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    Summary
    EudraCT Number:2010-023076-10
    Sponsor's Protocol Code Number:CP14B015
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2010-12-07
    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 ConcernedGermany - BfArM
    A.2EudraCT number2010-023076-10
    A.3Full title of the trial
    A Phase II Trial to Evaluate the Activity of Imetelstat (GRN163L) in Patients with Essential Thrombocythemia who Require Cytoreduction and Have Failed or Are Intolerant to Previous Therapy, or who Refuse Standard Therapy
    A.4.1Sponsor's protocol code numberCP14B015
    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 SponsorGeron 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 supportGeron Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGeron Corporation
    B.5.2Functional name of contact point
    B.5.3 Address:
    B.5.3.1Street Address149 Commonwealth Drive
    B.5.3.2Town/ cityMenlo Park
    B.5.3.3Post codeCA 94025
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 650-473-7725
    B.5.5Fax number+1 650-566-7210
    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 GRN163L
    D.3.4Pharmaceutical form Powder 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 codeGRN163L
    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 Yes
    D.3.11.13.1Other medicinal product typeSynthetic oligonucleotide (with a 5’-conjugated C16 lipid attached through an aminoglycerol linker)
    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
    Essential thrombocythemia
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    E.1.2Level PT
    E.1.2Classification code 10015493
    E.1.2Term Essential thrombocythaemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To obtain a preliminary estimate of efficacy of imetelstat, as measured by best hematologic response, in patients with ET who have failed or are intolerant to at least one prior therapy, or who have refused standard therapy.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are as follows:
    •To characterize the safety and tolerability of imetelstat in patients with ET;
    •To determine the durability of hematologic response;
    •To evaluate molecular response in patients with JAK2 V617F and/or MPL W515 mutations within 2 years of therapy;
    • To evaluate BM histologic response within the first 2 years of imetelstat therapy;
    • To evaluate clinicohematologic response within the first year of imetelstat therapy using the modified European LeukemiaNet response criteria
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Willing and able to sign an informed consent.
    -Male or female, aged 18 years or older.
    -Patients with ET requiring cytoreduction who have failed or are intolerant to at least one prior therapy, or who refuse standard therapy.
    -Confirmed diagnosis of ET by WHO criteria.
    -ECOG performance status 0–2.
    -Laboratory criteria (within 14 days of first study drug administration) in compliance with protocol limits.
    -Any clinically significant toxicity from previous cancer treatments and/or major surgery must have recovered to Grade 0-1 prior to initiation of study treatment.
    -Women of childbearing potential must have a negative pregnancy test and agree to use effective birth control during and for at least 12 weeks after the last study treatment with imetelstat.
    -Male patients must agree to use effective birth control for themselves or their partner during and for 12 weeks after the last study treatment with imetelstat.
    E.4Principal exclusion criteria
    -Women who are pregnant or breast feeding.
    -Prior stem cell transplantation.
    -Investigational therapy within 4 weeks prior to first study drug administration.
    -Clinically significant cardiovascular disease or condition including: Uncontrolled congestive heart failure (CHF); Need for antiarrhythmic therapy for a ventricular arrhythmia; Clinically significant severe conduction disturbance per the Investigator’s discretion; Ongoing angina pectoris requiring therapy; New York Heart Association (NYHA) Class II, III, or IV cardiovascular disease.
    -Known positive serology for human immunodeficiency virus (HIV).
    -Serious co-morbid medical conditions, including active or chronically recurrent bleeding, clinically relevant active infection, cirrhosis, and chronic obstructive or chronic restrictive pulmonary disease per the Investigator’s discretion.
    -Any other severe, acute, or chronic medical or psychiatric condition, laboratory abnormality, or difficulty complying with protocol requirements that may increase the risk associated with study participation or study drug administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the patient inappropriate for this study.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy will be determined by best overall hematologic response rate (CR + partial response [PR]) beginning within the first year of imetelstat therapy maintained for at least 4 consecutive weeks, defined as follows:
    -CR: Platelet count ≤ 400 x 103/μL in the absence of new thromboembolic events;
    -PR: ≤ 600 x 103/μL OR a 50% reduction in platelet counts (but > 400 x 103/μL) in the absence of new thromboembolic events.
    E.5.1.1Timepoint(s) of evaluation of this end point
    - Hematologic response prior to every imetelstat dose and molecular response every 12 (± 4) weeks
    - Clinicohematologic response (including evaluation of splenomegaly) at screening and every 12 (± 4) weeks
    - A repeat BM biopsy and aspirate will be performed after completing Cycle 6 (6 months) of study treatment when the patient is in a hematologic CR, and at the end of 12 cycles (12 months) or 1 year to assess histologic response. For patients who continue to receive treatment beyond 1 year, BM biopsies should be obtained every 6 months as long as the patient is in a hematologic CR
    - Additional BM biopsies and aspirates will be performed every 12 (± 4) weeks (after completion of Cycles 3 [Week 12], 9 [Week 36], and 12 [Week 48]) in patients at select study centers
    E.5.2Secondary end point(s)
    - Duration of hematologic response
    - Rate of molecular response (CR and PR) within 2 years of therapy in patients with a molecular mutation (JAK2 or MPL) at baseline
    - Rate of BM histologic response within 2 years of therapy
    - Rate of clinicohematologic response within the first year of therapy as defined by the modified European LeukemiaNet consensus criteria (Section 8.8.4.4 and Table 8), as measured by the investigator
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Hematologic response prior to every imetelstat dose and molecular response every 12 (± 4) weeks
    - Clinicohematologic response (including evaluation of splenomegaly) at screening and every 12 (± 4) weeks
    - A repeat BM biopsy and aspirate will be performed after completing Cycle 6 (6 months) of study treatment when the patient is in a hematologic CR, and at the end of 12 cycles (12 months) or 1 year to assess histologic response. For patients who continue to receive treatment beyond 1 year, BM biopsies should be obtained every 6 months as long as the patient is in a hematologic CR
    - Additional BM biopsies and aspirates will be performed every 12 (± 4) weeks (after completion of Cycles 3 [Week 12], 9 [Week 36], and 12 [Week 48]) in patients at select study centers
    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 Yes
    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 No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Yes
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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.8.3 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 No
    E.8.5.1Number of sites anticipated in the EEA5
    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
    Germany
    Switzerland
    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
    In addition to last visit of the last subject, Geron may terminate the study 1) if the incidence of severity of adverse events indicates a potential health hazard to patients, 2) patient enrollment is unsatisfactory, 3) data recording is inaccurate or incomplete, 4) lack of efficacy
    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 months2
    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 months5
    E.8.9.2In all countries concerned by the trial days0
    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.1.3Elderly (>=65 years) Yes
    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 15
    F.4.2.2In the whole clinical trial 40
    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 Decision2011-02-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-02-14
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-01-06
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