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    Clinical Trial Results:
    Phase Ib/II, open label study of sabatolimab as a treatment for patients with acute myeloid leukemia and presence of measurable residual disease after allogeneic stem cell transplantation. Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com for complete trial results.

    Summary
    EudraCT number
    		 2020-000869-17
    Trial protocol
    		 FR   DE   IT  
    Global end of trial date
    22 Aug 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    09 Mar 2025
    First version publication date
    09 Mar 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CMBG453F12201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04623216
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma, AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharmaceuticals, 41 613241111, novatis.email@novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharmaceuticals, 41 613241111, novatis.email@novartis.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    22 Aug 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    22 Aug 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    For adults only (from the safety run-in part): To determine whether sabatolimab as monotherapy at the two tested dose levels (400 mg and 800 mg Q4W) leads to an unacceptable level of toxicity when administered to adult participants with AML who are in complete remission but are MRD+ post-aHSCT. For adults only (from both safety run-in and expansion parts): To evaluate preliminary efficacy of sabatolimab (at the recommended dose level for expansion) as monotherapy and in combination with azacitidine on prevention of hematologic relapse by assessing the proportion of adult participants with AML and MRD+ post-aHSCT,) who remain with no evidence of hematologic relapse after 6 cycles of study treatment. For adolescents only: To determine whether sabatolimab as monotherapy at the recommended dose level for adults leads to an unacceptable level of toxicity when administered to adolescent participants with AML who are in complete remission but are MRD+ post-aHSCT.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    14 Sep 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 3
    Country: Number of subjects enrolled
    Germany: 8
    Country: Number of subjects enrolled
    Italy: 10
    Country: Number of subjects enrolled
    Spain: 3
    Worldwide total number of subjects
    24
    EEA total number of subjects
    24
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    1
    Adults (18-64 years)
    16
    From 65 to 84 years
    7
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 24 participants were enrolled in this study: 21 in Safety run-in sabatolimab monotherapy and 3 in Expansion part.

    Pre-assignment
    Screening details
    		 Due to the recruitment halt by Novartis, recruitment in the expansion phase was not completed.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Sabatolimab 400mg mono adults
    Arm description
    		 Safety run-in cohort: Adult participants in this arm received sabatolimab 400mg only.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sabatolimab
    Investigational medicinal product code
    MBG453
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    sabatolimab 400 mg/4ml (MBG453 400 mg liquid in vial 4 ml)

    Arm title
    		 Sabatolimab 800mg mono adults
    Arm description
    		 Safety run-in cohort: Adult participants in this arm received sabatolimab 800mg only.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sabatolimab
    Investigational medicinal product code
    MBG453
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    sabatolimab 800 mg/4ml (MBG453 400 mg liquid in vial 4 ml)

    Arm title
    		 Sabatolimab 800mg + Azacitidine adults
    Arm description
    		 Expansion cohort: Adult participants in this arm received sabatolimab 800 mg + azacitidine combination.
    Arm type
    		 Experimental

    Investigational medicinal product name
    azacitidine
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use, Subcutaneous use
    Dosage and administration details
    azacitidine 100 mg (azacitidine 100 mg lyophilizate in vial)

    Investigational medicinal product name
    Sabatolimab
    Investigational medicinal product code
    MBG453
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    sabatolimab 800 mg/4ml (MBG453 400 mg liquid in vial 4 ml)

    Arm title
    		 Sabatolimab 800mg mono adolescent
    Arm description
    		 Adolescent safety cohort: ≥12 to < 18-year-old adolescent participants in this arm received sabatolimab 800mg only.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sabatolimab
    Investigational medicinal product code
    MBG453
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    sabatolimab 800 mg/4ml (MBG453 400 mg liquid in vial 4 ml)

    Number of subjects in period 1
    		Sabatolimab 400mg mono adults Sabatolimab 800mg mono adults Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adolescent
    Started
    10
    11
    2
    1
    Didn't enter post-trtmnt follow-up (f/u)
    7
    9
    2
    1
    Entered post-treatment f/u, discontinued
    3
    2
    0
    0
    Completed treatment
    2
    0
    0
    0
    Completed
    1
    7
    0
    1
    Not completed
    9
    4
    2
    0
         Adverse event, serious fatal
    5
    2
    -
    -
         Terminated by Sponsor
    3
    2
    2
    -
         Physician decision
    1
    -
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Sabatolimab 400mg mono adults
    Reporting group description
    		 Safety run-in cohort: Adult participants in this arm received sabatolimab 400mg only.

    Reporting group title
    Sabatolimab 800mg mono adults
    Reporting group description
    		 Safety run-in cohort: Adult participants in this arm received sabatolimab 800mg only.

    Reporting group title
    Sabatolimab 800mg + Azacitidine adults
    Reporting group description
    		 Expansion cohort: Adult participants in this arm received sabatolimab 800 mg + azacitidine combination.

    Reporting group title
    Sabatolimab 800mg mono adolescent
    Reporting group description
    		 Adolescent safety cohort: ≥12 to < 18-year-old adolescent participants in this arm received sabatolimab 800mg only.

    Reporting group values
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adults Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adolescent Total
    Number of subjects
    		 10 11 2 1 24
    Age Categorical
    Units: Participants
        Category 1 : 12 - <18 years
    		 0 0 0 1 1
        Category 1 : 18 - <65 years
    		 8 7 1 0 16
        Category 1 : 65 - <85 years
    		 2 4 1 0 7
    Sex: Female, Male
    Units: Participants
        Female
    		 5 6 2 1 14
        Male
    		 5 5 0 0 10
    Race/Ethnicity, Customized
    Units: Subjects
        White
    		 9 9 2 1 21
        Unknown
    		 1 2 0 0 3

    End points

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    End points reporting groups
    Reporting group title
    Sabatolimab 400mg mono adults
    Reporting group description
    		 Safety run-in cohort: Adult participants in this arm received sabatolimab 400mg only.

    Reporting group title
    Sabatolimab 800mg mono adults
    Reporting group description
    		 Safety run-in cohort: Adult participants in this arm received sabatolimab 800mg only.

    Reporting group title
    Sabatolimab 800mg + Azacitidine adults
    Reporting group description
    		 Expansion cohort: Adult participants in this arm received sabatolimab 800 mg + azacitidine combination.

    Reporting group title
    Sabatolimab 800mg mono adolescent
    Reporting group description
    		 Adolescent safety cohort: ≥12 to < 18-year-old adolescent participants in this arm received sabatolimab 800mg only.

    Primary: Rate of dose limiting toxicities (Safety Run-in in adult sabatolimab 400mg & 800mg only)

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    End point title
    		 Rate of dose limiting toxicities (Safety Run-in in adult sabatolimab 400mg & 800mg only) [1] [2]
    End point description
    Assessment of tolerability of sabatolimab in adults and adolescents in the post allogenic stem cell transplantation setting. This was determined by the number of participants with at least one event - All grades. A dose-limiting toxicity (DLT) is defined as an adverse event or abnormal laboratory value considered by the Investigator to be at least possibly related to sabatolimab as a single contributor that occurs during the DLT observation period and meets the severity criteria as per protocol.
    End point type
    Primary
    End point timeframe
    From Cycle 1 Day 1 to end of Cycle 2; Cycle =28 Days
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No formal statistical test for this endpoint
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No formal statistical test for this endpoint
    End point values
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adults
    Number of subjects analysed
    8
    10
    Units: Participants
    0
    1
    No statistical analyses for this end point

    Primary: Percentage of adult subjects with absence of hematologic relapse per Investigator assessment (Safety Run-in and Expansion)

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    End point title
    		 Percentage of adult subjects with absence of hematologic relapse per Investigator assessment (Safety Run-in and Expansion) [3]
    End point description
    The percentage of adult participants for whom no evidence of hematologic relapse (no evidence of bone marrow blasts ≥5%, no evidence of reappearance of blasts in the blood; no evidence of development of extramedullary disease) has been documented after 6 cycles of study treatment or earlier discontinuation at the recommended dose of MBG453 800 mg.
    End point type
    Primary
    End point timeframe
    From Cycle 1 Day 1 to end of Cycle 6; Cycle = 28 Days
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No formal statistical test for this endpoint
    End point values
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adults Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adolescent
    Number of subjects analysed
    0 [4]
    11
    2
    0 [5]
    Units: Percentage of participants
        number (confidence interval 95%)
    ( to )
    36.4 (10.9 to 69.2)
    999 (999 to 999)
    ( to )
    Notes
    [4] - No subjects in this group were analyzed for this endpoint
    [5] - No subjects in this group were analyzed for this endpoint
    No statistical analyses for this end point

