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

Phase 2TerminatedNCT01628913

Novartis Pharmaceuticals62 enrolled

Overview

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).

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Pancreatic Neuroendocrine Tumors (pNET)

Interventions

BEZ235DRUG

BEZ235 400 mg bid p.o. (by mouth, twice daily)

EverolimusDRUG

Everolimus 10 mg qd p.o. (by mouth, daily)

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (26)

Cedars Sinai Medical Center SC-3

Los Angeles, California, United States

Outcomes

Primary Outcomes

Progression Free Survival (PFS)

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.

Time frame: up to approx. 18 months

Secondary Outcomes

Objective Response Rate

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.

Time frame: up to approx. 18 months

Overall Survival (OS)

Time from randomization to the date of death due to any cause

Time frame: up to approx. 30 months

Time to Treatment Failure (TTF)

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

Time frame: up to approx. 18 months

Data from ClinicalTrials.gov

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