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    Summary
    EudraCT Number:2012-001571-36
    Sponsor's Protocol Code Number:GS-US-322-0207
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2013-05-16
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2012-001571-36
    A.3Full title of the trial
    A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Assess the Efficacy and Safety of GS-6624 in Subjects with Idiopathic Pulmonary Fibrosis (RAINIER)
    Estudio de fase II, multicéntrico, aleatorizado, doble ciego, controlado con placebo, para evaluar la eficacia y la seguridad de GS 6624 en pacientes con fibrosis pulmonar idiopática (RAINIER)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate if a new drug is effective in preventing the progression of lung fibrosis
    Un estudio para evaluar si un nuevo medicamento es eficaz en la prevención de la progresión de la fibrosis pulmonar
    A.3.2Name or abbreviated title of the trial where available
    RAINER
    A.4.1Sponsor's protocol code numberGS-US-322-0207
    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 SponsorGilead Sciences, 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 supportGilead Sciences Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGilead Sciences Inc.
    B.5.2Functional name of contact pointMedical Monitor
    B.5.3 Address:
    B.5.3.1Street Address333 Lakeside Drive
    B.5.3.2Town/ cityFoster City, CA
    B.5.3.3Post code94404
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16505224707
    B.5.5Fax number+18664541397
    B.5.6E-mailjason.chien@gilead.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.3.2Product code GS-6624
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSimtuzumab
    D.3.9.1CAS number 1318075-13-6
    D.3.9.2Current sponsor codeGS-6624
    D.3.9.3Other descriptive nameGS-6624
    D.3.9.4EV Substance CodeSUB33243
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Idiopathic pulmonary fibrosis
    Fibrosis pulmonar idiopática
    E.1.1.1Medical condition in easily understood language
    Lung fibrosis of unknown cause
    Fibrosis pulmonar de origen desconocido
    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 16.0
    E.1.2Level PT
    E.1.2Classification code 10021240
    E.1.2Term Idiopathic pulmonary fibrosis
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is:
    - To determine the effect of GS-6624 on progression free survival
    (PFS) as determined by either a categorical decline in forced
    vital capacity (FVC) or all-cause mortality
    El objetivo principal de este estudio es:
    - Determinar el efecto de GS-6624 en la supervivencia sin progresión (SSP) determinado por una disminución categórica en la capacitad vital forzada (FVC) o la mortalidad por todas las causas.
    E.2.2Secondary objectives of the trial
    - Effects of GS-6624 on all-cause mortality
    - Predictive effects of baseline serum lysyl oxidase like 2
    (sLOXL2) levels
    - Effects of GS-6624 on change in lung function
    - Effects of GS-6624 on adjudicated acute exacerbations
    - Effects of GS-6624 on adjudicated respiratory hospitalizations
    - Effects of GS-6624 on 6-minute walk distance (6MWD)
    - Effects of GS-6624 on quality of life
    - The safety and tolerability of GS-6624

    Exploratory objectives include evaluation of:
    - Effect of GS-6624 on degree of fibrosis on high resolution
    computed tomography (HRCT) scans
    - Effect of GS-6624 on sLOXL2 levels compared to placebo
    - Prognostic effect of baseline sLOXL2 on primary and
    secondary objectives (among placebo treated subjects only)
    - Predictive effect of baseline sLOXL2 on the secondary
    objectives and subsequent sLOXL2 levels
    - Los efectos de GS-6624 en la mortalidad por todas las causas
    - Efectos predictivos de los niveles basales de la lisil oxidasa like 2 sérica (LOXL2s)
    - Efectos de GS-6624 sobre cambios en la función pulmonar
    - Efectos de GS-6624 sobre exacerbaciones agudas adjudicadas
    - Efectos de GS-6624 sobre hospitalizaciones por causas respiratorias adjudicadas
    - Efectos de GS-6624 sobre la prueba de la distancia caminada en 6 minutos (6MWD)
