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NCT04878432

STIMULUS MDS-US : Sabatolimab Added to HMA in Higher Risk MDS

Terminated Phase 2 Results posted Last updated 16 October 2025
What this trial tests

Phase 2 trial testing MBG453 in Myelodysplastic Syndrome (MDS) in 39 participants. Terminated before completion.

Timeline
17 March 2022
Primary endpoint
26 October 2023
1 September 2024

Quick facts

Lead sponsorNovartis Pharmaceuticals
PhasePhase 2
StatusTerminated
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment39
Start date17 March 2022
Primary completion26 October 2023
Estimated completion1 September 2024
Sites15 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Novartis Pharmaceuticals — full company profile →

Who can join

Adults 18 to 99, any sex, with Myelodysplastic Syndrome (MDS). Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events Primary · Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months.

An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a participant or clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product.

Adverse events (AEs)
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)39
Treatment-related Adverse events
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)30
AEs with grade ≥ 3
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)35
Treatment-related AEs with grade ≥ 3
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)25
Serious Adverse Events (SAEs)
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)23
Treatment-related SAEs
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)8
Fatal SAEs
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)0
Treatment-related Fatal SAEs
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)0
Number of Participants With Complete Remission According to International Working Group (IWG) for MDS (2006) With MBG453 (Sabatolimab) in Combination With HMAs (IV/SC/Oral) by 12 Months. Secondary · up to Month 12

Complete remission rate was assessed according to International Working Group (IWG) for MDS (2006) with MBG453 (sabatolimab) in combination with HMAs (IV/SC/Oral) in Participants with intermediate, high, or very high risk MDS by 12 months.

GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)1
Number of Participants With Progression Free Survival - Death and Disease Progression Secondary · up to Month 24

Defined as time from enrollment to disease progression (including transformation to leukemia per WHO 2016 classification), relapse from CR according to IWG-MDS or death due to any cause, whichever occurs first, as per investigator assessment by 12 months post last patient first treatment (LPFT).

Death
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)7
Disease Progression
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)9
Progression Free Survival - 50th Percentile Secondary · up to Month 24

Defined as time from enrollment to disease progression (including transformation to leukemia per WHO 2016 classification), relapse from CR according to IWG-MDS or death due to any cause, whichever occurs first, as per investigator assessment by 12 months post last patient first treatment (LPFT).

GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)11.564.24 – 16.59
Progression Free Survival - Percent Probability - Kaplan-Meier Probability Estimates Secondary · Months 6 and 12

Defined as time from enrollment to disease progression (including transformation to leukemia per WHO 2016 classification), relapse from CR according to IWG-MDS or death due to any cause, whichever occurs first, as per investigator assessment by 12 months post last patient first treatment (LPFT).

6 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)65.2843.18 – 80.53
12 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)43.2719.45 – 65.14
Overall Survival - Number of Participants Who Died Secondary · up to Month 24

Overall Survival (OS) is defined as the time from the first dose of study medication to death due to any cause.

GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)20
Overall Survival - 50th Percentile Secondary · up to Month 24

Overall Survival (OS) is defined as the time from the first dose of study medication to death due to any cause.

GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)15.778.74 – 19.19
Overall Survival - Percent Probability - Kaplan-Meier Probability Estimates Secondary · up to Month 24

Overall Survival (OS) is defined as the time from the first dose of study medication to death due to any cause.

6 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)82.9065.77 – 91.95
9 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)65.6646.24 – 79.50
12 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)61.5641.79 – 76.35
15 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)53.3533.57 – 69.65
18 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)35.5716.40 – 55.37
21 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)28.4510.67 – 49.37
24 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)21.346.07 – 42.68
Leukemia-free Survival - Number of Participants Who Died and Number of Participants Who Progressed to Leukemia Secondary · up to Month 24

Leukemia-free survival (LFS) is the time from the date of the first dose of medication to ≥ 20% blasts in bone marrow/peripheral blood (per WHO 2016 classification) or death due to any cause.

Death
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)16
Progressed to leukemia
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)6
Leukemia-free Survival - 50th Percentile Secondary · up to Month 24

Leukemia-free survival (LFS) is the time from the date of the first dose of medication to ≥ 20% blasts in bone marrow/peripheral blood (per WHO 2016 classification) or death due to any cause.

GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)11.306.01 – 16.59
Leukemia-free Survival - Percent Probability - Kaplan-Meier Probability Estimates Secondary · Months 6 and 12

Leukemia-free survival (LFS) is the time from the date of the first dose of medication to ≥ 20% blasts in bone marrow/peripheral blood (per WHO 2016 classification) or death due to any cause.

6 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)70.5850.67 – 83.65
12 months
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)48.0427.46 – 65.98
Percentage of Participants With Complete Remission, Marrow Complete Remission and/or Partial Remission Secondary · up to Month 24

Percentage of complete remission, marrow complete remission, and/or partial remission according to IWG-MDS remission criteria as per investigator assessment

Complete Remission (CR)
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)1
Marrow Complete Remission (mCR)
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)6
mCR with hematological improvement (HI)
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)4
Partial Remission (PR)
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)2
Stable Disease (SD)
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)11
SD with hematological improvement (HI)
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)1
CR+mCR+PR
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)9
CR+PR+HI
GroupValue95% CI
MBG453 (Sabatolimab) + Hypomethylating Agents (HMA)8

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events are reported from the first dose of study medication up to approximately 24 months plus 30 - 150 day safety follow up dependent on HMA, for a maximum timeframe of approximately 29 months.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

MBG453+HMA
Serious: 23/39 (59%)
Deaths: 20/39

Serious adverse events (35 terms)

