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TerminatedNCT04623216Updated Oct 16, 2025Results posted

Sabatolimab as a Treatment for Patients With Acute Myeloid Leukemia and Presence of Measurable Residual Disease After Allogeneic Stem Cell Transplantation.

A Phase 1/2 interventional study of Sabatolimab and Azacitidine in Acute Myeloid Leukemia, sponsored by Novartis Pharmaceuticals. Terminated at 9 sites in 4 countries. Open to participants aged 12 Years to 99 Years. Per ClinicalTrials.gov, last updated 2025-10-16.

Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 1/2, Interventional, and Treatment

Why this study was terminated
Study was stopped following a strategic decision from the Sponsor. It was not based on any safety findings or safety concerns with sabatolimab.
Phase
Phase 1/2
Study type
Interventional
Enrollment
24
Allocation
Randomized
Ages
12 Years to 99 Years
Sex
All
01

Study summary

The primary purpose of this study was to test the hypothesis that preemptive treatment with sabatolimab, alone or in combination with azacitidine, when administered to participants with Acute myeloid leukemia (AML)/secondary AML who were in complete remission with positive measurable residual disease post-allogeneic hematopoietic stem cell transplantation (Minimal residual disease (MRD)+ post- Allogeneic hematopoietic stem cell transplantation (aHSCT)), could enhance the graft versus leukemia (GvL) response and prevent or delay hematologic relapse without an unacceptable level of treatment-emergent toxicities, including clinically significant acute and/or chronic graft-versus-host disease (GvHD) and immune-related adverse events

Read the detailed description

This is a phase Ib/II, open label, multi-center study of sabatolimab as monotherapy and in combination with azacitidine, in participants with AML/secondary AML who had received one aHSCT and achieved complete remission but MRD+, by local assessment, anytime at >= Day 60 after aHSCT and at least 2 weeks after immunosuppressive medications had been tapered off.

The study was planned to enroll approximately 59 participants and be conducted in two parts:

Part 1 was a Safety Run-in of approximately 20 participants, to assess whether sabatolimab as monotherapy at the two tested dose levels (400 mg and 800 mg intravenously Q4W) was safe when administered in the post-aHSCT setting. For each dose level, once the required number of evaluable participants had been confirmed, enrollment would be halted until participants had completed the dose limiting toxicities (DLT) observation period (≥ 8 weeks following the first dose). Following the observation period for DLTs, a Safety Review Meeting was to be conducted after each dose level to assess safety and determine the recommended dose for expansion to proceed with enrollment of additional cohorts in Part 2 of the study.

Part 2 consisted of sabatolimab monotherapy expansion cohort of approximately 13 participants, sabatolimab in combination with azacitidine cohort of approximately 20 participants, and an adolescent cohort of approximately 6 participants (≥ 12 years but \< 18 years of age) with sabatolimab as monotherapy. Sabatolimab was to be administered at the recommended dose for expansion determined in Part 1.

After initiating Part 2, Novartis took the decision to put enrollment in permanent halt and terminate the sabatolimab program. This decision was not driven by any safety concerns.

02

Conditions studied

  • Acute Myeloid Leukemia

Keywords

  • Sabatolimab
  • MBG453
  • TIM-3
  • Azacitidine
  • Acute Myeloid Leukemia
  • AML
  • Allogeneic Hematopoietic Stem Cell Transplantation
  • aHSCT
  • Measurable Residual Disease
  • MRD
  • Phase Ib/II
  • Acute myeloid leukemia Hematopoietic stem cell transplantation
03

In context

Leukemia, Myeloid, Acute

2,971 studies on the registry are indexed under Leukemia, Myeloid, Acute; 745 are open to participants now.

This study's enrollment of 24 is below the median of 41 across 2,509 interventional studies indexed under Leukemia, Myeloid, Acute.

Browse Leukemia, Myeloid, Acute studies →

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.

Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.

