NCT03674411 · REGISTRY CONDITION SEARCH

Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase: PHASE2
Status at capture: ACTIVE NOT RECRUITING
Registry last update: 2026-01-06

This is an single arm, open label, interventional phase II trial evaluating the efficacy of umbilical cord blood (UCB) hematopoietic stem and progenitor cells (HSPC) expanded in culture with stimulatory cytokines (SCF, Flt-3L, IL-6 and thromopoietin) on lympho-hematopoietic recovery. Patients will receive a uniform myeloablative conditioning and post-transplant immunoprophylaxis.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Percentage of Participants With Neutrophil Recover

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outcome · POSTED

Number of Days Alive Without Hospitalization

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outcome · POSTED

Secondary Graft Failure

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outcome · POSTED

Platelet Recovery

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outcome · POSTED

Treatment Related Mortality (TRM)

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outcome · POSTED

Grades II-IV Acute GVHD

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outcome · POSTED

Grades III-IV Acute GVHD

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outcome · POSTED

Chronic GVHD

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outcome · POSTED

Relapse

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outcome · POSTED

Non-catheter Associated Bacterial Infections

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outcome · POSTED

Overall Survival (OS)

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outcome · POSTED

Event-Free Survival (EFS)

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part

eligibility Criteria: Age, Unit Cell Dose and HLA Match Criteria * Subjects must be ≤55 years of age * Subjects must weigh \>11 kg * Subjects must have a partially HLA matched UCB unit with a pre-cryopreserved TNC dose \>1.0 x 107 per kilogram recipient weight. HLA matching is initially based on a minimum of 5 of 8 HLA alleles at high resolution A, B, C, DRB1 typing; searches will be performed according to the current Magenta Cord Blood Search Algorithm. Eligible Diseases: * Acute myelogenous leukemia (AML) in morphological complete remission with: * Minimal residual disease (MRD) by flow cytometry, or * Intermediate to high risk leukemia in first (CR1) based on institutional criteria, eg. not favorable risk AML which is defined as having one of the following: * t(8,21) without cKIT mutation * inv(16) or t(16;16) without cKIT mutation * Normal karyotype with mutated NPM1 but FLT3-ITD wild type * Normal karyotype with double mutated CEBPA * Acute promyelocytic leukemia (APL) in first molecular remission at the end of consolidation * Any second or subsequent CR, or * Secondary AML with prior malignancy that has been in remission for at least 12 months. * Acute lymphocytic leukemia (ALL) at the following stages: * High risk first morphological, cytogenetic and molecular CR with: * MRD by flow cytometry, or * Diagnosis of Philadelphia chromosome (Ph)+ ALL, or * MLL rearrangement at diagnosis with slow early response at Day 14, or * Hypodiploidy (\< 44 chromosomes or DNA index \< 0.81) at diagnosis, or * End of induction M3 bone marrow, or * End of induction M2 with M2-3 at Day 42. * High risk second CR based on institutional criteria (eg, for children, bone marrow relapse \<36 months from induction or T-lineage bone marrow relapse or very early isolated central nervous system (CNS) relapse \<6 months from diagnosis, or slow re-induction (stage M2-3 at day 28 after induction) regardless of length remission. All patients with MRD by flow cytometry. * Any third or subsequent CR. * Secondary ALL * Biphenotypic/undifferentiated leukemia in morphological, cytogenetic and molecular CR . * Chronic Myelogenous Leukemia (CML) in high risk first chronic phase (failure of two tyrosine kinase inhibitors (TKI) or TKI intolerance), accelerated phase or second chronic phase. * Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt \<5% blasts) or other high risk features, including multiple cytopenias, high risk cytogenetics or lack of response to standard therapy.. * Relapsed large-cell lymphoma, mantle-cell lymphoma and Hodgkin lymphoma that is chemotherapy sensitive and ineligible for an autologous transplant. * Burkitt's lymphoma in CR2 or subsequent CR. * Relapsed T-cell lymphoma that is chemotherapy sensitive in CR/PR that is ineligible for an autologous transplant. Organ Specific Inclusion Criteria * Karnofsky score ≥70 (16 years and older), Lansky play score \>50 (children 2-16 years, or 'adequate' score for children \<2 years, as detailed in Appendix II. * Adequate organ function defined as: * Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then creatinine clearance \>40 ml/min or GFR ≥70 mL/min/1.73 m2.normal for age * Hepatic: Bilirubin \<3x upper limit of normal (ULN) and AST, ALT and alkaline phosphatase \<5x ULN. * Pulmonary function: DLCO, FEV1, FEC (diffusion capacity) \>5030% of predicted (corrected for hemoglobin); if unable to perform pulmonary function tests, then O2 saturation \>95% on room air. * Cardiac: No uncontrolled arrhythmia and left ventricular ejection fraction at rest must be \>3545%. * Available 'back-up' HSPC graft (e.g, second UCB unit, haploidentical related donor). * Females of child bearing potential and sexually active males must agree to use adequate birth control during study treatment. * Voluntary written consent signed (adult or parental) before performance of any study-related procedure not part of normal medical care. Exclusion Criteria * Patients with a HLA matched sibling donor or a HLA matched unrelated donor who is available for marrow or peripheral blood stem cell collection at the desired time of transplant. * Pregnant or breast feeding. The agents used in this study may be teratogenic to a fetus and there is no information on the excretion of agents into breast milk. Females of childbearing potential must have a blood test or urine study within 14 days prior to study enrollment to rule out pregnancy. * Evidence of human immunodeficiency virus (HIV) infection or known HIV positive serology. * Active bacterial, viral or fungal infection (currently taking medication and persistence of clinical signs and symptoms) with a minimum of 4 weeks of anti-fungal treatment * Prior autologous or allogeneic transplant. * Other active malignancy. * Subjects \>2 3 years of age unable to receive TBI 1320 cGy due to extensive prior therapy including \>12 months alkylator therapy or \>6 months alkylator therapy with extensive radiation, or prior Y-90 ibritumomab (Zevalin) or I-131 tostumomab (Bexxar), as part of their salvage therapy.
healthy Volunteers: false
maximum Age: 55 Years
sex: ALL
std Ages: CHILD
ADULT

