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    Summary
    EudraCT Number:2020-005554-23
    Sponsor's Protocol Code Number:1199-0378
    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:2021-10-05
    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 number2020-005554-23
    A.3Full title of the trial
    An open-label trial of the long-term safety and tolerability of nintedanib per os, on top of standard of care, over at least 2 years, in children and adolescents with clinically significant fibrosing Interstitial Lung Disease (InPedILD™-ON)
    Ensayo abierto de seguridad y tolerabilidad a largo plazo de nintedanib oral, añadido al tratamiento estándar, durante al menos 2 años, en niños y adolescentes con Enfermedad Pulmonar Intersticial fibrosante clínicamente significativa (InPedILD™-ON)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate long-term safety of nintedanib in children and adolescents with interstitial lung disease (InPedILD™-ON)
    Estudio para evaluar la seguridad a largo plazo de nintedanib en niños y adolescentes con Enfermedad Pulmonar Intersticial (InPedILD™-ON)
    A.3.2Name or abbreviated title of the trial where available
    InPedILD™-ON
    A.4.1Sponsor's protocol code number1199-0378
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/150/2019
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBoehringer Ingelheim España, S.A.
    B.1.3.4CountrySpain
    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 supportBoehringer Ingelheim España, S.A.
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBoehringer Ingelheim Pharma GmbH&Co KG
    B.5.2Functional name of contact pointCT Disclosure & Data Transparency
    B.5.3 Address:
    B.5.3.1Street AddressBinger Strasse 173
    B.5.3.2Town/ cityIngelheim am Rhein
    B.5.3.3Post code55216
    B.5.3.4CountryGermany
    B.5.4Telephone number+34 93 4045100
    B.5.5Fax number+34 93 4045580
    B.5.6E-mailclintriage.rdg@boehringer-ingelheim.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 Yes
    D.2.1.1.1Trade name Ofev®
    D.2.1.1.2Name of the Marketing Authorisation holderBoehringer Ingelheim International GmbH
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameNintedanib 150 mg capsules
    D.3.2Product code Nintedanib
    D.3.4Pharmaceutical form Capsule, soft
    D.3.4.1Specific paediatric formulation No
    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 INNNintedanib
    D.3.9.1CAS number 656247-17-5
    D.3.9.3Other descriptive nameNINTEDANIB
    D.3.9.4EV Substance CodeSUB120728
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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.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 Yes
    D.2.1.1.1Trade name Ofev®
    D.2.1.1.2Name of the Marketing Authorisation holderBoehringer Ingelheim International GmbH
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameNintedanib 100 mg capsules
    D.3.2Product code Nintedanib
    D.3.4Pharmaceutical form Capsule, soft
    D.3.4.1Specific paediatric formulation No
    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 INNNintedanib
    D.3.9.1CAS number 656247-17-5
    D.3.9.3Other descriptive nameNINTEDANIB
    D.3.9.4EV Substance CodeSUB120728
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 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.IMP: 3
    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 nameNintedanib 25 mg capsules
    D.3.2Product code Nintedanib
    D.3.4Pharmaceutical form Capsule, soft
    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 INNNintedanib
    D.3.9.1CAS number 656247-17-5
    D.3.9.3Other descriptive nameNINTEDANIB
    D.3.9.4EV Substance CodeSUB120728
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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
    Interstitial Lung Disease
    Enfermedad Pulmonar Intersticial
    E.1.1.1Medical condition in easily understood language
    Interstitial Lung Disease
    Enfermedad Pulmonar Intersticial
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10066393
    E.1.2Term Respiratory bronchiolitis-associated interstitial lung disease
    E.1.2System Organ Class 100000004855
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The trial will assess the safety and tolerability of long-term treatment with nintedanib in pediatric patients with clinically significant fibrosing ILD.
    The primary objective is to estimate the incidence of treatment emergent adverse events over the whole trial.
    El ensayo evaluará la seguridad y tolerabilidad del tratamiento a largo plazo con nintedanib en pacientes pediátricos con EPI fibrosante clínicamente significativa.
    El objetivo principal es estimar la incidencia de reacciones adversas derivadas del tratamiento durante todo el ensayo.
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplica
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    HRCT substudy:
    only at selected sites in the United States (US) for patients who already participated in the similar substudy in the parent trial
    Details of the planned HRCT analyses and endpoints will be described in a specific Statistical Analysis Plan (SAP) for the substudy and are considered exploratory.
    Subestudio de TCAR: solo en centros seleccionados en Estados Unidos para pacientes que ya participaron en el subestudio del ensayo parental.
    Los detalles de los análisis y los criterios de valoración planeados en el TCAR se describirán en un Plan de Análisis Estadístico (SAP) específico para el subestudio y se considerarán exploratorios.
    E.3Principal inclusion criteria
    For new patients:

