Acute Myeloid Leukemia, NUP98 Fusion-positive Acute Myeloid Leukemia, Pediatric Acute Myeloid Leukemia
Conditions
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.
Interventions
DRUGSelinexor
Induction Course 1: 40mg/m² orally on Days 1, 3, 8, and 10. Induction Course 2: 40mg/m² orally on Days 1 and 3.
DRUGZJCH-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).
Sponsors
The Children's Hospital of Zhejiang University School of Medicine
College of Pharmaceutical Sciences at Zhejiang University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Complete Remission (CR) Rate | 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. |
| Minimal Residual Disease (MRD) Negative Rate | 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
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival (OS) | 1 year. | The time from the date of enrollment to death from any cause. |
| Event-Free Survival (EFS) | 1 year. | The time from the date of enrollment to events including treatment failure, relapse, or death. |
Countries
China
Outcome results
None listed