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    Summary
    EudraCT Number:2010-023047-15
    Sponsor's Protocol Code Number:CC-4047-SSC-001
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-01-19
    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 number2010-023047-15
    A.3Full title of the trial
    A Phase 2, Proof-of-Concept, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Pomalidomide (CC-4047) In Subjects with Diffuse Cutaneous Systemic Sclerosis with Interstitial Lung Disease
    Estudio de fase II de prueba de concepto, multicéntrico, randomizado, doble ciego, controlado con placebo para evaluar la seguridad, tolerabilidad, farmacocinética, farmacodinámica y eficacia de pomalidomida (CC-4047) en pacientes con esclerosis sistémica cutánea difusa con enfermedad pulmonar intersticial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to see if pomalidomide is safe and works to treat patients with sclerosis affecting the skin on the whole body and that also have lung disease
    Estudio para comprobar si la pomalidomida es segura y funciona para tratar pacientes con esclerosis que afecta a la piel de todo el cuerpo y que tienen también afección pulmonar.
    A.4.1Sponsor's protocol code numberCC-4047-SSC-001
    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 SponsorCelgene Corporation
    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 supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinicalTrialDisclosure
    B.5.3 Address:
    B.5.3.1Street Address9900 W. 109th Street, Suite 300, Building 70, Overland Park
    B.5.3.2Town/ cityKansas
    B.5.3.3Post code66210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+18882601599
    B.5.5Fax number+19134513459
    B.5.6E-mailClinicalTrialDisclosure@celgene.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 namePomalidomida
    D.3.2Product code CC-4047
    D.3.4Pharmaceutical form Capsule, hard
    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 INNPomalidomida
    D.3.9.1CAS number 19171-19-8
    D.3.9.2Current sponsor codeCC-4047
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    diffuse cutaneous systemic sclerosis associated with interstitial lung disease
    Esclerosis sistémica cutánea difusa asociada con enfermedad pulmonar intersticial
    E.1.1.1Medical condition in easily understood language
    Tissue disease involving changes in the skin, blood vessels and internal organs. It is an'autoimmune disorder', meaning the immune system mistakenly attacks and destroys healthy body tissue.
    Enfermedad del tejido que supone cambios en la piel, vasos sanguíneos y órganos internos. Es un "desorden autoinmune" es decir el sistema inmune ataca por error y destruye el tejido corporal sano.
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10025109
    E.1.2Term Lung involvement in systemic sclerosis
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and tolerability of pomalidomide QD compared to placebo in subjects with dc-SSc-ILD

    To evaluate the change from Baseline (Week 0) in the FVC in dc-SSc-ILD subjects treated with pomalidomide QD compared to placebo

    To evaluate the change from Baseline (Week 0) in the mRSS in dc-SSc-ILD subjects treated with pomalidomide QD compared to placebo

    To evaluate changes from Baseline (Week 0) in GI symptomatology as measured by the UCLA SCTC GIT 2.0 total score in subjects treated with pomalidomide QD
    compared to placebo
    Evaluar la seguridad y tolerabilidad de pomalidomida una vez al día (UD) comparado con placebo en pacientes con ESCD-EPI

    Evaluar el cambio desde Visita Basal (Semana 0) en la capacidad vital forzada (CVF) en pacientes ESCD-EPI tratados con pomalidomida UD comparado con placebo

    Evaluar el cambio desde Visita Basal (Semana 0) en el índice de Afección Cutánea de Rodnan modificado (mRSS) en pacientes ESCD-EPI tratados con pomalidomida UD comparado con placebo
    E.2.2Secondary objectives of the trial
    To characterize the PK of pomalidomide in subjects with dc-SSc-ILD

    To evaluate the clinical efficacy of pomalidomide QD compared to placebo in other
    dc-SSc-ILD-related manifestations
    Caracterizar la farmacocinética (FC) de pomalidomida en pacientes con ESCD-EPI

