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

NCT ID: NCT00866281

Last Updated: 2015-12-22

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

22 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-09-30

Study Completion Date

2014-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Acute Myeloid Leukemia Acute Lymphoblastic Leukemia

Keywords

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Acute Myeloid Leukemia AML Acute Lymphoblastic Leukemia ALL midostaurin PKC412 Pediatric relapsed or refractory FLT3 positive Acute Myeloid Leukemia Pediatric relapsed or refractory Mixed-lineage leukemia gene rearranged Acute Lymphoblastic leukemia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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30 mg/m^2 bid

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.

Group Type EXPERIMENTAL

midostaurin

Intervention Type DRUG

Midostaurin 25 mg/mL oral solution was provided in bottles of 50 mL, administered with water. The pediatric starting dose of midostaurin was set at 30 mg/m2 bid and was not to exceed 60 mg/m2 bid.

60 mg/m^2 bid

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.

Group Type EXPERIMENTAL

midostaurin

Intervention Type DRUG

Midostaurin 25 mg/mL oral solution was provided in bottles of 50 mL, administered with water. The pediatric starting dose of midostaurin was set at 30 mg/m2 bid and was not to exceed 60 mg/m2 bid.

Interventions

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midostaurin

Midostaurin 25 mg/mL oral solution was provided in bottles of 50 mL, administered with water. The pediatric starting dose of midostaurin was set at 30 mg/m2 bid and was not to exceed 60 mg/m2 bid.

Intervention Type DRUG

Other Intervention Names

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PKC412

Eligibility Criteria

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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
Minimum Eligible Age

3 Months

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Seattle Children's Hospital CPKC412A2114

Seattle, Washington, United States

Site Status

Novartis Investigative Site

Paris, , France

Site Status

Novartis Investigative Site

Genova, GE, Italy

Site Status

Novartis Investigative Site

Monza, MB, Italy

Site Status

Novartis Investigative Site

Roma, RM, Italy

Site Status

Novartis Investigative Site

Torino, TO, Italy

Site Status

Novartis Investigative Site

Rotterdam, , Netherlands

Site Status

Novartis Investigative Site

Stockholm, , Sweden

Site Status

Countries

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United States France Italy Netherlands Sweden

Other Identifiers

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2008-006931-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CPKC412A2114

Identifier Type: -

Identifier Source: org_study_id