A Pharmacokinetic (PK) Study of Nilotinib in Pediatric Patients With Philadelphia Chromosome-positive (Ph+) Chronic Myelogenous Leukemia (CML) or Acute Lymphoblastic Leukemia (ALL)
Condition: Chronic Myeloid Leukemia; Acute Lymphoblastic Leukemia
MeSH terms: Leukemia; Leukemia, Myeloid; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Lymphoid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Intervention: Nilotinib
Intervention description: Nilotinib capsules were delivered in bottles with dose strengths of 50mg, 150mg and 200mg.
Patients were administered nilotinib 230 mg/m2 (per BSA) bid, orally, rounded to the nearest 50 mg (max single dose 400 mg) for 28 days (1 cycle) for up to 12 cycles prior to protocol amendment 3 and up to 24 cycles post amendment 3. Capsules were to be swallowed whole with water. Apple sauce (puréed apple) may have been used as a vehicle for dosing where capsules were not able to be swallowed whole with water.
Phase: Phase 1
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 15.0
Sex: All
Minimum age: 1 Year
Maximum age: 17 Years
Healthy volunteers: No
Keywords: Chronic Myeloid Leukemia; Acute Lymphoblastic Leukemia; Ph+ CML; Ph+ ALL; pediatric; nilotinib; imatinib; chronic phase; accelerated phase; newly diagnosed Ph+ CML; dasatinib

Summary:

      This study will assess the pharmacokinetics of nilotinib in Ph+ CML pediatric patients that
      are newly diagnosed or resistant or intolerant to imatinib or dasatinib or refractory or
      relapsed Ph+ ALL compared to the adult populations. It will also evaluate safety and activity
      of nilotinib as secondary objectives.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Must have one of the following: newly diagnosed CP Ph+CML, CP or AP resistant/
             intolerant to imatinib and/or dasatinib, or Ph+ ALL either relapsed after or
             refractory to standard therapy

          -  adequate renal, hepatic and pancreatic function

        Exclusion Criteria:

          -  patients receiving therapy with strong CYP3A4 inhibitors and/or inducers and
             treatments cannot be stopped or changed to a different medication at least 14 days
             prior to starting study drug

          -  patients receiving therapy with any medications with a known risk or possible risk to
             prolong the QT interval and the treatment cannot be either discontinued or switched to
             a different medication prior to starting study drug.

          -  gastrointestinal impairment or disease that may interfere with drug absorption

          -  liver, pancreatic or severe renal disease unrelated to disease under study

          -  impaired cardiac function

          -  patients who received dasatinib within 3 days of starting study drug

          -  patients who received imatinib within 5 days of starting study drug

          -  patients receiving hydroxyurea or corticosteroids that has not been discontinued at
             least 1 week after initiation of nilotinib

          -  patients who received hematopoietic growth factors within 7 days of starting study
             drug or Pegfilgrastim (Neulasta®) within 14 days of starting study drug

          -  patients with Stem Cell Transplant (SCT) or Rescue without TBI: Evidence of active
             graft vs. host disease and < 3 months since SCT

        Other protocol-defined inclusion/exclusion criteria may apply