BEZ235 Phase II Trial in Patients With Advanced Pancreatic Neuroendocrine Tumors (pNET) After Failure of mTOR Inhibitor Therapy.

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01658436
First received: July 6, 2012
Last updated: April 19, 2016
Last verified: April 2016
Results First Received: March 11, 2016  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Pancreatic Neuroendocrine Tumors (pNET)
Intervention: Drug: BEZ235 (Stage 1)

  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

This was a Phase II, two-stage, multicenter study, where Stage 1 was a singlearm,open label design and Stage 2 was planned to be a randomized, double-blind study.

However, at the end of Stage 1, the futility was met and hence the Stage 2 was not initiated.


Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Initially , the patients were started at BEZ235 400mg bid dose regimen. The preliminary safety & tolerability data fro first 3 patients treated at this dose showed all patients reported AEs leading to dose interruption. It was decided to decrease the dose of BEZ235 to 300mg bid dose regimen.

Reporting Groups
  Description
BEZ235 300 mg Oral BEZ235 300 mg bid was investigated in stage 1 of study
BEZ235 400 mg Bid Oral BEZ235 400 mg bid was investigated in stage 1 of study

Participant Flow:   Overall Study
    BEZ235 300 mg     BEZ235 400 mg Bid  
STARTED     20     11  
COMPLETED     0     0  
NOT COMPLETED     20     11  
Adverse Event                 6                 5  
Physician Decision                 1                 0  
Progressive disease                 12                 4  
Study terminated by sponsor                 0                 1  
Subject/guardian decision                 0                 1  
Death                 1                 0  



  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.
The Full Analysis Set (FAS) comprised of all patients who received at least one dose of study treatment (Stage 1). This set was known as ‘Analysis Set Stage 1’ (AS1).

Reporting Groups
  Description
BEZ235 300 mg/400 mg Bid Oral BEZ235 300 mg/400 mg bid was investigated in stage 1 of study

Baseline Measures
    BEZ235 300 mg/400 mg Bid  
Number of Participants  
[units: participants]
  31  
Age  
[units: Years]
Mean (Standard Deviation)
  60.1  (11.4)  
Gender  
[units: Participants]
 
Female     13  
Male     18  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Stage 1 - Progression Free Survival (PFS) Rate Analysis at 16 Weeks as Per Local Radiology Review   [ Time Frame: 16 weeks after the first BEZ235 administration. ]

2.  Secondary:   Stage 1- Overall Response Rate (ORR)   [ Time Frame: Baseline, every 8 weeks up to 31 months ]

3.  Secondary:   Stage 1 - Disease Control Rate   [ Time Frame: Baseline, every 8 weeks up to 31 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: Clinical Disclosure Office
Organization: Novartis Pharmaceuticals
phone: 862-778-8300
e-mail: trialandresults.registries@novartis.com



Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01658436     History of Changes
Other Study ID Numbers: CBEZ235F2201
2012-000675-16 ( EudraCT Number )
Study First Received: July 6, 2012
Results First Received: March 11, 2016
Last Updated: April 19, 2016
Health Authority: United States: Food and Drug Administration
Austria: Agency for Health and Food Safety
Belgium: Federal Agency for Medicinal Products and Health Products
France: ANSM
Germany: BfarM
Italy: AIFA
Netherlands: Dutch Healthcare Authority (NZA)
Spain: Spanish Agency of Medicines (AEMPS)
United Kingdom: Medicines and Healthcare Products Regulatory Agency