    - Efectos de GS-6624 sobre la calidad de vida
    - La seguridad y tolerabilidad de GS-6624

    Los objetivos exploratorios incluyen la evaluación de:
    - Efecto de GS-6624 sobre el grado de fibrosis en la tomografía computarizada de alta resolución (TCAR)
    - Efecto de GS-6624 sobre los niveles de LOXL2s en comparación con placebo
    - Efecto pronóstico de LOXL2s basal sobre los objetivos principal y secundarios (únicamente en pacientes tratados con placebo)
    - Efecto predictivo de LOXL2s basal sobre los objetivos secundarios y niveles posteriores de LOXL2s
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female subjects from 45 to 85 years of age
    2. Diagnosis of definite IPF according to American Thoracic
    Society/European Respiratory Society (ATS/ERS) Consensus Statement,
    using HRCT or surgical lung biopsy (SLB) (both to be confirmed by a
    central service); the diagnosis must be made within 3 years prior to
    screening
    3. 6MWD ? 50 meters (164 feet): use of ? 6 L/min supplemental O2 is
    required for all subjects
    4. Able to maintain oxygen saturation of ? 89% while breathing room air
    at rest
    5. Able to perform complete breath hold for diffusing lung capacity for
    carbon monoxide (DLCO) measurement
    6. Negative serum pregnancy test at screening and negative urine
    pregnancy test at randomization for female subjects of childbearing
    potential
    7. Willingness of female subjects of childbearing potential to undergo
    urine pregnancy tests every 28 days
    8. Females of childbearing potential must agree to use highly effective
    methods of contraception from the screening visit throughout the study
    period and for 90 days following the last dose of IMP. Please refer to
    Section 8.11.2 for protocol recommended methods of contraception;
    females of childbearing potential must have negative serum ?-hCG at
    screening.
    9. Non-vasectomized male subjects must agree to use a highly effective
    method of contraception if sexually active and refrain from sperm
    donation from Day 1 throughout the study period
    until 90 days following the last dose of investigational medicinal product
    (IMP)
    10. Lactating females must agree to discontinue nursing before enrolling
    in the study and for the duration of the study while receiving IMP
    11. Competency to understand the information given in the Institutional
    Review Board (IRB) or Independent Ethics Committee (IEC) approved
    Informed Consent Form (ICF); subjects must sign the ICF prior to the
    initiation of any study procedures/assessments, unless it is performed
    as standard of care for this disease
    1.Hombres o mujeres con edades entre 45 y 85 años
    2.Diagnóstico confirmado de FPI de acuerdo a la Declaración de Consenso de la Sociedad Torácica Norteamericana/Sociedad Europea de Enfermedades Respiratorias (ATS/ERS - American Thoracic Society/European Respiratory Society), mediante TCAR o biopsia pulmonar quirúrgica (BPQ) (debiendo ambas ser confirmadas por un servicio central); el diagnóstico debe haberse efectuado en los 3 años previos a la selección
    3.6MWD ? 50 metros (164 pies): es necesario que el uso de O2 suplementario sea ? 6 l/min en todos los pacientes
    4.Capacidad para mantener una saturación de oxígeno ? 89% al respirar aire ambiental en reposo
    5.Capacidad para realizar una retención completa de aire para determinar la capacidad de difusión pulmonar del monóxido de carbono (DLCO)
    6.Prueba de embarazo en suero negativa en la selección y prueba de embarazo en orina negativa en la aleatorización para pacientes mujeres en condiciones de quedar embarazadas
    7.Disposición de las pacientes en condiciones de quedar embarazadas a someterse a pruebas de embarazo en orina cada 28 días
    8.Las mujeres en condiciones de quedar embarazadas deben aceptar utilizar métodos anticonceptivos altamente efectivos a partir de la visita de selección, durante todo el periodo del estudio y en los 90 días posteriores a la última dosis del IMP. Por favor, refiérase al Apartado 8.11.2 sobre los métodos anticonceptivos recomendados en el protocolo; las mujeres en condiciones de quedar embarazadas deben tener una prueba de ? hCG sérica negativa en la selección
