Phase 2 Study of Enasidenib Alone or with Azacitidine to Treat Patients Aged 12 and Older with IDH2-Mutated High-Risk Myelodysplastic Syndrome

What this Trial Is About

Researchers are evaluating the safety, side effects, and effectiveness of azacitidine and enasidenib for patients with myelodysplastic syndrome MDS that has an IDH2 gene mutation. This phase II trial focuses on patients with high-risk MDS, including those who have never received hypomethylating agents HMA and those who have relapsed or are refractory to prior HMA therapy. The study aims to understand how these treatments impact cancer cell growth and patient outcomes over time. Participants are assigned to one of two groups based on their treatment history. Patients who are HMA-naive receive enasidenib orally daily for 28 days and azacitidine either intravenously or subcutaneously on days 1 through 7, with cycles repeating every 28 days if there is no disease progression or unacceptable side effects. Patients who have relapsed or are refractory to HMA therapy receive enasidenib orally daily for 28 days, with similar 28-day cycles. After finishing treatment, patients are followed every three months for up to three years. During the study, participants undergo various assessments including monitoring for side effects, measuring response rates, and evaluating overall and event-free survival. Researchers also study molecular markers related to tumor activity and resistance, as well as changes in DNA methylation and cellular differentiation. Quality of life is evaluated using a specific MDS questionnaire. Safety monitoring continues throughout the treatment and follow-up periods, which can last up to three years after treatment completion.

Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome