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Combining Selinexor and Ruxolitinib for Myelofibrosis Treatment

A Phase 1/3 Study to Evaluate Efficacy and Safety of Selinexor, a Selective Inhibitor of Nuclear Export, in Combination with Ruxolitinib in Treatment-naïve Patients with Myelofibrosis

Phase 3 Interventional Karyopharm Therapeutics Inc · NCT04562389

This study is testing if a new combination of two medications, selinexor and ruxolitinib, can help people with myelofibrosis who haven't been treated with JAK inhibitors before feel better.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment350 (estimated)
Ages18 Years and up
SexAll
SponsorKaryopharm Therapeutics Inc Industry-sponsored
Drugs / interventionsruxolitinib
Locations149 sites (Birmingham, Alabama and 148 other locations)
Trial IDNCT04562389 on ClinicalTrials.gov

What this trial studies

This global, multicenter clinical trial evaluates the efficacy and safety of selinexor in combination with ruxolitinib for patients with myelofibrosis who have not previously received JAK inhibitor treatment. The study consists of two phases: an initial open-label phase to determine the recommended dose and an ongoing double-blind phase comparing the combination therapy to a placebo with ruxolitinib. Participants will be monitored for symptom relief and overall treatment response.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with a diagnosis of primary myelofibrosis or post-essential thrombocythemia/polycythemia vera myelofibrosis and active symptoms.

Not a fit: Patients with prior treatment using JAK inhibitors or selinexor, or those with advanced disease characterized by high blast counts, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new effective option for patients with myelofibrosis, potentially improving their symptoms and quality of life.

How similar studies have performed: While this approach combines established therapies, the specific combination of selinexor and ruxolitinib in this context is novel and has not been extensively tested in prior studies.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Aged ≥ 18 years
* A diagnosis of primary MF or post-essential thrombocythemia (ET) or postpolycythemia- vera (PV) MF.
* Active symptoms of MF as determined by presence of at least 2 symptoms using the Myelofibrosis Symptom Assessment Form (MFSAF) V4.0.
* Participants with international prognostic scoring system (DIPSS) risk category of intermediate-1, or intermediate-2, or high-risk.
* Measurable splenomegaly during the screening period as demonstrated by spleen volume of greater than or equal to (\>=) 450 cubic centimeter (cm\^3) .
* Eastern Cooperative Oncology Group (ECOG) Performance Status less than or equal to (\<=) 2.

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.

Where this trial is running

Birmingham, Alabama and 148 other locations

+99 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 MyelofibrosisSelinexorRuxolitinibJanus kinase 2Myeloproliferative neoplasms
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.