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    Clinical Trial Results:
    A single-arm, open-label, Phase II study of sabatolimab in combination with azacitidine and venetoclax in adult participants with high or very high-risk myelodysplastic syndrome (MDS) as per IPSS-R criteria. 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/CtrdWeb/home.nov for complete trial results.

    Summary
    EudraCT number
    		 2020-003669-21
    Trial protocol
    		 HU   DE   BE   GR   IT  
    Global end of trial date
    08 May 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    06 Mar 2024
    First version publication date
    06 Mar 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CMBG453B12203
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04812548
    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 Pharma, AG, 41 613241111, novartis.email@novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma, AG, 41 613241111, novartis.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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    08 May 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    08 May 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    Safety run-in (Cohort 1 and Cohort 2 of Part 1): To determine whether sabatolimab is safe when added to azacitidine + venetoclax in participants with high or very high risk MDS per IPSS-R criteria. Cohort 2 of Safety run-in (Part 1) and Expansion (Part 2): To determine the complete remission (CR) rate of sabatolimab in combination with azacitidine and venetoclax in participants with high or very high risk MDS as per IPSS-R criteria treated with sabatolimab at 800 mg Q4W.
    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
    31 May 2021
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Efficacy
    Long term follow-up duration
    		 22 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 5
    Country: Number of subjects enrolled
    Belgium: 2
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Greece: 5
    Country: Number of subjects enrolled
    Hungary: 1
    Country: Number of subjects enrolled
    Italy: 2
    Country: Number of subjects enrolled
    Spain: 3
    Worldwide total number of subjects
    20
    EEA total number of subjects
    20
    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)
    0
    Adults (18-64 years)
    5
    From 65 to 84 years
    15
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Ten centers across 7 countries enrolled a total of 20 participants in this study.

    Pre-assignment
    Screening details
    		 Prior to dosing at Cycle 1 Day 1, participants who fulfilled all the inclusion/exclusion criteria were enrolled via IRT and a treatment number was provided for the study treatments sabatolimab, venetoclax, and azacitidine.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1)
    Arm description
    		 Part 1 Cohort 1: Safety run-in cohort of a lower dose of sabatolimab in combination with fixed dose of venetoclax and azacitidine.
    Arm type
    		 Experimental

    Investigational medicinal product name
    sabatolimab
    Investigational medicinal product code
    MBG453
    Other name
    Pharmaceutical forms
    Concentrate and solvent for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    administered intravenously at 400 mg during Safety run-in Cohort 1 on Day 8 (Q4W).

    Investigational medicinal product name
    venetoclax
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    administered orally at 400 mgdaily for 14 consecutive days,during Safety run-in (Part 1)

    Investigational medicinal product name
    azacitidine
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate and solvent for solution for infusion, Concentrate and solvent for solution for injection
    Routes of administration
    Intravenous use, Subcutaneous use
    Dosage and administration details
    azacitidine was to be administered intravenously or subcutaneously at 75 mg/m2on Days 1 to 7 (or, at discretion of the investigator on Days 1-5 and Day 8-9), during Safety run-in (Part 1)

    Arm title
    		 sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Arm description
    		 Part 1 Cohort 2: Safety run-in cohort of a higher dose of sabatolimab in combination with fixed dose of venetoclax and azacitidine.
    Arm type
    		 Experimental

    Investigational medicinal product name
    sabatolimab
    Investigational medicinal product code
    MBG453
    Other name
    Pharmaceutical forms
    Concentrate and solvent for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    administered intravenously at 800 mg during Safety run-in Cohort 2 on Day 8 (Q4W).

    Investigational medicinal product name
    venetoclax
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    administered orally at 400 mgdaily for 14 consecutive days,during Safety run-in (Part 1)

    Investigational medicinal product name
    azacitidine
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate and solvent for solution for infusion, Concentrate and solvent for solution for injection
    Routes of administration
    Intravenous use, Subcutaneous use
    Dosage and administration details
    azacitidine was to be administered intravenously or subcutaneously at 75 mg/m2on Days 1 to 7 (or, at discretion of the investigator on Days 1-5 and Day 8-9), during Safety run-in (Part 1)

    Number of subjects in period 1
    		sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Started
    5
    15
    Did not enter post-treatment follow-up
    1
    5
    Entered post-treatment follow-up
    4
    10
    Completed
    0
    0
    Not completed
    5
    15
         Adverse event, serious fatal
    -
    1
         Physician decision
    1
    -
         Consent withdrawn by subject
    -
    1
         Adverse event, non-fatal
    2
    7
         Post Study Access to Treatment
    -
    1
         Progressive Disease
    -
    2
         HSCT Planned
    2
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1)
    Reporting group description
    		 Part 1 Cohort 1: Safety run-in cohort of a lower dose of sabatolimab in combination with fixed dose of venetoclax and azacitidine.

