A Phase 3, Randomized, Placebo-controlled, Multicenter, Double-blind Study to Evaluate the Safety and Efficacy of Telotristat Etiprate (LX1606) in Patients With Carcinoid Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 76
- 试验地点
- 2
- 主要终点
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs) in the Open-Label Extension Period
研究概览
简要总结
The purpose of the study is to evaluate the effect of telotristat etiprate versus placebo on the incidence of treatment-emergent adverse events and on 5-hydroxyindoleacetic acid (5-HIAA) levels.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
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
- •Patients ≥ 18 years of age
- •All patients of reproductive potential must agree to use an adequate method of contraception during the study and for 12 weeks after the Follow-up visit.
- •Histopathologically-confirmed, well-differentiated metastatic neuroendocrine tumor
- •Documented history of carcinoid syndrome
- •Patient is able and willing to provide written informed consent prior to participation
排除标准
- •Presence of diarrhea attributed to any condition other than carcinoid syndrome.
- •Presence of 12 or more watery bowel movements per day
- •Positive stool examination for enteric pathogens, pathogenic ova or parasites, of Clostridium difficile at Screening
- •Karnofsky Performance Status ≤ 60%
- •Presence of any clinically significant laboratory, medical history, or physical examination findings deemed unacceptable by the Investigator
- •A history of short bowel syndrome
- •History of constipation within 2 years of Screening
- •Life expectancy < 12 months from Screening
研究组 & 干预措施
250 mg Telotristat Etiprate
Following a 3 to 4-week run-in period, participants were randomized to receive one 250 mg telotristat etiprate tablet and one placebo-matching telotristat etiprate tablet administered three times daily for 12 weeks, followed by a 36 week open-label extension period.
干预措施: Telotristat etiprate (Drug)
250 mg Telotristat Etiprate
Following a 3 to 4-week run-in period, participants were randomized to receive one 250 mg telotristat etiprate tablet and one placebo-matching telotristat etiprate tablet administered three times daily for 12 weeks, followed by a 36 week open-label extension period.
干预措施: Placebo (Drug)
500 mg Telotristat Etiprate
Following a 3 to 4-week run-in period, participants were randomized to receive one 250 mg telotristat etiprate tablet and one placebo-matching telotristat etiprate tablet administered three times daily for one week, followed by two 250 mg telotristat etiprate tablets administered three times daily for 11 weeks in the 12 week double-blind treatment period, followed by a 36 week open-label extension period.
干预措施: Telotristat etiprate (Drug)
500 mg Telotristat Etiprate
Following a 3 to 4-week run-in period, participants were randomized to receive one 250 mg telotristat etiprate tablet and one placebo-matching telotristat etiprate tablet administered three times daily for one week, followed by two 250 mg telotristat etiprate tablets administered three times daily for 11 weeks in the 12 week double-blind treatment period, followed by a 36 week open-label extension period.
干预措施: Placebo (Drug)
Placebo
Following a 3 to 4-week run-in period, participants were randomized to receive two placebo-matching telotristat etiprate tablets administered three times daily for 12 weeks, followed by a 36 week open-label extension period.
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) in the Open-Label Extension Period
时间窗: First dose of study drug to within 30 days of last dose of study drug in the Open-Label Extension Period (Up to 52.6 Weeks)
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. A TEAE was an AE reported after the first dose of randomized treatment on Day 1.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) in the Double-Blind Treatment Period
时间窗: First dose of study drug to within 30 days of last dose of study drug in the Double-Blind Treatment Period (Up to 17.1 Weeks)
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. A TEAE was an AE reported after the first dose of randomized treatment on Day 1.
Primary: Percent Change From Baseline in Urinary 5-hydroxyindoleacetic Acid (u5-HIAA) Levels
时间窗: Baseline and 12 Weeks
u5-HIAA is a standard test used in clinical practice to assess neuroendocrine tumor (NET) activity and is collected as a 24-hour urine specimen. A negative change from Baseline indicates improvement.
次要结局
- Change From Baseline in Abdominal Pain Averaged Across All Time-Points(Baseline and 12 Weeks)
- Change in the Frequency of Rescue Short-acting, Somatostatin Analog (SSA) Used to Treat Carcinoid Syndrome Symptoms Averaged Across All Time-Points(Baseline and 12 weeks)
- Change From Baseline in Stool Form/Consistency Averaged Across All Time-Points(Baseline and 12 Weeks)
- Change From Baseline in the Number of Bowel Movements (BMs) Per Day Averaged Over 12 Weeks(Baseline and 12 weeks)
- Change From Baseline in the Number of Daily Cutaneous Flushing Episodes Averaged Across All Time-Points(Baseline and 12 Weeks)
- Change From Baseline in the Number of Daily BMs Averaged Over the 12-Week Double-Blind Period, Among Participants Who Were Not Receiving SSA Therapy at Baseline(Baseline and 12 Weeks)