    Evaluar la eficacia clínica de pomalidomida UD comparado con placebo en otras manifestaciones relacionadas con ESCD-EPI.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Male or female subjects between 18 and 80 (inclusive) at the time of signing the ICD
    2.Body weight >=80pounds (36.3kg) at Screening and Baseline
    3.Understand and voluntarily sign ICDs prior to the initiation of any study-related assessments / procedures
    4.Able to adhere to the study visit schedule and other protocol requirements
    5.Diagnosis of dc-SSc as defined by ACR criteria
    6.Onset of the first non-Raynaud?s manifestation of SSc within 5 years of Screening
    7.FVC>=45% and <=80% predicted at Screening and Baseline. Source documentation confirming a >=5% decrease in FVC based on 3 or more assessments within 18months of Baseline (Visit 2) is required. Two assessments may be done during the Screening Phase provided the assessments are completed at least 2 weeks apart.
    8.Repeat FVC at Baseline (Visit 2) within 5% of the FVC measured at Screening
    9.DLco >=40% and <=80% of predicted value at Screening
    10.Abnormalities on HRCT consistent with sclerodermatous involvement of the lung
    11.Meet the following laboratory criteria:
    Hemoglobin >=10.0 g/dL
    White blood cell (WBC) count >=3000/µL(3.0x10^9/L) and <=14,000/µL
    (14x109/µL)
    Absolute neutrophil count (ANC)>=2x10^9/L
    Platelet count>=150,000/µL(>=150x10^9/L)
    Serum creatinine<=1.5 mg/dL(<=132.6?mol/L)
    Total bilirubin<=2.0 mg/dL
    Albumin>=3.0g/dL
    AST and ALT<=1.5X upper limit of normal
    12.Females of childbearing potential (FCBP) must undergo pregnancy testing based and results must be negative
    13.Unless practicing complete abstinence from heterosexual intercourse, sexually active FCBP must use adequate contraceptive methods
    14.Males (including those who have had a vasectomy) must use barrier contraception when engaging in sexual activity with FCBP
    15.Males must agree not to donate semen or sperm
    16.All subjects must:
    Understand that the IP could have a potential teratogenic risk
    Agree to abstain from donating blood while taking IP and following discontinuation of IP
    Agree not to share IP with another person
    Other than the subject, FCBP and males able to father a child should not handle the IP or touch the capsules, unless gloves are worn.
    Be counseled about pregnancy precautions and risks of fetal exposure
    17.Use of concomitant medications for the treatment of SSc-related symptoms is permitted (Proton pump inhibitors or other gastroesophageal reflux disease therapies, Angiotensin receptor blocker, Angiotensin-converting enzyme inhibitor, Selective serum reuptake inhibitors, Calcium channel blockers, Non-sedating oral or intranasal antihistamine agents, PDE5 inhibitors, <=10mg/day of oral prednisone, Oral statin agents for hyperlipidemia, Analgesic or opioid pain medications, Subjects are required to follow a low-dose aspirin regimen (<=100mg/day) unless contraindicated
    1.Pacientes mujeres y varones con edades comprendidas entre 18 y 80 años (inclusive) en el momento de la firma del CI.
    2.Peso corporal >= 80 libras (36.3 Kg) en la Selección y Basal
    3.Debe entender y firmar voluntariamente un CI antes de que se realice ningún procedimiento o evaluación relativa al estudio
    4.Ser capaz de someterse al programa de visitas del estudio y otros requisitos del protocolo
    5.Diagnóstico de ESCD según la definición de los criterios de ACR (Apéndice A)
    6.Inicio de la primera manifestación no Raynaud de esclerosis sistémica en un plazo de 5 años desde la selección
    7.CVF >= 45% y <= 80% pronosticado en la selección y en Basal. Es necesario documentación fuente que confirme una disminución de >= 5% en CVF según 3 o más evaluaciones en el plazo de 18 meses desde la Basal (Visita 2). Podrán realizarse dos evaluaciones durante la fase de selección siempre que las evaluaciones se hayan realizado al menos con 2 semanas de diferencia.
    8.Repetir CVF en la Basal (Visita 2) y esté dentro del 5% del CVF medido en la selección (la evaluación más reciente si se han realizado varias evaluaciones durante la selección)
    9.DLco >= 40% y <= 80% de valor pronosticado en la selección
    10.Anomalías en HRCT coherente con la afección esclerodérmica de los pulmones (por ejemplo, patrón de opacificación en vidrio esmerilado, pulmón en panal de abejas)
    11.Debe cumplir los siguientes criterios de laboratorio:
    Hemoglobina >= 10.0 g/dL
    Recuento de leucocitos >= 3000 /µL (>= 3.0 x 109/L) y <= 14.000/µL (<= 14 x 109/L)
    Recuento absoluto de neutrófilos (RAN) >= 2 x 109/L
    Recuento de plaquetas >= 150.000 /µL (>= 150 X 109/L)
    Creatinina sérica <= 1.5 mg/dL (<= 132.6 ?mol/L)
    Bilirrubina total <= 2.0 mg/dL
    Albumina >= 3.0 g/dL
    Aspartato transaminasa (AST [ SGOT]) y alanina transaminasa (ALT [SGPT]) <= 1.5 X límite superior de normalidad (ULN)
    12. Las mujeres en edad fértil (MEF)* deben someterse a pruebas de embarazo según la frecuencia descrita en Apéndice F y los resultados de embarazo deben ser negativos.
    13. A menos que esté practicando la abstinencia completa de relaciones heterosexuales, las MEF sexualmente activas deben aceptar el uso de métodos anticonceptivos adecuados según se especifica en Apéndice F.
    14. Los varones (incluyendo a los que se le haya realizado una vasectomía) deben usar un anticonceptivo de barrera (preservativos) cuando tengan relaciones sexuales con MEF según se especifica en Apéndice F.
    15.Los varones deben aceptar no donar semen ni esperma 16.Todos los pacientes deben:
    Entender que el producto en investigación puede tener potencialmente un riesgo teratogénico.
    Acordar abstenerse de donar sangre mientras toman el producto en investigación y tras la interrupción del producto en investigación
    Acordar no compartir la medicación en estudio con nadie más.
    Aparte del paciente, las MEF y los varones capaces de ser padres no deben manejar el producto en investigación ni tocar las cápsulas, a menos que se usen guantes.
    Recibir asesoramiento acerca de las precauciones de embarazo y los riesgos de la exposición fetal.
    17.Está permitido el uso de medicaciones concomitantes para el tratamiento de síntomas relacionados con esclerosis sistémica. Estos incluyen:
    Terapias con inhibidores de bomba de protones u otra enfermedad del reflujo gastroesofágico (ERGE)
    Terapias con antagonistas de los receptores de la angiotensina
    Terapias con inhibidores de la enzima convertidora de angiotensina (IECA)
    Inhibidores selectivos de la recaptación de serotonina (ISRS)
    Bloqueantes de canal de calcio
    Agentes antihistamínicos intranasales u orales no sedativos (uso intermitente).
    Inhibidores PDE5 (si no se toma para hipertensión arterial pulmonar [HAP])
    <= 10 mg/día de prednisona oral (o equivalente)
    Agentes de estatina orales para hiperlipidemia (si se ha tomado la medicación durante
    >= 84 días [12 semanas] antes de la selección)
    Medicaciones analgésicas u opioides contra el dolor.
    E.4Principal exclusion criteria
    1.Oxygen saturation<92%(room air [sea level] at rest) at Screening or Baseline
    2.Known diagnosis of obstructive lung disease as defined by FEV1/FVC ratio <0.65
    3.Diagnosis of pulmonary arterial hypertension (PAH) requiring treatment
    4.Known diagnosis of other significant respiratory disorders
