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

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.

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

Study Overview

• Status: Completed
• Conditions: Pancreatic Neuroendocrine Tumors
• Intervention / Treatment: Drug: everolimus

• Study Type: Interventional
• Enrollment (Actual): 61
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100730
    • Novartis Investigative Site
  • Beijing, China, 100039
    • Novartis Investigative Site
  • Guangzhou, China, 510060
    • Novartis Investigative Site
  • Shanghai, China, 200032
    • Novartis Investigative Site
  • Shanghai
    • Shanghai, Shanghai, China, 200032
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: everolimus (single arm); 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; 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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): March 14, 2016
• Primary Completion (Actual): February 22, 2024
• Study Completion (Actual): February 22, 2024

Study Registration Dates

• First Submitted: June 16, 2016
• First Submitted That Met QC Criteria: July 22, 2016
• First Posted (Estimated): July 25, 2016

Study Record Updates

• Last Update Posted (Actual): March 15, 2024
• Last Update Submitted That Met QC Criteria: March 14, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Endocrine System Diseases; Digestive System Neoplasms; Endocrine Gland Neoplasms; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Pancreatic Diseases; Adenoma; Pancreatic Neoplasms; Neuroendocrine Tumors; Adenoma, Islet Cell; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Protein Kinase Inhibitors; MTOR Inhibitors; Everolimus
• Other Study ID Numbers: CRAD001PCN31

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No