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

Tracking Information

• First Submitted Date: December 31, 2013
• First Posted Date: January 1, 2014
• Results First Submitted Date: August 22, 2019
• Results First Posted Date: September 17, 2019
• Last Update Posted Date: September 17, 2019
• Actual Study Start Date: January 14, 2014
• Actual Primary Completion Date: September 12, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 22, 2019)

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.

• Original Primary Outcome Measures (submitted: December 31, 2013): Incidence of Treatment-Emergent Adverse Events [ Time Frame: 50 weeks ]

Current Secondary Outcome Measures (submitted: August 22, 2019)

• 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).
• 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).
• 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.
• 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.

Original Secondary Outcome Measures (submitted: December 31, 2013)

Quality of Life measures [ Time Frame: Baseline and every 24 weeks ]

EORTC QLQ-C30 (a questionnaire developed to assess the quality of life of cancer patients) and GI.NET21 (module intended for use among patients with G.I.-related neuroendocrine tumours) quality of life questionnaires

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Telotristat Etiprate - Expanded Treatment for Patients With Carcinoid Syndrome Symptoms
• Official Title: A Multicenter, Long-term Extension Study to Further Evaluate the Safety and Tolerability of Telotristat Etiprate (LX1606)
• Brief Summary: The primary objective of this study is to evaluate the long-term safety and tolerability of orally administered telotristat etiprate.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Carcinoid Syndrome

Intervention

Drug: Telotristat etiprate

Telotristat etiprate tablet (250 mg)

Other Name: LX1606

Study Arms

• Experimental: 250 mg Telotristat Etiprate
  One telotristat etiprate (250 mg) tablet administered three times daily.
  Intervention: Drug: Telotristat etiprate
• Experimental: 500 mg Telotristat Etiprate
  Two telotristat etiprate (250 mg) tablets administered three times daily.
  Intervention: Drug: Telotristat etiprate

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 12, 2019): 124
• Original Estimated Enrollment (submitted: December 31, 2013): 160
• Actual Study Completion Date: September 12, 2018
• Actual Primary Completion Date: September 12, 2018 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Australia, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Spain, Sweden, United Kingdom, United States

Administrative Information

• NCT Number: NCT02026063
• Other Study ID Numbers: LX1606.1-302-CS; LX1606.302 ( Other Identifier: Lexicon Pharmaceuticals, Inc. ); 2013-002596-18 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

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

• IPD Sharing Statement: Not Provided
• Responsible Party: Lexicon Pharmaceuticals
• Study Sponsor: Lexicon Pharmaceuticals
• Collaborators: Not Provided

Investigators

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

• PRS Account: Lexicon Pharmaceuticals
• Verification Date: August 2019