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    Summary
    EudraCT Number:2015-000946-41
    Sponsor's Protocol Code Number:63935937MYF2001
    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:2015-06-01
    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 number2015-000946-41
    A.3Full title of the trial
    A Randomized, Single-Blind, Multicenter Phase 2 Study to Evaluate the Activity of 2 Dose Levels of Imetelstat in Subjects with Intermediate-2 or High-Risk Myelofibrosis (MF) Relapsed/Refractory to Janus Kinase (JAK) Inhibitor
    Estudio Fase 2, aleatorizado, ciego, multicéntrico, para evaluar la actividad de 2 niveles de dosis de Imetelstat (GRN163L) en sujetos con mielofibrosis (MF) de riesgo intermedio-2 o alto riesgo en recaída o refractarios a inhibidores de la quinasa de Janus (JAK).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Evaluate Activity of 2 Dose Levels of Imetelstat in Participants with Intermediate-2 or High-Risk Myelofibrosis (MF) Previously Treated with Janus Kinase (JAK) Inhibitor
    Estudio para evaluar la actividad de 2 niveles de dosis de Imetelstat en sujetos con mielofibrosis (MF) de riesgo intermedio-2 o alto riesgo tratados previamente con inhibidores de la quinasa de Janus (JAK).
    A.4.1Sponsor's protocol code number63935937MYF2001
    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 S.A.
    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+34917228100
    B.5.5Fax number+34917228628
    B.5.6E-mailagonza45@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 nameImetelstat sodium
    D.3.2Product code JNJ-63935937
    D.3.4Pharmaceutical form Lyophilisate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNImetelstat sodium
    D.3.9.1CAS number 1007380-31-5
    D.3.9.2Current sponsor codeJNJ-63935937
    D.3.9.3Other descriptive nameIMETELSTAT SODIUM
    D.3.9.4EV Substance CodeSUB174121
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number210
    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
    Myelofibrosis (MF) is classified as a myeloproliferative neoplasm (MPN)
    La Mielofibrosis (MF) se clasifica como una neoplasia mieloproliferativa (MPN)
    E.1.1.1Medical condition in easily understood language
    Myelofibrosis (MF)
    Mielofibrosis
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level PT
    E.1.2Classification code 10028537
    E.1.2Term Myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    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 objectives of this study are to evaluate spleen response rate and symptom response rate of 2 dose regimens of imetelstat (9.4 mg/kg and 4.7 mg/kg imetelstat given intravenous [IV] every 3 weeks) in subjects with intermediate-2 or high-risk MF who are relapsed after or refractory to JAK inhibitor treatment.
    Los objetivos principales de este estudio son: evaluar la tasa de respuesta esplénica y la tasa de respuesta sintomática ante dos niveles de dosis de imetelstat (9,4 mg/kg y 4,7 mg/kg de imetelstat por vía intravenosa [iv] cada 3 semanas) en sujetos con MF intermedia 2 o de alto riesgo recidivantes o resistentes al tratamiento con inhibidores de JAK.
    E.2.2Secondary objectives of the trial
    The secondary objectives are:
    - To assess the safety of imetelstat
    - To assess CR or PR, CI, spleen response, symptoms response, and anemia response per modified 2013 IWG-MRT criteria, duration of responses, and overall survival (OS).
