Myelodysplastic SyndromesPhase 2NCT04823624
MBG453 in Lower Risk MDS
A Phase 2 study testing MBG453 in people with Myelodysplastic Syndromes, run by Massachusetts General Hospital at 3 sites in United States.
Trial summary
This research study is assessing the efficacy of MBG-453, a humanized monoclonal antibody, in treating myelodysplastic syndromes (MDS). The name of the study drug involved in this study is MBG453. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- MBG453
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 3 sitesUnited States
- Study dates
- 27 Jan 2022to 1 Nov 2025
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Lower risk MDS patients (IPSS-R score ≤ 3.5 at diagnosis) who have progressed or are refractory to/intolerant of prior therapy and meet one of the following categories:
- RBC transfusion dependent according to IWG criteria must either be unresponsive to prior ESA therapy or have an EPO level > 500
- Prior HMA therapy
- Patients with the following cytopenias who otherwise are felt to require treatment per the treating physician:
- Platelets < 50k/uL
- ANC < 500 cells/uL
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- Patients with MDS with isolated del(5q) ("5q- syndrome") must have progressed on or not tolerated lenalidomide
- Patients who are not felt to be candidates for or lack other standard treatment options. Patients with prior luspatercept exposure are eligible.
- Patients with dysplastic type CMML (WBC < 13,000 cells/uL) meeting the above criteria are eligible; responses will be assessed using MDS criteria
- Age ≥18 years. Because no dosing or adverse event data are currently available on the use of MGB453 in participants <18 years of age, children are excluded from this study, but will be eligible for future pediatric trials.
- ECOG performance status ≤2 (see Appendix A).
- Participants must have adequate organ and marrow function as defined below within 21 days of treatment:
- Total bilirubin ≤ 2 mg/dL (unless due to Gilbert's in which case it must be <3 mg/dL)
- AST(SGOT)/ALT(SGPT) ≤3 × institutional ULN
- Creatinine clearance ≥30 mL/min/1.73 m2 (by MDRD calculation)
- Human immunodeficiency virus (HIV)-infected participants on effective anti-retroviral therapy with undetectable viral load over the prior 6 months are eligible for this trial.
- Participants with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, participants should be class 2B or better.
- Ability to understand and the willingness to sign a written informed consent document.
Who cannot join
- Participants who have had chemotherapy or radiotherapy within 14 days or five half-lives, whichever is shorter, prior to the first dose of study treatment.
- Participants who are receiving any other investigational agents; a washout of 14 days or 5 half-lives, whichever is longer, is required. The washout period for biologic agents should be 28 days since the last dose.
- Prior exposure to a TIM-3 inhibitor.
- Active autoimmune disease requiring > 10 mg per day of prednisone or the equivalent. Inactive or controlled autoimmune disease is allowed.
- Prior solid organ transplant is exclusionary. Patients with prior hematopoietic cell transplant are eligible if they are over 6 months from transplant and not on any related immunosuppressive therapy.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to MBG453.
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- Active untreated or concurrent malignancy that is distinct in primary site or histology, excluding:
- The following will not be exclusionary: non-melanoma skin cancer, noninvasive colonic polyps, superficial bladder tumors, cervical cancer in-situ, ductal carcinoma in situ of the breast, monoclonal B-cell lymphocytosis, or monoclonal gammopathy of undetermined significance.
- Hormonal therapy is allowed.
- History of other malignancy is allowed if not requiring active management.
- Other malignancies that were treated with curative intent at least 1 year prior to study screening and without evidence of active disease will be allowed.
- Participants with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial pending discussion with the principal investigator.
- Participants with uncontrolled intercurrent illness.
- Participants must not have clinically active HBV or HCV; testing is not required
- Receipt of a live vaccination within 28 days of cycle 1 day 1
- Participants with psychiatric illness/social situations that would limit compliance with study requirements.
