Efficacy and Safety of Nilotinib (AMN107) Compared With Current Treatment Options in Patients With GIST Who Have Failed Both Imatinib and Sunitinib (ENEST)

Inclusion criteria (Core Phase):

• Age ≥18 years
• Radiological confirmation of disease progression during imatinib and sunitinib therapy OR intolerance to imatinib and/or sunitinib
• At least one measurable site of disease on CT/MRI scan
• Physically fit even if not able to work
• Normal organ, electrolyte, and bone marrow function

Inclusion criteria (Extension Phase):

• Patients whose tumors had progressed on the control arm and had crossed over to the nilotinib arm.
• The study was stopped due to meeting the primary efficacy endpoint of PFS at the interim analysis.
• Patients who were still being treated at the close of the Core study on the control arm or nilotinib arm (whose tumors have not progressed at the time of the end of the Core study).
• Patients must have had documented, confirmed stable, partial or complete response as defined by the RECIST criteria at the time of entry into the Extension study with the exception of patients who had progressed on the control arm.

Exclusion criteria (Core Phase):

• Previous treatment with nilotinib or any other drug in this class or other targeted therapy
• Treatment with any cytotoxic and/or investigational cytotoxic drug ≤ 4 weeks prior to study entry
• Impaired cardiac function
• Use of coumarin derivatives (i.e. warfarin, acenocoumarol, phenprocoumon)
• Women who are pregnant or lactating

Exclusion criteria (Extension Phase):

• Use of other anticancer treatments or investigational drugs (with exception of the study drugs)
• Patients with a history of noncompliance with study drug treatment in the Core study protocol.

Other protocol-defined inclusion/exclusion criteria applied