New hope for myelofibrosis: selinexor trial targets spleen and symptoms
NCT ID NCT04562870
First seen Jun 25, 2026 · Last updated Jul 02, 2026 · Updated 2 times
Summary
This phase 2 study tests the drug selinexor against standard treatments in 112 people with myelofibrosis who have already tried JAK inhibitors. The main goal is to see if selinexor can shrink the spleen and improve symptoms. Participants are randomly assigned to receive either selinexor or their doctor's choice of therapy.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- selinexor
- What this could lead to
- If successful, selinexor could offer a new treatment option for myelofibrosis patients who have not responded well to standard JAK inhibitor therapy.
- What could go wrong
- This is a phase 2 trial with only 112 participants, so results may not be definitive. Selinexor may cause side effects like nausea, fatigue, or low blood counts, and it may not prove better than existing treatments.
Why investors are watching
Karyopharm is running a Phase 2 trial of its drug selinexor against standard treatments chosen by doctors in 112 patients with myelofibrosis who have already tried a JAK inhibitor. For a micro-cap company, this readout is a major test of whether selinexor can work in a new disease area and support the company's future value.
If it works: If selinexor shows better safety or effectiveness than the doctor's choice, Karyopharm could gain a new treatment option for myelofibrosis and strengthen its drug pipeline. A positive result might also attract partnership interest or support further development.
If it fails: Phase 2 trials often fail to show a clear benefit, and a negative or unclear result could hurt the company's prospects and stock. Delays in enrollment or data readout could also weigh on investor confidence.
AI-written from the trial record. Speculative, and not investment advice.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 112 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2021
- Expected to finish
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Sep 2026
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * A diagnosis of primary MF or post-essential thrombocythemia (ET) or post-polycythemia (PV) MF according to the 2016 World Health Organization (WHO) classification of myeloproliferative neoplasms (MPN), by the most recent local pathology report. * Previous treatment with JAK inhibitors for at least 6 months. * Measurable splenomegaly during the screening period as demonstrated by spleen volume of ≥450 centimeter cube (cm\^3) by magnetic resonance imaging (MRI) or computerized tomography (CT) scan. * Relapsed, Refractory or Intolerant to JAK inhibitors as defined as meeting one of the criteria below: * less than (\<) 35% spleen volume reduction by MRI or CT-scan (from baseline) or * \<50% decrease in spleen size by palpation (from baseline) or an increase of at least 3 cm with the spleen at least 5 cm below the left costal margin or * Spleen volume increase greater than (\>) 25% from nadir or a return to within 10% of baseline after any initial response or * Treatment with JAK inhibitor was complicated by development of red blood cells (RBC) transfusion requirement (2 units per month for 2 month); or grade 3 thrombocytopenia, anemia, hematoma/hemorrhage; or grade 2 non-hematologic toxicity while on JAK inhibitors * Participants ≥18 years of age. * Eastern Cooperative Oncology Group (ECOG) less than or equal to (≤) 2. * Platelet count ≥75\*10\^9 per liter (/L). * Absolute neutrophil count (ANC) ≥1.5\*10\^9/L. * Serum direct bilirubin ≤1.5\*upper limit of normal (ULN); aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5\*ULN. * Calculated creatinine clearance (CrCl) \>15 milliliter (mL)/minute (min) based on the Cockcroft and Gault formula. * Participants with active hepatitis B virus (HBV) are eligible if antiviral therapy for hepatitis B has been given for \>8 weeks and viral load is \<100 International Units (IU)/mL. * Participants with untreated hepatitis C virus (HCV) are eligible if there is a documentation of negative viral load per institutional standard. * Participants with history of human immunodeficiency virus (HIV) are eligible if they have cluster of differentiation 4 (CD4)+ T-cell counts ≥350 cells/microliter (mcL), negative viral load per institutional standard, and no history of acquired immunodeficiency syndrome (AIDS)-defining opportunistic infections in the last year. * Female participants of childbearing potential must have a negative serum pregnancy test at screening and agree to use highly effective methods of contraception throughout the study and for at least 90 days after the last dose of selinexor, or for the duration as stated on the label (SmPC/USPI) for those on the comparator drug (physician's choice arm). Childbearing potential excludes: Age \>50 years and naturally amenorrhoeic for \>1 year, or previous bilateral salpingo-oophorectomy, or hysterectomy. * Male participants who are sexually active must use highly