    9.Los pacientes varones que no se han sometido a una vasectomía deben aceptar utilizar un método anticonceptivo altamente efectivo si son sexualmente activos y abstenerse de donar esperma desde el Día 1, durante todo el periodo del estudio y hasta los 90 días posteriores a la última dosis del medicamento en investigación (IMP)
    10.Las mujeres en periodo de lactancia deben aceptar dejar de amamantar antes de ser incluidas en el estudio y a lo largo del estudio mientras reciban el IMP
    11.Capacidad para comprender la información proporcionada en el Documento de Consentimiento Informado (DCI) aprobado por el Comité Ético de Investigación Clínica (CEIC); los pacientes deben firmar el DCI antes de que se inicie cualquier procedimiento/evaluación del estudio, salvo que éstos se realicen como parte del tratamiento estándar para la enfermedad
    E.4Principal exclusion criteria
    Related to other medical conditions or diagnoses:
    1. Pregnant or breastfeeding
    2. Clinically significant respiratory diseases other than IPF, including
    asbestosis, other pneumoconiosis or hypersensitivity pneumonitis
    3. Obstructive lung disease determined by Pulmonary Function Test
    (PFTs) or HRCT as follows:
    ? Evidence of reactive airway disease by absolute increase in forced
    expiratory volume in 1 second (FEV1) of > 12% following bronchodilator
    challenge OR
    ? FEV1/FVC ratio < age adjusted lower limit of normal (LLN) OR
    ? Residual volume (RV) > 120% by plethysmography or significant
    emphysema on HRCT defined as more emphysema than fibrosis
    interpreted by a central radiology service
    4. Hemoglobin corrected, not volume corrected, DLCO <25% of predicted
    normal
    5. Surgical lung biopsy (SLB) showing, pneumoconiosis, hypersensitivity
    pneumonitis nonspecific pneumonia or other idiopathic interstitial lung
    disease
    6. Any collagen vascular disease
    7. History of aortic aneurysm ? 3.5cm in diameter
    8. History of cerebrovascular accident (stroke) within the preceding 26
    weeks
    9. Clinically significant heart disease defined as a myocardial infarction
    documented by an ST elevation (STEMI) on electrocardiogram (ECG)
    within 6 months prior to screening, percutaneous coronary intervention
    or coronary artery bypass surgery within 6 months prior to screening,
    unstable angina pectoris, congestive heart failure (NYHA class III/IV or known left ventricular ejection fraction < 25%), right heart failure, significant right ventricular hypertrophy, or uncontrolled arrhythmia
    10. Require pharmaceutical treatment for pulmonary hypertension
    11. Hospitalization for acute respiratory illness < 26 weeks prior to
    screening
    12. Aspiration pneumonia < 26 weeks prior to screening
    13. Active or recent (< 4 weeks prior to enrollment) respiratory
    bacterial, viral, fungal, or other infection (defined as exhibiting ongoing
    signs/symptoms related to the infection and without improvement)
    14. History of cancer, precancerous state (eg, familial polyposis, BRCA1,
    BRCA2), other than non-melanomatous skin cancer, within 5 years prior
    to screening, or active work-up for suspected lung cancer
    15. History of human immunodeficiency virus (HIV), hepatitis C, or
    hepatitis B
    16. Co-morbid condition or illness limiting life expectancy to < 2 years at
    time of screening
    17. Major surgery, as defined by the investigator, < 30 days prior to
    screening or scheduled elective surgery during the expected duration of
    the study
    18. History or evidence of a clinically significant disorder, condition, or
    disease that, in the opinion of the investigator and the Gilead medical
    monitor, would pose a risk to subject safety or interfere with the study
    evaluations, procedures, or completion
    19. Current excessive (as determined by the investigator) consumption
    of alcohol or use of illegal drugs Related to physiologic or organ specific
