Trial · NCT00471328

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

NCT00471328, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2011-05-17

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy and Safety of Nilotinib (AMN107) Compared With Current Treatment Options in Patients With GIST Who Have Failed Both Imatinib and Sunitinib. Trials, record NCT00471328. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00471328

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Efficacy and Safety of Nilotinib (AMN107) Compared With Current Treatment Options in Patients With GIST Who Have Failed Both Imatinib and Sunitinib
NCT number: NCT00471328
Status: Completed
Phases: Phase 3
Enrollment: 248 participants, actual
Lead sponsor: Novartis Pharmaceuticals
Conditions: Gastrointestinal Stromal Tumors
Interventions: Nilotinib, Best Supportive Care (BSC) +/- imatinib or sunitinib
First posted: 2007-05-09
Last updated: 2012-06-12
Other registries' IDs: eudract:2006-002267-11
Results first posted: 2011-05-17

Source ClinicalTrials.gov

Registered outcomes9

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Progression-free Survival (PFS) From Central Radiology Review Based on Primary Analysis (Data Cut-off: June, 2008)	Up to 16 months	Progression-free survival (PFS) is the time from date of randomization to the date of first documented progression or death due to any cause. If a participant has not had an event, progression-free survival is censored at the time of last adequate tumor assessment. Progression is defined according to Modified Response Evaluation Criteria in Solid Tumors (modified RECIST) criteria as at least a 20% increase in the sum of the longest diameter of target lesions, worsening of the non-target lesions or the appearance of one or more new lesions.
Primary	Progression-free Survival (PFS) From Local Investigator's Assessment Based on Treatment Crossover Analysis Set	Up to 34 months	PFS is defined as the time from the first date of cross-over to nilotinib therapy from the control arm to the date of the first observation of documented disease progression. Tumor assessment was based on the local investigator's measurement using Modified Response Evaluation Criteria in Solid Tumors (modified RECIST) criteria. Progression is defined according to modified RECIST criteria as at least a 20% increase in the sum of the longest diameter of target lesions, worsening of the non-target lesions or the appearance of one or more new lesions.
Secondary	Overall Survival Based on Primary Analysis (Data Cut-off:June, 2008)	Up to 16 months	Overall survival (OS) is defined as the time from date of randomization to date of death due to any cause. If a participant is not known to have died, survival will be censored at the date of last contact.
Secondary	Overall Survival During Core and Extension Phases of the Study	Up to 50 months (including core, extension and follow up period)	Overall survival (OS) is defined as the time from date of randomization to date of death due to any cause. If a participant is not known to have died, survival will be censored at the date of last contact. This analysis included both Core and Extension data as well as survival follow up data.
Secondary	Overall Survival for Treatment Crossover Analysis Set	Up to 34 months	For patients who crossed-over to nilotinib from the control arm, the overall survival was the time from the first dose date of nilotinib after switching from control arm to the date of death due to any cause. If death was not observed, the OS was censored at the latest date the patient was known to be alive.
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)	Up to 16 months	The best overall response is the best response recorded from randomization until disease progression. The CR/PR must be confirmed by at least two determinations at least 4 weeks apart before progression. Using modified RECIST criteria, a Complete Response is defined as disappearance of all lesions, and a Partial Response is defined as either 1) at least a 30% decrease in the sum of the longest diameter of target lesions and no new lesions or progression of non-target lesions or 2) disappearance of all target lesions but persistence of one or more non-target lesion(s).
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	Up to 34 months	The best overall response (CR/PR) must be confirmed by at least two determinations at least 4 weeks apart before progression. Using modified RECIST criteria, a Complete Response is defined as disappearance of all lesions, and a Partial Response is defined as either 1) at least a 30% decrease in the sum of the longest diameter of target lesions and no new lesions or progression of non-target lesions or 2) disappearance of all target lesions but persistence of one or more non-target lesion(s).
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)	Up to 16 months	The overall clinical benefit includes the best overall responses of CR, PR, or SD. The best overall responses of CR/PR must be confirmed by at least two determinations at least 4 weeks apart before progression. The best overall response of SD must have at least one SD (or better) at least 6 weeks (or 6 months or 12 months as applicable) after randomization but before progression.
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	Up to 34 months	The overall clinical benefit includes the best overall responses of CR, PR, or SD. The best overall responses of CR/PR must be confirmed by at least two determinations at least 4 weeks apart before progression. The best overall response of SD must have at least one SD (or better) at least 6 weeks (or 6 months or 12 months as applicable) after randomization but before progression.

