A Study of the Safety and Preliminary Efficacy of Oral Midostaurin (PKC412) in Relapsed or Refractory Pediatric Leukemia

A Phase I/II, Open-label, Dose-escalating Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Twice Daily Oral Midostaurin and to Evaluate the Preliminary Clinical and Pharmacodynamic Response in Pediatric Patients With Relapsed or Refractory Leukemia

This is a phase I/II pediatric dose-ranging study that will evaluate the safety, tolerability, clinical response, pharmacokinetics and pharmacodynamics of midostaurin in patients <18 years of age who have relapsed or refractory acute leukemias that may benefit from administration of midostaurin, including MLL-rearranged ALL and FLT3 positive AML.

Inclusion Criteria:

• Mixed-lineage leukemia (MLL) gene rearranged Acute Lymphoblastic Leukemia (ALL), that does not respond to treatment or has relapsed from prior treatment; or FLT3 mutated Acute Myeloid Leukemia (AML) that does not respond to a second treatment or has relapsed from 2 prior treatments
• Normal organ function, and chest x-ray
• Expected survival greater than 8 weeks
• Can care for most of personal needs and perform at least minimum activity

Exclusion Criteria:

• Patients with symptomatic leukemic central nervous system involvement or isolated extramedullary leukemia
• Patients must not have received other treatments for leukemia within a predefined time period, 72 hours for medications, 2 months for transplants
• Patients with heart function that is not normal
• Patients with HIV or hepatitis
• Patients with another severe disease or medical condition besides leukemia Other protocol-defined inclusion/exclusion criteria may apply