Myeloproliferative NeoplasmsPhase 2NCT07626021
Selinexor Monotherapy for Cytoreduction in BCR::ABL1-Negative Myeloproliferative Neoplasms
A Phase 2 study testing Selinexor in people with Myeloproliferative Neoplasms, run by Zhongshan Hospital (Xiamen), Fudan University.
Trial summary
Myeloproliferative neoplasms are chronic blood cancers in which the bone marrow produces too many blood cells. Patients with Philadelphia chromosome-negative myeloproliferative neoplasms, including polycythemia vera, essential thrombocythemia, and primary myelofibrosis, may need treatment to reduce high blood cell… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Myeloproliferative Neoplasms
- What is being tested?
- Selinexor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- No sites listed yet
- Study dates
- 1 Jul 2026to 31 Aug 2026
- 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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged 18 to 80 years at the time of informed consent.
- Diagnosis of Philadelphia chromosome-negative myeloproliferative neoplasm, including polycythemia vera, essential thrombocythemia, or primary myelofibrosis, according to World Health Organization criteria.
- Presence of an indication for cytoreductive therapy, including at least one of the following:
- Extreme thrombocytosis, defined as platelet count >1500 x 10^9/L.
- Progressive leukocytosis, defined as white blood cell count >25 x 10^9/L.
- Symptomatic splenomegaly documented by imaging.
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- Severe disease-related symptoms, such as significant weight loss within the past 6 months.
- Unwillingness or inability to tolerate standard cytoreductive therapies, such as hydroxyurea or interferon.
- Eastern Cooperative Oncology Group performance status of 0 to 2.
- Adequate organ function, including renal, hepatic, cardiac, and coagulation function, as determined by laboratory tests and clinical evaluation in the opinion of the investigator.
- Adequate baseline hematologic function without recent transfusion or growth factor support.
- Ability to comply with study procedures, visits, and assessments.
- Ability to understand and willingness to sign a written informed consent form.
- For women of childbearing potential, a negative pregnancy test before study entry.
- For participants of reproductive potential, agreement to use effective contraception during the study and for at least 120 days after the last dose of study drug.
Who cannot join
- Diagnosis of Philadelphia chromosome-positive myeloproliferative neoplasm or chronic myeloid leukemia.
- Concurrent acute leukemia or other active hematologic malignancy.
- Severe or uncontrolled comorbid condition, including but not limited to significant cardiac or pulmonary disease, decompensated liver disease, or end-stage renal disease.
- Active uncontrolled bacterial, viral, or fungal infection.
- Active gastrointestinal disorder associated with significant bleeding or impaired drug absorption.
- Significant neurologic or psychiatric disorder that may interfere with study participation or compliance.
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- Known hypersensitivity to selinexor or any of its components.
- Receipt of another investigational agent or participation in another interventional clinical trial within 4 weeks before enrollment.
- Recent receipt of cytotoxic chemotherapy, radiotherapy, immunotherapy, or other cytoreductive treatment before study entry.
- History of substance abuse, alcohol dependence, or illicit drug use that may interfere with adherence to study requirements.
- Another active malignancy requiring systemic treatment.
- Clinically significant bleeding disorder, severe coagulopathy, or requirement for long-term therapeutic anticoagulation.
- Receipt of a live vaccine within 30 days before the first dose of study drug, or planned receipt of a live vaccine during the study.
- Known active infection with human immunodeficiency virus or active viral hepatitis.
- Estimated life expectancy of less than 12 weeks.
- Inability to provide informed consent due to cognitive impairment or severe psychiatric illness.
- Any condition that, in the investigator's judgment, would make the participant unsuitable for participation in this study.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: An Open-Label, Single-Arm Phase II Study of Selinexor Monotherapy for Cytoreduction in Patients With BCR::ABL1-Negative Myeloproliferative Neoplasms
Myeloproliferative neoplasms are chronic blood cancers in which the bone marrow produces too many blood cells. Patients with Philadelphia chromosome-negative myeloproliferative neoplasms, including polycythemia vera, essential thrombocythemia, and primary myelofibrosis, may need treatment to reduce high blood cell counts, relieve disease-related symptoms, and lower the risk of complications. However, currently available cytoreductive treatments may be ineffective, poorly tolerated, or inconvenient for some patients.
Selinexor is an oral selective inhibitor of nuclear export that has shown antitumor activity in several hematologic malignancies. This study will evaluate the effectiveness and safety of selinexor used alone as cytoreductive treatment in patients with Philadelphia chromosome-negative myeloproliferative neoplasms who have an indication for cytoreductive therapy.
This is a prospective, single-arm, open-label phase II study conducted at a single center. Eligible participants will receive oral selinexor, with dose adjustments based on tolerability and blood cell counts. Participants will be followed for treatment response, symptom improvement, and side effects for up to 6 months. The results of this study may help determine whether selinexor could provide a potential treatment option for patients with Philadelphia chromosome-negative myeloproliferative neoplasms who have limited cytoreductive therapy choices.
