ADVL1521: A Phase 2 Study of the MEK inhibitor Trametinib (IND #119346, NSC# 763093) in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia


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This study is for children with Juvenile Myelomonocytic Leukemia (JMML) that has come back (relapsed) or not responded to treatment (refractory). The overall goal is to find out what effects, good and/or bad, the study drug, Trametinib, has on people with JMML. In addition, the study is trying to determine how well Trametinib works to treat JMML. Trametinib is a drug that works to target certain gene changes (mutations) commonly found in JMML leukemia cells.

Are you Eligible? (Inclusion Criteria)

  • Patient must be greater than 1 month and less than 22 years of age at study entry
  • Patient must have a proven diagnosis of juvenile myelomonocytic leukemia (JMML) at original diagnosis and currently have relapsed or refractory disease.
  • Patient must have previously had at least one cycle of intensive therapy or at least 2 cycles of treatment with a DNA demethylating agent and have persistence of disease.
  • Patients must also meet all other eligibility criteria as outlined in the study protocol

Specialty Area(s)

Childhood and Adolescent Cancers (Pediatric), Pediatric: Leukemia/Lymphoma

Trial Location(s)

CUIMC/Herbert Irving Pavilion
161 Fort Washington Avenue
New York, NY 10032