A Study That Switched Patients From Imatinib to Nilotinib and Then Was Followed by Treatment Cessation (ENESTgoal)

Novartis

Status and phase: Completed; Phase 2

Conditions: CML

Treatments: Drug: nilotinib

Study type: Interventional

Funder types: Industry

Identifiers

NCT01744665

CAMN107AUS37

Details and patient eligibility

About

To evaluate molecular relapse free rates 6 months after stopping nilotinib therapy in patients who achieve MR4.5

Full description

Study protocol included criteria for study termination that was met when > 2 patients lost CCyR during TFR phase (> 1% BCR-ABL); This study was terminated early as > 2 cases of confirmed loss of complete cytogenetic response were reported despite BCR-ABL monitoring during the TFR Phase. All cases achieved MR4.5 after Nilotinib treatment re-initiation and maintained until end of study; trial did not mandate re-initiation within 4 weeks after loss of MMR_ that was a requirement in other Nilotinib TFR trials Initial sample size was 300 patients with CML-CP; Amendment #2 in June 2015 reduced sample size to 59 due to recruitment challenges; Study endpoint analysis and interpretations of data were challenging due to small sample size for early study closure..

Enrollment: 59 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• diagnosis of CML
• Treated with at least 1 year of imatinib
• Bcr-Abl level by PCR must be less than or equal to 0.1% and greater than 0.0032% by PCR reported on the International scale confirmed during screening
• Written informed consent obtained prior to any screening procedures performed

Exclusion criteria

• T315I mutation
• Prior imatinib failure or had accelerated phase or blast crisis CML
• Impaired cardiac function
• Pregnant or lactating women

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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59 participants in 1 patient group

Treatment Free Remission

Experimental group

Description:

Patients entered a monitoring phase for 2 years and received 300 mg nilotinib mg bid. Patients who achieved MR4.5 entered a Consolidation Phase and were treated with nilotinib for 2 years. If MR4.5 was sustained during the Consolidation phase, patients were eligible to stop taking niltoinib during the treatment-free remission (TFR) phase.

Treatment:

Drug: nilotinib