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    Summary
    EudraCT Number:2021-001855-15
    Sponsor's Protocol Code Number:QBGJ398-203
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-09-15
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2021-001855-15
    A.3Full title of the trial
    Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL OLE
    Estudio de extensión de fase II, abierto, a largo plazo de infigratinib, un inhibidor selectivo de la tirosina quinasa de los receptores del factor de crecimiento fibroblástico 1-3 (FGFR 1-3), en niños con acondroplasia: PROPEL OLE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate long-term Infigratinib in children with Achondroplasia
    Un estudio para evaluar Infigratinib a largo plazo en niños con acondroplasia
    A.3.2Name or abbreviated title of the trial where available
    PROPEL OLE
    A.4.1Sponsor's protocol code numberQBGJ398-203
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/331/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorQED Therapeutics, Inc.
    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 supportQED Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationQED Therapeutics
    B.5.2Functional name of contact pointDaniela Rogoff
    B.5.3 Address:
    B.5.3.1Street Address8000 Marina Blvd, Suite 400 Brisbane,
    B.5.3.2Town/ cityCalifornia
    B.5.3.3Post code94005
    B.5.3.4CountryUnited States
    B.5.4Telephone number0012144066899
    B.5.6E-mailDaniela.Rogoff@qedtx.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameInfigratinib
    D.3.2Product code BGJ398 (also known as BBP-831)
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNINFIGRATINIB
    D.3.9.1CAS number 872511-34
    D.3.9.2Current sponsor codeBGJ398 (also known as BBP-831)
    D.3.9.4EV Substance CodeSUB186757
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number0.1 to 1.0
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Achondroplasia in Children
    Acondroplasia en niños
    E.1.1.1Medical condition in easily understood language
    Disproportionate short stature with rhizomelia (shortened proximal limbs).
    Estatura desproporcionádamente corta con rizomelia (miembros proximales acortados)
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10000452
    E.1.2Term Achondroplasia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and tolerability of long-term administration of daily doses of oral infigratinib in subjects with ACH.

    To evaluate the efficacy of long-term administration of daily doses of oral infigratinib in subjects with ACH as assessed as changes over time in standing height Z-score.
    Evaluar la seguridad y tolerabilidad de la administración a largo plazo de dosis diarias de infigratinib oral en sujetos con acondroplasia

    Evaluar la eficacia de la administración a largo plazo de dosis diarias de infigratinib en sujetos con acondroplasia conforme a los cambios en el tiempo en la puntuación en la escala heigh-Z
    E.2.2Secondary objectives of the trial
    To evaluate changes in other indicators of growth and development in subjects with ACH receiving long-term treatment with oral infigratinib, such as:
    ○ Change over time in height velocity (HV) Z-score in relation to ACH and non-ACH growth charts.
    ○ Changes over time in other anthropometric parameters after administration of oral infigratinib.
    ○ Age at puberty onset and progression of pubertal development.

    To evaluate changes over time in ACH disease burden with long-term administration of oral doses of infigratinib.
    Evaluar los cambios en otros indicadores de crecimiento y desarrollo en sujetos con acondroplasia que reciben tratamiento a largo plazo con infigratinib oral como:
    _Cambios en el tiempo en la escala Height Velocity-Z en relación con gráficos de crecimiento de sujetos con acondroplasia y sin ella
    _Cambios en el tiempo en otros parámetros tras la administración oral de infigratinib
    _Edad de establecimiento de la pubertad y progresión del desarrollo de la misma

    Evaluar los cambios en el tiempo en la carga acondroplasia con la administración a largo plazo de dosis orales de infigratinib
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion Criteria for Rollover Subjects
    1. Pediatric subjects with ACH who have completed study activities in a previous QED-sponsored interventional study with infigratinib.
    2. Subjects and parent(s) or legally authorized representatives (LARs) are willing and able to comply with study visits and study procedures.
    3. In girls ≥10 years of age or girls of any age who have experienced menarche, having a negative pregnancy test.
    4. If sexually active, subject must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug.
    5. The PI, or a person designated by the PI, will obtain written informed consent from each subject’s Legally Authorised Representative and the subject’s assent, when applicable, before any study-specific activity is performed.

    Inclusion Criteria for Treatment Naïve Subjects
    1. Subject must be 3 to <18 years of age (inclusive) at screening and have growth potential (as defined by HV ≥1.5 cm/year, Tanner stage ≤4 of pubertal development, and bone age of ≤13 years in females and ≤15 years in males.
    2. Subjects and parent(s) or LARs are willing and able to comply with study visits and study procedures.
    3. Subjects are able to swallow oral medication.
    4. Subjects who have a diagnosis of ACH, documented clinically and confirmed by genetic testing.
    5. Subjects have at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry.
    6. In girls ≥10 years of age or girls of any age who have experienced menarche, having a negative pregnancy test.
    7. If sexually active, subject must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug.
    8. The PI, or a person designated by the PI, will obtain written informed consent from each subject’s Legally Authorised Representative and the subject’s assent, when applicable, before any study-specific activity is performed.
    Criterios de inclusión para sujetos en extensión
    1. Sujetos pediátricos con acondroplasia que han completado las actividades de un estudio intervencionista previo con infigratinib promovido por QED
    2. Sujetos o padres/representantes legales autorizados que deseen participar y sean capaces de cumplir con las visitas y procedimientos del estudio.
    3. En niñas mayores de 10 años o de cualquier edad que hayan experimentado menarquia, con un test de embarazo negativo
    4. Si el sujeto es sexualmente activo debe aceptar el uso de un anticonceptivo altamente eficaz mientras recibie la medicación del estudio y durante un mes tras la última dosis
    5. El investigador o persona designada por el investigador, obtendrá el consentimiento informado por escrito de cada representante legal autorizado y el asentimiento del sujeto, cuando aplique, antes de que se realize cualquier actividad del estudio.

    Criterios de inclusión para sujetos nuevos en el tratamiento
    1. Sujetos entre 3 y <18 años en la selección con potencial de crecimiento (velocidad de crecimiento ≥1.5 cm/año, escala de Tanner de desarrollo de la pubertad ≤4 y edad ósea ≤13 años en mujeres y ≤15 años en varones.
    2. Los sujetos y los padres o representantes legales autorizados desean participar y son capaces de cumplir con las visitas y procedimientos del estudio.
    3. Los sujetos son capaces de tragar medicación oral
    4. Sujetos con diagnóstico de acondroplasia, clinicamente documentado y confirmado mediante test genético
    5. Los sujetos tienen al menos un periode de 6 meses de seguimiento del crecimiento en el estudio PROPEL (protocolo QBGJ398-001) antes de entrar en el estudio
    6. En chicas ≥10 años o de cualquier edad que hayan experimentado menarquia, test de embarazo negativo
    7. Si el sujeto es sexualmente activo debe aceptar utilizar un método anticonceptivo altamente eficaz mientras recibe la medicación del estudio y durante 1 mes tras la última dosis
    8. El investigador principal o persona delegada obtendrá el consentimiento escrito de cada representante legal autorizado del sujeto y el asentimiento del sujeto, cuando aplique, antes de realizar cualquier actividad del estudio.
    E.4Principal exclusion criteria
    Exclusion Criteria for Rollover Subjects
    1. Subject has concurrent circumstance, disease, or condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations.
    2. Subjects who developed a medical condition that will require the initiation of treatment with a prohibited medication.
    3. Subjects that prematurely discontinued a prior QED-sponsored interventional study with infigratinib.
    4. Subjects that have reached final height or near final height (as defined by HV <1.5cm/year and Tanner stage ≥4).

