MDSPhase 2NCT06765928
Selinexor Combined With Venetoclax Maintenance Therapy After Allo-HSCT
A Phase 2 study testing selinexor in combination with venetoclax in people with MDS and AML, Adult, run by Ruijin Hospital at 1 site in China.
Trial summary
A multicenter, single-arm clinical study of evaluate the efficacy and safety of selinexor combined with venetoclax as maintenance therapy following allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia patients. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- MDS, AML, Adult
- What is being tested?
- selinexor in combination with venetoclax
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 73 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2025to 1 Jan 2028
- 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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- de novo AML in the ELN2022 high-risk group or MDS in the IPSS-M intermediate-high/high/very-high risk group
- Age 18-75 years old, gender is not limited
- First hematopoietic stem cell transplant with at least one eligible donor
- ECOG physical status score of 0-2
- The subject has received an allogeneic hematopoietic stem cell transplant within 90-120 days and STR-PCR shows complete donor chimerism;
- Have appropriate organ function, and laboratory results within 7 days prior to the start of trial treatment need to meet the following criteria:
Show all 9Show fewer
- AST and ALT) ≤ 3x ULN; Total serum bilirubin ≤ 1.5x ULN unless the patient has Gilbert syndrome; patients with Gilbert-Meulengracht syndrome with bilirubin ≤ 3.0 times the upper limit of normal and direct bilirubin ≤ 1.5 times the upper limit of normal may be included; HB ≥ 70 g/L (had not received a red blood cell transfusion within 1 week prior to administration); ANC ≥ 0.8 x 10^9/L (had not received long-acting colony-stimulating factor (LACSF) within 1 week prior to administration and short-acting colony-stimulating factor (SACSF) within 3 days prior to administration); Platelet count ≥ 20 x 10^9/L (had not received a platelet transfusion within 1 week prior to administration); serum creatinine ≤ 1.5x ULN or creatinine clearance ≥ 60 mL/min; Coagulation function: International Normalized Ratio (INR) ≤1.5×ULN, Activated Partial Thromboplastin Time (APTT) ≤1.5×ULN; Left ventricular ejection fraction (LVEF) ≥45%;
- Life expectancy ≥ 12 weeks;
- Voluntarily sign the informed consent form and understand and comply with the requirements of the study.
Who cannot join
- bone marrow examination after allo-HSCT suggestive of relapse or measurable residual disease (MRD) before the initiation of maintenance therapy;
- Other malignant tumors within 5 years prior to screening, except adequately treated carcinoma in situ of the cervix, basal cell or squamous epithelial cell skin cancers, post-radical thyroid cancer, and post-radical ductal carcinoma in situ;
- Current active cardiovascular disease of clinical significance, such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease as determined by the New York Heart Association (NYHA) functional class, or a history of myocardial infarction within the 6 months prior to screening;
- Other serious medical conditions that may limit the patient's participation in this trial (e.g., active infection, uncontrolled diabetes);
- Known HIV infection, or chronic infection with hepatitis B virus (HBsAg-positive) or hepatitis C virus (anti-HCV-positive) that cannot be controlled by medications;
- Patients with other tumors in combination, not cured;
Show all 12Show fewer
- Patients with neurologic or psychiatric disorders; clinically significant active cerebrovascular disease (e.g., cerebral edema, posterior reversible encephalopathy syndrome);
- Those who are allergic to the test drug;
- Those who are unable to understand or comply with the study protocol or are unable to sign the informed consent form;
- Those who have received other maintenance drugs after hematopoietic stem cell transplantation or have the desire to receive other maintenance therapy;
- Participation in other clinical studies within 3 months prior to screening that have not interfered with the safety and efficacy of the study drug as assessed by the investigator is permitted for inclusion in the study, e.g., non-interventional observational studies;
- Patients who, in the investigator's judgment and/or clinical criteria, have contraindications to any of the study procedures or have other medical conditions that may place them at unacceptable risk.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Ruijin Hospital of Shanghai Jiaotong University
Shanghai, Shanghai Municipality
About this study
Official title: A Multicenter, Single-arm Study of the Efficacy and Safety of Selinexor Combined With Venetoclax as Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia/Myelodysplastic Syndrome
A multicenter, single-arm clinical study of evaluate the efficacy and safety of selinexor combined with venetoclax as maintenance therapy following allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia patients.
Read the detailed description
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a curative treatment for intermediate- to high-risk acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).However, there are still some patients who relapse after transplantation, resulting in treatment failure. Therefore, post-transplantation maintenance therapy is needed for AML/MDS patients with poor prognosis to further reduce recurrence and prolong survival. In in vitro studies, XPO1 inhibitor combined with venetoclax was found to have a promising anti-leukemic effect, and venetoclax increased ROS levels in the bone marrow microenvironment and improved the post-transplant immune microenvironment. Accordingly, we propose to carry out XPO1 inhibitor combined with venetoclax as maintenance therapy after allo-HSCT in patients with intermediate- to high-risk AML/MDS.
There is a lack of prospective, controlled studies to clarify the efficacy and safety of XPO1 inhibitor combined with venetoclax as maintenance therapy after allo-HSCTin patients with intermediate- to high-risk AML/MDS, especially those with out specific gene mutation which would be targeted with commerically available inhibitors. Therefore, this multicenter, single-arm study is designed to assess the efficacy and safety of selinexor combined with venetoclax as maintenance therapy after intermediate to high-risk MDS/AML after allo-HSCT, with the aim of providing a reference for clinical treatment.
