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

• ClinicalTrials.gov Identifier: NCT02026063
• Recruitment Status: Completed
• First Posted: January 1, 2014
• Results First Posted: September 17, 2019
• Last Update Posted: September 17, 2019
• Sponsor: Lexicon Pharmaceuticals
• Information provided by (Responsible Party): Lexicon Pharmaceuticals

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Carcinoid Syndrome	Drug: Telotristat etiprate	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 124 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Multicenter, Long-term Extension Study to Further Evaluate the Safety and Tolerability of Telotristat Etiprate (LX1606)
• Actual Study Start Date: January 14, 2014
• Actual Primary Completion Date: September 12, 2018
• Actual Study Completion Date: September 12, 2018

Arms and Interventions

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 Name: 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 Name: LX1606

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: First dose of study drug (Day 1) up to 15 days post last dose (approximately up to 236 weeks) ]
   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.

Secondary Outcome Measures :

1. Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ-C30) Score at Each Visit [ Time Frame: Baseline, Weeks 24, 48, 72 and 84 ]
   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).
2. Change From Baseline in Gastrointestinal Symptoms of Carcinoid Neuroendocrine Tumors (GI.NET21) Score at Each Visit [ Time Frame: Baseline, Weeks 24, 48, 72 and 84 ]
   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).
3. Percentage of Participants With Adequate Relief as Per Subjective Global Assessment Question [ Time Frame: Baseline, Weeks 12, 24, 36, 48, 60, 72 and 84 ]
   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.
4. Change From Baseline in Subjective Global Assessment of Carcinoid Syndrome Symptoms on 11-Point Numeric Scale at Each Visit [ Time Frame: Baseline, Weeks 12, 24, 36, 48, 60, 72 and 84 ]
   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.

Eligibility Criteria

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

Criteria

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

Contacts and Locations

Locations

United States, Alabama

Lexicon Investigational Site

Mobile, Alabama, United States, 36604

United States, California

Lexicon Investigational Site

Stanford, California, United States, 94305

United States, Iowa

Lexicon Investigational Site

Iowa City, Iowa, United States, 52242

United States, Kentucky

Lexicon Investigational Site

Lexington, Kentucky, United States, 40536

United States, Massachusetts

Lexicon Investigational Site

Boston, Massachusetts, United States, 02215

United States, New York

Lexicon Investigational Site

New York, New York, United States, 10029

United States, Pennsylvania

Lexicon Investigational Site

Philadelphia, Pennsylvania, United States, 19104

Australia, New South Wales

Lexicon Investigational Site

St. Leonards, New South Wales, Australia, 2065

Australia, Queensland

Lexicon Investigational Site

Herston, Queensland, Australia, 4029

Australia, Victoria

Lexicon Investigational Site

East Melbourne, Victoria, Australia, 3002

Belgium

Lexicon Investigational Site

Edegem, Belgium, B-2650

Lexicon Investigational Site

Ghent, Belgium, 9000

Lexicon Investigational Site

Yvoir, Belgium, 5530

Canada, Alberta

Lexicon Investgational Site

Calgary, Alberta, Canada, T2N4N2

Canada, Nova Scotia

Lexicon Investigational Site

Halifax, Nova Scotia, Canada, B0J1N0

France

Lexicon Investigational Site

Lille, France, 59037

Lexicon Investigational Site

Lyon, France, 69437

Lexicon Investigational Site

Villejuif, France, 94805

Germany

Lexicon Investigational Site

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

Israel

Lexicon Investigational Site

Jerusalem, Israel, 91120

Italy

Lexicon Investigational Site

Milano, Italy, 20089

Lexicon Investigational Site

Milano, Italy, 20141

Lexicon Investigational Site

Pisa, Italy, 56124

Lexicon Investigational Site

Torino, Italy, 10043

Netherlands

Lexicon Investigational Site

Amsterdam, Netherlands, 1105AZ

Lexicon Investigational Site

Noord Holland, Netherlands, 1066CX

Lexicon Investigational Site

Noord-Brahant, Netherlands, 5631BM

Spain

Lexicon Investigational Site

Barcelona, Spain, 08035

Lexicon Investigational Site

Madrid, Spain, 28034

Lexicon Investigational Site

Seville, Spain, 41013

Sweden

Lexicon Investigational Site

Lund, Sweden, 22185

Lexicon Investigational Site

Uppsala, Sweden, 75185

United Kingdom

Lexicon Investigational Site

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

Sponsors and Collaborators

Lexicon Pharmaceuticals

Investigators

• Study Director: Pablo Lapuerta, MD; Lexicon Pharmaceuticals, Inc.

  Study Documents (Full-Text)

Documents provided by Lexicon Pharmaceuticals:

Study Protocol  [PDF] November 2, 2015

Statistical Analysis Plan  [PDF] November 24, 2014

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

• Responsible Party: Lexicon Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT02026063
• Other Study ID Numbers: LX1606.1-302-CS; LX1606.302 ( Other Identifier: Lexicon Pharmaceuticals, Inc. ); 2013-002596-18 ( EudraCT Number )
• First Posted: January 1, 2014
• Results First Posted: September 17, 2019
• Last Update Posted: September 17, 2019
• Last Verified: August 2019

• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Carcinoid Tumor; Malignant Carcinoid Syndrome; Syndrome; Serotonin Syndrome; Disease; Pathologic Processes; Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Drug-Related Side Effects and Adverse Reactions; Chemically-Induced Disorders