-Silver, mutazioni/delezioni SHOX e displasie scheletriche.
    10. Somministrazione concomitante di altri trattamenti con possibile effetto sulla crescita, come steroidi anabolizzanti, steroidi sessuali, ad eccezione dei farmaci per l’ADHD o della terapia ormonale sostitutiva (tiroxina, idrocortisone, desmopressina (DDAVP))
    11. Bambini/e che necessitano di terapia con glucocorticoidi (ad esempio per l’asma) che assumono cronicamente dosi superiori a 400 µg/die dibudesonide per via inalatoria o farmaco equivalente come riportato in Allegato J.
    12. Gravi condizioni mediche e/o controindicazioni al trattamento con r-hGH.
    13. Epifisi chiuse
    14. Paziente con HIV accertata o sospetta, o con patologia in stato avanzato come AIDS o tubercolosi.
    15. Abuso di droghe, sostanze o alcool.
    16. Ipersensibilità nota ai componenti del farmaco di studio.
    17. Altre cause di bassa statura, come celiachia, ipotiroidismo primario non controllato e rachitismo.
    18. Potenziale inadempienza del paziente e/o del genitore/tutore legale alle linee di condotta dello studio.
    19. Partecipazione ad altri studi clinici con farmaci sperimentali nei 30 giorni precedenti alla firma dell’ICF (inclusa la somministrazione di un farmaco sperimentale).
    E.5 End points
    E.5.1Primary end point(s)
    Annual Height Velocity (HV) in cm/year
    Velocità di crescita annuale (HV) in cm/anno
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and after 12 months of treatment
    Baseline e dopo 12 mesi di trattamento
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints (Auxology/Clinical):
    • Annualized height velocity after 6 months of treatment
    • Change in height SDS at 6 and 12 months, compared to baseline
    • Change in bone maturation (BM) at the end of 12 months, compared to Screening bone age (calculated as BA/CA)
    Secondary endpoints (Biochemical):
    • Absolute IGF-I levels on day 4(-1) after MOD-4023 dosing across study visits
    • IGF-I SDS on day 4(-1) after MOD-4023 dosing across study visits
    •IGFBP-3 levels and IGFBP-3 SDS on day 4(-1) after MOD-4023 dosing across study visits
    Additional Endpoints
    •QoL endpoint measured by the QoLISSY core questionnaire at baseline and month 12 or early termination in specific countries per Appendix L.
    Endpoint secondari di efficacia (auxologici/clinici):
    •Velocità di crescita annualizzata dopo 6 mesi di trattamento.
    •SDS del cambiamento di statura a 6 e 12 mesi rispetto al basale.
    •Cambiamento di maturazione ossea (MO) al termine dei 12 mesi rispetto all’età ossea allo screening (calcolata come rapporto EO/EC)
    Endpoint secondari (biochimici):
    •Livelli assoluti di IGF-I al giorno 4 (-1) dopo il dosaggio di MOD-4023 durante le visite di studio.
    •SDS di IGF-I al giorno 4 (-1) dopo il dosaggio di MOD-4023 durante le visite di studio.
    •Livelli di IGFBP-3 e SDS di IGFBP-3 al giorno 4 (-1) dopo il dosaggio di MOD-4023 durante le visite di studio.
    Endpoint addizionale:
    •QoL endpoint misurato con il QoLISSY core questionnaire al baseline ed al mese 12 o in caso di prematura discontinuazione in specifiche nazioni come da Appendix L.
    E.5.2.1Timepoint(s) of evaluation of this end point
    as above
    come sopra
    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 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) 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA68
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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 years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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) 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: 220
    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 No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Children of age 3-11
    Bambini/e tra i 3 e gli 11 anni
    F.4 Planned number of subjects to be included
    F.4.1In the member state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 68
    F.4.2.2In the whole clinical trial 220
    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)
    Standard care or participation in other study until marketing approval.
    Terapia standard o partecipazione in un altro studio sino alla autorizzazione alla commercializzazione
    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 Decision2017-06-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-04-26
    P. End of Trial
    P.End of Trial StatusOngoing
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