A Randomized Phase III Study to Assess the Effect of a Longer Duration of Consolidation Treatment With Nilotinib on TFR in CP CML.
Condition: Philadelphia Chromosome Positive (PH+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP)
MeSH terms: Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Philadelphia Chromosome
Intervention: Nilotinib
Intervention description: Participants received a daily oral nilotinib dose of 300 mg BID, given as two 150 mg capsules BID.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: All participants received 24 months of study treatment. After 24 months of treatment, eligible participants (i.e. those deemed to have achieved sustained molecular response) were randomized to one of the two study arms on a continued open-label basis.
Participants not achieving sustained molecular response after 24 months of treatment were not randomized but remained in the study until the 5-year study period was completed (Not randomized arm).
Masking: None (Open Label)
Enrollment: 620.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Adult; Chronic myelogenous leukemia (CML); Chronic myeloid leukemia (CML); Chronic myelocytic leukemia (CML); Acute Lymphoblastic Leukemia (ALL) Philadelphia chromosome positive; Acute Lymphoid Leukemia; Suboptimal molecular response

Summary:

      This study aimed to assess the optimal duration of nilotinib 300 mg twice daily (BID)
      consolidation treatment in patients with Philadelphia chromosome-positive (Ph+) chronic
      myeloid leukemia (CML), in order that patients remained in treatment-free remission (≥MR4.0)
      without molecular relapse 12 months after starting the Treatment-Free Remission (TFR) phase.
    

Eligibility criteria:

        Key Inclusion Criteria:

          -  Confirmed diagnosis of chronic phase Ph+ CML

          -  Previous first-line treatment with imatinib for a minimum of 2 years;

          -  Patient in complete cytogenetic response;

        Key Exclusion Criteria:

          -  Previous achievement of MR4.0 at study entry;

          -  Previous treatment with other target cells inhibitors other than imatinib;

          -  Patients with any history of detectable atypical Leukemia transcripts or patients with
             detectable atypical leukemia transcripts at screening;

          -  Previous anticancer agents for Chronic myeloid leukemia other than imatinib except for
             cytoreduction;

          -  Severe and/or uncontrolled concurrent medical disease that in the opinion of the
             investigator could cause unacceptable safety risks or compromise compliance with the
             protocol;

          -  History of other active malignancies within the 5 years prior to study entry with the
             exception of previous or concomitant basal cell skin cancer and previous carcinoma in
             situ treated curatively;

          -  Patients who have not recovered from prior surgery;

          -  Treatment with other investigational agents within 4 weeks of Day 1;

          -  Impairment of gastrointestinal (GI) function or GI disease that may significantly
             alter the absorption of study drug

        Other inclusion/exclusion criteria might apply.