Busulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide for Hematological Malignancies Hematocrit (HCT)

Myelodysplastic Syndrome Clinical Trial

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT01339988
• Other study ID #: TASMC-11-MB-442-CTIL
• Status: Not yet recruiting
• Phase: Phase 4
• First received: April 20, 2011
• Last updated: April 20, 2011
• Start date: June 2011
• Est. completion date: June 2019

Study information

• Verified date: April 2011
• Source: Tel-Aviv Sourasky Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: Israel: Ethics Commission
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 10
• Est. completion date: June 2019
• Est. primary completion date: June 2014
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 21 Years

Eligibility

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

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Acute Leukemias
• Chronic Leukemias
• Juvenile Myelomonocytic Leukemia
• Leukemia
• Leukemia, Myelomonocytic, Acute
• Leukemia, Myelomonocytic, Chronic
• Leukemia, Myelomonocytic, Juvenile
• Myelodysplastic Syndrome
• Myelodysplastic Syndromes
• Preleukemia

Intervention

Drug:
• Busulfan/Cyclophosphamide
Conditioning regimen: Cyclophosphamide 50mg/kg/day for 4 days + Busulfan 0.8-1.0mg/kg/day for 4 days.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Tel-Aviv Sourasky Medical Center

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall survival		Five years	Yes
Secondary	Disease free survival		Five years	Yes