    Primary: Rate of dose limiting toxicities (Safety confirmation in adolescent cohort only)

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    End point title
    		 Rate of dose limiting toxicities (Safety confirmation in adolescent cohort only) [6] [7]
    End point description
    Assessment of tolerability of sabatolimab in adolescent participants in the post allogeneic stem cell transplantation setting. This was determined by the number of participants with at least one event - All grades. A dose-limiting toxicity (DLT) is defined as an adverse event or abnormal laboratory value considered by the Investigator to be at least possibly related to sabatolimab as a single contributor that occurs during the DLT observation period and meets the severity criteria as per protocol.
    End point type
    Primary
    End point timeframe
    From Cycle 1 Day 1 to end of Cycle 2; Cycle =28 Days
    Notes
    [6] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No formal statistical test for this endpoint
    [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: No formal statistical test for this endpoint
    End point values
    		 Sabatolimab 800mg mono adolescent
    Number of subjects analysed
    1
    Units: Participants
    999
    No statistical analyses for this end point

    Secondary: Incidence of moderate to severe Chronic GVHD (cGvHD)

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    End point title
    		 Incidence of moderate to severe Chronic GVHD (cGvHD)
    End point description
    Assessment of the treatment emergent moderate or severe cGvHD. Chronic GvHD: Moderate chronic GvHD of the lungs, Severe chronic GvHD.
    End point type
    Secondary
    End point timeframe
    From start of treatment up to 36 months from last patient first treatment
    End point values
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adults Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adolescent
    Number of subjects analysed
    10
    11
    2
    1
    Units: Participants
    999
    999
    999
    999
    No statistical analyses for this end point

    Secondary: Incidence of grade III or IV acute Graft versus Host Disease (aGvHD)

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    End point title
    		 Incidence of grade III or IV acute Graft versus Host Disease (aGvHD)
    End point description
    Assessment of the treatment emergent grade III or IV aGvHD. Acute GvHD: Grade IV acute GvHD, Stage ≥3 lower GI acute GvHD (consistent with Grade III acute GvHD) or Stage ≥3 liver acute GvHD (consistent with Grade III GvHD).
    End point type
    Secondary
    End point timeframe
    From start of treatment up to 36 months from last patient first treatment
    End point values
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adults Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adolescent
    Number of subjects analysed
    10
    11
    2
    1
    Units: Participants
    999
    999
    999
    999
    No statistical analyses for this end point

    Secondary: Peak of Serum Concentration (Cmax) sabatolimab

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    End point title
    		 Peak of Serum Concentration (Cmax) sabatolimab
    End point description
    Cmax is the maximal serum concentration of sabatolimab.
    End point type
    Secondary
    End point timeframe
    Cycle 1 Day 5 (end of infusion) and Cycle 3 Day 1 or Day 5 (end of infusion) and Cycle 24 Day 1 (end of infusion)
    End point values
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adults Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adolescent
    Number of subjects analysed
    6
    7
    1
    0 [8]
    Units: ug/ml
    geometric mean (geometric coefficient of variation)
        Cycle1 Day5 at 2 hrs (end of infusion) (n=0,0,1,0)
    999 ( 999 )
    999 ( 999 )
    256 ( 0.0 )
    ( )
        Cycle3 Day1 at 2 hr (end of infusion) (n=6,7,0, 0)
    137 ( 52.7 )
    304 ( 31.4 )
    999 ( 999 )
    ( )
        Cycle3 Day5 at 2 hr (end of infusion) (n=0,0,1,0)
    999 ( 999 )
    999 ( 999 )
    315 ( 0.0 )
    ( )
        Cycle24 Day1 at 2 hr (end of infusion) (n=2,0,0,0)
    163 ( 23.7 )
    999 ( 999 )
    999 ( 999 )
    ( )
    Notes
    [8] - No subjects in this group were analyzed for this endpoint
    No statistical analyses for this end point