ReactionSystemMBG453+HMA
Febrile neutropeniaBlood and lymphatic system disorders
PneumoniaInfections and infestations
Atrial fibrillationCardiac disorders
Gastrointestinal haemorrhageGastrointestinal disorders
PyrexiaGeneral disorders
Urinary tract infectionInfections and infestations
FallInjury, poisoning and procedural complications
Immune thrombocytopeniaBlood and lymphatic system disorders
Angina pectorisCardiac disorders
Sinus tachycardiaCardiac disorders
TachycardiaCardiac disorders
PhotopsiaEye disorders
HaematocheziaGastrointestinal disorders
ProctitisGastrointestinal disorders
Small intestinal obstructionGastrointestinal disorders
MalaiseGeneral disorders
Physical deconditioningGeneral disorders
BacteraemiaInfections and infestations
COVID-19Infections and infestations
CellulitisInfections and infestations
Device related infectionInfections and infestations
Enterocolitis infectiousInfections and infestations
SepsisInfections and infestations
Post procedural haemorrhageInjury, poisoning and procedural complications
HypervolaemiaMetabolism and nutrition disorders
Other adverse events (68 terms — click to expand)

ReactionSystemMBG453+HMA
AnaemiaBlood and lymphatic system disorders
Platelet count decreasedInvestigations
White blood cell count decreasedInvestigations
ConstipationGastrointestinal disorders
Neutrophil count decreasedInvestigations
FatigueGeneral disorders
HeadacheNervous system disorders
NauseaGastrointestinal disorders
DiarrhoeaGastrointestinal disorders
Oedema peripheralGeneral disorders
DizzinessNervous system disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
PruritusSkin and subcutaneous tissue disorders
HypertensionVascular disorders
StomatitisGastrointestinal disorders
ChillsGeneral disorders
FallInjury, poisoning and procedural complications
HypokalaemiaMetabolism and nutrition disorders
Back painMusculoskeletal and connective tissue disorders
InsomniaPsychiatric disorders
CoughRespiratory, thoracic and mediastinal disorders
PyrexiaGeneral disorders
ArthralgiaMusculoskeletal and connective tissue disorders
PollakiuriaRenal and urinary disorders
EpistaxisRespiratory, thoracic and mediastinal disorders
HyperhidrosisSkin and subcutaneous tissue disorders
ThrombocytopeniaBlood and lymphatic system disorders
ContusionInjury, poisoning and procedural complications
Infusion related reactionInjury, poisoning and procedural complications
Abnormal loss of weightMetabolism and nutrition disorders
Decreased appetiteMetabolism and nutrition disorders
HyponatraemiaMetabolism and nutrition disorders
MyalgiaMusculoskeletal and connective tissue disorders
Pain in extremityMusculoskeletal and connective tissue disorders
DysgeusiaNervous system disorders
Febrile neutropeniaBlood and lymphatic system disorders
NeutropeniaBlood and lymphatic system disorders
VomitingGastrointestinal disorders
Lymphocyte count decreasedInvestigations
HypomagnesaemiaMetabolism and nutrition disorders

Most-reported serious reactions: Febrile neutropenia, Pneumonia, Atrial fibrillation, Gastrointestinal haemorrhage, Pyrexia, Urinary tract infection, Fall, Immune thrombocytopenia.

Data from ClinicalTrials.gov NCT04878432 adverse events section.

Sponsor's own description

The main objective of this study was to assess the safety profile of MBG453 (sabatolimab) in combination with FDA approved hypomethylating agents (HMAs) of investigator's choice (IV Decitabine or Azacitidine /SC Azacitidine /Oral Decitabine (cedazuridine combination (INQOVI))).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Drugging the efferocytosis process: concepts and opportunities.
    Mehrotra P, Ravichandran KS. · · 2022 · cited 319× · PMID 35650427 · DOI 10.1038/s41573-022-00470-y
  2. Targeting LAG-3, TIM-3, and TIGIT for cancer immunotherapy.
    Cai L, Li Y, Tan J, Xu L, et al · · 2023 · cited 232× · PMID 37670328 · DOI 10.1186/s13045-023-01499-1
  3. Immune checkpoint modulators in cancer immunotherapy: recent advances and emerging concepts.
    Wang Y, Zhang H, Liu C, Wang Z, et al · · 2022 · cited 179× · PMID 35978433 · DOI 10.1186/s13045-022-01325-0
  4. LAG-3, TIM-3, and TIGIT: Distinct functions in immune regulation.
    Joller N, Anderson AC, Kuchroo VK. · · 2024 · cited 159× · PMID 38354701 · DOI 10.1016/j.immuni.2024.01.010
  5. Recent advances in targeted therapies in acute myeloid leukemia.
    Bhansali RS, Pratz KW, Lai C. · · 2023 · cited 151× · PMID 36966300 · DOI 10.1186/s13045-023-01424-6
  6. Harnessing the tumor microenvironment: targeted cancer therapies through modulation of epithelial-mesenchymal transition.
    Glaviano A, Lau HS, Carter LM, Lee EHC, et al · · 2025 · cited 90× · PMID 39806516 · DOI 10.1186/s13045-024-01634-6
  7. Immune Checkpoint Inhibition in Acute Myeloid Leukemia and Myelodysplastic Syndromes.
    Abaza Y, Zeidan AM. · · 2022 · cited 61× · PMID 35883692 · DOI 10.3390/cells11142249
  8. TIM-3 in Leukemia; Immune Response and Beyond.
    Rezaei M, Tan J, Zeng C, Li Y, et al · · 2021 · cited 53× · PMID 34660319 · DOI 10.3389/fonc.2021.753677

Verify or expand the search:

Other trials of MBG453

Trials testing the same drug.

Other recruiting trials for Myelodysplastic Syndrome (MDS)

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Trials by the same sponsor.

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04878432.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing