Myelodysplastic SyndromesPhase 2STIMULUS-MDS1NCT03946670
A Study of MBG453 in Combination With Hypomethylating Agents in Subjects With IPSS-R Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS).
A Phase 2 study testing MBG453 and Hypomethylating agents in people with Myelodysplastic Syndromes, run by Novartis Pharmaceuticals at 48 sites in 17 countries.
Trial summary
This Phase II was a multicenter, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to hypomethylating agents (azacitidine or decitabine) in adult subjects with IPSS-R intermediate, high or very high risk myelodysplastic syndrome (MDS) not eligible for Hematopoietic… Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- MBG453 and Hypomethylating agents
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 127 participants
- Where
- 48 sitesin 17 countries
- Study dates
- 4 Jun 2019to 15 Jul 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed informed consent must be obtained prior to participation in the study.
- Age ≥ 18 years at the date of signing the informed consent form (ICF)-. Morphologically confirmed diagnosis of a myelodysplastic syndrome (MDS) based on 2016 WHO classification (Arber et al 2016) by investigator assessment with one of the following Prognostic Risk Categories, based on the International Prognostic Scoring System (IPSS-R):
- Very high
- High
- Intermediate with at least ≥ 5% bone marrow blast
- Not eligible at the time of screening, for intensive chemotherapy according to the investigator, based on local standard medical practice and institutional guidelines for treatment decisions
- Not eligible at the time of screening, for hematopoietic stem-cell transplantation (HSCT) according to the investigator, based on local standard medical practice and institutional guidelines for treatment decisions.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
Who cannot join
- Prior exposure to TIM-3 directed therapy at any time. Prior therapy with immune check point inhibitors (e.g. anti-CTLA4, anti-PD-1, anti-PD-L1, or anti-PD-L2), cancer vaccines are allowed except if the drug was administered within 4 months prior to randomization.
- Previous first-line treatment for higher risk MDS with chemotherapy or any other antineoplastic agents including lenalidomide and hypomethylating agent (HMAs) such as decitabine or azacitidine.
- History of severe hypersensitivity reactions to any ingredient of the study treatment (azacitidine, decitabine or MGB453) or their excipients, or to monoclonal antibodies (mAbs).
- Currently using or used within 14 days prior to randomization of systemic, steroid therapy (> 10 mg/day prednisone or equivalent) or any immunosuppressive therapy. Topical, inhaled, nasal, ophthalmic steroids are allowed. Replacement therapy, steroids given in the context of a transfusion are allowed and not considered a form of systemic treatment.
- Investigational treatment for MDS received within 4 weeks prior to randomization. In case of a checkpoint inhibitor: 4 months minimum prior to randomization interval is necessary to allow enrollment.
- Active autoimmune disease requiring systemic therapy (e.g.corticosteroids).
- Live vaccine administered within 30 Days prior to randomization.
Trial locations and contacts
48 sites in 17 countries.
48 sites in 17 countries
Japan9 sites
Novartis Investigative Site
Bunkyo Ku, Tokyo, 113-8677
Novartis Investigative Site
Fukuoka, Fukuoka, 812-8582
Novartis Investigative Site
Fukushima, Fukushima, 960 1295
Novartis Investigative Site
Gifu, 500 8513
Novartis Investigative Site
Isehara, Kanagawa, 259-1193
Novartis Investigative Site
Kumamoto, Kumamoto, 860-0008
Novartis Investigative Site
Nagasaki, Nagasaki, 852-8501
Novartis Investigative Site
Osaka, 545-8586
Novartis Investigative Site
Sendai, Miyagi, 983 8520
United States7 sites
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Ohio State Comprehensive Cancer Center James Cancer Hospital
Columbus, Ohio, 73210
Mary Crowley Cancer Research
Dallas, Texas, 75251
City Of Hope National Med Center
Duarte, California, 91010
City of Hope National Medical Center Medical Oncology & Therapeutic
Duarte, California, 91010
The Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
Yale University School Of Medicine
New Haven, Connecticut, 06520
Italy5 sites
Novartis Investigative Site
Florence, FI, 50134
Novartis Investigative Site
Milan, MI, 20162
Novartis Investigative Site
Reggio Calabria, RC, 89124
Novartis Investigative Site
