Enasidenib for Acute Myeloid Leukemia
Trial Summary
What is the purpose of this trial?
This phase II trial studies the side effects of enasidenib and sees how well it works in treating pediatric patients with acute myeloid leukemia that has come back after treatment (relapsed) or has been difficult to treat with chemotherapy (refractory). Patients must also have a specific genetic change, also called a mutation, in a protein called IDH2. Enasidenib may stop the growth of cancer cells by blocking the mutated IDH2 protein, which is needed for leukemia cell growth.
Will I have to stop taking my current medications?
The trial requires that you stop taking certain medications before starting enasidenib. Specifically, you must discontinue drugs with a narrow therapeutic range that are sensitive to certain enzymes, as well as the drug rosuvastatin, more than 5 half-lives before the first dose of enasidenib. Additionally, you cannot be on other anti-cancer agents or investigational drugs during the trial.
What data supports the effectiveness of the drug Enasidenib for treating acute myeloid leukemia?
Research shows that Enasidenib, a drug targeting specific genetic mutations in acute myeloid leukemia, has been effective in some patients, with 40.3% showing positive responses and a median survival of 9.3 months. It is approved for use in patients with relapsed or refractory acute myeloid leukemia with IDH2 mutations.12345
Is Enasidenib safe for humans?
What makes the drug Enasidenib unique for treating acute myeloid leukemia?
Enasidenib is unique because it specifically targets and inhibits mutant IDH2 proteins, which are involved in the development of certain types of acute myeloid leukemia. It is taken orally and is particularly used for patients with relapsed or refractory AML who have an IDH2 mutation, offering a targeted approach compared to traditional chemotherapy.23567
Research Team
Sara Zarnegar-Lumley
Principal Investigator
Children's Oncology Group
Eligibility Criteria
This trial is for patients with acute myeloid leukemia that's returned or hasn't responded to chemo, and have an IDH2 mutation. They should be able to perform daily activities at least halfway normally, not have certain AML types like Down syndrome-related or t(15;17), no known allergies to enasidenib, and mustn't be pregnant or breastfeeding. Participants need proper kidney function, acceptable blood counts, and can't be on conflicting medications.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive enasidenib orally once daily on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.
Follow-up
Participants are monitored for safety and effectiveness after treatment completion, with follow-up at 30 days, then periodically up to 1 year.
Treatment Details
Interventions
- Enasidenib
Enasidenib is already approved in United States, European Union for the following indications:
- Relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation
- Acute myeloid leukaemia (AML) with an isocitrate dehydrogenase 2 (IDH2) mutation
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Oncology Group
Lead Sponsor
National Cancer Institute (NCI)
Collaborator