Study of Selinexor+Ruxolitinib for efficacy/safety in myelofibrosis
Research type
Research Study
Full title
A Phase 1/3 Study To Evaluate The Efficacy And Safety Of Selinexor, A Selective Inhibitor Of Nuclear Export, In Combination With Ruxolitinib In Treatment-Naïve Patients With Myelofibrosis
IRAS ID
1008333
Contact name
Demetrice Avery
Contact email
Sponsor organisation
Karyopharm Therapeutics Inc.
Eudract number
2020-003883-19
Clinicaltrials.gov Identifier
Research summary
Myelofibrosis (MF) is an uncommon type of cancer in the bone marrow. Bone marrow is found in the centre of most bones, where new healthy blood cells are made. In myelofibrosis, the normal production of blood cells in the bone marrow is disrupted leading to anaemia – a lack of red blood cells that can cause weakness and fatigue. MF may also cause an enlarged spleen, a part of your body’s immune system. Current MF treatments work by blocking a protein (Janus kinase 2 [JAK2]) that causes cells to multiply, helping to improve symptoms and reduce the size of the spleen.
Many patients stop their current available treatments due to intolerance to the medicine or lack of response, so there is a need for alternative treatments that work differently. Selinexor is an experimental medication for MF that works by blocking a protein, exportin-1 (XPO1). This study aims to look at the effects and safety of selinexor in combination with ruxolitinib. Ruxolitinib is an approved medication for treating MF that inhibits JAK2.
This is a phase 3, double-blind study. A screening period of 4 weeks will assess if patients can take part. The treatment period will see patients divided into two groups by chance and receive either selinexor with ruxolitinib, or placebo with ruxolitinib. Patients will receive selinexor as 60 mg once a week with ruxolitinib or placebo with ruxolitinib. The starting dose of ruxolitinib will be either 15 or 20 mg two times a day. Each cycle will be 28 days long. A follow-up period will include phone calls to the patient at 30 days after the last treatment and then every 3 months for 1 year and then every 6 months until either the patient withdrawals consent, starts new therapy, has disease progression, or the end of the study.
Around 300 to 450 patients in approximately 19 countries will take part in the study. The study is anticipated to last approximately 48 months.
Karyopharm Therapeutics Inc. is the sponsor and will organise and fund the study.
REC name
London - Fulham Research Ethics Committee
REC reference
23/LO/0804
Date of REC Opinion
20 Nov 2023
REC opinion
Further Information Favourable Opinion