Trial Outcomes & Findings for MBG453 in Lower Risk MDS (NCT NCT04823624)

Last Updated: 2026-02-27

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

6 months

Results posted on

2026-02-27

Participant Flow

10 patients were enrolled

A total of 10 patients were enrolled.

Participant milestones

Participant milestones
Measure	MG MBG453 Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

study ended early

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	MG MBG453 n=10 Participants Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
Age, Continuous	72 years n=24 Participants
Sex: Female, Male Female	1 Participants n=24 Participants • study ended early
Sex: Female, Male Male	9 Participants n=24 Participants • study ended early
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=24 Participants • study ended early
Race (NIH/OMB) Asian	2 Participants n=24 Participants • study ended early
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=24 Participants • study ended early
Race (NIH/OMB) Black or African American	0 Participants n=24 Participants • study ended early
Race (NIH/OMB) White	8 Participants n=24 Participants • study ended early
Race (NIH/OMB) More than one race	0 Participants n=24 Participants • study ended early
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=24 Participants • study ended early
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=24 Participants • study ended early
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=24 Participants • study ended early
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=24 Participants • study ended early
Transfusion Burden High Transfusion Burden	8 Participants n=24 Participants
Transfusion Burden Low Transfusion Burden	2 Participants n=24 Participants

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	MG MBG453 n=10 Participants Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
Overall Response Rate (ORR)	2 Participants

SECONDARY outcome

Timeframe: during the intervention, an average of 1 year

The number of participants with adverse events, graded as defined by CTCAE version 5.0 will be tabulated by type and grade.

Outcome measures

Outcome measures
Measure	MG MBG453 n=10 Participants Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE Version 5.0	2 Participants

SECONDARY outcome

Timeframe: 1 year

1 year overall survival rate estimated using the Kaplan and Meier method.

Outcome measures

Outcome measures
Measure	MG MBG453 n=10 Participants Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
Overall Survival (OS) 1-year	64 percentage Interval 39.0 to 100.0

SECONDARY outcome

Timeframe: through study completion, an average of 1 year

1 year PFS estimated using the Kaplan and Meier method.

Outcome measures

Outcome measures
Measure	MG MBG453 n=10 Participants Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
Progression Free Survival (PFS)	47 percentage Interval 23.0 to 94.0

SECONDARY outcome

Timeframe: through study completion, an average of 1 year

Median time from treatment start until disease progression or death, estimated using the Kaplan and Meier method.

Outcome measures

Outcome measures
Measure	MG MBG453 n=10 Participants Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
Time to Disease Progression	5.9 months Interval 4.8 to not reached

SECONDARY outcome

Timeframe: through study completion, an average of 1 year

Estimated using the Kaplan and Meier method.

Outcome measures

Outcome measures
Measure	MG MBG453 n=2 Participants Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
Duration of Response	5.25 months Interval 3.68 to 6.83

Adverse Events

MG MBG453

Serious events: 3 serious events

Other events: 7 other events

Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure	MG MBG453 n=10 participants at risk Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
Blood and lymphatic system disorders neutropenia	20.0% 2/10 • Adverse Events were monitored/assessed from enrollment through end of treatment, an average of 6 months. All-Cause Mortality was monitored/assessed for up to 1 year following end of treatment.
Blood and lymphatic system disorders thrombocytopenia	10.0% 1/10 • Adverse Events were monitored/assessed from enrollment through end of treatment, an average of 6 months. All-Cause Mortality was monitored/assessed for up to 1 year following end of treatment.
Cardiac disorders chest pain - cardiac	10.0% 1/10 • Adverse Events were monitored/assessed from enrollment through end of treatment, an average of 6 months. All-Cause Mortality was monitored/assessed for up to 1 year following end of treatment.

Other adverse events

Other adverse events
Measure	MG MBG453 n=10 participants at risk Participants will be given MBG453 On Day 1 of each cycle 28 days (4 weeks) study cycle
Blood and lymphatic system disorders anemia	20.0% 2/10 • Adverse Events were monitored/assessed from enrollment through end of treatment, an average of 6 months. All-Cause Mortality was monitored/assessed for up to 1 year following end of treatment.
Hepatobiliary disorders alkaline phosphatase increased	10.0% 1/10 • Adverse Events were monitored/assessed from enrollment through end of treatment, an average of 6 months. All-Cause Mortality was monitored/assessed for up to 1 year following end of treatment.
Blood and lymphatic system disorders neutropenia	40.0% 4/10 • Adverse Events were monitored/assessed from enrollment through end of treatment, an average of 6 months. All-Cause Mortality was monitored/assessed for up to 1 year following end of treatment.
Blood and lymphatic system disorders thrombocytopenia	10.0% 1/10 • Adverse Events were monitored/assessed from enrollment through end of treatment, an average of 6 months. All-Cause Mortality was monitored/assessed for up to 1 year following end of treatment.

Additional Information

Andrew Brunner, MD

Massachusetts General Hospital

Phone: 617-724-1124

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place