Clinical trial NCT00516152: Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing MUD SCT
Official title: Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Chronic Myeloid Leukemia
Acute Myelogenous Leukemia
Myelodysplasia
Acute Lymphocytic Leukemia
Severe Aplastic Anemia
Non-Hodgkin's Lymphoma
Lymphoproliferative Disease
Multiple Myeloma
Advanced Myeloproliferative Disease
Keywords: Matched Unrelated Donor
Stem Cell Transplantation
Busulfan
fludarabine
Hematopoietic Disorder
Lead sponsor: University of California, San Francisco
Sponsor class: OTHER
First posted: 2007-08-15
Enrollment: 36
Sex: ALL
Minimum age (years): 15
Maximum age (years): 61
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to assess the safety and efficacy of performing unrelated stem cell transplants using intravenous busulfan and fludarabine as preparative therapy and tacrolimus plus methotrexate as the GVHD prophylaxis regimen. The goal is to demonstrate safety, aiming for a transplant related mortality rate (TRM) of \< or equal to 40% at 100 days. A TRM of \> or equal to 60% will be considered unacceptable. Another goal is to demonstrate efficacy by showing and overall survival of \>40% at 1-year following transplant.


Eligibility criteria: Inclusion Criteria:

* No fully or single-antigen mismatched sibling donor is available to donate stem cells.
* Age \>15 and \<61
* ECOG PS \< or equal to 2
* Adequate renal function with serum creatinine \<2.0 mg/dl
* Pulmonary diffusing capacity \>40% of predicted
* Cardiac ejection fraction \>40% as measured by radionuclide wall motion study or echocardiography
* No active liver disease. Total bilirubin must be \< or equal to 2.0 mg/dl. Alkaline phosphatase and AST must be less than three times the upper limit of normal. Patients with hepatitis C and active hepatitis B are eligible only if a liver biopsy is performed and there is \< or equal to grade 2 inflammation. Patients wtih a history of HBV infection should be tested for HBeAg, antiHBe and HBV DNA (quantitative). Patients with active HBV viral replication should receive anti-viral therapy.
* Negative serology for the human immunodeficiency virus (HIV)
* Available HLA-matched donor (see HLA compatibility requirements below)
* Signed informed consent from the recipient

Exclusion Criteria:

* Ongoing active infection
* Pregnancy and/or nursing
* Active, uncontrolled CNS leukemia
* Opinion of BMT Committee that autologous or mini-allogeneic transplant would be the preferable form of treatment
* Receipt of any chemotherapy within 3 weeks of study entry except for hydroxyurea or imatinib mesylate. Use of interferon within 3 months of starting therapy.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
Study officials:
affiliation: University of California, San Francisco
name: Thomas G. Martin, M.D.
role: PRINCIPAL_INVESTIGATOR
References: