This is a Phase II study in 2 stages, evaluating BEZ235 plus best supportive care (BSC) versus placebo plus BSC in patients with advanced pancreatic neuroendocrine tumors (pNET) after failure of mTOR inhibitor therapy. Study design: This was a Phase II, two-stage, multicenter study, where Stage 1 was a single arm, 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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
31
The investigational study drug used in this trial was BEZ235, which was supplied as 50mg, 200mg, 300mg, and 400mg solid dispersion sachets. Supply as 200mg and 50mg were provided for dose reduction. Patients were instructed to take the contents of one sachet of BEZ235 twice a day in the morning within 30 minutes after a light meal (breakfast).
Indiana University SC
Indianapolis, Indiana, United States
Dana Farber Cancer Institute GastrointestionalCancer Clinic
Boston, Massachusetts, United States
Montefiore Medical Center SC-2
The Bronx, New York, United States
Ohio State Comprehensive Cancer Center/James Cancer Hospital SC
Columbus, Ohio, United States
Novartis Investigative Site
Vienna, Austria
Novartis Investigative Site
Leuven, Belgium
Novartis Investigative Site
Lyon, France
Novartis Investigative Site
Villejuif, France
Novartis Investigative Site
Essen, Germany
Novartis Investigative Site
Florence, FI, Italy
...and 7 more locations
Stage 1 - Progression Free Survival (PFS) Rate Analysis at 16 Weeks as Per Local Radiology Review
PFS rate at 16 weeks was defined as a binary variable. Patients were considered as 'progression free' after 16 weeks if they had an overall lesion response of complete response (CR) partial response ('PR) or stable disease (SD)' and "progressed" if they had an overall lesion response of 'Progressive disease (PD) at the scan which occurred on day 105 after start of treatment, or later. Patients whose 16 weeks tumor assessment was unknown, missing or outside the window was not considered as 'progression free' and was considered a "failure" and counted only in the denominator for the estimation of the 16 week progression free rate.
Time frame: 16 weeks after the first BEZ235 administration.
Stage 1- Overall Response Rate (ORR)
Overall Response rate was defined as the proportion of patients with a best overall response of complete response or partial response, based on investigator's assessment as per RECIST criteria version 1.1. Based on futility analysis conducted at the end of stage 1, stage 2 was not initiated.
Time frame: Baseline, every 8 weeks up to 31 months
Stage 1 - Disease Control Rate
Disease control rate was defined as the proportion of patients with a best overall response of Complete Response, Partial response, or Stable disease, based on the investigator's assessment per RECIST version 1.1. Based on futility analysis conducted at the end of stage 1, stage 2 was not initiated.
Time frame: Baseline, every 8 weeks up to 31 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.