Roma, RM, 00133
Novartis Investigative Site
Rozzano, MI, 20089
Greece3 sites
Novartis Investigative Site
Alexandroupoli, 681 00
Novartis Investigative Site
Larissa, 411 10
Novartis Investigative Site
Pátrai, 265 04
Spain3 sites
Novartis Investigative Site
L'Hospitalet de Llobregat, Barcelona, 08907
Novartis Investigative Site
Málaga, 29010
Novartis Investigative Site
Santander, Cantabria, 39008
Turkey (Türkiye)3 sites
Novartis Investigative Site
Izmir, 35100
Novartis Investigative Site
Köseköy, Kocaeli, 41380
Novartis Investigative Site
Samsun, Atakum, 55200
Belgium2 sites
Novartis Investigative Site
Brasschaat, 2930
Novartis Investigative Site
Leuven, 3000
Canada2 sites
Novartis Investigative Site
Québec, Quebec, G1J 1Z4
Novartis Investigative Site
Toronto, Ontario, M5G 2M9
Czechia2 sites
Novartis Investigative Site
Brno, 625 00
Novartis Investigative Site
Prague, 128 08
Germany2 sites
Novartis Investigative Site
Berlin, 13353
Novartis Investigative Site
Leipzig, Saxony, 04103
Hungary2 sites
Novartis Investigative Site
Debrecen, Hajdu Bihar Megye, 4032
Novartis Investigative Site
Nyíregyháza, 4400
South Korea2 sites
Novartis Investigative Site
Seoul, Korea, 03080
Novartis Investigative Site
Seoul, 06351
Taiwan2 sites
Novartis Investigative Site
Kaohsiung City, 83301
Novartis Investigative Site
Taipei, 10002
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Phase II Multi-center Study of Intravenous MBG453 Added to Hypomethylating Agents in Adult Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R Criteria
This Phase II was a multicenter, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to hypomethylating agents (azacitidine or decitabine) in adult subjects with IPSS-R intermediate, high or very high risk myelodysplastic syndrome (MDS) not eligible for Hematopoietic Stem Cell Transplant (HSCT) or intensive chemotherapy.
Read the detailed description
The 2 primary objectives were as follows:
To determine if MBG453 combined with standard HMA therapy improved complete remission in subjects with intermediate, high, or very high risk MDS.
To determine if MBG453 combined with standard HMA therapy improved progression free survival (PFS) in subjects with intermediate, high or very high risk MDS.
This Phase II study was a multicenter, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to hypomethylating agents (azacitidine or decitabine, as per investigators' choice based on local standard of care (SOC)) in adult subjects with IPSS-R intermediate, high or very high risk MDS not eligible for HSCT or intensive chemotherapy. A total of 127 subjects were randomized in a 1:1 ratio to treatment arms as follows:
- MBG453 400 mg IV Q2W and decitabine or azacitidine
- Placebo IV Q2W and decitabine or azacitidine
The randomization was stratified by 2 stratification factors: a) HMA (decitabine or azacitidine) selected by the investigator as per the local SOC and b) IPSS-R prognostic risk categories (intermediate, high or very high) at randomization. Crossover between treatment arms was not permitted at any time during the study.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- MBG453, Placebo, Hypomethylating agents
- Keywords
- Sabatolimab, Placebo, Phase II, MBG453, TIM-3, decitabine, azacitidine, myelodysplastic syndrome (MDS)
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MBG453Drug
Also called Sabatolimab
MBG453 is being administered i.v.
PlaceboDrug
Placebo is being administered i.v.
Hypomethylating agentsDrug
Decitabine is being administered i.v. Azacitidine is being administered i.v or s.c.
| Study group | Role | What they receive |
|---|---|---|
| MBG453 + hypomethylating agents | Experimental | Patients are taking MBG453 plus hypomethylating agents Receives: Drug: MBG453; Drug: Hypomethylating agents |
| Placebo + hypomethylating agents | Placebo comparator | Patients are taking placebo plus hypomethylating agents Receives: Drug: Placebo; Drug: Hypomethylating agents |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Remission (CR) Rate
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%.
Modified response criteria According to International Working Group (IWG) and as per World Health Organization (WHO) criteria for Myelodysplastic syndromes (MDS) as per investigator assessment.
Measured: average of 7 months
Progression Free Survival (PFS)
PFS is defined as time from randomization to disease progression (including transformation to acute 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.
