Trial · NCT04266301
Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2)
NCT04266301, Phase 3, Terminated, Novartis Pharmaceuticals; results first posted 2025-12-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2). Trials, record NCT04266301. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04266301
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2)
- NCT number
- NCT04266301
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 530 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
- Interventions
- Sabatolimab, Azacitidine, Placebo
- First posted
- 2020-02-12
- Last updated
- 2026-01-13
- Other registries' IDs
- eudract:2019-002089-11
- Results first posted
- 2025-12-12
- Limitations and caveats
A total of 530 participants were randomized (265 to sabatolimab+AZA, 265 to placebo+AZA), but 1 participant in each arm did not receive any study treatment and were excluded from the Safety Set. Another sabatolimab-assigned participant received only AZA and thus was summarized in the placebo+AZA arm in all safety analyses. Therefore, the Serious and Other Adverse Events were shown for 263 sabatolimab+AZA and 265 placebo+AZA participants.
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Survival (OS) (Primary Efficacy Results) | up to approx. 39 months | OS is the time from randomization until death due to any cause. |
| Primary | Overall Survival (OS) (Final Efficacy Results) | up to approx. 52 months | OS is the time from randomization until death due to any cause. |
| Secondary | Key Secondary Endpoint 1: Time to Definitive Deterioration of Fatigue Using Functional Assessment of Cancer Therapy (FACIT)-Fatigue Score | up to approx. 52 months | FACIT-Fatigue score is a 13-item questionnaire designed to assess fatigue in cancer participants. All items use a 5-point scale ranging from 0 to 4 (0=Not at All to 4=Very Much). The total score ranges from 0 to 52 with higher values representing better quality of life. Time to definitive deterioration of fatigue is defined as time from randomization to at least 3 points worsening from baseline in FACIT-fatigue scores with no subsequently observed improvement above this threshold, or death due to any cause, whichever occurred first. |
| Secondary | Key Secondary Endpoint 2: Red Blood Cell (RBC) Annualized Transfusion Free Rate for Transfusion | up to approx. 52 months | Annualized transfusion free rate is defined as the average number of days in RBC transfusion-free intervals in a year (i.e., the total number of days in RBC transfusion-free intervals divided by the total days in the study multiplied by 365.25), where RBC transfusion-free intervals correspond to cumulative times of intervals with no evidence of RBC transfusion for at least 8 weeks at any point after randomization until death due to any cause. |
| Secondary | Key Secondary Endpoint 3: Percentage of Participants With at Least 3 Point Confirmed Improvement From Baseline in FACIT-fatigue Scores | up to approx. 52 months | FACIT-Fatigue score is a 13-item questionnaire designed to assess fatigue in cancer participants. All items use a 5-point scale ranging from 0 to 4 (0=Not at All to 4=Very Much). The total score ranges from 0 to 52 with higher values representing better quality of life. The responder is defined as having 3 points improvement from baseline confirmed by a second improvement of 3 points at any time, regardless of preceding worsening. A participant who could not improve was considered as a non-responder. |
| Secondary | Key Secondary Endpoint 4: Percentage of Participants With at Least 10 Point Confirmed Improvement From Baseline in Physical Functioning Using European Organization for Research and Treatment of Cancer's Core Quality of Life Questionnaire (EORTC QLQ-C30) | up to approx. 52 months | EORTC-QLQ-C30 is a 30-item questionnaire developed to assess the quality of life of cancer participants. Participants' responses to 5 questions about their physical functioning (Items 1-5) are scored on a 4-point scale (1=Not at All to 4=Very Much). Using linear transformation, raw scores are standardized, so that scores range from 0 to 100. A high score indicates a high / healthy level of functioning. The responder is defined as having 10 points improvement from baseline confirmed by a second improvement of 10 points at any time, regardless of preceding worsening. A participant who could not improve was considered as a non-responder. |
| Secondary | Key Secondary Endpoint 5: Percentage of Participants With at Least 10 Point Confirmed Improvement From Baseline in Emotional Functioning Using EORTC-QLQ-C30 | up to approx. 52 months | EORTC-QLQ-C30 is a 30-item questionnaire developed to assess the quality of life of cancer participants. Participants' responses to 4 questions about their emotional functioning (Items 21-24) are scored on a 4-point scale (1=Not at All to 4=Very Much). Using linear transformation, raw scores are standardized, so that scores range from 0 to 100. A high score indicates a high / healthy level of functioning. The responder is defined as having 10 points improvement from baseline confirmed by a second improvement of 10 points at any time, regardless of preceding worsening. A participant who could not improve was considered as a non-responder. |
