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    Summary
    EudraCT Number:2015-000946-41
    Sponsor's Protocol Code Number:63935937MYF2001
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Temporarily Halted
    Date on which this record was first entered in the EudraCT database:2018-02-06
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2015-000946-41
    A.3Full title of the trial
    Study to Evaluate the Activity of 2 Dose
    Levels of Imetelstat in Participants with Intermediate-2 or High-Risk Myelofibrosis (MF) previously treated with Janus Kinase (JAK) Inhibitor
    Uno studio di fase 2 multicentrico, randomizzato, in singolo cieco per valutare l’attività di 2 livelli di dosaggio di Imetelstat in soggetti con mielofibrosi (MF) a rischio intermedio-2 o alto rischio, recidivante o refrattaria all'inibitore della Janus chinasi (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 Subjects with Intermediate-2 or High-Risk Myelofibrosis (MF)
    previously treated with Janus Kinase (JAK) Inhibitor
    Uno studio per valutare l’attività di 2 livelli di dosaggio di Imetelstat in soggetti con mielofibrosi (MF) a rischio intermedio-2 o alto rischio, precedentemente trattati con un inibitore della Janus chinasi (JAK)
    A.3.2Name or abbreviated title of the trial where available
    MYF2001
    MYF2001
    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 CILAG SPA
    B.4.2CountryItaly
    B.4.1Name of organisation providing supportJANSSEN CILAG INTERNATIONAL NV
    B.4.2CountryBelgium
    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 pointCLINICAL REGISTRY GROUP
    B.5.3 Address:
    B.5.3.1Street AddressARCHIMEDESWEG 29
    B.5.3.2Town/ cityLEIDEN
    B.5.3.3Post code2333 -CM
    B.5.3.4CountryNetherlands
    B.5.4Telephone number0031 715242166
    B.5.5Fax number0031 715242110
    B.5.6E-mailclinicaltrialseu@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 Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    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 nameIMTELSTAT 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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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)
    Mielofibrosi (MF) classificata come neoplasia mieloproliferativa (MPN)
    E.1.1.1Medical condition in easily understood language
    Myelofibrosis
    Mielofibrosi
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    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.
    Gli obiettivi primari di questo studio sono la valutazione del tasso di risposta della milza e del tasso di risposta dei sintomi a 2 regimi di dosaggio di Imetelstat (9,4 mg/kg e 4,7 mg/kg di Imetelstat somministrati per via endovenosa [IV] ogni 3 settimane) in soggetti con mielofibrosi (MF) a rischio intermedio-2 o alto, recidivi o refrattari al trattamento con inibitore di 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)
    Gli obiettivi secondari consistono nel: determinare la sicurezza di Imetelstat; determinare remissione completa (CR) o remissione parziale (PR), miglioramento clinico (CI), risposta della milza, risposta dei sintomi e risposta dell'anemia secondo i criteri dell’International Working Group – Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) modificati nel 2013, durata delle risposte e sopravvivenza generale (OS); valutare la farmacocinetica (PK) di Imetelstat; valutare il rapporto tra la PK/risposta e la farmacodinamica (PD) con i fattori tra cui la concentrazione di emoglobina, le dimensioni della milza e la conta piastrinica; valutare l'immunogenicità di Imetelstat; determinare l'effetto del trattamento in base ai risultati riportati dal paziente (PRO).
    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 least5 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
    -Diagnosi di mielofibrosi primaria (PMF) sulla base dei criteri WHO revisionati o PET-MF o PPV-MF sulla base dei criteri IWG-MRT
    -Rischio di MF intermedio-2 o alto secondo il sistema DIPSS
    - Splenomegalia misurabile antecedente all'ingresso nello studio, dimostrata da:
    • Milza al di sotto del margine costale sinistro (>= a 5cm) alla palpazione
    OPPURE
    • volume della milza ≥ 450 cm3 misurato tramite risonanza magnetica (RMN)
    -Sintomi attivi di MF dimostrati da un punteggio in base ai sintomi di almeno 5 punti (su una scala da 0 a 10) per almeno uno dei sintomi, oppure pari a, o superiore, 3 per almeno 2 dei sintomi
    -Progressione di malattia documentata durante o dopo la terapia con l'inibitore di JAK
    -Performance status ECOG pari a 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
    -Conta di blasti nel sangue periferico ≥10% oppure conta di blasti nel midollo osseo >=10%.
    -Precedente trattamento con Imetelstat.
    -Intervento chirurgico importante nelle 4 settimane precedenti la randomizzazione.
    -Infezione da epatite di qualsiasi tipo passata o in atto o di epatopatia acuta o cronica conosciuta, inclusa la cirrosi.
    -Eventuale storia precedente di trapianto di cellule staminali ematopoietiche.
    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.)
    Percentuale dei partecipanti che raggiungono la riduzione del volume dalle milza alla settimana 24 maggiore o uguale al 35% (il tasso di risposta della milza e definito come la percentuale dei partecipanti che raggiungono la riduzione del volume dalle milza alla settimana 24 maggiore o uguale al 35% dal basale mediante imaging)

    Percentuale dei partecipanti che raggiungono la riduzione del punteggio complessivo dei sintomi ≥50%, alla settimana 24 (il tasso di risposta dei sintomi è definite come Percentuale dei partecipanti che raggiungono la riduzione del punteggio complessivo dei sintomi ≥50%, alla settimana 24 dal basale , misurato secondo il diario Myelofibrosis Symptom Assessment Form (MFSAF) v2.0)
    E.5.1.1Timepoint(s) of evaluation of this end point
    both at 24 weeks
    entrambi a 24 settimane
    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 halflife
    (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).)
    -Remissione completa o remissione parziale per criteri IWG-MRT modificati 2013
    - Miglioramento clinico (CI) come da criteri 2013 IWG-MRT
    -Numero di partecipanti con risposta come da 2013 IWG-MRT modificati (saranno valutati la risposta della milza, la risposta dei sintomi, la risposta di anemia come da criteri 2013 IWG-MRT modificati)
    -Durata della risposta al trattamento e remissione (saranno riportati la durata della risposta della milza, dei sintomi, CR o PR, CI, anemia)
    - Sopravvivenza complessiva (definita come tempo dalla randomizzazione al decesso per qualsiasi causa)
    -Punteggio come da European Organization for Research and treatment of Cancer (EORTC)
    QLQ-C30
    -Punteggio come da EuroQol-EQ-5D (EQ-5D-5L) Health Questionnaire
    -Punteggio come da Brief Pain Inventory- Short Form (BPI)
    -Valutazione come da Patient's Global Impression of Change (PGIC)
    -Numero dei partecipanti con EA
    -Concentrazione plasmatica massima (Cmax) di Imetestat
    -Tempo per raggiungere Cmax di Imetelstat
    -Area sotto la curva concentrazione plasmatica/tempo da tempo 0 a 24 ore (AUC[0-24H]) di Imetelstat
    -Emivita (t[1/2] Lambda) di Imetelstat
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to 3 years
    Fino a 3 anni
    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
    Immunogenicità
    Biomarcatori
    Patient Reported Outcome
    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 Yes
    E.8.1.2Open No
    E.8.1.3Single blind Yes
    E.8.1.4Double blind No
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    GRUPPI PARALLELI
    PARALLEL GROUPS
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the 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 concerned9
    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 Information not present in EudraCT
    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, Democratic People's 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.
    Lo studio è considerato concluso 18 mesi dopo l'arruolamento dell'ultimo soggetto o qualora lo Sponsor decidesse di chiudere lo studio a seconda dell'evento che si verifica per primo
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days9
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 1
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    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 Yes
    F.3.3.2Women of child-bearing potential using contraception No
    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 state18
    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)
    The sponsor will ensure that subjects benefiting from treatment with
    imetelstat will be able to continue treatment after the end of the study.
    Lo Sponsor assicura che i soggetti che avranno un beneficio dal trattamento con Imetelstat potranno continuare il trattamento dopo la fine dello studio
    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 Decision2015-08-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-09-11
    P. End of Trial
    P.End of Trial StatusTemporarily Halted
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