Selinexor for Myelofibrosis
(ESSENTIAL Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the effectiveness of selinexor for individuals with myelofibrosis, a rare bone marrow disorder that causes severe symptoms and an enlarged spleen. It targets those who haven't responded to or can't tolerate current treatments such as ruxolitinib. Participants should have primary or secondary myelofibrosis and continue to experience significant symptoms despite previous treatment. The trial aims to determine if selinexor, a new potential drug, can improve their condition safely and effectively. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.
Do I need to stop my current medications to join the trial?
The trial requires a washout period (time without taking certain medications) for any prohibited medications before starting the study treatment. If you are taking ruxolitinib or other JAK1/2 inhibitors, you must stop at least 3 days or 5 half-lives before the trial begins. It's best to discuss your specific medications with the trial team.
Is there any evidence suggesting that selinexor is likely to be safe for humans?
Research has shown that selinexor is generally well-tolerated by patients with myelofibrosis. In earlier studies, selinexor proved effective both alone and in combination with ruxolitinib. Safety information from these studies indicates that most patients managed the treatment without serious issues. Common side effects included fatigue, nausea, and reduced appetite, but these were usually manageable.
Early results from a study with 61 patients demonstrated that selinexor was safe for more than 12 months, suggesting it might be safe for longer use as well. However, each person's experience can differ, so discussing potential risks with a healthcare provider is always advisable.12345Why do researchers think this study treatment might be promising for myelofibrosis?
Unlike the standard treatments for myelofibrosis, which often include JAK inhibitors like ruxolitinib, selinexor offers a different approach by targeting nuclear export. Selinexor works by inhibiting a protein called XPO1, which helps transport cancer-promoting molecules out of the cell nucleus. By blocking this process, selinexor can potentially restore normal cell function and slow disease progression. Researchers are excited because this unique mechanism of action could provide new hope for patients who may not respond well to existing therapies.
What evidence suggests that selinexor might be an effective treatment for myelofibrosis?
Research has shown that selinexor might improve symptoms in patients with myelofibrosis. Studies have found that patients taking selinexor had higher hemoglobin levels and required fewer blood transfusions. Additionally, selinexor has been associated with smaller spleen sizes, which can alleviate symptoms. In laboratory studies, selinexor decreased the number of active cancer cells and slowed their growth. These findings suggest that selinexor could be a promising treatment option for those who haven't had success with other therapies.16789
Who Is on the Research Team?
Srinivas Tantravahi
Principal Investigator
University of Utah
Are You a Good Fit for This Trial?
Adults diagnosed with primary or secondary myelofibrosis who haven't responded well to, or can't tolerate, JAK1/2 inhibitors like ruxolitinib. They should be relatively active (ECOG ≤ 2), have a spleen enlarged by at least 5 cm if symptomatic or 10 cm regardless of symptoms, and meet specific blood count and organ function criteria.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive selinexor for up to six cycles to assess efficacy and safety
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term follow-up
Participants are monitored for adverse events and overall survival
What Are the Treatments Tested in This Trial?
Interventions
- Selinexor
Selinexor is already approved in United States, Canada for the following indications:
- Multiple myeloma
- Diffuse large B-cell lymphoma
- Multiple myeloma
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Utah
Lead Sponsor
Karyopharm Therapeutics Inc
Industry Sponsor
Richard Paulson
Karyopharm Therapeutics Inc
Chief Executive Officer since 2021
MBA from the University of Toronto's Rotman School of Management
Reshma Rangwala
Karyopharm Therapeutics Inc
Chief Medical Officer since 2023
MD, PhD