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    Summary
    EudraCT Number:2019-002113-19
    Sponsor's Protocol Code Number:42756493CAN2002
    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:2019-11-06
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2019-002113-19
    A.3Full title of the trial
    A Phase 2 Study of Erdafitinib in Subjects with Advanced Solid Tumors and FGFR Gene Alterations
    Estudio fase 2 de erdafitinib en sujetos con tumores sólidos avanzados y alteraciones en el gen FGFR
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2 Study of Erdafitinib in Subjects with Advanced Solid Tumors and FGFR Gene Alterations
    Estudio fase 2 de erdafitinib en sujetos con tumores sólidos avanzados y alteraciones en el gen FGFR
    A.4.1Sponsor's protocol code number42756493CAN2002
    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 SponsorJanssen-Cilag International NV
    B.1.3.4CountryBelgium
    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 supportJanssen Research & Development LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJanssen-Cilag International NV
    B.5.2Functional name of contact pointGlobal Clinical Operations Spain
    B.5.3 Address:
    B.5.3.1Street AddressPaseo de las Doce Estrellas, 5- 7
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28042
    B.5.3.4CountrySpain
    B.5.4Telephone number+31917226532
    B.5.5Fax number+31917228628
    B.5.6E-mailgbadenes@its.jnj.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 nameErdafitinib
    D.3.2Product code JNJ-42756493
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNErdafitinib
    D.3.9.1CAS number 1346242-81-6
    D.3.9.2Current sponsor codeJNJ-42756493
    D.3.9.3Other descriptive nameERDAFITINIB
    D.3.9.4EV Substance CodeSUB167731
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3
    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 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 nameErdafitinib
    D.3.2Product code JNJ-42756493
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNErdafitinib
    D.3.9.1CAS number 1346242-81-6
    D.3.9.2Current sponsor codeJNJ-42756493
    D.3.9.3Other descriptive nameERDAFITINIB
    D.3.9.4EV Substance CodeSUB167731
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    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 nameErdafitinib
    D.3.2Product code JNJ-42756493
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNErdafitinib
    D.3.9.1CAS number 1346242-81-6
    D.3.9.2Current sponsor codeJNJ-42756493
    D.3.9.3Other descriptive nameERDAFITINIB
    D.3.9.4EV Substance CodeSUB167731
    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 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
    Advanced solid tumors (other than Urothelial tumors), and FGFR gene alterations.
    Tumores sólidos avanzados (distintos de los tumores uroteliales), y alteraciones del gen FGFR
    E.1.1.1Medical condition in easily understood language
    Advanced solid tumors (other than Urothelial tumors), and FGFR gene alterations.
    Tumores sólidos avanzados (distintos de los tumores uroteliales), y alteraciones del gen FGFR
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10065143
    E.1.2Term Malignant solid tumour
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of erdafitinib in terms of overall response rate (ORR) as assessed by the Independent Review Committee (IRC) in subjects with advanced solid tumors with target FGFR mutations and any gene fusions (Broad Panel Cohort), or in a pre-specified subgroup of subjects with a selected panel of FGFR markers (Core Panel Cohort), or in both cohorts
    Evaluar la eficacia de erdafitinib en cuanto a la tasa de respuesta global (TRG) evaluada por el Comité Independiente de revisión (IRC) en sujetos con tumores sólidos avanzados con mutaciones seleccionadas del FGFR y cualquier fusión génica (cohorte del panel de marcadores seleccionados), o bien en un subgrupo previamente especificado de sujetos con un perfil seleccionado de marcadores de FGFR (cohorte del panel de marcadores central) o en ambas cohortes.