effective methods of contraception throughout the study and for at least 90 days after the last dose of selinexor, or for the duration as stated on the label (SmPC/USPI) for those on the comparator drug (physician's choice arm). Male participants must agree not to donate sperm during the study treatment period. * Participants must sign written informed consent in accordance with federal, local and institutional guidelines. Exclusion Criteria: * \>5% blasts in peripheral blood or \>10% blasts in bone marrow (i.e., accelerated phase). * Previous treatment with selinexor or other exportin 1 (XPO1) inhibitors. * Use of any standard or experimental anti-MF therapy \<21 days prior to Cycle 1 Day 1 (hydroxyurea or growth factors are allowed). * Impairment of gastrointestinal (GI) function or GI disease that could significantly alter the absorption of selinexor (Example: vomiting, or diarrhea that is Common Terminology Criteria for Adverse Events (CTCAE) grade \>1). * Received strong cytochrome P450 3A (CYP3A) inhibitors ≤7 days prior to selinexor dosing or strong CYP3A inducers ≤14 days prior to selinexor dosing. * Major surgery \<28 days prior to cycle 1 day 1 (C1D1). * Uncontrolled (ie, clinically unstable) infection requiring parenteral antibiotics, antivirals, or antifungals within 7 days prior to first dose of study treatment; however, prophylactic use of these agents is acceptable (including parenteral). * Any life-threatening illness, medical condition, or organ system dysfunction which, in the Investigator's opinion, could compromise the participants safety, prevent the participant from giving informed consent, or being compliant with the study procedures. * Female participants who are pregnant or lactating. * Participants with contraindications to use of selinexor or all the drugs intended to be used in the comparative treatment arm.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Affiliated Hospital of Nantong University
Nantong, Jiangsu, 226001, China
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Asst Settelaghi, Ospedale Di Circolo E Fondazione Macchi
Varese, 21100, Italy
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Azienda Unita Sanitaria Locale Latina - Ospedale Santa Maria Goretti
Latina, 4100, Italy
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Centre Hospitalier Universitaire d'Angers (CHU Angers)
Angers, 49933, France
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Hospital Universitario 12 de Octubre
Madrid, Madrid, 28041, Spain
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Illinois Cancer Specialist
Niles, Illinois, 60714, United States
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Institut de Cancéro-Hématologie
Brest, Brittany Region, 29609, France
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Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IRST) IRCCS
Meldola, Forlì-Cesena, 47014, Italy
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Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730, China
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Pratia Onkologia Katowice
Katowice, 40-519, Poland
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Rocky Mountain Cancer Centers, LLP
Aurora, Colorado, 80012, United States
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Sir Run Run Shaw Hospital - Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310016, China
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Suzhou University -The First Affiliated Hospital
Suzhou, Jiangsu, 215007, China
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Texas Oncology - Northeast Texas
Tyler, Texas, 75702, United States
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The First Hospital of Jilin University
Changchun, Jilin, 130021, China
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The Oncology Institute of Hope and Innovation
Pasadena, California, 91105, United States
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The Second Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215004, China
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Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, 430022, China
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University General Hospital "ATTIKON"
Athens, Attica, 12462, Greece
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University of Perugia Department of Medicine Hematology Section
Perugia, 6132, Italy
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Università degli Studi di Firenze - Azienda Ospedaliero - Universitaria Careggi - Dipartimento di medicina sperimentale e clinica
Florence, 50134, Italy
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Half-Matched stem cells tested as cure for myelofibrosis
- Can blood tests predict transplant complications?
- Can a new daily pill shrink the spleen and ease myelofibrosis symptoms?
- Can a new pill tame myelofibrosis?
- MRI as a window into bone marrow disease: a new biomarker test?
- Can a menin inhibitor tame myelofibrosis when standard drugs fall short?