Source ClinicalTrials.gov

Outcomes9

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Progression-free Survival (PFS) From Central Radiology Review Based on Primary Analysis (Data Cut-off: June, 2008)	Up to 16 months	The intent to treat (ITT) population was defined as all randomized patients and was used as the primary efficacy population.	days	MEDIAN
SECONDARY	Overall Survival Based on Primary Analysis (Data Cut-off:June, 2008)	Up to 16 months	The intent to treat (ITT) population was defined as all randomized participants and was used as the primary efficacy population.	days	MEDIAN
SECONDARY	Overall Survival During Core and Extension Phases of the Study	Up to 50 months (including core, extension and follow up period)	Core Full Analysis Set (Core FAS): included all randomized patients included in the Core study. Analyses based on Core FAS does not account for treatment crossover i.e.pooling all data both before and after crossover. This analysis set was used to conduct an overall survival analysis.	days	MEDIAN
SECONDARY	Overall Survival for Treatment Crossover Analysis Set	Up to 34 months	Treatment Crossover Analysis Set: All patients who crossed over from control arm to nilotinib after completing the Core study or during the Extension study after disease progression.	days	MEDIAN
PRIMARY	Progression-free Survival (PFS) From Local Investigator's Assessment Based on Treatment Crossover Analysis Set	Up to 34 months	Treatment Crossover Analysis Set: All patients who crossed over from control arm to nilotinib after completing the Core study or during the Extension study after disease progression.	days	MEDIAN
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)	Up to 16 months	The intent to treat (ITT) population was defined as all randomized patients and was used as the primary efficacy population.	Participants	NUMBER
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	Up to 34 months	Treatment Crossover Analysis Set: All patients who crossed over from control arm to nilotinib after completing the Core study or during the Extension study after disease progression.	Participants	NUMBER
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)	Up to 16 months	The intent to treat (ITT) population was defined as all randomized patients and was used as the primary efficacy population.	Percentage of Participants	NUMBER
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	Up to 34 months	Treatment Crossover Analysis Set: All patients who crossed over from control arm to nilotinib after completing the Core study or during the Extension study after disease progression.	Percentage of Participants	NUMBER

Source ClinicalTrials.gov

Results by group20

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Progression-free Survival (PFS) From Central Radiology Review Based on Primary Analysis (Data Cut-off: June, 2008)	Nilotinib	109.0	95% Confidence Interval 61.0 to 113.0	days
Progression-free Survival (PFS) From Central Radiology Review Based on Primary Analysis (Data Cut-off: June, 2008)	Control/Cross Over to Nilotinib	111.0	95% Confidence Interval 60.0 to 116.0	days
Overall Survival Based on Primary Analysis (Data Cut-off:June, 2008)	Nilotinib	332.0	95% Confidence Interval 246.0 to NA	days
Overall Survival Based on Primary Analysis (Data Cut-off:June, 2008)	Control/Cross Over to Nilotinib	280.0	95% Confidence Interval 240.0 to 372.0	days
Overall Survival During Core and Extension Phases of the Study	Nilotinib	361	95% Confidence Interval 265 to 425	days
Overall Survival During Core and Extension Phases of the Study	Control/Cross Over to Nilotinib	300	95% Confidence Interval 246 to 386	days
Overall Survival for Treatment Crossover Analysis Set	Control/Cross Over to Nilotinib	231	95% Confidence Interval 196 to 327	days
Progression-free Survival (PFS) From Local Investigator's Assessment Based on Treatment Crossover Analysis Set	Control/Cross Over to Nilotinib	84	95% Confidence Interval 56 to 109	days
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)	Nilotinib	1	0.0 to 3.3	Participants
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)	Control/Cross Over to Nilotinib	0	0.0 to 0.0	Participants

1 to 10 of 20Source ClinicalTrials.gov

See all 20 results by group

Participant flow20

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Core Phase	STARTED	Nilotinib	165
Core Phase	STARTED	Control/Cross Over to Nilotinib	83
Core Phase	Crossover to Nilotinib:Started Extension	Nilotinib	0
Core Phase	Crossover to Nilotinib:Started Extension	Control/Cross Over to Nilotinib	53
Core Phase	COMPLETED	Nilotinib	58
Core Phase	COMPLETED	Control/Cross Over to Nilotinib	16
Core Phase	NOT COMPLETED	Nilotinib	107
Core Phase	NOT COMPLETED	Control/Cross Over to Nilotinib	67
Extension Phase	STARTED	Nilotinib	41
Extension Phase	STARTED	Control/Cross Over to Nilotinib	67

1 to 10 of 20Source ClinicalTrials.gov

See all 20 participant flow

Adverse events495

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Anaemia	Blood and lymphatic system disorders	Nilotinib(Core +Extension)	39	165
other	Anaemia	Blood and lymphatic system disorders	Control(Core + Extension)	17	83
other	Anaemia	Blood and lymphatic system disorders	Crossover Nilotinib Therapy	12	67
other	Neutropenia	Blood and lymphatic system disorders	Nilotinib(Core +Extension)	1	165
other	Neutropenia	Blood and lymphatic system disorders	Control(Core + Extension)	7	83
other	Neutropenia	Blood and lymphatic system disorders	Crossover Nilotinib Therapy	0	67
other	Eyelid oedema	Eye disorders	Nilotinib(Core +Extension)	3	165
other	Eyelid oedema	Eye disorders	Control(Core + Extension)	7	83
other	Eyelid oedema	Eye disorders	Crossover Nilotinib Therapy	0	67
other	Abdominal distension	Gastrointestinal disorders	Nilotinib(Core +Extension)	17	165

1 to 10 of 495Source ClinicalTrials.gov

See all 495 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Nilotinib(Core +Extension)
Total, all-cause mortality	Control(Core + Extension)
Total, all-cause mortality	Crossover Nilotinib Therapy
Total, other adverse events	Nilotinib(Core +Extension)	159	165
Total, other adverse events	Control(Core + Extension)	73	83
Total, other adverse events	Crossover Nilotinib Therapy	58	67
Total, serious adverse events	Nilotinib(Core +Extension)	81	165
Total, serious adverse events	Control(Core + Extension)	27	83
Total, serious adverse events	Crossover Nilotinib Therapy	32	67

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
Nilotinib vs Control/Cross Over to Nilotinib: 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)

Source Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT00471328

GET https://conductscience.org/api/trials/v1/NCT00471328/events?page=2

API reference For AI assistants