Prospective multicenter clinical study evaluating efficacy and safety of Nilotinib in Ph chromosome-positive (Ph+) CML patients with Imatinib resistance or intolerance

UMIN ID: UMIN000002201

Registered date:15/07/2009

Basic Information

• Recruitment status: Complete: follow-up complete
• Health condition(s) or Problem(s) studied: Chronic myelogenous leukemia
• Date of first enrollment: 2009/03/06
• Target sample size: 40
• Countries of recruitment: Japan
• Study type: Interventional
• Intervention(s): Nilotinib will be administered twice daily at a dose of 400 mg (800 mg/day) for 1 year.

Outcome(s)

• Primary Outcome: To assess the efficacy of twice daily administration of Nilotinib at a dose of 400 mg in Ph chromosome-positive (Ph+) CML patients with Imatinib resistance or intolerance based on the rate of major molecular response at 12 months after starting treatment.
• Secondary Outcome: 1) To assess the rate of complete cytogenetic response (CCyR) at 12 months. 2) To assess the times to achieve a CCyR and a major molecular response (MMR) every 3 months. 3) To assess durations of MMR every 3 months. 4) To assess the progression-free survival (PFS), the event-free survival (EFS), and the overall survival (OS) at 5 years. 5) To assess the actual dose intensity at 12 months in the groups which have achieved a CCyR or a MMR and the groups which have not achieved a CCyR or a MMR. 6) To assess patient profiles such as BCR-ABL point mutations and the blood levels of Imatinib before Nilotinib treatment. 7) To assess profiles of BCR-ABL point mutations at 12 months in the groups which have achieved a CCyR and a MMR and in the groups which have not achieved a CCyR and a MMR. 8) To assess the safety of twice daily administration of Nilotinib 400 mg dosage.

Key inclusion & exclusion criteria

• Age minimum: 20years-old
• Age maximum: 80years-old
• Gender: Male and Female
• Exclude criteria: 1) Patient who exhibits the T315I BCR-ABL mutation. 2) Patient who is pregnant or breastfeeding. 3) Patient does not agree to use contraceptive methods to prevent pregnancy. 4) Patient who has any cardiac disturbances including the following conditions. (1) Unmeasurable QT interval by ECG (2) Complete left bundle branch block (3) Use of a ventricular pacemaker (4) Congenital long QT syndrome or family history of long QT syndrome (5) History or complication of serious ventricular or atrial tachycardia (6) Clinically serious resting bradycardia(<50 bpm) (7) History of clinically diagnosed myocardial infarction (8) History of unstable angina within 12 months before start of study (9) Other clinically important heart diseases 5) Patient who has a history of non-adherence to medications or patient from whom an informed consent can not be obtained. 6) Patient who has any gastrointestinal disorders or diseases which may affect the absorption of the study drug. 7) Patients who has acute or chronic hepatic disease, pancreatic disease, or severe renal disease unrelated to the primary disease.

Related Information

• Primary Sponsor: East Japan CML Study Group (EJCML)
• Source(s) of Monetary Support: NPO Ibaraki hematology, Oncology & Palliative Expert Meeting (IB-HOPE)