    E.2.2Secondary objectives of the trial
    - To evaluate the efficacy of erdafitinib, in terms of the ORR, as assessed by investigator
    - To evaluate the efficacy of erdafitinib in terms of DOR
    - To evaluate other measures of efficacy including DCR, PFS, and OS
    - To evaluate erdafitinib PK
    - To evaluate Health-Related Quality of Life
    - Evaluar la eficacia de erdafitinib, en términos de TRG, según lo evaluado por el investigador
    - Evaluar la eficacia de erdafitinib en cuanto a la duración de la respuesta (DR)
    - Evaluar otras mediciones de eficacia, incluidas la tasa de control de la enfermedad (TCE), la supervivencia libre de progresión (SLP) y la supervivencia global (SG)
    - Evaluar la farmacocinética (FC) de erdafitinib
    - Evaluar la seguridad y tolerabilidad de erdafitinib
    - Evaluar la calidad de vida (CdV) relacionada con la salud
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. ≥12 years of age
    2. Criterion amended per Amendment 1
    2.1 Histologic demonstration of an unresectable, locally advanced, or metastatic solid tumor malignancy bearing an FGFR mutation or fusion, as determined by local* or central laboratory screening (Section 8.1.1.1). Note that central creening for breast cancer is limited to women with hormone-sensitive (ie, estrogen positive [ER]/progesterone positive [PR]) breast cancer
    *Subjects with target FGFR mutations or any** FGFR gene fusions are eligible for enrollment in the Broad Panel Cohort (The List of Target FGFR Mutations is provided in Section 10.11).
    *Subjects with other FGFR mutations not captured in the Broad Panel Cohort are eligible for enrollment in the Exploratory Cohort
    *local testing from tissue or blood with NGS tests performed in Clinical laboratory Improvement Amendments (CLIA)-certified or equivalent laboratories or results from commercially available NGS tests
    ** FGFR gene fusions must have an intact FGFR kinase domain (include exon 16, 17, or 18). FGFR gene identifiers are provided below for reference (see protocol).
    3 Measurable disease according to RECIST v1.1 or RANO for primary brain tumors
    4. Subject must have received at least one prior line of systemic therapy in the metastatic setting
    5. Subject does not have standard of care options that have shown meaningful clinical benefit for the relevant underlying histology and line of therapy or the subject is unable to tolerate the therapy
    6. Documented progression of disease, defined as any progression that requires a change in treatment, prior to full study screening
    7. Toxicities from previous anticancer therapies should have resolved to baseline levels or to Grade 1 or less except for alopecia, peripheral neuropathy, and Grade 2 laboratory values eligible per Inclusion Criterion 9.
    8. For adults (≥18 years of age), ECOG performance status Grade 0 or 1 (Section 10.5)
    For adolescents (≥12 to <16 years of age), Lansky Score of ≥80 (Section 10.6).
    For adolescents (≥16 to <18 years of age), Karnofsky Score of ≥80 (Section 10.6).
    9. Criterion amended per Amendment 1
    9.1 Adequate bone marrow, liver, and renal function:
    a. Bone marrow function (without the support of cytokines or erythropoiesis-stimulating agent in preceding 2 weeks):
    -Asolute neutrophil count (ANC) ≥1,000/mm3
    -Patelet count ≥75,000/mm3
    -Hemoglobin ≥8.0 g/dL
    b. Liver function:
    -Total bilirubin ≤1.5 x institutional ULN OR direct bilirubin ≤ULN for subjects with total bilirubin levels >1.5xULN
    -Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5x nstitutional ULN or ≤5x institutional ULN for subjects with liver metastases
    c. Renal function: Creatinine clearance >30 mL/min/1.73m2 either directly measured via 24-hour urine collection or calculated using the Cockcroft-Gault formula for adult subjects or the CKiD (Chronic Kidney Disease in Children)Schwartz formula for adolescent subjects (Section 10.7)
    d. Phosphate: <ULN within 14 days of treatment and prior to Cycle 1 Day 1 medical management allowed)
    10. Must sign an ICF (or their legally acceptable representative must sign)idicating that he or she understands the nature, significance, and purpose of the study, and procedures required for the study, and consequence of the study; and is willing to participate in the study. For adolescent subjects, parent(s) (preferably both if available or as per local requirements) (or their legally acceptable representative) must sign an ICF indicating that he or she understands the purpose of, and procedures required for, the study and is willing to allow the child to participate in the study. Assent is also required of adolescent subjects as described in Informed Consent Process in Section 10.3
    Regulatory, Ethical, and Study Oversight Considerations.
    11. A female of childbearing potential must have a negative pregnancy test (β-human chorionic gonadotropin [hCG]) at Screening (urine or serum).
    12. Criterion amended per Amendment 1
    12.1 Contraceptive use by male or female subjects should be consistent with local regulations regarding the use of contraceptive methods for subject participating in clinical studies. For females of childbearing potential (defined as: fertile, following menarche and until becoming post-menopausal unless permanently sterile [for the purpose of this study]. Permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy). Please refer to protocol section 5.1 for continuation of criterium 12
    1.≥12 años de edad
    2.Criterio modificado según la enmienda 1
    2.1Demostración histológica de un tumor sólido maligno no resecable, localmente avanzado o metastásico con una mutación o fusión en el FGFR, según se determine mediante selección por el lab. local* o central (apdo 8.1.1.1).Téngase en cuenta que la selección centralizada para el cáncer de mama se limita a las mujeres con cáncer de mama hormonosensible (es decir, positivo en estrógenos [ER]/positivo en progesterona [PR]).
