Real-world Evidence Study EvaLuating PAtient-Reported Outcomes With XERMELO

Completed

• Conditions: Carcinoid Syndrome
• Interventions: Drug: Xermelo

• Registration Number: NCT03223428
• Lead Sponsor: TerSera Therapeutics LLC

Brief Summary

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).

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 Timeline

• Study Start: 2017-06-22
• Study First Submitted: 2017-07-18
• Study First Submitted QC: 2017-07-18
• Study First Posted: 2017-07-21
• Primary Completion: 2022-01-14
• Study Completion: 2022-01-14
• Results First Submitted: 2023-04-19
• Results First Submitted QC: 2024-01-30
• Status Verified: 2024-07
• Results First Posted: 2024-07-15
• Last Update Submitted: 2024-07-15
• Last Update Posted: 2024-07-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 223

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

Read More

Exclusion Criteria

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

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Carcinoid Syndrome patients initiating Xermelo	Xermelo	Patients with Carcinoid Syndrome who are initiating treatment with XERMELO.

Primary Outcome Measures

Name	Time	Method
Percentage of Patients Who Are Satisfied With Their Overall Symptom Control	Baseline to 6 months	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.

Secondary Outcome Measures

Name	Time	Method
Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC)	Baseline to 6 months	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)
Number of Patients Reporting Reduction in Rescue SSA Use	Baseline to 6 months	Number of participants reporting a reduction in rescue SSA use at 6 months after initiating treatment with Xermelo.
Percentage of Patients Reporting Reduction in the Dose of Long-acting SSA Injection	Baseline to 6 months	Percentage of patients reporting a reduction in the dose of long-acting SSA injection 6 months after patients initiated treatment with Xermelo
Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection	Baseline to 6 months	Estimate Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection 6 months after patients initiated treatment with Xermelo
Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control	Baseline to 6 months	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?"
Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control	Baseline to 6 months	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?"
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	Baseline to 6 months	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.
Percentage of Patients Reporting Weight Gain	Baseline to 6 months	Estimate of the percentage of patients reporting weight gain after initiating Xermelo therapy.

Trial Locations

Locations (1)

RTI-HS; 🇺🇸 Research Triangle Park, North Carolina, United States