This Phase 3 study is evaluating the safety and effectiveness of midostaurin (Rydapt) combined with chemotherapy for patients with newly-diagnosed acute myeloid leukemia (AML). The main outcome to be measured will be event-free survival at 5 years.
Previous studies have shown that midostaurin combined with chemotherapy is more effective than midostaurin alone for the treatment of AML. Midostaurin is a type of targeted therapy. This type of treatment binds to cancer cells and blocks their growth. This leads to cancer cell death. Midostaurin is approved for treating patients with AML who have an abnormal gene called FLT3. Whether patients who lack this abnormal gene may benefit from midostaurin remains unclear.
This study is evaluating the safety and effectiveness of midostaurin combined with chemotherapy for newly-diagnosed AML. The main outcome will be measured as the amount of time from treatment until relapse or death from any cause.
Who are they looking for?
This study is recruiting 502 patients. Patients should have adequate lab test results. Patients should be able to receive intensive chemotherapy treatment. Patients should have AML that does not have the FLT3 abnormal gene.
Patients should not have received prior treatment for leukemia. Patients should not have secondary AML due to prior therapy. Patients should not have received prior treatment with agents like midostaurin.
How will it work
Patients will be divided into two groups. The first group will receive midostaurin. The second group will receive a placebo (a substance with no active effect). Both groups will receive chemotherapy. Patients will be followed-up for 5 years.
At follow-up, effectiveness will also be measured as overall survival (time from treatment until death from any cause). The number of patients who achieve a complete response (disappearance of all signs of cancer) will also be measured.