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NCT Number: NCT07094464

Maintenance Therapy With Selinexor and Azacitidine in TP53 Mutant AML/MDS After Transplantation

This study aims to explore the safety and efficacy of selinexor combined with azacitidine for maintenance therapy in TP53 mutant AML/MDS patients following transplantation.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

TP53-mutated MDS and AML Allogeneic Hematopoietic Stem Cell Transplantation Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Neoplasms Neoplasms by Histologic Type

Age range

14 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Chinese PLA General Hospital

Beijing, Beijing Municipality, 100853, China

About this study

This research targets high-risk recurrence patients with TP53 mutations in AML/MDS, administering low-dose azacitidine combined with selinexor for maintenance treatment post-allo-HCT (Allogeneic Hematopoietic Cell Transplantation). The goal is to observe the safety and tolerability of this drug combination as maintenance therapy post-transplant. The primary outcome measure will be post-transplant recurrence rate and non-relapse survival rate, while secondary outcomes include overall survival and non-relapse mortality. Previous studies have indicated that azacitidine and selinexor are safe and effective in maintenance therapy for AML/MDS. This study aims to reduce the risk of recurrence and prolong disease-free survival.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • (1) Voluntary participation in the clinical study: Participant or legal guardian understands and signs the Informed Consent Form (ICF) and is willing to comply with all trial procedures.

(2) Age below 75 years at screening; gender not restricted. (3) Diagnosed with AML or MDS with TP53 mutation (VAF ≥ 2%) and post-allo-HCT. (4) No severe allergic constitution. (5) Liver function: ALT and AST ≤ 2.5 times the upper limit of normal, bilirubin ≤ 2 times the upper limit of normal.

(6) Renal function: creatinine ≤ upper limit of normal. (7) No uncontrolled infections or severe psychiatric disorders. (8) ECOG performance score of 0-3; life expectancy of 4 months or more. (9) Peripheral blood counts for granulocytes and platelets.

Exclusion criteria

  • (1) Patients with known allergies or contraindications to the study drugs. (2) Pregnant or breastfeeding women. (3) Patients with uncontrolled active infections or active GVHD. (4) Patients with a long history of smoking or alcohol abuse affecting trial outcome evaluation.

(5) Patients with psychiatric disorders or conditions preventing informed consent, unable to comply with treatment and assessment requirements.

(6) Patients who underwent major surgery on important organs less than 6 weeks prior.

(7) Abnormal liver function: ALT or AST > 2.5 times the upper limit of normal; bilirubin > 2 times the upper limit of normal; renal function: creatinine > upper limit of normal.

(8) Patients deemed unsuitable for this clinical trial by the investigator (e.g., poor compliance, substance abuse).

Treatment and study plan

Maintenance Therapy with Selinexor and Azacitidine

Drug
  • Selinexor: Oral administration in 4 cycles:
  • Dose Escalation Phase: Cohort 1: Selinexor, 20 mg, twice a week for 2 weeks Cohort 2: Selinexor, 40 mg, twice a week for 2 weeks Cohort 3: Selinexor, 60 mg, twice a week for 2 weeks
  • Dose Expansion: MTD/RP2D will be determined based on safety.
  • Azacitidine: 35 mg/m² for 5 days.

Primary outcomes

  1. Disease relapse

    Time frame: 1 year after transplantation

    The definition of disease relapse: After remission, patients present with one of the following three conditions: (1) ≥5% of primary lymphocytes and immature lymphocytes in the bone marrow; (2) Extramedullary leukemia occurs; (3) Leukemia cells were found in peripheral blood smears. The definition of MRD recurrence: Leukemia cells are detected by flow cytometry or molecular biology.

Secondary outcomes

  1. Non-relapse mortality (NRM)

    Time frame: 1 year after transplantation

    From the first day after stem cell infusion to the last follow-up time, the number of NRM patients/the total number of cases ×100%. Death not caused by recurrence in the CR state.

  2. Overall survival (OS)

    Time frame: 1 year after transplantation

    The time from transplantation to death of any cause or the last follow-up day.

  3. Progress-free survival (PFS)

    Time frame: 1 year after transplantation

    The time from the date of transplantation to the recurrence of leukemia or death for any reason.

  4. Drug-related adverse events

    Time frame: 1 month after the last maintenance treatment ends

    All adverse events with a causal relationship to the study intervention (Selinexor + Azacitidine), assessed by investigators according to CTCAE v5.0 criteria.

  5. Measurable Residual Disease (MRD) in bone marrow

    Time frame: 1 year after transplantation

    Quantitative assessment of residual leukemic cells using: FCM-MRD and TP53 mutation NGS testing

Sponsors and collaborators

Lead sponsor

Daihong Liu

Other

Registry information

Official study title

A Single-Arm, Open-Label Phase I Clinical Study of Selinexor Combined With Azacitidine for Maintenance Therapy in TP53 Mutant AML/MDS Patients Post-Transplant

Important dates

Study start
2025
Primary completion
2027
Study completion
2028
First posted
Jul 30, 2025
Registry last updated
Jul 30, 2025

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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