NCT02960646

Engineered Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Phase I Clinical Trial Using an Engineered Peripheral Blood Graft for Haploidentical Transplantation

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This pilot phase I trial studies the side effects of engineered donor stem cell transplant in treating patients with hematologic malignancies. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft-versus-host disease). Using T cells specially selected from donor blood in the laboratory for transplant may stop this from happening.

Detailed description

PRIMARY OBJECTIVE: I. To assess the safety of a modified peripheral blood (PB) graft for haploidentical transplantation, obtained by using depletion of naive, cluster of differentiation (CD)45RA+ T cells. SECONDARY OBJECTIVES: I. To estimate the proportion of patients with engraftment/graft failure. II. To determine the day 100 and 6 month non-relapse mortality (NRM). III. To estimate the cumulative incidence of grade 2-4 and 3-4 acute graft versus (vs.) host disease (aGVHD). IV. To assess the rate of chronic GVHD within the first year post transplantation. V. To assess immune reconstitution and the incidence of infectious episodes. VI. To assess disease response, disease-free survival (DFS) and overall survival (OS) after transplantation. VII. To compare results with a retrospective cohort of patients treated with bone marrow graft on protocol 2009-0266. OUTLINE: Patients receive melphalan intravenously (IV) over 30 minutes on day -6 and fludarabine phosphate IV over 1 hour on days -6 to -3. Patients undergo total-body irradiation (TBI) on day -2 and CD45RA depleted peripheral blood stem cell transplantation on day 0. Patients also receive cyclophosphamide IV over 3 hours on days 3-4. Beginning on day 5, patients receive tacrolimus IV for 2 weeks and orally (PO) for at least 4 months. Beginning on day 7, patients receive filgrastim subcutaneously (SC) daily. Patients with CD20 positive lymphoma may receive rituximab IV on days -13, -6, 1, and 8. After completion of study treatment, patients are followed up periodically.

Conditions

• Acute Lymphoblastic Leukemia
• Acute Myeloid Leukemia
• Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
• Aplastic Anemia
• Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
• Chronic Myelomonocytic Leukemia
• Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
• Lymphoblastic Lymphoma
• Myelodysplastic Syndrome
• Myeloproliferative Neoplasm
• Plasma Cell Myeloma
• Recurrent Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
• Recurrent Hodgkin Lymphoma
• Recurrent Non-Hodgkin Lymphoma
• Recurrent Plasma Cell Myeloma
• Therapy-Related Myelodysplastic Syndrome

Interventions

Type	Name	Description
DRUG	Cyclophosphamide	Given IV
BIOLOGICAL	Filgrastim	Given SC
DRUG	Fludarabine Phosphate	Given IV
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Melphalan	Given IV
PROCEDURE	Peripheral Blood Stem Cell Transplantation	Undergo CD45RA depleted peripheral blood stem cell transplantation
BIOLOGICAL	Rituximab	Given IV
DRUG	Tacrolimus	Given IV or PO
RADIATION	Total-Body Irradiation	Undergo TBI

Timeline

• Start date: 2017-01-18
• Primary completion: 2023-02-13
• Completion: 2023-02-13
• First posted: 2016-11-09
• Last updated: 2023-02-16

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study