NCT01524783

Everolimus Plus Best Supportive Care vs Placebo Plus Best Supportive Care in the Treatment of Patients With Advanced Neuroendocrine Tumors (GI or Lung Origin)

A Randomized, Double-blind, Multicenter, Phase III Study of Everolimus (RAD001) Plus Best Supportive Care Versus Placebo Plus Best Supportive Care in the Treatment of Patients With Advanced NET of GI or Lung Origin

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 302 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to compare the antitumor activity of everolimus plus best supportive care versus placebo plus best supportive care in patients with progressive nonfunctional neuroendocrine tumor (NET) of gastrointestinal (GI) or lung origin without a history of, or current symptoms of carcinoid syndrome.

Detailed description

This was a prospective, multi-center, randomized, double-blind, parallel-group, placebo-controlled, two-arm Phase III study comparing the efficacy and safety of everolimus 10 mg daily to placebo in patients with advanced NET of GI or lung origin without a history of, or current symptoms of carcinoid syndrome. After assessment of eligibility, participants qualifying for the study were randomized in a 2:1 ratio to receive either everolimus or matching placebo. Participants received daily oral doses of 10 mg everolimus or matching placebo as study drug. In both arms, the study drug was combined with best supportive care and treatment cycles were defined as 28 days. Participants were treated until disease progression as per Response Evaluation Criteria In Solid Tumors (RECIST) 1.0, intolerable toxicity, death, lost to follow-up or consent withdrawal. Regardless of the reason for study drug discontinuation, participants had a safety follow-up visit scheduled 30 days after the last dose of the study drug. Per data monitoring committee recommendation, all participants on treatment with placebo were allowed to crossover to open-label treatment with everolimus. This change was implemented through protocol amendment 3 (issued on 06-May-2016) after which remaining participants entered into open-label phase of the study.

Conditions

• Advanced NET of GI Origin
• Advanced NET of Lung Origin
• Neuroendocrine Tumors

Interventions

Type	Name	Description
DRUG	Everolimus	Participants were treated with everolimus 10 mg (two 5 mg tablets) once daily orally taken
DRUG	Placebo	Participants were treated with two tablets of matching placebo once daily orally taken.
OTHER	Best suportive care (BSC)	Best supportive care includes all care provided to participants deemed necessary by the treating physician, such as but not restricted to anti-diarrheals and analgesics. The optimal care of the patient is based on the treating physician's best medical judgment.

Timeline

• Start date: 2012-03-30
• Primary completion: 2014-11-28
• Completion: 2020-08-07
• First posted: 2012-02-02
• Last updated: 2021-08-05
• Results posted: 2016-12-28

Locations

97 sites across 25 countries: United States, Austria, Belgium, Canada, China, Colombia, Czechia, Germany, Greece, Hungary, Italy, Japan, Lebanon, Netherlands, Poland, Russia, Saudi Arabia, Slovakia, South Africa, South Korea, Spain, Taiwan, Thailand, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study