    •Los sujetos con mutaciones seleccionadas del FGFR o cualquier** fusión de genes FGFR son elegibles para su inclusión en la cohorte del panel de marcadores seleccionados (la Lista de Mutaciones del FGFR seleccionadas se proporciona en apdo. 10.11).
    •Los sujetos con cualquier otra mutación del FGFR que no formen parte de la cohorte del panel de marcadores seleccionados son elegibles para su inclusión en la cohorte exploratoria.
    *Pruebas locales de sangre o tejido con pruebas NGS realizadas en labs certificados por Clinical Laboratory Improvement Amendments (CLIA) o equivalentes o bien resultados de pruebas NGS disponibles en el mercado.
    ** Las fusiones de genes FGFR debe tener un dominio de quinasa FGFR intacto (incluye el exón 16, 17, o 18). Se proporcionan identificadores del gen FGFR como referencia (ver protocol)
    Enfermedad medible según RECIST v1.1 o RANO para tumores cerebrales primarios.
    4.El sujeto debe haber recibido al menos una línea anterior de tmto sistémico para enfermedad metastásica.
    5.El sujeto no tiene opciones de tmto de referencia que hayan mostrado un beneficio clínico significativo para la histología subyacente y la línea de tmto pertinentes, o el sujeto es incapaz de tolerar el tmto.
    6.Progresión documentada de la enfermedad, definida como cualquier progresión que requiera un cambio en el tmto, antes del periodo de selección completo.
    7.La toxicidad del tmto antineoplásico anterior debe haber vuelto a los niveles iniciales o haber disminuido hasta el grado 1 o menos, salvo la alopecia y la neuropatía periférica, y valores de laboratorio de grado 2 son admisibles de acuerdo al criterio de inclusión 9.
    8.En adultos (≥18 años de edad), estado funcional del ECOG de grado 0 o 1 (apdo 10.5).
    En adolescentes (de ≥12 a <16 años), puntuación de Lansky ≥80 (apdo 10.6).
    En adolescentes (de ≥16 a <18 años), puntuación de Karnofsky ≥80 (apdo 10.6).
    9.Criterio modificado según la enmienda 1
    9.1Función renal, hepática y de la médula ósea adecuadas:
    a.Función de la médula ósea (sin el apoyo de citoquinas o de un fármaco estimulador de la eritropoyesis en las 2sms anteriores):
    –Recuento absoluto de neutrófilos (RAN) ≥1000/mm3Recuento plaquetario ≥75 000/mm3Hemoglobina ≥8,0 g/dl
    b. Función hepática:
    –Bilirrubina total ≤1,5 veces el LSN institucional O bilirrubina directa ≤LSN en sujetos con concentraciónde bilirrubina total >1,5 veces el LSN
    ALT y AST ≤2,5 veces el LSN institucional o ≤5 veces el LSN institucional en sujetos con metástasis hepáticas
    c.Función renal: aclaramiento de creatinina >30 ml/min/1.73m2, medida directamente mediante la recogida de orina de 24horas o calculada usando la fórmula de Cockcroft-Gault para sujetos adultos o la fórmula CKiD (Enfermedad Renal Crónica en Niños) Schwartz para adolescentes (apdo. 10.7)
    d.Fosfato: <LSN en los 14 días del tmto y antes del día 1 del ciclo 1 (se permite el tmto médico)
    10.Debe firmar el Formulario de CI (o su representante legal debe firmarlo) indicando que entiende la naturaleza, significado y propósito del estudio, los procedimientos requeridos y las consecuencias de este, y está dispuesto a participar en el estudio. En el caso de sujetos adolescentes, los progenitores (preferiblemente ambos si están disponibles o según los requisitos locales) (o su representante legal) deben firmar un CI indicando que entienden el propósito del estudio y los procedimientos requeridos para este, y que están dispuestos a permitir que el niño participe en el estudio.También se requiere la aceptación de los sujetos adolescentes como se describe en el proceso de CI o en el apdo 10.3, consideraciones reglamentarias, éticas y de supervisión del estudio.
