Clinical trial NCT03223428: Real-world Evidence Study EvaLuating PAtient-Reported Outcomes With XERMELO
Official title: An Observational Study to Evaluate the Real-world Experience of Patients Who Are Initiating Treatment With Telotristat Ethyl (XERMELO™)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Carcinoid Syndrome
Keywords: neuroendocrine tumors, carcinoid syndrome, telotristat ethyl
Lead sponsor: TerSera Therapeutics LLC
Sponsor class: INDUSTRY
First posted: 2017-07-21
Enrollment: 223
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The study will include patients with CS and who are initiating treatment with XERMELO.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Percentage of Patients Who Are Satisfied With Their Overall Symptom Control
timeFrame: Baseline to 6 months
Secondary outcomes:
measure: Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control
timeFrame: Baseline to 6 months
measure: Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control
timeFrame: Baseline to 6 months
measure: Number of Patients Reporting Reduction in Rescue SSA Use
timeFrame: Baseline to 6 months
measure: Percentage of Patients Reporting Reduction in the Dose of Long-acting SSA Injection
timeFrame: Baseline to 6 months
measure: Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection
timeFrame: Baseline to 6 months
measure: Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC)
timeFrame: Baseline to 6 months
measure: 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
timeFrame: Baseline to 6 months
measure: Percentage of Patients Reporting Weight Gain
timeFrame: Baseline to 6 months
Other outcomes:
Locations:
city: Research Triangle Park
country: United States
facility: RTI-HS
state: North Carolina
Study officials:
affiliation: TerSera Therapeutics LLC
name: Janine North, BS
role: STUDY_DIRECTOR
References:
citation: 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.