Study of Telotristat Etiprate (LX1606) in Participants With Symptomatic Carcinoid Syndrome
2019년 2월 26일 업데이트: 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.
연구 개요
연구 유형
중재적
등록 (실제)
15
단계
- 2 단계
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
연구 장소
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Bad Berka, 독일
- Lexicon Investigational Site
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Berlin, 독일
- Lexicon Investigational Site
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Halle, 독일
- Lexicon Investigational Site
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Lubeck, 독일
- Lexicon Investigational Site
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Marburg, 독일
- Lexicon Investigational Site
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Munich, 독일
- Lexicon Investigational Site
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Basingstoke, 영국
- Lexicon Investigational Site
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Cambridge, 영국
- Lexicon Investigational Site
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London, 영국
- Lexicon Investigational Site
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Manchester, 영국
- Lexicon Investigational Site
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참여기준
연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.
자격 기준
공부할 수 있는 나이
18년 이상 (성인, 고령자)
건강한 자원 봉사자를 받아들입니다
아니
연구 대상 성별
모두
설명
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
공부 계획
이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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실험적: 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.
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Telotristat etiprate capsules orally three times daily.
다른 이름들:
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실험적: 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.
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Telotristat etiprate capsules orally three times daily.
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Core Phase
기간: Baseline up to Week 12 in the Core Phase
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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.
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Baseline up to Week 12 in the Core Phase
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Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Extension Period
기간: Up to 124 Weeks in the Extension Period
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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.
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Up to 124 Weeks in the Extension Period
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Change From Baseline in Number of Bowel Movements (BMs)
기간: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Change From Baseline in Stool Form/Consistency
기간: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Change From Baseline in Percentage of Days With Sensation of Urgency to Defecate
기간: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Change From Baseline in Sensation/Severity of Nausea Using 100 mm Visual Analog Scale (VAS)
기간: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Number of Participants With an Improvement in Global Assessment of Symptoms Associated With Carcinoid Syndrome
기간: Core Phase: Weeks 9-12; Extension Period: Week 24
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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.
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Core Phase: Weeks 9-12; Extension Period: Week 24
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Change From Baseline in Daily Severity of Abdominal Pain or Discomfort Using 100 mm Visual Analog Scale (VAS)
기간: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Change From Baseline in Daily Number of Cutaneous Flushing Episodes
기간: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Number of Participants Achieving Clinically Meaningful Symptom Reduction in the Core Phase
기간: Baseline to Week 12
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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.
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Baseline to Week 12
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Change From Baseline in Urinary 5-Hydroxyindoleacetic Acid (HIAA) Levels
기간: Core Phase: Baseline to Week 12; Extension Period: Baseline to Weeks 20-21
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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.
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Core Phase: Baseline to Week 12; Extension Period: Baseline to Weeks 20-21
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공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
수사관
- 연구 책임자: Pablo LaPuerta, MD, Lexicon Pharmaceuticals, Inc.
간행물 및 유용한 링크
연구에 대한 정보 입력을 담당하는 사람이 자발적으로 이러한 간행물을 제공합니다. 이것은 연구와 관련된 모든 것에 관한 것일 수 있습니다.
연구 기록 날짜
이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.
연구 주요 날짜
연구 시작 (실제)
2010년 6월 15일
기본 완료 (실제)
2014년 2월 12일
연구 완료 (실제)
2014년 2월 12일
연구 등록 날짜
최초 제출
2010년 4월 12일
QC 기준을 충족하는 최초 제출
2010년 4월 13일
처음 게시됨 (추정)
2010년 4월 15일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
2019년 3월 15일
QC 기준을 충족하는 마지막 업데이트 제출
2019년 2월 26일
마지막으로 확인됨
2019년 2월 1일
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- LX1606.1-203-CS
- LX1606.203, LX1032 (기타 식별자: Lexicon Pharmaceuticals, Inc.)
- 2009-016973-13 (EudraCT 번호)
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
예
미국 FDA 규제 기기 제품 연구
아니
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .