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

NCT01339988 | Unknown Phase 4

• 1 other identifier: TASMC-11-MB-442-CTIL
• Study Type: interventional
• Target: 10
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Acute Leukemias; Chronic Leukemias; Myelodysplastic Syndrome; Juvenile Myelomonocytic Leukemia

Keywords

Stem-cells; Transplantation; Busulfex; Cyclophosphamide; Total-Body-Ittadiation

Outcome Measures

Primary Outcomes (1)

• Overall survival

  Five years

Secondary Outcomes (1)

• Disease free survival

  Five years

Study Arms (1)

Busulfan/Cyclophosphamide

EXPERIMENTAL

Drug: Busulfan/Cyclophosphamide

Interventions

Busulfan/Cyclophosphamide DRUG

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

Also known as: Cytoxan

Busulfan/Cyclophosphamide

Eligibility Criteria

• Age: Up to 21 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Tel-Aviv Sourasky Medical Center: lead

MeSH Terms

Conditions

Myelodysplastic Syndromes; Leukemia, Myelomonocytic, Juvenile

Interventions

Busulfan; Cyclophosphamide

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myelodysplastic-Myeloproliferative Diseases

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV

Study Record Dates

• First Submitted: April 20, 2011
• First Posted: April 21, 2011
• Study Start: June 1, 2011
• Primary Completion: June 1, 2014
• Study Completion: June 1, 2019
• Last Updated: April 21, 2011

Record last verified: 2011-04