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Study on the Effectiveness and Safety of Selinexor for Patients with Myelofibrosis and Moderate Thrombocytopenia Who Have Not Used JAK Inhibitors

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effectiveness and safety of a treatment for people with a condition called myelofibrosis, which is a type of blood cancer that affects the bone marrow. The study is specifically for those who have not previously been treated with a type of medication known as a JAK inhibitor and who have a condition called moderate thrombocytopenia, which means they have a lower than normal number of platelets in their blood. The treatment being tested is a medication called selinexor, which is taken as a tablet.

The purpose of the study is to evaluate how well selinexor works on its own in treating myelofibrosis. Participants in the study will take selinexor orally for a period of up to 105 days. During this time, they will have regular check-ups to monitor their health and see how the treatment is affecting their condition. The study will also compare the effects of selinexor with those of a placebo, which is a substance with no active medication, to better understand its effectiveness.

Throughout the study, participants will undergo various assessments, including imaging tests like MRI or CT scans, to measure changes in their spleen size and other health indicators. The study aims to gather information on the overall response to the treatment, including any changes in symptoms and side effects. This research will help determine if selinexor is a beneficial treatment option for people with myelofibrosis who have not been treated with JAK inhibitors before.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, a diagnosis of myelofibrosis (MF) or related conditions is confirmed through a pathology report.

    Eligibility is assessed based on specific health criteria, including liver function, platelet count, and absence of other malignancies.

    A bone marrow biopsy sample is required, which may have been obtained up to three months prior to the first dose of the study medication.

  2. Step 2

    Initial screening

    A comprehensive screening process is conducted to ensure all health criteria are met.

    A negative pregnancy test is required for female participants of childbearing potential.

    Participants must agree to use effective contraception methods during the study and for 90 days after the last dose of the study medication.

  3. Step 3

    Medication administration

    The study involves the administration of selinexor as a single-agent treatment.

    Selinexor is taken orally in tablet form.

    The dosage and frequency of selinexor administration are determined by the study protocol and monitored by the study team.

  4. Step 4

    Monitoring and assessments

    Regular monitoring of health status and response to treatment is conducted throughout the study.

    Participants are required to complete daily symptom assessments using a specific tool designed for this study.

    Imaging tests such as MRI or CT scans are performed to measure spleen volume and assess treatment efficacy.

  5. Step 5

    End of treatment evaluation

    At the end of the treatment period, a final evaluation is conducted to assess the overall response to selinexor.

    The primary endpoint is the proportion of participants achieving a significant reduction in spleen volume by week 24.

    Secondary endpoints include the assessment of symptom improvement, anemia response, and overall survival.

Who can join the trial?

22 criteria

  • Must have a diagnosis of MF (myelofibrosis) or related conditions confirmed by a recent pathology report.
  • If you have active hepatitis B, you must have been on antiviral treatment for more than 8 weeks, and the virus level should be very low.
  • If you have had hepatitis C, you must have completed treatment, and the virus should not be detectable.
  • If you have HIV, your immune cell count should be above a certain level, the virus should not be detectable, and you should not have had serious infections related to AIDS in the past year. You should also be on HIV treatment for at least 4 weeks.
  • Women who can have children must have a negative pregnancy test and agree to use effective birth control during and after the study.
  • Men who are sexually active must use a barrier method of birth control and agree not to donate sperm during and after the study.
  • Must sign a consent form agreeing to participate in the study.
  • Must have active symptoms of MF, as determined by a specific symptom score.
  • Must provide bone marrow samples for the study.
  • Must have a life expectancy of more than 6 months, according to the study doctor.
  • Must not have other cancers, except for certain types that have been treated.
  • Must have an enlarged spleen, as shown by a scan.
  • Must not be a candidate for stem cell transplantation.
  • Must be willing to complete a daily symptom questionnaire during the study.
  • Must be able to provide written consent, or have a representative do so if unable.
  • Must have a certain risk level of MF, as determined by a specific scoring system.
  • Must be 18 years of age or older.
  • Must have a performance status that allows participation in the study, as determined by a specific scale.
  • Must have a platelet count within a certain range without recent transfusions.
  • Must have a certain level of neutrophils, a type of white blood cell, without recent growth factor treatment.
  • Must have adequate liver function, as shown by specific blood tests.
  • Must have a certain level of kidney function, as calculated by a specific formula.

Who cannot join the trial?

2 criteria

  • Patients with **moderate thrombocytopenia** cannot participate. This means having a lower than normal number of platelets, which are cells that help your blood clot.
  • Patients who have not previously been treated with a **JAK inhibitor** for **myelofibrosis** are excluded. A JAK inhibitor is a type of medication, and myelofibrosis is a condition where scar tissue forms in the bone marrow, affecting blood cell production.
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Investigated drugs

Selinexor is a medication being studied for its effectiveness and safety in treating patients with myelofibrosis, a type of bone marrow cancer. This trial focuses on patients who have not previously been treated with JAK inhibitors and have moderate thrombocytopenia, which means they have a lower than normal number of platelets in their blood. Selinexor is being tested as a single-agent therapy, meaning it is used alone without combining it with other treatments. The goal is to see how well it works in reducing the size of the spleen, which is a common issue in myelofibrosis patients.

What is already known about the treatment

Selinexor – Selinexor is administered orally in the form of tablets. It is currently being studied in clinical trials for its effectiveness and safety in treating patients with myelofibrosis who have not previously received JAK inhibitors and have moderate thrombocytopenia. The main therapeutic indication for selinexor in this context is myelofibrosis, a type of bone marrow cancer. At the molecular level, selinexor works by inhibiting the export of tumor suppressor proteins from the nucleus of cancer cells, leading to cell death. It is classified as a selective inhibitor of nuclear export (SINE) compound.

Investigated diseases

  • Moderate Thrombocytopenia

    This condition is characterized by a lower than normal number of platelets in the blood, which are essential for blood clotting. Individuals with moderate thrombocytopenia may experience symptoms such as easy bruising, frequent nosebleeds, or prolonged bleeding from cuts. The condition can arise from various causes, including certain medications, autoimmune diseases, or bone marrow disorders. It is important to monitor platelet levels to prevent complications. The progression of the condition depends on the underlying cause and the individual's overall health.

  • JAK Inhibitor-Naïve Myelofibrosis

    Myelofibrosis is a rare bone marrow disorder that disrupts the body's normal production of blood cells, leading to severe anemia, weakness, and an enlarged spleen. In patients who have not previously been treated with JAK inhibitors, the disease may present with symptoms such as fatigue, night sweats, and weight loss. The condition is caused by the abnormal growth of fibrous tissue in the bone marrow, which impairs its ability to produce blood cells. Over time, myelofibrosis can lead to significant complications, including an increased risk of infections and bleeding. The progression of the disease varies among individuals, and regular monitoring is essential.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-511309-47-00Protocol codeXPORT-MF-044Estimated enrolment58 patientsSponsorKaryopharm Therapeutics Inc.

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