- Conditions
- MDS, AML, Adult
- Being tested
- selinexor in combination with venetoclax
- Keywords
- MDS/AML, allogeneic hematopoietic stem cell transplantation, maintenance therapy
- 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.
selinexor in combination with venetoclaxDrug
Also called XPO1 inhibitor combined with Bcl-2 inhibitor
After allo-HSCT, intermediate-high risk MDS/AML patients are maintenanced with selinexor given in combination with venetoclax for 2 years
| Study group | Role | What they receive |
|---|---|---|
| Experimental arm | Experimental | Post allo-HSCT maintenance with selinexor in combination with venetoclax for intermediate to high-risk MDS/AML Receives: Drug: selinexor in combination with venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progress-free survival
disease progression or death as a result of any causes
Measured: through study completion, an average of 2 year
Secondary outcomes (2)
Overall survival
death as a result of any causes
Measured: through study completion, an average of 2 year
Non-relapse survial
death without disease progression or relapse
Measured: through study completion, an average of 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2025
- Study start1 Jan 2025 (estimated)
- Primary completion1 Jan 2026 (estimated)
- Study completion1 Jan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MDSStudies of the same condition, open and under way.
- More trials run by Ruijin HospitalThe 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
- 73 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hu Xiaoxia, Ruijin Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06765928
- Sponsor's protocol ID
- RJBMT-010
- Status last verified
- Jan 2025
- First submitted
- 5 Jan 2025
- Last updated
- 9 Jan 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Single-arm Study of the Efficacy and Safety of Selinexor Combined With Venetoclax as Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia/Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT06765928
- Acronym / sponsor ID
- RJBMT-010
- Conditions
- MDS, AML, Adult
- Keywords
- MDS/AML, allogeneic hematopoietic stem cell transplantation, maintenance therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 73 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Study start
- 1 Jan 2025 (estimated)
- Primary completion
- 1 Jan 2026 (estimated)
- Study completion
- 1 Jan 2028 (estimated)
- First posted
- 9 Jan 2025 (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.
A multicenter, single-arm clinical study of evaluate the efficacy and safety of selinexor combined with venetoclax as maintenance therapy following allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia patients.
Detailed description
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a curative treatment for intermediate- to high-risk acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).However, there are still some patients who relapse after transplantation, resulting in treatment failure. Therefore, post-transplantation maintenance therapy is needed for AML/MDS patients with poor prognosis to further reduce recurrence and prolong survival. In in vitro studies, XPO1 inhibitor combined with venetoclax was found to have a promising anti-leukemic effect, and venetoclax increased ROS levels in the bone marrow microenvironment and improved the post-transplant immune microenvironment. Accordingly, we propose to carry out XPO1 inhibitor combined with venetoclax as maintenance therapy after allo-HSCT in patients with intermediate- to high-risk AML/MDS.
There is a lack of prospective, controlled studies to clarify the efficacy and safety of XPO1 inhibitor combined with venetoclax as maintenance therapy after allo-HSCTin patients with intermediate- to high-risk AML/MDS, especially those with out specific gene mutation which would be targeted with commerically available inhibitors. Therefore, this multicenter, single-arm study is designed to assess the efficacy and safety of selinexor combined with venetoclax as maintenance therapy after intermediate to high-risk MDS/AML after allo-HSCT, with the aim of providing a reference for clinical treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental arm | Experimental | Drug: selinexor in combination with venetoclaxPost allo-HSCT maintenance with selinexor in combination with venetoclax for intermediate to high-risk MDS/AML |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- de novo AML in the ELN2022 high-risk group or MDS in the IPSS-M intermediate-high/high/very-high risk group
- Age 18-75 years old, gender is not limited
- First hematopoietic stem cell transplant with at least one eligible donor
- ECOG physical status score of 0-2
- The subject has received an allogeneic hematopoietic stem cell transplant within 90-120 days and STR-PCR shows complete donor chimerism;
Key exclusion criteria (12 listed)
- bone marrow examination after allo-HSCT suggestive of relapse or measurable residual disease (MRD) before the initiation of maintenance therapy;
- Other malignant tumors within 5 years prior to screening, except adequately treated carcinoma in situ of the cervix, basal cell or squamous epithelial cell skin cancers, post-radical thyroid cancer, and post-radical ductal carcinoma in situ;
- Current active cardiovascular disease of clinical significance, such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease as determined by the New York Heart Association (NYHA) functional class, or a history of myocardial infarction within the 6 months prior to screening;
- Other serious medical conditions that may limit the patient's participation in this trial (e.g., active infection, uncontrolled diabetes);
- Known HIV infection, or chronic infection with hepatitis B virus (HBsAg-positive) or hepatitis C virus (anti-HCV-positive) that cannot be controlled by medications;
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 |
|---|---|---|
| selinexor in combination with venetoclaxAlso: XPO1 inhibitor combined with Bcl-2 inhibitorAfter allo-HSCT, intermediate-high risk MDS/AML patients are maintenanced with selinexor given in combination with venetoclax for 2 years | Drug | Experimental arm |
Primary outcome
Progress-free survival
Time frame: through study completion, an average of 2 year
disease progression or death as a result of any causes
2 secondary outcomes
Overall survival
Time frame: through study completion, an average of 2 year
Non-relapse survial
Time frame: through study completion, an average of 2 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 9 Jan 2025.
Related trials for MDS
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 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
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
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.
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 in combination with venetoclax in people with MDS and AML, Adult, run by Ruijin Hospital 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 9 inclusion and 12 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: opening soon, as of the sponsor's last update on 9 Jan 2025. 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?
Contact Xiaoxia HU, Doctor (02164370045, hxx12276@rjh.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progress-free survival (time frame: through study completion, an average of 2 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 9 Jan 2025.