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

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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)

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

Trial contacts and locations

8

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Data sourced from clinicaltrials.gov

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