NCT01985061

Evaluation of 3 Different Doses of IV Busulfan

Prospective and Multicentre Evaluation of 3 Different Doses of IV Busulfan Associated With Fludarabine and Thymoglobuline in the Conditioning of Allogeneic Stem Cell Transplantation (SCT) From a Matched Related or Unrelated Donor in Patients With Poor Prognosis Myeloid Malignancies

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 177 (estimated)
• Sponsor: Institut Paoli-Calmettes · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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

Conditions

• Myelodysplastic Syndrome
• Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	BX2
DRUG	BX3
DRUG	BX4-Suspended	Suspended

Timeline

• Start date: 2013-12-01
• Primary completion: 2026-07-07
• Completion: 2026-07-07
• First posted: 2013-11-15
• Last updated: 2025-06-15

Locations

1 site across 1 country: France