NCT00796068

Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation in Treating Patients With Hematological Cancer Who Are Undergoing Umbilical Cord Blood Transplant

Transplantation of Umbilical Cord Blood in Patients With Hematological Malignancies Using a Treosulfan Based Preparative Regimen

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 130 (actual)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 65 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well giving treosulfan together with fludarabine phosphate and total-body irradiation (TBI) works in treating patients with hematological cancer who are undergoing umbilical cord blood transplant (UCBT). Giving chemotherapy, such as treosulfan and fludarabine phosphate, and TBI before a donor UCBT helps stop the growth of cancer cells and helps stop the patient's immune system from rejecting the donor's stem cells. When the stem cells from a related or unrelated donor, that do not exactly match the patient's blood, 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 also make an immune response against the body's normal cells. Giving cyclosporine (CsA) and mycophenolate mofetil (MMF) after the transplant may stop this from happening.

Detailed description

PRIMARY OBJECTIVES: I. Graft failure/rejection and secondary graft failure. II. Day -200 non-relapse mortality. SECONDARY OBJECTIVES: I. Platelet engraftment by six months. II. Grade II-IV and III-IV acute graft-versus-host disease (GVHD) at day 100 and one year. III. Chronic GVHD. IV. Clinically significant infections. V. Overall survival. VI. Relapse or disease progression. VII. Immune reconstitution (Fred Hutchinson Cancer Research Center \[FHCRC\] only). VIII. Emergence of a dominant unit (FHCRC only). OUTLINE: Patients are assigned to 1 of 2 arms. ARM I (low risk for graft failure): Patients receive a conditioning regimen comprising fludarabine phosphate intravenously (IV) over 1 hour once daily (QD) on days -6 to -2 and treosulfan IV over 120 minutes on days - 6 to -4. Patients undergo TBI on day -1. Patients then undergo donor UCBT on day 0. Patients receive GVHD prophylaxis comprising cyclosporine IV over 1 hour or orally (PO) 2-3 times daily on days -3 to 100, followed by a taper in the absence of GVHD. Patients also receive mycophenolate mofetil IV 3 times daily on days 0 to 40, followed by a taper in the absence of GVHD. ARM II (high risk for graft failure): Patients receive a conditioning regimen, TBI, donor UCBT, GVHD prophylaxis, and mycophenolate mofetil as in Arm I. After completion of the study treatment, patients are followed up at 6 months and 1 and 2 years.

Conditions

• Acute Biphenotypic Leukemia
• Acute Lymphoblastic Leukemia in Remission
• Acute Myeloid Leukemia in Remission
• Blasts Under 5 Percent of Bone Marrow Nucleated Cells
• Chronic Myelogenous Leukemia, BCR-ABL1 Positive
• Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
• Myelodysplastic Syndrome (MDS)

Interventions

Type	Name	Description
DRUG	Cyclosporine	Given IV or PO
DRUG	Fludarabine Phosphate	Given IV
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Mycophenolate Mofetil	Given IV
RADIATION	Total-Body Irradiation	TBI administered day -1: 200 cGy (or escalated to 300 cGy, 400 cGy, or 450 cGy per protocol statistical section)
DRUG	Treosulfan	Given IV
PROCEDURE	Umbilical Cord Blood Transplantation	Undergo single or double unit UCBT

Timeline

• Start date: 2009-02-24
• Primary completion: 2021-12-14
• Completion: 2021-12-14
• First posted: 2008-11-24
• Last updated: 2023-02-17
• Results posted: 2023-02-17

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study