An Open Label, Multicenter, Single Arm Study of Pasireotide LAR in Patients With Rare Tumors of Neuroendocrine Origin
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 118
- 试验地点
- 7
- 主要终点
- Percentage of Responders at Month 6 - Pooled Pancreatic NETs (PNETs)
研究概览
简要总结
This study will assess the effectiveness and safety of pasireotide long-acting release in patients who have rare tumors of neuroendocrine origin.
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
- •Male and Female Patients at least 18 years old
- •Patient who have rare tumors of neuroendocrine origin, such as tumors of the:
- •pituitary glands
- •Nelson syndrome
- •ectopic-ACTH secreting tumor
- •Patients who have failed standard of care treatment or for whom no standard of care treatment exist
- •Signed Informed Consent
排除标准
- •Patients with active gallbladder disease
- •Patients with any ongoing or planned anti-neoplastic or interferon therapy
- •Poorly controlled diabetes mellitus
- •Female patients who are pregnant or lactating, or are of childbearing potential and not practicing a medically acceptable method of birth control
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
pasireotide LAR 60mg
Patients received pasireotide LAR at 60 mg approximately once every 28 days for 6 months during the core treatment period and additional treatment cycles up to a total of 48 months during the extension phase.
干预措施: pasireotide LAR (Drug)
结局指标
主要结局
Percentage of Responders at Month 6 - Pooled Pancreatic NETs (PNETs)
时间窗: 6 months
The primary efficacy endpoint was defined as the percentage of responders at Month 6 among pooled PNET patients (insulinoma, gastrinoma, VIPoma, and glucagonoma). A responder was defined as a patient who either attained normalization or had a greater than 50% reduction from baseline of the level of the primary biochemical tumor marker at Month 6 (M6). Four insulinoma pts were excluded from analysis because of unavailability of normal ranges for the associated primary biochemical tumor marker (insulin-to-glucose ratio). One patient with VIPoma with a normal baseline was also excluded. As a result, only 20 out of 25 patients with PNET were included in the assessment of the primary endpoint, which was less than the planned sample size of 34. Therefore, the primary objective could not be assessed with sufficient power. Patients with missing Month 6 assessment were considered as non-responders. Responder analyses are reported only for indications with minimum of 6 patients.
次要结局
- PiNETs: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker(Baseline, month 6)
- Percentage of Responders With Probability of Success at Month 6 - Individual NETs(6 months)
- PNETs: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker(Baseline, month 6)
- Nelson's Syndrome: Number of Patients Attaining Normalization or a More Than 50% Reduction in Primary Biochemical Tumor Marker(Baseline, month 6)
- Percentage of Responders at Month 6 - Individual NETs(6 months)