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

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.