    Reporting group title
    sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Reporting group description
    		 Part 1 Cohort 2: Safety run-in cohort of a higher dose of sabatolimab in combination with fixed dose of venetoclax and azacitidine.

    Reporting group values
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2) Total
    Number of subjects
    		 5 15 20
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 2 3 5
        From 65-84 years
    		 3 12 15
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 67.2 ± 11.28 69.3 ± 9.45 -
    Sex: Female, Male
    Units: Participants
        Female
    		 0 4 4
        Male
    		 5 11 16
    Race/Ethnicity, Customized
    Units: Subjects
        White
    		 4 14 18
        Unknown
    		 1 1 2

    End points

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    End points reporting groups
    Reporting group title
    sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1)
    Reporting group description
    		 Part 1 Cohort 1: Safety run-in cohort of a lower dose of sabatolimab in combination with fixed dose of venetoclax and azacitidine.

    Reporting group title
    sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Reporting group description
    		 Part 1 Cohort 2: Safety run-in cohort of a higher dose of sabatolimab in combination with fixed dose of venetoclax and azacitidine.

    Primary: Incidence of dose limiting toxicities (DLTs) - All grades (Safety run-in patients only)

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    End point title
    		 Incidence of dose limiting toxicities (DLTs) - All grades (Safety run-in patients only) [1]
    End point description
    Assessment of tolerability of sabatolimab (MBG453) in combination with venetoclax and azacitidine
    End point type
    Primary
    End point timeframe
    From Cycle 1 Day 8 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 statistical analysis was done for this endpoint
    End point values
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    4
    13
    Units: Participants
        Number of participants with at least one event
    0
    2
        Blood and lymph. syst disorders (Thrombocytopenia)
    0
    1
        Nerv. Syst. disorders (Haemorrhage intracranial)
    0
    1
    No statistical analyses for this end point

    Primary: Percentage of participants (receiving 800mg sabatolimab) achieving complete remission (CR) per investigator assessment

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    End point title
    		 Percentage of participants (receiving 800mg sabatolimab) achieving complete remission (CR) per investigator assessment [2] [3]
    End point description
    This endpoint assessed Complete Remission (CR) Rate of participants from Cohort 2 of Part 1 and Part 2 according to Investigator assessment per modified IWG-MDS - Cheson 2006 criteria. CR is defined as follows: bone marrow blasts <=5%, hemoglobin level ≥ 10 g/dL, platelets count ≥ 100*10^9/L, neutrophils count ≥ 1.0*10^9/L, absence of blasts in peripheral blood.
    End point type
    Primary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    Notes
    [2] - 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 statistical analysis was done for this endpoint
    [3] - 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 statistical analysis was done for this endpoint
    End point values
    		 sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    15
    Units: Participants
    1
    No statistical analyses for this end point

    Secondary: Duration of transfusion independence

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    End point title
    		 Duration of transfusion independence
    End point description
    Sum of each period of the transfusion independence for participants with at least one period of transfusion independence post-baseline by dose level for the safety run-in part (Cohort 1 (400 mg Q4W) and Cohort 2 (800 mg Q4W)) and for participants treated with sabatolimab 800 mg (Q4W) (Cohort 2 of safety run-in and expansion parts) for both red blood cells and platelets.
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    End point values
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    5
    15
    Units: Weeks
    arithmetic mean (standard deviation)
        Packed Red Blood Cells (n = 2, 5)
    16.43 ± 0.808
    23.11 ± 12.636
        Platelets (n = 2, 7)
    18.93 ± 2.727
    24.02 ± 16.423
    No statistical analyses for this end point

    Secondary: Percentage of participants who are RBC/platelets transfusion independent