    abnormalities:
    20. Use of supplemental O2 > 6L/min during activity
    21. Inadequate organ function reflected by any of the following:
    ? Platelets < 100 x 109/L
    ? Hemoglobin < 11.0 g/dL
    ? Absolute Neutrophil Count (ANC) < 1.5 x 109/L
    ? PT/INR and PTT > 1.5 x upper limit of normal (ULN)
    ? AST/ALT > 3 x ULN
    ? Total bilirubin > 1.5 x ULN
    ? Serum creatinine > 2.0 mg/dL
    Related to treatment for IPF or investigational drugs:
    22. Treatment with immunosuppressive, cytotoxic, or antifibrotic drugs
    including pirfenidone, colchicine, cyclosporine A, TNF-? antagonists,
    tyrosine kinase inhibitor, interferon-gamma,
    cyclophosphamide, or azathioprine < 28 days prior to randomization are
    not permitted
    ? N-acetylcysteine is permitted provided the subject has been on stable
    dose for > 12 weeks prior to screening
    ? Once a subject meets the FVC criteria for PFS, the subject may at the
    investigators discretion be prescribed any drug that is approved for
    treatment of IPF in the subject's country of
    residence, including pirfenidone
    23. Chronic use (> 28 days) of moderate or high dose oral
    corticosteroids (equivalent of > 10 mg of prednisone per day); short
    courses of higher corticosteroid doses within 1-6 months prior to
    randomization are permitted but limited to ? 28 days
    24. Participation in an investigational drug or device trial < 30 days prior
    to screening or within 5 times the half-life of the investigational agent in
    the other clinical study, if known
    25. Treatment with GS-6624 in another clinical study
    26. Listed as active on a lung transplant waiting list
    1.Embarazo o lactancia.2.Enfermedades respiratorias clínicamente significativas aparte de FPI, entre otras, asbestosis, otras neumoconiosis o neumonitis por hipersensibilidad.3.Enfermedad pulmonar obstructiva determinada mediante Pruebas de Función Pulmonar (PFP) o TCAR de la forma siguiente:?Enfermedad reactiva de las vías respiratorias evidenciada por un incremento absoluto del volumen espiratorio forzado en el primer segundo (FEV1) > 12% posterior a una prueba broncodilatadora
    O ?cociente FEV1/FVC < límite inferior de normalidad (LIN) ajustado por edad O ?Volumen residual (RV) > 120% mediante pletismografía o enfisema significativo en TCAR definido más como enfisema que fibrosis, interpretado por un servicio radiológico central.4.DLCO <25% del valor normal previsto, corregido por hemoglobina, no corregido por volumen.5.Biopsia pulmonar quirúrgica (BPQ) que muestra neumoconiosis, neumonitis por hipersensibilidad, neumonía intersticial no específica u otra enfermedad pulmonar intersticial idiopática.6.Cualquier enfermedad colágeno-vascular.7.Antecedentes de aneurisma aórtico ? 3,5cm de diámetro.8.Antecedentes de accidente cerebrovascular en las 26 semanas anteriores.9.Cardiopatía clínicamente significativa definida como infarto de miocardio documentado por elevación del segmento ST (IAMEST) en el electrocardiograma (ECG) en los 6 meses anteriores a la selección, intervención coronaria percutánea o cirugía de bypass arterial coronario en los 6 meses anteriores a la selección, angina de pecho inestable, insuficiencia cardiaca congestiva (clase NYHA III/IV o fracción de eyección ventricular izquierda conocida < 25%, insuficiencia cardiaca derecha, hipertrofia ventricular derecha significativa o arritmia no controlada
    10.Requiere tratamiento farmacológico para hipertensión pulmonar
    11.Hospitalización por enfermedad respiratoria aguda < 26 semanas antes de la selección.12.Neumonía por aspiración < 26 semanas antes de la selección.13.Infección respiratoria bacteriana, vírica, fúngica u otra (definida como la manifestación de signos/síntomas persistentes relacionados a la infección y sin mejoría), activa o reciente (< 4 semanas antes de la inclusión).14.Antecedentes de cáncer, estado precanceroso (p. ej. poliposis familiar, BRCA1, BRCA2), aparte de cáncer de piel no melatomatoso, en los 5 años previos a la selección, o en estudio diagnóstico activo por sospecha de cáncer de pulmón.15.Antecedentes de infección por virus de la inmunodeficiencia humana (VIH), hepatitis C o hepatitis B.16.Afección comórbida o enfermedad que limite la expectativa de vida a < 2 años en el momento de la selección.17.Cirugía mayor, definida según criterio del investigador, < 30 días antes de la selección o cirugía electiva programada durante la duración prevista del estudio.18.Antecedentes o evidencia de un trastorno clínicamente significativo, condición o enfermedad que, a criterio del investigador y del monitor médico de Gilead, pondrían en riesgo la seguridad del paciente o interferirían con las evaluaciones, procedimientos o finalización del estudio
