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

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.

Conditions

• Acute Myeloid Leukemia
• Acute Lymphocytic Leukemia
• Biphenotypic/Undifferentiated Leukemia
• Chronic Myelogenous Leukemia
• Myelodysplasia
• Relapsed Large Cell Lymphoma
• Mantle Cell Lymphoma
• Hodgkin Lymphoma
• Burkitt Lymphoma
• Relapsed T-Cell Lymphoma
• Lymphoplasmacytic Lymphoma

Interventions

DRUG

Fludarabine (FLU)

25 mg/m2 IV over 1 hour (\<10 kg: 0.83 mg/kg IV over 1 hour)

DRUG

Cyclophosphamide (CY)

60 mg/kg IV over 2 hours

DRUG

Total Body Irradiation (TBI)

165 cGy twice daily

DRUG

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

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

Granulocyte Colony-Stimulating Factor (G-CSF)

5 ug/kg/d until the absolute neutrophil count (ANC) is \>2500/uL for 2 consecutive days

DRUG

Busulfan (BU)

BU IV once daily with dose based on Pharmacokinetics (PK) calculator over 3 hours

DRUG

Melphalan

50 mg/m2/day (1.7 mg/kg/day if \< 10 kg) IV over 30 min

DRUG

MGTA 456 Infusion

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.

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota

  lead OTHER

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Max Age

55 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2019-01-02

Primary Completion

2020-06-23

Completion

2026-12-01

FDA Drug

Yes

Countries

• United States

Study Locations