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

• Status: Terminated
• Trial end date: 2014-09-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Treatments: 4'-N-benzoylstaurosporine; Midostaurin; Staurosporine

Criteria

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