An Open-label, Multi-center, Expanded Access Study of Everolimus in Participants With Advanced Neuroendocrine Tumors (NETs) (Core Study) and an Extension Study to the Open-label, Multi-center, Expanded Access Study of Everolimus in Patients With Advanced NETs (E1)
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 246
- 试验地点
- 1
- 主要终点
- Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core)
研究概览
简要总结
This record combines the results of CRAD001K24133 and CRAD001K24133E1. The purpose of the CRAD001K24133 study was to evaluate the safety profile of everolimus in patients with advanced neuroendocrine tumors of pancreatic origin (pNETs) and to provide access of everolimus to this patient population. Everolimus was taken by participants until disease progression, unacceptable toxicity, death, discontinuation from the trial for any other reason, or when it became commercially available for this indication, or until May 30, 2012, whichever came first. Prior to amendment 1, the study enrolled participants with NET of the lung (L-NETs) and gastrointestinal (GI) (GI-NETs) origin. The core study was stopped (per protocol) because everolimus was approved for pNETs. All ongoing patients with pNETs were switched to commercially available everolimus. For GI and lung NETs, everolimus was not approved at the time the core study was stopped. Therefore, patients with GI or lung NETs were not able to switch to commercial drug. To provide study medication access to these patients beyond 30 May 2012, the open label extension study, CRAD001K24133E1, was conducted. In the extension study, RAD001K24133E1, participants with GI or lung NETs who had not progressed during therapy with everolimus in the core study and who had not suffered from intolerable toxicity, were enrolled and treated with everolimus in order to provide data on long-term safety and efficacy. Patients were treated until it became commercially available in the respective indication or until documented tumor progression, unacceptable toxicity, any other reason or until study end on 31 May 2017, whichever came first.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •The patient must provide a signed Informed Consent Form (ICF) for the extension study prior to any study related procedures
- •Age ≥18 years old
- •Completion of the whole treatment period in the CRAD001K24133 study
- •Neuroendocrine tumor of gastrointestinal or pulmonary origin (pancreatic neuroendocrine tumors are excluded)
- •No tumor progression during therapy with everolimus during CRAD001K24133 study (checked via radiologically assessment)
- •No intolerable toxicity during therapy everolimus, or during combination therapy of everolimus and somatostatin analogues
研究组 & 干预措施
Everolimus (RAD001)
Participants received Everolimus 10 mg orally once daily until documented tumor progression, unacceptable toxicity or any other reason.
干预措施: Everolimus (RAD001) (Drug)
结局指标
主要结局
Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths (Core)
时间窗: from the day of first treatment up to 19 months
The number of participants with AEs, SAEs and deaths were assessed.
Number and Percentage of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths During the Extension Phase (E1)
时间窗: from the first day of treatment in the extension up to 4 years
The number of participants with AEs, SAEs and deaths were assessed.
次要结局
- Investigator-assessed Progression Free Survival (PFS) (Core)(from the day of first treatment up to 19 months)
- Mean EORTC QLQ-G.I. NET21 Score (Core)(Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82)
- Number and Percentage of Participants With Ratings of 'no Problem, 'Some Problem' and 'Extreme Problem' in the EuroQol Five Dimensions Questionnaire (EQ-5D) (Core)(Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82)
- Mean European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 (EORTC QLQ-C30) Score (Core)(Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82)
- Investigator-assessed Best Overall Response (Core)(from the start of treatment, every 12 weeks for the first year and then every 6 months up to 19 months)
- Mean EQ-5D Visual Analogue Scale (VAS) Score (Core)(Baseline, weeks 4, 8, 20, 32, 44, and end of treatment (EOT) up to week 82)
- Investigator-assessed Progression Free Survival (PFS) (E1)(from first date of treatment in the extension up to 4 years)
- Change in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-C30 (EORTC QLQ-C30) Score at the End of Treatment From Baseline (Baseline = First Day of Treatment in the Extension) (E1)(baseline, every 12 weeks and up to 4 years)
- Change in EORTC QLQ-G.I. NET21 Score at the End of Treatment From Baseline (Baseline = First Day of Treatment in the Extension) (E1)(baseline, every 12 weeks and up to 4 years)
- Change in EuroQol Five Dimensions Questionnaire (EQ-5D) Score at the End of Treatment From Baseline (Baseline = First Day of Treatment in the Extension) (E1)(baseline, every 12 weeks and up to 4 years)
- Investigator-assessed Best Overall Response During the Extension Phase (E1)(from the start of treatment, every 12 weeks for the first year and then every 6 months up to 4 years)