    11.Las mujeres en edad fértil debe tener un resultado negativo en una prueba de embarazo (gonadotropina coriónica humana [hCG]) en el momento de la selección (orina o suero).
    12.Criterio modificado según la enmienda 1
    12.1El uso de anticonceptivos por los sujetos de sexo masculino o femenino debe ser congruente con las normativas locales respecto al uso de métodos anticonceptivos en sujetos que participan en estudios clínicos.Para las mujeres con potencial de maternidad (definidas como: fértiles, después de la menarquia y hasta llegar a ser postmenopáusicas, a menos que sean permanentemente estériles [para los fines de este estudio]). Los métodos de esterilización permanente incluyen histerectomía, salpingectomía bilateral y ooforectomía bilateral). Véase el apdo 5.1 del protocolo para continuación del criterio 12
    E.4Principal exclusion criteria
    1. Has had prior chemotherapy, targeted therapy, or treatment with an investigational anticancer agent within 30 days or ≤5 half-lives of the agent (whichever is longer) before the first dose of erdafitinib. Has had prior immunotherapy within 30 days before the first dose of erdafitinib and/or has an ongoing Grade ≥2 immunotherapyrelated toxicity
    2. The presence of FGFR gatekeeper and resistance alterations. Mutations in the following positions: FGFR1 V561; FGFR2 V564; FGFR2 N549; FGFR3 V555; and FGFR4 V550
    3. Criterion amended per Amendment 1
    3.1 For NSCLC subjects only - pathogenic somatic mutations or gene fusions in the following genes: EGFR, ALK, ROS1, NTRK and BRAF V600E (PIK3CA and PTEN alteration status must be known at the time of enrollment for retrospective data analysis)
    4. Histologic demonstration of transitional cell carcinoma of the urothelium (ie, bladder cancer)
    5. Hematologic malignancy (ie, myeloid and lymphoid neoplasms)
    6. Active malignancies other than for disease requiring therapy
    7. Symptomatic central nervous system metastases (except for subjects with primary CNS tumors)
    8. Received prior FGFR inhibitor treatment
    9. Known allergies, hypersensitivity, or intolerance to erdafitinib or its excipients.
    10. Current central serous retinopathy (CSR) or retinal pigment epithelial detachment of any grade.
    11. Criterion amended per Amendment 1
    11.1 History of uncontrolled cardiovascular disease include:
    -Unstable angina, myocardial infarction, ventricular fibrillation, Torsades de Pointes, cardiac arrest, or known congestive heart failure Class III-V (Section 10.8) within the preceding 3 months; cerebrovascular accident or transient ischemic attack within the preceding 3 months.
    -QTc prolongation (Fridericia: QTc >480 milliseconds; or for adolescent subjects, Bazett: QTc >440 milliseconds)
    12. Known history of AIDS (human immunodeficiency virus (HIV) infection), unless the subject has been on a stable anti-retroviral therapy regimen for the last 6 months or more, has had no opportunistic infections in the last 6 months, and has CD4 count >350.
    13. Criterion amended per Amendment 1
    13.1 Evidence of active hepatitis B or C infection (for example, subjects with history of hepatitis C infection but normal hepatitis C virus polymerase chain reaction test and subjects with hepatitis B with positive HBsAg antibody are allowed)
    14. Not recovered from reversible toxicity of prior anticancer therapy (except toxicities which are not clinically significant such as alopecia, skin discoloration, neuropathy, hearing loss).
    15. Impaired wound healing capacity defined as skin/decubitus ulcers, chronic leg ulcers, known gastric ulcers, or unhealed incisions.
    16. Major surgery within 4 weeks before first dose of erdafitinib.
    17. Palliative radiation within 2 weeks before the first dose of erdafitinib.
    18. Pregnant, or breast-feeding, or planning to become pregnant while enrolled in this study or within 3 months after the last dose of drug.
    19. Plans to father a child while enrolled in this study or within 3 months after the last dose of drug.
    20. Any condition for which, in the opinion of the investigator, participation would not be in the best interest of the subject (eg, compromise the well-being) or that could prevent, limit, or confound the protocol-specified assessments. Examples include ongoing active infection requiring systemic therapy and uncontrolled ongoing medical conditions.
