Clinical trial NCT06829472: Conditioning Regimen of Melphalan-busulfan and Fludarabine: a Randomized Study of Different Dose of Melphalan
Official title: Conditioning Regimen of Melphalan-busulfan and Fludarabine: a Randomize Study Comparing 100mg/m2 to 140mg/m2 Melphalan for Adult Patients With Acute Myeloid Leukemia or Myelodysplasia Syndrome.
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Toxicities
Survivorship
Keywords: melphalan, AML, MDS, allogeneic stem cell transplantation
Lead sponsor: Shanghai Jiao Tong University School of Medicine
Sponsor class: OTHER
Collaborators: name: Shanghai Clinical Research Center
First posted: 2025-02-17
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Treatment
Primary purpose: TREATMENT

Brief summary: Conditioning regimen with melphalan-busulfan and fludarabine (MBF) achieved low relapse rate in patients with acute myeloid leukemia (AML) and myelodysplasia syndrome (MDS). In our previous retrospective study, we demonstrated that melphalan dose at 100 or 140mg/m2 had similar incidence of relapse but it might had lower toxicities. In this prospective randomize study, we aim to compare the transplantation outcome in adult patients with AML/MDS receiving either MBF with 100 or 140 mg/m2 melphalan.

Detailed description: Intensity of conditioning regimen plays a important role in allogeneic stem cell transplantation (allo-HSCT) for patents with AML and MDS. IN our previous prospective study, we demonstrated that condoning regimen of dual alkylating agents with melphalan-busulfan and fludarabine (MBF) achieved a very low relapse rate (\~6%) in patients with acute myeloid leukemia (AML) and myelodysplasia syndrome (MDS). In that study, adult patients received melphalan 140mg/m2 while patients \>=60 or with high transplantation risk in terms of HCT-CI received reduced dose of melphalan (100mg/m2). Overall, it was shown that 100 or 140mg/m2 melphalan had similar incidence of relapse while the 100mg/m2 melphalan presented lower incidence of toxicities.In this prospective randomize study, we aim to compare the transplantation toxicities and outcomes in young adults (18\~55) with AML/MDS receiving either 100 or 140 mg/m2 melphalan as conditioning regimen.

Eligibility criteria: Inclusion Criteria:

* Patients with AML in first remission
* Patients with MDS with bone marrow blast \>5% but remain less than 20% before transplantation
* Donor available: HLA matched sibling donor, 9\~10/10 matched unrelated donor or haplo-identical donor
* Inform consent provided

Exclusion Criteria:

* Patients with active infection (bacteria, fungal or viral)
* Patients with abnormal liver, renal and cardiac function

Primary outcomes:
measure: Toxicities
timeFrame: up to 100 days
Secondary outcomes:
measure: Relapse
timeFrame: 2 years
measure: Non-relapse mortality
timeFrame: 100 days
measure: Non-relapse mortality
timeFrame: 2 years
measure: disease-free survival
timeFrame: 2 years
measure: overall survival
timeFrame: 2 years
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Rui Jin Hospital
state: Shanghai
city: Shanghai
country: China
facility: Zhaxin Hospital, Go Broad Health Care
state: Shanghai
Study officials:
affiliation: Go Broad Health Care
name: chun Wang
role: STUDY_DIRECTOR
References: