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Efficacy of Everolimus Alone or in Combination With Pasireotide LAR in Advanced PNET

Phase 2
Terminated
Conditions
Islet Cell Tumor
Interventions
Registration Number
NCT01374451
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

This study will estimate the treatment effect of everolimus in combination with pasireotide LAR relative to everolimus alone on progression-free survival (PFS) in patients with advanced progressive PNET.

A planned primary analysis was completed with data cut of 02-Apr-2014. The study did not meet its primary objective, which was based on progression-free survival (PFS) as per local radiology assessment and was prematurely terminated with the last patient last visit on 19-Feb-2015. However, it is important to note that the data did not reveal any new safety concerns. It was decided to stop the study and this decision was shared with the study sites on 31-Jul-2014.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
160
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
  • Patients currently requiring somatostatin analog treatment
  • Prior therapy with mTOR inhibitors or pasireotide
  • Patients with more than 2 prior systemic treatment regimens
  • Previous cytotoxic chemotherapy, targeted therapy, somatostatin analogs, or biotherapy within the last 4 weeks

Other protocol-defined inclusion/exclusion criteria may apply

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
EverolimusEverolimuseverolimus 10 mg once daily po alone
Paseriotide LAR + EverolimusEverolimuseverolimus 10 mg once daily po in combination with pasireotide LAR 60 mg every 28 days (q28d) im
Paseriotide LAR + EverolimusPasireotide LAReverolimus 10 mg once daily po in combination with pasireotide LAR 60 mg every 28 days (q28d) im
Primary Outcome Measures
NameTimeMethod
Progression-free Survival (PFS) Per Local Radiological ReviewOnce 80 PFS events had occurred aproximately after 24 months

PFS per RECIST 1.0. (Response Evaluation Criteria in Solid Tumors). PFS was defined as the time from the date of randomization to the date of the first radiologically documented disease progression or death due to any cause.

Secondary Outcome Measures
NameTimeMethod
Disease Control Rate (DCR) as Per Radiology ReviewOnce 80 PFS events had occurred

Disease control rate is the percentage of patients with a best overall response of CR or PR or stable disease (SD) determined by the local radiologist according to the Response Evaluation Criteria In Solid Tumors Criteria (RECIST) Version 1.0. CR: Disappearance of all nontarget lesions. PR: At least a 30% decrease in the sum of the longest diameter of all target lesions, taking as reference the smallest sum of the longest diameter of all target lesions recorded at or after baseline. SD: Neither sufficient shrinkage to qualify for PR or CR nor an increase in lesions which would qualify for progressive disease (PD). PD: Any progression ≤ 18 weeks after randomization (and not qualifying for CR, PR or stable disease SD.

Overall Survival (OS) Using Kaplan Meier MethodOnce 80 PFS events had occurred

Overall survival was defined as the time from date of randomization/start of treatment to date of death due to any cause. If a patient is not known to have died, survival was to be censored at the date of last contact.

PFS and the Predictive Probability of Success in Phase IIIOnce 105 PFS events had occurred occurred

105 PFS events expected after approximately 36 months

Summary of Pharmacokinetics (PK) for Everolimus for AUClastCycle 2 Day 1
Duration of Response (DoR)Once 80 PFS events had occurred

80 PFS are expected after approximately 24 months. Kaplan Meier was initially planned to be used to depict duration of response by treatment group and by stratum. Later based on the mode of action of everolimus and pasireotide and based on study experience, only a low number of objective responses per RECIST were expected. Therefore, protocol was amended to only list duration of response, and confirmed responses were flagged in the listing. Hence, statistical analyses were not planned and such data are not available for the following table.

Summary of Pharmacokinetics (PK) for Everolimus for TmaxCycle 2 Day 1
Summary of Pasireotide Concentrations Following Intramuscular Injection of Pasireotide LAR 60mgCycle 1 Day 21, Cycle 2 Day 29
Safety and Tolerability Profile of Everolimus Alone or in Combination With Pasireotide LAROnce 80 PFS events had occurred

Consisted of monitoring and recording the rate, type, severity, and causal relationship of adverse events (AEs) and serious AEs (SAEs) to treatment. The safety analysis was based mainly on the frequency of AEs or SAEs and on the number of laboratory values that fell outside of pre-determined range.

Objective Response Rate (ORR) as Per Radiology ReviewOnce 80 PFS events had occurred

Objective response was determined by the local radiologist according to the RECIST Version 1.0. ORR is the percentage of patients with a best overall response of complete response (CR) or partial response (PR). This is also referred to as Overall response rate.

CR: Disappearance of all nontarget lesions. PR: At least a 30% decrease in the sum of the longest diameter of all target lesions, taking as reference the smallest sum of the longest diameter of all target lesions recorded at or after baseline.

Summary of Pharmacokinetics (PK) for Everolimus for CL/FCycle 2 Day 1
Summary of Pharmacokinetics (PK) for Everolimus for Cmax and CminCycle 2 Day 1

Trial Locations

Locations (5)

Dana Farber Cancer Institute SC-2

🇺🇸

Boston, Massachusetts, United States

Montefiore Medical Center MMC

🇺🇸

Bronx, New York, United States

Oregon Health & Science University Knight Cancer Institute

🇺🇸

Portland, Oregon, United States

Novartis Investigative Site

🇬🇧

Manchester, United Kingdom

University of Texas/MD Anderson Cancer Center UT MD Anderson Cancer Ctr

🇺🇸

Houston, Texas, United States

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