- Female contraception is required. Pregnant women are excluded from this study because MBG453 is an agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with MBG453, breastfeeding should be discontinued. Women of child-bearing potential should use highly effective methods of contraception during treatment and for 150 days after the last dose of MBG453.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114
Brigham and Women's Hospital
Boston, Massachusetts, 02215
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
About this study
Official title: TIM3 Inhibition With MBG453 for Patients With Lower Risk MDS: an Adaptive Two-Stage Phase II Clinical Trial
This research study is assessing the efficacy of MBG-453, a humanized monoclonal antibody, in treating myelodysplastic syndromes (MDS).
The name of the study drug involved in this study is MBG453.
Read the detailed description
This is an adaptive two-stage phase II clinical trial to assess the activity of the anti-TIM-3, (T cell immunoglobulin domain and mucin domain) antibody, MBG453, in patients with lower-risk myelodysplastic syndromes (MDS), not eligible for or progressing on frontline therapy.
The U.S. Food and Drug Administration (FDA) has not approved MBG453 for myelodysplastic syndromes (MDS), but it has been approved for other uses.
The study drug (MBG453) may interact with TIM-3 (an antibody which is a protein that attaches to foreign infectious/invading cells and signals the immune system) which might aid the immune system's response by helping immune cells recognize, find, and destroy cancer cells in the body.
The research study procedures include screening for eligibility and study treatment, including evaluations and follow up visits.
Participants will receive study treatment for as long as they and their doctor believe they are benefiting from the study drug. Participants will then be followed for 12 months after their last dose of the study drug or until they withdraw their consent to be contacted.
It is expected that about 20 people will take part in this research study
- Conditions
- Myelodysplastic Syndromes
- Being tested
- MBG453
- Keywords
- Myelodysplastic Syndromes
- 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
intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| MG MBG453 | Experimental | Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle Receives: Drug: MBG453 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
Assessed on the proposal for the modification of the International Working Group (IWG) response criteria in myelodysplasia (Cheson et al., 2006), but modified to include complete remission with partial hematologic improvement CRh (Bloomfield et al., 2018), and the 2018 proposed update for hematologic responses and transfusion independence (TI) (Platzbecker et al., 2019). Patients with dysplastic-type CMML will use MDS risk-stratification and response assessment criteria.
Measured: 6 months
Secondary outcomes (5)
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE Version 5.0
The number of participants with adverse events, graded as defined by CTCAE version 5.0 will be tabulated by type and grade.
Measured: during the intervention, an average of 1 year
Overall Survival (OS) 1-year
1 year overall survival rate estimated using the Kaplan and Meier method.
Measured: 1 year
Progression Free Survival (PFS)
1 year PFS estimated using the Kaplan and Meier method.
Measured: through study completion, an average of 1 year
Time to Disease Progression
Median time from treatment start until disease progression or death, estimated using the Kaplan and Meier method.
Measured: through study completion, an average of 1 year
Duration of Response
Estimated using the Kaplan and Meier method.
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2021
- Study start27 Jan 2022
- Primary completion12 Nov 2024
- Study completion1 Nov 2025
Why it stopped: The trial closed early due to changes in support for the study drug.
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.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
- Responsible party
- Principal investigator, Andrew Mark Brunner, MD, Massachusetts General Hospital
- Investigators
- Andrew Brunner, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04823624
- Sponsor's protocol ID
- 20-637
- Status last verified
- Sep 2026
- First submitted
- 9 Mar 2021
- Last updated
- 20 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- TIM3 Inhibition With MBG453 for Patients With Lower Risk MDS: an Adaptive Two-Stage Phase II Clinical Trial
- ClinicalTrials.gov ID
- NCT04823624
- Acronym / sponsor ID
- 20-637
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Myelodysplastic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The trial closed early due to changes in support for the study drug.
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- 27 Jan 2022 (actual)
- Primary completion
- 12 Nov 2024 (actual)
- Study completion
- 1 Nov 2025 (actual)
- First posted
- 1 Apr 2021 (actual)
- Results
- Posted 27 Feb 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is assessing the efficacy of MBG-453, a humanized monoclonal antibody, in treating myelodysplastic syndromes (MDS).