Browse Novartis Pharmaceuticals studies →All Novartis studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years to 99 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Signed informed consent must be obtained prior to participation in the study.
  • At the date of signing the informed consent form (ICF), eligible participants must be ≥ 18 years for the adult cohorts; and ≥ 12 years old but \< 18 years old for the adolescent cohort (cohort 5), which will open after completion of Safety Run-in.
  • Participants in complete remission (\< 5% bone marrow blasts, absence of circulating blasts, and absence of extramedullary disease) with MRD positivity by local assessment or by central assessment where required (e.g., USA sites), any time at ≥ Day 60 after aHSCT
  • Diagnosis of AML/secondary AML and received one prior aHSCT performed to control AML
  • Ability to provide a fresh bone marrow aspirate sample collected within 28 days from enrollment/randomization, and immediately shipped to a Novartis designated central laboratory for MRD testing.
  • Systemic GvHD (graft versus host disease) prophylaxis or treatment [immunosuppressive treatment (IST)] completely tapered for at least two weeks prior to study entry. Prednisone dose ≤ 5 mg/day or equivalent corticosteroid dose is allowed.
  • Participants who are found with MRD positivity while still on or tapering systemic GvHD prophylaxis or treatment, MRD positivity must be re-confirmed at least 2 week after the last dose of IST
  • For the adult cohorts, participants must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.

For the adolescent cohort, participants must have a Karnofsky (age ≥ 16 years) or Lansky (age \< 16 years) performance status score ≥ 50%.

Exclusion criteria

Exclusion Criteria:

  • Prior exposure to TIM-3 directed therapy at anytime.
  • History of severe hypersensitivity reactions to any ingredient of study drug(s) (azacitidine, sabatolimab) or monoclonal antibodies (mAbs) and/or their excipients
  • Active Hepatitis B (HBV) or Hepatitis C (HCV) infection. Participants whose disease is controlled under antiviral therapy should not be excluded.
  • Active acute GvHD grade III-IV according to standard criteria (Harris 2016).
  • Active moderate chronic GvHD of the lungs according to NIH consensus criteria. Active severe chronic GvHD according to NIH consensus criteria.
  • History of another primary malignancy that is currently clinically significant or currently requires active intervention. Participants who are receiving adjuvant therapy, such as hormone therapy, are eligible
  • Any concurrent severe and/or active uncontrolled infection requiring parenteral antibacterial, antiviral or antifungal therapy (such as severe pneumonia, meningitis, or septicemia)
  • Active autoimmune disease requiring systemic therapy (e.g. corticosteroids). Topical, inhaled, nasal and ophthalmic steroids are not prohibited. Replacement corticosteroids therapy is allowed and not considered a form of systemic treatment
  • Live vaccine administered within 30 days prior to the first day of study treatment (Cycle 1 Day 1)
  • Other concurrent severe and/or uncontrolled medical conditions (e.g. uncontrolled diabetes mellitus, chronic obstructive or chronic restrictive pulmonary disease including dyspnoea at rest from any cause) or history of serious organ dysfunction or disease involving the heart, kidney, or liver
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
24 participants (actual)

Study arms

  • Experimental
    Sabatolimab 400mg

    Safety cohort 1: Participants in this arm will receive sabatolimab 400mg intravenously every 4 weeks.

    Biological: Sabatolimab

  • Experimental
    Sabatolimab 800mg

    Safety cohort 2: Participants in this arm will receive sabatolimab 800mg intravenously every 4 weeks.

    Biological: Sabatolimab

  • Experimental
    Sabatolimab + Azacitidine

    Expansion cohort 3: Participants in this arm will receive sabatolimab at the recommended dose for expansion in combination with azacitidine.

    Biological: Sabatolimab · Drug: Azacitidine

  • Experimental
    Sabatolimab

    Expansion cohort 4: Participants in this arm will receive sabatolimab at the recommended dose for expansion.

    Biological: Sabatolimab

  • Experimental
    Sabatolimab (adolescent cohort)

    Adolescent safety cohort (cohort 5): ≥12 to \< 18 year old adolescent participants in this arm will receive sabatolimab at the recommended dose for expansion.

    Biological: Sabatolimab

Interventions

  • BiologicalSabatolimab

    Sabatolimab is a solution in vial for IV infusion

    Also known as: MBG453

  • DrugAzacitidine

    Azacitidine comes in Vial for IV infusion or subcutaneous administration

06

What researchers measure

Primary outcomes

  1. Rate of Dose Limiting Toxicities (Safety Run-in in Adult Sabatolimab 400mg & 800mg Only)

    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.

