Research and Clinical Trials

Brief Description  
The primary objective is to characterize the PK of nilotinib in pediatric patients with imatinib resistant / intolerant Ph+ CML CP or AP, or with Ph+ ALL refractory/relapsed to standard therapy. This study will be conducted as a multi-center, open-label study to characterize the PK of nilotinib in the study population receiving the adult recommended dose (400mg twice daily) scaled to body surface area (BSA) (230 mg/m2 twice daily). Patients will receive one to twelve courses of therapy, based on
Who may be Eligible  

  1. Male or female patients less than 18 years of age at study entry.
  2. Patients must have the diagnosis of imatinib resistant or intolerant Ph+ CML CP or AP or Ph+ ALL either relapsed after or refractory to standard therapy.
  1. Imatinib resistance in Ph+ CML is defined as:
    • Increasing WBC or platelet count while on imatinib therapy indicative of a hematological relapse or primary resistance to imatinib.
    • Cytogenetic or molecular response consistent with suboptimal response or failure(Refer to Post-Text Supplement 2 for definition of suboptimal response).
    • Progression to accelerated phase or blast crisis while on imatinib therapy.
    • Reappearance of Ph+ bone marrow cells after a complete cytogenetic response to imatinib.
    • A greater than 30% increase in Ph+ cells on bone marrow cytogenetics while on imatinib therapy.
    • Loss of molecular response on imatinib therapy.
  2. Imatinib intolerance (at any dose or duration) is defined as the development of AEs requiring discontinuation of imatinib therapy.
  3. Ph+ ALL - relapsed or refractory to prior standard therapy.
Speciality/Disorder  
Pediatric Cancer
IRB Number  
02-11-02A
Principal Investigator  
Oesterheld, Javier

For More Information, Contact  Julie  A, Chasnis
Phone:  (704) 446-5154  Fax:  (704) 355-1188  
Email:  julie.chasnis@carolinashealthcare.org
Address:1025 Morehead Medical Drive Suite 600 Charlotte, NC 28204
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