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

Sponsor
The Children's Hospital of Zhejiang University School of Medicine
Study ID
NCT07437170
Phase
PHASE2
Status
Active Not Recruiting

Conditions

Eligibility Criteria

Sex
ALL
Age
1 Year - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • Selinexor — DRUG
    Induction Course 1: 40mg/m² orally on Days 1, 3, 8, and 10. Induction Course 2: 40mg/m² orally on Days 1 and 3.
  • ZJCH-AML Standardized Chemotherapy — DRUG
    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).

Study Details

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.

Key Dates

Start date
Jan 1, 2024
Status verified
Feb 2026
Primary completion
Dec 31, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
10 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Selinexor + ZJCH-AML
    All enrolled patients receive Selinexor combined with the Zhejiang Children's Hospital (ZJCH) AML induction chemotherapy protocol.

Primary Outcome Measure

Complete Remission (CR) Rate [ Time Frame: Day 14 of Induction Course 2 (approximately 2 months). ]

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