NCT07437170

Selinexor Combined With Induction Chemotherapy for Children With NUP98-positive AML

Clinical Study of Selinexor Combined With ZJCH-AML Induction Chemotherapy for the Treatment of NUP98-positive Pediatric Acute Myeloid Leukemia

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: The Children's Hospital of Zhejiang University School of Medicine · Academic / Other
• Sex: All
• Age: 1 Year – 18 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective, single-arm, open-label clinical study to evaluate the efficacy and safety of Selinexor combined with a standardized induction chemotherapy regimen (ZJCH-AML) for children with NUP98-fusion positive Acute Myeloid Leukemia (AML). NUP98-fusion positive AML is a high-risk subtype in pediatric patients, characterized by poor prognosis and high relapse rates under traditional chemotherapy. Preliminary research suggests that Selinexor, an XPO1 inhibitor, can selectively inhibit NUP98-driven leukemia cells. This study aims to determine if adding Selinexor to standardized chemotherapy can increase the complete remission rate and improve the overall survival for these specific high-risk patients.

Conditions

• Acute Myeloid Leukemia
• Pediatric Acute Myeloid Leukemia
• NUP98 Fusion-positive Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	Selinexor	Induction Course 1: 40mg/m² orally on Days 1, 3, 8, and 10. Induction Course 2: 40mg/m² orally on Days 1 and 3.
DRUG	ZJCH-AML Standardized Chemotherapy	Course 1 (HAG): Homoharringtonine (HHT) 2mg/m²/d, Cytarabine (Ara-C) 10mg/m² q12h, and G-CSF 200μg/m²/d for 14 days. Course 2 (Risk-stratified): Based on the response to Course 1, patients receive HDA (for CR), DAC+HDA (for PR), or CLAG (for NR).

Timeline

• Start date: 2024-01-01
• Primary completion: 2027-12-31
• Completion: 2027-12-31
• First posted: 2026-02-27
• Last updated: 2026-02-27

Locations

1 site across 1 country: China