    5.Any significant medical condition, laboratory abnormality or psychiatric illness that would prevent the subject from participating in the study
    6. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study
    7.Any condition that confounds the ability to interpret data from the study
    8.Pregnant or lactating females
    9.Current clinical diagnosis of another inflammatory connective tissue disease
    10.History of a thromboembolic event or hypercoagulable state
    11.Family history of genetic disease associated with deep vein thrombosis or
    thromboembolism
    12. History of clinically significant endocrinologic, pulmonary (other than SSc-related), GI (other than SSc-related), neurologic, psychiatric, hepatic, renal, hematologic, cardiovascular, immunologic or other major uncontrolled disease
    13.History or current diagnosis of peripheral neuropathy
    14.History of alcohol or drug abuse
    15.History of any of the following cardiac conditions within 6 months of Screening: acute myocardial infarction, acute coronary syndrome, new onset atrial fibrillation, new onset atrial flutter, second- or third-degree atrioventricular block, ventricular fibrillation, ventricular tachycardia, heart failure, cardiac surgery, interventional cardiac catheterization (with or without a stent placement), interventional electrophysiology procedure, presence of implanted permanent pacemaker or presence of implanted defibrillator
    16.History of additional risk factors for torsade de pointes
    17.Corrected QTcF>440msec at Screening or Baseline
    18. Presence of any of the following on 2 of the 3 Screening or Baseline ECGs (pre-dose) at rest: heart rate<45beats/min or >110beats/min, PR interval>220ms, QRS duration>110ms
    19.Clinically significant abnormality on any Screening or Baseline ECG
    20.History of tuberculosis
    21.History of HIV infection
    22.History of congenital and acquired immunodeficiencies
    23.Hepatitis B surface antigen positive or hepatitis B core antibody positive at Screening
    24.Antibodies to hepatitis C at Screening
    25.History of malignancy
    26.Use of concomitant medication(s) which could increase the risk for developing deep vein thrombosis
    27.Use of melphalan within 52 weeks of Screening
    28.Use of concomitant medications which prolong the QT/QTc interval
    29.Use of any anti-coagulant or anti-thrombotic medications
    30.Use of any cytotoxic/immunosuppressive agent
    31.Use of prostaglandin analogues
    32.Use of any biologic agent within 84 days or 5 half-lives of Screening
    33.Use of endothelin-1 inhibitors
    34.Use of medications with putative scleroderma disease-modifying properties within 28 days of Screening
    35.Smoking within 168 days of Screening
    36.Ingestion of grapefruit, grapefruit juice or grapefruit-containing products within 14 days of randomization
    37.Use of any investigational drug within 28 days of Screening or 5 PD/PK
    half-lives
    1.Saturación de oxígeno (SpO2) < 92% (aire ambiente [a nivel del mar] en descanso) en la selección o visita basal
    2.Diagnóstico conocido de enfermedad pulmonar obstructiva según la definición de relaciónVEF1/CVF < 0.65
    3.Diagnóstico de hipertensión arterial pulmonar (HAP) que requiera tratamiento
    4.Diagnóstico conocido de otros trastornos respiratorios importantes (por ejemplo asma, tuberculosis, sarcoidosis, aspergilosis, bronquitis crónica, enfermedad neoplásica, fibrosis cística, etc.)
    5.Cualquier trastorno medico significativo, anomalía de laboratorio o enfermedad psiquiátrica que pueda evitar la participación del paciente en el estudio.
    6.Cualquier trastorno que incluya la presencia de anomalías de laboratorio, que ponga al paciente en un riesgo inaceptable si él/ella participara en el estudio.
