Acute Myeloid LeukemiaPhase 2NCT03674411

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

A Phase 2 study testing Fludarabine (FLU), Cyclophosphamide (CY) and Total Body Irradiation (TBI) in people with Acute Myeloid Leukemia and Acute Lymphocytic Leukemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages Up to 55 years
Conditions the study is for: Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Biphenotypic/Undifferentiated Leukemia
What is being tested?: Fludarabine (FLU), Cyclophosphamide (CY) and Total Body Irradiation (TBI)
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 22 participants
Where: 1 siteUnited States
Study dates: 2 Jan 2019to 1 Dec 2026
Phase: Phase 2

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 Up to 55 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

None listed.

Who cannot join

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.

Trial locations and contacts

1 site in 1 country.

United States1 site

Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, 55455

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About this study

Official title: Single-Arm, Open Label, Interventional Phase II Clinical Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy

Conditions: Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Biphenotypic/Undifferentiated Leukemia, Chronic Myelogenous Leukemia, Myelodysplasia, Relapsed Large Cell Lymphoma
Being tested: Fludarabine (FLU), Cyclophosphamide (CY), Total Body Irradiation (TBI), Tacrolimus (Tac), Mycophenolate Mofetil (MMF)
Keywords: AML, ALL, CML, MCL
Medical subject headings: Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Anemia, Refractory, with Excess of Blasts, Dendritic Cell Sarcoma, Interdigitating, Lymphoma, Mantle-Cell, Hodgkin Disease, Burkitt Lymphoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Fludarabine (FLU)Drug
25 mg/m2 IV over 1 hour (<10 kg: 0.83 mg/kg IV over 1 hour)
Cyclophosphamide (CY)Drug
60 mg/kg IV over 2 hours
Total Body Irradiation (TBI)Drug
165 cGy twice daily
Tacrolimus (Tac)Drug
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.
Mycophenolate Mofetil (MMF)Drug
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.
Granulocyte Colony-Stimulating Factor (G-CSF)Drug
5 ug/kg/d until the absolute neutrophil count (ANC) is >2500/uL for 2 consecutive days
Busulfan (BU)Drug
BU IV once daily with dose based on Pharmacokinetics (PK) calculator over 3 hours
MelphalanDrug
50 mg/m2/day (1.7 mg/kg/day if < 10 kg) IV over 30 min
MGTA 456 InfusionDrug
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.

Study groups in NCT03674411
Study group	Role	What they receive
FLU, CY, TBI + MGTA-456 infusion	Experimental	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) Receives: 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
BU,FLU, MEL + MGTA-456 infusion	Experimental	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 Receives: Drug: Fludarabine (FLU); Drug: Mycophenolate Mofetil (MMF); Drug: Granulocyte Colony-Stimulating Factor (G-CSF); Drug: Busulfan (BU); Drug: Melphalan; Drug: MGTA 456 Infusion

What is being measured

The primary outcome is the main question the study is designed to answer.

Percentage of Participants With Neutrophil Recover
Percentage of participants with neutrophil recovery by day 14 after transplantation in recipients of MGTA-456.
Measured: Day 14

Secondary outcomes (11)

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

2 Jan 20191 Dec 2026

First posted17 Sep 2018
Study start2 Jan 2019
Primary completion23 Jun 2020
Study completion1 Dec 2026 (estimated)

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 Acute Myeloid LeukemiaStudies of the same condition, open and under way.
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: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 22 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03674411
Sponsor's protocol ID: 2018LS051
Other IDs: MT2018-06
Status last verified: Dec 2025
First submitted: 14 Sep 2018
Last updated: 6 Jan 2026
Results posted: Yes, on ClinicalTrials.gov
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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