NCT03399773

Infusion of Expanded Cord Blood Cells in Addition to Single Cord Blood Transplant in Treating Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplastic Syndromes

Pilot Study: Infusion of Off-the-Shelf Ex Vivo Expanded Cryopreserved Progenitor Cells (Dilanubicel) in the Setting of Single Cord Blood Transplantation for Patients With Hematologic Malignancies

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 10 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well donor umbilical cord blood transplant with ex-vivo expanded cord blood progenitor cells (dilanubicel) works in treating patients with blood cancer. Before the transplant, patients will receive chemotherapy (fludarabine, cyclophosphamide and in some cases thiotepa) and radiation therapy. Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.

Detailed description

OUTLINE: Patients receive either regimen A or regimen B. REGIMEN A: Patients (10 through 45 years old) receive fludarabine intravenously (IV) over 30 minutes on days -8 to -6 and cyclophosphamide IV on days -7 and -6. Patients undergo total body irradiation (TBI) twice daily (BID) on days -4 to -1. Patients receive unmanipulated cord blood unit IV followed by dilanubicel IV within the next 24 hours on day 0. REGIMEN B: Patients (10 through 65 years old) receive fludarabine IV over 30-60 minutes on days -6 to -3 and IV over 30 minutes on day -2, cyclophosphamide IV on day -6, and thiotepa IV over 2-4 hours on days -5 and -4. Patients undergo TBI once daily (QD) on days -2 and -1. Patients receive unmanipulated cord blood unit IV followed by dilanubicel IV within the next 24 hours on day 0. All patients undergo bone marrow aspirate and biopsy as clinically indicated during screening and on study. Patients undergo multigated acquisition scan (MUGA) or echocardiography (ECHO), and computed tomography (CT) during screening. Patients also undergo blood sample collection on study. After completion of study treatment, patients are followed up at 180 days, 1 year, and 2 years.

Conditions

• Acute Biphenotypic Leukemia
• Acute Lymphoblastic Leukemia
• Chronic Myelogenous Leukemia, BCR-ABL1 Positive
• Hematopoietic and Lymphoid Cell Neoplasm
• Myelodysplastic Syndrome
• Myelodysplastic Syndrome With Excess Blasts

Interventions

Type	Name	Description
BIOLOGICAL	Dilanubicel	Given IV
DRUG	Cyclophosphamide	Given IV
DRUG	Fludarabine	Given IV
DRUG	Thiotepa	Given IV
RADIATION	Total-Body Irradiation	Undergo TBI
PROCEDURE	Umbilical Cord Blood Transplantation	Given IV
OTHER	Laboratory Biomarker Analysis	Correlative studies
PROCEDURE	Biospecimen Collection	Undergo blood sample collection
PROCEDURE	Bone Marrow Aspirate	Undergo bone marrow aspirate and biopsy
PROCEDURE	Bone Marrow Biopsy	Undergo bone marrow aspirate and biopsy
PROCEDURE	Multigated Acquisition Scan	Undergo MUGA
PROCEDURE	Electrocardiography	Undergo ECHO
PROCEDURE	Computed Tomography	Undergo CT

Timeline

• Start date: 2022-05-10
• Primary completion: 2025-06-09
• Completion: 2027-04-02
• First posted: 2018-01-16
• Last updated: 2025-08-26

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study