Measured: approx. 32 months
Secondary outcomes (13)
Progression Free Survival (PFS) - Final PFS
PFS isdefined as time from randomization to disease progression (including transformation to acute leukemia per WHO 2016 classification), relapse from CR according to IWG-MDS or death due to any cause. This is an update of the Primary Outcome Measure PFS with data collected after assessment of the primary results.
Measured: approx. 48 months
Overall Survival (OS)
Time from randomization to death due to any cause
Measured: approx. 48 months
Event-free Survival (EFS)
EFS is defined as the time from randomization to lack of reaching complete response (CR) within the first 6 months, relapse from CR or death due to any cause, whichever occurs first. CR and relapse from CR were defined according to International Working Group (IWG) for Myelodysplastic Syndromes (MDS) as per Investigator assessment. For participants not reaching CR within the first 6 months, an EFS event at day 1 was considered.
Measured: approx. 48 months
Leukemia-free Survival (LFS)
LFS is defined as the time from randomization to ≥ 20% blasts in bone marrow/peripheral blood (per World Health Organization (WHO) 2016 classification) or diagnosis of extramedullary acute leukemia or death due to any cause.
Measured: approx. 48 months
Response Rate of Complete Remission (CR)/Marrow Complete Remission (mCR)/Partial Remission (PR)/Hematopoietic Improvement (HI))
Percentage of complete remission (CR)/marrow Complete Remission (mCR)/partial remission (PR) \& Hematological improvement (HI) as per investigator assessment according to IWG-MDS. 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: where the Bone marrow ≤5% blasts and blast count decrease by ≥50% compared to baseline 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. HI definition is based on modified Hematological Improvement per IWG-MDS criteria in MDS \& is the combination of Erythroid response (HI-E), Platelet response (HI-P) \& Neutrophil response (HI-N).
Measured: approx. 32 months
Duration of Complete Remission
Duration of complete response is the time from the date of the first documented CR to the date of first documented relapse from CR or death due to any cause, whichever occurs first.
Measured: approx. 48 months
Time to Complete Remission
Time from randomization to the first documented CR
Measured: Average of 7 months
Percent of Participants Who Are Red Blood Cells (RBC)/Platelets Transfusion Independent After Randomization as Per IWG-MDS
Improvement in RBC/platelets transfusion independence. RBC/platelets transfusion independence rate is defined as the percentage of participants having received no RBC/platelets transfusions during at least 8 consecutive weeks after randomization.
Measured: approx. 48 months
Red Blood Cells (RBC) Transfusion Independence Duration After Randomization
The total duration of all transfusion independence periods is the sum of each period of the transfusion independence.
RBC transfusions independence period is defined as the period for which participants having received no RBC transfusions during at least 8 consecutive weeks after randomization.
Measured: approx. 48 months
Platelets Transfusion Independence Duration After Randomization
The total duration of all transfusion independence periods is the sum of each period of the transfusion independence.
Platelets transfusions independence period is defined as the period for which participants having received no platelets transfusions during at least 8 consecutive weeks after randomization.
Measured: approx. 48 months
Serum Concentrations for MBG453
Pharmacokinetics (PK) of MBG453 when given in combination with hypomethylating agents (HMA)
Measured: 0hr pre-dose on Day 8 of each cycle until cycle 6 and on Day 8 of cycles 9, 12, 18 and 24, 2hr post-dose on Day 8 of C1 and C3, EOT (approx. 48 months) and up to 150 day of the safety follow up period; 1 cycle = 28 days
Immunogenicity (IG) of MBG453 When Given in Combination of Hypomethylating Agents: ADA Prevalence
Number of participants with at least one sample meeting the criteria at baseline. Anti-drug Antibody (ADA) prevalence equals ADA-positive at baseline.
Measured: at baseline
Immunogenicity of MBG453 When Given in Combination of Hypomethylating Agents: ADA-positive Participants
Anti-drug Antibody (ADA) positive participants on-treatment was calculated as the number of participants with at least 1 on-treatment ADA-positive sample divided by the number of participants with a determinant baseline IG sample and at least one determinant post-baseline IG sample.