    7.Cualquier trastorno que confunda la capacidad de interpretar los datos del estudio
    8.Mujeres embarazadas o lactantes
    9.Diagnóstico clínico actual de otra enfermedad de tejidos conectivos inflamatorios
    10.Historial de un acontecimiento tromboembólico o estado hipercoagulable.
    11.Historial familiar de enfermedad genética asociada a trombosis venosa profunda o tromboembolismo
    12.Historial de enfermedad clínicamente significativa (según determine el investigador) endocrinológica, pulmonar (que no esté relacionada con esclerosis sistémica), neurológica, psiquiátrica, hepática, renal, hematológica, cardiovascular, inmunológica u otra principal no controlada.
    13.Historial o diagnóstico actual de neuropatía periférica
    14.Historial de abuso de alcohol o drogas
    15.Historia de cualquiera de los siguientes trastornos cardiacos en el plazo de 6 meses desde la selección: infarto agudo de miocardio, síndrome coronario agudo, nuevo episodio de fibrilación auricular, nuevo episiodio de flutter auricular, bloqueo atrioventricular de segundo o tercer grado, fibrilación ventricular, taquicardia ventricular, paro cardiaco, cirugía cardiaca, cateterismo cardíaco intervencionista (con o sin colocación de stent), procedimiento intervencionista de electrofisiología, presencia de un marcapasos permanente implantado o presencia de un desfibrilador implantado.
    16.Historial de factores de riesgo adicionales por síndrome de QT largo (es decir, fallo cardiaco, hipocalemia, historia familiar de síndrome de QT largo)
    17.QTcF corregido > 440 msec en la selección o visita basal (pre-dosis)
    18.Presencia de cualquiera de lo siguiente en 2 de los 3 electrocardiogramas en selección o basal (pre-dosis) en descanso:
    Ritmo cardiaco < 45 latidos por minuto
    Ritmo cardiaco > 110 latidos por minuto
    Intervalo PR > 220 ms
    Duración QRS > 110 ms
    19.Anomalía clínicamente significativa en cualquiera de los electrocardiogramas de selección o basal
    20.Historial de tuberculosis (TB).
    21.Historial de infección del virus de inmunodeficiencia humano (VIH).
    22.Historial de inmunodeficiencias adquiridas o congénitas 23.Antígeno de superficie de hepatitis B positivo o anticuerpo core positivo de hepatitis B en la selección
    24.Anticuerpos a hepatitis C en la selección
    25.Historial de neoplasias
    26.Uso de medicación(es) concomitante(s) que pueda aumentar el riesgo de desarrollo trombosis venosa profunda
    27.Uso de melfalán en el plazo de 52 semanas desde la Selección.
    28.Uso de medicaciones concomitantes que prolonguen el intervalo QT/QTc
    29.Uso de medicaciones antitrombóticas o anti-coagulantes (que no sea aspirina a dosis bajas
    30.Uso de cualquier agente citotóxico/inmunosupresivo
    31.Uso de análogos de prostaglandina
    32.Uso de cualquier agente biológico en el plazo de 84 días (12 semanas) o 5 medias vidas desde la selección.
    33.Uso de inhibidores de endotelina-1
    34.Uso de medicaciones con propiedades putativas de modificación de la enfermedad esclerodérmica en el plazo de 28 días (4 semanas) desde la selección
    35.Fumar puros, pipas o cigarrillos en el plazo de 168 días (24 semanas) desde la selección
    36.Ingestión de pomelo, zumo de pomelo o productos que contengan pomelo en el plazo de 14 días (2 semanas) anteriores a la randomización.
    37.Uso de cualquier medicamento en investigación en los 28 días (4 semanas) desde selección o 5 medias vidas de farmacocinética/farmacodinámica
    E.5 End points
    E.5.1Primary end point(s)
    Safety (type, frequency, severity and relationship of AEs to pomalidomide,
    laboratory, ECG, physical exam or other changes) and tolerability to pomalidomide