    Time frame: From Cycle 1 Day 1 to end of Cycle 2; Cycle =28 Days

  2. Percentage of Adult Subjects With Absence of Hematologic Relapse Per Investigator Assessment (Safety Run-in and Expansion)

    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 sabatolimab 800 mg.

    Time frame: From Cycle 1 Day 1 to end of Cycle 6; Cycle = 28 Days

  3. Rate of Dose Limiting Toxicities (Safety Confirmation in Adolescent Cohort Only)

    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.

    Time frame: From Cycle 1 Day 1 to end of Cycle 2; Cycle =28 Days

Secondary outcomes

  1. Incidence of Grade III or IV Acute Graft Versus Host Disease (aGvHD)

    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).

    Time frame: From start of treatment to up to 36 months from last patient first treatment.

  2. Incidence of Moderate to Severe Chronic GVHD (cGvHD)

    Assessment of the treatment emergent moderate or severe cGvHD. Chronic GvHD: Moderate chronic GvHD of the lungs, Severe chronic GvHD.

    Time frame: From start of treatment to up to 36 months from last patient first treatment.

  3. Peak of Serum Concentration (Cmax) Sabatolimab

    Cmax is the maximal serum concentration of sabatolimab.

    Time frame: 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); Cycle =28 Days

  4. Trough Serum Concentration of (Cmin) Sabatolimab

    Cmin is the concentration of sabatolimab prior to next dosing or after end of treatment.

    Time frame: 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

  5. Graft Versus Host Disease (GvHD)-Free/Relapse-free Survival (GRFS)

    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

    Time frame: From start of treatment to up to 36 months from last patient first treatment

  6. Relapse-free Survival (RFS)

    Time from start of treatment to the date of first documented hematologic relapse or death due to any cause, whichever occurs first.

    Time frame: From start of treatment to up to 36 months from last patient first treatment

  7. Percentage of Participants With Measurable Residual Disease (MRD) Positive at Baseline Who Become MRD Negative

    Percentage of participants with centrally confirmed MRD+ status at baseline converting to MRD- within the first 6 cycles of study treatment.

    Time frame: From start of treatment until end of Cycle 6 (Cycle = 28 Days)

07

Results

Posted Jun 3, 2025

Participant flow

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

Participant flow — Overall Study
MilestoneSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
Started101121
Did not enter post-treatment follow-up (f/u)7921
Entered post-treatment f/u, discontinued3200
Completed treatment2000
Completed1701
Not completed9420
Withdrew: Death5200
Withdrew: Terminated by sponsor3220
Withdrew: Physician decision1000

Outcome measures

PrimaryRate of Dose Limiting Toxicities (Safety Run-in in Adult Sabatolimab 400mg & 800mg Only)

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.

Time frame:
From Cycle 1 Day 1 to end of Cycle 2; Cycle =28 Days
Reported as:
Count of participants · Participants
Rate of Dose Limiting Toxicities (Safety Run-in in Adult Sabatolimab 400mg & 800mg Only)
ParticipantsSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono Adults
Rate of Dose Limiting Toxicities (Safety Run-in in Adult Sabatolimab 400mg & 800mg Only)01
PrimaryPercentage of Adult Subjects With Absence of Hematologic Relapse Per Investigator Assessment (Safety Run-in and Expansion)

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 sabatolimab 800 mg.

Time frame:
From Cycle 1 Day 1 to end of Cycle 6; Cycle = 28 Days
Reported as:
Number · Percentage of participants
Percentage of Adult Subjects With Absence of Hematologic Relapse Per Investigator Assessment (Safety Run-in and Expansion)
Percentage of participantsSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
Percentage of Adult Subjects With Absence of Hematologic Relapse Per Investigator Assessment (Safety Run-in and Expansion)—36.4 (10.9 to 69.2)——
PrimaryRate of Dose Limiting Toxicities (Safety Confirmation in Adolescent Cohort Only)

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.

Time frame:
From Cycle 1 Day 1 to end of Cycle 2; Cycle =28 Days
Reported as:
Count of participants · Participants
Rate of Dose Limiting Toxicities (Safety Confirmation in Adolescent Cohort Only)
ParticipantsSabatolimab 800mg Mono Adolescent
Rate of Dose Limiting Toxicities (Safety Confirmation in Adolescent Cohort Only)0
SecondaryIncidence of Grade III or IV Acute Graft Versus Host Disease (aGvHD)

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).