Measured: approx. 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2019
- Study start4 Jun 2019
- Primary completion26 Apr 2022
- Study completion15 Jul 2024
Why it stopped: In December 2023, Novartis decided to terminate the sabatolimab clinical development program early after Phase II (MDS1) and Phase III (MDS2) studies failed to meet their primary objectives. The termination was not due to safety concerns.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 127 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03946670
- Sponsor's protocol ID
- CMBG453B12201
- Other IDs
- 2018-004479-11, 2018
- Acronym
- STIMULUS-MDS1
- Status last verified
- Dec 2025
- First submitted
- 8 May 2019
- Last updated
- 13 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Publications
Zeidan AM, Ando K, Rauzy O, Turgut M, Wang MC, Cairoli R, Hou HA, Kwong YL, Arnan M, Meers S, Pullarkat V, Santini V, Malek K, Kiertsman F, Niolat J, Ramos PM, Menssen HD, Fenaux P, Miyazaki Y, Platzbecker U. Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Haematol. 2024 Jan;11(1):e38-e50. doi: 10.1016/S2352-3026(23)00333-2. Epub 2023 Dec 5.
PubMed 38065203
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Phase II Multi-center Study of Intravenous MBG453 Added to Hypomethylating Agents in Adult Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R Criteria
- ClinicalTrials.gov ID
- NCT03946670
- Acronym / sponsor ID
- STIMULUS-MDS1 / CMBG453B12201
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Sabatolimab, Placebo, Phase II, MBG453, TIM-3, decitabine, azacitidine, myelodysplastic syndrome (MDS)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: In December 2023, Novartis decided to terminate the sabatolimab clinical development program early after Phase II (MDS1) and Phase III (MDS2) studies failed to meet their primary objectives. The termination was not due to safety concerns.
- Enrolment
- 127 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 4 Jun 2019 (actual)
- Primary completion
- 26 Apr 2022 (actual)
- Study completion
- 15 Jul 2024 (actual)
- First posted
- 13 May 2019 (actual)
- Results
- Posted 7 Jul 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Phase II was a multicenter, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to hypomethylating agents (azacitidine or decitabine) in adult subjects with IPSS-R intermediate, high or very high risk myelodysplastic syndrome (MDS) not eligible for Hematopoietic Stem Cell Transplant (HSCT) or intensive chemotherapy.
Detailed description
The 2 primary objectives were as follows:
To determine if MBG453 combined with standard HMA therapy improved complete remission in subjects with intermediate, high, or very high risk MDS.
To determine if MBG453 combined with standard HMA therapy improved progression free survival (PFS) in subjects with intermediate, high or very high risk MDS.
This Phase II study was a multicenter, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to hypomethylating agents (azacitidine or decitabine, as per investigators' choice based on local standard of care (SOC)) in adult subjects with IPSS-R intermediate, high or very high risk MDS not eligible for HSCT or intensive chemotherapy. A total of 127 subjects were randomized in a 1:1 ratio to treatment arms as follows:
- MBG453 400 mg IV Q2W and decitabine or azacitidine
- Placebo IV Q2W and decitabine or azacitidine
The randomization was stratified by 2 stratification factors: a) HMA (decitabine or azacitidine) selected by the investigator as per the local SOC and b) IPSS-R prognostic risk categories (intermediate, high or very high) at randomization. Crossover between treatment arms was not permitted at any time during the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MBG453 + hypomethylating agents | Experimental | Drug: MBG453, Drug: Hypomethylating agentsPatients are taking MBG453 plus hypomethylating agents |
| Placebo + hypomethylating agents | Placebo comparator | Drug: Placebo, Drug: Hypomethylating agentsPatients are taking placebo plus hypomethylating agents |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Signed informed consent must be obtained prior to participation in the study.
- Age ≥ 18 years at the date of signing the informed consent form (ICF)-. Morphologically confirmed diagnosis of a myelodysplastic syndrome (MDS) based on 2016 WHO classification (Arber et al 2016) by investigator assessment with one of the following Prognostic Risk Categories, based on the International Prognostic Scoring System (IPSS-R):
- Not eligible at the time of screening, for intensive chemotherapy according to the investigator, based on local standard medical practice and institutional guidelines for treatment decisions
- Not eligible at the time of screening, for hematopoietic stem-cell transplantation (HSCT) according to the investigator, based on local standard medical practice and institutional guidelines for treatment decisions.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
Key exclusion criteria (7 listed)
- Prior exposure to TIM-3 directed therapy at any time. Prior therapy with immune check point inhibitors (e.g. anti-CTLA4, anti-PD-1, anti-PD-L1, or anti-PD-L2), cancer vaccines are allowed except if the drug was administered within 4 months prior to randomization.
