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NCT Number: NCT07437170

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

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.

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Key information

Conditions

Acute Myeloid Leukemia Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute NUP98 Fusion-positive Acute Myeloid Leukemia Neoplasms Neoplasms by Histologic Type Pediatric Acute Myeloid Leukemia

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310000, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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).

Primary outcomes

  1. 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.

  2. 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.

Secondary outcomes

  1. Overall Survival (OS)

    Time frame: 1 year.

    The time from the date of enrollment to death from any cause.

  2. Event-Free Survival (EFS)

    Time frame: 1 year.

    The time from the date of enrollment to events including treatment failure, relapse, or death.

Sponsors and collaborators

Lead sponsor

The Children's Hospital of Zhejiang University School of Medicine

Other

Collaborators

  • College of Pharmaceutical Sciences at Zhejiang University

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Feb 27, 2026
Registry last updated
Feb 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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