Brief Summary
This was a multicenter, open label, randomized phase II study to evaluate the efficacy and safety of BEZ235 as compared to everolimus in patients with advanced, low to intermediate grade pancreatic neuroendocrine tumor (pNET).
Brief Title
Efficacy and Safety of BEZ235 Compared to Everolimus in Patients With Advanced Pancreatic Neuroendocrine Tumors
Detailed Description
Patients with advanced (unresectable or metastatic), low to intermediate grade (histologically confirmed well and moderately differentiated) pancreatic neuroendocrine tumor (pNET) were randomized to either BEZ235 or everolimus. The study was planned to include 140 patients, with 70 patients in the BEZ235 treatment group and 70 patients in the everolimus treatment group. An interim analysis was conducted on 62 randomized patients. The study was terminated as the BEZ235 treatment did not demonstrate a progression free survival advantage to everolimus treatment.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Pancreatic Neuroendocrine Tumors (pNET)
Eligibility Criteria
Inclusion Criteria:
* Advanced histologically confirmed well differentiated pancreatic neuroendocrine tumor
* Progressive disease within the last 12 months
* Measurable disease per RECIST Version 1.0 determined by multiphase MRI or triphasic CT
Exclusion Criteria:
* Prior treatment with mTOR or PI3K inhibitors
* Patients with more than 2 prior systemic treatment regimens
* Previous cytotoxic chemotherapy, targeted therapy, or biotherapy within the last 4 weeks
Other protocol-defined inclusion/exclusion criteria may apply
* Advanced histologically confirmed well differentiated pancreatic neuroendocrine tumor
* Progressive disease within the last 12 months
* Measurable disease per RECIST Version 1.0 determined by multiphase MRI or triphasic CT
Exclusion Criteria:
* Prior treatment with mTOR or PI3K inhibitors
* Patients with more than 2 prior systemic treatment regimens
* Previous cytotoxic chemotherapy, targeted therapy, or biotherapy within the last 4 weeks
Other protocol-defined inclusion/exclusion criteria may apply
Inclusion Criteria
Inclusion Criteria:
* Advanced histologically confirmed well differentiated pancreatic neuroendocrine tumor
* Progressive disease within the last 12 months
* Measurable disease per RECIST Version 1.0 determined by multiphase MRI or triphasic CT
inclusion/
* Advanced histologically confirmed well differentiated pancreatic neuroendocrine tumor
* Progressive disease within the last 12 months
* Measurable disease per RECIST Version 1.0 determined by multiphase MRI or triphasic CT
inclusion/
Gender
All
Gender Based
false
Keywords
Pancreatic
Neuroendocrine tumors
PNET
BEZ235
Everolimus
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Estimated
Last Update Submit Date
Minimum Age
18 Years
NCT Id
NCT01628913
Org Class
Industry
Org Full Name
Novartis
Org Study Id
CBEZ235Z2401
Overall Status
Terminated
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
Randomized Phase II Study of BEZ235 or Everolimus in Advanced Pancreatic Neuroendocrine Tumors
Primary Outcomes
Outcome Description
PFS is defined as the time from the date of randomization until the date of the first radiologically documented disease progression or death due to any cause. PFS is based on local investigator assessment. Patients will be followed up for the duration of the study and for an expected average of every 12 weeks after randomization. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of all target lesions, or unequivocal progression of non-target lesions, or the appearance of new lesions.
Outcome Measure
Progression Free Survival (PFS)
Outcome Time Frame
up to approx. 18 months
Secondary Ids
Secondary Id
CBEZ235Z2401
Secondary Id
2012-000769-19
Secondary Outcomes
Outcome Description
Proportion of patients with a best overall response during the study of complete response (CR) or partial response (PR), based on the investigator assessment. 2. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for all target and non-target lesions, as well as new lesions as assessed by CT or MRI: Complete Response (CR), Disappearance of all target and non-target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of all target lesions; Overall Response (OR) = CR + PR.
Outcome Time Frame
up to approx. 18 months
Outcome Measure
Objective Response Rate
Outcome Description
Time from randomization to the date of death due to any cause
Outcome Time Frame
up to approx. 30 months
Outcome Measure
Overall Survival (OS)
Outcome Description
Time from randomization to the date of the first of the following events:death due to any cause or progressive disease, treatment discontinuation due to toxicity or treatment discontinuation due to patient preference
Outcome Time Frame
up to approx. 18 months
Outcome Measure
Time to Treatment Failure (TTF)
Start Date
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Steven Libutti
Investigator Email
slibutti@montefiore.org
Investigator Phone