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    Summary
    EudraCT Number:2022-001253-23
    Sponsor's Protocol Code Number:4202-ONC-203
    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:2022-08-10
    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 number2022-001253-23
    A.3Full title of the trial
    A Phase 2 Open-Label Study to Evaluate Etavopivat for the Treatment of Anemia in Patients with Myelodysplastic Syndromes (MDS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Etavopivat study in the treatment of Anemia in Patients with Myelodysplastic Syndromes (MDS)
    A.4.1Sponsor's protocol code number4202-ONC-203
    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 SponsorForma 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 supportForma Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationForma Therapeutics, Inc.
    B.5.2Functional name of contact pointMedical Lead
    B.5.3 Address:
    B.5.3.1Street Address300 North Beacon Street, Suite 501
    B.5.3.2Town/ cityWatertown
    B.5.3.3Post code02472
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1-857-327-8141
    B.5.6E-mail4202-203Clinical@formatherapeutics.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/3/20/2335 EMA-OD-0000033107
    D.3 Description of the IMP
    D.3.1Product nameEtavopivat
    D.3.2Product code FT-4202
    D.3.4Pharmaceutical form Tablet
    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 INNEtavopivat
    D.3.9.2Current sponsor codeFT-4202
    D.3.9.4EV Substance CodeSUB207913
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200.0
    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
    Anemia of myelodysplastic syndromes (MDS), inherited hemolytic anemias, and other refractory anemias
    E.1.1.1Medical condition in easily understood language
    Adult patients with very low, low risk, or intermediate risk MDS per the International Prognostic Scoring System-Revised (IPSS-R) classification
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess hematologic improvement based on an erythroid response (HI E) ≥ 8 weeks duration in patients with MDS after 16 weeks of etavopivat treatment
    E.2.2Secondary objectives of the trial
    • To assess HI-E ≥ 8 weeks duration in this population of patients after 24 and 48 weeks of etavopivat treatment
    • To assess HI-E ≥ 16 weeks duration in this population of patients after 24 and 48 weeks of etavopivat treatment
    • To assess the safety and tolerability of etavopivat in patients with MDS
    • To assess additional measures demonstrating potential clinical benefit
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient has provided documented informed consent; the informed consent form (ICF) must be reviewed and signed by each patient prior to any study-related assessments/procedures being conducted.
    2. Age ≥ 18 years at time of first dose
    3. Patients, if female and of childbearing potential, must agree to use acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and who if male are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug.
    4. Documented diagnosis of idiopathic/de novo MDS according to WHO classification that meets IPSS-R classification of very low, low, or intermediate risk disease, and:
    • < 5% blasts and no Auer rods in bone marrow based on local pathology review
    • < Intermediate risk cytogenetic abnormalities per IPSS-R
    5. Anemia defined as:
    • NTD: Subjects with mean Hb concentration < 10.0 g/dL of 2 measurements (1 performed within 3 days prior to Day 1 and the other performed 7 to 28 days prior to Day 1, not influenced by RBC transfusion within 7 days of measurement) and < 3 RBC transfusions for anemia in the prior 16 weeks before Day 1 of etavopivat dosing
    OR
    • Transfusion dependent: Subjects having received ≥ 3 units of RBCs for the treatment of anemia within 16 weeks prior to Day 1
    6. Serum erythropoietin level > 200 U/L, OR, if ≤ 200 U/L, subject is non-responsive, refractory, or intolerant to erythropoiesis-stimulating agents, or erythropoiesis-stimulating agents are contraindicated or unavailable.
    7. Eastern Cooperative Oncology Group performance status of ≤ 2
    8. Subject is non-responsive, refractory, or intolerant to luspatercept, or luspatercept is contraindicated or not indicated.
    9. No alternative treatment options are available and/or appropriate for the subject, at the discretion of the investigator.
    10. Patient is willing and able to adhere to the study visit schedule and other protocol requirements
    E.4Principal exclusion criteria
    MDS History
    1. MDS associated with del 5q cytogenetic abnormality and known TP53 abnormality
    2. Therapy-associated MDS (eg. t-MDS) that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases
    3. Known history of AML
    Medical Conditions
    4. Female who is breast feeding or pregnant
    5. Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
    6. Absolute neutrophil count < 500/µL (0.5 × 109/L)
    7. Platelet count < 50,000/µL (50 × 109/L) without transfusion within 2 weeks
    8. Hepatic dysfunction characterized by:
    • Alanine aminotransferase (ALT) > 5.0 × ULN
    • Total bilirubin > 3.0 × ULN
    • History of cirrhosis
    9. Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the local laboratory < 30 mL/min/1.73 m2) or on chronic dialysis.
    10. Patients with clinically significant and active bacterial, fungal, parasitic, or viral infection.
    • Patients with acute bacterial, fungal, parasitic, or viral infection requiring systemic therapy should delay Screening/ enrollment until active therapy has been completed.
    • Patients with acute viral infections without available therapies (eg, coronavirus disease 2019 [COVID-19]) should delay Screening/ enrollment until the acute infection has resolved.
    Note: Infection prophylaxis is allowed (see concomitant medication restrictions).
    11. Known human immunodeficiency virus (HIV) positivity
    12. Active infection with hepatitis B virus (hepatitis B surface antigen [HepBsAg] and hepatitis B core antibody [HepBcAb] positive)
    13. Active hepatitis C infection
    14. History of malignancy, other than MDS, within the past 2 years prior to treatment Day 1 requiring systemic chemotherapy and/or radiation.
    • Patients with malignancy considered surgically cured are eligible (eg, non-melanoma skin cancer, carcinoma in situ of the cervix, or carcinoma in situ of the breast)
    • Patients with incidental histologic findings of prostate cancer (T1a or T1b) are eligible
    15. History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:
    • Unstable angina pectoris or myocardial infarction or elective coronary intervention
    • Heart disease, heart failure as classified by the New York Heart Association classification 3 or higher, or significant arrhythmia requiring treatment,
    • Pulmonary fibrosis or pulmonary hypertension which are clinically significant ie, ≥ Grade 3 National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 (or higher)
    16. Uncontrolled hypertension, defined as repeated elevation of diastolic blood pressure ≥ 100 mmHg despite adequate treatment
    17. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable).
    Prior/Concomitant Therapy
    18. Prior treatment with azacitidine (injectable or oral) or decitabine
    19. Use of erythropoietin, other hematopoietic growth factor treatment or lenalidomide within 30 days of starting study treatment or anticipated need for such agents during the study.
    20. Prior use of luspatercept:
    • NTD patients must not have received luspatercept within 30 days prior to Day 1 treatment
    • TD patients must not have received luspatercept within 16 weeks prior to Day 1 treatment
    21. Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP)3A4/5 (see Appendix F) within 2 weeks of starting study treatment or anticipated need for such agents during the study.
    22. Prior allogeneic or autologous stem cell transplant
    23. Initiation of a new chelation therapy within 3 months before the first dose of study treatment
    Prior/Concurrent Clinical Study Experience
    24. Participated in another clinical trial of an investigational agent (or medical device) within 30 days or 5 half-lives of date of informed consent, whichever is longer, or is currently participating in another trial of an investigational agent (or medical device).
    Other Exclusions
    25. Medical, psychological, or behavioral conditions, which, in the opinion of the Investigator, may preclude safe participation, confound study interpretation, interfere with compliance, or preclude informed consent.
    E.5 End points
    E.5.1Primary end point(s)
    • The HI-E ≥ 8 weeks after 16 weeks of etavopivat
    This endpoint will be based on the combined incidence of:
    o Non-transfusion dependent (NTD) patients: ≥ 1.5 g/dL increase in Hb from baseline maintained ≥ 8 consecutive weeks
    o Low transfusion burden (LTB) patients: absence of any transfusion for ≥ 8 consecutive weeks
    o High transfusion burden (HTB) patients: reduction by ≥ 50% of red blood cell (RBC) units for ≥ 8 consecutive weeks
    E.5.1.1Timepoint(s) of evaluation of this end point
    16 week of treatment
    E.5.2Secondary end point(s)
    • The HI-E ≥ 8 weeks after 24 and 48 weeks of etavopivat
    This endpoint will be based on the combined incidence of:
    o NTD patients: ≥ 1.5 g/dL increase in Hb from baseline maintained ≥ 8 consecutive weeks
    o LTB patients: absence of any transfusion for ≥ 8 consecutive weeks
    o HTB patients: reduction by ≥ 50% of RBC units for ≥ 8 consecutive weeks

    • The HI-E ≥ 16 weeks after 24 and 48 weeks of etavopivat
    This endpoint will be based on the combined incidence of:
    o NTD patients: ≥ 1.5 g/dL increase in Hb from baseline maintained ≥ 16 consecutive weeks
    o LTB patients: absence of any transfusion for ≥ 16 consecutive weeks
    o HTB patients: reduction by ≥ 50% of RBC units for ≥ 16 consecutive weeks
    • Incidence of AEs, serious adverse events (SAEs), and AEs related to etavopivat
    • Number of premature discontinuations, dose interruptions, and dose reductions
    • Changes from baseline in vital signs, clinical laboratory assessments, and physical examination findings
    • Overall response rate (2006 International Working Group [IWG] Criteria)
    • Duration of response (2006 IWG Criteria)
    • Reduction in RBC transfusions and rate of RBC transfusion independence in patients with LTB or HTB at study entry
    • Increase in neutrophils and/or platelets
    • Decrease in ferritin
    • Decrease in iron chelation therapy
    • Overall survival
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary objectives:
    24 and 48 weeks
    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 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.8.3 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 Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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
    Canada
    United States
    France
    Germany
    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 will end when all patients who entered the treatment period complete study treatment and the End of Study (EOS) visit or discontinue study participation
    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 days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months4
    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: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 30
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 45
    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)
    There will not be any specific treatment will be provided to Subject once they ended the participation
    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 Decision2022-09-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-09-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2024-07-15
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