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Study of Telotristat Etiprate (LX1606) in Participants With Symptomatic Carcinoid Syndrome

2019年2月26日 更新者：Lexicon Pharmaceuticals

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

The purpose of the study is to evaluate the safety and tolerability of orally administered telotristat etiprate (LX1606) in participants with symptomatic carcinoid syndrome.

研究概览

地位

完全的

条件

干预/治疗

研究类型

介入性

注册 (实际的)

15

阶段

  • 阶段2

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 德国
      • Bad Berka、德国
        • Lexicon Investigational Site
      • Berlin、德国
        • Lexicon Investigational Site
      • Halle、德国
        • Lexicon Investigational Site
      • Lubeck、德国
        • Lexicon Investigational Site
      • Marburg、德国
        • Lexicon Investigational Site
      • Munich、德国
        • Lexicon Investigational Site
  • 英国
      • Basingstoke、英国
        • Lexicon Investigational Site
      • Cambridge、英国
        • Lexicon Investigational Site
      • London、英国
        • Lexicon Investigational Site
      • Manchester、英国
        • Lexicon Investigational Site

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

18年 及以上 (成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • ≥ 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

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：单组作业
  • 屏蔽：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
实验性的：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.
药品：Telotristat etiprate
Telotristat etiprate capsules orally three times daily.
其他名称：
  • LX1606 马尿
实验性的：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.
药品：Telotristat etiprate
Telotristat etiprate capsules orally three times daily.
其他名称：
  • LX1606 马尿

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Core Phase
大体时间：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.
Baseline up to Week 12 in the Core Phase
Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Extension Period
大体时间：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.
Up to 124 Weeks in the Extension Period

次要结果测量

结果测量
措施说明
大体时间
Change From Baseline in Number of Bowel Movements (BMs)
大体时间：Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Participants recorded the number of bowel movements in a daily diary. The change from baseline value was calculated as the difference between mean numbers of BMs of the post-baseline interval (Weeks 9 to 12) and baseline. A negative change from baseline indicates improvement. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Change From Baseline in Stool Form/Consistency
大体时间：Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Participants assessed stool form/consistency in a daily diary using a 6-point scale (0-none, 1-hard, 2-firm, 3-soft, 4-loose, 5-watery). The change from the baseline value was calculated as the difference between a mean score of the post-baseline interval (Weeks 9 to 12) and baseline. 0 indicates the best score and 5 indicates the worst score. A negative change from baseline indicates improvement. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
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
大体时间：Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Participants assessed the urgency to defecate using a daily diary response to the following question, "Have you felt or experienced a sense of urgency to pass stool today?". The change from the baseline value was calculated as the difference between the mean score (percentage of days) of the post-baseline interval (Weeks 9 to 12) and baseline. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Change From Baseline in Sensation/Severity of Nausea Using 100 mm Visual Analog Scale (VAS)
大体时间：Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Sensation/severity of nausea was measured using a 100 mm VAS. Participants rated their perception of the sensation/severity of nausea experienced by marking a single vertical line on a VAS scale from 0 to 100 mm, where 0 = No vomiting and 100 = vomiting. The change from the baseline value was calculated as the difference between the mean score of the post-baseline interval (Weeks 9 to 12) and baseline. 0 indicates the best score, 100 indicates the worst score. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
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
大体时间：Core Phase: Weeks 9-12; Extension Period: Week 24
Participants assessed their symptoms using a weekly subjective response to the following question, "In the past 7 days, have you had adequate relief of your carcinoid syndrome bowel complaints such as diarrhea, urgent need to have a bowel movement, abdominal pain, or discomfort?". The values for improvement in global assessment of symptoms associated with carcinoid syndrome in the Core Phase were averaged from Weeks 9 to 12.
Core Phase: Weeks 9-12; Extension Period: Week 24
Change From Baseline in Daily Severity of Abdominal Pain or Discomfort Using 100 mm Visual Analog Scale (VAS)
大体时间：Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
The severity of abdominal pain was measured using a 100 mm VAS. Participants rated their perception of the sensation/severity of abdominal pain or experienced by marking a single vertical line on a VAS scale from 0 to 100 mm, where 0 = No vomiting and 100 = vomiting. The change from the baseline value was calculated as the difference between the mean score of the post-baseline interval (Weeks 9 to 12) and baseline. 0 indicate the best score, 100 indicates the worst score. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Change From Baseline in Daily Number of Cutaneous Flushing Episodes
大体时间：Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Participants recorded the number of daily cutaneous flushing episodes experienced in the daily diary. The change from baseline value was calculated as the difference between the mean numbers of cutaneous flushing episodes of the post-baseline interval (Weeks 9 to 12) and baseline. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
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
大体时间：Baseline to Week 12
Clinically meaningful symptom reduction was defined as either: a) an average of < 4 bowel movements per day over 15 consecutive days, b) a 50% reduction from baseline in the number of bowel movements, c) a positive response to the question regarding adequate relief, or d) a 50% reduction from baseline in the number of daily flushing episodes.
Baseline to Week 12
Change From Baseline in Urinary 5-Hydroxyindoleacetic Acid (HIAA) Levels
大体时间：Core Phase: Baseline to Week 12; Extension Period: Baseline to Weeks 20-21
Urinary 5-HIAA (u5-HIAA) is a standard test used in clinical practice to assess the neuroendocrine tumor (NET) activity and is collected as a 24-hour urine specimen. The change from baseline value for the Extension Period was calculated as the difference between mean change in 5-HIAA of the post-baseline interval (Weeks 20 to 21) and baseline. A negative change from baseline indicates improvement. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
Core Phase: Baseline to Week 12; Extension Period: Baseline to Weeks 20-21

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Lexicon Pharmaceuticals

调查人员

  • 研究主任：Pablo LaPuerta, MD、Lexicon Pharmaceuticals, Inc.

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

一般刊物

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2010年6月15日

初级完成 (实际的)

2014年2月12日

研究完成 (实际的)

2014年2月12日

研究注册日期

首次提交

2010年4月12日

首先提交符合 QC 标准的

2010年4月13日

首次发布 (估计)

2010年4月15日

研究记录更新

最后更新发布 (实际的)

2019年3月15日

上次提交的符合 QC 标准的更新

2019年2月26日

最后验证

2019年2月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • LX1606.1-203-CS
  • LX1606.203, LX1032 (其他标识符：Lexicon Pharmaceuticals, Inc.)
  • 2009-016973-13 (EudraCT编号)

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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