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Joint Clinical Trials Office

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

Clinical Trial Details

The main purpose of this study with the corresponding optional expansion is to evaluate the efficacy of selinexor in JAKi-naïve participants with myelofibrosis (MF) and moderate thrombocytopenia based on spleen volume reduction (SVR). Additional efficacy and safety parameters will also be assessed during the study. The purpose of this research study is to see if Selinexor is an effective treatment for MF.

Selinexor is an experimental drug that is not approved by the U.S. Food and Drug Administration (FDA) for the treatment of MF. Selinexor  is an oral drug.
   
Participants will be assigned to one of two groups. One group will receive Selinexor 40mg and the other will receive Selinexor 60mg. Dosing and dosing schedules will be discussed with the study team.

During Cycles 1 and 2, participants will need to visit the research center every 2 weeks. From Cycle 3 onwards, they will visit the research center every 4 weeks on the first day of each cycle. These study visits may take approximately 2-3 hours. There will also be a follow-up period after completing treatment where participants will receive a phone call to discuss how they're feeling and answer other questions from the study team.

Total study participation will last approximately 3 years. 

Key Eligibility: 

 Key Inclusion Criteria:

  1. A diagnosis of myelofibrosis (MF) or post-essential thrombocythemia (ET) or post-polycythemia vera (PV) 
  2. Measurable splenomegaly during the screening period as demonstrated by spleen volume of greater than or equal to 450 cubic square centimeters by MRI or CT scan 
  3. Participants with DIPSS risk category of intermediate-1 with symptoms, or intermediate-2, or high-risk.
  4. ECOG Performance Status less than or equal to 2
  5. Platelet count of 50 to less than 100 x 10^9/L without platelet transfusion within 7 days prior to the first dose of selinexor.

   
Key Exclusion Criteria:

  1. More than 10% blasts in peripheral blood or bone marrow (accelerated or blast phase).
  2. Previous treatment with JAK inhibitors for MF.
  3. Previous treatment with selinexor or other XPO1 inhibitors.
  4. Female participants who are pregnant or lactating.
  5. Prior splenectomy, or splenic radiation within 6 months prior to C1D1.

 Detailed eligibility will be discussed when reaching out to the study team.

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Study contact by location

Upper East Side - Manhattan

Contact(s)

Victoria Mendez
212-746-4829
vib9049@med.cornell.edu

Primary Investigator(s)

Ellen K. Ritchie, MD

Protocol ID(s)

Weill Cornell Medicine IRB #:

2402027025

ClinicalTrials.gov:

NCT05980806

Sponsor:

XPORT-MF-044

Status

Open to Enrollment

Age Group

Adult

Sponsor

  • Karyopharm Therapeutics

Disease

Thrombocytopenia
Myeloproliferative Neoplasms
Myelofibrosis