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.
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
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
About this study
Official title: Single-Arm, Open Label, Interventional Phase II Clinical Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy
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.
- 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 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.
- 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)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
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
Study at a glance
From the registry record, last updated by the sponsor on 6 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single-Arm, Open Label, Interventional Phase II Clinical Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy
- ClinicalTrials.gov ID
- NCT03674411
- Acronym / sponsor ID
- 2018LS051
- Conditions
- Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Biphenotypic/Undifferentiated Leukemia, Chronic Myelogenous Leukemia, Myelodysplasia, Relapsed Large Cell Lymphoma
- Keywords
- AML, ALL, CML, MCL
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 2 Jan 2019 (actual)
- Primary completion
- 23 Jun 2020 (actual)
- Study completion
- 1 Dec 2026 (estimated)
- First posted
- 17 Sep 2018 (actual)
- Results
- Posted 30 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| FLU, CY, TBI + MGTA-456 infusion | Experimental | 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 InfusionAll 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) |
| BU,FLU, MEL + MGTA-456 infusion | Experimental | Drug: Fludarabine (FLU), Drug: Mycophenolate Mofetil (MMF), Drug: Granulocyte Colony-Stimulating Factor (G-CSF), Drug: Busulfan (BU), Drug: Melphalan, Drug: MGTA 456 InfusionAll 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 |
Who may qualify
- Ages
- Up to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (0 listed)
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fludarabine (FLU)25 mg/m2 IV over 1 hour (<10 kg: 0.83 mg/kg IV over 1 hour) | Drug | BU,FLU, MEL + MGTA-456 infusion, FLU, CY, TBI + MGTA-456 infusion |
| Cyclophosphamide (CY)60 mg/kg IV over 2 hours | Drug | FLU, CY, TBI + MGTA-456 infusion |
| Total Body Irradiation (TBI)165 cGy twice daily | Drug | FLU, CY, TBI + MGTA-456 infusion |
| Tacrolimus (Tac)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. | Drug | FLU, CY, TBI + MGTA-456 infusion |
| Mycophenolate Mofetil (MMF)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. | Drug | BU,FLU, MEL + MGTA-456 infusion, FLU, CY, TBI + MGTA-456 infusion |
| Granulocyte Colony-Stimulating Factor (G-CSF)5 ug/kg/d until the absolute neutrophil count (ANC) is >2500/uL for 2 consecutive days | Drug | BU,FLU, MEL + MGTA-456 infusion, FLU, CY, TBI + MGTA-456 infusion |
| Busulfan (BU)BU IV once daily with dose based on Pharmacokinetics (PK) calculator over 3 hours | Drug | BU,FLU, MEL + MGTA-456 infusion |
| Melphalan50 mg/m2/day (1.7 mg/kg/day if < 10 kg) IV over 30 min | Drug | BU,FLU, MEL + MGTA-456 infusion |
| MGTA 456 InfusionThe 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. | Drug | BU,FLU, MEL + MGTA-456 infusion, FLU, CY, TBI + MGTA-456 infusion |
Primary outcome
Percentage of Participants With Neutrophil Recover
Time frame: Day 14
Percentage of participants with neutrophil recovery by day 14 after transplantation in recipients of MGTA-456.
11 secondary outcomes
Number of Days Alive Without Hospitalization
Time frame: Day 0 and Day 100
Secondary Graft Failure
Time frame: 2 Years
Platelet Recovery
Time frame: Day 42
Treatment Related Mortality (TRM)
Time frame: 6 Months
Grades II-IV Acute GVHD
Time frame: Day 100
Grades III-IV Acute GVHD
Time frame: Day 100
Chronic GVHD
Time frame: 1 Year
Relapse
Time frame: 2 Years
Non-catheter Associated Bacterial Infections
Time frame: Day 100
Overall Survival (OS)
Time frame: 2 Years
Event-Free Survival (EFS)
Time frame: 2 Years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jan 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 6 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Jan 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With Neutrophil Recover (time frame: Day 14). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jan 2026.