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    End point title
    		 Percentage of participants who are RBC/platelets transfusion independent
    End point description
    Improvement in red blood cells (RBC)/platelets transfusion post-baseline as per International Working Group - Myelodysplastic syndromes (IWG-MDS) by dose level for the safety run-in part (Cohort 1 (400 mg Q4W) and Cohort 2 (800 mg Q4W)) and for participants treated with sabatolimab 800 mg (Q4W) (Cohort 2 of safety run-in and expansion parts).
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    End point values
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    5
    15
    Units: Participants
        RBC
    2
    5
        Platelets
    2
    7
    No statistical analyses for this end point

    Secondary: Overall Response Rate (ORR) of participants who achieved hematologic improvement (HI) or better as best response

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    End point title
    		 Overall Response Rate (ORR) of participants who achieved hematologic improvement (HI) or better as best response
    End point description
    The percentage of participants achieving [CR + mCR + partial remission (PR) + hematologic improvement (HI)], per modified IWG-MDS Cheson 2006 criteria
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    End point values
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    5
    15
    Units: Participants
    4
    13
    No statistical analyses for this end point

    Secondary: Percentage of subjects achieving a complete remission (CR) + morphologic complete remission (mCR): Safety run-in (Part 1)

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    End point title
    		 Percentage of subjects achieving a complete remission (CR) + morphologic complete remission (mCR): Safety run-in (Part 1)
    End point description
    Assessed the durability of complete remission (CR) or morphologic complete remission (mCR) rate (defined as the percentage of participants with best overall response of either CR or mCR).
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    End point values
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    5
    15
    Units: Participants
    4
    13
    No statistical analyses for this end point

    Secondary: Trough Serum Concentration (Cmin) sabatolimab

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    End point title
    		 Trough Serum Concentration (Cmin) sabatolimab
    End point description
    Concentration of sabatolimab prior to next dosing or after end of treatment by dose level for the safety run-in part (Cohort 1 (400 mg Q4W) and Cohort 2 (800 mg Q4W)).
    End point type
    Secondary
    End point timeframe
    Continuously collected for patients during treatment with sabatolimab up to 150 days after last treatment, approx. 14.2 months
    End point values
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    5
    13
    Units: ug/ml
    geometric mean (geometric coefficient of variation)
        Cycle (C) 1 Day (D) 8 (n = 4, 12)
    0.0 ± 0.0
    0.0 ± 0.0
        C2D8 (n = 4, 10)
    23.0 ± 137.0
    30.7 ± 41.7
        C3D8 (n = 1, 6)
    0.0 ± 0.0
    34.4 ± 72.2
        C6D8 (n = 0, 3)
    999 ± 999
    68.6 ± 11.5
        C9D8 (n = 0, 2)
    999 ± 999
    71.2 ± 33.8
        C12D (n = 0, 1)
    999 ± 999
    0.0 ± 0.0
    No statistical analyses for this end point

    Secondary: Peak Serum Concentration (Cmax) of sabatolimab

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    End point title
    		 Peak Serum Concentration (Cmax) of sabatolimab
    End point description
    Maximal concentration of sabatolimab for participants treated with sabatolimab by dose level for the safety run-in part (Cohort 1 (400 mg Q4W) and Cohort 2 (800 mg Q4W)).
    End point type
    Secondary
    End point timeframe
    Continuously collected for patients during treatment with sabatolimab up to 150 days after last treatment, approx. 14.2 months
    End point values
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    0 [4]
    0 [5]
    Units: ug/ml
        geometric mean (geometric coefficient of variation)
    ±
    ±
    Notes
    [4] - No subjects were analyzed for this endpoint
    [5] - No subjects were analyzed for this endpoint
    No statistical analyses for this end point

    Secondary: Anti-drug Antibody (ADA) prevalence at baseline and ADA incidence on-treatment by dose level

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    End point title
    		 Anti-drug Antibody (ADA) prevalence at baseline and ADA incidence on-treatment by dose level
    End point description
    Immunogenicity of sabatolimab prior to sabatolimab exposure and during treatment
    End point type
    Secondary
    End point timeframe
    Continuously collected for patients during treatment with sabatolimab up to 150 days after last treatment, approx. 14.2 months
    End point values
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    4
    13
    Units: Participants
        ADA prevalence at baseline
    0
    2
        ADA incidence (i.e., ADA positive) on-treatment
    1
    2
        Treatment-induced ADA-positive
    1
    1
        Treatment-boosted ADA-positive
    0
    1
    No statistical analyses for this end point

