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Open Label, Phase II Study to Evaluate Efficacy and Safety of Oral Nilotinib in Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia (CML) Pediatric Patients.

Trial Status: Complete

To evaluate the safety, efficacy and pharmacokinetics of nilotinib over time in the Ph+ chronic myelogenous leukemia (CML) in pediatric patients (from 1 to <18 years).

Inclusion Criteria

  • Newly diagnosed and untreated Ph+ CML CP or Ph+ CML CP or AP resistant or intolerant to either imatinib or dasatinib
  • Karnofsky ≥ 50% for patients > 10 years of age and Lansky ≥ 50 for patients ≤ 10 years of age
  • Adequate renal, hepatic and pancreatic function
  • Potassium, magnesium, phosphorus and total calcium values ≥ LLN (lower limit of normal)
  • Written informed consent

Exclusion Criteria

  • Treatment with strong CYP3A4 inhibitors or inducers
  • Use or planned use of any medications that have a known risk or possible risk to prolong the QT interval
  • Acute or chronic liver, pancreatic or severe renal disease
  • History of pancreatitis or chronic pancreatitis.
  • Impaired cardiac function
  • No evidence of active graft vs host and <3mo since Stem Cell Transplant
  • Total body irradiation (TBI) or craniospinal radiation therapy <6months
  • Hypersensitivity to the active ingredient or any of the excipients including lactose.
  • the criteria regarding pregnancy and contraception
  • Active or systemic bacterial, fungal, or viral infection
  • known Hepatitis B, Hepatitis C, or HIV infection

California

Duarte
City of Hope Comprehensive Cancer Center
Status: ADMINISTRATIVELY_COMPLETE
Los Angeles
Children's Hospital Los Angeles
Status: CLOSED_TO_ACCRUAL
Palo Alto
Stanford Cancer Institute Palo Alto
Status: CLOSED_TO_ACCRUAL
San Diego
Rady Children's Hospital - San Diego
Status: CLOSED_TO_ACCRUAL

Georgia

Atlanta
Children's Healthcare of Atlanta - Egleston
Status: ADMINISTRATIVELY_COMPLETE

Illinois

Chicago
Lurie Children's Hospital-Chicago
Status: CLOSED_TO_ACCRUAL

Indiana

Indianapolis
Riley Hospital for Children
Status: COMPLETED

Maryland

Baltimore
Johns Hopkins University / Sidney Kimmel Cancer Center
Status: COMPLETED

Minnesota

Minneapolis
University of Minnesota / Masonic Cancer Center
Status: COMPLETED

North Carolina

Chapel Hill
UNC Lineberger Comprehensive Cancer Center
Status: CLOSED_TO_ACCRUAL
Durham
Duke University Medical Center
Status: COMPLETED

Ohio

Cleveland
Case Comprehensive Cancer Center
Status: ADMINISTRATIVELY_COMPLETE
Columbus
Nationwide Children's Hospital
Status: ACTIVE
Ohio State University Comprehensive Cancer Center
Status: ACTIVE

Tennessee

Memphis
Saint Jude Children's Research Hospital
Status: CLOSED_TO_ACCRUAL

Texas

Dallas
UT Southwestern / Simmons Cancer Center-Dallas
Status: CLOSED_TO_ACCRUAL

Utah

Salt Lake City
Huntsman Cancer Institute / University of Utah
Status: COMPLETED

Virginia

Richmond
Virginia Commonwealth University / Massey Cancer Center
Status: ADMINISTRATIVELY_COMPLETE

Wisconsin

Madison
University of Wisconsin Hospital and Clinics
Status: COMPLETED

The study was designed as a multi-center, open-label, non-controlled phase II study to assess

efficacy, safety and PK parameters of 230 mg/m2 twice daily nilotinib in pediatric patients

(1 to <18 years old). The study population consisted of three cohorts of Ph+ CML pediatric

patients:

- Cohort 1: Ph+ CML-CP patients resistant or intolerant to either imatinib or dasatinib

- Cohort 2: Ph+ CML-AP patients resistant or intolerant to either imatinib or dasatinib

- Cohort 3: Newly-diagnosed Ph+ CML-CP patients in first chronic phase A minimum number of

50 pediatric patients (from 1 to <18 years) were enrolled in the study. Of them, at

least 15 patients were Ph+ CML-CP patients resistant or intolerant to either imatinib or

dasatinib, and at least 15 were newly-diagnosed Ph+ CML-CP patients in first chronic

phase patients. There was no minimum number of patients required for Ph+ CML-AP patients

resistant or intolerant to either imatinib or dasatinib.

Based on enrollment forecasts as of Jan 2015, and to reflect the agreements with the US FDA

and the PDCO, the study remained open for enrollment until the targeted number of 50 patients

with at least 15 newly diagnosed Ph+CML patients was achieved or until 31May2015, whichever

was later.

Patients who completed the study were treated with nilotinib for a total of 66 cycles of 28

days unless the patient prematurely discontinued study treatment.

The primary analysis cut-off date was the date when all patients enrolled in the trial either

completed their visit for treatment cycle 12 or had discontinued study treatment early

(EoT/early discontinuation visit). These analyses were reported in the 12-cycle clinical

study report (CSR). A 24-cycle analysis was done when all patients had either completed their

24-cycle treatment visit or had discontinued study treatment early.

At trial end, a final comprehensive CSR of all data collected during the trial was produced.

Trial Phase Phase II

Trial Type Treatment

Lead Organization
Novartis Pharmaceuticals Corporation

  • Primary ID CAMN107A2203
  • Secondary IDs NCI-2014-01948, 2013-000200-41
  • Clinicaltrials.gov ID NCT01844765