Read the detailed description
This is a prospective, open-label, single-arm phase II study designed to evaluate the efficacy and safety of selinexor monotherapy as cytoreductive treatment in participants with Philadelphia chromosome-negative myeloproliferative neoplasms who have an indication for cytoreductive therapy.
Eligible participants will receive oral selinexor once weekly, starting at 40 mg. Dose escalation to 60 mg or 80 mg once weekly may be considered according to tolerability, hematologic response, and investigator judgment. Dose interruption or dose reduction will be permitted for hematologic or non-hematologic toxicities according to the protocol.
The primary objective is to evaluate the proportion of participants who achieve cytoreductive response based on disease-relevant peripheral blood count parameters, including platelet count, white blood cell count, and hematocrit where applicable. Secondary objectives include evaluation of symptom improvement, spleen-related response where applicable, safety, tolerability, and treatment discontinuation.
Participants will be followed for treatment response, adverse events, and overall clinical status for up to 6 months after treatment initiation. Safety assessments will include clinical evaluation, laboratory testing, and monitoring of treatment-emergent adverse events.
- Conditions
- Myeloproliferative Neoplasms
- Being tested
- Selinexor
- Medical subject headings
- Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SelinexorDrug
Also called KPT-330
Selinexor will be administered orally at an initial dose of 40 mg once weekly. Treatment may be continued for up to 3 months, with a planned total follow-up period of 6 months. Dose escalation to 60 mg or 80 mg once weekly is permitted in participants without significant hematologic or non-hematologic toxicity, according to the investigator's judgment. Dose interruption and dose reduction are allowed based on safety and tolerability. In the event of grade 3 or higher hematologic toxicity or clinically significant non-hematologic toxicity, selinexor will be temporarily withheld and resumed at a reduced dose after recovery. Treatment will be permanently discontinued if unacceptable toxicity persists despite dose modification.
| Study group | Role | What they receive |
|---|---|---|
| Selinexor Monotherapy | Experimental | Participants in this arm will receive oral selinexor monotherapy as cytoreductive treatment. Selinexor will be administered once weekly, with dose adjustment based on safety, tolerability, and hematologic response. Receives: Drug: Selinexor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Participants Achieving Disease-Relevant Peripheral Blood Count Control
Disease-relevant peripheral blood count control will be assessed using hematologic parameters, including platelet count, white blood cell count, and hematocrit where applicable. A participant will be considered to have achieved cytoreductive response if the elevated disease-relevant blood count parameter at baseline normalizes or shows a clinically meaningful reduction from baseline according to the study protocol.
Measured: Up to 6 months after initiation of study treatment
Secondary outcomes (2)
Incidence of Treatment-Emergent Adverse Events
Treatment-emergent adverse events will be assessed according to clinical evaluation and laboratory testing during the study period.
Measured: Up to 6 months after initiation of study treatment
Proportion of Participants With Dose Interruption, Dose Reduction, or Treatment Discontinuation Due to Adverse Events
Change From Baseline in Disease-Related Symptom Burden Assessed by the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score
Measured: Up to 6 months after initiation of study treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2026
- Study start1 Jul 2026 (estimated)
- Primary completion31 Aug 2026 (estimated)
- Study completion31 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myeloproliferative NeoplasmsStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhongshan Hospital (Xiamen), Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zheng Wei, MD, Zhongshan Hospital (Xiamen), Fudan University
- Investigators
- Zheng Wei, Principal investigator, Zhongshan Hospital (Xiamen), Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07626021
- Sponsor's protocol ID
- B2025-039R
- Status last verified
- Jul 2025
- First submitted
- 17 Jan 2026
- Last updated
- 4 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- 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 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Single-Arm Phase II Study of Selinexor Monotherapy for Cytoreduction in Patients With BCR::ABL1-Negative Myeloproliferative Neoplasms
- ClinicalTrials.gov ID
- NCT07626021
- Acronym / sponsor ID
- B2025-039R
- Conditions
- Myeloproliferative Neoplasms
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Zhongshan Hospital (Xiamen), Fudan UniversityUniversity, hospital or other
- Study start
- 1 Jul 2026 (estimated)
- Primary completion
- 31 Aug 2026 (estimated)
- Study completion
- 31 Aug 2026 (estimated)
- First posted
- 4 Jun 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.
Myeloproliferative neoplasms are chronic blood cancers in which the bone marrow produces too many blood cells. Patients with Philadelphia chromosome-negative myeloproliferative neoplasms, including polycythemia vera, essential thrombocythemia, and primary myelofibrosis, may need treatment to reduce high blood cell counts, relieve disease-related symptoms, and lower the risk of complications. However, currently available cytoreductive treatments may be ineffective, poorly tolerated, or inconvenient for some patients.