    1. Children and adolescents 6 to 17 years old at Visit 2.
    2. Signed and dated written informed consent and assent, where applicable, in accordance with ICH-GCP and local legislation prior to admission to the trial.
    3. Male or female patients. Female of childbearing potential (WOCBP1) must confirm that sexual abstinence is standard practice and will be continued until 3 months after last drug intake, or be ready and able to use a highly effective method of birth control per ICH M3 (R2) that results in a low failure rate of less than 1% per year when used consistently and correctly, in combination with one barrier method, from 28 days prior to initiation of study treatment, during treatment and until 3 months after last drug intake. Sexual abstinence is defined as abstinence from any sexual act that may result in pregnancy.
    4. Patients with evidence of fibrosing ILD on HRCT within 12 months of Visit 1 as assessed by the investigator and confirmed by central review.
    5. Patients with FVC % predicted ≥25% at Visit 2.
    6. Patients with clinically significant disease at Visit 2, as assessed by the investigator based on any of the following:
    - Fan score ≥3, or
    - Documented evidence of clinical progression over time based on either
    o a 5-10% relative decline in FVC% predicted accompanied by worsening symptoms, or
    o a ≥10% relative decline in FVC % predicted, or
    o increased fibrosis on HRCT, or
    o other measures of clinical worsening attributed to progressive lung disease

    For roll-over patients from the InPedILD™ study:

    Only criteria 2 and 3 listed for new patients are applicable with the following additional inclusion criterion:
    7. Patients who completed the InPedILD™ trial as planned and who did not permanently prematurely discontinue study treatment.

    For patients who discontinued treatment permanently in 1199-0337 but are potentially eligible:

    Criteria for new patients are applicable except criteria 4, and 6
    Para pacientes nuevos:
    1. Niños y adolescentes de 6 a 17 años de edad en la visita 2.
    2. Consentimiento informado por escrito firmado, y asentimiento, cuando aplique, de acuerdo con las ICH-GCP y la legislación local, antes de la inclusión en el ensayo.
    3. Pacientes mujeres o hombres. Las mujeres potencialmente fértiles deben confirmar que la abstinencia sexual es su práctica habitual y que será así hasta 3 meses después de la administración del fármaco, o deben estar dispuestas a utilizar métodos anticonceptivos altamente eficaces según la directriz M3(R2) de la ICH, es decir, aquellos con una tasa de fracaso baja, inferior al 1 %, cuando se utilizan correctamente y de forma sistemática así como métodos de barrera durante 28 días antes de y 3 meses después de la administración del tratamiento del ensayo. La abstinencia sexual se define como abstinencia de ningún acto sexual que derive en embarazo.
    4. Pacientes con signos de EPI fibrosante en la TCAR en los 12 meses previos a la visita 1, comprobado por el investigador y confirmado después de revisión de centralizada.
    5. Pacientes con % de CVF predicha ≥25 % en la Visita 2.
    6. Pacientes con enfermedad clínicamente significativa en la Visita 2, comprobada por el investigador en base a lo siguiente: - Puntuación Fan ≥ 3 o - Evidencia documentada de progresión clínica a lo largo del tiempo basada en: o bien una reducción del 5-10% del %CVF predicho acompañado de un empeoramiento de los síntomas, o ≥10% de reducción relativa del %CVF, o fibrosis aumentada en TCAR, u otras medidas de empeoramiento clínico atribuido a la progresión de la enfermedad pulmonar.

