Phase 4
N=1,089
Nilotinib in Newly Diagnosed Adult Philadelphia Chromosome & /or BCR-ABL Positive Chronic Myeloid Leukaemia in Chronic Phase
CML in Chronic Phase
Bottom Line
View on ClinicalTrials.gov: NCT01061177 ↗Enrolled (actual)
1,089
Serious AEs
19.0%
Results posted
Feb 2017
Primary outcome: Primary: Percentage of Participants With Molecular Response (MR4^0) at 18 Months — 38.3 Percentage of Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Nilotinib (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Jul 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Molecular Response (MR4^0) at 18 Months |
38.3 | — |
| SECONDARY Percentage of Participants Free From Progression to Accelerated Phase/Blast Crisis (AP/BC) at 12 and 24 Months |
99.4; 99.4 | — |
| SECONDARY Rate of Event Free Survival at 12 and 24 Months |
71.7; 69.1 | — |
| SECONDARY Percentage of Participants With Major Molecular Response (MMR) at, as Well as by, 12 and 24 Months |
56.2; 61.1; 68.8; 80.3 | — |
| SECONDARY Percentage of Participants With Complete Cytogenetic Response (CCyR) at, as Well as by, 12 and 24 Months |
72.4; 65.6; 82.5; 89.0 | — |
| SECONDARY Percentage of Participants With Major Cytogenetic Response (MCyR) at, as Well as by, 12 and 24 Months |
73.8; 66.2; 86.7; 91.4 | — |
| SECONDARY Percentage of Participants Free From Progression to AP/BC With MR4^0 at 12 Months |
100.0 | — |
| SECONDARY Percentage of Participants With Event Free Survival in Participants Achieving MR4^0 at 12 Months |
87.0 | — |
| SECONDARY Percentage of Participants With Progression Free Survival (PFS) at 12 and 24 Months |
99.2; 99.0 | — |
| SECONDARY Rate of Molecular Response (MR4^0) at, as Well as by, 12 and 24 Months |
30.7; 40.2; 36.9; 55.0 | — |
| SECONDARY Rate of Molecular Response (MR4^5) at, as Well as by, 12 and 24 Months |
15.2; 21.9; 20.6; 38.4 | — |
| SECONDARY Rate of Complete Hematologic Response (CHR) at, as Well as by, 12 and 24 Months |
89.1; 82.7; 75.5; 86.2 | — |
| SECONDARY Percentage of Participants With Overall Survival at 12 and 24 Months |
99.6; 98.9 | — |
| SECONDARY Rate of Molecular Response (MR4^0) by 18 Months |
48.5 | — |
| SECONDARY Rate of Molecular Response (MR4^5) by 18 Months |
31.6 | — |
| SECONDARY Percentage of Participants With Progression Free Survival in Participants Achieving MR4^0 at 12 Months |
99.2; 99.0 | — |
Summary
This study will assess the efficacy and safety of nilotinib in adult patients with newly diagnosed Philadelphia chromosome positive/BCR-ABL positive chronic myeloid leukaemia in chronic phase. The aim of the study is to confirm the rates of complete molecular remission (CMR) of nilotinib in newly diagnosed CML chronic phase patients in a pan-European population using the EUTOS standardized laboratories.
Eligibility Criteria
Inclusion Criteria
- Patients with diagnosis of CP-CML with cytogenetic confirmation of Philadelphia (Ph) chromosome
- Ph negative cases or patients with variant translocations who are BCR-ABL positive in multiplex PCR are also eligible
- WHO performance status 0-2
- Laboratory assessments within normal limits
- Written informed consent prior to any study procedures being performed
Exclusion Criteria
- Known impaired cardiac function
- History of acute or chronic pancreatitis
- Impaired gastrointestinal function or disease that may alter the absorption of study drug
- Concomitant medications with potential QT prolongation, or known to interact with CYP450 isoenzymes (CYP3A4, CYP2C9, and CYP2C8)
- Patients who have undergone major surgery ≤ 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy
- Patients who are pregnant or breast feeding, or females of reproductive potential not employing an effective method of birth control. Female patients must agree to employ an effective barrier method of birth control throughout the study and for up to 3 months following discontinuation of study drug
Other protocol-defined inclusion/exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT01061177). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.