    Exclusion Criteria for Treatment Naïve Subjects
    Medical Conditions
    1. Subjects who have hypochondroplasia or short stature condition other than ACH (eg, trisomy 21, pseudoachondroplasia, psychosocial short stature).
    2. Subjects who have significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the subject or would interfere with study participation or safety evaluations, including but not limited to cardiac or vascular disease; hyperthyroidism; abnormal thyroid levels or recently diagnosed hypothyroidism that has not been stable on therapy for at least 3 months; uncontrolled diabetes mellitus (hemoglobin A1c [HbA1c] >9%); adrenal insufficiency; autoimmune inflammatory disease; inflammatory bowel disease; diagnosis of severe sleep apnea that will require surgery or the initiation of continuous positive airway pressure (CPAP) machine use. Subjects with previously diagnosed obstructive sleep apnea (OSA) who are already using a CPAP machine at Screening and whose OSA is stable are eligible provided they continue to be compliant with CPAP use.
    3. Subjects who have a history and/or current evidence of extensive ectopic tissue calcification.
    4. Subjects who have a history of malignancy.
    5. Subjects who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4 or medications that alter the pH of the gastrointestinal tract, including antacids, H2 antagonists (eg, ranitidine), and proton-pump inhibitors (eg, omeprazole); or enzyme-inducing anti-epileptic drugs, including carbamazepine, phenytoin, phenobarbital, and primidone.
    6. Subjects who have current evidence of endocrine alterations of calcium/phosphorus homeostasis (Vitamin D supplementation or Vitamin D treatment for Vitamin D insufficiency or deficiency with normal calcium and phosphorus levels is allowed).
    7. Subjects who have received regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid therapy (ie, >15 mg/m2/day of hydrocortisone or equivalence) or treatment with glucocorticoids at anti-inflammatory doses for over 3 weeks within 6 months of the screening visit (low-dose ongoing inhaled steroid for asthma is acceptable).
    8. Subjects who have received treatment with growth hormone, insulin-like growth factor 1 (IGF 1), anabolic steroids or any investigational or approved drug for the treatment of ACH in the previous 6 months. Subjects that received the last dose of any of these growth-promoting agents ≥6 months before screening are eligible.
    9. Subjects who have significant abnormality in screening laboratory results, including but not limited to the following:
    a. Hemoglobin <10.0 g/dL.
    b. Total bilirubin >1.5× upper limit of normal (ULN).
    c. Aspartate aminotransferase/serum glutamic-oxaloacetic transaminase (AST/SGOT) or alanine aminotransferase/serum glutamic-pyruvic transaminase (ALT/SGPT) >2× ULN.
    d. Glomerular filtration rate <60 mL/min/1.73m2 calculated using the Schwartz formula.

    Other Exclusions
    10. Subjects who have an allergy to any components of the study drug.
    11. Subjects who have had a fracture within 6 months of screening.
    12. Subjects who are eligible to enroll in any other ongoing QED-sponsored interventional study with infigratinib.
    Criterios de exclusión para sujetos en extensión
    1. Cualquier circunstancia, enfermedad o condición concurrentes que a juicio del investigador principal y/o el promotor pudiera interferir con la participación en el estudio o las evaluaciones de seguridad.
    2. Sujetos que desarrollaron una condición médica que requiera el inicio de un tratamiento con una medicación no permitida.
    3. Sujetos que discontinuaron prematuramente de un estudio intervencional promovido por QED con infigratinib
    4. Sujetos que han alcanzado su altura final o están cerca de la misma ( velocidad de crecimiento <1.5cm/año y escala de Tanner ≥4)

    Criterios de inclusión para sujetos nuevos en el tratamiento
    Condiciones médicas
    1. Sujetos con hipocondroplasia o baja estatura no relacionada con la acondroplasia (p.ej.: trisoma 21, pseudoacondroplasia, baja estatura psicosocial)
    2. Sujetos con una enfermedad o condición concurrente significativas que a juicio del investigador y/o el promotor pudieran representar un mayor riesgo para el sujeto o pudiera interferir con la participación en el estudio o las evaluaciones de seguridad, incluyendo pero no limitadas a enfermedad cardiaca o vascular; hipertiroidismo, niveles tiroideos anormales o hipotiroidismo de diagnóstico reciente inestable en tratamiento por al menos 3 meses; diabetes melitus incontrolada (hemoglobina A1c [HbA1c] >9%); insuficiencia adrenal; enfermedad autoimmune inflamatoria; enfermedad intestinal inflamatoria; diagnóstico de apnea del sueño severa que pueda requerir cirugía o inicio de suplemento continuo de oxígeno artificial. Los sujetos con diagnóstico de apnea del sueño obstructiva que necesitan suplemento continuo de oxígeno artificial durante la selección y cuya condición es estable son elegibles mientras continúen el uso del mismo.
    3. Sujetos con antecedentes y/o evidencia actual de calcificación ectópica de tejidos
    4. Sujetos con antecedentes de cáncer
    5. Sujetos que actualmente reciben tratamiento con agentes conocidos como inductores o inhibidores del citocromo P450 (CYP) 3A4 fuertes, o medicación que altere el PH del tracto gastrointestinal incluyendo antiácidos, antagonistas H2 (p.ej: ranitidina), inhibidores bomba de protones (p.ej: omeprazol); o anti epilécticos inductores de enzimas incluyendo carbamacepina, fenitoina, fenobarbital y primidona.
    6. Sujetos con evidencia actual de alteraciones endocrinas de homeostasis de calcio/fósforo (la suplementación de vitamina D o tratamiento con vitamina D para la insuficiencia de la misma o deficiencia de los niveles normales de calcio y fósforo están permitidos)
    7. Sujetos que han recibido tratamiento regular a largo plazo (≥3 semanas) con dosis suprafisiológicas de terapia con glucocorticoides (p.ej: >15 mg/m2/día de hidrocortisona o equivalente) o tratamiento con glucocorticoides a dosis anti inflamatorias durante más de 3 semanas en los 6 meses anteriores a la visita de selección (las dosis bajas de esteroides inalados para el asma son aceptables)
    8. Sujetos que han recibido tratamiento con hormona de crecimiento, factor de crecimiento 1 (IGF 1) con insulina, esteroides anabolizantes o cualquier medicación aprobada o en investigación para el tratamiento de la acondroplasia los 6 meses anteriores. Los sujetos que recibieron la última dosis de cualquiera de estos agentes de crecimiento ≥6 antes de la visita de selección son elegibles.
    9. Sujetos con abnormalidades significativas en los resultados de laboratorio en la selección incluyendo pero no limitadas a:
    a. Hemoglobina <10.0 g/dL.
    b. Bilirrubina total >1.5× por encima del límite normal
    c. Aminotransferasa aspartato/AST/SGOT o ALT/SGPT >2× por encima del límite normal.
    d. Tasa de filtrado glomerular <60 mL/min/1.73m2 calculada según la fórmula de Schwartz

    Otras exclusiones
    10. Sujetos con alergia a algunos de los componentes de la medicación en estudio
    11. Sujetos que hayan tenido alguna fractura ósea en los 6 meses anteriores a la selección
    12. Sujetos elegibles para cualquier otro ensayo intervencional en marcha promovido por QED con infigratinib
    E.5 End points
    E.5.1Primary end point(s)
    • Safety evaluations including incidence, type, severity, and causality of adverse events (AEs), serious adverse events (SAEs), laboratory test results (urinalysis, chemistry, hematology), and clinically significant changes in vital signs, physical examinations, ophthalmic and dental evaluations, and imaging (x-rays, dual x-ray absorptiometry [DXA] scan).
    • Changes over time in height Z-score in relation to ACH and non-ACH growth charts.
    - Evaluaciones de seguridad incluyendo la incidencia, tipo, gravedad y causalidad de los acontecimientos adversos (AEs), acontecimientos adversos graves (SAEs), resultados de laboratorio (urinálisis, química, hematología) y cambios clínicamente significativos en los signos vitales, exploraciones físicas, oftalmológicas y dentales así como pruebas de imagen (rayos x, escáner DXA)
    - Cambios en el tiempo en la escala height-Z en relación a las gráficas de crecimiento de acondroplasia y no acondroplasia
    E.5.1.1Timepoint(s) of evaluation of this end point
    Every year throughout the duration of the study.
    Cada año a lo largo de la duración del estudio
    E.5.2Secondary end point(s)
    • Change over time in HV Z-score in relation to ACH and non-ACH growth charts.
    • Changes over time in body proportions that may include, but may not be limited to, upper to lower body segment ratio, upper arm to forearm length ratio, upper leg to lower leg length ratio, arm span to standing height ratio, and head circumference to standing height ratio.
    • Change over time in weight Z-score and body mass index (BMI).
    • Age of puberty onset and time to Tanner stage ≥4.
    Changes over time in disease-specific complications that may include (but are not limited to):
    ○ Number of episodes of otitis media per year.
    ○ Number of episodes and/or severity of sleep apnea.
    ○ Changes over time in range of motion (hip, knee and elbow).
    ○ Changes over time in skeletal abnormalities of the lower extremities and spine (ie, tibial bowing, interpedicular narrowing, lumbar hyperlordosis, etc.).
    ○ Quality of life [QoL] as assessed by Pediatric Quality of Life Inventory (PedsQL [generic core scale short form, child and parent reports]).
    - Cambios en el tiempo en puntuación en la escala de velocidad de crecimiento (HV-Z) en relación a las gráficas de crecimiento con acondroplasia y sin ella
    -Cambios en el tiempo en las proporciones corporales que pueden incluir, aunque no se limitan a, relación entre los segmentos superior e inferior, parte superior e inferior del brazo, parte superior e inferior de la pierna, lapso del brazo frente a altura y circunferencia de la cabeza frente a altura
    - Cambio en el tiempo en la puntuación de la escala de peso Weigh Z e índice de masa corporal (IMC)
    - Edad de establecimiento de la pubertad y momento hasta nivel 4 en la escala de Tanner

    Cambios en el tiempo en complicaciones específicas de la enfermedad que pueden incluir, aunque no se limitan a:
    ○ Número de episodios de otitis media al año
    ○ Número de episodios y/o gravedad de apena del sueño
    ○ Cambios en el tiempo en el rango de movimiento (cadera, rodilla y codo)
    ○ Cambios en el tiempo en deficiencias óseas en las extremidades inferiores y columna (p.ej: desviación tibial, estrechamiento interpedicular, hiperlordosis lumbar, etc...)
    ○ Calidad de vida evaluada mediante PedsQL (escala genérica reducida, reportes de padres y sujeto)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Every year throughout the duration of the study.

    For the complete list please refer to the protocol
    Cada año a lo largo de la duración del estudio
    Para una lista completa revisar protocolo
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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.8.3 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Australia
    Canada
    France
    Spain
    United Kingdom
    United States
    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
    The end of the study is defined as the date of the last visit of the last subject (LVLS) in the study.
    El fin del estudio se define como la fecha de la última visita del último paciente (LVLS)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years10
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years10
    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: 280
    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: 200
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 80
    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 Yes
    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 below the age limit to provide consent
    Sujetos sin la edad para proporcionar consentimiento
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 180
    F.4.2.2In the whole clinical trial 280
    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)
    Continued access to study drug is not applicable because subjects enrolled in this study are expected to complete linear growth and reach final height while participating in this study.
    El acceso continuado al tratamiento no aplica dado que se espera que los sujetos incluidos en este estudio completen su crecimiento y alcancen su altura final durante la participación en el mismo.
    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 Decision2022-02-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-30
    P. End of Trial
    P.End of Trial StatusOngoing
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