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    Summary
    EudraCT Number:2015-002874-19
    Sponsor's Protocol Code Number:63935937MDS3001
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2015-09-04
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2015-002874-19
    A.3Full title of the trial
    A Study to Evaluate Imetelstat (JNJ-63935937) in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) that is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Evaluate Imetelstat (JNJ-63935937) in Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS).
    A.4.1Sponsor's protocol code number63935937MDS3001
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorJanssen-Cilag International NV
    B.1.3.4CountryBelgium
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportJanssen Research & Development, LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJanssen-Cilag International NV
    B.5.2Functional name of contact pointClinical Registry Group
    B.5.3 Address:
    B.5.3.1Street AddressArchimedesweg 29
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333CM
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31(0)71524 21 66
    B.5.5Fax number+31(0)71524 21 10
    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 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
    Myelodysplastic syndrome (MDS)
    E.1.1.1Medical condition in easily understood language
    Myelodysplastic syndrome (MDS)
    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 HLT
    E.1.2Classification code 10028536
    E.1.2Term Myelodysplastic syndromes
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part 1: To evaluate the efficacy and safety of imetelstat in transfusion dependent subjects with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment.

    Part 2: To compare the efficacy, in terms of RBC TI, of imetelstat to placebo in transfusion dependent subjects with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment.
    E.2.2Secondary objectives of the trial
    - To assess the safety of imetelstat in subjects with MDS
    - To assess the time to RBC TI and duration of RBC TI
    - To assess the rate of hematologic improvement
    - To assess the rates of CR or PR
    - To assess OS
    - To assess time to progression to AML
    - To assess the rate and amount of supportive care, including transfusions and myeloid growth factors
    - To evaluate the pharmacokinetics and immunogenicity of imetelstat in subjects with MDS
    - To assess the effect of imetelstat treatment on patient reported outcomes (PROs)
    - To assess the effect of treatment on medical resource utilization
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Man or woman greater than or equal to (>=) 18 years of age
    - Diagnosis of myelodysplastic syndrome (MDS) according to WHO criteria or French-American-British (FAB) classification confirmed by bone marrow aspirate and biopsy within 12 weeks prior to Study Entry. A local laboratory report from this diagnostic bone marrow aspirate and biopsy must be reviewed and approved by the sponsor
    - International Prognostic Scoring System (IPSS) low Risk or intermediate-1 risk MDS
    - Red blood cell (RBC) transfusion dependent, defined as requiring 4 units RBC over 8 weeks during the 12 weeks prior to Study Entry; pre-transfusion hemoglobin (Hb) should be less than equal to 9.0 gram per deciliter (g/dL) to count towards the 4 units total
    - Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2
    E.4Principal exclusion criteria
    - Participant has known allergies, hypersensitivity, or intolerance to imetelstat or its excipients
    - Participant has received an investigational drug or used an invasive investigational medicaldevice within 30 days prior to Study Entry or is currently enrolled in an investigational study
    - Prior treatment with imetelstat
    - Have received any chemotherapy, immunomodulatory or immunosuppressive therapy, corticosteroids greater than 30 milligram per day prednisone or equivalent, or growth factor treatment within 28 days prior to study entry
    - Have received other treatments for MDS within 4 weeks prior to Study Entry
    E.5 End points
    E.5.1Primary end point(s)
    Percentage of participants without any red blood cell (RBC) transfusion during any consecutive 8 week period.
    E.5.1.1Timepoint(s) of evaluation of this end point
    8 weeks
    E.5.2Secondary end point(s)
    1/ Number of Participants with Adverse Events (AEs)
    2/ Percentage of participants without any red blood cell (RBC) transfusion during any consecutive 24 week period
    3/ Time to the 8-week RBC transfusion independence (TI)
    4/ Duration of RBC TI
    5/ Percentage of Participants with hematologic improvement
    6/ Percentage of Participants with Complete remission (CR) or Partial remission (PR) as Per International Working Group (IWG) Response CriteriaI 2006
    7/ Overall survival
    8/ Time to Progression to Acute Myeloid Leukemia
    9/ Percentage of Participants with Transfusion
    10/ Amount of Transfusions
    11/ Percentage of Participants receiving any myeloid growth factors
    12/ Change from baseline in Functional Assessment of Cancer Therapy -Anemia-Related Effects (FACT-An) Score and EuroQol-EQ-5D-5L ESA (EQ-5D-5L) Score
    13/ Maximum Observed Plasma Concentration (Cmax)
    14/ Area under the drug concentrationplasma time curve from time zero to last measurable concentration (AUC0-t)
    15/ Percentage of Participants with antibodies to imetelstat
    16/ Medical resource utilization data
    E.5.2.1Timepoint(s) of evaluation of this end point
    1/ up to follow-up (30 days posttreatment [approximately 2 years])
    2-12/ up to 2 years after enrollment of the last participant
    13-14/ During treatment (approximately 2 years)
    15-16/ up to 2 years after enrollment of the last participant
    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
    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) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA56
    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
    France
    Germany
    Italy
    Korea, Republic of
    Mexico
    Netherlands
    Russian Federation
    Spain
    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
    The End of the Study is defined as 2 years after Study Entry of the last subject 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 months8
    E.8.9.1In the Member State concerned days29
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days28
    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: 60
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 140
    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 state27
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 140
    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.
    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 Decision2015-11-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-11-23
    P. End of Trial
    P.End of Trial StatusOngoing
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