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    Summary
    EudraCT Number:2020-000869-17
    Sponsor's Protocol Code Number:CMBG453F12201
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-07
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2020-000869-17
    A.3Full title of the trial
    A phase Ib/II, open label study of sabatolimab as a treatment for patients with acute myeloid leukemia and presence of measurable residual disease after allogenic stem cell transplantation
    Studio di Fase Ib/II, in aperto, con sabatolimab nel trattamento dei pazienti con leucemia mieloide acuta e presenza di malattia residua misurabile dopo trapianto allogenico di cellule staminali
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    sabatolimab as a treatment for patients with acute myeloid leukemia and presence of measurable residual disease after allogeneic stem cell transplantation
    sabatolimab come trattamento in pazienti con leucemia mieloide acuta e presenza di malattia residua misurabile dopo trapianto allogenico di cellule staminali
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    A.4.1Sponsor's protocol code numberCMBG453F12201
    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 SponsorNOVARTIS PHARMA AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Farma
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressLargo Umberto Boccioni, 1 -
    B.5.3.2Town/ cityOriggio (VA)
    B.5.3.3Post code21040
    B.5.3.4CountryItaly
    B.5.4Telephone number0296543289
    B.5.5Fax number029659066
    B.5.6E-mailinfo.studiclinici@novartis.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.2.1.1.1Trade name -
    D.2.1.1.2Name of the Marketing Authorisation holder-
    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 namesabatolimab
    D.3.2Product code [MBG453]
    D.3.4Pharmaceutical form Concentrate 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 INNsabatolimab
    D.3.9.2Current sponsor codeMBG453
    D.3.9.3Other descriptive nameMBG453
    D.3.9.4EV Substance CodeSUB178459
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 Yes
    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.IMP: 2
    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 Yes
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameazacitidine
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Powder for suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    Intravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAZACITIDINA
    D.3.9.1CAS number 320-67-2
    D.3.9.2Current sponsor code-
    D.3.9.4EV Substance CodeSUB05624MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Participants with AML/secondary AML who are in complete remission with positive measurable residual disease post-allogeneic hematopoietic stem cell transplantation (MRD+ post aHSCT)
    -
    E.1.1.1Medical condition in easily understood language
    Participants with AML/secondary AML who are in complete remission with positive measurable residual disease post-allogeneic hematopoietic stem cell transplantation (MRD+ post aHSCT)
    -
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10000886
    E.1.2Term Acute myeloid leukemia
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    adults only (safety run-in & expansion): to evaluate preliminary efficacy of MBG453 as monotherapy and in combination with aza on prevention of hematologic relapse by assessing proportion of adult participants with AML and MRD+ post aHSCT who remain with no evidence of hematologic relapse after 6 cycles adults only safety run-in: determine if MBG453 as monotherapy at the 2 tested dose levels leads to unacceptable toxicity when administered to adult participants who are in complete remission but are MRD+ post aHSCT adolescents only: to determine if MBG453 as monotherapy at the recommended dose level for adults leads to unacceptable toxicity when administered to adolescent participants who are in complete remission but are MRD+ post aHSCT
    -
    E.2.2Secondary objectives of the trial
    1. to assess the incidence of Grade III or IV aGvHD and moderate to severe cGvHD
    2. to characterize PK of MBG453
    3. to assess GvHD-free/relapse free survival (GRFS)
    4. to assess relapse-free survival (RFS)
    5. to determine safety and tolerability of MBG453 in monotherapy and in combination with azacitidine
    6. To assess severe immune-related adverse events not attributed to GvHD
    7. To assess the proportion of participants with centrally confirmed MRD+ at screening who become MRD- during the first 6 cycles of study treatment
    -
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed informed consent must be obtained prior to participation in the study.
    2. At the date of signing the informed consent form (ICF), eligible participants must be = 18 years for the adult cohorts; and = 12 years old but < 18 years old for the adolescent cohort (cohort 5), which will open after completion of Safety Run-in.
    3. Diagnosis of AML/secondary AML and received one prior aHSCT performed to control AML
    4. Participants in complete remission (< 5% bone marrow blasts, absence of circulating blasts, and absence of extramedullary disease) with MRD positivity by local assessment, at any time between day 100 and day 365 after aHSCT.
    5. Ability to provide a fresh bone marrow aspirate sample collected within 28 days from enrollment/randomization, and immediately shipped to a Novartis designated central laboratory for MRD testing.
    6. Systemic GvHD prophylaxis or treatment [immunosuppressive treatment (IST)] completely tapered for at least two weeks prior to study entry. Prednisone dose = 5 mg/day or equivalent corticosteroid dose is allowed.