    Para los pacientes provenientes del estudio InPedILD™:

    Sólo los criterios 2 y 3 enumerados para los nuevos pacientes son aplicables con el siguiente criterio de inclusión adicional:
    7. Pacientes que completaron el ensayo InPedILD™ como estaba previsto y que no interrumpieron de forma permanente prematura el tratamiento del estudio.

    Para los pacientes que interrumpieron el tratamiento de forma permanente en 1199-0337, pero que son potencialmente elegibles:

    Se aplican los criterios para los pacientes nuevos, excepto los criterios 4 y 6.
    E.4Principal exclusion criteria
    For new patients:
    1. AST and/or ALT >1.5 x ULN at Visit 1.
    2. Bilirubin >1.5 x ULN at Visit 1.
    3. eGFR <30 mL/min calculated by Schwartz formula at Visit 1
    4. Patients with underlying chronic liver disease (Child Pugh A, B or C hepatic impairment) at Visit 1.
    5. Other investigational therapy received within 1 month or 5 half-lives (whichever is shorter but ≥1 week) prior to Visit 2 except investigational therapy received in InPedILD™ trial.
    6. Significant pulmonary arterial hypertension (PAH) defined by any of the following:
    a. Previous clinical or echocardiographic evidence of significant right heart failure
    b. History of right heart catheterization showing a cardiac index ≤2 l/min/m²
    c. PAH requiring parenteral therapy with epoprostenol/treprostinil
    7. In the opinion of the Investigator, other clinically significant pulmonary abnormalities.
    8. Cardiovascular diseases, any of the following:
    a. Severe hypertension, uncontrolled under treatment, within 6 months of Visit 1.
    Uncontrolled hypertension is defined as
    i. In children 6 to ≤12 years old: ≥95th percentile + 12 mm Hg or ≥140/90 mm Hg
    (whichever is lower) (systolic or diastolic blood pressure equal to or greater than
    the calculated target value) (please refer to Appendix 10.5)
    ii. In adolescents 13 to 17 years old: systolic blood pressure ≥140 mm Hg or
    diastolic blood pressure ≥90 mm Hg (please refer to Appendix 10.5)
    b. Myocardial infarction within 6 months of Visit 1
    c. Unstable cardiac angina within 6 months of Visit 1
    9. Bleeding risk, any of the following:
    a. Known genetic predisposition to bleeding
    b. Patients who require
    i. Fibrinolysis, full-dose therapeutic anticoagulation (e.g. vitamin K antagonists, direct thrombin inhibitors, heparin, hirudin)
    ii. High dose antiplatelet therapy
    c. History of haemorrhagic central nervous system (CNS) event within 12 months of Visit 1
    d. Any of the following within 3 months of Visit 1:
    i. Haemoptysis or haematuria
    ii. Active gastro-intestinal (GI) bleeding or GI – ulcers
    iii. Major injury or surgery (investigator’s judgment)
    e. Any of the following coagulation parameters at Visit 1:
    i. International normalized ratio (INR) >2
    ii. Prolongation of prothrombin time (PT) by >1.5 x ULN
    iii. Prolongation of activated partial thromboplastin time (aPTT) by >1.5 x ULN
    10. History of thrombotic event (including stroke and transient ischemic attack) within 12 months of Visit 1.
    11. Known hypersensitivity to the trial medication or its components (i.e. soya lecithin).
    12. Patients with documented allergy to peanut or soya.
    13. Other disease that may interfere with testing procedures or in the judgment of the investigator may interfere with trial participation or may put the patient at risk when participating in this trial.
    14. Life expectancy for any concomitant disease other than ILD <2.5 years (investigator assessment).
    15. Female patients who are pregnant, nursing, or who plan to become pregnant while in the trial.
    16. Patients not able or willing to adhere to trial procedures, including intake of study medication.
    17. Patients who must or wish to take any drug considered likely to interfere with the safe conduct of the trial according to investigator’s benefit-risk assessment for the individual patient
    18. Patients with any diagnosed growth disorder such as growth hormone deficiency or any genetic disorder that is associated with short stature (e.g. Turner Syndrome, Noonan Syndrome, Russell-Silver Syndrome) and/or treatment with growth hormone therapy
    within 6 months before Visit 2. Patients with short stature considered by the investigator to be due to glucocorticoid therapy may be included.
    19. Patients <13.5 kg of weight at Visit 1 (same threshold to be used for male and female patients).

