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    Summary
    EudraCT Number:2013-000672-15
    Sponsor's Protocol Code Number:09-07
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-04-22
    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 number2013-000672-15
    A.3Full title of the trial
    A Single-arm Study to Assess the Efficacy and Safety of Oral Rigosertib in Transfusion-dependent, Low or Intermediate-1, Myelodysplastic Syndrome Patients Based on the International Prognostic Scoring System
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the efficacy and safety of oral rigosertib in patients with myelodysplastic syndrome (a blood disease) who are dependent on transfusions.
    A.4.1Sponsor's protocol code number09-07
    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 SponsorOnconova Therapeutics, Inc.
    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 supportOnconova Therapeutics, Inc.
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportLeukemia and Lymphoma Society
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOckham Oncology
    B.5.2Functional name of contact pointProject Manager 09-07
    B.5.3 Address:
    B.5.3.1Street AddressThe Logan Building, Roslin Biocentre
    B.5.3.2Town/ cityRoslin, Midlothian
    B.5.3.3Post codeEH25 9TT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44131200 6320
    B.5.5Fax number+44131200 6322
    B.5.6E-mailcontact@ockham.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/12/987
    D.3 Description of the IMP
    D.3.1Product namerigosertib
    D.3.2Product code ON 01910.Na
    D.3.4Pharmaceutical form Capsule, soft
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNrigosertib
    D.3.9.1CAS number 592542-60-4
    D.3.9.2Current sponsor codeON 01910.NA
    D.3.9.4EV Substance CodeSUB32475
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number70 to 280
    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
    E.1.1.1Medical condition in easily understood language
    Myelodysplastic syndromes (MDSs) are a group of disorders in which the red blood cells, white blood cells and platelets produced by the bone marrow do not grow and mature normally.
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10028533
    E.1.2Term Myelodysplastic syndrome
    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
    The primary efficacy objective is to determine the onset of hematological improvement according to the 2006 International Working Group (IWG) criteria.
    E.2.2Secondary objectives of the trial
    The secondary efficacy objectives are to evaluate rigosertib with respect to:
    Efficacy:
    - Overall response (complete and partial responses) according to 2006 IWG criteria including the onset and duration of response;
    - Bone marrow response according to 2006 IWG criteria.
    Safety will also be evaluated.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Male and female patients who meet all of the following criteria are eligible for enrollment in the trial:
    a. ≥18 years of age;
    b. Diagnosis of MDS according to World Health Organization (WHO) criteria (Appendix 2) or French-American-British (FAB) classification, that must be confirmed by BM aspirate and/or biopsy within 6 weeks prior to Screening;
    c. MDS classified as Low risk or Int-1 risk, according to IPSS classification; in addition, patients should never have been classified as Int-2 or High-risk since their MDS was diagnosed;
    d. Transfusion dependency defined by transfusion of at least 4 units of RBC within 56 days (ie, 8 weeks) before Screening (pre-transfusion Hgb values values must be ≤ 9 g/dL to be taken into account);
    e. Refractory to 8- to 12-week course of ESA administered within the past 2 years before enrollment, or EPO level > 500 mU/mL and off ESA for at least 8 weeks before Screening;
    f. Off all other treatments for MDS (AZA, decitabine, lenalinomide, ESA, chemotherapy, immunotherapy) for at least 2 weeks prior to Screening;
    g. ECOG performance status of 0, 1 or 2;
    h. Willing to adhere to the prohibitions and restrictions specified in this protocol;
    i. The patient must sign an informed consent form (ICF) indicating that s/he understands the purpose of, and procedures required for, the study and is willing to participate.