    Secondary: Trough serum concentration of (Cmin) sabatolimab

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    End point title
    		 Trough serum concentration of (Cmin) sabatolimab
    End point description
    Cmin is the concentration of sabatolimab prior to next dosing or after end of treatment.
    End point type
    Secondary
    End point timeframe
    Adult cohorts: Pre-dose on Day 1 (safety run-in) or Day 5 (expansion) of Cycle 1, 3, 6 and 24 (safety run-in only); Adolescent cohort: Pre-dose on Day 1 of Cycle 1, 2, 3 and 6; Cycle = 28 Days
    End point values
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adults Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adolescent
    Number of subjects analysed
    6
    7
    2
    1
    Units: µg/ml
    geometric mean (geometric coefficient of variation)
        0-hour (hr) Pre-dose at Cycle1 Day1 (n=6,7,0,1)
    0.00 ( 0.0 )
    0.00 ( 0.0 )
    999 ( 999 )
    0.00 ( 0.0 )
        0 hr (pre-dose) at Cycle1 Day5 (0, 0, 2, 0)
    999 ( 999 )
    999 ( 999 )
    0.00 ( 0.0 )
    999 ( 999 )
        0 hr (pre-dose) at Cycle2 Day1 (0, 0, 0, 1)
    999 ( 999 )
    999 ( 999 )
    999 ( 999 )
    70.7 ( 0.0 )
        0 hr (pre-dose) at Cycle3 Day1 (6, 7, 0, 1)
    40.4 ( 20.2 )
    70.7 ( 47.5 )
    999 ( 999 )
    129 ( 0.0 )
        0 hr (pre-dose) at Cycle 3 Day 5 (0, 0, 1, 0)
    999 ( 999 )
    999 ( 999 )
    42.9 ( 0.0 )
    999 ( 999 )
        0 hr (pre-dose) at Cycle 6 Day 1 (4, 5, 0, 1)
    46.4 ( 66.4 )
    99.8 ( 52.1 )
    999 ( 999 )
    219 ( 0.0 )
        0 hr (pre-dose) at Cycle 6 Day 5 (0, 0, 1, 0)
    999 ( 999 )
    999 ( 999 )
    71.9 ( 0.0 )
    999 ( 999 )
        0 hr (pre-dose)at Cycle 24 Day 1 (2, 0, 0, 0)
    49.7 ( 44.1 )
    999 ( 999 )
    999 ( 999 )
    999 ( 999 )
    No statistical analyses for this end point

    Secondary: Graft versus host disease (GvHD)-free/relapse-free survival (GRFS)

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    End point title
    		 Graft versus host disease (GvHD)-free/relapse-free survival (GRFS)
    End point description
    Time from start of treatment to the date of first documented occurrence or worsening of treatment emergent grade III or IV aGvHD or moderate to severe cGvHD requiring initiation of systemic treatment, morphologic/hematologic relapse, or death due to any cause, whichever occurs first
    End point type
    Secondary
    End point timeframe
    From start of treatment to up to 36 months from last patient first treatment
    End point values
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adults Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adolescent
    Number of subjects analysed
    10
    11
    2
    1
    Units: Months
        median (confidence interval 95%)
    999 (999 to 999)
    999 (999 to 999)
    999 (999 to 999)
    999 (999 to 999)
    No statistical analyses for this end point

    Secondary: Relapse-free survival (RFS)

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    End point title
    		 Relapse-free survival (RFS)
    End point description
    Time from start of treatment to the date of first documented hematologic relapse or death due to any cause, whichever occurs first.
    End point type
    Secondary
    End point timeframe
    From start of treatment to up to 36 months from last patient first treatment.
    End point values
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adults Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adolescent
    Number of subjects analysed
    10
    11
    2
    1
    Units: Months
        median (confidence interval 95%)
    2.56 (0.95 to 999)
    6.74 (0.95 to 999)
    999 (999 to 999)
    7.16 (0 to 999)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events and deaths are described from the start of treatment up to 30 days after the last dose of treatment for a max. approx. 25 months
    Adverse event reporting additional description
    An Adverse Event is any sign or symptom occurring during a trial and safety follow-up. Death data covers all enrolled patients, while Adverse Event data was reported from those receiving at least one study drug dose.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.0
    Reporting groups
    Reporting group title
    Sabatolimab 400mg mono adults
    Reporting group description
    		 Safety run-in cohort: Adult participants in this arm received sabatolimab 400mg only.

    Reporting group title
    Sabatolimab 800mg mono adolescent
    Reporting group description
    		 Adolescent safety cohort: ≥12 to < 18-year-old adolescent participants in this arm received sabatolimab 800mg only.

    Reporting group title
    Sabatolimab 800mg + Azacitidine adults
    Reporting group description
    		 Expansion cohort: Adult participants in this arm received sabatolimab 800 mg + azacitidine combination.