Selinexor is an oral selective inhibitor of nuclear export that has shown antitumor activity in several hematologic malignancies. This study will evaluate the effectiveness and safety of selinexor used alone as cytoreductive treatment in patients with Philadelphia chromosome-negative myeloproliferative neoplasms who have an indication for cytoreductive therapy.
This is a prospective, single-arm, open-label phase II study conducted at a single center. Eligible participants will receive oral selinexor, with dose adjustments based on tolerability and blood cell counts. Participants will be followed for treatment response, symptom improvement, and side effects for up to 6 months. The results of this study may help determine whether selinexor could provide a potential treatment option for patients with Philadelphia chromosome-negative myeloproliferative neoplasms who have limited cytoreductive therapy choices.
Detailed description
This is a prospective, open-label, single-arm phase II study designed to evaluate the efficacy and safety of selinexor monotherapy as cytoreductive treatment in participants with Philadelphia chromosome-negative myeloproliferative neoplasms who have an indication for cytoreductive therapy.
Eligible participants will receive oral selinexor once weekly, starting at 40 mg. Dose escalation to 60 mg or 80 mg once weekly may be considered according to tolerability, hematologic response, and investigator judgment. Dose interruption or dose reduction will be permitted for hematologic or non-hematologic toxicities according to the protocol.
The primary objective is to evaluate the proportion of participants who achieve cytoreductive response based on disease-relevant peripheral blood count parameters, including platelet count, white blood cell count, and hematocrit where applicable. Secondary objectives include evaluation of symptom improvement, spleen-related response where applicable, safety, tolerability, and treatment discontinuation.
Participants will be followed for treatment response, adverse events, and overall clinical status for up to 6 months after treatment initiation. Safety assessments will include clinical evaluation, laboratory testing, and monitoring of treatment-emergent adverse events.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Selinexor Monotherapy | Experimental | Drug: SelinexorParticipants in this arm will receive oral selinexor monotherapy as cytoreductive treatment. Selinexor will be administered once weekly, with dose adjustment based on safety, tolerability, and hematologic response. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Aged 18 to 80 years at the time of informed consent.
- Diagnosis of Philadelphia chromosome-negative myeloproliferative neoplasm, including polycythemia vera, essential thrombocythemia, or primary myelofibrosis, according to World Health Organization criteria.
- Presence of an indication for cytoreductive therapy, including at least one of the following:
- Extreme thrombocytosis, defined as platelet count >1500 x 10^9/L.
- Progressive leukocytosis, defined as white blood cell count >25 x 10^9/L.
Key exclusion criteria (17 listed)
- Diagnosis of Philadelphia chromosome-positive myeloproliferative neoplasm or chronic myeloid leukemia.
- Concurrent acute leukemia or other active hematologic malignancy.
- Severe or uncontrolled comorbid condition, including but not limited to significant cardiac or pulmonary disease, decompensated liver disease, or end-stage renal disease.
- Active uncontrolled bacterial, viral, or fungal infection.
- Active gastrointestinal disorder associated with significant bleeding or impaired drug absorption.
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 |
|---|---|---|
| SelinexorAlso: KPT-330Selinexor will be administered orally at an initial dose of 40 mg once weekly. Treatment may be continued for up to 3 months, with a planned total follow-up period of 6 months. Dose escalation to 60 mg or 80 mg once weekly is permitted in participants without significant hematologic or non-hematologic toxicity, according to the investigator's judgment. Dose interruption and dose reduction are allowed based on safety and tolerability. In the event of grade 3 or higher hematologic toxicity or clinically significant non-hematologic toxicity, selinexor will be temporarily withheld and resumed at a reduced dose after recovery. Treatment will be permanently discontinued if unacceptable toxicity persists despite dose modification. | Drug | Selinexor Monotherapy |
Primary outcome
Proportion of Participants Achieving Disease-Relevant Peripheral Blood Count Control
Time frame: Up to 6 months after initiation of study treatment
Disease-relevant peripheral blood count control will be assessed using hematologic parameters, including platelet count, white blood cell count, and hematocrit where applicable. A participant will be considered to have achieved cytoreductive response if the elevated disease-relevant blood count parameter at baseline normalizes or shows a clinically meaningful reduction from baseline according to the study protocol.
2 secondary outcomes
Incidence of Treatment-Emergent Adverse Events
Time frame: Up to 6 months after initiation of study treatment
Proportion of Participants With Dose Interruption, Dose Reduction, or Treatment Discontinuation Due to Adverse Events
Time frame: Up to 6 months after initiation of study treatment
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 4 Jun 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.
Zheng Wei principal investigator
Zhongshan Hospital (Xiamen), Fudan University
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Selinexor in people with Myeloproliferative Neoplasms, run by Zhongshan Hospital (Xiamen), Fudan University.
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 15 inclusion and 17 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 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
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 Zheng Wei (+86-13916563166, wei.zheng@zs-hospital.sh.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Participants Achieving Disease-Relevant Peripheral Blood Count Control (time frame: Up to 6 months after initiation of study treatment). 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 4 Jun 2026.