Selinexor Monotherapy for Cytoreduction in BCR::ABL1-Negative Myeloproliferative Neoplasms

NCT07626021 | Not Yet Recruiting Phase 2

• 1 other identifier: B2025-039R
• Study Type: interventional
• Target: 15
• Locations: 0 countries
• Sites: N/A

Brief 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 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.

Conditions

Myeloproliferative Neoplasms

Outcome Measures

Primary Outcomes (1)

• 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.

  Up to 6 months after initiation of study treatment

Secondary Outcomes (2)

• Incidence of Treatment-Emergent Adverse Events

  Up to 6 months after initiation of study treatment

• Proportion of Participants With Dose Interruption, Dose Reduction, or Treatment Discontinuation Due to Adverse Events

  Up to 6 months after initiation of study treatment

Study Arms (1)

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.

Drug: Selinexor

Interventions

Selinexor DRUG

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.

Also known as: KPT-330

Selinexor Monotherapy

Eligibility Criteria

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

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Zhongshan Hospital (Xiamen), Fudan University: lead

MeSH Terms

Conditions

Myeloproliferative Disorders

Interventions

selinexor

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Zheng Wei

  Zhongshan Hospital (Xiamen), Fudan University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Zheng Wei

CONTACT

+86-13916563166; wei.zheng@zs-hospital.sh.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Chief Physician

Study Record Dates

• First Submitted: January 17, 2026
• First Posted: June 4, 2026
• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): August 31, 2026
• Study Completion (Estimated): August 31, 2026
• Last Updated: June 4, 2026

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share