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Evaluating Selinexor for Myelofibrosis with Low Platelet Counts

A Phase 2 Study to Evaluate the Efficacy and Safety of Selinexor Monotherapy in Subjects With JAK Inhibitor-naïve Myelofibrosis and Moderate Thrombocytopenia

Phase 2 Interventional Karyopharm Therapeutics Inc · NCT05980806

This study is testing if a new drug called selinexor can help people with myelofibrosis and low platelet counts feel better and reduce their spleen size.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment58 (estimated)
Ages18 Years and up
SexAll
SponsorKaryopharm Therapeutics Inc Industry-sponsored
Drugs / interventionsradiation
Locations70 sites (Duarte, California and 69 other locations)
Trial IDNCT05980806 on ClinicalTrials.gov

What this trial studies

This study aims to assess the effectiveness of selinexor in patients with myelofibrosis who have not previously received JAK inhibitors and have moderate thrombocytopenia. Participants will be evaluated based on the reduction in spleen volume and other efficacy and safety parameters. The study will involve administering different doses of selinexor and monitoring the participants' responses over time. The goal is to provide insights into the potential benefits of selinexor for this specific patient population.

Who should consider this trial

Good fit: Ideal candidates include individuals diagnosed with myelofibrosis who have moderate thrombocytopenia and have not previously been treated with JAK inhibitors.

Not a fit: Patients with severe thrombocytopenia or those who have previously received JAK inhibitor therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could offer a new therapeutic option for patients with myelofibrosis and moderate thrombocytopenia.

How similar studies have performed: While there have been studies on myelofibrosis treatments, the specific use of selinexor in JAK inhibitor-naïve patients is a novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

* A diagnosis of MF or post-ET or post-PV MF according to the 2016 World Health Organization (WHO) classification of MPN, confirmed by the most recent local pathology report
* Measurable splenomegaly during the screening period as demonstrated by spleen volume of greater than or equal to (\>=) 450 cubic square centimeter (cm\^3) by MRI or CT scan (results from MRI or CT imaging performed within 28 days prior to C1D1 are acceptable)
* DIPSS risk category of intermediate-1 with symptoms, or intermediate-2, or high-risk
* ECOG Performance Status less than or equal to (\<=) 2
* Platelet count of greater than or equal to (\>=) 50 x 10\^9/L without platelet transfusion within 7 days prior to the first dose of selinexor
* Absolute neutrophil count (ANC) \>=1.0 × 10\^9/L without need for growth factors within 7 days prior to the first dose of selinexor
* Adequate liver function as defined by the following: aspartate transaminase (AST) and alanine transaminase (ALT) \<= 2.5 × upper limit normal (ULN) and serum total bilirubin \<= 3×ULN
* Calculated creatinine clearance (CrCl) greater than (\>) 15 milliliter per minute (mL/min) based on the Cockcroft and Gault formula
* Active symptoms of MF as determined by presence of at least 2 symptoms with an average score \>= 5 or total score of \>= 12 at screening (at least 5 of 7 consecutive days immediately preceding C1D1) using the MFSAF V4.0
* Must provide bone marrow biopsy samples (samples obtained up to 3 months prior to C1D1 are permitted) at screening and during the study
* Currently not eligible for stem cell transplantation
* Must be willing to complete the MFSAF V4.0 daily during the study for evaluating the symptom response (i.e., TSS50)

Key Exclusion Criteria:

* More than 10% blasts in peripheral blood or bone marrow (accelerated or blast phase)
* Previous treatment with JAK inhibitors for MF
* Previous treatment with selinexor or other XPO1 inhibitors
* Females who are pregnant or lactating
* Prior splenectomy, splenic radiation, or a splenic embolization within 6 months prior to C1D1
* History of myocardial infarction, unstable angina, percutaneous transluminal coronary angioplasty (PTCA), coronary artery bypass graft (CABG), cerebrovascular accident (transient ischemic attack \[TIA\]), ventricular arrhythmias, congestive heart failure class \> 2 per New York Heart Association (NYHA) within 6 months of C1D1
* Unable to tolerate two forms of antiemetics prior to each dose for the first two cycles

Where this trial is running

Duarte, California and 69 other locations

+20 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions MyelofibrosisModerate ThrombocytopeniaMild ThrombocytopeniaSelinexorTotal Symptom ScoreMyelofibrosis Symptom Assessment FormSpleen Volume ReductionTSS50
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.