A Phase 2, Open-Label, Multi-Center, Serial Ascending-Dose, Dose-Finding Study to Evaluate the Safety and Tolerability of LX1606 in Subjects With Symptomatic Carcinoid Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Core Phase
研究概览
简要总结
The purpose of the study is to evaluate the safety and tolerability of orally administered telotristat etiprate (LX1606) in participants with symptomatic carcinoid syndrome.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 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
- •Males and females, aged 18 and older
- •Biopsy-proven metastatic carcinoid tumor of the gastrointestinal (GI) tract with disease extent confirmed by computed tomography (CT), magnetic resonance imaging (MRI), or radionuclide imaging
- •Symptomatic carcinoid syndrome (≥4 bowel movements per day)
- •Ability to provide written informed consent
排除标准
- •≥ 12 high-volume, watery bowel movements per day
- •Sponsor-unacceptable clinical laboratory values for hematology and liver function tests at screening
- •Karnofsky status ≤70% - unable to care for self
- •Surgery within 60 days prior to screening
- •A history of short bowel syndrome
- •Life expectancy < 12 months
- •History of substance or alcohol abuse within 2 years prior to screening
- •Administration of any investigational drug within 30 days of screening or any therapeutic protein or antibody within 90 days of screening
研究组 & 干预措施
Telotristat etiprate - Core Phase
Following a 2-week Run-In Period, participants received telotristat etiprate capsules at a starting dose of 150 mg, orally three times daily (TID) for 14 days in the Core Phase. Dose escalations (250 mg, 350 mg, 500 mg) occurred serially every 14 days, up to a maximum dosage of telotristat etiprate 500 mg TID, as guided by specific clinical criteria for dose escalation. Upon completion of 12 weeks of treatment, participants were eligible to receive telotristat etiprate in the optional Open-label Extension Period.
干预措施: Telotristat etiprate (Drug)
Telotristat etiprate - Extension Period
Participants received telotristat etiprate at their highest tolerated dose (250 mg or 500 mg), orally, TID for 124 weeks in the Open-label Extension Period. If neither dose was tolerated participants were discontinued from the study and completed the 2-week Follow-up Visit.
干预措施: Telotristat etiprate (Drug)
结局指标
主要结局
Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Core Phase
时间窗: Baseline up to Week 12 in the Core Phase
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. Treatment-emergent AEs were defined as any AEs reported after the first dose of treatment on Day 1.
Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Extension Period
时间窗: Up to 124 Weeks in the Extension Period
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. Treatment-emergent AEs were defined as any AEs reported after the first dose of treatment on Day 1.
次要结局
- Change From Baseline in Number of Bowel Movements (BMs)(Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24)
- Number of Participants With an Improvement in Global Assessment of Symptoms Associated With Carcinoid Syndrome(Core Phase: Weeks 9-12; Extension Period: Week 24)
- Change From Baseline in Sensation/Severity of Nausea Using 100 mm Visual Analog Scale (VAS)(Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24)
- Change From Baseline in Percentage of Days With Sensation of Urgency to Defecate(Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24)
- Change From Baseline in Daily Number of Cutaneous Flushing Episodes(Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24)
- Change From Baseline in Urinary 5-Hydroxyindoleacetic Acid (HIAA) Levels(Core Phase: Baseline to Week 12; Extension Period: Baseline to Weeks 20-21)
- Change From Baseline in Stool Form/Consistency(Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24)
- Change From Baseline in Daily Severity of Abdominal Pain or Discomfort Using 100 mm Visual Analog Scale (VAS)(Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24)
- Number of Participants Achieving Clinically Meaningful Symptom Reduction in the Core Phase(Baseline to Week 12)