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

NCT07437170 | Active Not Recruiting Phase 2

• 1 other identifier: 2025-IRB-0134-P-01
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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

Outcome Measures

Primary Outcomes (2)

• Complete Remission (CR) Rate

  Percentage of patients achieving CR after Induction Course 1 or 2. CR is defined as bone marrow blasts \<5%, ANC \> 1\\times10\^9/L, and PLT \> 80\\times10\^9/L.

  Day 14 of Induction Course 2 (approximately 2 months).

• Minimal Residual Disease (MRD) Negative Rate

  The proportion of patients reaching MRD negativity (threshold \< 0.1%) measured by flow cytometry or molecular methods.

  Day 14 of Induction Course 1 (approximately 28 days).

Secondary Outcomes (2)

• Overall Survival (OS)

  1 year.

• Event-Free Survival (EFS)

  1 year.

Study Arms (1)

Selinexor + ZJCH-AML

EXPERIMENTAL

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

Drug: Selinexor; Drug: ZJCH-AML Standardized Chemotherapy

Interventions

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).

Selinexor + ZJCH-AML

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.

Selinexor + ZJCH-AML

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosed with Acute Myeloid Leukemia (AML).
• Confirmed NUP98 fusion gene positivity.
• Pediatric patients.

You may not qualify if:

• Pediatric patients with AML who did not receive induction chemotherapy according to the Zhejiang Province pediatric AML protocol.

Sponsors & Collaborators

• The Children's Hospital of Zhejiang University School of Medicine: lead
• College of Pharmaceutical Sciences at Zhejiang University: collaborator

Study Sites (1)

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310000, China

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

selinexor

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2026
• First Posted: February 27, 2026
• Study Start: January 1, 2024
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: February 27, 2026

Record last verified: 2026-02

Locations

CN (1)