Time frame:
From start of treatment to up to 36 months from last patient first treatment.
Reported as:
Number · Participants
Incidence of Grade III or IV Acute Graft Versus Host Disease (aGvHD)
ParticipantsSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
Incidence of Grade III or IV Acute Graft Versus Host Disease (aGvHD)0000
SecondaryIncidence of Moderate to Severe Chronic GVHD (cGvHD)

Assessment of the treatment emergent moderate or severe cGvHD. Chronic GvHD: Moderate chronic GvHD of the lungs, Severe chronic GvHD.

Time frame:
From start of treatment to up to 36 months from last patient first treatment.
Reported as:
Count of participants · Participants
Incidence of Moderate to Severe Chronic GVHD (cGvHD)
ParticipantsSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
Incidence of Moderate to Severe Chronic GVHD (cGvHD)0100
SecondaryPeak of Serum Concentration (Cmax) Sabatolimab

Cmax is the maximal serum concentration of sabatolimab.

Time frame:
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); Cycle =28 Days
Reported as:
Geometric mean · ug/ml
Peak of Serum Concentration (Cmax) Sabatolimab
ug/mlSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
Cycle 1 Day 5 at 2 hours (hr) (end of infusion)——256 ± 0.0—
Cycle 3 Day 1 at 2 hr (end of infusion)137 ± 52.7304 ± 31.4——
Cycle 3 Day 5 at 2 hr (end of infusion)——315 ± 0.0—
Cycle 24 Day 1 at 2 hr (end of infusion)163 ± 23.7———
SecondaryTrough Serum Concentration of (Cmin) Sabatolimab

Cmin is the concentration of sabatolimab prior to next dosing or after end of treatment.

Time frame:
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
Reported as:
Geometric mean · µg/ml
Trough Serum Concentration of (Cmin) Sabatolimab
µg/mlSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
0-hour (hr) Pre-dose at Cycle 1 Day 10.00 ± 0.00.00 ± 0.0—0.00 ± 0.0
0 hr (pre-dose) at Cycle 1 Day 5——0.00 ± 0.0—
0 hr (pre-dose) at Cycle 2 Day 1———70.7 ± 0.0
0 hr (pre-dose) at Cycle 3 Day 140.4 ± 20.270.7 ± 47.5—129 ± 0.0
0 hr (pre-dose) at Cycle 3 Day 5——42.9 ± 0.0—
0 hr (pre-dose) at Cycle 6 Day 146.4 ± 66.499.8 ± 52.1—219 ± 0.0
0 hr (pre-dose) at Cycle 6 Day 5——71.9 ± 0.0—
0 hr (pre-dose) at Cycle 24 Day 149.7 ± 44.1———
SecondaryGraft Versus Host Disease (GvHD)-Free/Relapse-free Survival (GRFS)

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

Time frame:
From start of treatment to up to 36 months from last patient first treatment
Reported as:
Median · Months
Graft Versus Host Disease (GvHD)-Free/Relapse-free Survival (GRFS)
MonthsSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
Graft Versus Host Disease (GvHD)-Free/Relapse-free Survival (GRFS)NA (NA to NA)NA (NA to NA)NA (NA to NA)NA (NA to NA)
SecondaryRelapse-free Survival (RFS)

Time from start of treatment to the date of first documented hematologic relapse or death due to any cause, whichever occurs first.

Time frame:
From start of treatment to up to 36 months from last patient first treatment
Reported as:
Median · Months
Relapse-free Survival (RFS)
MonthsSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
Relapse-free Survival (RFS)2.56 (0.95 to NA)6.74 (0.95 to NA)NA (NA to NA)7.16 (NA to NA)
SecondaryPercentage of Participants With Measurable Residual Disease (MRD) Positive at Baseline Who Become MRD Negative

Percentage of participants with centrally confirmed MRD+ status at baseline converting to MRD- within the first 6 cycles of study treatment.

