Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With an IDH2 Mutation

Official Title

An Open-Label Feasibility Study to Assess the Safety and Pharmacokinetics of Enasidenib in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (R/R-AML) With an Isocitrate Dehydrogenase-2 (IDH2) Mutation

Summary:

This trial studies the side effects of enasidenib and to see how well it works in treating 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 cell growth.

Trial Description

Primary Outcome:

• Incidence of adverse events of enasidenib
• Area under the plasma concentration versus time curve of enasidenib
• Total plasma clearance of enasidenib
• Elimination half-life of enasidenib
• Maximum concentration of enasidenib

Secondary Outcome:

• Plasma 2-HG levels of enasidenib
• Composite complete remission rate (complete remission [CR]/ CR with incomplete hematologic recovery [CRi])
• Anti-tumour activity of enasidenib
• Duration of remission of enasidenib
• Duration of response of enasidenib
• Event-free survival of enasidenib
• Overall survival of enasidenib
• Time to remission of enasidenib
• Time to response of enasidenib

PRIMARY OBJECTIVES:

• To determine the safety of treatment with enasidenib mesylate (enasidenib) administered at continuous daily oral dosing for a 28-day cycle up to 12 cycles in pediatric patients with IDH2-mutant relapsed/refractory (R/R)-acute myeloid leukemia (AML).
• To characterize the plasma pharmacokinetic (PK) profile of enasidenib in pediatric patients with IDH2-mutant R/R-AML.

SECONDARY OBJECTIVES:

• To investigate the pharmacodynamic (PD) relationship of oncogenic metabolite 2-hydroxyglutarate (2-HG) to enasidenib treatment in pediatric patients with IDH2-mutant R/R-AML.
• To describe the clinical activity of enasidenib in pediatric patients with IDH2-mutant R/R-AML.

OUTLINE: Patients receive enasidenib orally (PO) once daily (QD) on days 1-28. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 30 days, then periodically up to 1 year.

View this trial on ClinicalTrials.gov