- Previous first-line treatment for higher risk MDS with chemotherapy or any other antineoplastic agents including lenalidomide and hypomethylating agent (HMAs) such as decitabine or azacitidine.
- History of severe hypersensitivity reactions to any ingredient of the study treatment (azacitidine, decitabine or MGB453) or their excipients, or to monoclonal antibodies (mAbs).
- Currently using or used within 14 days prior to randomization of systemic, steroid therapy (> 10 mg/day prednisone or equivalent) or any immunosuppressive therapy. Topical, inhaled, nasal, ophthalmic steroids are allowed. Replacement therapy, steroids given in the context of a transfusion are allowed and not considered a form of systemic treatment.
- Investigational treatment for MDS received within 4 weeks prior to randomization. In case of a checkpoint inhibitor: 4 months minimum prior to randomization interval is necessary to allow enrollment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MBG453Also: SabatolimabMBG453 is being administered i.v. | Drug | MBG453 + hypomethylating agents |
| PlaceboPlacebo is being administered i.v. | Drug | Placebo + hypomethylating agents |
| Hypomethylating agentsDecitabine is being administered i.v. Azacitidine is being administered i.v or s.c. | Drug | MBG453 + hypomethylating agents, Placebo + hypomethylating agents |
Primary outcome
Complete Remission (CR) Rate
Time frame: average of 7 months
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%. Modified response criteria According to International Working Group (IWG) and as per World Health Organization (WHO) criteria for Myelodysplastic syndromes (MDS) as per investigator assessment.
Progression Free Survival (PFS)
Time frame: approx. 32 months
PFS is defined as time from randomization to disease progression (including transformation to acute 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.
13 secondary outcomes
Progression Free Survival (PFS) - Final PFS
Time frame: approx. 48 months
Overall Survival (OS)
Time frame: approx. 48 months
Event-free Survival (EFS)
Time frame: approx. 48 months
Leukemia-free Survival (LFS)
Time frame: approx. 48 months
Response Rate of Complete Remission (CR)/Marrow Complete Remission (mCR)/Partial Remission (PR)/Hematopoietic Improvement (HI))
Time frame: approx. 32 months
Duration of Complete Remission
Time frame: approx. 48 months
Time to Complete Remission
Time frame: Average of 7 months
Percent of Participants Who Are Red Blood Cells (RBC)/Platelets Transfusion Independent After Randomization as Per IWG-MDS
Time frame: approx. 48 months
Red Blood Cells (RBC) Transfusion Independence Duration After Randomization
Time frame: approx. 48 months
Platelets Transfusion Independence Duration After Randomization
Time frame: approx. 48 months
Serum Concentrations for MBG453
Time frame: 0hr pre-dose on Day 8 of each cycle until cycle 6 and on Day 8 of cycles 9, 12, 18 and 24, 2hr post-dose on Day 8 of C1 and C3, EOT (approx. 48 months) and up to 150 day of the safety follow up period; 1 cycle = 28 days
Immunogenicity (IG) of MBG453 When Given in Combination of Hypomethylating Agents: ADA Prevalence
Time frame: at baseline
Immunogenicity of MBG453 When Given in Combination of Hypomethylating Agents: ADA-positive Participants
Time frame: approx. 48 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38065203Zeidan AM, Ando K, Rauzy O, Turgut M, Wang MC, Cairoli R, Hou HA, Kwong YL, Arnan M, Meers S, Pullarkat V, Santini V, Malek K, Kiertsman F, Niolat J, Ramos PM, Menssen HD, Fenaux P, Miyazaki Y, Platzbecker U. Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Haematol. 2024 Jan;11(1):e38-e50. doi: 10.1016/S2352-3026(23)00333-2. Epub 2023 Dec 5.
- A Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
48 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MBG453 and Hypomethylating agents in people with Myelodysplastic Syndromes, run by Novartis Pharmaceuticals at 48 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 48 sites in Japan, United States, Italy, Greece, Spain and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Jul 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete Remission (CR) Rate (time frame: average of 7 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03946670, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jan 2026.