    - To evaluate the pharmacokinetics of imetelstat
    - To evaluate the PK/response and pharmacodynamic (PD) relationships with factors that include hemoglobin concentration, spleen size, and platelet count
    - To evaluate the immunogenicity of imetelstat
    - To assess the effect of treatment on PROs (patient-reported outcomes)
    Los objetivos secundarios son: evaluar la seguridad de imetelstat; evaluar la remisión completa (RC) o la remisión parcial (RP), la mejoría clínica (MC), la respuesta esplénica, la respuesta sintomática y la respuesta de la anemia según los criterios de investigación y tratamiento de las neoplasias mieloproliferativas del Grupo de trabajo internacional (GTI-TMP) modificados en 2013, la duración de las respuestas y la supervivencia global (SG); evaluar la farmacocinética (FC) de imetelstat; evaluar la relación de la respuesta FC y farmacodinámica (FD) con factores como la concentración de hemoglobina, el tamaño del bazo y el recuento de plaquetas; evaluar la inmunogenicidad de imetelstat; y evaluar el efecto del tratamiento sobre los resultados comunicados por los pacientes (RCP).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Diagnosis of PMF according to the revised WHO criteria; or PET-MF or PPV-MF according to the IWG-MRT criteria
    - DIPSS intermediate-2 or high risk MF
    - Measurable splenomegaly prior to study entry as demonstrated by palpable spleen measuring greater than or equal to (>=) 5 cm below the left costal margin OR spleen volume of >= 450 cm^3 measured by MRI
    - Active symptoms of MF as demonstrated by a symptom score of at least 5 points (on a 0 to 10 scale) on at least one of the symptoms or a score of 3 or greater on at least 2 of the symptoms
    - Documented progressive disease during or after JAK inhibitor therapy
    - ECOG performance status 0, 1 or 2
    - Diagnóstico de MFP según los criterios revisados de la OMS; PTE-MF o PPV-MF según los criterios de IWG-MRT
    - MF intermedia 2 o de alto riesgo según el Sistema internacional de puntuación pronóstica (DIPSS)
    - Esplenomegalia medible antes del ingreso en el estudio, demostrable mediante medición del bazo palpable a 5cm o más (>=) por debajo del reborde costal o volumen del bazo de >= 450 cm^3 medido mediante RM.
    - Síntomas activos de MF demostrables mediante una puntuación de los síntomas de al menos 5 puntos ( en una escala de 0 a 10) en al menos uno de los síntomas, o una puntuación de 3 o más en al menos 2 de los síntomas.
    - Enfermedad progresiva documentada durante o después del tratamiento con un inhibidor de JAK
    - Estado funcional del Eastern Cooperative Oncology Group de 0, 1 o 2
    E.4Principal exclusion criteria
    - Peripheral blood blast count of >= 10% or bone marrow blast count of >=10%
    - Prior treatment with imetelstat
    - Major surgery within 4 weeks of randomization
    - Past or present active hepatitis infection of any type or known acute or chronic liver disease including cirrhosis
    - Prior history of hematopoietic stem cell transplant
    - Recuento de blastos en sangre periférica de >= 10% o recuento de blastos en médula ósea de >=10%
    - Tratamiento previo con Imetelstat
    - Cirugía mayor en las 4 semanas previas a la aleatorización
    - Hepatitis activa pasada o presente o cualquier tipo de enfermedad hepática aguda o crónica incluyendo cirrosis
    - Historia previa de trasplante de células progenitoras hematopoyéticas
    E.5 End points
    E.5.1Primary end point(s)
    - Percentage of participants who Achieve Greater than or equal to 35 percent (%) Reduction in Spleen Volume at Week 24 (Spleen response rate is defined as the percentage of participants who achieve >= 35% reduction in spleen volume at Week 24 from baseline as measured by imaging scans.)

    - Percentage of participants who Achieve Greater than or equal to 50 percent (%) Reduction in Total Symptom Score at Week 24 (Symptom response rate is defined as the percentage of participants who achieve >= 50% reduction in TSS at Week 24 from baseline as measured by the modified Myelofibrosis Symptom Assessment Form (MFSAF) version 2.0 diary.)
    Porcentaje de pacientes participantes que consiguen una reducción del volumen del bazo mayor o igual al 35 % en la semana 24 (La tasa de respuesta esplénica se define como la proporción de pacientes que consiguen una reducción del volumen del bazo mayor o igual al 35 % en la semana 24 con respecto al momento basal determinada mediante pruebas de imagen).

    Porcentaje de pacientes participantes que consiguen una reducción del volumen del bazo mayor o igual al 50 % en la puntuación total de síntomas en la semana 24 (La tasa de respuesta sintomática se define como la proporción de pacientes que consiguen una reducción mayor o igual al 50 % en la puntuación total de síntomas en la semana 24 con respecto al momento basal, determinada por el diario Formulario de evaluación de los síntomas de mielofibrosis (MFSAF) versión 2.0)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Both at Week 24
    Ambos en la semana 24
    E.5.2Secondary end point(s)
    - Complete remission (CR) or partial remission (PR) per modified 2013 IWG-MRT criteria
    - Clinical improvement (CI) per modified 2013 IWG-MRT criteria
    - Number of Participants with Responses per 2013 IWG-MRT (Spleen response, symptoms response, and anemia response per modified 2013 IWG-MRT will be assessed.)
