NCT06297772

Compare the Efficacy and Safety of Dec-FB4 and FB4 as Conditioning Regimen for AML-MR

Comparing the Efficacy and Safety of Decitabine-Fludarabine-busulfan (Dec-FB4) and Fludarabine-busulfan (FB4) as Conditioning Regimens for AML-MR

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 220 (estimated)
• Sponsor: Ruijin Hospital · Academic / Other
• Sex: All
• Age: 16 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

A multicenter, randomized, controlled clinical study comparing the efficacy and safety of allogeneic hematopoietic stem cell transplantation with decitabine-Fludarabine- busulfan (Dec-FB4) and Fludarabine-busulfan (FB4) as pretreatment regimens for the treatment of acute myeloid leukemia in adults with MR gene abnormalities

Detailed description

AML-MR is one of high-risk AML with aggressive disease progression and poor prognosis. The median survival of AML-MR is only 10.5 months. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective post-remission therapy for high-risk acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS). Selection of an appropriate pretreatment regimen that balances the risk of relapse and reduces the risk of non-relapse mortality is a key component of successful HSCT. FB4 regimen consisting of Fludarabine (FLU) combined with busulfan (BU) has become the most commonly used myeloablative preconditioning regimen for allo-HSCT in patients with AML/MDS, and prospective studies have demonstrated a similar relapse rate and lower treatment-related mortality than the Bu/cy regimen. Decitabine is also approved for the treatment of AML and MDS as an inhibitor of DNA methyltransferase I, which allows for the expression of silenced oncogenes and terminal differentiation of leukemia cells, and as a single agent is superior to supportive therapy for patients with MDS. In addition, decitabine has been shown to attenuate GVHD by enhancing the function of regulatory T cells, and in myeloid tumors, several prospective single-arm and retrospective clinical studies have demonstrated that modified pretreatment regimens containing decitabine have a lower relapse rate, as well as a reduction in the incidence and severity of GVHD, with satisfactory survival data in allogeneic hematopoietic stem cell transplantation. The investigators have not yet obtained direct evidence in high-risk patients to confirm the superiority of decitabine-containing pretreatment regimens over standard pretreatment regimens (FB4). There is a lack of prospective, controlled studies to clarify the efficacy and safety of decitabine-containing transplantation preconditioning in high-risk patients, especially those with specific AML-MR.Therefore, this multicenter, randomized controlled study was designed to compare the efficacy and safety of Dec-FB4 and FB4 regimens as pretreatment regimens for allogeneic HSCT in adults with AML-MR and to assess whether Dec-FB4 is superior or noninferior to FB4, in order to provide a reliable evidence-based medical basis for guiding the therapeutic choices for AML-MR/MDS patients.

Conditions

• AML, Adult

Interventions

Type	Name	Description
DRUG	Decitabine	decitabine 20mg/m2 per day for consecutive 5 days
DRUG	Fludarabine Injection	fludarabine 30mg/m2 per day for consecutive 5 days
DRUG	Busulfan Injection	Busulfan 3.2mg/kg per day for consecutive 4 days

Timeline

• Start date: 2024-06-01
• Primary completion: 2026-03-01
• Completion: 2028-05-01
• First posted: 2024-03-07
• Last updated: 2024-06-04

Locations

1 site across 1 country: China