ADVL1521, A Phase 2 Study of the MEK inhibitor Trametinib (IND #119346, NSC# 763093) in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Study ID: STU 102017-033
Based on the genetics and predicted biochemical sequelae of increased signaling through the Ras/MaPK pathway, MeK inhibition is a logical treatment approach for patients with JMML. We will collect sequential samples from all patients enrolled to assess the clinical and molecular response at study entry and at disease progression if applicable. This trial will provide an opportunity to interrogate the genetic, biochemical, and functional perturbations of response and resistance to trametinib in patients with JMML.