NCT00445744

Cyclophosphamide and Busulfan Followed by Donor Stem Cell Transplant in Treating Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome

Cyclophosphamide Followed by Intravenous Busulfan as Conditioning for Hematopoietic Cell Transplantation in Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome.

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 52 (actual)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 65 Years
• Healthy volunteers: Not accepted

Summary

This trial is studying the side effects and how well giving cyclophosphamide and busulfan followed by donor stem cell transplant works in treating patients with myelofibrosis, acute myeloid leukemia, or myelodysplastic syndrome. Giving chemotherapy, such as cyclophosphamide and busulfan, before a donor stem cell transplant helps stops the growth of cancer cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving tacrolimus and methotrexate after the transplant may stop this from happening

Detailed description

PRIMARY OBJECTIVES: I. To estimate the incidence of hepatotoxicity with a conditioning regimen of CY (cyclophosphamide)/tBU (busulfan) in patients receiving hematopoietic cell transplant (HCT). SECONDARY OBJECTIVES: I. To determine overall and non-relapse mortality at day +200 after HCT. II. To determine the peak bilirubin levels through day +20 after HCT. III. To determine the incidence of pulmonary toxicity in the form of idiopathic pulmonary syndrome (IPS). IV. To determine the rate of graft failure. V. To determine the time to engraftment. VI. To determine the rate of relapse. VII. To determine the incidence and severity of graft-versus-host disease (GVHD). VIII. To evaluate the pharmacokinetics/dynamics of BU and CY. X. To evaluate the pharmacogenomics of response, toxicity and pharmacokinetics of CY/tBU. OUTLINE: CONDITIONING REGIMEN: Patients receive cyclophosphamide intravenously (IV) on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2. TRANSPLANTATION: Patients undergo allogeneic peripheral blood stem cell transplant on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacrolimus IV or orally (PO) twice daily on days -1 to 200 with taper on day 56 and methotrexate on days 1, 3, 6, and 11. After completion of study treatment, patients are followed periodically.

Conditions

• Adult Acute Myeloid Leukemia in Remission
• Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
• Adult Acute Myeloid Leukemia With Del(5q)
• Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
• Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
• Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
• Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)
• Childhood Acute Myeloid Leukemia in Remission
• Childhood Myelodysplastic Syndromes
• de Novo Myelodysplastic Syndromes
• Essential Thrombocythemia
• Myelodysplastic Syndrome With Isolated Del(5q)
• Polycythemia Vera
• Previously Treated Myelodysplastic Syndromes
• Primary Myelofibrosis
• Recurrent Adult Acute Myeloid Leukemia
• Recurrent Childhood Acute Myeloid Leukemia
• Secondary Acute Myeloid Leukemia
• Secondary Myelodysplastic Syndromes
• Secondary Myelofibrosis
• Untreated Adult Acute Myeloid Leukemia
• Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies

Interventions

Type	Name	Description
DRUG	cyclophosphamide	Given IV
DRUG	busulfan	Given IV
DRUG	tacrolimus	Given IV or PO
DRUG	methotrexate	Given IV
GENETIC	cytogenetic analysis	Correlative studies
OTHER	flow cytometry	Correlative studies
OTHER	pharmacological study	Correlative studies
OTHER	pharmacogenomic studies	Correlative studies
PROCEDURE	peripheral blood stem cell transplantation	Undergo PBPC transplantation
PROCEDURE	allogeneic hematopoietic stem cell transplantation	Undergo allogeneic transplantation

Timeline

• Start date: 2006-12-01
• Primary completion: 2011-06-01
• Completion: 2013-06-01
• First posted: 2007-03-09
• Last updated: 2018-01-02
• Results posted: 2018-01-02

Locations

1 site across 1 country: United States