    Secondary: Time to complete remission(CR)/marrow complete remission (mCR)

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    End point title
    		 Time to complete remission(CR)/marrow complete remission (mCR) [6]
    End point description
    Time to CR/mCR is defined as time from start of treatment to first occurrence of CR or mCR as per investigator assessment for the safety run-in part (Cohort 2 (800 mg Q4W)).
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    Notes
    [6] - 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 statistical analysis was done for this endpoint
    End point values
    		 sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    15
    Units: months
        median (confidence interval 95%)
    2.33 (1.61 to 2.83)
    No statistical analyses for this end point

    Secondary: Duration of complete remission (CR)

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    End point title
    		 Duration of complete remission (CR) [7]
    End point description
    Duration of CR is defined as time from first occurrence of CR to relapse from CR, progression or death due to any cause whichever occurs first for the safety run-in part (Cohort 2 (800 mg Q4W)).
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    Notes
    [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 statistical analysis was done for this endpoint
    End point values
    		 sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    1
    Units: months
        median (confidence interval 95%)
    999 (999 to 999)
    No statistical analyses for this end point

    Secondary: Duration of response for participants who achieved hematologic improvement (HI) or better

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    End point title
    		 Duration of response for participants who achieved hematologic improvement (HI) or better [8]
    End point description
    The duration of response was derived for participants treated with sabatolimab at the higher dose who achieved HI or better as per investigator assessment and is defined from the first occurrence of complete response (CR), marrow complete response (mCR), partial response (PR) or hematologic improvement (HI) until relapse, progression or death due to any reason for the safety run-in part (Cohort 2 (800 mg Q4W)).
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    Notes
    [8] - 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 statistical analysis was done for this endpoint
    End point values
    		 sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    13
    Units: months
        median (confidence interval 95%)
    5.60 (4.14 to 99)
    No statistical analyses for this end point

    Secondary: Leukemia-Free Survival (LFS)

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    End point title
    		 Leukemia-Free Survival (LFS) [9]
    End point description
    Time from start of treatment to transformation to acute leukemias per investigator assessment [as defined as ≥ 20% blasts in bone marrow/ peripheral blood (per WHO 2016 classification) or diagnosis of extramedullary acute leukemia or death due to any cause, whichever occurs first for the safety run-in part (Cohort 2 (800 mg Q4W)).
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    Notes
    [9] - 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 statistical analysis was done for this endpoint
    End point values
    		 sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    15
    Units: months
        median (confidence interval 95%)
    7.59 (4.24 to 999)
    No statistical analyses for this end point

    Secondary: Progression-Free Survival (PFS)

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    End point title
    		 Progression-Free Survival (PFS) [10]
    End point description
    Time from start of treatment to disease progression (including transformation to acute leukemia per WHO 2016 classification), relapse from CR or death due to any cause, whichever occurs first for the safety run-in part (Cohort 2 (800 mg Q4W)).
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    Notes
    [10] - 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 statistical analysis was done for this endpoint
    End point values
    		 sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    15
    Units: months
        median (confidence interval 95%)
    6.77 (3.71 to 999)
    No statistical analyses for this end point

    Secondary: Event-Free Survival (EFS)

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    End point title
    		 Event-Free Survival (EFS) [11]
    End point description
    Time from start of treatment to lack of reaching CR within the first 6 cycles, relapse from CR or death due to any cause, whichever occurs first for the safety run-in part (Cohort 2 (800 mg Q4W)).
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    Notes
    [11] - 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 statistical analysis was done for this endpoint
    End point values
    		 sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    15
    Units: months
        median (confidence interval 95%)
    0.03 (0.009 to 999)
    No statistical analyses for this end point

    Secondary: Duration of complete response (CR)/marrow complete response (mCR)

