Conditions: Advanced NET of GI Origin Advanced NET of Lung Origin Neuroendocrine Tumors
Keywords: Neuroendocrine tumor NET progressive advanced gastrointestinal GI or lung origin nonfunctional everolimus Advanced NET of GI origin Advanced NET of lung origin
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 302
Healthy volunteers: no

Brief 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.

Arms:
arm 1: Participants received everolimus 10 mg once daily plus best supportive care (BSC) throughout the study arm 2: Participants received matching placebo once daily plus best supportive care (BSC) during the blinded period. Participants were allowed to crossover to treatment with everolimus 10mg once daily plus BSC during the open-label period.

Interventions: intervention 1: Everolimus intervention 2: Placebo intervention 3: Best suportive care (BSC)
Intervention descriptions: intervention 1: Participants were treated with everolimus 10 mg (two 5 mg tablets) once daily orally taken intervention 2: Participants were treated with two tablets of matching placebo once daily orally taken. intervention 3: 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.

Protocol document: