NCT06013423

Cord Blood Transplant, Cyclophosphamide, Fludarabine, and Total-Body Irradiation in Treating Patients With High-Risk Hematologic Diseases

Optimized Cord Blood Transplantation for the Treatment of High-Risk Hematologic Malignancies in Adults and Pediatrics

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 54 (estimated)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 6 Months – 65 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well giving an umbilical cord blood transplant together with cyclophosphamide, fludarabine, and total-body irradiation (TBI) works in treating patients with hematologic diseases. Giving chemotherapy, such as cyclophosphamide, fludarabine and thiotepa, and TBI before a donor cord blood transplant (CBT) helps stop the growth of cancer and abnormal cells and helps 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after transplant may stop this from happening in patients with high-risk hematologic diseases.

Detailed description

OUTLINE: Patients are assigned to 1 of 2 arms. ARM I: Patients aged 6 months through 30 years old receive myeloablative conditioning comprising fludarabine intravenously (IV) over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 and -6, and undergo high-dose TBI twice daily (BID) on days -4 to -1. Patients then undergo UCBT on day 0. Patients undergo blood sample collection throughout the study. Patients undergo echocardiography (ECHO) or multigated acquisition scan (MUGA) and diagnostic imaging during screening and as clinically indicated on study. Patients also undergo blood sample collection throughout the study and bone marrow aspirate during screening and on study. ARM II: Patients aged 6 months through 65 years old receive myeloablative conditioning comprising fludarabine IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 2-4 hours on days -5 and -4, and middle-intensity TBI once daily (QD) on days -2 and -1. Patients undergo ECHO or MUGA and diagnostic imaging during screening and as clinically indicated on study. Patients also undergo blood sample collection throughout the study and bone marrow aspirate during screening and on study. All patients receive GVHD prophylaxis comprising cyclosporine IV over 1 hour every 8 or 12 hours, then cyclosporine orally (PO) (if tolerated), on days -3 to 100 with taper on day 101. Patients also receive mycophenolate mofetil IV every 8 hours on days 0 to 7 and then PO (if tolerated) three times daily (TID) on days 8-30. Mycophenolate mofetil is tapered to BID on day 30 or 7 days after engraftment if there is no acute GVHD, and then tapered over 2-3 weeks beginning on day 45 (or 15 days after engraftment if engraftment occurred \> day 30) after engraftment if there continues to be no evidence of acute GVHD. After completion of study treatment, patients are followed up at day 180, 1 year, and 2 years.

Conditions

• Acute Leukemia of Ambiguous Lineage
• Acute Lymphoblastic Leukemia
• Acute Myeloid Leukemia
• Blastic Plasmacytoid Dendritic Cell Neoplasm
• Hematopoietic and Lymphatic System Neoplasm
• Mixed Phenotype Acute Leukemia
• Myelodysplastic Syndrome
• Myeloproliferative Neoplasm
• Non-Hodgkin Lymphoma
• Chronic Myeloid Leukemia, BCR-ABL1 Positive

Interventions

Type	Name	Description
PROCEDURE	Biospecimen Collection	Undergo blood sample collection
PROCEDURE	Bone Marrow Aspirate	Undergo bone marrow aspirate
DRUG	Cyclophosphamide	Receive IV
DRUG	Cyclosporine	Receive IV or PO
PROCEDURE	Diagnostic Imaging	Undergo diagnostic imaging
PROCEDURE	Echocardiography	Undergo ECHO
DRUG	Fludarabine Phosphate	Receive IV
PROCEDURE	Multigated Acquisition Scan	Undergo MUGA
DRUG	Mycophenolate Mofetil	Receive IV
OTHER	Survey Administration	Ancillary studies
DRUG	Thiotepa	Receive IV
RADIATION	Total-Body Irradiation	Undergo high-dose or middle-intensity TBI
PROCEDURE	Umbilical Cord Blood Transplantation	Undergo UCBT

Timeline

• Start date: 2024-07-23
• Primary completion: 2031-10-31
• Completion: 2032-10-31
• First posted: 2023-08-28
• Last updated: 2026-01-22

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study