Acute Myeloid Leukaemia (AML)Phase 1/2TP53NCT07779031
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
A Phase 1/2 study testing Azacitidine (AZA) Days 1 - 5 and Selinexor in people with Acute Myeloid Leukaemia (AML) and MDS (Myelodysplastic Syndrome), run by Liping Dou at 1 site in China.
Trial summary
To explore the safety and efficacy of selinexor combined with azacitidine as post-transplant maintenance therapy for TP53-mutated AML/MDS. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Acute Myeloid Leukaemia (AML), MDS (Myelodysplastic Syndrome)
- What is being tested?
- Azacitidine (AZA) Days 1 - 5 and Selinexor
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 58 participants
- Where
- 1 siteChina
- Study dates
- 1 May 2025to 1 May 2028
- Phase
- Phase 1/2
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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).
Who cannot join
- (1) Patients with known allergy or contraindications to the investigational drugs.
- Pregnant or breastfeeding female patients.
- Presence of uncontrolled active infection or active graft-versus-host disease (GVHD).
- Patients with long-term smoking or heavy alcohol consumption that may interfere with the evaluation of trial results.
- Patients with psychiatric disorders or other conditions that preclude obtaining informed consent, or who are unable to comply with treatment and examination procedures.
- Patients who have undergone major organ surgery within less than 6 weeks prior to enrollment.
- Abnormal liver function: ALT and AST > 2.5 × upper limit of normal (ULN), or bilirubin > 2 × ULN; abnormal renal function: serum creatinine > ULN.
- Patients deemed unsuitable for this clinical trial by the investigator (e.g., poor compliance, drug abuse, etc.).
Trial locations and contacts
1 site in 1 country.
About this study
To explore the safety and efficacy of selinexor combined with azacitidine as post-transplant maintenance therapy for TP53-mutated AML/MDS.
Read the detailed description
This study focuses on AML/MDS patients with TP53 mutations who are at high risk of relapse. Following allogeneic hematopoietic stem cell transplantation, low-dose azacitidine combined with selinexor is administered as maintenance therapy. The objective is to evaluate the safety and tolerability of the combination of azacitidine and selinexor as post-transplant maintenance treatment. The primary endpoints are post-transplant relapse rate and non-relapse mortality. The secondary endpoints are overall survival and non-relapse mortality. Previous studies have reported that azacitidine and selinexor are safe and effective as post-transplant maintenance therapy for AML/MDS. In this study, the two-drug combination is administered post-transplant with the aim of reducing the risk of relapse and prolonging disease-free survival.
- Being tested
- Azacitidine (AZA) Days 1 - 5, Selinexor
- Medical subject headings
- Leukemia, Myeloid, Acute, Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Azacitidine (AZA) Days 1 - 5Drug
azacitidine 35mg/m2
SelinexorDrug
selinexor 20mg q2w
| Study group | Role | What they receive |
|---|---|---|
| azacitidine combined with selinexor | Experimental | azacitidine 35mg/m2,d1-5, selinexor Receives: Drug: Azacitidine (AZA) Days 1 - 5; Drug: Selinexor |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1 year
Secondary outcomes (3)
overall survival (OS)
Overall survival (OS) refers to the time from the start of treatment to the death of the patient for any reason.
Measured: 1 year
event free survival (EFS)
Event Free Survival (EFS) is a commonly used endpoint indicator in clinical trials to evaluate the survival time of patients without any adverse events during a specific time period. These adverse events include but are not limited to disease progression, death, treatment plan changes, and the occurrence of serious side effects
Measured: 1 year
Cumulative Incidence of Relapse, CIR
Cumulative incidence of relapse (CIR) is the estimated probability of disease recurrence over time, calculated using a competing-risk model that accounts for non-relapse mortality as a competing event rather than censoring it.
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 posted21 Aug 2026
- Study start1 May 2025
- Primary completion30 Apr 2028 (estimated)
- Study completion1 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukaemia (AML)Studies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 58 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Liping Dou
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Liping Dou, Chinese PLA General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07779031
- Sponsor's protocol ID
- S2025-055-01-tp53
- Acronym
- TP53
- Status last verified
- Jun 2026
- First submitted
- 21 Jun 2026
- Last updated
- 21 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07779031
- Acronym / sponsor ID
- TP53 / S2025-055-01-tp53
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 58 participants (estimated)
- Lead sponsor
- Liping DouUniversity, hospital or other
- Study start
- 1 May 2025 (actual)
- Primary completion
- 30 Apr 2028 (estimated)
- Study completion
- 1 May 2028 (estimated)
- First posted
- 21 Aug 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.
To explore the safety and efficacy of selinexor combined with azacitidine as post-transplant maintenance therapy for TP53-mutated AML/MDS.
Detailed description
This study focuses on AML/MDS patients with TP53 mutations who are at high risk of relapse. Following allogeneic hematopoietic stem cell transplantation, low-dose azacitidine combined with selinexor is administered as maintenance therapy. The objective is to evaluate the safety and tolerability of the combination of azacitidine and selinexor as post-transplant maintenance treatment. The primary endpoints are post-transplant relapse rate and non-relapse mortality. The secondary endpoints are overall survival and non-relapse mortality. Previous studies have reported that azacitidine and selinexor are safe and effective as post-transplant maintenance therapy for AML/MDS. In this study, the two-drug combination is administered post-transplant with the aim of reducing the risk of relapse and prolonging disease-free survival.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| azacitidine combined with selinexor | Experimental | Drug: Azacitidine (AZA) Days 1 - 5, Drug: Selinexorazacitidine 35mg/m2,d1-5, selinexor |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (8 listed)
- (1) Patients with known allergy or contraindications to the investigational drugs.
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 |
|---|---|---|
| Azacitidine (AZA) Days 1 - 5azacitidine 35mg/m2 | Drug | azacitidine combined with selinexor |
| Selinexorselinexor 20mg q2w | Drug | azacitidine combined with selinexor |
Primary outcome
relapse free survival
Time frame: 1 year
Relapse free survival (RFS) refers to the time from treatment to the first disease progression or death of the patient for any reason.
3 secondary outcomes
overall survival (OS)
Time frame: 1 year
event free survival (EFS)
Time frame: 1 year
Cumulative Incidence of Relapse, CIR
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 21 Aug 2026.
- Individual participant dataSharing planned: No
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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 1/2 study testing Azacitidine (AZA) Days 1 - 5 and Selinexor in people with Acute Myeloid Leukaemia (AML) and MDS (Myelodysplastic Syndrome), run by Liping Dou 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 11 inclusion and 8 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 21 Aug 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
relapse free survival (time frame: 1 year). 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 21 Aug 2026.