    1. Haber recibido quimioterapia previa, tratamiento dirigido o tratamiento con un fármaco anticancerígeno en investigación en un plazo de 30 días o un periodo de semidesintegración del fármaco ≤5 semividas (el que sea más largo) antes de la primera dosis de erdafitinib. Haber recibido inmunoterapia previa en los 30 días anteriores a la primera dosis de erdafitinib o presentar una toxicidad relacionada con la inmunoterapia de grado ≥2 en curso.
    2. La presencia de alteraciones de resistencia y sincronización de FGFR. Mutaciones en las siguientes posiciones: FGFR1 V561; FGFR2 V564; FGFR2 N549; FGFR3 V555; y FGFR4 V550.
    3. Criterio modificado según la enmienda 1
    3.1 Solo para sujetos con CPCNP - mutaciones somáticas patogénicas o fusiones génicas en los siguientes genes: EGFR, ALK, ROS1, NTRK y BRAF V600E (el estado de modificación de PIK3CA y PTEN debe conocerse en el momento de la inclusión para el análisis de datos retrospectivo).
    4. Demostración histológica de carcinoma de células transicionales del urotelio (es decir, cáncer de vejiga).
    5. Neoplasias malignas hematológicas (es decir, neoplasias mieloides y linfoides).
    6. Neoplasias activas diferentes a la enfermedad a tratar. .
    7. Metástasis sintomáticas del sistema nervioso central (excepto en sujetos con tumores primarios del SNC).
    8. Haber recibido tratamiento previo con inhibidores del FGFR.
    9. Alergias, hipersensibilidad o intolerancia conocidas a erdafitinib o a sus excipientes.
    10. Retinopatía serosa central (RSC) actual o desprendimiento epitelial de pigmento de la retina de cualquier grado.
    11. Criterio modificado según la enmienda 1
    11.1 Antecedentes de enfermedad cardiovascular no controlada, entre las que se incluyen:
    • Angina inestable, infarto de miocardio, fibrilación ventricular, Torsade de Pointes, paro cardíaco o insuficiencia cardíaca congestiva conocida de clase III-V (apartado 10.8) en los 3 meses anteriores; accidente cerebrovascular o ataque isquémico transitorio en los 3 meses anteriores.
    • QTc (Fridericia; QTc >480 milisegundos; o para sujetos adolescentes, Bazett: QTc >440 milisegundos))).
    12. Antecedentes conocidos de SIDA (infección por el virus de la inmunodeficiencia humana [VIH]), a menos que el sujeto haya recibido una pauta de terapia antirretroviral estable durante los últimos 6 meses o más, no haya tenido infecciones oportunistas en los últimos 6 meses y tenga un recuento de CD4 >350.
    13. Criterio modificado según la enmienda 1
    13.1 Evidencia de hepatitis B o C activa (por ejemplo, se permiten sujetos con antecedentes de infección por hepatitis C pero con una prueba normal de reacción en cadena de la polimerasa del virus de la hepatitis C y sujetos con hepatitis B con anticuerpos HBsAg positivos).
    14. No haberse recuperado de la toxicidad reversible de tratamientos anteriores contra el cáncer (excepto las toxicidades que no son clínicamente significativas como la alopecia, la decoloración de la piel, la neuropatía y la pérdida de audición).
    15. Deterioro de la capacidad de cicatrización de heridas definida como úlceras de piel/decúbito, úlceras crónicas en las piernas, úlceras gástricas conocidas o incisiones no cicatrizadas.
    16. Cirugía mayor en las 4 semanas anteriores a la primera dosis de erdafitinib.
    17. Radiación paliativa en las 2 semanas anteriores a la primera dosis de erdafitinib.
    18. Embarazo o lactancia materna, o tener intención de quedarse embarazada durante la participación en este estudio o antes de que pasen 3 meses tras la última dosis del fármaco.
    19. Tener intención de engendrar un hijo durante la participación en este estudio o antes de que pasen 3 meses tras la última dosis del fármaco del estudio.
