Trial Outcomes & Findings for Real-world Evidence Study EvaLuating PAtient-Reported Outcomes With XERMELO (NCT NCT03223428)
NCT ID: NCT03223428
Last Updated: 2024-07-24
Results Overview
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.
COMPLETED
223 participants
Baseline to 6 months
2024-07-24
Participant Flow
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.
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
223
|
|
Overall Study
COMPLETED
|
125
|
|
Overall Study
NOT COMPLETED
|
98
|
Reasons for withdrawal
| Measure |
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.
|
|---|---|
|
Overall Study
No longer taking Xermelo
|
27
|
|
Overall Study
Declined Consent
|
1
|
|
Overall Study
Non-responders
|
56
|
|
Overall Study
Withdrawal by Subject
|
14
|
Baseline Characteristics
Age was not collected for one (1) patient.
Baseline characteristics by cohort
| Measure |
Carcinoid Syndrome Patients Initiating Xermelo
n=223 Participants
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.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=222 Participants • Age was not collected for one (1) patient.
|
|
Age, Categorical
Between 18 and 65 years
|
138 Participants
n=222 Participants • Age was not collected for one (1) patient.
|
|
Age, Categorical
>=65 years
|
84 Participants
n=222 Participants • Age was not collected for one (1) patient.
|
|
Age, Continuous
|
60.8 years
STANDARD_DEVIATION 10.99 • n=222 Participants • One patient did not provide age during the baseline survey
|
|
Sex: Female, Male
Female
|
135 Participants
n=223 Participants
|
|
Sex: Female, Male
Male
|
88 Participants
n=223 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
13 Participants
n=223 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
208 Participants
n=223 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=223 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=223 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=223 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=223 Participants
|
|
Race (NIH/OMB)
Black or African American
|
16 Participants
n=223 Participants
|
|
Race (NIH/OMB)
White
|
199 Participants
n=223 Participants
|
|
Race (NIH/OMB)
More than one race
|
4 Participants
n=223 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=223 Participants
|
|
Region of Enrollment
United States
|
223 participants
n=223 Participants
|
|
Baseline Weight
|
80.9 kilogram
STANDARD_DEVIATION 22.5 • n=223 Participants
|
PRIMARY outcome
Timeframe: Baseline to 6 monthsThe 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.
Outcome measures
| Measure |
Carcinoid Syndrome Patients Initiating Xermelo
n=125 Participants
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.
|
|---|---|
|
Percentage of Patients Who Are Satisfied With Their Overall Symptom Control
Very Satisfied with symptom control
|
37 Participants
|
|
Percentage of Patients Who Are Satisfied With Their Overall Symptom Control
Somewhat satisfied with symptom control
|
58 Participants
|
|
Percentage of Patients Who Are Satisfied With Their Overall Symptom Control
Neither satisfied or dissatisfied with symptom control
|
22 Participants
|
|
Percentage of Patients Who Are Satisfied With Their Overall Symptom Control
Somewhat dissatisfied with symptom control
|
5 Participants
|
|
Percentage of Patients Who Are Satisfied With Their Overall Symptom Control
Very dissatisfied with symptom control
|
3 Participants
|
SECONDARY outcome
Timeframe: Baseline to 6 monthsEstimate 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?"
Outcome measures
| Measure |
Carcinoid Syndrome Patients Initiating Xermelo
n=125 Participants
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.
|
|---|---|
|
Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control
Very satisfied
|
46 Participants
|
|
Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control
Somewhat satisfied
|
51 Participants
|
|
Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control
Neither satisfied nor dissatisfied
|
12 Participants
|
|
Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control
Somewhat dissatisfied
|
11 Participants
|
|
Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control
Very dissatisfied
|
5 Participants
|
SECONDARY outcome
Timeframe: Baseline to 6 monthsEstimate 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?"
Outcome measures
| Measure |
Carcinoid Syndrome Patients Initiating Xermelo
n=108 Participants
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.
|
|---|---|
|
Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control
Very satisfied
|
24 Participants
|
|
Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control
Somewhat satisfied
|
39 Participants
|
|
Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control
Neither satisfied nor dissatisfied
|
34 Participants
|
|
Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control
Somewhat dissatisfied
|
7 Participants
|
|
Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control
Very dissatisfied
|
4 Participants
|
SECONDARY outcome
Timeframe: Baseline to 6 monthsNumber of participants reporting a reduction in rescue SSA use at 6 months after initiating treatment with Xermelo.
Outcome measures
| Measure |
Carcinoid Syndrome Patients Initiating Xermelo
n=104 Participants
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.
|
|---|---|
|
Number of Patients Reporting Reduction in Rescue SSA Use
|
21 participants
|
SECONDARY outcome
Timeframe: Baseline to 6 monthsPercentage of patients reporting a reduction in the dose of long-acting SSA injection 6 months after patients initiated treatment with Xermelo
Outcome measures
| Measure |
Carcinoid Syndrome Patients Initiating Xermelo
n=125 Participants
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.
|
|---|---|
|
Percentage of Patients Reporting Reduction in the Dose of Long-acting SSA Injection
|
7 Participants
|
SECONDARY outcome
Timeframe: Baseline to 6 monthsPopulation: There was a discrepancy between how many patients answered the survey for "Reduction in dose of long-acting SSA" (n=125) and "Reduction in frequency of long-acting SSA" (n=123). Two people did not answer the latter question but did answer the former.
Estimate Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection 6 months after patients initiated treatment with Xermelo
Outcome measures
| Measure |
Carcinoid Syndrome Patients Initiating Xermelo
n=123 Participants
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.
|
|---|---|
|
Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection
|
9 Participants
|
SECONDARY outcome
Timeframe: Baseline to 6 monthsEstimate 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)
Outcome measures
| Measure |
Carcinoid Syndrome Patients Initiating Xermelo
n=125 Participants
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.
|
|---|---|
|
Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC)
Very much improved
|
20 Participants
|
|
Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC)
Much improved
|
28 Participants
|
|
Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC)
Somewhat improved
|
49 Participants
|
|
Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC)
No change
|
20 Participants
|
|
Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC)
Somewhat worse
|
6 Participants
|
|
Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC)
Much worse
|
1 Participants
|
|
Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC)
very much worse
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline to 6 monthsPopulation: Only 124 patients answered the work activity and impairment questionnaire. Not all patients were employed and only a small subset were eligible to answer the Absenteeism, Presenteeism and Work Productivity Loss. Only 34, 32 and 32 patients answered those questionnaires, respectively.
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.
Outcome measures
| Measure |
Carcinoid Syndrome Patients Initiating Xermelo
n=125 Participants
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.
|
|---|---|
|
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
Absenteeism
|
4.1 percentage change baseline to Month 6
Standard Deviation 21.86
|
|
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
Presenteeism
|
-9.7 percentage change baseline to Month 6
Standard Deviation 20.76
|
|
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
Work productivity loss
|
-10.1 percentage change baseline to Month 6
Standard Deviation 19.60
|
|
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
Activity impairment
|
-8.1 percentage change baseline to Month 6
Standard Deviation 26.86
|
SECONDARY outcome
Timeframe: Baseline to 6 monthsEstimate of the percentage of patients reporting weight gain after initiating Xermelo therapy.
Outcome measures
| Measure |
Carcinoid Syndrome Patients Initiating Xermelo
n=125 Participants
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.
|
|---|---|
|
Percentage of Patients Reporting Weight Gain
|
24 Participants
|
Adverse Events
Carcinoid Syndrome Patients Initiating Xermelo
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Noran Osman, PharmD, MHS, Associate Director, External Research and Medical
TerSera Therapeutics LLC
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place