New oral drug could tame overactive bone marrow in rare blood cancers
NCT ID NCT07626021
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a pill called selinexor in 15 adults with chronic blood cancers (MPN) whose blood cell counts are too high. The goal is to see if selinexor can safely bring those counts down and ease symptoms. Participants take the drug once a week for up to 3 months and are followed for 6 months total.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- selinexor (KPT-330), an oral drug that blocks a protein to help control cancer cell growth
- What this could lead to
- If it works, selinexor could offer a new oral option for patients with certain blood cancers who need to lower their blood cell counts but have few other choices.
- What could go wrong
- This is a very small, early-phase trial with only 15 people, so results may not apply widely. The drug can cause side effects like low blood counts or other toxicities, and it may not work for everyone.
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 15 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jul 2026
An estimate. Start dates often move.
- Expected to finish
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Aug 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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 to 80 years
- 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: * Aged 18 to 80 years at the time of informed consent. * Diagnosis of Philadelphia chromosome-negative myeloproliferative neoplasm, including polycythemia vera, essential thrombocythemia, or primary myelofibrosis, according to World Health Organization criteria. * Presence of an indication for cytoreductive therapy, including at least one of the following: * Extreme thrombocytosis, defined as platelet count \>1500 x 10\^9/L. * Progressive leukocytosis, defined as white blood cell count \>25 x 10\^9/L. * Symptomatic splenomegaly documented by imaging. * Severe disease-related symptoms, such as significant weight loss within the past 6 months. * Unwillingness or inability to tolerate standard cytoreductive therapies, such as hydroxyurea or interferon. * Eastern Cooperative Oncology Group performance status of 0 to 2. * Adequate organ function, including renal, hepatic, cardiac, and coagulation function, as determined by laboratory tests and clinical evaluation in the opinion of the investigator. * Adequate baseline hematologic function without recent transfusion or growth factor support. * Ability to comply with study procedures, visits, and assessments. * Ability to understand and willingness to sign a written informed consent form. * For women of childbearing potential, a negative pregnancy test before study entry. * For participants of reproductive potential, agreement to use effective contraception during the study and for at least 120 days after the last dose of study drug. Exclusion Criteria: * Diagnosis of Philadelphia chromosome-positive myeloproliferative neoplasm or chronic myeloid leukemia. * Concurrent acute leukemia or other active hematologic malignancy. * Severe or uncontrolled comorbid condition, including but not limited to significant cardiac or pulmonary disease, decompensated liver disease, or end-stage renal disease. * Active uncontrolled bacterial, viral, or fungal infection. * Active gastrointestinal disorder associated with significant bleeding or impaired drug absorption. * Significant neurologic or psychiatric disorder that may interfere with study participation or compliance. * Known hypersensitivity to selinexor or any of its components. * Receipt of another investigational agent or participation in another interventional clinical trial within 4 weeks before enrollment. * Recent receipt of cytotoxic chemotherapy, radiotherapy, immunotherapy, or other cytoreductive treatment before study entry. * History of substance abuse, alcohol dependence, or illicit drug use that may interfere with adherence to study requirements. * Another active malignancy requiring systemic treatment. * Clinically significant bleeding disorder, severe coagulopathy, or requirement for long-term therapeutic anticoagulation. * Receipt of a live vaccine within 30 days before the first dose of study drug, or planned receipt of a live vaccine during the study. * Known active infection with human immunodeficiency virus or active viral hepatitis. * Estimated life expectancy of less than 12 weeks. * Inability to provide informed consent due to cognitive impairment or severe psychiatric illness. * Any condition that, in the investigator's judgment, would make the participant unsuitable for participation in this study.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
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