NCT07094464

Maintenance Therapy With Selinexor and Azacitidine in TP53 Mutant AML/MDS After Transplantation

A Single-Arm, Open-Label Phase I Clinical Study of Selinexor Combined With Azacitidine for Maintenance Therapy in TP53 Mutant AML/MDS Patients Post-Transplant

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Daihong Liu · Academic / Other
• Sex: All
• Age: 14 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This study aims to explore the safety and efficacy of selinexor combined with azacitidine for maintenance therapy in TP53 mutant AML/MDS patients following transplantation.

Detailed description

This research targets high-risk recurrence patients with TP53 mutations in AML/MDS, administering low-dose azacitidine combined with selinexor for maintenance treatment post-allo-HCT (Allogeneic Hematopoietic Cell Transplantation). The goal is to observe the safety and tolerability of this drug combination as maintenance therapy post-transplant. The primary outcome measure will be post-transplant recurrence rate and non-relapse survival rate, while secondary outcomes include overall survival and non-relapse mortality. Previous studies have indicated that azacitidine and selinexor are safe and effective in maintenance therapy for AML/MDS. This study aims to reduce the risk of recurrence and prolong disease-free survival.

Conditions

• TP53-mutated MDS and AML
• Allogeneic Hematopoietic Stem Cell Transplantation

Interventions

Type	Name	Description
DRUG	Maintenance Therapy with Selinexor and Azacitidine	1. Selinexor: Oral administration in 4 cycles: \- Dose Escalation Phase: Cohort 1: Selinexor, 20 mg, twice a week for 2 weeks Cohort 2: Selinexor, 40 mg, twice a week for 2 weeks Cohort 3: Selinexor, 60 mg, twice a week for 2 weeks \- Dose Expansion: MTD/RP2D will be determined based on safety. 2. Azacitidine: 35 mg/m² for 5 days.

Timeline

• Start date: 2025-06-01
• Primary completion: 2027-06-30
• Completion: 2028-12-30
• First posted: 2025-07-30
• Last updated: 2025-07-30

Locations

1 site across 1 country: China