or
forgot password

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


Phase 1/Phase 2
3 Months
18 Years
Open (Enrolling)
Both
Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia

Thank you

Trial Information

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


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

Type of Study:

Interventional

Study Design:

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

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

Outcome Time Frame:

primarily the first cycle of treatment

Safety Issue:

Yes

Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals

Authority:

United States: Food and Drug Administration

Study ID:

CPKC412A2114

NCT ID:

NCT00866281

Start Date:

September 2009

Completion Date:

November 2013

Related Keywords:

  • Acute Myeloid Leukemia
  • Acute Lymphoblastic Leukemia
  • 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
  • Leukemia
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid

Name

Location
Children's Hospital of Orange County CHOC Cancer Institute Orange, California  92868-3874
University of Colorado Children's Hospital Colorado Aurora, Colorado  80045
Ann & Robert H. Lurie Children's Hospital of Chicago SC Chicago, Illinois  60611
Dana Farber Cancer Institute Deptof DanaFarberCancerInst(4) Boston, Massachusetts  02115
Seattle Children's Hospital CPKC412A2114 Seattle, Washington  98105