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    End point title
    		 Duration of complete response (CR)/marrow complete response (mCR) [12]
    End point description
    Duration of CR/mCR is defined as time from first occurrence of CR/mCR to relapse from CR, progression or death due to any cause whichever occurs first for the safety run-in part (Cohort 2 (800 mg Q4W)).
    End point type
    Secondary
    End point timeframe
    Throughout study completion, approx. 22.4 months
    Notes
    [12] - 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 statistical analysis was done for this endpoint
    End point values
    		 sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    13
    Units: months
        median (confidence interval 95%)
    5.60 (4.14 to 999)
    No statistical analyses for this end point

    Secondary: Overall Survival (OS)

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    End point title
    		 Overall Survival (OS) [13]
    End point description
    Time from start of treatment to death due to any cause for the safety run-in part (Cohort 2 (800 mg Q4W)).
    End point type
    Secondary
    End point timeframe
    Date of start of treatment to date of death due to any reason, for up to approx. 22.4 months
    Notes
    [13] - 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 statistical analysis was done for this endpoint
    End point values
    		 sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    15
    Units: months
        median (confidence interval 95%)
    999 (677 to 999)
    No statistical analyses for this end point

    Post-hoc: All Collected Deaths

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    End point title
    		 All Collected Deaths
    End point description
    On-treatment deaths were collected from start of study treatment (FPFT) up to 30 days after study drug discontinuation, for a maximum duration of approx. 13 months. Post-treatment survival follow-up deaths were collected from Day 31 after last dose of study treatment to end of study up to approx. 18.4 months All deaths refer to the sum of on-treatment and post-treatment survival follow-up deaths, approx. 22.4 months.
    End point type
    Post-hoc
    End point timeframe
    On-treatment deaths: up to approx. 13 months, post-treatment deaths: up to approx 18.4 months
    End point values
    		 sabatolimab (MBG453) 400 mg + AZA + VEN (Part 1 Cohort 1) sabatolimab (MBG453) 800 mg + AZA + VEN (Part 1 Cohort 2)
    Number of subjects analysed
    5
    15
    Units: Participants
        All deaths
    1
    6
        On-treatment deaths
    0
    1
        Post-treatment deaths
    1
    5
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Timeframe for AE
    Adverse event reporting additional description
    AE additional description
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    MBG453 800 mg@+ AZA + VEN
    Reporting group description
    		 MBG453 800 mg@+ AZA + VEN

    Reporting group title
    MBG453 400 mg@+ AZA + VEN
    Reporting group description
    		 MBG453 400 mg@+ AZA + VEN

