Conditioning Regimen of Melphalan-busulfan and Fludarabine: a Randomized Study of Different Dose of Melphalan

Phase 3

Recruiting

• Conditions: Toxicities; Survivorship
• Interventions: Drug: melphalan

• Registration Number: NCT06829472
• Lead Sponsor: Shanghai Jiao Tong University School of Medicine

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.

Study Timeline

• Study First Submitted: 2023-08-12
• Study Start: 2023-12-01
• Status Verified: 2025-02
• Study First Submitted QC: 2025-02-14
• Last Update Submitted: 2025-02-14
• Study First Posted: 2025-02-17
• Last Update Posted: 2025-02-17
• Primary Completion: 2026-07-01
• Study Completion: 2026-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 120

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
MBF100	melphalan	Patients receive condoning regimen with melphalan (100mg/m2) with busulfan and fludarabine.
MBF140	melphalan	Patients receive condoning regimen with melphalan (140mg/m2) with busulfan and fludarabine.

Primary Outcome Measures

Name	Time	Method
Toxicities	up to 100 days	Toxicities with grade III or over

Secondary Outcome Measures

Name	Time	Method
disease-free survival	2 years	event defined as documentation of AML and MDS relapse or death of any causes
overall survival	2 years	event defined as death of any causes
Relapse	2 years	Documentation of bone marrow blasts \>5% or extra medullary disease
Non-relapse mortality	2 years	Death without documentation of AML and MDS disease

Trial Locations

Locations (2)

Rui Jin Hospital; 🇨🇳 Shanghai, Shanghai, China

Zhaxin Hospital, Go Broad Health Care; 🇨🇳 Shanghai, Shanghai, China