    For roll-over patients from the InPedILD™ study:

    Only criteria 11, 12, 13, 15, 16, 17 and 19, listed for new patients are applicable with the following additional exclusion criterion:

    20. Patient not compliant in parent trial (InPedILD™), with trial medication or trial visits, according to investigator’s judgement. Roll-over patients may qualify for participation even though other exclusion criteria may have been met during the participation in InPedILD™, if the investigator’s benefit-risk assessment for the individual patient remains favourable.

    For patients who discontinued treatment permanently in 1199-0337 but are potentially eligible:

    All exclusion criteria for new patients are applicable with following additional exclusion criterion:
    21. Patients who experienced drug-related adverse events during parent trial leading to permanent study treatment discontinuation.
    Para pacientes nuevos:
    1. AST y/o ALT >1,5 x ULN - Visita 1.
    2. Bilirrubina >1,5 x ULN - Visita 1.
    3. eGFR <30 mL/min calculado mediante la fórmula de Schwartz - Visita 1
    4. Pacientes con enfermedad hepática crónica subyacente - Visita 1.
    5. Haber tomado otro tratamiento en investigación en un 1 mes o 5 semividas (lo que sea más corto, pero ≥1 semana) antes de la Visita 2, excepto el tratamiento del ensayo InPedILD™.
    6. Hipertensión arterial pulmonar (HAP) significativa definida por cualquiera de los siguientes factores:
    a. Evidencia clínica previa o ecocardiográfica de insuficiencia cardiaca derecha significativa
    b. Antecedentes de cateterismo cardíaco derecho con un índice cardíaco ≤2 l/min/m²
    c. HAP con tratamiento parenteral con epoprostenol/treprostinil
    7. En opinión del Investigador, otras anormalidades pulmonares clínicamente significativas.
    8. Enfermedades cardiovasculares, como:
    a. Hipertensión grave, no controlada durante el tratamiento, en los 6 meses antes de la Visita 1.
    La hipertensión no controlada se define como
    i. En niños de 6 a ≤12 años: ≥percentil 95 + 12 mm Hg o ≥ 140/90 mm Hg (el que sea inferior)
    ii. En adolescentes de 13 a 17 años: presión arterial sistólica ≥140 mm Hg o presión arterial diastólica ≥90 mm Hg
    b. Infarto de miocardio en los 6 meses de la Visita 1
    c. Angina cardíaca inestable en los 6 meses de la Visita 1
    9. Riesgo de hemorragia, como:
    a. Predisposición genética
    b. Pacientes que requieren: i. Fibrinólisis, anticoagulación terapéutica a dosis completas; ii. Alta dosis de terapia antiplaquetaria
    c. Antecedentes de episodios hemorrágicos del SNC en los 12 meses de la Visita 1
    d. Cualquiera de los siguientes en los 3 meses siguientes a la Visita 1:
    i. Hemoptisis o hematuria
    ii. Hemorragia gastrointestinal activa o úlceras gastrointestinales
    iii. Lesión mayor o cirugía
    e. Cualquiera de los siguientes parámetros de coagulación en la Visita 1:
    i. Ratio internacional normalizado (INR) >2
    ii. Prolongación del tiempo de protrombina (PT) >1,5 x ULN
    iii. Prolongación del tiempo de tromboplastina parcial activada (aTTP) >1,5 x ULN
    10. Antecedentes de episodios trombóticos (incluyendo accidente cerebrovascular y ataque isquémico transitorio) en los 12 meses de la Visita 1.
    11. Hipersensibilidad conocida al medicamento del ensayo o a sus componentes (p.ej., lecitina de soja).
    12. Pacientes con alergia documentada al cacahuete o a la soja.
    13. Otras enfermedades que puedan interferir con los procedimientos del ensayo o a juicio del investigador puedan interferir con la participación en el ensayo o puedan poner al paciente en riesgo cuando participe en este ensayo.
    14. Esperanza de vida para cualquier enfermedad concomitante distinta de la EPI <2,5 años (evaluación del investigador).
    15. Pacientes femeninas que están embarazadas, en periodo de lactancia, o que planean quedarse embarazadas durante el ensayo.