    19.Consumo actual excesivo (a criterio del investigador) de alcohol o consumo de drogas.Relacionados con alteraciones fisiológicas o de órganos específicos.20.Uso de O2 suplementario > 6 l/min en actividad.21.Funcionamiento orgánico inadecuado reflejado en cualquiera de los resultados siguientes:-Plaquetas < 100 x 109/l -Hemoglobina < 11,0 g/dl - Recuento absoluto de neutrófilos (RAN) < 1,5 x 109/l -TP/INR y TTP > 1,5 x límite superior de normalidad (LSN) -AST/ALT > 3 x LSN -Bilirrubina total >1,5 LSN -Creatinina sérica > 2,0 mg/dl
    22.No se permiten tratamientos con fármacos inmunosupresores, citotóxicos o antifibróticos, entre otros, pirfenidona, colchicina, ciclosporina A, antagonistas TNF-?, inhibidores de la tirosincinasa, interferón-gamma, ciclofosfamida o azatioprina < 28 días antes de la aleatorización. -Se permite la toma de N-acetilcisteína siempre y cuando el paciente haya estado recibiendo una dosis estable durante > 12 semanas antes de la selección -Una vez que un paciente cumpla los criterios de FVC para SSP, este paciente podrá recibir, a criterio de los investigadores, cualquier fármaco que esté aprobado para el tratamiento de FPI en el país de residencia del paciente, incluida la pirfenidona.23.Uso crónico (> 28 días) de dosis moderadas o altas de corticoesteroides orales (equivalentes a > 10 mg de prednisona al día); se permiten tratamientos cortos de corticoesteroides a dosis más altas en los 1-6 meses previos a la aleatorización pero de duración ? 28 días.24.Participación en un ensayo con un fármaco o un dispositivo en investigación < 30 días antes de la selección o en el periodo equivalente a cinco veces la semivida del agente experimental en el otro ensayo clínico, en caso de que se conozca.25.Tratamiento con GS-6624 en otro ensayo clínico.26.Estar inscrito como activo en una lista de espera para trasplante de pulmón
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is:
    -PFS among intent-to-treat (ITT) subjects. PFS is defined as time between randomization and all-cause mortality or a categorical decrease in FVC % predicted (? 10% relative decrease in FVC and a ? 5% absolute decrease in FVC).
    La variable principal es:
    -SSP en los pacientes por intención de tratar (ITT). La SSP se define como el tiempo transcurrido entre la aleatorización y la mortalidad por todas las causas o una disminución categórica en la FVC como % del valor previsto (disminución relativa de FVC ? 10% y disminución absoluta de FVC ? 5%).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Following randomization (Day 0), abbreviated study visits will occur at Weeks 2, 6, 14, 18,26, 30, 38, 42, 50, 54, 62, 66, 74, 78, 82, 94, 106, 118, 130, 142, 154, 166, and 178. The interval for abbreviated study visits will be increased to every 12 weeks if a subject meets the
    FVC component of the PFS endpoint and chooses to remain in the study to receive blinded study drug.
    Following randomization (Day 0), Full Study visits will occur at Weeks 10, 22, 34, 46, 58, and 70. Full study visits will be discontinued when a subject meets a PFS event.
    Después de la aleatorización (día 0), las visitas del estudio abreviadas se realizarán en las semanas 2, 6, 14, 18, 26, 30, 38, 42, 50, 54, 62, 66, 74, 78, 82, 94, 106, 118, 130, 142, 154, 166 y 178. Si un paciente cumple con el componente de FVC de la variable SSP y decide permanecer en el estudio para recibir el fármaco del estudio enmascarado, se aumentará el intervalo entre las visitas del estudio abreviadas a 12 semanas.
