NCT07101497

Phase 2 Trial of BN104 as Post-HSCT Maintenance in Acute Leukemia

Phase 2 Trial of Menin Inhibitor BN104 as Post Hematopoietic Stem Cell Transplantation Maintenance in Patients With Acute Leukemia

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: The First Affiliated Hospital of Soochow University · Academic / Other
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 2, open label, single arm trial. This study aims to assess the efficacy and safety of menin inhibitor BN104 as maintenance therapy in patients with acute leukemia harboring specific genetic alterations who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT). Eligible patients will be screened at 30-180 days post allo-HSCT. Participants will take BN104 100-200mg orally, twice a day, 28 days a cycle for 24-36 cycles. The primary endpoint is 2-year relapse-free survival rate since enrollment. The secondary endpoints included overall survival, event-free survival, cumulative incidence of relapse, non relpase related mortality and safety.

Detailed description

AML with KMT2A rearrangements are grouped as a high risk subtype. Allogenic hematopoietic stem cell transplantation is the only curative strategy for this subtype. However, patients with KMT2A rearrangements have a high incidence of relapse rates. BN104 is a menin inhibitor. It has shown promising remission rates in patients with relpased or refractory patients haboring specific fusions including KMT2A rearrangements. The role of BN104 in post-HSCT maintenance therapy remains unknown. This is a phase 2, open label, single arm trial. This study aims to assess the efficacy and safety of menin inhibitor BN104 as maintenance therapy in patients with acute leukemia harboring specific genetic alterations who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT). Eligible patients will be screened at 30-180 days post allo-HSCT. Participants will take BN104 100-200mg orally, twice a day, 28 days a cycle for 24-36 cycles. The primary endpoint is 2-year relapse-free survival rate since enrollment. The secondary endpoints included overall survival, event-free survival, cumulative incidence of relapse, non relpase related mortality, incidence of acute and chronic graft versus host diseas, and safety.

Conditions

• Menin Inhibitors
• Post Hematopoietic Stem Cell Transplantation
• Maintenance Therapy
• Acute Leukemia
• KMT2A Rearrangement
• NPM1 Mutation
• NUP98 Gene Rearrangement

Interventions

Type	Name	Description
DRUG	BN104 monotherapy	BN104 will be started at 200 mg or 300mg, to a target dose of 400mg, twice daily, orally, 28 days a cycle, for a total of 24 to 36 cycles . When used in combination with posaconazoles and voriconazole, the dose of BN104 should be reduced by 50%.

Timeline

• Start date: 2025-08-01
• Primary completion: 2027-12-31
• Completion: 2029-12-30
• First posted: 2025-08-03
• Last updated: 2025-08-03

Locations

1 site across 1 country: China