    E.4Principal exclusion criteria
    Patients with any of the following will not be enrolled in the study:
    a. Ongoing clinically significant anemia due to factors such as iron, vitamin B12, or folate deficiencies, auto-immune or hereditary hemolysis, or gastrointestinal (GI) bleeding;
    b. Serum ferritin < 50 ng/mL;
    c. Hypoplastic MDS (cellularity <10%);
    d. Any active malignancy within the past year, except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix or breast;
    e. Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia;
    f. Active infection not adequately responding to appropriate therapy;
    g. Total bilirubin ≥ 2.0 mg/dL not related to hemolysis or Gilbert’s disease;
    h. Alanine transaminase (ALT) or aspartate transaminase (AST) ≥ 2.5 x the upper limit of normal (ULN);
    i. Serum creatinine ≥ 2.0 mg/dL;
    j. Ascites requiring active medical management including paracentesis;
    k. Hyponatremia (defined as serum sodium value of < 130 mEq/L);
    l. Female patients who are pregnant or lactating;
    m. Patients who are unwilling to follow strict contraception requirements (including 2 reliable methods in combination: one non-hormonal, highly-reliable method [diaphragm, condoms with spermicidal foam or jelly, or sterilization] plus one additional reliable method [birth control pills, intrauterine device, contraceptive injections, or contraceptive patches]) before entry and throughout the study, up to and including the 30-day nontreatment follow-up period;
    n. Female patients with reproductive potential who do not have a negative urine pregnancy test at Screening;
    o. Major surgery without full recovery or major surgery within 3 weeks of Screening;
    p. Uncontrolled hypertension (defined as a systolic pressure ≥ 160 mmHg and/or a diastolic pressure ≥ 110 mmHg);
    q. New onset seizures (within 3 months prior to the first dose of rigosertib) or poorly controlled seizures;
    r. Any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy;
    s. Chronic use (> 2 weeks) of corticosteroids (> 10 mg/24 hr equivalent prednisone) within 4 weeks of Screening;
    t. Investigational therapy within 4 weeks of Screening;
    u. Psychiatric illness or social situation that would limit the patient’s ability to tolerate and/or comply with study requirements.
    E.5 End points
    E.5.1Primary end point(s)
    Haematological improvement will be assessed according to the 2006 IWG criteria to include:
    1. RBC transfusion independence, ie no RBC transfusions administered over a period of 8 consecutive weeks.
    2. erythroid response, ie transfusion reduction of at least 4 RBC units over 8 weeks after Baseline/First Dose compared to the number of transfusions in the 8 weeks before treatment
    3. neutrophil or platelet response
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. RBC transfusion independence and 2. erythroid response:
    transfusion history to be recorded at screening, baseline (Day 1) and weekly throughout study for a maximum period of 48 weeks.
    3. neutrophil or platelet response: complete blood count will be measured at screening, baseline (Day 1) and every week throughout study.
    E.5.2Secondary end point(s)
    Efficacy response will be assessed according to the following outcomes:
    1. Number and dates of transfusions of red blood cell (RBC) units
    2. Hgb values before each RBC transfusion
    3. Number and dates of transfusions of platelet (PLT) units
    4. Platelet counts before each PLT transfusion
    5. Overall survival
    6. Blastic response in bone marrow (BM)
    7. Change in BM cytogenetics
    8. Changes in Hgb and in RBC transfusion requirements
    9. Changes in absolute neutrophil count (ANC)
    10. Changes in PLT count and PLT transfusion requirements

    Safety will be assessed as treatment emergent adverse events
    E.5.2.1Timepoint(s) of evaluation of this end point
    1, 3: Transfusion history to be recorded at screening, baseline (Day 1) and weekly throughout study;
    2, 4, 8, 9, 10: Complete blood count will be measured at screening, baseline (Day 1) and every week throughout study;
    5: throughout study
    6: Bone marrow aspirate and/or biopsy will be performed at screening and for patients with more than 5% pre-treatment BMBL every 15 weeks throughout study;
    7: Bone marrow cytogenetics will be performed at screening and if abnormal will repeated every 15 weeks throughout study

    Safety will be monitored throughout study
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    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) 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 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.8.2.4Number 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    France
    Germany
    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
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months4
    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.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 40
    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)
    Normal treatment of the condition
    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 Decision2013-05-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-03-18
    P. End of Trial
    P.End of Trial StatusCompleted
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