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| ID | Type | Description | Link |
|---|---|---|---|
| LX1606.401 | Other Identifier | TerSera Therapeutics |
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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Carcinoid Syndrome patients initiating Xermelo | Patients with Carcinoid Syndrome who are initiating treatment with XERMELO. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Xermelo | Drug | This study will include patients with carcinoid syndrome who are initiating treatment with XERMELO. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control | 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?" |
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Inclusion Criteria:
Exclusion Criteria:
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The study will include patients with CS and who are initiating treatment with XERMELO.
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| Name | Affiliation | Role |
|---|---|---|
| Janine North, BS | TerSera Therapeutics LLC | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| RTI-HS | Research Triangle Park | North Carolina | 27709 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36262750 | Result | 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. |
| Label | URL |
|---|---|
| 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. | View source |
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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.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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The analysis population will include all patients who provide consent, are enrolled into the study, and have data available for 1 or more of the primary and secondary endpoints.
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Age was not collected for one (1) patient. |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Posted | Count of Participants | Participants | Baseline to 6 months |
|
Adverse events not collected as part of the survey.
All-cause mortality, serious and other (not serious) adverse events were not collected or assessed during this study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
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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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Noran Osman, PharmD, MHS, Associate Director, External Research and Medical | TerSera Therapeutics LLC | +1-224-754-4605 | nosman@tersera.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 6, 2020 | Apr 19, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 19, 2019 | Apr 19, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D020230 | Serotonin Syndrome |
| D018358 | Neuroendocrine Tumors |
| ID | Term |
|---|---|
| D064420 | Drug-Related Side Effects and Adverse Reactions |
| D064419 | Chemically-Induced Disorders |
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
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| ID | Term |
|---|---|
| C000621725 | telotristat ethyl |
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| Baseline to 6 months |
| Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control | 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?" | Baseline to 6 months |
| Number of Patients Reporting Reduction in Rescue SSA Use | Number of participants reporting a reduction in rescue SSA use at 6 months after initiating treatment with Xermelo. | Baseline to 6 months |
| Percentage of Patients Reporting Reduction in the Dose of Long-acting SSA Injection | Percentage of patients reporting a reduction in the dose of long-acting SSA injection 6 months after patients initiated treatment with Xermelo | Baseline to 6 months |
| Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection | Estimate Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection 6 months after patients initiated treatment with Xermelo | Baseline to 6 months |
| Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC) | 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) | Baseline to 6 months |
| 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 | 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. | Baseline to 6 months |
| Percentage of Patients Reporting Weight Gain | Estimate of the percentage of patients reporting weight gain after initiating Xermelo therapy. | Baseline to 6 months |
| Withdrawal by Subject |
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| Count of Participants |
| Participants |
| No |
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| Age, Continuous | One patient did not provide age during the baseline survey | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Baseline Weight | Weight collected in kilograms. | Mean | Standard Deviation | kilogram |
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| Secondary | Percentage of Patients Reporting Satisfaction of CS-related Diarrhea Control | 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?" | Posted | Count of Participants | Participants | Baseline to 6 months |
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|
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| Secondary | Percentage of Patients Reporting Satisfaction of CS- Related Flushing Control | 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?" | Posted | Count of Participants | Participants | Baseline to 6 months |
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| Secondary | Number of Patients Reporting Reduction in Rescue SSA Use | Number of participants reporting a reduction in rescue SSA use at 6 months after initiating treatment with Xermelo. | Posted | Number | participants | Baseline to 6 months |
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| Secondary | Percentage of Patients Reporting Reduction in the Dose of Long-acting SSA Injection | Percentage of patients reporting a reduction in the dose of long-acting SSA injection 6 months after patients initiated treatment with Xermelo | Posted | Count of Participants | Participants | Baseline to 6 months |
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| Secondary | Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection | Estimate Percentage of Patients Reporting Reduction in the Frequency of Long-acting SSA Injection 6 months after patients initiated treatment with Xermelo | 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. | Posted | Count of Participants | Participants | Baseline to 6 months |
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|
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| Secondary | Percentage of Patients Reporting an Overall Improvement in CS Symptom Control Based on Patient Global Impression of Change (PGIC) | 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) | Posted | Count of Participants | Participants | Baseline to 6 months |
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| Secondary | 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 | 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. | 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. | Posted | Mean | Standard Deviation | percentage change baseline to Month 6 | Baseline to 6 months |
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| Secondary | Percentage of Patients Reporting Weight Gain | Estimate of the percentage of patients reporting weight gain after initiating Xermelo therapy. | Posted | Count of Participants | Participants | Baseline to 6 months |
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| 0 |
| 0 |
| 0 |
| 0 |
| 0 |
| 0 |
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| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| Somewhat dissatisfied |
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| Very dissatisfied |
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| Somewhat dissatisfied |
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| Very dissatisfied |
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| No change |
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| Somewhat worse |
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| Much worse |
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| very much worse |
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| Work productivity loss |
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| Activity impairment |
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