    Después de la aleatorización (día 0), las visitas del estudio completas se realizarán en las semanas 10, 22, 34, 46, 58 y 70. Se suspenderán las visitas del estudio completas cuando un paciente cumpla un acontecimiento de SSP.
    E.5.2Secondary end point(s)
    Key secondary endpoints are:
    -All cause mortality among ITT subjects
    -PFS among subjects with ?high? sLOXL2 at baseline
    -All-cause mortality among subjects with ?high? sLOXL2 at baseline
    Other secondary endpoints are:
    -Adjudicated acute exacerbations
    -Adjudicated respiratory hospitalizations
    -Change in FVC % predicted
    -Change in DLCO % predicted
    -Absolute change in 6MWD
    -Change in SGRQ score
    -Change in TDI score
    -Change in CPI score
    The exploratory endpoints are:
    -Change in HRCT findings since baseline
    -Change in sLOXL2 levels
    -Determine whether baseline sLOXL2 levels are prognostic for disease progression (evaluated in placebo arm)
    -Evaluate the predictive effects of baseline sLOXL2 levels on various clinical endpoints outlined as secondary objectives
    Las variables secundarias clave incluyen:
    -Mortalidad por todas las causas en los pacientes ITT
    -SSP en los sujetos con LOXL2s "elevada" en la visita basal
    -Mortalidad por todas las causas en pacientes con LOXL2s "elevada" en la visita basal
    Otras variables secundarias son:
    -Exacerbaciones agudas adjudicadas
    -Hospitalizaciones por causas respiratorias adjudicadas
    -Cambio en FVC % previsto
    -Cambio en DLCO % previsto
    -Cambio absoluto en 6MWD
    -Cambio en la puntuación SGRQ
    -Cambio en la puntuación TDI
    -Cambio en la puntuación CPI
    -Cambio en los resultados de TCAR respecto a la evaluación basal
    -Cambio en los niveles de LOXL2s
    -Determinar si los niveles basales de LOXL2s tienen valor pronóstico para la progresión de la enfermedad (evaluada en el grupo placebo)
    -Evaluar los efectos predictivos de los niveles basales de LOXL2s en varias variables clínicas descritas como objetivos secundarios
    E.5.2.1Timepoint(s) of evaluation of this end point
    Following randomization (Day 0), abbreviated study visits will occur at Weeks 2, 6, 14, 18,26, 30, 38, 42, 50, 54, 62, 66, 74, 78, 82, 94, 106, 118, 130, 142, 154, 166, and 178. The
    interval for abbreviated study visits will be increased to every 12 weeks if a subject meets the FVC component of the PFS endpoint and chooses to remain in the study to receive blinded
    study drug.
    Following randomization (Day 0), Full Study visits will occur at Weeks 10, 22, 34, 46, 58,
    and 70. Full study visits will be discontinued when a subject meets a PFS event.
    Después de la aleatorización (día 0), las visitas del estudio abreviadas se realizarán en las semanas 2, 6, 14, 18, 26, 30, 38, 42, 50, 54, 62, 66, 74, 78, 82, 94, 106, 118, 130, 142, 154, 166 y 178. Si un paciente cumple con el componente de FVC de la variable SSP y decide permanecer en el estudio para recibir el fármaco del estudio enmascarado, se aumentará el intervalo entre las visitas del estudio abreviadas a 12 semanas.
    Después de la aleatorización (día 0), las visitas del estudio completas se realizarán en las semanas 10, 22, 34, 46, 58 y 70. Se suspenderán las visitas del estudio completas cuando un paciente cumpla un acontecimiento de SSP.
    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 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA60
    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
    Israel
    Korea, Republic of
    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
    Last patient, last visit.
    Ultima visita último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 150
    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 state19
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 200
    F.4.2.2In the whole clinical trial 500
    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
    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 Decision2013-05-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-04-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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