A Single-Arm, Open Phase I Clinical Study of Selinexor in Combination With Azacitidine for the Treatment of High-Risk AML/MDS Patients After Allo-HSCT

NCT07779031 | Active Not Recruiting Phase 1 DMC

• 1 other identifier: S2025-055-01-tp53
• Study Type: interventional
• Target: 58
• Locations: 1 country
• Sites: 1

Brief Summary

To explore the safety and efficacy of selinexor combined with azacitidine as post-transplant maintenance therapy for TP53-mutated AML/MDS.

Conditions

Acute Myeloid Leukaemia (AML); MDS (Myelodysplastic Syndrome)

Outcome Measures

Primary Outcomes (1)

• relapse free survival

  Relapse free survival (RFS) refers to the time from treatment to the first disease progression or death of the patient for any reason.

  1 year

Secondary Outcomes (3)

• overall survival (OS)

  1 year

• event free survival (EFS)

  1 year

• Cumulative Incidence of Relapse, CIR

  1 year

Study Arms (1)

azacitidine combined with selinexor

EXPERIMENTAL

azacitidine 35mg/m2,d1-5, selinexor

Drug: Azacitidine (AZA) Days 1 - 5; Drug: Selinexor

Interventions

Azacitidine (AZA) Days 1 - 5 DRUG

azacitidine 35mg/m2

azacitidine combined with selinexor

Selinexor DRUG

selinexor 20mg q2w

azacitidine combined with selinexor

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Voluntary participation in the clinical study: The subject or their legal guardian fully understands the study, provides informed consent by signing the Informed Consent Form (ICF), and is willing to comply with and complete all trial procedures.
• Age \< 75 years at screening, regardless of gender.
• Post-transplant MRD-negative acute myeloid leukemia / myelodysplastic syndrome with TP53 mutation.
• No history of severe allergic constitution.
• Liver function: ALT and AST ≤ 2.5 × upper limit of normal (ULN), and bilirubin ≤ 2 × ULN.
• Renal function: Serum creatinine ≤ ULN.
• No uncontrolled infection or severe psychiatric/psychological disorders.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-3, with an estimated life expectancy of ≥ 4 months.
• Bone marrow flow cytometry confirming MRD negativity prior to enrollment.
• Successful myeloid and platelet engraftment.
• No active graft-versus-host disease (GVHD).

You may not qualify if:

• (1) Patients with known allergy or contraindications to the investigational drugs.
• (2) Pregnant or breastfeeding female patients.
• (3) Presence of uncontrolled active infection or active graft-versus-host disease (GVHD).
• (4) Patients with long-term smoking or heavy alcohol consumption that may interfere with the evaluation of trial results.
• (5) Patients with psychiatric disorders or other conditions that preclude obtaining informed consent, or who are unable to comply with treatment and examination procedures.
• (6) Patients who have undergone major organ surgery within less than 6 weeks prior to enrollment.
• (7) Abnormal liver function: ALT and AST \> 2.5 × upper limit of normal (ULN), or bilirubin \> 2 × ULN; abnormal renal function: serum creatinine \> ULN.
• (8) Patients deemed unsuitable for this clinical trial by the investigator (e.g., poor compliance, drug abuse, etc.).

Sponsors & Collaborators

• Liping Dou: lead

Study Sites (1)

Chinese PLA General Hospital

Beijing, China

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Anemia, Refractory, with Excess of Blasts

Interventions

Azacitidine; selinexor

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Anemia, Refractory; Anemia; Myelodysplastic Syndromes; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Dr.

Study Record Dates

• First Submitted: June 21, 2026
• First Posted: August 21, 2026
• Study Start: May 1, 2025
• Primary Completion (Estimated): April 30, 2028
• Study Completion (Estimated): May 1, 2028
• Last Updated: August 21, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)