Phase IV Study of the Safety and Efficacy of Everolimus in Adult Patients With Progressive pNET in China

Study Overview
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Brief Summary
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To evaluate safety and efficacy of everolimus (Afinitor®) in Chinese adult patients with local advanced or metastatic, well differentiated progressive pancreatic neuroendocrine tumors.

Official Title
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Phase IV, Open-label, Multi-center, Single-arm Study of the Safety and Efficacy of Everolimus (Afinitor) in Adult Patients With Local Advanced or Metastatic, Well Differentiated Progressive Pancreatic Neuroendocrine Tumors (pNET) in China.

Conditions
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Pancreatic Neuroendocrine Tumors

Intervention / Treatment
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* Drug: everolimus


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients must have histological confirmed G1 or G2 pancreatic neuroendocrine tumors(pNETs) (WHO 2010) Patients must have radiological documentation of progression of disease per RECIST 1.1 within 12 months prior to enrollment. Measurable disease per RECIST 1.1 criteria using triphasic computed tomography (CT) scan or multiphase MRI for radiologic assessment. everolimus treatment which is recommended by the treating physician Exclusion Criteria: Hypersensitivity to everolimus, to other rapamycin derivatives, or to any of the excipients. Patient who is unwilling to receive Afinitor treatment due to any reason. Pregnant or nursing (lactating) women, Prior therapy with mTOR inhibitors (e.g. sirolimus, temsirolimus, everolimus). Use of an investigational drug within the 30 days prior to enrollment

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: everolimus (single arm)<br>Everolimus is taken at a starting dose of 10 mg orally once daily.Patients will be provided with adequate supply of study treatment for self-administration at home until at least their next scheduled study visit.All patients will be followed for adverse events and serious adverse events for 30 days following the last dose of study drug. Beyond these 30 days, any serious adverse events that are suspected to be related to the study drug will also be collected | Drug: everolimus<br>* Everolimus is taken at a starting dose of 10 mg orally once daily.Patients will be provided with adequate supply of study treatment for self-administration at home until at least their next scheduled study visit.All patients will be followed for adverse events and serious adverse events for 30 days following the last dose of study drug. Beyond these 30 days, any serious adverse events that are suspected to be related to the study drug will also be collected<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants with Adverse Events as a Measure of Safety and Tolerability | Incidences of AEs, AE suspected to be related to Afinitor, Grade 3/4 AEs and SAEs | 5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Survival | Overall Survival is defined as time from study treatment to death due to any cause | Baseline, Week 4, every 3 months during treatment, every 6 months during survival through study completion which is defined as 75% completed survival (death) or ended treatment 5 years earlier (approximately 5 years) | 
| Progression free survival | Progression free survival is defined as time from first dose of study treatment to progression or death due to any cause | Baseline, WK 4, every 3 MO during treatment until disease progression, unacceptable toxicity or discontinue treatment, every 6 MO during survival to study completion ( defined as 75% completed survival (death) or ended treatment 5 YRs earlier |