Selinexor in Myelofibrosis Refractory or Intolerant to JAK1/2 Inhibitors (ESSENTIAL)
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|ClinicalTrials.gov Identifier: NCT03627403|
Recruitment Status : Recruiting
First Posted : August 13, 2018
Last Update Posted : May 9, 2022
|Condition or disease||Intervention/treatment||Phase|
|Primary Myelofibrosis Post-essential Thrombocythemia Myelofibrosis Post-polycythemia Vera Myelofibrosis||Drug: Selinexor||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||56 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Open label, non-randomized, prospective, single-arm study|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Study to Evaluate the Efficacy and Safety of Selinexor in Patients With Myelofibrosis Refractory or Intolerant to JAK1/2 Inhibitors|
|Actual Study Start Date :||May 10, 2019|
|Estimated Primary Completion Date :||March 14, 2024|
|Estimated Study Completion Date :||March 14, 2025|
Experimental: Selinexor, all patients
Single Arm Study, all patients will get selinexor
Selinexor will be administered orally at a dose of 80 mg once weekly until IWG-MR disease progression, intolerable toxicity, or no clinical benefit per treating physician's discretion whichever occurs first.
For patients enrolled after Protocol v7, Selinexor will be administered by oral route beginning at 40mg once weekly. . Prior to protocol version 7, the starting dose of sSelinexor was 60 mg and 80 mg once weekly.
- Change in spleen volume [ Time Frame: Up to 6 months ]
To assess the efficacy of selinexor on spleen volume reduction in subjects with myelofibrosis (PMF, PET-MF, or PPV-MF) refractory or intolerant to ruxolitinib and/or any other experimental JAK1/2 inhibitors.
Change and percentage change in spleen volume from baseline to EOT as measured by MRI or CT (in applicable patients).
- Adverse Events that Occur [ Time Frame: Up to 6 months ]
To assess the safety/tolerability and further characterize the safety profile of selinexor in PMF, PET-MF, or PPV-MF patients refractory or intolerant to ruxolitinib and/or any other experimental JAK1/2 inhibitors.
Secondary Endpoints: rate of adverse events (AEs) and serious adverse events (SAEs).
- Change in symptoms score [ Time Frame: Up to 6 months ]
Proportion of patients with ≥ 50% reduction of total symptoms score as measured by the MPN-Symptoms Assessment Form (MPN-SAF) from baseline after 6 cycles of treatment.
The score will be reported as one mean score of all of the questions in the questionnaire. The higher the score, the worse the symptom.
- Overall response [ Time Frame: Up to 6 months ]Overall response rate according to the 2013 IWG-MRT consensus criteria for treatment response in primary and secondary MF (post-PV and post-ET).
- Overall Survival [ Time Frame: Up to 24 months ]Overall survival at 24 months from the initiation of study therapy.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03627403
|Contact: Priscilla Blosser, RN||801-213-6117||Priscilla.email@example.com|
|United States, Utah|
|Huntsman Cancer Institute/University of Utah||Recruiting|
|Salt Lake City, Utah, United States, 84112|
|Contact: Priscilla Blosser, RN 801-213-6117 Priscilla.firstname.lastname@example.org|
|Principal Investigator:||Srinivas Tantravahi, MD||University of Utah|