Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT00471328
Registration number
NCT00471328
Ethics application status
Date submitted
26/04/2007
Date registered
9/05/2007
Date last updated
12/06/2012
Titles & IDs
Public title
Efficacy and Safety of Nilotinib (AMN107) Compared With Current Treatment Options in Patients With GIST Who Have Failed Both Imatinib and Sunitinib
Query!
Scientific title
A Randomized, Open-label, Multi-center Study to Evaluate the Efficacy of Nilotinib Versus Best Supportive Care With or Without a Tyrosine Kinase Inhibitor (Investigator's Choice) in Adult Patients With Gastrointestinal Stromal Tumors Resistant to Both Imatinib and Sunitinib
Query!
Secondary ID [1]
0
0
2006-002267-11
Query!
Secondary ID [2]
0
0
CAMN107A2201
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
ENEST
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Gastrointestinal Stromal Tumors
0
0
Query!
Condition category
Condition code
Cancer
0
0
0
0
Query!
Stomach
Query!
Cancer
0
0
0
0
Query!
Bowel - Small bowel (duodenum and ileum)
Query!
Cancer
0
0
0
0
Query!
Bowel - Back passage (rectum) or large bowel (colon)
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Nilotinib
Other interventions - Best Supportive Care (BSC) +/- imatinib or sunitinib
Experimental: Nilotinib - 400mg twice daily in core and extension phases of the study.
Active Comparator: Control/cross-over to Nilotinib - In core study phase, patients in this arm received Best Supportive Care (BSC) with or without imatinib or sunitinib at the last tolerated dose or at the investigator's choice until documented disease progression followed by cross-over to nilotinib arm.
Patients entering the extension study on this control arm were permitted to cross over to nilotinib arm only upon documented disease progression.
Treatment: Drugs: Nilotinib
Nilotinib 400 mg twice daily (bid)
Other interventions: Best Supportive Care (BSC) +/- imatinib or sunitinib
Can include pain medication, localized radiotherapy, nutritional support, and/or oxygen therapy and blood transfusions. Imatinib or sunitinib can be administered at the last tolerated dose and regimen or at the Investigator's choice.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Intervention code [2]
0
0
Other interventions
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Progression-free Survival (PFS) From Central Radiology Review Based on Primary Analysis (Data Cut-off: June, 2008)
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Up to 16 months
Query!
Primary outcome [2]
0
0
Progression-free Survival (PFS) From Local Investigator's Assessment Based on Treatment Crossover Analysis Set
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
Up to 34 months
Query!
Secondary outcome [1]
0
0
Overall Survival Based on Primary Analysis (Data Cut-off:June, 2008)
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Up to 16 months
Query!
Secondary outcome [2]
0
0
Overall Survival During Core and Extension Phases of the Study
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
Up to 50 months (including core, extension and follow up period)
Query!
Secondary outcome [3]
0
0
Overall Survival for Treatment Crossover Analysis Set
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
Up to 34 months
Query!
Secondary outcome [4]
0
0
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)
Query!
Assessment method [4]
0
0
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).
Query!
Timepoint [4]
0
0
Up to 16 months
Query!
Secondary outcome [5]
0
0
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
Query!
Assessment method [5]
0
0
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).
Query!
Timepoint [5]
0
0
Up to 34 months
Query!
Secondary outcome [6]
0
0
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)
Query!
Assessment method [6]
0
0
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.
Query!
Timepoint [6]
0
0
Up to 16 months
Query!
Secondary outcome [7]
0
0
Overall Clinical Benefit (Complete Response [CR]/Partial Response [PR] or Stable Disease [SD]) From Local Investigator's Assessment Based on Treatment Crossover Analysis Set
Query!
Assessment method [7]
0
0
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.
Query!
Timepoint [7]
0
0
Up to 34 months
Query!
Eligibility
Key inclusion 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.
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
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
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/03/2007
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
1/06/2011
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
248
Query!
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Query!
Recruitment hospital [1]
0
0
Novartis Investigative Site - Auchenflower
Query!
Recruitment hospital [2]
0
0
Novartis Investigative Site - East Melbourne
Query!
Recruitment postcode(s) [1]
0
0
4066 - Auchenflower
Query!
Recruitment postcode(s) [2]
0
0
3002 - East Melbourne
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
District of Columbia
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Florida
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Illinois
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Massachusetts
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Michigan
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Missouri
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
New York
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
North Carolina
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Pennsylvania
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Texas
Query!
Country [12]
0
0
Austria
Query!
State/province [12]
0
0
Vienna
Query!
Country [13]
0
0
Canada
Query!
State/province [13]
0
0
Toronto
Query!
Country [14]
0
0
Czech Republic
Query!
State/province [14]
0
0
Praha 5
Query!
Country [15]
0
0
France
Query!
State/province [15]
0
0
Bordeaux
Query!
Country [16]
0
0
France
Query!
State/province [16]
0
0
Lyon
Query!
Country [17]
0
0
France
Query!
State/province [17]
0
0
Marseille
Query!
Country [18]
0
0
Germany
Query!
State/province [18]
0
0
Berlin
Query!
Country [19]
0
0
Germany
Query!
State/province [19]
0
0
Duesseldorf
Query!
Country [20]
0
0
Germany
Query!
State/province [20]
0
0
Essen
Query!
Country [21]
0
0
Germany
Query!
State/province [21]
0
0
Frankfurt
Query!
Country [22]
0
0
Germany
Query!
State/province [22]
0
0
Hannover
Query!
Country [23]
0
0
Germany
Query!
State/province [23]
0
0
Koln
Query!
Country [24]
0
0
Germany
Query!
State/province [24]
0
0
Mannheim
Query!
Country [25]
0
0
Germany
Query!
State/province [25]
0
0
Muenchen
Query!
Country [26]
0
0
Germany
Query!
State/province [26]
0
0
Tubingen
Query!
Country [27]
0
0
Italy
Query!
State/province [27]
0
0
Bologna
Query!
Country [28]
0
0
Italy
Query!
State/province [28]
0
0
Milan
Query!
Country [29]
0
0
Korea, Republic of
Query!
State/province [29]
0
0
Seoul
Query!
Country [30]
0
0
Netherlands
Query!
State/province [30]
0
0
Leiden
Query!
Country [31]
0
0
Poland
Query!
State/province [31]
0
0
Warszawa
Query!
Country [32]
0
0
Spain
Query!
State/province [32]
0
0
Madrid
Query!
Country [33]
0
0
Switzerland
Query!
State/province [33]
0
0
Chur
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Novartis Pharmaceuticals
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief 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.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT00471328
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Novartis Pharmaceuticals
Query!
Address
0
0
Novartis Pharmaceuticals
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT00471328
Download to PDF