Real-world Evidence Study EvaLuating PAtient-Reported Outcomes With XERMELO (RELAX)

An Observational Study to Evaluate the Real-world Experience of Patients Who Are Initiating Treatment With Telotristat Ethyl (XERMELO™)

The primary objective of the study is to estimate the proportion of carcinoid syndrome (CS) patients who are satisfied with their overall symptom control, 6 months after initiating treatment with telotristat ethyl (XERMELO).

Study Overview

• Status: Completed
• Conditions: Carcinoid Syndrome
• Intervention / Treatment: Drug: Xermelo

Detailed Description

This study will evaluate patient-reported outcomes for adults initiating telotristat ethyl (XERMELO) for carcinoid syndrome diarrhea (CSD) not adequately controlled by somatostatin analogs in US clinical practice. The primary objective is satisfaction with overall CS symptom control 6 months after starting XERMELO. Secondary objectives will evaluate satisfaction with control of CSD and flushing, CS symptoms, work productivity and activity impairment, SSA use, and weight. This study will provide real-world patient-reported outcomes in CS with XERMELO treatment for at least 6 months.

• Study Type: Observational
• Enrollment (Actual): 223

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Research Triangle Park, North Carolina, United States, 27709
      • RTI-HS

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Probability Sample

Study Population

The study will include patients with CS and who are initiating treatment with XERMELO.

Description

Inclusion Criteria:

• Adult, ≥18 years of age at the time of informed consent
• A new, valid prescription for XERMELO
• Initiating XERMELO for the treatment of carcinoid syndrome
• Able and willing to provide informed consent prior to participation in the study

Exclusion Criteria:

• Unable to understand and read English
• Unable to access the internet
• Prior exposure to XERMELO

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Carcinoid Syndrome patients initiating Xermelo; Patients with Carcinoid Syndrome who are initiating treatment with XERMELO.	Drug: Xermelo; This study will include patients with carcinoid syndrome who are initiating treatment with XERMELO.; Other Names: Telotristat ethyl

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Patients Who Are Satisfied With Their Overall Symptom Control	The percentage of CS patients who are satisfied with their overall symptom control, 6 months after initiating treatment with telotristat ethyl (XERMELO). Satisfaction with how Xermelo has controlled diarrhea, flushing, and overall CS symptoms was assessed at the initial 6-month follow-up. The questionnaire allowed participants to choose a response on a 5-level Likert scale ranging from "Very dissatisfied" (level 1) to "Very satisfied" (level 5). For the assessments of satisfaction with control of diarrhea and flushing, "Not applicable" (level 0) was a response option for participants who do not have the particular symptom that is being assessed.	Baseline to 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control	Estimate percentage of patients reporting satisfaction with CS-related diarrhea control from Baseline to 6 months after starting Xermelo. At baseline participants were asked, "Currently, how satisfied are you with how your diarrhea is controlled?" At the 6-month follow-up survey, participants were asked, "Currently, how satisfied are you with how Xermelo has controlled your diarrhea?"	Baseline to 6 months
Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control	Estimate percentage of patients reporting satisfaction with CS-related flushing control from Baseline to 6 Months. At baseline, participants who had reported flushing as a symptom were asked, "Currently, how satisfied are you with how your flushing is controlled?" At 6 months, participants who had reported flushing as a symptom at Baseline were asked, "Currently, how satisfied are you with how Xermelo has controlled your flushing?"	Baseline to 6 months
Number of Patients Reporting Reduction in Rescue SSA Use	Number of participants reporting a reduction in rescue SSA use at 6 months after initiating treatment with Xermelo.	Baseline to 6 months
Percentage of Patients Reporting Reduction in the Dose of Long-acting SSA Injection	Percentage of patients reporting a reduction in the dose of long-acting SSA injection 6 months after patients initiated treatment with Xermelo	Baseline to 6 months
Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection	Estimate Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection 6 months after patients initiated treatment with Xermelo	Baseline to 6 months
Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC)	Estimate percentage of patients reporting an overall improvement in CS symptom control (diarrhea, flushing, frequency of BMs) after initiating Xermelo based on Patient Global Impression of Change (PGIC)	Baseline to 6 months
Percentage Change of Patients Who Had Reduction in Work-related Absenteeism, Presenteeism, Activity Impairment, and Overall Productivity Loss From Baseline to 6 Months After Initiating Telotristat Ethyl Based on WPAI-SHP	Estimate of the percentage of patients who had reduction in work-related absenteeism, presenteeism, activity impairment, and overall productivity loss based on Worker Productivity and Activity Impairment: Specific Health Problem v2.0 (WPAI- SHP). The percentage change was measured from baseline to 6 months after treatment with telotristat ethyl.	Baseline to 6 months
Percentage of Patients Reporting Weight Gain	Estimate of the percentage of patients reporting weight gain after initiating Xermelo therapy.	Baseline to 6 months

Collaborators and Investigators

• Sponsor: TerSera Therapeutics LLC
• Investigators: Study Director: Janine North, BS, TerSera Therapeutics LLC

Publications and helpful links

General Publications

• Li D, Darden C, Osman N, Sayeed S, Jackson L, Garbinsky D, Chauhan A. Real-World Clinical and Patient-Reported Outcomes from the Longitudinal Telotristat Ethyl Treatment Registry of Patients with Neuroendocrine Tumors. Cancer Manag Res. 2022 Oct 17;14:3009-3020. doi: 10.2147/CMAR.S386419. eCollection 2022.

Helpful Links

• Prospective, observational, single-arm study evaluated long-term patient-reported outcomes in adults initiating TE in US clinical practice. The primary objective was satisfaction with overall CS symptom control at 6 months.

Study record dates

Study Major Dates

• Study Start (Actual): June 22, 2017
• Primary Completion (Actual): January 14, 2022
• Study Completion (Actual): January 14, 2022

Study Registration Dates

• First Submitted: July 18, 2017
• First Submitted That Met QC Criteria: July 18, 2017
• First Posted (Actual): July 21, 2017

Study Record Updates

• Last Update Posted (Actual): July 24, 2024
• Last Update Submitted That Met QC Criteria: July 15, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: neuroendocrine tumors, carcinoid syndrome, telotristat ethyl
• Additional Relevant MeSH Terms: Chemically-Induced Disorders; Neoplasms by Histologic Type; Neoplasms; Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroendocrine Tumors; Drug-Related Side Effects and Adverse Reactions; Carcinoid Tumor; Malignant Carcinoid Syndrome; Serotonin Syndrome
• Other Study ID Numbers: LX1606.1-401-CS; LX1606.401 (Other Identifier: TerSera Therapeutics)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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