Evaluation of 3 Different Doses of IV Busulfan

Albeit the safety of the stem cell transplantation procedure has been greatly improved, further refining the intensity of the conditioning is an important issue to explore, especially in patients with poor prognosis, the goal being to maintain the very favorable safety profile and improve the disease control. This is the goal our prospective trial; we aim to prospectively evaluate in a prospective multicenter trial the efficacy of different conditioning regimens in patients with high-risk myeloid malignancies. The study is a phase II trial randomizing patients between a prospective active control arm (BX2) and two experimental arms (BX3 and BX4). A standard group was kept in this clinical trial in order to avoid the limitations induced by the comparison with historical controls in the context of continuously improving practice. Each experimental arm will be conducted in parallel according to a standard phase II trial design. In addition, this trial will associate four ancillary studies to the main clinical objective: 1/ a prospective assessment of the quality of life of the patients over a period of 2 years 2/ an analysis of the cost effectiveness of the procedure, assessed over a period of 2 years 3/ an observational busulfan pharmacokinetic study 4/ a busulfan pharmacogenomic study

NCT Number	NCT01985061
Lead Sponsor	Institut Paoli-Calmettes
Conditions	Myelodysplastic Syndrome, Acute Myeloid Leukemia
Enrollment	177 participants
Start Date	2013-12
Primary Completion	2026-07-07 (estimated)
Study Completion	2026-07-07 (estimated)
Updated on ClinicalTrials.gov	2025-06-15