    Change from Baseline (Week 0) of the FVC at Week 52

    Change from Baseline (Week 0) of the mRSS score at Week 52

    Change from Baseline (Week 0) of the UCLA SCTC GIT 2.0 total score at Week 52
    Seguridad (tipo, frecuencia, intensidad y relación de AAs a la pomalidomida, laboratorio, electrocardiograma, exploración física u otros cambios) y tolerabilidad a la pomalidomida.

    Cambio desde Basal (Semana 0) de CVF en la Semana 52

    Cambio desde Basal (Semana 0) de puntuación mRSS en la Semana 52

    Cambio desde Basal (Semana 0) de la puntuación total del UCLA SCTC GIT 2.0 en la Semana 52
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety:
    - complete physical exam: screening, week 24, week 52, week 56
    - vital signs: screening, baseline, every week from week 1 to 56
    - ECG: screening, baseline, week 4, 6, 8, 12, 20, 28, 36, 44, 52 and 56
    - adverse events: screening, baseline, everyweek from week 1 to 56
    - survival: week 56

    FVC: screening, baseline, weeks 12, 24, 36, 48, 52 and 56

    mRSS: baseline, weeks 12, 24, 52, 56

    UCLA SCTC GIT: baseline, weeks 12, 24, 52, 56
    Seguridad:
    -examen físico completo: visita de selección, semana 24, semana 52, semana 56
    -signos vitales: visita de selección, visita basal, cada semana desde semana 1 a 56
    -ECG: selección, basal, semanas 4, 6, 8, 12, 20, 28, 36, 44, 52 y 56
    -reacciones adversas: selección, basal, cada semana desde semana 1 a 56
    -supervivencia: semana 56

    FVC: selección, basal, semanas 12, 24, 36, 48, 52 y 56.

    mRSS: basal, semanas 12, 24, 52, 56

    UCLA SCTC BIT: basal, semanas 12, 24, 52, 56
    E.5.2Secondary end point(s)
    Estimation of pomalidomide PK parameters in plasma, eg, AUCt, Cmax, and tmax,

    Change from Baseline (Week 0) of the FVC at Weeks 12, 24, 36 and 48

    Change from Baseline (Week 0) of the DLco at Weeks 24 and 52

    Change from Baseline (Week 0) of the mRSS at Weeks 12 and 24

    Change from Baseline (Week 0) of the UCLA SCTC GIT 2.0 total score at Weeks 12, and 24

    Change from Baseline (Week 0) UCLA SCTC GIT 2.0 subscale scores (Reflux,
    Distention/Bloating, Fecal Soilage, Diarrhea, Social functioning, Emotional Wellbeing and Constipation) at Weeks 12, 24 and 52

    Change from Baseline (Week 0) of the oxygen saturation (as measured by pulse
    oximetry) at Weeks 12, 24 and 52

    Change from Baseline (Week 0) dyspnea (as measured by the Transition Dyspnea Index) at Weeks 12, 24 and 52
    Estimación de los parámetros farmacocinéticos de pomalidomida en plasma, ejemplo AUCt, Cmax y tmax.

    Cambio desde la visita basal (semana 0) de la FVC a las semanas 12, 24, 36, 48

    Cambio desde la visita basal (semana 0) de la DLco a las semanas 24 y 52

    Cambio desde la visita basal (semana 0) de la mRSS a las semanas 12 y 24

    Cambio desde la visita basal (semana 0) de la puntuación total de UCLA SCTC GIT 2.0 a las semanas 12 y 24

    Cambio desde la visita basal (semana 0) de las puntuaciones de la subescala UCLA SCTC GIT 2.0 (reflujo, distensión/hinchazón, incontinencia fecal, diarrea, funcionamiento social, bienestar emocional y estreñimiento) en las semanas 12, 24 y 52

    Cambio de la visita basal (semana 0) de la saturación de oxígeno (medido mediante pulsometría) en las semanas 12, 24 y 52

    Cambio desde la visita basal (semana 0) de disnea (medido por el índice de transición de disnea) en las semanas 12, 24 y 52
    E.5.2.1Timepoint(s) of evaluation of this end point
    PK assessments: baseline, weeks 1 to 4, weeks 6 and 8

    FVC: baseline, Weeks 12, 24, 36 and 48

    DLco: baseline, weeks 24 and 52

    mRSS: baseline, weeks 12 and 24

    UCLA SCTC GIT: baseline, weeks 12 and 24
    Medición farmacocinética: basal, semanas de la 1 a la 4, semanas 6 y 8

    CVF: basal, semanas 12, 24, 36 y 48

    DLco: basal, semanas 24 y 52

    mRSS: basal, semanas 12 y 24

    UCLA SCTC GIT: basal, semanas 12 y 24
    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 Yes
    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
    tolerability
    tolerabilidad
    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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA35
    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
    France
    Germany
    Italy
    Poland
    Spain
    Sweden
    Switzerland
    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
    last visit of the last subject during the follow-up phase
    Última visita del último sujeto durante la fase de seguimiento
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days7
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days7
    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: 70
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 18
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2012-01-19. Yes
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 54
    F.4.2.2In the whole clinical trial 88
    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)
    Subjects that are still alive will return to their physician to receive standard of care
    Los sujetos que continúen con vida volverán a su médico para recibir tratamiento estándar.
    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 Decision2012-03-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-11-03
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