    Serious adverse events
    		 MBG453 800 mg@+ AZA + VEN MBG453 400 mg@+ AZA + VEN
    Total subjects affected by serious adverse events
         subjects affected / exposed
    14 / 15 (93.33%)
    3 / 5 (60.00%)
         number of deaths (all causes)
    1
    0
         number of deaths resulting from adverse events
    1
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Paraneoplastic syndrome
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Lung adenocarcinoma
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Investigations
    Medical observation
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Limb injury
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Wound haemorrhage
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Haemorrhage intracranial
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Febrile neutropenia
         subjects affected / exposed
    5 / 15 (33.33%)
    2 / 5 (40.00%)
         occurrences causally related to treatment / all
    3 / 5
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Febrile bone marrow aplasia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Anaemia
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Neutropenia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pancytopenia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune system disorders
    Autoinflammatory disease
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Large intestine perforation
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Bacterial sepsis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Peritonitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis viral
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Escherichia bacteraemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 5 (20.00%)
         occurrences causally related to treatment / all
    1 / 2
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Septic shock
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    1 / 1
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 MBG453 800 mg@+ AZA + VEN MBG453 400 mg@+ AZA + VEN
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 15 (100.00%)
    5 / 5 (100.00%)
    Vascular disorders
    Thrombosis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Hypotension
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Hypertension
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Haematoma
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Infusion site haematoma
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Injection site erythema
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Injection site pain
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Injection site reaction
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Oedema peripheral
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Hyperthermia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Fatigue
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 5 (20.00%)
         occurrences all number
    3
    1
    Device related thrombosis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Chills
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Catheter site inflammation
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Asthenia
         subjects affected / exposed
    3 / 15 (20.00%)
    1 / 5 (20.00%)
         occurrences all number
    3
    1
    Peripheral swelling
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Pyrexia
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Reproductive system and breast disorders
    Uterine haemorrhage
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Heavy menstrual bleeding
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Cervical polyp
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Pleural effusion
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Epistaxis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Cough
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Psychiatric disorders
    Confusional state
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Anxiety
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Investigations
    Blood bilirubin increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Blood folate decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    3
    0
    Blood urea increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    C-reactive protein increased
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Neutrophil count decreased
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 5 (20.00%)
         occurrences all number
    4
    1
    Platelet count decreased
         subjects affected / exposed
    3 / 15 (20.00%)
    0 / 5 (0.00%)
         occurrences all number
    5
    0
    Serum ferritin decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Troponin T increased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Weight decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    White blood cell count decreased
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    3
    0
    Cardiac disorders
    Sinus bradycardia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Haemorrhage intracranial
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Dizziness
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    7 / 15 (46.67%)
    1 / 5 (20.00%)
         occurrences all number
    17
    1
    Bone marrow failure
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Febrile neutropenia
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 5 (0.00%)
         occurrences all number
    3
    0
    Thrombocytosis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Thrombocytopenia
         subjects affected / exposed
    7 / 15 (46.67%)
    3 / 5 (60.00%)
         occurrences all number
    10
    4
    Pancytopenia
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Neutropenia
         subjects affected / exposed
    8 / 15 (53.33%)
    3 / 5 (60.00%)
         occurrences all number
    19
    3
    Leukopenia
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Haemolysis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 15 (13.33%)
    2 / 5 (40.00%)
         occurrences all number
    2
    4
    Constipation
         subjects affected / exposed
    4 / 15 (26.67%)
    2 / 5 (40.00%)
         occurrences all number
    4
    2
    Abdominal pain upper
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Abdominal pain
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Eructation
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Vomiting
         subjects affected / exposed
    3 / 15 (20.00%)
    1 / 5 (20.00%)
         occurrences all number
    3
    1
    Toothache
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Stomatitis
         subjects affected / exposed
    3 / 15 (20.00%)
    1 / 5 (20.00%)
         occurrences all number
    8
    1
    Odynophagia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Nausea
         subjects affected / exposed
    6 / 15 (40.00%)
    1 / 5 (20.00%)
         occurrences all number
    10
    1
    Lower gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Haemorrhoidal haemorrhage
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Flatulence
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Haemorrhoids
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Hepatobiliary disorders
    Cholestasis
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 5 (20.00%)
         occurrences all number
    2
    2
    Hepatic cytolysis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Hepatotoxicity
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Hyperbilirubinaemia
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 5 (20.00%)
         occurrences all number
    1
    1
    Hypertransaminasaemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Jaundice
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Erythema
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Rash pruritic
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Rash maculo-papular
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Purpura
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Pruritus
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 5 (20.00%)
         occurrences all number
    2
    1
    Dermatitis allergic
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Renal impairment
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Tendonitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Pain in extremity
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Myalgia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Arthralgia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Cellulitis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Clostridium bacteraemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Escherichia bacteraemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Oral herpes
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Sepsis
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Tooth abscess
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Vascular device infection
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Vitamin K deficiency
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Tumour lysis syndrome
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Malnutrition
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Hypophosphataemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    2
    0
    Hypomagnesaemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Hypokalaemia
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 5 (20.00%)
         occurrences all number
    4
    3
    Cell death
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Decreased appetite
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Gout
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 5 (20.00%)
         occurrences all number
    0
    1
    Hyperkalaemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Hypernatraemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0
    Hyperuricaemia
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 5 (0.00%)
         occurrences all number
    1
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    17 May 2021
    The purpose of this amendment was to address Health Authorities’ requests (from Belgium and France) to specify that women of childbearing potential using a hormonal contraception should add a barrier method, as stated in the venetoclax SmPC, and to add a cross-reference to venetoclax local label in the prohibited medication section. Additionally, preliminary results of the first cohort of the safety run-in of CMBG453C12201 study were included. The pharmaceutical dose form and route of administration terms for azacitidine were updated to be in alignment with other sabatolimab protocols.
    06 Oct 2021
    The purpose of this amendment was to clarify DLT criteria to specify that prolonged cytopenias beyond Day 42 from the start of a study treatment cycle should be considered DLTs and to clarify language around intercurrent events in the estimand section as well as analysis of duration of response. Additionally, language around COVID-19 vaccines and time frame for SAE follow-up reporting was added.

    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/CtrdWeb/home.nov 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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