    16. Pacientes que no pueden o no quieren seguir los procedimientos del ensayo, incluida la toma de medicamentos del ensayo.
    17. Pacientes que deben o desean tomar cualquier medicamento considerado susceptible de interferir con la realización segura del ensayo según la evaluación beneficio-riesgo del investigador para el paciente individual
    18. Pacientes con cualquier trastorno del crecimiento diagnosticado, como deficiencia de la hormona del crecimiento o cualquier trastorno genético que esté asociado con una estatura corta (por ejemplo, síndrome de Turner, síndrome de Noonan, síndrome de Russell-Silver) y/o tratamiento con terapia con hormona del crecimiento dentro de los 6 meses previos a la Visita 2. Los pacientes con estatura corta que el investigador considere que es debido a la terapia con glucocorticoides pueden ser incluidos.
    19. Pacientes de <13,5 kg de peso en la Visita 1 (mismo umbral que se utilizará para pacientes masculinos y femeninos).
    Para los pacientes provenientes del estudio InPedILD™: Sólo son aplicables los criterios 11, 12, 13, 15, 16, 17 y 19 enumerados para los nuevos pacientes, con el siguiente criterio de exclusión adicional:
    20. Paciente que no haya cumplido en el ensayo parental (InPedILD™), con la medicación del ensayo o las visitas del ensayo, según el juicio del investigador. Los pacientes del ensayo parental pueden calificar para la participación en este ensayo aunque otros criterios de exclusión pueden haberse dado durante la participación en InPedILD™, si la evaluación beneficio-riesgo del investigador para el paciente individual sigue siendo favorable.
    Para los pacientes que interrumpieron el tratamiento de forma permanente en 1199-0337, pero que son potencialmente elegibles:
    Todos los criterios de exclusión para los nuevos pacientes son aplicables con el siguiente criterio de exclusión adicional:
    21. Pacientes que experimentaron reacciones adversas relacionadas con el fármaco durante el ensayo parental que condujeron a la interrupción del tratamiento en el estudio de forma permanente.
    E.5 End points
    E.5.1Primary end point(s)
    1) The primary endpoint is the incidence of treatment emergent adverse events over the whole trial.
    1) La variable principal es la incidencia de acontecimientos adversos surgidos a lo largo de todo el ensayo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1) Whole trial duration
    1) Durante todo el ensayo
    E.5.2Secondary end point(s)
    None
    Ninguno
    E.5.2.1Timepoint(s) of evaluation of this end point
    None
    Ninguno
    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 Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 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.8.2.4Number 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    Argentina
    Australia
    Belgium
    Brazil
    Canada
    Czechia
    Denmark
    Finland
    France
    Germany
    Greece
    Hungary
    Italy
    Mexico
    Netherlands
    Norway
    Poland
    Portugal
    Russian Federation
    Spain
    Ukraine
    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
    LVLS
    Última visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days25
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days1
    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: 60
    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: 18
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 38
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 4
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 60
    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)
    None
    Ninguno
    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 Decision2021-12-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-22
    P. End of Trial
    P.End of Trial StatusOngoing
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