Telotristat Etiprate - Expanded Treatment for Patients With Carcinoid Syndrome Symptoms (TELEPATH)

A Multicenter, Long-term Extension Study to Further Evaluate the Safety and Tolerability of Telotristat Etiprate (LX1606)

The primary objective of this study is to evaluate the long-term safety and tolerability of orally administered telotristat etiprate.

Study Overview

• Status: Completed
• Conditions: Carcinoid Syndrome
• Intervention / Treatment: Drug: Telotristat etiprate

• Study Type: Interventional
• Enrollment (Actual): 124
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • St. Leonards, New South Wales, Australia, 2065
      • Lexicon Investigational Site
  • Queensland
    • Herston, Queensland, Australia, 4029
      • Lexicon Investigational Site
  • Victoria
    • East Melbourne, Victoria, Australia, 3002
      • Lexicon Investigational Site
• Belgium
  • Edegem, Belgium, B-2650
    • Lexicon Investigational Site
  • Ghent, Belgium, 9000
    • Lexicon Investigational Site
  • Yvoir, Belgium, 5530
    • Lexicon Investigational Site
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N4N2
      • Lexicon Investgational Site
  • Nova Scotia
    • Halifax, Nova Scotia, Canada, B0J1N0
      • Lexicon Investigational Site
• France
  • Lille, France, 59037
    • Lexicon Investigational Site
  • Lyon, France, 69437
    • Lexicon Investigational Site
  • Villejuif, France, 94805
    • Lexicon Investigational Site
• Germany
  • Bad Berka, Germany, 99437
    • Lexicon Investigational Site
  • Berlin, Germany, 13353
    • Lexicon Investigational Site
  • Essen, Germany, 45147
    • Lexicon Investigational Site
  • Hamburg, Germany, 20246
    • Lexicon Investigational Site
  • Marburg, Germany, 35043
    • Lexicon Investigational Site
• Israel
  • Jerusalem, Israel, 91120
    • Lexicon Investigational Site
• Italy
  • Milano, Italy, 20089
    • Lexicon Investigational Site
  • Milano, Italy, 20141
    • Lexicon Investigational Site
  • Pisa, Italy, 56124
    • Lexicon Investigational Site
  • Torino, Italy, 10043
    • Lexicon Investigational Site
• Netherlands
  • Amsterdam, Netherlands, 1105AZ
    • Lexicon Investigational Site
  • Noord Holland, Netherlands, 1066CX
    • Lexicon Investigational Site
  • Noord-Brahant, Netherlands, 5631BM
    • Lexicon Investigational Site
• Spain
  • Barcelona, Spain, 08035
    • Lexicon Investigational Site
  • Madrid, Spain, 28034
    • Lexicon Investigational Site
  • Seville, Spain, 41013
    • Lexicon Investigational Site
• Sweden
  • Lund, Sweden, 22185
    • Lexicon Investigational Site
  • Uppsala, Sweden, 75185
    • Lexicon Investigational Site
• United Kingdom
  • Coventry, United Kingdom, CV22DX
    • Lexicon Investigational Site
  • Glasgow, United Kingdom, G120YN
    • Lexicon Investigational Site
  • London, United Kingdom, NW32QG
    • Lexicon Investigational Site
  • London, United Kingdom, SE59RS
    • Lexicon Investigational Site
  • London, United Kingdom, W120HS
    • Lexicon Investigational Site
  • Manchester, United Kingdom, M204BX
    • Lexicon Investigational Site
  • Newcastle-Upon-Tyne, United Kingdom, NE14LP
    • Lexicon Investigational Site
• United States
  • Alabama
    • Mobile, Alabama, United States, 36604
      • Lexicon Investigational Site
  • California
    • Stanford, California, United States, 94305
      • Lexicon Investigational Site
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • Lexicon Investigational Site
  • Kentucky
    • Lexington, Kentucky, United States, 40536
      • Lexicon Investigational Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Lexicon Investigational Site
  • New York
    • New York, New York, United States, 10029
      • Lexicon Investigational Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Lexicon Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Ongoing participation in a Phase 2 [LX1606.1-202-CS (NCT00853047), LX1606.1-203-CS (NCT01104415)] or Phase 3 [LX1606.1-301-CS (NCT01677910), LX1606.1-303-CS (NCT02063659)] study
• Patients of childbearing potential must agree to use an adequate method of contraception (defined as having a failure rate of <1% per year) during the study and for 12 weeks after the Follow-up visit.
• Ability and willingness to provide written informed consent

Exclusion Criteria:

• Major protocol violations or tolerability concerns in a Phase 2 (eg, LX1606.1-202-CS, LX1606.1-203-CS) or Phase 3 (eg, LX1606.1-301-CS, LX1606.1-303-CS) study
• Positive pregnancy test
• Presence of any clinically significant findings at entry for medical history, laboratory values, or physical examination

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 250 mg Telotristat Etiprate; One telotristat etiprate (250 mg) tablet administered three times daily.	Drug: Telotristat etiprate; Telotristat etiprate tablet (250 mg); Other Names: LX1606
Experimental: 500 mg Telotristat Etiprate; Two telotristat etiprate (250 mg) tablets administered three times daily.	Drug: Telotristat etiprate; Telotristat etiprate tablet (250 mg); Other Names: LX1606

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	An adverse event (AE) is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE includes any noxious, pathological, or unintended change in anatomical, physiological, or metabolic functions as indicated by physical signs or symptoms occurring in any phase of the clinical study whether or not considered related to the study medication. A TEAE is an AE that occurs or worsens after receiving study drug.	First dose of study drug (Day 1) up to 15 days post last dose (approximately up to 236 weeks)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ-C30) Score at Each Visit	QLQ-C30 is a standardized 30-item scale to assess health-related quality of life composed of 5 functional scales (physical functioning [5 items], role functioning [2 items], emotional functioning [4 items], cognitive functioning [2 items], and social functioning [2 items]); 3 symptom scales (fatigue [3 items], nausea/vomiting [2 items], and pain [2 items]); a global health status (GHS) /quality of life (QOL) scale [2 items]; 6 single items (dyspnoea, insomnia, appetite loss, constipation, diarrhoea, and financial difficulties). 28 questions answered:1 (not at all) to 4 (very much) and 2 questions on overall health/QOL answered:1 (poor) to 7 (excellent). All of the scales and single-item measures are transformed to a score:0 to 100. For functioning scales and global QOL higher scores indicate better functioning (a positive change from Baseline indicates improvement); for symptom scales higher scores indicate more severe symptoms (a negative change from Baseline indicates improvement).	Baseline, Weeks 24, 48, 72 and 84
Change From Baseline in Gastrointestinal Symptoms of Carcinoid Neuroendocrine Tumors (GI.NET21) Score at Each Visit	GI.NET21 is a standardized 21-item scale composed of both multi-item scales and single-item measures that include 5 functional scales (gastrointestinal (GI) [5 items], endocrine [3 items], treatment-related [3 items], social functioning [3 items], and disease-related worries scale [DRWS] [3 items]) and 4 single items (muscle and bone pain symptom (BPS), sexual functioning, communication function (CF), body image and information about the disease). Each item is scored from 1 (not at all) to 4 (very much). All of the scales and single-item measures are transformed to a score of 0 to 100. For functioning scales higher scores indicate better functioning (a positive change from Baseline indicates improvement); for symptom scales higher scores indicate more severe symptoms (a negative change from Baseline indicates improvement).	Baseline, Weeks 24, 48, 72 and 84
Percentage of Participants With Adequate Relief as Per Subjective Global Assessment Question	Participants were asked to respond 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 percentage of participants reporting adequate relief (answered Yes) were reported.	Baseline, Weeks 12, 24, 36, 48, 60, 72 and 84
Change From Baseline in Subjective Global Assessment of Carcinoid Syndrome Symptoms on 11-Point Numeric Scale at Each Visit	Participants were asked the following question to assess global symptoms associated with carcinoid syndrome (CS) on an 11-point scale: "Rate the severity of your overall carcinoid symptoms over the past 7 days on a scale from 0 to 10, where 0=no symptoms and 10=worst symptoms ever experienced. A negative change from baseline indicated improvement.	Baseline, Weeks 12, 24, 36, 48, 60, 72 and 84

Collaborators and Investigators

• Sponsor: Lexicon Pharmaceuticals
• Investigators: Study Director: Pablo LaPuerta, MD, Lexicon Pharmaceuticals, Inc.

Publications and helpful links

General Publications

• Horsch D, Anthony L, Gross DJ, Valle JW, Welin S, Benavent M, Caplin M, Pavel M, Bergsland E, Oberg K, Kassler-Taub KB, Binder P, Banks P, Lapuerta P, Kulke MH. Long-Term Treatment with Telotristat Ethyl in Patients with Carcinoid Syndrome Symptoms: Results from the TELEPATH Study. Neuroendocrinology. 2022;112(3):298-310. doi: 10.1159/000516958. Epub 2021 May 3.

Study record dates

Study Major Dates

• Study Start (Actual): January 14, 2014
• Primary Completion (Actual): September 12, 2018
• Study Completion (Actual): September 12, 2018

Study Registration Dates

• First Submitted: December 31, 2013
• First Submitted That Met QC Criteria: December 31, 2013
• First Posted (Estimate): January 1, 2014

Study Record Updates

• Last Update Posted (Actual): September 17, 2019
• Last Update Submitted That Met QC Criteria: August 22, 2019
• Last Verified: August 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Chemically-Induced Disorders; Pathologic Processes; Neoplasms by Histologic Type; Neoplasms; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Disease; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroendocrine Tumors; Drug-Related Side Effects and Adverse Reactions; Syndrome; Carcinoid Tumor; Malignant Carcinoid Syndrome; Serotonin Syndrome
• Other Study ID Numbers: LX1606.1-302-CS; LX1606.302 (Other Identifier: Lexicon Pharmaceuticals, Inc.); 2013-002596-18 (EudraCT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated device product: No