Acute Myeloid LeukemiaPhase 2NCT07437170
Selinexor Combined With Induction Chemotherapy for Children With NUP98-positive AML
A Phase 2 study testing Selinexor and ZJCH-AML Standardized Chemotherapy in people with Acute Myeloid Leukemia and Pediatric Acute Myeloid Leukemia, run by The Children's Hospital of Zhejiang University School of Medicine at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Acute Myeloid Leukemia, Pediatric Acute Myeloid Leukemia, NUP98 Fusion-positive Acute Myeloid Leukemia
- What is being tested?
- Selinexor and ZJCH-AML Standardized Chemotherapy
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2024to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed with Acute Myeloid Leukemia (AML).
- Confirmed NUP98 fusion gene positivity.
- Pediatric patients.
Who cannot join
- Pediatric patients with AML who did not receive induction chemotherapy according to the Zhejiang Province pediatric AML protocol.
Trial locations and contacts
1 site in 1 country.
China1 site
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310000
About this study
Official title: 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.
- Conditions
- Acute Myeloid Leukemia, Pediatric Acute Myeloid Leukemia, NUP98 Fusion-positive Acute Myeloid Leukemia
- Being tested
- Selinexor, ZJCH-AML Standardized Chemotherapy
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SelinexorDrug
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 ChemotherapyDrug
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 group | Role | What they receive |
|---|---|---|
| Selinexor + ZJCH-AML | Experimental | All enrolled patients receive Selinexor combined with the Zhejiang Children's Hospital (ZJCH) AML induction chemotherapy protocol. Receives: Drug: Selinexor; Drug: ZJCH-AML Standardized Chemotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Day 14 of Induction Course 1 (approximately 28 days).
Secondary outcomes (2)
Overall Survival (OS)
The time from the date of enrollment to death from any cause.
Measured: 1 year.
Event-Free Survival (EFS)
The time from the date of enrollment to events including treatment failure, relapse, or death.
Measured: 1 year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2026
- Study start1 Jan 2024
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by The Children's Hospital of Zhejiang University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- College of Pharmaceutical Sciences at Zhejiang University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07437170
- Sponsor's protocol ID
- 2025-IRB-0134-P-01
- Status last verified
- Feb 2026
- First submitted
- 22 Feb 2026
- Last updated
- 27 Feb 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of Selinexor Combined With ZJCH-AML Induction Chemotherapy for the Treatment of NUP98-positive Pediatric Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT07437170
- Acronym / sponsor ID
- 2025-IRB-0134-P-01
- Conditions
- Acute Myeloid Leukemia, Pediatric Acute Myeloid Leukemia, NUP98 Fusion-positive Acute Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- The Children's Hospital of Zhejiang University School of MedicineUniversity, hospital or other
- Collaborators
- College of Pharmaceutical Sciences at Zhejiang University
- Study start
- 1 Jan 2024 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 27 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Selinexor + ZJCH-AML | Experimental | Drug: Selinexor, Drug: ZJCH-AML Standardized ChemotherapyAll enrolled patients receive Selinexor combined with the Zhejiang Children's Hospital (ZJCH) AML induction chemotherapy protocol. |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Diagnosed with Acute Myeloid Leukemia (AML).
- Confirmed NUP98 fusion gene positivity.
- Pediatric patients.
Key exclusion criteria (1 listed)
- Pediatric patients with AML who did not receive induction chemotherapy according to the Zhejiang Province pediatric AML protocol.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SelinexorInduction Course 1: 40mg/m² orally on Days 1, 3, 8, and 10. Induction Course 2: 40mg/m² orally on Days 1 and 3. | Drug | Selinexor + ZJCH-AML |
| ZJCH-AML Standardized ChemotherapyCourse 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). | Drug | Selinexor + ZJCH-AML |
Primary outcome
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.
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.
2 secondary outcomes
Overall Survival (OS)
Time frame: 1 year.
Event-Free Survival (EFS)
Time frame: 1 year.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2026.
Related trials for Acute Myeloid Leukemia
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingPhase 1/2
Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
Acute Biphenotypic Leukemia, Acute Myeloid Leukemia
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Selinexor and ZJCH-AML Standardized Chemotherapy in people with Acute Myeloid Leukemia and Pediatric Acute Myeloid Leukemia, run by The Children's Hospital of Zhejiang University School of Medicine at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 27 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete Remission (CR) Rate (time frame: Day 14 of Induction Course 2 (approximately 2 months).). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2026.