Treatment arms and interventions

arm Groups

description
All patients will receive MGTA-456 on the day of transplantation after myeloablative conditioning. 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)
intervention Names
Drug: Fludarabine (FLU)
Drug: Cyclophosphamide (CY)
Drug: Total Body Irradiation (TBI)
Drug: Tacrolimus (Tac)
Drug: Mycophenolate Mofetil (MMF)
Drug: Granulocyte Colony-Stimulating Factor (G-CSF)
Drug: MGTA 456 Infusion
label
FLU, CY, TBI + MGTA-456 infusion
type
EXPERIMENTAL
description
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
intervention Names
Drug: Fludarabine (FLU)
Drug: Mycophenolate Mofetil (MMF)
Drug: Granulocyte Colony-Stimulating Factor (G-CSF)
Drug: Busulfan (BU)
Drug: Melphalan
Drug: MGTA 456 Infusion
label
BU,FLU, MEL + MGTA-456 infusion
type
EXPERIMENTAL

interventions

arm Group Labels
BU,FLU, MEL + MGTA-456 infusion
FLU, CY, TBI + MGTA-456 infusion
description
25 mg/m2 IV over 1 hour (\<10 kg: 0.83 mg/kg IV over 1 hour)
name
Fludarabine (FLU)
type
DRUG
arm Group Labels
FLU, CY, TBI + MGTA-456 infusion
description
60 mg/kg IV over 2 hours
name
Cyclophosphamide (CY)
type
DRUG
arm Group Labels
FLU, CY, TBI + MGTA-456 infusion
description
165 cGy twice daily
name
Total Body Irradiation (TBI)
type
DRUG
arm Group Labels
FLU, CY, TBI + MGTA-456 infusion
description
Tacrolimus will start day -3 and will be administered as a continuous IV infusion at a starting dose of 0.03 mg/kg/day. Goal trough levels will be 10-15 ng/mL for the first 14 days post-transplant and then decreased to a goal of 5-10 ng/ml thereafter.
name
Tacrolimus (Tac)
type
DRUG
arm Group Labels
BU,FLU, MEL + MGTA-456 infusion
FLU, CY, TBI + MGTA-456 infusion
description
MMF 3 gram/day IV/PO for adult patients divided in 2 or 3 doses. Pediatric patients will receive MMF at the dose of 15 mg/kg/dose (max 1 gram per dose) every 8 hours beginning day -3.
name
Mycophenolate Mofetil (MMF)
type
DRUG
arm Group Labels
BU,FLU, MEL + MGTA-456 infusion
FLU, CY, TBI + MGTA-456 infusion
description
5 ug/kg/d until the absolute neutrophil count (ANC) is \>2500/uL for 2 consecutive days
name
Granulocyte Colony-Stimulating Factor (G-CSF)
type
DRUG
arm Group Labels
BU,FLU, MEL + MGTA-456 infusion
description
BU IV once daily with dose based on Pharmacokinetics (PK) calculator over 3 hours
name
Busulfan (BU)
type
DRUG
arm Group Labels
BU,FLU, MEL + MGTA-456 infusion
description
50 mg/m2/day (1.7 mg/kg/day if \< 10 kg) IV over 30 min
name
Melphalan
type
DRUG
arm Group Labels
BU,FLU, MEL + MGTA-456 infusion
FLU, CY, TBI + MGTA-456 infusion
description
The target cell dose is \>10 x 106 CD34/kg with a maximum TNC 2.7 x 108/kg for children (\<18 years) and 8.1 × 108 cells/kg \[expanded product only\] for adults based on the highest cell dose windows evaluated in prior studies.
name
MGTA 456 Infusion
type
DRUG

Study design

allocation

NON_RANDOMIZED

intervention Model

SINGLE_GROUP

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 22
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
Percentage of participants with neutrophil recovery by day 14 after transplantation in recipients of MGTA-456.
measure
Percentage of Participants With Neutrophil Recover
time Frame
Day 14

secondary Outcomes

description
Number of days alive without hospitalization between days 0 and 100 after transplantation
measure
Number of Days Alive Without Hospitalization
time Frame
Day 0 and Day 100
description
Incidence of secondary graft failure
measure
Secondary Graft Failure
time Frame
2 Years
description
Incidence of platelet recovery at day 42
measure
Platelet Recovery
time Frame
Day 42
description
Incidence of TRM at 6 months
measure
Treatment Related Mortality (TRM)
time Frame
6 Months
description
Incidence of grades II-IV acute GVHD at day 100
measure
Grades II-IV Acute GVHD
time Frame
Day 100
description
Incidence of grades III-IV acute GVHD at day 100
measure
Grades III-IV Acute GVHD
time Frame
Day 100
description
Incidence of chronic GVHD at 1 year
measure
Chronic GVHD
time Frame
1 Year
description
Incidence of relapse at 2 years
measure
Relapse
time Frame
2 Years
description
Incidence of non-catheter associated bacterial infections by day 100
measure
Non-catheter Associated Bacterial Infections
time Frame
Day 100
description
Incidence of overall survival (OS) at 2 years
measure
Overall Survival (OS)
time Frame
2 Years
description
Incidence of event-free survival (EFS) at 2 years
measure
Event-Free Survival (EFS)
time Frame
2 Years

Full study description

brief Summary: This is an single arm, open label, interventional phase II trial evaluating the efficacy of umbilical cord blood (UCB) hematopoietic stem and progenitor cells (HSPC) expanded in culture with stimulatory cytokines (SCF, Flt-3L, IL-6 and thromopoietin) on lympho-hematopoietic recovery. Patients will receive a uniform myeloablative conditioning and post-transplant immunoprophylaxis.

Source references

Source notices and limitations

Discovery and provenance

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Preserved source evidence · Not medical advice