Study to Assess Efficacy and Safety of Nilotinib 300mg Twice Daily in Patients With Philadelphia Positive Chronic Myeloid Leukaemia (CML) in Chronic Phase Who Are Intolerant to Prior Tyrosine Kinase Inhibitors.
Condition: Philidelphia Positive Chronic Myeloid Leukaemia
MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Intervention: Nilotinib
Intervention description: Nilotinib 150mg hard gelatin capsules taken orally
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 20.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: CML; Ph-CML; leukaemia; leukemia; chronic myeloid leukemia; chronic myeloid leukaemia; TKI intolerance; tyrosine kinase inhibitor; tyrosine kinase inhibitor intolerance

Summary:

      The purpose of this Australian study was to assess the efficacy and safety of nilotinib 300mg
      twice daily in patients with chronic myeloid leukemia chronic phase who were intolerant but
      responsive to 1st line treatment with imatinib or dasatinib. Eligible patients have been
      previously treated with imatinib or dasatinib for at least 3 months and are experiencing
      non-hematologic toxicity whilst having documented responses that meet PBS authority for 1st
      line treatment of CML without current MR4.5.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Written informed consent prior to screening procedures

          2. Eastern Cooperative Oncology Grou (ECOG) Performance Status of 0, 1, or 2.

          3. Patient with diagnosis of Ph+ CML-CP associated with BCR-ABL quantifiable by RQ-PCR
             (IS).

          4. Patient has received a minimum of 3 months of imatinib or dasatinib treatment (any
             dose) since initial diagnosis with a documented response.

          5. Patient is eligible for Pharmaceutical Benefits Scheme (PBS) reimbursed 1st line TKI
             treatment.

          6. Patient has experienced non-hematological Adverse Events (AE(s)) of any grade, which
             persisted for at least 1 month despite supportive care or recurred at any grade at
             least once. Patients who, at the Investigator's discretion, require immediate
             discontinuation due to the severity of the adverse event are also eligible.

          7. No other current or planned anti-leukemia therapies.

          8. Adequate organ function.

          9. Potassium, Magnesium and Total Calcium above Lower limit of normal.

         10. life expectancy of more than 12 months in the absence of any intervention

        Key Exclusion Criteria:

          1. Prior treatment with nilotinib.

          2. Prior Accelerated Phase (AP), Blast Crisis (BC) or allogeneic-transplant (unless the
             patient received an autologous transplant and was in Chrionic Phase (CP) prior to
             transplant and never in AP or BC).

          3. Patient has documented Molecular Response (MR) 4.5 at the time of study entry

          4. Patients with atypical BCR-ABL transcript not quantifiable by standard RQ-PCR.

          5. Known impaired cardiac function.

          6. Impairment of gastrointestinal (GI) function or GI disease that may significantly
             alter the absorption of study drug.

          7. Pregnant or breast feeding (lactating) women.

          8. Women of child-bearing potential unwilling or unable to use highly effective
             contraception.