Time frame:
From start of treatment until end of Cycle 6 (Cycle = 28 Days)
Reported as:
Number · Percentage of participants
Percentage of Participants With Measurable Residual Disease (MRD) Positive at Baseline Who Become MRD Negative
Percentage of participantsSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
Percentage of Participants With Measurable Residual Disease (MRD) Positive at Baseline Who Become MRD Negative0 (0.0 to 30.8)0 (0.0 to 28.5)0 (0.0 to 84.2)0 (0.0 to 97.5)

Adverse events

Collected over Adverse Events were collected from start of treatment (FPFT) up to 30 days after the last dose of study treatment (sabatolimab or azacitidine), for a maximum duration of approx. 25 months (max. duration of treatment 24 months + 30 days). Deaths were collected on-treatment period up to 30 days and after Day 31 after last dose of study treatment.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Sabatolimab 400mg Mono Adults5/10 (50%)2/10 (20%)9/10 (90%)
Sabatolimab 800mg Mono Adults2/11 (18.2%)3/11 (27.3%)9/11 (81.8%)
Sabatolimab 800mg + Azacitidine Adults0/2 (0%)0/2 (0%)2/2 (100%)
Sabatolimab 800mg Mono Adolescent0/1 (0%)0/1 (0%)1/1 (100%)
Most frequent serious events
Most frequent serious events
EventSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
BronchitisInfections and infestations1/100/110/20/1
Febrile infectionInfections and infestations1/100/110/20/1
PneumoniaInfections and infestations1/100/110/20/1
Femoral neck fractureInjury, poisoning and procedural complications1/100/110/20/1
Acute kidney injuryRenal and urinary disorders1/100/110/20/1
MyocarditisCardiac disorders0/101/110/20/1
Oral herpesInfections and infestations0/101/110/20/1
Nervous system disorderNervous system disorders0/101/110/20/1
Most frequent other events
Showing 10 of 72
Most frequent other events
EventSabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono Adolescent
NauseaGastrointestinal disorders1/101/112/20/1
COVID-19Infections and infestations2/102/111/21/1
RhinitisInfections and infestations0/100/111/21/1
HyperkalaemiaMetabolism and nutrition disorders0/100/110/21/1
AstheniaGeneral disorders1/101/111/20/1
FatigueGeneral disorders1/101/111/20/1
Injection site painGeneral disorders0/100/111/20/1
Non-cardiac chest painGeneral disorders0/100/111/20/1
Oral bacterial infectionInfections and infestations0/100/111/20/1
Oral candidiasisInfections and infestations0/100/111/20/1

Baseline characteristics

Full Analysis Set (FAS) included all participants who received at least one dose of any component of the study treatment (i.e. at least one dose of sabatolimab or at least one dose of azacitidine).

Age, Customized
Age, Customized(Participants)Sabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono AdolescentTotal
Category 1 : 12 - <18 years00011
Category 1 : 18 - <65 years871016
Category 1 : 65 - <85 years24107
Sex: Female, Male
Sex: Female, Male(Participants)Sabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono AdolescentTotal
Female562114
Male550010
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Sabatolimab 400mg Mono AdultsSabatolimab 800mg Mono AdultsSabatolimab 800mg + Azacitidine AdultsSabatolimab 800mg Mono AdolescentTotal
White992121
Unknown12003
08

Study locations

9 sites
  • Novartis Investigative Site
    Marseille, 13273, France
  • Novartis Investigative Site
    Freiburg im Breisgau, 79106, Germany
  • Novartis Investigative Site
    Hamburg, 20246, Germany
  • Novartis Investigative Site
    Münster, 48149, Germany
  • Novartis Investigative Site
    Bergamo, BG 24127, Italy
  • Novartis Investigative Site
    Bologna, BO 40138, Italy
  • Novartis Investigative Site
    Brescia, BS 25123, Italy
  • Novartis Investigative Site
    Roma, RM 00165, Italy
  • Novartis Investigative Site
    Barcelona, Catalonia 08035, Spain
09

References and documents

Study documents

  • Study protocol · Mar 24, 2022
  • Statistical analysis plan · Jul 3, 2024

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 16, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT04623216
Lead sponsor
Novartis Pharmaceuticals
Responsible party
Sponsor
First posted
Nov 10, 2020
Start date
Sep 14, 2021
Primary completion
Aug 22, 2024
Completion
Aug 22, 2024
Results posted
Jun 3, 2025
Last update
Oct 16, 2025

Study contacts

Novartis Pharmaceuticals
study director · Novartis Pharmaceuticals

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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