    - Duration of Treatment Response and Remission (Duration of spleen response, duration of symptoms response, duration of CR or PR, duration of CI, and duration of anemia response will be reported.)
    - Overall Survival (Overall survival is defined as the time from randomization to date of death from any cause.)
    - European Organization for Research and treatment of Cancer (EORTC) QLQ-C30 Score
    - EuroQol-EQ-5D (EQ-5D-5L) Health Questionnaire Score
    - Brief Pain Inventory- Short Form (BPI) Score
    - Patient?s Global Impression of Change (PGIC)
    - Number of Participants with Adverse Events (AEs)
    - Maximum Plasma Concentration (Cmax) of Imetelstat (The Cmax is the maximum observed plasma concentration.)
    - Time to Reach Maximum Concentration (tmax) of Imetelstat (The tmax is time to reach the maximum observed plasma concentration.)
    - Area Under the Plasma Concentration-Time Curve From Time Zero to 24 Hours (AUC [0-24h]) of Imetelstat (AUC 0-24h is area under the plasma concentration-time curve from time 0 to 24 hours postdose.)
    - Elimination Half-Life (t [1/2] Lambda) of Imetelstat (Elimination half-life (t [1/2] Lambda) is associated with the terminal slope (lambda [z]) of the semi logarithmic drug concentration-time curve, calculated as 0.693/lambda(z).)
    - Remisión completa (RC) o la remisión parcial (RP) según los criterios GTI-TMP modificados en 2013.
    - Mejoría clínica (MC) según los criterios GTI-TMP 2013 modificados en 2013 .
    - Números de participantes con respuesta esplénica, la respuesta sintomática y la respuesta de la anemia según los criterios GTI-TMP modificados en 2013.
    - Duración de la respuesta y remisión al tratamiento (duración de la respuesta esplénica, de la respuesta sintomática, de la RC o RP, de la MC, y la duración de la respuesta de la anemia serán reportadas).
    - Supervivencia global (SG) (la supervivencia global se define como el tiempo transcurrido desde la fecha de aleatorización hasta la muerte por cualquier causa).
    - Puntuación del “European Organization for Research and treatment of Cancer (EORTC) QLQ-C30”.
    - Puntuación del cuestionario de salud EuroQol-EQ-5D (EQ-5D-5L)
    - Puntuación del cuestionario BPI (Brief Pain Inventory)
    - Cuestionario PGIC (Impresión Global del cambio del paciente)
    - Concentración Plasmática máxma (Cmax) de Imetelstat (El Cmax es la concentración plásmatica máxima observada).
    - Tiempo para alcanzar la concentración máxima (tmax) de Imetelstat (El tmax es el tiempo que tarda en alcanzarse la máxima concentración plasmática observada)
    -Área bajo la curva de la Concentración Plasmática – Tiempo desde 0 a 24 horas (AUC [0-24h]) de Imetelstat ( AUC 0-24h es el área bajo la curva de la concentración plasmática-tiempo desde las 0-24 horas postdosis)
    - Vida media de eliminación (t [1/2] Lambda) de Imetelstat (la Vida media de eliminación (t [1/2] Lambda) está asociado con la pendiente final de la curva semilogaritmica concentración-tiempo del fármaco, calculada como 0.639/lambda(z).)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to 3 years
    Hasta 3 años
    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 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
    Immunogenicity
    Biomarker
    Patient Reported Outcome
    Imnunogenicidad
    Biomarcadores
    Resultado reportado del paciente
    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 Yes
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Se comparan dos dosis de Imetelstat
    Two dosis of Imetelstat are compared
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA47
    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
    Belgium
    Canada
    France
    Germany
    Israel
    Italy
    Korea, Republic of
    Spain
    Switzerland
    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 study is considered completed 18 months after the last subject is enrolled or anytime the sponsor terminates the study, whichever comes first.
    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 months2
    E.8.9.1In the Member State concerned days21
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days21
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 102
    F.4.2.2In the whole clinical trial 200
    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 Decision2015-08-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-07-14
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-02-07
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