    20. Cualquier enfermedad para la cual, en opinión del investigador, la participación no redunde en el mejor interés del sujeto (por ejemplo, que ponga en peligro su bienestar) o que pudiera evitar, limitar o confundir las evaluaciones especificadas en el protocolo. Los ejemplos incluyen infección activa en curso que requiera tratamiento sistémico y afecciones médicas en curso no controladas
    E.5 End points
    E.5.1Primary end point(s)
    The proportion of subjects who achieve a complete response (CR) or partial response (PR) based on RECIST v1.1. or RANO as assessed by IRC
    La proporción de sujetos que alcanzan una respuesta completa (RC) o respuesta parcial (RP) basada en RECIST v1.1 o RANO según lo evaluado por el IRC
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the study
    A lo largo del estudio
    E.5.2Secondary end point(s)
    - The proportion of subjects who achieve a CR or PR based on RECIST v1.1. or RANO as assessed by investigator
    - DOR: the duration from the date of initial documentation of a response to the date of first documented evidence of progressive disease (or relapse for subjects who experience CR during the study) or death, whichever comes first. Data from subjects who are progression-free and alive or have unknown status will be censored at the last tumor assessment
    - DCR: the proportion of subjects with CR, PR or SD
    - PFS: the duration from the date of the first dose of study drug until the date of first documented evidence of progressive disease (or relapse for subjects who experience CR during the study) or death, whichever comes first. Data from subjects who are progression-free and alive or have unknown status will be censored at the last tumor assessment
    - OS: measured from the date of first dose of study drug to the date of the subject’s death. If the subject is alive or the vital status is unknown, the subject’s data will be censored at the date the subject was last known to be alive
    - PK exposure parameters derived using existing population PK model
    - Incidence and severity of AEs
    - Change from baseline in patient-reported health status and physical functioning scales of the EORTC-QLQ-C30, PGIS, PGIC, and EQ-5D-5L.
    - La proporción de sujetos que alcanzan una RC o RP basado en RECIST v1.1. o RANO según lo evaluado por el investigador
    - Duración de la Respuesta: la duración desde la fecha de la documentación inicial de una respuesta hasta la fecha de la primera evidencia documentada de enfermedad progresiva (o recaída en sujetos que experimentan RC durante el estudio) o muerte, lo que ocurra primero. Los datos de los sujetos que están libres de progresión y vivos o tienen un estado desconocido serán censurados en la última evaluación del tumor
    - Tasa de Control de la Enfermedad: la proporción de sujetos con RC, RP o enfermedad estable
    - Supervivencia libre de progresión: la duración desde la fecha de la primera dosis del fármaco del estudio hasta la fecha de la primera evidencia documentada de enfermedad progresiva (o recaída en sujetos que experimentan RC durante el estudio) o muerte, lo que ocurra primero. Los datos de los sujetos que están libres de progresión y vivos o tienen un estado desconocido serán censurados en la última evaluación del tumor
    SG: medido desde la fecha de la primera dosis del fármaco del estudio hasta la fecha de la muerte del sujeto. Si el sujeto está vivo o se desconoce su estado vital, los datos del sujeto serán censurados en la fecha en que se supo que el sujeto estaba vivo por última vez
    - Parámetros de exposición FC derivados del uso del modelo FC de población existente
    - Incidencia y gravedad de los AAs
    - Cambio desde el inicio en el estado de salud informado por el paciente y las escalas de funcionamiento físico de EORTC-QLQ-C30, PGIS, PGIC y EQ-5D-5L.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study
    A lo largo del estudio
    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 Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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 Yes
    E.6.13.1Other scope of the trial description
    Biomarkers, PROs, Medical Resource Utilization
    Biomarcadores, Cuestionarios de paciente, Utilización de recursos médicos
    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 concerned10
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA65
    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
    China
    France
    Germany
    Italy
    Japan
    Korea, Republic of
    Poland
    Spain
    Taiwan
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The end of study is defined as 4 years from enrollment of the last subject into the study or the last follow-up visit of the last patient, whichever occurs first.
    El final del estudio se define como 4 años desde el reclutamiento del último sujeto en el estudio o la última visita de seguimiento del último paciente, lo que ocurra primero.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months0
    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: 20
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 190
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 70
    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
    For adolescents between 12-17 yrs old
    Para adolescentes entre 12 - 17 años de edad
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state33
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 114
    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)
    The sponsor will ensure that subjects benefiting from treatment with erdafitinib continue to receive treatment after the end of the study. Subsequent therapy is left to the investigator's discretion.
    El promotor asegurará que los sujetos beneficiados del tratamiento con erdafitinib continúan recibiendo tratamiento después del final del estudio. La terapia posterior se deja a discreción del investigador.
    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 Decision2019-11-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-09-30
    P. End of Trial
    P.End of Trial StatusOngoing
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