The Protein Tyrosine Kinase Inhibitor Nilotinib as First-line Treatment of Ph+, BCR-, ABL+ Chronic Myeloid Leukemia (CML) in Early Chronic Phase: a Phase IIIb, Multicenter Study to Assess the Complete Molecular Response
This study is an open-label, multicentric, phase IIIb study of NILOTINIB administred orally
at the dose of 300 mg twice daily (total daily dose 600 mg daily) for 24 months (study
core), and indefinitely if it is in the interest of the patient (the drug will be given
free-of-charge after 24 months to all those patients achieving the CMR4 at 24 months and
in absence of safety concerns). Nilotinib dose is increased to 400 mg BID in case of
suboptimal response or failure (with the exception of patients who will fail for progression
to ABP: in case of progression to ABP, the patient will not be treated with study drug and
the choise of the treatment will be up to the physician).
Study duration is estimated in 6 years, 1 year of estimated enrollment, 2 years therapy
duration. Thereafter, information on course and survival is due for other 3 years.
The main data analysis will be performed when all patients will complete 24 months of
treatment (or discontinued earlier). Safety and tolerability profile will be assessed by
collecting hematologic and non-hematologic adverse events, laboratory examinations and ECG
data. The molecular response will be assessed using the GIMEMA standardized molecular
laboratories (Labnet network).
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Complete molecular response
To assess the complete molecular response (CMR4) rate at 24 months of treatment. For the purpose of this protocol, CMR is defined as a negative results of quantitative RT-PCR for BCR-ABL transcripts in a peripheral blood sample of at least 10 ml with a minimum sensitivity of 1:10,000, that corresponds to at least a 4-log reduction (hence, CMR4)
At 24 months of treatment
Italy: Ethics Committee