Trial Outcomes & Findings for Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (NCT NCT03674411)
NCT ID: NCT03674411
Last Updated: 2026-01-06
Results Overview
Percentage of participants with neutrophil recovery by day 14 after transplantation in recipients of MGTA-456.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
22 participants
Primary outcome timeframe
Day 14
Results posted on
2026-01-06
Participant Flow
Participant milestones
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Overall Study
STARTED
|
20
|
2
|
|
Overall Study
COMPLETED
|
16
|
2
|
|
Overall Study
NOT COMPLETED
|
4
|
0
|
Reasons for withdrawal
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Overall Study
Non-treatment
|
4
|
0
|
Baseline Characteristics
Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy
Baseline characteristics by cohort
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
5 Participants
n=37 Participants
|
2 Participants
n=56 Participants
|
7 Participants
n=82 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
11 Participants
n=82 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=82 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=37 Participants
|
1 Participants
n=56 Participants
|
6 Participants
n=82 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=37 Participants
|
1 Participants
n=56 Participants
|
12 Participants
n=82 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
1 Participants
n=82 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=37 Participants
|
2 Participants
n=56 Participants
|
15 Participants
n=82 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
2 Participants
n=82 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
2 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
2 Participants
n=82 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=82 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=82 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
1 Participants
n=82 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
10 Participants
n=82 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=37 Participants
|
2 Participants
n=56 Participants
|
3 Participants
n=82 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
2 Participants
n=82 Participants
|
|
Region of Enrollment
United States
|
16 participants
n=37 Participants
|
2 participants
n=56 Participants
|
18 participants
n=82 Participants
|
PRIMARY outcome
Timeframe: Day 14Percentage of participants with neutrophil recovery by day 14 after transplantation in recipients of MGTA-456.
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Percentage of Participants With Neutrophil Recover
|
31 Percentage of participants
Interval 14.0 to 60.0
|
50 Percentage of participants
Interval 9.0 to 99.0
|
SECONDARY outcome
Timeframe: Day 0 and Day 100Number of days alive without hospitalization between days 0 and 100 after transplantation
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Number of Days Alive Without Hospitalization
|
67.5 Days
Interval 18.0 to 85.0
|
33 Days
Interval 0.0 to 66.0
|
SECONDARY outcome
Timeframe: 2 YearsIncidence of secondary graft failure
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Secondary Graft Failure
|
37.5 Percentage of participants
|
0 Percentage of participants
|
SECONDARY outcome
Timeframe: Day 42Incidence of platelet recovery at day 42
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Platelet Recovery
|
56 Percentage of participants
Interval 33.0 to 80.0
|
50 Percentage of participants
Interval 1.0 to 99.0
|
SECONDARY outcome
Timeframe: 6 MonthsIncidence of TRM at 6 months
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Treatment Related Mortality (TRM)
|
6 Percentage of participants
Interval 0.0 to 18.0
|
50 Percentage of participants
Interval 15.0 to 85.0
|
SECONDARY outcome
Timeframe: Day 100Incidence of grades II-IV acute GVHD at day 100
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Grades II-IV Acute GVHD
|
25 Percentage of participants
Interval 4.0 to 46.0
|
0 Percentage of participants
Interval 0.0 to 100.0
|
SECONDARY outcome
Timeframe: Day 100Incidence of grades III-IV acute GVHD at day 100
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Grades III-IV Acute GVHD
|
19 Percentage of participants
Interval 0.0 to 37.0
|
0 Percentage of participants
Interval 0.0 to 100.0
|
SECONDARY outcome
Timeframe: 1 YearIncidence of chronic GVHD at 1 year
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Chronic GVHD
|
13 Percentage of participants
Interval 0.0 to 28.0
|
0 Percentage of participants
Interval 0.0 to 100.0
|
SECONDARY outcome
Timeframe: 2 YearsIncidence of relapse at 2 years
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Relapse
|
25 Percentage of participants
Interval 4.0 to 46.0
|
50 Percentage of participants
Interval 1.0 to 99.0
|
SECONDARY outcome
Timeframe: Day 100Incidence of non-catheter associated bacterial infections by day 100
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Non-catheter Associated Bacterial Infections
|
6 Percentage of participants
|
0 Percentage of participants
|
SECONDARY outcome
Timeframe: 2 YearsIncidence of overall survival (OS) at 2 years
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Overall Survival (OS)
|
69 Percentage of participants
|
0 Percentage of participants
|
SECONDARY outcome
Timeframe: 2 YearsIncidence of event-free survival (EFS) at 2 years
Outcome measures
| Measure |
FLU, CY, TBI + MGTA-456 Infusion
n=16 Participants
All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF)
|
BU,FLU, MEL + MGTA-456 Infusion
n=2 Participants
All young children \<3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF
|
|---|---|---|
|
Event-Free Survival (EFS)
|
69 Percentage of participants
|
0 Percentage of participants
|
Adverse Events
FLU, CY, TBI + MGTA-456 Infusion
Serious events: 0 serious events
Other events: 0 other events
Deaths: 5 deaths
BU,FLU, MEL + MGTA-456 Infusion
Serious events: 0 serious events
Other events: 0 other events
Deaths: 2 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Dr. Margaret MacMillan
University of Minnesota, Masonic Cancer Center
Phone: (612) 626-5654
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place