Clinical Trial: Efficacy and Safety of BEZ235 Compared to Everolimus in Patients With Advanced Pancreatic Neuroendocrine Tumors

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Randomized Phase II Study of BEZ235 or Everolimus in Advanced Pancreatic Neuroendocrine Tumors

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).

Detailed Summary: 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.
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Progression Free Survival (PFS) [ Time Frame: up to approx. 18 months ]

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.


Original Primary Outcome: Progression Free Survival (PFS) [ Time Frame: up to approx. 18 months ]

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 8 weeks after randomisation


Current Secondary Outcome:

  • Objective Response Rate [ Time Frame: up to approx. 18 months ]
    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.
  • Overall Survival (OS) [ Time Frame: up to approx. 30 months ]
    Time from randomization to the date of death due to any cause
  • Time to Treatment Failure (TTF) [ Time Frame: up to approx. 18 months ]
    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


Original Secondary Outcome:

  • Type, frequency and severity of adverse events [ Time Frame: at minimum at each study visit and up to approx. 18 months ]
    Safety will be determined by type, frequency and severity of adverse events per CTCAEv4.03 and type, frequency and severity of laboratory toxicities per CTCAEv4.03. Patients will be followed up for the duration of the study
  • Objective Response Rate [ Time Frame: up to approx. 18 months ]
    Proportion of patients with a best overall response during the study of complete response (CR) or partial response (PR), based on the investigator assessment
  • Overall Survival (OS) [ Time Frame: up to approx. 30 months ]
    Time from randomization to the date of death due to any cause
  • Time to Treatment Failure (TTF) [ Time Frame: up to approx. 18 months ]
    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


Information By: Novartis

Dates:
Date Received: June 25, 2012
Date Started: October 2012
Date Completion:
Last Updated: March 9, 2016
Last Verified: March 2016