| Secondary | Percentage of Participants With Either CR, or mCR, or PR, or HI in Each Treatment Arm According to International Working Group for MDS (IWG-MDS) as Per Investigator Assessment | up to approx. 52 months | Response rate of participants with complete remission (CR), or marrow remission (mCR), or partial remission (PR), or hematologic improvement (HI). CR: where the Bone marrow: ≤ 5% blasts with normal maturation of all cell lineages and Peripheral blood: where Hgb ≥ 10 g/dL AND Platelets ≥ 100\*109/L AND Neutrophils ≥ 1.0\*109/L AND Peripheral blasts 0%. mCR: Bone marrow: ≤ 5% blasts and blast count decrease by ≥ 50% compared to baseline; Peripheralblood/transfusion: Marrow CR may be achieved with or without improved blood counts or with or without transfusions PR: All CR criteria except bone marrow: ≥50% decrease from baseline in blasts in bone marrow AND blast count in bone marrow \>5%. HI: restoration or enhancement of the function of the body's blood cell-producing system that must last as least 8 weeks. |
| Secondary | Percentage of Participants With Stable Disease (SD) According to International Working Group for MDS (IWG-MDS) as Per Investigator Assessment | up to approx. 52 months | Response rate of participants with stable disease. SD is the failure to achieve at least partial response (PR), but no evidence of progression for \>8 weeks. |
| Secondary | Progression Free Survival (PFS) | up to approx. 52 months | PFS is defined as the time from randomization to disease progression (including transformation to acute leukemia per WHO 2016), relapse from CR (IWG-MDS), or death. Disease progression: bone marrow blasts increase ≥ 50% over baseline, to \> 5% if initially \< 5%, \> 10% if 5%-\<10%, or \> 20% if 10%-\<20%. Includes a peripheral blood count decrease ≥ 50% from maximum remission/response levels: neutrophils \< 1.0x109/L, platelets \< 100x109/L, or hemoglobin drop ≥ 2 g/dL to \< 10 g/dL, becoming transfusion-dependent. Relapse from CR: baseline bone marrow blast % return, neutrophils decrease ≥ 50% to \< 1.0x109/L, platelets decrease ≥ 50% to \< 100x109/L, or hemoglobin drop ≥ 1.5 g/dL to \< 10 g/dL, becoming transfusion-dependent. Leukemia transformation: \> 20% blasts per WHO 2016 (Arber et al 2016). |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Survival (OS) (Primary Efficacy Results) | up to approx. 39 months | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Months | MEDIAN |
| PRIMARY | Overall Survival (OS) (Final Efficacy Results) | up to approx. 52 months | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Months | MEDIAN |
| SECONDARY | Key Secondary Endpoint 1: Time to Definitive Deterioration of Fatigue Using Functional Assessment of Cancer Therapy (FACIT)-Fatigue Score | up to approx. 52 months | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Months | MEDIAN |
| SECONDARY | Key Secondary Endpoint 2: Red Blood Cell (RBC) Annualized Transfusion Free Rate for Transfusion | up to approx. 52 months | FAS comprised of all participants to whom study treatment had been assigned by randomization. | days per year | MEDIAN |
| SECONDARY | Key Secondary Endpoint 3: Percentage of Participants With at Least 3 Point Confirmed Improvement From Baseline in FACIT-fatigue Scores | up to approx. 52 months | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Percentage of participants with response | NUMBER |
| SECONDARY | Key Secondary Endpoint 4: Percentage of Participants With at Least 10 Point Confirmed Improvement From Baseline in Physical Functioning Using European Organization for Research and Treatment of Cancer's Core Quality of Life Questionnaire (EORTC QLQ-C30) | up to approx. 52 months | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Percentage of participants with response | NUMBER |
| SECONDARY | Key Secondary Endpoint 5: Percentage of Participants With at Least 10 Point Confirmed Improvement From Baseline in Emotional Functioning Using EORTC-QLQ-C30 | up to approx. 52 months | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Either CR, or mCR, or PR, or HI in Each Treatment Arm According to International Working Group for MDS (IWG-MDS) as Per Investigator Assessment | up to approx. 52 months | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Stable Disease (SD) According to International Working Group for MDS (IWG-MDS) as Per Investigator Assessment | up to approx. 52 months | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Percentage of participants | NUMBER |
| SECONDARY | Progression Free Survival (PFS) | up to approx. 52 months | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Months | MEDIAN |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group110
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Survival (OS) (Primary Efficacy Results) | Sabatolimab (MBG453) + Azacitidine | 22.31 | 95% Confidence Interval 19.55 to 24.74 | Months | |
| Overall Survival (OS) (Primary Efficacy Results) | Placebo + Azacitidine | 18.83 | 95% Confidence Interval 15.38 to 23.72 | Months | |
| Overall Survival (OS) (Final Efficacy Results) | Sabatolimab (MBG453) + Azacitidine | 22.18 | 95% Confidence Interval 19.55 to 24.41 | Months | |
| Overall Survival (OS) (Final Efficacy Results) | Placebo + Azacitidine | 18.83 | 95% Confidence Interval 15.38 to 23.72 | Months | |
| Key Secondary Endpoint 1: Time to Definitive Deterioration of Fatigue Using Functional Assessment of Cancer Therapy (FACIT)-Fatigue Score | Sabatolimab (MBG453) + Azacitidine | 13.37 | 95% Confidence Interval 11.96 to 15.90 | Months | |
| Key Secondary Endpoint 1: Time to Definitive Deterioration of Fatigue Using Functional Assessment of Cancer Therapy (FACIT)-Fatigue Score | Placebo + Azacitidine | 11.76 | 95% Confidence Interval 10.15 to 13.86 | Months | |
| Key Secondary Endpoint 2: Red Blood Cell (RBC) Annualized Transfusion Free Rate for Transfusion | Sabatolimab (MBG453) + Azacitidine | 184.3 | Full Range 0 to 365 | days per year | |
| Key Secondary Endpoint 2: Red Blood Cell (RBC) Annualized Transfusion Free Rate for Transfusion | Placebo + Azacitidine | 175.7 | Full Range 0 to 365 | days per year | |
| Key Secondary Endpoint 3: Percentage of Participants With at Least 3 Point Confirmed Improvement From Baseline in FACIT-fatigue Scores | Sabatolimab (MBG453) + Azacitidine | 39.6 | 95% Confidence Interval 33.7 to 45.8 | Percentage of participants with response | |
| Key Secondary Endpoint 3: Percentage of Participants With at Least 3 Point Confirmed Improvement From Baseline in FACIT-fatigue Scores | Placebo + Azacitidine | 40.8 | 95% Confidence Interval 34.8 to 46.9 | Percentage of participants with response |
1 to 10 of 110Source ClinicalTrials.gov
See all 110 results by groupParticipant flow22
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sabatolimab (MBG453) + Azacitidine | 265 | |
| Overall Study | STARTED | Placebo + Azacitidine | 265 | |
| Overall Study | Participants Treated | Sabatolimab (MBG453) + Azacitidine | 264 | |
| Overall Study | Participants Treated | Placebo + Azacitidine | 264 | |
| Overall Study | Participants Not treated | Sabatolimab (MBG453) + Azacitidine | 1 | |
| Overall Study | Participants Not treated | Placebo + Azacitidine | 1 | |
| Overall Study | Discontinued from treatment | Sabatolimab (MBG453) + Azacitidine | 264 | |
| Overall Study | Discontinued from treatment | Placebo + Azacitidine | 264 | |
| Overall Study | Entered post-treatment | Sabatolimab (MBG453) + Azacitidine | 68 | |
| Overall Study | Entered post-treatment | Placebo + Azacitidine | 71 |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 participant flowAdverse events584
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Sabatolimab (MBG453) + Azacitidine | 107 | 263 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo + Azacitidine | 91 | 265 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Sabatolimab (MBG453) + Azacitidine | 19 | 263 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Placebo + Azacitidine | 21 | 265 | |
| other | Leukopenia | Blood and lymphatic system disorders | Sabatolimab (MBG453) + Azacitidine | 17 | 263 | |
| other | Leukopenia | Blood and lymphatic system disorders | Placebo + Azacitidine | 22 | 265 | |
| other | Neutropenia | Blood and lymphatic system disorders | Sabatolimab (MBG453) + Azacitidine | 96 | 263 | |
| other | Neutropenia | Blood and lymphatic system disorders | Placebo + Azacitidine | 76 | 265 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Sabatolimab (MBG453) + Azacitidine | 67 | 263 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Placebo + Azacitidine | 58 | 265 |
1 to 10 of 584Source ClinicalTrials.gov
See all 584 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sabatolimab (MBG453) + Azacitidine | 161 | 265 |
| Total, all-cause mortality | Placebo + Azacitidine | 160 | 265 |
| Total, other adverse events | Sabatolimab (MBG453) + Azacitidine | 256 | 263 |
| Total, other adverse events | Placebo + Azacitidine | 255 | 265 |
| Total, serious adverse events | Sabatolimab (MBG453) + Azacitidine | 174 | 263 |
| Total, serious adverse events | Placebo + Azacitidine | 164 | 265 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37083373 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Sabatolimab (MBG453) + Azacitidine vs Placebo + Azacitidine: Number of Participants Who Become Red Blood Cells (RBC) Transfusion Independent After Randomization | Trial comparisons |
| Sabatolimab (MBG453) + Azacitidine vs Placebo + Azacitidine: Number of Participants Who Become Platelets Transfusion Independent After Randomization | Trial comparisons |
| Sabatolimab (MBG453) + Azacitidine vs Placebo + Azacitidine: deaths from any cause | Trial comparisons |
| Sabatolimab (MBG453) + Azacitidine vs Placebo + Azacitidine: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04266301
GET https://conductscience.org/api/trials/v1/NCT04266301/results?page=2