A Phase 2 interventional study of fludarabine and treosulfan in Acute Myeloid Leukemia, sponsored by Institut Paoli-Calmettes. Not yet recruiting. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-05-20.
Sponsored by Institut Paoli-Calmettes · Phase 2, Interventional, and Treatment
Acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) predominantly affect older adults, and their incidence continues to rise with advanced age. For many patients, allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative option capable of providing long-term disease control through the graft-versus-leukemia (GVL) effect. Historically, however, allo-HSCT was rarely offered to patients older than 50 years because of the high morbidity and mortality associated with myeloablative conditioning regimens and limited supportive care strategies. Over the past two decades, advances in reduced-intensity conditioning (RIC), infection prophylaxis, and donor availability have profoundly transformed the landscape, allowing increasing numbers of older patients to access transplantation.
Multiple studies have demonstrated that allo-HSCT confers a survival benefit in older AML patients in complete remission compared with consolidation chemotherapy alone.
The intensity of conditioning profoundly influences both relapse risk and non-relapse mortality (NRM). myeloablative conditioning (NMAC) regimens are attractive for older adults due to their low toxicity but rely solely on the immunologic GVL effect and thus carry a higher relapse risk. Reduced-intensity conditioning (RIC) regimens, incorporating intermediate-dose alkylating agents such as busulfan, melphalan, or thiotepa, offer stronger anti-leukemic effect but at the cost of greater toxicity.
These observations underscore the central question: can a conditioning regimen combine strong anti-leukemic potency with the low toxicity required for older patients undergoing Haplo-SCT? The main objective is to evaluate the efficacy of FT-RIC regimen before Haplo-SCT for older and/or frail patients diagnosed with AML, who are not eligible for a myeloablative conditioning (MAC) regimen.
To achieve this objective, the investigators will assess Progression Free Survival (PFS) defined as the time from allo-HSCT to AML relapse or death.
This is a Multicenter trial, single arm prospective of phase II. Once the conditioning has been administered and the transplant performed, the patient will receive standard routine follow-up care, with the addition of questionnaires, and for patients followed at the Institut Paoli Calmettes only, blood samples will be collected.
Exclusion Criteria:
After screening and inclusion, patients will be given a RIC regimen based on the market-approved association of fludarabine and treosulfan (FT-RIC): * Fludarabine (concentrate for solution for injection/infusion) is a marketed purine analogue and antineoplastic agent. * Treosulfan (powder for solution for infusion) is a marketed alkylating medication
Drug: fludarabine and treosulfan
As per standard practices, patients will be hospitalized during the treatment period. The treatment is administered by the nurses of the department under the responsibility of the investigator.Fludarabine (30 mg/m²/day from day-6 to day-2), iv andTreosulfan (10 g/m²/day from day-4 to day-2), iv
The main objective is to evaluate the efficacy of FT-RIC regimen before Haplo-SCT for older and/or frail patients diagnosed with AML, who are not eligible for a MAC regimen.
Progression Free Survival (PFS) defined as the time from allo-HSCT to AML relapse or death
Time frame: through study completion an average of 4 years
To evaluate adverse events related to the FT combination according to CTCAE V6.0
Conditioning related toxicity according to CTCAE V.6.0
Time frame: through study completion an average of 4 years
To evaluate engraftment after FT-RIC
rate of graft failure
Time frame: through study completion an average of 4 years
To evaluated hematological recovery after FT-RIC
Cumulative incidence of neutrophil and platelet recovery
Time frame: after hematological recovery
to evaluate incidence of both acute and chronic GVHD after FT-RIC
Cumulative incidence of acute GVHD and Cumulative incidence of chronic GVHD
Time frame: through study completion an average of 4 years
To evaluate survival, non-relapse mortality and cause of death after FT-RIC
Probability of Overall Survival and Probability of GVHD
Time frame: through study completion an average of 4 years
To evaluate the immunosuppressive therapy duration after FT-RIC
Prevalence of immunosuppressive therapy (IST) and GVHD at 3, 6, 9, 12 months
Time frame: through study completion an average of 4 years
No study locations are listed for this record.
This study is not yet recruiting, as verified in May 2026. You cannot join it, but the record below documents what was studied.
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Institut Paoli-Calmettes