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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00471328
First received: April 26, 2007
Last updated: June 5, 2012
Last verified: June 2012
Results First Received: January 12, 2011  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Gastrointestinal Stromal Tumors
Interventions: Drug: Nilotinib
Other: Best Supportive Care (BSC) +/- imatinib or sunitinib

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Patients ongoing on treatment at primary analysis had option to enter extension. Patients in control arm were allowed cross over to Nilotinib arm at disease progression and considered as part of extension. Patients entering the extension part on control arm were permitted to cross over to the nilotinib only upon documented disease progression.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Nilotinib 400 mg was taken orally twice daily in core and extension phase of the study
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.


Participant Flow for 2 periods

Period 1:   Core Phase
    Nilotinib   Control/Cross Over to Nilotinib
STARTED   165   83 
Crossover to Nilotinib:Started Extension   0   53 
COMPLETED   58 [1]   16 [2] 
NOT COMPLETED   107   67 
Adverse Event                18                6 
Death                8                4 
Withdrawal by Subject                2                2 
Abnormal laboratory value                1                0 
Disease Progression                78                53 
Administrative Problem                0                1 
Protocol Deviation                0                1 
[1] Ongoing on treatment during completion of core.
[2] Ongoing on treatment during completion of core

Period 2:   Extension Phase
    Nilotinib   Control/Cross Over to Nilotinib
STARTED   41 [1]   67 
Started:CrossOver Before PrimaryAnalysis   0   53 
Started:CrossOver After PrimaryAnalysis   0   4 
Started:CrossOver During Extension   0   10 
COMPLETED   0   0 
NOT COMPLETED   41   67 
Adverse Event                3                12 
Withdrawal by Subject                0                2 
Death                1                7 
Protocol Deviation                3                1 
Treatment duration as per protocol                0                1 
Disease Progression                32                44 
New cancer Therapy                1                0 
Administrative Problem                1                0 
[1] Out of 58 ongoing patients, 17 patients had progression or died before they could enter extension



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Nilotinib 400 mg was taken orally twice daily in core and extension phase of the study
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.

Total Total of all reporting groups

Baseline Measures
   Nilotinib   Control/Cross Over to Nilotinib   Total 
Overall Participants Analyzed 
[Units: Participants]
 165   83   248 
Age 
[Units: Participants]
     
<=18 years   0   0   0 
Between 18 and 65 years   118   57   175 
>=65 years   47   26   73 
Age 
[Units: Years]
Mean (Standard Deviation)
 57.4  (12.69)   58.6  (10.57)   57.8  (12.01) 
Gender 
[Units: Participants]
     
Female   64   36   100 
Male   101   47   148 
Race (NIH/OMB) 
[Units: Participants]
     
American Indian or Alaska Native   0   0   0 
Asian   21   11   32 
Native Hawaiian or Other Pacific Islander   2   0   2 
Black or African American   2   4   6 
White   134   67   201 
More than one race   0   0   0 
Unknown or Not Reported   6   1   7 


  Outcome Measures
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1.  Primary:   Progression-free Survival (PFS) From Central Radiology Review Based on Primary Analysis (Data Cut-off: June, 2008)   [ Time Frame: Up to 16 months ]

2.  Primary:   Progression-free Survival (PFS) From Local Investigator’s Assessment Based on Treatment Crossover Analysis Set   [ Time Frame: Up to 34 months ]

3.  Secondary:   Overall Survival Based on Primary Analysis (Data Cut-off:June, 2008)   [ Time Frame: Up to 16 months ]

4.  Secondary:   Overall Survival During Core and Extension Phases of the Study   [ Time Frame: Up to 50 months (including core, extension and follow up period) ]

5.  Secondary:   Overall Survival for Treatment Crossover Analysis Set   [ Time Frame: Up to 34 months ]

6.  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)   [ Time Frame: Up to 16 months ]

7.  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   [ Time Frame: Up to 34 months ]

8.  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)   [ Time Frame: Up to 16 months ]

9.  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   [ Time Frame: Up to 34 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862-778-8300



Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00471328     History of Changes
Obsolete Identifiers: NCT00488150
Other Study ID Numbers: CAMN107A2201
2006-002267-11 ( EudraCT Number )
Study First Received: April 26, 2007
Results First Received: January 12, 2011
Last Updated: June 5, 2012
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Belgium: The Federal Public Service (FPS) Health, Food Chain Safety and Environment
Czech Republic: State Institute for Drug Control
Canada: Health Canada
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medical Devices
South Korea: Korea Food and Drug Administration (KFDA)
Italy: The Italian Medicines Agency
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Spain: Ministry of Health and Consumption
Switzerland: Swissmedic
Taiwan: Department of Health
United States: Food and Drug Administration