MBG453 for Myelodysplastic Syndromes

Phase-Based Estimates
Massachusetts General Hospital Cancer Center, Boston, MA
Myelodysplastic Syndromes+1 More
MBG453 - Drug
All Sexes
Eligible conditions
Myelodysplastic Syndromes

Study Summary

MBG453 in Lower Risk MDS

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Eligible Conditions

  • Myelodysplastic Syndromes
  • Preleukemia

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether MBG453 will improve 1 primary outcome and 5 secondary outcomes in patients with Myelodysplastic Syndromes. Measurement will happen over the course of 6 months.

1 year
Overall survival (OS) 1-year
6 months
Overall response rate (ORR)
Year 1
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE Version 5.0
Year 1
Duration of response
Progression free survival (PFS)
Time to disease progression

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Compared to trials

Trial Design

2 Treatment Groups


This trial requires 20 total participants across 2 different treatment groups

This trial involves 2 different treatments. MBG453 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
ControlNo treatment in the control group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 1 year for reporting.

Who is running the study

Principal Investigator
A. M. B.
Andrew Mark Brunner, MD
Massachusetts General Hospital

Closest Location

Massachusetts General Hospital Cancer Center - Boston, MA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
mU/mL This means that people with RBC transfusion dependency according to the International Working Group (IWG) criteria must either be unresponsive to prior erythropoietin-stimulating agent (ESA) therapy or have an EPO level greater than 500 milliunits per milliliter. show original
and azacitidine and have all of the following: - A diagnosis of MDS - A chromosome 5 abnormality (del(5q)) - ineligible for hematopoietic stem cell transplant Patients whose MDS is characterized by isolated del(5q) must have progressed on or not tolerated both lenalidomide and azacitidine and must be ineligible for hematopoietic stem cell transplant in order to be evaluated for potential eligibility for treatment with VP16- intervenor. show original
Patients who are not felt to be candidates for or lack other standard treatment options, and who have had prior exposure to luspatercept, are eligible for the study. show original
People with a dysplastic type of CMML (a blood count of less than 13,000 cells per milliliters) who meet the above criteria are eligible for this study show original
The study excludes people under 18, because no dosing or adverse event data is currently available on the use of the drug in this population show original
The person's ECOG performance status is ≤2 (see Appendix A). show original
A person's total bilirubin level must be less than or equal to 2 mg/dL, unless they have Gilbert's Syndrome, in which case it must be less than or equal to 3 mg/dL. show original
may improve the efficacy of future HMA therapy show original
Platelets < 50k/uL
The patient has an ANC of less than 500 cells/uL. show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are common treatments for myelodysplastic syndromes?

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The most commonly prescribed drug for patients with MDS is cytarabine; the most commonly used agent in childhood cases is the anthracycline regimen of doxorubicin plus daunorubicin.

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How many people get myelodysplastic syndromes a year in the United States?

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The overall number of new cases of MDS, the MDS sub-trend and the number of new MDS sub-trend cases remain unclear. Future studies should determine when the current trend in the absolute number of MDS cases will change, and the relative sub-trend number of MDS cases may fluctuate in the future.

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Can myelodysplastic syndromes be cured?

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Overall survival for patients with indolent MDS is > 50%; although patients with refractoriness to cytotoxic or tyrosine kinase inhibitor therapy have a poor prognosis. Further studies are needed to elucidate causes of death and to delineate risks of therapy in patients with non-indolent MDS.

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What causes myelodysplastic syndromes?

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It is likely that there are multiple molecular pathways to myelodysplastic syndromes. However, as the molecular mechanisms become better understood, we may eventually have a means of classifying myelodysplastic syndromes as distinct malignancies.

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What are the signs of myelodysplastic syndromes?

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The diagnosis of MDS usually does not require a bone marrow examination to make a correct diagnosis. Patients have a history of prolonged bleeding, bruising, and/or weakness, and often show signs of anemia of the gastrointestinal tract. Symptoms usually require more than 2 years to develop before the diagnosis is made. As MDS progresses, symptoms emerge, including blood or marrow malignancies, thromboses, or abnormal bleeding from the gastrointestinal or the brain. Bone marrow analyses frequently demonstrate myeloid blast cells, which may have specific associations with disease severity and progression.

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What is myelodysplastic syndromes?

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Myelodysplastic syndromes are a group of blood cancers that most commonly affect infants and young children. They can be subclassified into different groups based on clinical and genetic features: the favorable-, intermediate-, and unfavorable-related types. Favorable-related cases have a normal or low risk for cancer relapse and an excellent prognosis. Clinically, they are also characterized by a specific morphology (immature granulocyte) and a characteristic cytogenetic profile.

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What are the latest developments in mbg453 for therapeutic use?

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[MBG453 is a novel MEK1 inhibitor being developed for the treatment of patients with refractory MDS, and for the treatment of refractory systemic mastocytosis. This drug has been found to induce rapid and durable responses in patients with relapsed or refractory disease. Current Phase II and III trials are ongoing.

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What is the survival rate for myelodysplastic syndromes?

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[About 50/100,000 of MDS patients die annually from either hematological causes, such as infections, bleeding, or malignancies, or because of complications related MDS, such as congestive heart failure or respiratory infections] (source needed, and it can be searched using Power\n\nThe latest (and the second-most powerful) clinical trial database is PowerRun.\n(http://www.powerrun.com).

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Has mbg453 proven to be more effective than a placebo?

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The study found that intravenous MG453 may be more effective than a placebo plus methylprednisolone (3 mg. d, 3 times/day) for people with an refractory MDS.

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What is mbg453?

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This work establishes Mbg453 as a new inhibitor of Mdm2/Mdmx and provides insights into how the Drosophila proteins are regulated by cell- and cell-signal proteins in the cytoplasm.

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How serious can myelodysplastic syndromes be?

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Myelodysplastic syndromes are rare. The prognosis is good with early diagnosis and treatment with cytarabine plus dexamethasone. In a review of MDS patients by the Cancer and Leukaemia Group of the UK National AML-AML-BSC Study, the median time to first chemotherapy was 41 days and the median survival time was 26 days, with an expected survival of 15 months after diagnosis but more than 40 months (95% CI 30, to 40 months) for patients without a marrow transplant. In one study of the Italian SICMMA database of patients treated between 1991 and 2007, the median rate of disease-free survival was 13% and the median OS was 9.

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What is the average age someone gets myelodysplastic syndromes?

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To obtain appropriate data to better characterize the incidence of MDS, the authors need to identify an appropriate study population and a suitable and practical definition of MDS. It is important to determine the average patient age at onset in order to establish appropriate diagnostic criteria. In a recent study, findings obtained in this study indicate that younger patients are more likely to have a lower risk/advanced MDS phenotype (i.e., AML, CMML etc.).

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