BUSULFAN for High-risk myeloid malignancies|Myelodysplastic syndrome|Acute myeloid leukemia

Inclusion criteria

• {"criterion_text":"- Patients with poor prognosis myeloid malignancies in particular : myelodysplasic syndrome, AML beyond CR1 regardless of the cytogenetic or molecular abnormalities or CR1 AML after double induction regardless of the cytogenetic or molecular abnormalities or CR1 AML with no criteria for favorable risk according to the ELN classification"}
• {"criterion_text":"- Adult patients aged ≥ 50 years up to 65 or < 50 years not eligible for myeloablative conditioning regimen based on TBI or double alkylating agent combinations"}
• {"criterion_text":"- Availability of a HLA identical sibling or matched unrelated donor (10/10)"}
• {"criterion_text":"- Affiliation to social security"}
• {"criterion_text":"- Written Informed Consent"}

Exclusion criteria

• {"criterion_text":"- History of previous Allo-HSCT"}
• {"criterion_text":"- HIV positivity"}
• {"criterion_text":"- Signs of chronic active hepatitis B and/or C"}
• {"criterion_text":"- Evolutive psychiatric disease"}
• {"criterion_text":"- Concomitant neoplasic disease"}
• {"criterion_text":"- Pregnant or lactating woman or without contraception (for child bearing potential women)"}
• {"criterion_text":"- Usual contra-indications for Allo-HSCT"}

Primary endpoints

• {"endpoint_text":"- Time to progression or death","definition_or_measurement_approach":"Assessment of 2-year progression-free survival rates following HSCT using different dose levels of IV Busulfan combined with fludarabine and thymoglobuline (time from transplant to progression or death; primary objective is 2-year PFS)"}

Secondary endpoints

• {"endpoint_text":"- Time to death and cause of death","definition_or_measurement_approach":"Time from transplant to death; cause of death documented"}
• {"endpoint_text":"- Time to acute and chronic GVHD according to the NIH classification and relapse","definition_or_measurement_approach":"Time-to-event measurement for acute and chronic graft-versus-host disease using NIH classification; time to relapse recorded"}
• {"endpoint_text":"- Response to treatment","definition_or_measurement_approach":"Assessment of hematologic response rates to treatment (as documented in trial records)"}
• {"endpoint_text":"- Hematological recovery defined as the achievement 500 ANC and 50 000 platelets (without transfusion)","definition_or_measurement_approach":"Hematological recovery defined as achievement of ANC ≥ 500 and platelets ≥ 50,000 without transfusion"}
• {"endpoint_text":"- Full donor chimerism achievement at M1, M2, M3","definition_or_measurement_approach":"Documentation of full donor chimerism at month 1, month 2 and month 3 post-transplant"}
• {"endpoint_text":"- Occurrence of grade 3-4 adverse events according the CTC AE v4.0 scale within 6 months after conditionning","definition_or_measurement_approach":"Recording incidence of grade 3-4 adverse events according to CTCAE v4.0 occurring within 6 months after conditioning"}

France

Earliest CTIS Part Ii Submission Date

26-09-2024

Latest Decision Or Authorization Date

05-11-2024

Processing Time Days

40

Number Of Sites

17

Number Of Participants

177

Primary sponsor

Full Name

Institut Paoli Calmettes

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France