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    Summary
    EudraCT Number:2012-003120-21
    Sponsor's Protocol Code Number:GFM-Acadesine
    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:2013-04-19
    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 number2012-003120-21
    A.3Full title of the trial
    A phaseI-II trial of acadesine in ipss high and int 2 myelodysplastic syndromes, acute myeloid leukemia with 20-30% marrow blasts and chronic myelomonocytic leukemia type 2 not responding to azacitidine or decitabine for at least 6 courses or relapsing after a response
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Acadesine treatment of patients with myelodysplastic syndrome high risk,
    acute myeloid leukemia and chronic myelomonocytic leukemia type 2.
    A.4.1Sponsor's protocol code numberGFM-Acadesine
    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 SponsorGroupe Francophone des Myélodysplasies (GFM)
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAdvancell
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Yes
    D.2.5.1Orphan drug designation numberORPHA65972
    D.3 Description of the IMP
    D.3.1Product nameACADESINE
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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 SYNDROMES
    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 14.1
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1.Phase I:
    To determine the maximal tolerated dose (MTD) and dose limiting toxicities (DLTs) of increasing doses of IV Acadesine administered on D1, D3, D5, D8, D10 and D12 of a 28 to 56 day-course (cycle length based on the toxicities recovery).

    2.Phase II:
    To confirm safety and hematological toxicity in 18 additional patients
    E.2.2Secondary objectives of the trial
    1.Phase I:
    To determine response rates, as defined by the 2006 modified IWG criteria,
    •To evaluate response duration, time to IPSS progression, and loss of RBC transfusion independence in these patients.
    •To evaluate hospitalization duration, rates of rehospitalization for non-hematological toxicities, severe bleeding or febrile neutropenia.

    2.Phase II:
    To determine:
    •response rate as defined by the 2006 modified IWG criteria
    •toxicity profile and safety
    •response duration
    •rate of progression to AML
    •overall survival
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all of the following criteria to participate in the study:
    1.Myelodysplastic syndrome including the following categories of the WHO classification: refractory anemia with excess blasts (RAEB), non-proliferative chronic myelomonocytic leukemia (CMML) (leukocytes < 13 G/L but > 10% marrow blasts), AML with 20-30% marrow blasts (RAEB-T according to the FAB classification)

    2.Prior treatment with azacitidine or Decitabine for at least 6 courses without response (CR, PR, marrow CR or stable disease with HI according to IWG 2006 criteria) or relapsing after a response

    3.IPSS score >1 (IPSS: Int-2 or High);

    4.Age ≥ 18 years;

    5.Normal liver function, defined by total bilirubin and transaminases less than 1.5 time the upper limit of normal;

    6.Normal renal function, defined by creatinine less than 1.5 time the upper limit of normal, creatinine clearance ≥ 50 mL/min.

    7.Patient ineligible for allogeneic hematopoietic stem cell transplantation;

    8.Written informed consent;

    9.Patient must understand and voluntarily sign consent form;

    10.Patient must be able to adhere to the visit schedule as outlined in the study and follow protocol requirements;

    11.ECOG performance status between 0-2 at the time of screening;

    12.Women of childbearing potential must: Agree to use effective contraception without interruption throughout the study and for a further 1 month after the end of treatment;

    13.Men must: Agree to not conceive during the treatment and to use effective contraception during the treatment period (including periods of dose reduction or temporary suspension) and for a further 1 month after the end of treatment if their partner is of childbearing potential.
    E.4Principal exclusion criteria
    1.Severe infection or any other uncontrolled severe condition

    2.Significant cardiac disease - NYHA Class III or IV or having suffered a myocardial infarction in the last 6 months

    3.Less than 30 days since prior treatment with growth factors (EPO, G-CSF)

    4.Use of investigational agents within 30 days or any anticancer therapy within 2 weeks before study entry with the exception of hydroxyurea. The patient must have recovered from all acute toxicities from any previous therapy.

    5.Active cancer, or cancer during the year prior to trial entry other than basal cell carcinoma or carcinoma in situ of the cervix or breast;

    6.Patient already enrolled in another therapeutic trial of an investigational drug;

    7.HIV infection or active hepatitis B or C;

    8.Women who are or could become pregnant or who are currently breastfeeding;

    9.Any medical or psychiatric contraindication that would prevent the patient from understanding and signing the informed consent form;

    10.Patient eligible for allotransplantation.

    11.Known allergy to acadesine or any of its excipients

    12.No affiliation to an insurance system
    E.5 End points
    E.5.1Primary end point(s)
    Phase I:
    Safety of Acadesine. It will be analyzed after 1 cycle, and the decision to continue to the dose level above and to enroll 3 additional patients will be taken after assessment of the first patients enrolled by an independent data safety monitoring committee (DSMC), in association with the investigators.

    Phase II:
    Number of complete Remission (CR), Partial Remission (PR), Marrow CR and stable disease with Hematological Improvement (HI) according to IWG 2006 criteria (see appendix 6).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Phase I: Toxicity, evaluated using NCI-CTCAE V4.0 criteria.

    Phase II: response, according to IWG 2006 criteria.
    E.5.2Secondary end point(s)
    •Total percentage of response, including all patients achieving CR, PR, marrow CR or stable disease with hematologic improvement, evaluated according to IWG 2006 criteria (after 2, 4 and 6 treatment cycles).
    •Duration of response, measured from the date an objective response was achieved to the date of relapse or progression or the date of last contact if no event occurred.
    E.5.2.1Timepoint(s) of evaluation of this end point
    to determine:
    - the duration of the response
    - overall survival
    - the safety profile
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Testing three levels of dose of Acadesine
    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 No
    E.8.1.2Open Yes
    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.8.3 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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    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 months
    E.8.9.1In the Member State concerned days
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    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 state30
    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 Decision2013-03-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-04-15
    P. End of Trial
    P.End of Trial StatusOngoing
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