This phase 3 trial aims to evaluate the efficacy and safety of AG-221 as a treatment for older patients with late stage IDH2 mutated acute myeloid leukemia (AML). The main outcome to be investigated is overall survival.
The details
Mutations in the isocitrate dehydrogenase enzyme 2 (IDH2) are present in roughly 15% of AML patients. AG-221 is a dehydrogenase inhibitor. It works by blocking dehydrogenase that is needed to encourage tumor formation and growth. It has previously been used as an investigational drug in other clinical trials for AML. It is thought that it could be effective in older patients with late stage IDH2 mutated AML.
The study will evaluate the efficacy and safety of AG-221 by looking at overall survival (time from start of study to death from any cause) and various other outcomes.
Who are they looking for?
This study will recruit 316 patients aged 60 and over who have primary or secondary AML who are being treated for the second or third time. Patients should have the IDH2 mutated AML. All patients must have adequate organ function to take part. Patients must agree to the use of contraceptives for the duration of the study.
Patients with acute promyelocytic leukemia and central nervous system leukemia will be excluded. Patients who have received drugs against IDH2, anticancer therapy, radiotherapy or investigational drugs recently will not be eligible. Those with heart problems, hypertension, HIV, hepatitis or active infections will not be allowed take part.
How will it work
Patients will be randomly assigned to one of two groups. One group will receive AG-221 with the best available supportive care (BSC). They will receive AG-221 once a day for 28 days along with best supportive care. The other group will receive a conventional care regimen. This will involve one of the following: 28 day cycles of BSC only; azacitidine (Vidaza) subcutaneously (injected under skin) plus BSC; low-dose cytarabine (Cytosar-U) subcutaneously plus BSC; intermediate dose cytarabine subcutaneously plus BSC; or intermediate dose cytarabine intravenously (injected into vein) plus BSC.
The effectiveness and safety will be measured by overall survival rates. This will be done by following up with patients for at least 28 months.
