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

February 22, 2026 updated by: The Children's Hospital of Zhejiang University School of Medicine

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Zhejiang
      • Hangzhou, Zhejiang, China, 310000
        • Children's Hospital, Zhejiang University School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

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

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Selinexor + ZJCH-AML
All enrolled patients receive Selinexor combined with the Zhejiang Children's Hospital (ZJCH) AML induction chemotherapy protocol.
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).

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete Remission (CR) Rate
Time Frame: Day 14 of Induction Course 2 (approximately 2 months).
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
Time Frame: Day 14 of Induction Course 1 (approximately 28 days).
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall Survival (OS)
Time Frame: 1 year.
The time from the date of enrollment to death from any cause.
1 year.
Event-Free Survival (EFS)
Time Frame: 1 year.
The time from the date of enrollment to events including treatment failure, relapse, or death.
1 year.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The Children's Hospital of Zhejiang University School of Medicine

Collaborators

College of Pharmaceutical Sciences at Zhejiang University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2024

Primary Completion (Estimated)

December 31, 2027

Study Completion (Estimated)

December 31, 2027

Study Registration Dates

First Submitted

February 22, 2026

First Submitted That Met QC Criteria

February 22, 2026

First Posted (Actual)

February 27, 2026

Study Record Updates

Last Update Posted (Actual)

February 27, 2026

Last Update Submitted That Met QC Criteria

February 22, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2025-IRB-0134-P-01

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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