    Reporting group title
    Sabatolimab 800mg mono adults
    Reporting group description
    		 Safety run-in cohort: Adult participants in this arm received sabatolimab 800mg only.

    Serious adverse events
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adolescent Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adults
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    3 / 11 (27.27%)
         number of deaths (all causes)
    1
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Femoral neck fracture
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Myocarditis
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Nervous system disorder
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Bronchitis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Febrile infection
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Oral herpes
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Sabatolimab 400mg mono adults Sabatolimab 800mg mono adolescent Sabatolimab 800mg + Azacitidine adults Sabatolimab 800mg mono adults
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 10 (90.00%)
    1 / 1 (100.00%)
    2 / 2 (100.00%)
    9 / 11 (81.82%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Basal cell carcinoma
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Vascular disorders
    Hypertensive crisis
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Oedema peripheral
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    2
    Asthenia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    2
    1
    Chest pain
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Fatigue
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    1
    1
    Injection site pain
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Mucosal inflammation
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Oedema
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Immune system disorders
    Chronic graft versus host disease
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    1
    0
    0
    2
    Lung disorder
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Dyspnoea exertional
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Investigations
    Blood cholesterol increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Blood alkaline phosphatase increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    1
    1
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    1
    1
    Amylase increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    1
    1
    C-reactive protein increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    1
    1
    Brain natriuretic peptide increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Blood potassium increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Blood creatinine increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Lipase increased
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    1
    0
    White blood cell count increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Troponin T increased
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Immunisation reaction
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Anaemia postoperative
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Nervous system disorders
    Neuropathy peripheral
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Paraesthesia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Somnolence
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Neutropenia
         subjects affected / exposed
    2 / 10 (20.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    2
    0
    0
    1
    Thrombocytopenia
         subjects affected / exposed
    3 / 10 (30.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    3
    0
    0
    0
    Disseminated intravascular coagulation
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    2
    Vomiting
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Nausea
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    2 / 2 (100.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    2
    1
    Stomatitis
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Pruritus
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Intertrigo
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Telangiectasia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    1 / 11 (9.09%)
         occurrences all number
    1
    0
    1
    1
    Bone pain
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Arthralgia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Myalgia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Infections and infestations
    Influenza
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    COVID-19
         subjects affected / exposed
    2 / 10 (20.00%)
    1 / 1 (100.00%)
    1 / 2 (50.00%)
    2 / 11 (18.18%)
         occurrences all number
    2
    1
    1
    2
    Viral infection
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Pneumonia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Otitis media
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Folliculitis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Febrile infection
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Clostridium difficile colitis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Bronchitis
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Rhinitis
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 1 (100.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Herpes zoster
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Metapneumovirus infection
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Oral bacterial infection
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Oral candidiasis
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    1 / 2 (50.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    0 / 10 (0.00%)
    1 / 1 (100.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Hypertriglyceridaemia
         subjects affected / exposed
    0 / 10 (0.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    0
    1
    Cachexia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Hyperferritinaemia
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Tumour lysis syndrome
         subjects affected / exposed
    1 / 10 (10.00%)
    0 / 1 (0.00%)
    0 / 2 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    16 Apr 2021
    Amendment #1: - In response to a request from health authorities, the list of inclusion and exclusion criteria was revised. The modification includes the addition of required minimum hemoglobin level, as well as restrictions for prior cancer-directed treatment or investigational modalities and for patient with BCR-ABL mutations eligible for post-transplant tyrosine kinase inhibitor therapies; - Clarification was provided on the dose used in cohorts 3-5, to define DLTs and dose modifications handling for sabatolimab by the request of health authorities; - Revisions were made to the visit schedule assessments tables to add/correct the visit windows. additionally, amendment was implemented to differentiate the collection of antineoplastic therapies/medications since discontinuation which was previously included in collection concomitant medication and therapies; - Changes included removal of immunogenicity (IG) sample collection after sabatolimab post-infusion at Cycle 24, as pre-infusion collected sample at this visit was sufficient.
    24 Mar 2022
    Amendment #2: - The restrictive list of inclusion and exclusion criteria was modified per feedback provided by the Principal Investigators to increase the recruitment rate. These changes did not substantially impact the study design or the primary objectives, which included safety and preliminary efficacy assessments. An amended protocol was implemented to include the determination of eligibility for enrollment based on central MRD assay. These modifications allowed for a more comprehensive assessment of patient eligibility during the course of the study.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com for complete trial results.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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