Phase 3
N=248
Efficacy and Safety of Nilotinib (AMN107) Compared With Current Treatment Options in Patients With GIST Who Have Failed Both Imatinib and Sunitinib
Gastrointestinal Stromal Tumors
Bottom Line
View on ClinicalTrials.gov: NCT00471328 ↗Enrolled (actual)
248
Serious AEs
44.4%
Results posted
May 2011
Primary outcome: Primary: Progression-free Survival (PFS) From Central Radiology Review Based on Primary Analysis (Data Cut-off: June, 2008) — 109.0; 111.0 days
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Nilotinib (Drug); Best Supportive Care (BSC) +/- imatinib or sunitinib (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Aug 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Progression-free Survival (PFS) From Central Radiology Review Based on Primary Analysis (Data Cut-off: June, 2008) |
109.0; 111.0 | — |
| PRIMARY Progression-free Survival (PFS) From Local Investigator's Assessment Based on Treatment Crossover Analysis Set |
84 | — |
| SECONDARY Overall Survival Based on Primary Analysis (Data Cut-off:June, 2008) |
332.0; 280.0 | — |
| SECONDARY Overall Survival During Core and Extension Phases of the Study |
361; 300 | — |
| SECONDARY Overall Survival for Treatment Crossover Analysis Set |
231 | — |
| SECONDARY Number of Responders With Confirmed Best Overall Response of Complete Response (CR) or Partial Response (PR) From Central Radiology Review During Primary Analysis (Data Cut-off: June, 2008) |
1; 0 | — |
| SECONDARY Number of Responders With Confirmed Best Overall Response of Complete Response (CR) or Partial Response (PR) From Local Investigator's Assessment Based on Treatment Crossover Analysis Set |
1 | — |
| SECONDARY Overall Clinical Benefit (Complete Response [CR]/Partial Response [PR] or Stable Disease [SD]) From Central Radiology Review Based on Primary Analysis (Data Cut-off: June, 2008) |
52.7; 44.6; 7.3; 1.2; 0.6; 0 | — |
| SECONDARY Overall Clinical Benefit (Complete Response [CR]/Partial Response [PR] or Stable Disease [SD]) From Local Investigator's Assessment Based on Treatment Crossover Analysis Set |
37.3; 7.5; 6.0 | — |
Summary
The study evaluated the safety and efficacy of nilotinib versus current treatment in adults with gastrointestinal stromal tumors (GIST) who have either progressed or who were intolerant to the first and second line treatments.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00471328). 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.