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

NCT03223428 | Completed Results FDA Drug

• 2 other identifiers: LX1606.1-401-CSLX1606.401
• Study Type: observational
• Target: 223
• Locations: 1 country
• Sites: 1

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).

Conditions

Carcinoid Syndrome

Keywords

neuroendocrine tumors, carcinoid syndrome, telotristat ethyl

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (8)

• Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control

  Baseline to 6 months

• Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control

  Baseline to 6 months

• Number of Patients Reporting Reduction in Rescue SSA Use

  Baseline to 6 months

• Percentage of Patients Reporting Reduction in the Dose of Long-acting SSA Injection

  Baseline to 6 months

• Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection

  Baseline to 6 months

Study Arms (1)

Carcinoid Syndrome patients initiating Xermelo

Patients with Carcinoid Syndrome who are initiating treatment with XERMELO.

Drug: Xermelo

Interventions

Xermelo DRUG

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

Also known as: Telotristat ethyl

Carcinoid Syndrome patients initiating Xermelo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• TerSera Therapeutics LLC: lead

Study Sites (1)

RTI-HS

Research Triangle Park, North Carolina, 27709, United States

Location

Related Publications (1)

• 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.

  PMID: 36262750 RESULT

Related 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.

MeSH Terms

Conditions

Serotonin Syndrome; Neuroendocrine Tumors

Interventions

telotristat ethyl

Condition Hierarchy (Ancestors)

Drug-Related Side Effects and Adverse Reactions; Chemically-Induced Disorders; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue

Limitations and Caveats

A potential for responder bias was possible as results were self-reported. Decreased number of responders presented challenges to interpretation after approx. 24-30 months, hence findings should be interpreted with caution. Adverse event safety data collection: Out of scope of this study. Reported adverse events, if occurred, were to be collected and reported to the Sponsor outside of the survey collection process.

Results Point of Contact

• Title: Noran Osman, PharmD, MHS, Associate Director, External Research and Medical
• Organization: TerSera Therapeutics LLC

nosman@tersera.com

+1-224-754-4605

Study Officials

• Janine North, BS

  TerSera Therapeutics LLC

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 3 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 18, 2017
• First Posted: July 21, 2017
• Study Start: June 22, 2017
• Primary Completion: January 14, 2022
• Study Completion: January 14, 2022
• Last Updated: July 24, 2024
• Results First Posted: July 15, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)