Clinical trial NCT01339988: Busulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide for Hematological Malignancies Hematocrit (HCT)
Official title: Treatment Plan for Hematologic Malignancies Using Intravenous Busulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide to Examine Results, Success and Side Effects of Treatment With Chemotherapy Only, as a Preparative Therapy for Patients With Cord Blood Transplants
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Acute Leukemias
Chronic Leukemias
Myelodysplastic Syndrome
Juvenile Myelomonocytic Leukemia
Keywords: Stem-cells
Transplantation
Busulfex
Cyclophosphamide
Total-Body-Ittadiation
Lead sponsor: Tel-Aviv Sourasky Medical Center
Sponsor class: OTHER_GOV
First posted: 2011-04-21
Enrollment: 10
Sex: MALE
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Long-term follow-up studies have demonstrated significant late toxicities of total body irradiation (TBI), which are most marked in children radiated at a young age. Growth failure, decline in cognitive function, and endocrine abnormalities have all been described. Good outcomes can be achieved with alkylating agents only as a preparative regimen. This plan will use a combination of busulfan and cyclophosphamide (Bu/Cy) with or without antithymocyte globulin (ATG) to reduce the late toxicities of therapy that includes TBI.


Eligibility criteria: Inclusion Criteria:

* Suitable cord blood from matched unrelated or related donor.
* Cardiac (Echo/EKG): shortening fraction ≥ 27%
* Electrolytes within normal CCHMC limits.
* Pulmonary function tests: DLCO ≥ 50%
* Renal: creatinine clearance/GFR ≥ 60 ml/minute/1.73m2
* Lumbar puncture: no leukemic infiltrate.
* CBC: ANC ≥ 1000ml and unsupported platelet count of ≥ 50,000/ml
* Documented HSV and CMV titers, Hepatitis B surface antigen, Hepatitis C by serology, HIV by serology: all negative.
* Hepatic transaminases \< 2.5x normal; Total bilirubin \< 2 mg/dl Patients who do not meet above organ function criteria (liver, cardiac, renal), due to the presence of a tumor compromising these organs, may have exception made for these criteria and remain eligible for treatment plan after consultation with Program Director of Blood and Marrow Transplant

Exclusion Criteria:

* Patients with neoplastic or non-neoplastic disease of any major organ system that would compromise their ability to withstand the pre-transplant conditioning regimen.
* Patients with uncontrolled (culture or biopsy positive) infections requiring intravenous antivirals, antibiotics, or antifungals. Patients on prolonged antifungal therapy with a history of fungal infection should be considered for a non-myeloablative protocol.
* Patients who are pregnant or lactating. Patients of childbearing potential must practice an effective method of birth control while participating on this treatment plan.
* HIV seropositive patients

Primary outcomes:
measure: Overall survival
timeFrame: Five years
Secondary outcomes:
measure: Disease free survival
timeFrame: Five years
Other outcomes:
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Study officials:
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