Selinexor Versus Treatment of Physician's Choice in Patients With Previously Treated Myelofibrosis
A PHASE 2, RANDOMIZED, OPEN-LABEL, MULTICENTER STUDY TO EVALUATE SAFETY AND EFFICACY OF SINGLE AGENT SELINEXOR VERSUS TREATMENT OF PHYSICIAN’S CHOICE IN PATIENTS WITH PREVIOUSLY TREATED MYELOFIBROSIS
Karyopharm Therapeutics Inc
Myelofibrosis (MF) is a type of bone marrow cancer which disrupts the normal production of blood cells, causing scarring in the bone marrow. It may also cause the number of blood-clotting cells (platelets) to be reduced, increasing the risk of bleeding. The clinical signs and symptoms of MF are an enlarged spleen, anaemia, weakness and fatigue. Currently, the only potentially curative treatment for MF is allogeneic hematopoietic stem cell transplant.
Selinexor is an investigative drug (i.e. not yet approved by a regulatory agency) being developed for the treatment of MF.
Adults with MF aged 18 or older, who have been treated the Janus Kinas (JAK) inhibitors for at least 6 months can take part if they meet the following conditions: they have relapsed (their MF has got worse), they are refractory (their MF does not respond to the treatment) or they are unable to tolerate the treatment.
There will be two treatment arms, Arm S (Selinexor) and Arm PC (Physician’s choice). The study design is randomized and open-label. The objective is to test whether selinexor is safe and effective.
Selinexor will be compared to the choice of treatment by the participant’s physician. The physician’s choice may include ruxolitinib retreatment, fedratinib, chemotherapy (e.g., hydroxyurea), anagrelide, corticosteroid, hematopoietic growth factor, androgen, interferon, and may include supportive care only with no MF treatment. However, the physician may not choose and alternative investigational therapy.
The effect of the treatment will be checked by medical assessments, blood tests, magnetic resonance imaging (MRI) or computed tomography (CT) scan, bone marrow tests, checking for side effects, and completing questionnaires. Participants will be able to crossover from Arm PC to Arm S if they develop progressive disease.
The study will take place at approximately 24 sites in Europe and at 10 sites in the United States. Approximately 112 participants will be enrolled worldwide.
West of Scotland REC 1
Date of REC Opinion
10 Aug 2022
Further Information Favourable Opinion