Phase I Study of Oral AG-120 for Advanced Blood Cancers with IDH1 Mutation Evaluating Safety and Effectiveness

What this Trial Is About

Researchers are evaluating AG-120, an oral drug, in adults with advanced blood cancers that have an IDH1 mutation. This Phase I, multicenter study aims to assess the safety, how the drug moves and works in the body, and its clinical activity. The study includes patients with relapsed or refractory acute myeloid leukemia AML, untreated AML, other IDH1-mutated blood cancers, and myelodysplastic syndromes, focusing on those with specific gene mutations to better understand treatment options for these conditions. Participants receive AG-120 daily as a single oral dose in 28-day cycles. The study is divided into two parts first, a dose escalation phase where doses increase to find the maximum tolerated or recommended Phase II dose, followed by a dose expansion phase with four patient groups receiving the identified dose to further evaluate safety and activity. There is also a substudy focused on patients with relapsed or refractory myelodysplastic syndrome to assess similar outcomes. Treatment continues until the disease worsens or unacceptable side effects occur. During the study, participants undergo regular evaluations including bone marrow biopsies, blood and urine sampling, and safety monitoring. Researchers track adverse events, dose tolerance, drug levels in the body, and the drugs effect on disease markers. Clinical activity is assessed using established criteria for AML and myelodysplastic syndromes. The study period for these assessments typically lasts up to 26 weeks. Participants are closely monitored for safety and treatment effects throughout their involvement.

Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation