A Randomized, open label, phase 2 study of the selective inhibitor of nuclear export (SINE) SELINEXOR (KPT-330) versus specified physician's choice in patients * 60 years old with relapsed/refractory acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy and/or transplantation.
- Conditions
- acute myeloid leukemiaform of blood cancer10024324
- Registration Number
- NL-OMON41919
- Lead Sponsor
- Karypharm Europe GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 19
Patients age * 60 years with relapsed/refractory AML (defined using WHO criteria) of any type except for acute promyelocytic leukemia (APL; AML M3), who have poor prognosis (intermediate or adverse risk) cytogenetics, with relapsed or refractory AML, after at least one prior AML therapy (must have included an adequate trial of a hypomethylating agent with at least 2 cycles), who have never undergone, and who are not currently eligible for stem cell transplantation, and are currently deemed unfit for intensive chemotherapy.
Patients with AML M3, known central nervous system (CNS) leukemia, who are in blast transformation of chronic myeloid leukemia (CML), or whose AML is classified as favorable according to the European LeukemiaNet (ELN) disease risk assessment will be excluded from this study.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary efficacy endpoint: Overall survival.</p><br>
- Secondary Outcome Measures
Name Time Method <p>* The proportion of patients whose OS is at least 3 months (OS3.0)<br /><br>* The complete remission rate (CRR), including complete remission with full<br /><br>hematologic recovery (CR), and median disease free survival (DFS) for patients<br /><br>who achieve CR<br /><br>* The modified CRR (mCRR), including CR or CRi (including CRp), and median DFS<br /><br>for patients who achieve CR or CRi (including CRp)<br /><br>* The overall response rate (ORR) and duration of overall response (DOR),<br /><br>including CR, CRi, MLFS, and partial remission (PR)<br /><br>* The disease control rate (DCR) defined as ORR + stable disease for * 4 weeks<br /><br>(SD), and duration of DCR<br /><br>* Quality of life and patient reported outcomes (FACT-Leukemia and EQ-5D-5L)<br /><br>(QoL)<br /><br><br /><br>The safety and tolerability of selinexor and PC will be evaluated by means of<br /><br>drug-related AE reports, physical examinations, and laboratory safety<br /><br>evaluations.</p><br>