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

This study is currently recruiting participants.

Verified September 2012 by Novartis

Sponsor:

Information provided by (Responsible Party):

Novartis ( Novartis Pharmaceuticals )

ClinicalTrials.gov Identifier:

NCT00866281

First received: March 19, 2009

Last updated: September 5, 2012

Last verified: September 2012

History of Changes

Tracking Information

First Received Date ^ICMJE

March 19, 2009

Last Updated Date

September 5, 2012

Start Date ^ICMJE

September 2009

Estimated Primary Completion Date

April 2013 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

to determine the maximum tolerated dose for two age groups (3 months to 2 years; and >2 years to <18 years) based on the rate of dose-limiting toxicity (DLT) within the equivalent dose ranges studied in adults [ Time Frame: primarily the first 7 days of treatment ] [ Designated as safety issue: Yes ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT00866281 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

to characterize acute and chronic safety and tolerability [ Time Frame: Continuous ] [ Designated as safety issue: Yes ]
to characterize the population pharmacokinetics and trough of single and repeated doses in the pediatric population [ Time Frame: Continous ] [ Designated as safety issue: No ]
to determine the preliminary efficacy, including response rates, time to relapse and overall survival [ Time Frame: Continuous ] [ Designated as safety issue: No ]
to determine the presence and correlate baseline levels of activating mutations or WT-overexpression of the FLT3 gene in AMl and MLL rearranged ALL samples [ Time Frame: Predose, Day 3, and end of treatment ] [ Designated as safety issue: No ]
to evaluate changes in FLT3 phosphorylation following treatment, and correlate with changes in clinical outcome and pharmacokinetics [ Time Frame: Predose, Day 3, and end of treatment ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ^ICMJE

to characterize acute and chronic safety and tolerability [ Time Frame: Continuous ] [ Designated as safety issue: Yes ]
to characterize the pharmacokinetics of single and repeated doses in the pediatric population [ Time Frame: Continous ] [ Designated as safety issue: No ]
to determine the preliminary efficacy, including response rates, time to relapse and overall survival [ Time Frame: Continuous ] [ Designated as safety issue: No ]
to determine the presence and correlate baseline levels of activating mutations or WT-overexpression of the FLT3 gene in AMl and MLL rearranged ALL samples [ Time Frame: Predose, Day 3, and end of treatment ] [ Designated as safety issue: No ]
to evaluate changes in FLT3 phosphorylation following treatment, and correlate with changes in clinical outcome and pharmacokinetics [ Time Frame: Predose, Day 3, and end of treatment ] [ Designated as safety issue: No ]

Current Other Outcome Measures ^ICMJE

Original Other Outcome Measures ^ICMJE

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

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

Brief 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.

Detailed Description

Study Type ^ICMJE

Interventional

Study Phase

Phase 1

Study Design ^ICMJE

Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia

Intervention ^ICMJE

Drug: midostaurin

Other Name: PKC412

Study Arm (s)

Experimental: Midostaurin

Intervention: Drug: midostaurin

Publications *

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

April 2013

Estimated Primary Completion Date

April 2013 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Gender

Both

Ages

3 Months to 18 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Novartis Pharmaceuticals

+1(800)340-6843

Location Countries ^ICMJE

United States, France, Italy, Netherlands, Sweden

Administrative Information

NCT Number ^ICMJE

NCT00866281

Other Study ID Numbers ^ICMJE

CPKC412A2114, 2008-006931-11

Has Data Monitoring Committee

Responsible Party

Novartis ( Novartis Pharmaceuticals )

Study Sponsor ^ICMJE

Novartis Pharmaceuticals

Collaborators ^ICMJE

Investigators ^ICMJE

Study Director:

Novartis Pharmaceuticals

Information Provided By

Novartis

Verification Date

September 2012

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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