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

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00866281
First received: March 19, 2009
Last updated: August 21, 2014
Last verified: August 2014

March 19, 2009
August 21, 2014
September 2009
April 2016   (final data collection date for primary outcome measure)
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 cycle of treatment ] [ Designated as safety issue: Yes ]
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 ]
Complete list of historical versions of study NCT00866281 on ClinicalTrials.gov Archive Site
  • 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 ]
  • 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 ]
Not Provided
Not Provided
 
A Study of the Safety and Preliminary Efficacy of Oral Midostaurin (PKC412) in Relapsed or Refractory Pediatric Leukemia
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

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.

Not Provided
Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Acute Myeloid Leukemia
  • Acute Lymphoblastic Leukemia
Drug: midostaurin
Other Name: PKC412
Experimental: Midostaurin
Intervention: Drug: midostaurin
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
26
April 2016
April 2016   (final data collection date for primary outcome measure)

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
Both
3 Months to 18 Years
No
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals
United States,   France,   Italy,   Netherlands,   Sweden
 
NCT00866281
CPKC412A2114, 2008-006931-11
Not Provided
Novartis ( Novartis Pharmaceuticals )
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Novartis
August 2014

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