Sorafenib Maintenance for Prophylaxis of Leukemia Relapse in Allo-HSCT Recipients With FLT3 Negative Acute Leukemia

Phase 2

• Conditions: Acute Leukemia; Relapse; Hematopoietic Stem Cell Transplantation
• Interventions: Drug: Sorafenib

• Registration Number: NCT04674345
• Lead Sponsor: Nanfang Hospital, Southern Medical University

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of sorafenib maintenance after allo-HSCT in FLT3-negative acute leukemia patients.

Detailed Description

Sorafenib is a multikinase inhibitor that blocks multiple pathways involved in the development and progression of acute leukemia, such as FLT3-ITD, the RAS and RAF gene families, KIT, and the VEGF and PDGF receptors. Recently, two back-to-back randomized controlled trials both reveal that sorafenib maintenance after allo-HSCT could prevent relapse in patients with FLT3-ITD AML, resulting in a survival benefit. Sorafenib has also been explored in the treatment of acute leukemia without FLT3 mutations and shown promising results. Currently, relapse remains the major cause of transplant failure, especially for high-risk and refractory acute leukemia patients. Once patients relapse after allo-HSCT, the prognosis is dismal. Therefore, prevention of relapse is of great importance to improve the prognosis. Based on the current research status, we plan to conduct a prospective, multicenter, phase 2 randomized controlled trial to explore the efficacy and safety of sorafenib maintenance after allo-HSCT in FLT3-negative acute leukemia patients.

Study Timeline

• Status Verified: 2020-12
• Study First Submitted: 2020-12-14
• Study First Submitted QC: 2020-12-14
• Study Start: 2020-12-15
• Study First Posted: 2020-12-19
• Last Update Submitted: 2021-10-30
• Last Update Posted: 2021-11-08
• Primary Completion: 2022-12-31
• Study Completion: 2023-12-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 346

Inclusion Criteria

• Patients with FLT3-negative acute leukemia undergoing first allo-HSCT
• Age 18 to 65 years old with ECOG performance status 0-2
• Hematopoietic recovery within 60 days post-transplantation
• Sign informed consent form, have the ability to comply with study and follow-up procedures

Exclusion Criteria

• Acute promyelocytic leukemia (AML subtype M3)
• Acute leukemia with FLT3-ITD or FLT3-TKD mutations
• Philadelphia-positive acute lymphoblastic leukemia
• Chronic myelogenous leukemia with blast crisis
• Intolerance to sorafenib pre-transplantation
• Life expectancy less than 30 days post-transplantation
• Active aGVHD or uncontrolled infections within 60 days post-transplantation
• Cardiac dysfunction (particularly congestive heart failure, unstable coronary artery disease and serious cardiac ventricular arrhythmias requiring antiarrhythmic therapy)
• Respiratory failure ( PaO2 ≤60mmHg)
• Hepatic abnormalities (total bilirubin ≥3 mg/dL, aminotransferase >2 times the upper limit of normal)
• Renal dysfunction (creatinine clearance rate < 30 mL/min)
• ECOG performance status 3, 4 or 5
• With any conditions not suitable for the trial (investigators' decision)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Sorafenib group	Sorafenib	Sorafenib will be administered at 45-60 days post-transplantation and taken for one year.

Primary Outcome Measures

Name	Time	Method
Incidence of leukemia relapse	1 year

Secondary Outcome Measures

Name	Time	Method
Adverse effects	1 year
Overall survival	1 year
Leukemia-free survival	1 year

Trial Locations

Locations (1)

Department of Hematology,Nanfang Hospital, Southern Medical University; 🇨🇳 Guangzhou, Guangdong, China