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

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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03223428

Acronym

RELAX

Enrollment

223

Registered

2017-07-21

Start date

2017-06-22

Completion date

2022-01-14

Last updated

2024-07-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Carcinoid Syndrome

Keywords

neuroendocrine tumors, carcinoid syndrome, telotristat ethyl

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.

Interventions

DRUGXermelo

This study will include patients with carcinoid syndrome who are initiating treatment with XERMELO.

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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?
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 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 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)
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.
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

Countries

United States

Participant flow

Recruitment details

Eligible patients were invited to participate in the RELAX study by the enrolling Specialty Pharmacies EnvoyHealth (also known as Diplomat Specialty Pharmacy and Optum Specialty Pharmacy) and Biologics Specialty Pharmacy.

Participants by arm

Arm	Count
Carcinoid Syndrome Patients Initiating Xermelo Patients with Carcinoid Syndrome who are initiating treatment with XERMELO. Xermelo: This study will include patients with carcinoid syndrome who are initiating treatment with XERMELO.	223
Total	223

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Declined Consent	1
Overall Study	No longer taking Xermelo	27
Overall Study	Non-responders	56
Overall Study	Withdrawal by Subject	14

Baseline characteristics

Characteristic	Carcinoid Syndrome Patients Initiating Xermelo
Age, Categorical <=18 years	0 Participants
Age, Categorical >=65 years	84 Participants
Age, Categorical Between 18 and 65 years	138 Participants
Age, Continuous	60.8 years STANDARD_DEVIATION 10.99
Baseline Weight	80.9 kilogram STANDARD_DEVIATION 22.5
Ethnicity (NIH/OMB) Hispanic or Latino	13 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	208 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants
Race (NIH/OMB) Asian	1 Participants
Race (NIH/OMB) Black or African American	16 Participants
Race (NIH/OMB) More than one race	4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants
Race (NIH/OMB) White	199 Participants
Region of Enrollment United States	223 participants
Sex: Female, Male Female	135 Participants
Sex: Female, Male Male	88 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 0
other Total, other adverse events	0 / 0
serious Total, serious adverse events	0 / 0

Outcome results

Primary

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.