NCT03881735

Enasidenib in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia With an IDH2 Gene Mutation

A Phase II Study of Intensive Salvage Therapy Followed by Enasidenib for Patients With AML Harboring Mutations in IDH2 Who Have Failed or Been Refractory to One Prior Line of Therapy

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Roswell Park Cancer Institute · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well enasidenib works in treating in patients with acute myeloid leukemia with an IDH2 gene mutation that has come back or has not responded to treatment. Enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. In this study we are investing if enasidenib can be used as maintenance therapy post salvage induction chemotherapy.

Detailed description

PRIMARY OBJECTIVES: I. Evaluate the effect of IDH2 inhibition following conventional high dose salvage chemotherapy with detectable IDH2 mutations on event free survival (EFS) in patients with relapsed/refractory acute myeloid leukemia (AML). SECONDARY OBJECTIVES: I. Evaluate the effect of IDH2 inhibitor maintenance therapy following intensive salvage therapy: i.e., rate of hematocrit (HCT), duration of maintenance therapy and overall survival in patients with IDH2 mutant relapsed/refractory AML. EXPLORATORY OBJECTIVES: I. Evaluate the changes in IDH2 mutational variant allelic frequency and deoxyribonucleic acid (DNA) methylation signature while on enasidenib therapy. OUTLINE: Patients are assigned to 1 of 2 cohorts. COHORT I: Patients receive enasidenib orally (PO) once daily (QD). Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Patients then undergo a hematopoietic cell transplantation (HCT) 7-14 days after treatment. Within 30-100 days following the transplant, patients receive enasidenib QD. Treatment repeats every 28 days for 24 cycles in the absence of disease progression or unacceptable toxicity. COHORT II: Patients receive enasidenib PO QD. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at 6 months and then yearly for 5 years.

Conditions

• Blasts Under 5 Percent of Peripheral Blood White Cells
• Bone Marrow Blasts Decreased by 50 Percent or More Compared to Pretreatment Level
• IDH2 Gene Mutation
• Recurrent Acute Myeloid Leukemia
• Refractory Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	Enasidenib	Given PO
PROCEDURE	Hematopoietic Cell Transplantation	Undergo HCT

Timeline

• Start date: 2019-12-02
• Primary completion: 2021-11-19
• Completion: 2022-11-19
• First posted: 2019-03-19
• Last updated: 2021-03-10

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study