    7. Participants who are found with MRD positivity while still on or tapering systemic GvHD prophylaxis or treatment, MRD positivity must be re-confirmed at least 2 week after the last dose of IST
    8. For the adult cohorts, participants must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.
    For the adolescent cohort, participants must have a Karnofsky (age = 16
    years) or Lansky (age < 16 years) performance status score = 50%.
    Additional inclusion criteria as per full protocol may apply
    -
    E.4Principal exclusion criteria
    1. Prior exposure to TIM-3 directed therapy at anytime
    2. History of severe hypersensitivity reactions to any ingredient of study drug(s) (azacitidine, sabatolimab) or monoclonal antibodies (mAbs) and/or their excipients
    3. Active Hepatitis B (HBV) or Hepatitis C (HCV) infection. Participants whose disease is controlled under antiviral therapy should not be excluded.
    4. Active acute GvHD grade III-IV according to standard criteria (Harris 2016).
    5. Active moderate chronic GvHD of the lungs according to NIH consensus criteria. Active severe chronic GvHD according to NIH consensus criteria.
    6. History of another primary malignancy that is currently clinically significant or currently requires active intervention.
    7. Any concurrent severe and/or active uncontrolled infection requiring parenteral antibacterial, antiviral or antifungal therapy (such as severe pneumonia, meningitis, or septicemia
    8. Active autoimmune disease requiring systemic therapy (e.g. corticosteroids). Topical, inhaled, nasal and ophthalmic steroids are not prohibited. Replacement corticosteroids therapy is allowed and not considered a form of systemic treatment
    9. Live vaccine administered within 30 days prior to the first day of study treatment (C1D1)
    10. Other concurrent severe and/or uncontrolled medical conditions (e.g. uncontrolled diabetes mellitus, chronic obstructive or chronic restrictive pulmonary disease including dyspnoea at rest from any cause) or history of serious organ dysfunction or disease involving the heart, kidney, or liver
    Additional exclusion criteria as per full protocol may apply
    -
    E.5 End points
    E.5.1Primary end point(s)
    1. incidence of dose limiting toxicities (safety run-in in adult cohorts 1 and 2 only)
    2. percentage of adult subjects with absence of haematologic relapse per investigator assessment (safety run-in and expansion)
    3. incidence of dose limiting toxicities (safety confirmation in adolescent cohort 5 only)
    -
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. from Cycle 1 Day 1 to end of Cycle 6 (cycle = 28 days)
    2. from Cycle 1 Day 1 to end of Cycle 6 (cycle = 28 days)
    3. from Cycle 1 Day 1 to end of Cycle 2 (cycle = 28 days)
    -
    E.5.2Secondary end point(s)
    1. Incidence of Grade III or IV acute Graft versus Host Disease (aGvHD)
    2. incidence of moderate to severe chronic Graft versus Host Disease (cGvHD)
    3. Peak of serum concentration (Cmax) sabatolimab
    4. Trough serum concentration (Cmin) sabatolimab
    5. Anti-drug antibody (ADA) prevalence on-treatment
    6. ADA prevalence at baseline
    7. time from start of treatment to the date of first documented GvHDfree/ relapse-free survival
    8. time from start of treatment to the date of first documented haematologic relapse or death due to any cause, whichever occurs first
    9. incidence of Grade 3 immune-related adverse events not attributed to GvHD
    10. percentage of participants with measurable residual disease (MRD) positive at baseline (BL) who become MRD negative
    -
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. from start of treatment to up to 36 m from LPFT
    2. from start of treatment to up to 36 m from LPFT
    3. C1 D1 or 5 (end of infusion) and C3 D1 or 5 (EoI) for adult cohorts and C1 D1 (EoI) and C3 D1 (end of infusion) for adolescent cohort (cycle = 28d)
    4. D1 or D5 of C1, 3,6 and 24 for adult cohorts and D1 of C1, 2, 3, 6, 9, 12, 18 and 24 for adolescent cohort, and through treatment completion, and average of 15 m
    5. through study till 150d safety followup
    6. prior to first dose of MBG453 on C1
    7. every 2 w until W9, every 4 w until W25, and then every 8 w until end of treatment and then every 12 w for up to 36 m from LPFT
    8. every 4 w until W13, then every 12 w till W49, every 24 w after up to 36 m from LPFT
    9. through study till 150d safety FU
    10. start of treatment to end C6
    -
    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) 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
    -
    -
    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 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 trial3
    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 EEA7
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    France
    Germany
    Italy
    Spain
    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
    LVLS
    -
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days21
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days21
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 32
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 21
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 59
    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)
    none
    -
    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 Decision2021-07-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-03-16
    P. End of Trial
    P.End of Trial StatusOngoing
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