Open Label, Phase II Study to Evaluate Efficacy and Safety of Oral Nilotinib in Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia (CML) Pediatric Patients.
Condition: Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia
MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Study type: Interventional
Phase: Phase 2

Summary: 
      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).
    

Detailed description: 
      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.
    

Intervention name(s): nilotinib
Intervention type(s): Drug
Intervention description(s): Nilotinib supplied in 50mg, 150mg, and 200mg capsules. It was administered orally at 230mg/m2, twice daily for up to 66 cycles (1 cycle = 28 days). Dose administration was rounded to the nearest 50mg dose (to a maximum dose of 400mg).
Other intervention name(s): AMN107
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Tasigna; nilotinib treatment; chronic phase; Ph+ CML; accelerated phase; newly diagnosed Ph+ CML; pediatric; leukemia; leukemia, pediatric; leukemia, myleiod; leukemia, mylegenous, chronic; leukemia, mylegenous, accelerated; BCR-ABL positive; myeloproliferative disorder; bone marrow disease; hematologic diseases; neoplastic processes; imatinib; dasatinib; enzyme inhibitor; protein kinase inhibitor