The name of the study drug involved in this study is MBG453.
Detailed description
This is an adaptive two-stage phase II clinical trial to assess the activity of the anti-TIM-3, (T cell immunoglobulin domain and mucin domain) antibody, MBG453, in patients with lower-risk myelodysplastic syndromes (MDS), not eligible for or progressing on frontline therapy.
The U.S. Food and Drug Administration (FDA) has not approved MBG453 for myelodysplastic syndromes (MDS), but it has been approved for other uses.
The study drug (MBG453) may interact with TIM-3 (an antibody which is a protein that attaches to foreign infectious/invading cells and signals the immune system) which might aid the immune system's response by helping immune cells recognize, find, and destroy cancer cells in the body.
The research study procedures include screening for eligibility and study treatment, including evaluations and follow up visits.
Participants will receive study treatment for as long as they and their doctor believe they are benefiting from the study drug. Participants will then be followed for 12 months after their last dose of the study drug or until they withdraw their consent to be contacted.
It is expected that about 20 people will take part in this research study
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MG MBG453 | Experimental | Drug: MBG453Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Lower risk MDS patients (IPSS-R score ≤ 3.5 at diagnosis) who have progressed or are refractory to/intolerant of prior therapy and meet one of the following categories:
- Age ≥18 years. Because no dosing or adverse event data are currently available on the use of MGB453 in participants <18 years of age, children are excluded from this study, but will be eligible for future pediatric trials.
- ECOG performance status ≤2 (see Appendix A).
- Participants must have adequate organ and marrow function as defined below within 21 days of treatment:
- Human immunodeficiency virus (HIV)-infected participants on effective anti-retroviral therapy with undetectable viral load over the prior 6 months are eligible for this trial.
Key exclusion criteria (17 listed)
- Participants who have had chemotherapy or radiotherapy within 14 days or five half-lives, whichever is shorter, prior to the first dose of study treatment.
- Participants who are receiving any other investigational agents; a washout of 14 days or 5 half-lives, whichever is longer, is required. The washout period for biologic agents should be 28 days since the last dose.
- Prior exposure to a TIM-3 inhibitor.
- Active autoimmune disease requiring > 10 mg per day of prednisone or the equivalent. Inactive or controlled autoimmune disease is allowed.
- Prior solid organ transplant is exclusionary. Patients with prior hematopoietic cell transplant are eligible if they are over 6 months from transplant and not on any related immunosuppressive therapy.
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 |
|---|---|---|
| MBG453intravenous infusion | Drug | MG MBG453 |
Primary outcome
Overall Response Rate (ORR)
Time frame: 6 months
Assessed on the proposal for the modification of the International Working Group (IWG) response criteria in myelodysplasia (Cheson et al., 2006), but modified to include complete remission with partial hematologic improvement CRh (Bloomfield et al., 2018), and the 2018 proposed update for hematologic responses and transfusion independence (TI) (Platzbecker et al., 2019). Patients with dysplastic-type CMML will use MDS risk-stratification and response assessment criteria.
5 secondary outcomes
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE Version 5.0
Time frame: during the intervention, an average of 1 year
Overall Survival (OS) 1-year
Time frame: 1 year
Progression Free Survival (PFS)
Time frame: through study completion, an average of 1 year
Time to Disease Progression
Time frame: through study completion, an average of 1 year
Duration of Response
Time frame: through study completion, an average of 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: [contact information for Sponsor Investigator or designee]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
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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.
Andrew Brunner, MD principal investigator
Massachusetts General Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MBG453 in people with Myelodysplastic Syndromes, run by Massachusetts General Hospital at 3 sites in United States.
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 18 inclusion and 17 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 20 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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. 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 27 Feb 2026). 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?
Overall Response Rate (ORR) (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 20 Oct 2026.