Table of Contents

• Trial overview
• Myelofibrosis studies
• Multiple myeloma studies
• Lymphoma studies
• Other conditions studied
• Main outcomes used in the trials
• Study status and what it means

Trial overview

The data show interventional trials, which means researchers give a treatment and watch what happens.^[1] The studies of Selinexor cover several diseases, with the most common being myelofibrosis and multiple myeloma.^{[1][2] The trial phases range from Phase 1 to Phase 4, so the research includes early dose-finding studies and later comparison studies.[1][2]}

Myelofibrosis studies

Several studies focus on myelofibrosis, a bone marrow disease that can cause spleen enlargement and symptoms such as tiredness.^{[1][2] One Phase 2 study looks at Selinexor monotherapy, which means Selinexor is used alone, in people with JAK inhibitor-naïve myelofibrosis and moderate thrombocytopenia, a lower-than-normal platelet count.[1] That study measures the proportion of patients with spleen volume reduction of 35% or more at Week 24, using MRI or CT scans.[1]}

Another Phase 3 study tests Selinexor plus ruxolitinib in treatment-naïve patients with myelofibrosis and compares it with placebo plus ruxolitinib.^[2] Its main outcomes are spleen volume reduction at Week 24 and change in symptom score, measured with the Myelofibrosis Symptom Assessment Form.^[2] A separate Phase 2 study looks at Selinexor alone versus physician’s choice in previously treated myelofibrosis, and it measures the rate of spleen volume reduction of 35% or more.^[8]

Multiple myeloma studies

Multiple myeloma is studied in several Selinexor trials, including patients with relapsed or refractory disease, which means the disease came back or did not respond well to earlier treatment.^{[3][5][7] One Phase 2 study looks at Selinexor with dexamethasone and bortezomib in people with penta-refractory or triple-class refractory multiple myeloma.[7] The main outcome is overall response rate, which counts complete and partial responses.[7]}

Two Phase 3 studies also focus on relapsed or refractory multiple myeloma.^{[5][9] One compares etentamig with standard available therapies and includes Selinexor among the listed treatment options.[5] Its outcomes are progression-free survival and overall response rate, both assessed by International Myeloma Working Group criteria.[5] Another Phase 3 study compares selinexor pomalidomide dexamethasone with elotuzumab pomalidomide dexamethasone, and its main outcome is progression-free survival.[9]}

Lymphoma studies

Selinexor is also being studied in diffuse large B-cell lymphoma, a fast-growing type of lymphoma.^[4] One Phase 4 study compares R-GDP with or without Selinexor in patients with relapsed or refractory diffuse large B-cell lymphoma.^[4] The trial looks at overall response rate in Phase 2 and progression-free survival in Phase 3, using the Lugano 2014 criteria, which are standard rules for measuring lymphoma response.^[4]

A smaller Phase 2 pilot study tests Selinexor with ifosfamide, etoposide, and dexamethasone in relapsed or refractory peripheral T-cell lymphomas.^[6] The main outcome is overall response rate after 4 treatment courses.^[6]

Other conditions studied

One Phase 3 study looked at Selinexor as maintenance therapy after combination chemotherapy in patients with advanced or recurrent endometrial cancer.^[7] Its primary outcome was progression-free survival, measured from randomization until disease progression or death.^[7]

Another Phase 1 study explored Selinexor plus gemcitabine in selected advanced soft-tissue sarcomas, including leiomyosarcoma and malignant peripheral nerve sheath tumor.^{[3][10] In this study, Phase 1 focused on dose-limiting toxicities, which are side effects severe enough to limit dosing, and Phase II would have looked at progression-free survival rate at 6 months.[3] A second record for the same soft-tissue sarcoma study was withdrawn.[10]}

Main outcomes used in the trials

The studies use several common outcomes to judge benefit.^{[1][2][4][5][6][7][8][9]}

• Overall response rate shows how many patients have a meaningful improvement in their disease, such as complete or partial response.^[4][5][6][7]
• Progression-free survival measures how long patients live without the disease getting worse.^[4][5][7][9]
• Overall survival measures how long patients stay alive after randomization in the study.^[3]
• Spleen volume reduction is used in myelofibrosis to see whether the spleen becomes smaller on scan.^[1][2][8]
• Myelofibrosis Symptom Assessment Form records how symptoms change over time.^[2]
• Dose-limiting toxicities help researchers find the highest dose that can be given safely.^[3]

Study status and what it means

Most of the listed Selinexor trials are authorised, which means they have been approved to start or continue.^{[1][2][4][5][6][8][9] Some studies are completed, such as the endometrial cancer trial and the allogeneic stem cell transplantation study in multiple myeloma.[3][7] One soft-tissue sarcoma trial is withdrawn, meaning it did not continue as planned.[10]}