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

Novartis

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia

Treatments: Drug: midostaurin

Study type: Interventional

Funder types: Industry

Identifiers

NCT00866281

CPKC412A2114

2008-006931-11 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 22 patients

Sex: All

Ages: 3 months to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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22 participants in 2 patient groups

30 mg/m^2 bid

Experimental group

Description:

Participants received bodyweight and body surface area (BSA) stratified dose of midostaurin 30 mg/m\^2 twice daily (bid) through oral route. The total daily dose in 30 mg/m\^2 bid cohort was 60 mg/m\^2.

Treatment:

Drug: midostaurin

60 mg/m^2 bid

Experimental group

Description:

Participants received bodyweight and BSA stratified dose of midostaurin 60 mg/m\^2 bid through oral route. The total daily dose in 60 mg/m\^2 bid cohort was 120 mg/m\^2.

Treatment:

Drug: midostaurin