Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| NCI-2021-00852 | Registry Identifier | CTRP (Clinical Trial Reporting Program) | |
| 2019-1205 | Other Identifier | M D Anderson Cancer Center |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This phase III trial compares the effect of telotristat ethyl and the current standard of care somatostatin analog therapy or somatostatin analog therapy alone in treating patients with neuroendocrine tumor that has spread to other places in the body (metastatic). Telotristat ethyl and somatostatin analog therapy may help to control carcinoid syndrome and carcinoid heart disease.
PRIMARY OBJECTIVE:
I. To estimate the percent change in N-terminal pro B-type natriuretic peptide (NT-proBNP) at 6 month visit from baseline after initiation of study drug in each arm and to compare the percent change between the two study arms.
SECONDARY OBJECTIVES:
I. To evaluate the change in functional capacity from baseline at 3 and 6 month visits as assessed by a 6 minute walk test (6MWT) in each arm.
II. To evaluate changes in echocardiographic parameters (Carcinoid Valvular Heart Disease [CVHD] score, global longitudinal myocardial strain assessment of the left and right ventricle/tricuspid annular plane systolic excursion [TAPSE]) from baseline to 3 and 6 month visits in each arm.
III. To evaluate the change from baseline to 3 and 6 month visits in plasma 5-hydroxyindoleacetic acid (5-HIAA) levels in each arm.
IV. To evaluate the change from baseline to 3 and 6 month visits in high sensitivity troponin T in each arm.
V. To evaluate the change from baseline to 3 and 6 month visits in health related quality of life with using the MD Anderson Symptom Inventory (MDASI) in each arm.
VI. To evaluate compliance of medications.
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM A: Patients receive telotristat ethyl orally (PO) three times daily (TID) and somatostatin analog therapy (SSA) for 6 months in the absence of disease progression or unacceptable toxicity.
ARM B: Patients receive placebo PO TID and SSA for 6 months in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for 30 days.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm A (telotristat ethyl, SSA) | Experimental | Patients receive telotristat ethyl PO TID and SSA for 6 months in the absence of disease progression or unacceptable toxicity. |
|
| Arm B (placebo, SSA) | Active Comparator | Patients receive placebo PO TID and SSA for 6 months in the absence of disease progression or unacceptable toxicity. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo Administration | Drug | Given PO |
| |
| Questionnaire Administration |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change in NTproBNP at 6 Months Visit From Baseline | Percent change in N-terminal pro B-type natriuretic peptide (NT-proBNP) from baseline to 6 months defined as (NTproBNP at 6 month - NTproBNP at baseline)/(NTproBNP at baseline)*100 | Baseline and at 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in 6MWT at 3 Month Visit | Change in functional capacity from baseline to 3-month visit, assessed by a 6-minute walk test (6MWT), where 6MWT is defined as the distance walked in 6 minutes, and the change is calculated as: 6MWT at 3 months minus 6MWT at baseline | Baseline and 3 month follow-up |
| Change in 6MWT at 6 Month Visit |
Not provided
Inclusion Criteria:
Patients who are >= 18 years old will be eligible for the study
Histopathologically-confirmed,metastatic neuroendocrine tumor and/or locally/regionally advanced neuroendocrine tumor
Documented history of carcinoid syndrome based on clinical parameters
Currently receiving stable-dose somatostatin analog (SSA) therapy defined as >= 2 months
Dose of long-acting release (LAR) or depot SSA therapy and on at least:
Ability and willingness to provide written informed consent
Patients of childbearing potential must agree to use an adequate method of contraception during the study and for 30 days after the last dose of telotristat ethyl
Eastern Cooperative Oncology Group (ECOG) 0-2
Exclusion Criteria:
Previous exposure to telotristat ethyl (XERMELO) in the last 3 months
History of active treatment for malignancy, other than neuroendocrine tumor (malignancies that in the opinion of the Investigator are considered cured, may participate)
Treatment with any tumor directed therapy, including interferon, chemotherapy, mechanistic target of rapamycin (mTOR) inhibitors < 4 weeks prior to screening, or hepatic embolization, radiotherapy, peptide receptor radionuclide therapy, and/or tumor debulking < 12 weeks prior to screening
History of short bowel syndrome or other known causes of diarrhea unrelated to carcinoid syndrome
Clinically significant (as per primary investigators judgement) cardiac arrhythmia, bradycardia, tachycardia that would compromise patient safety or the outcome of the study
Estimated glomerular filtration rate estimated glomerular filtration rate (eGFR) < 30 ml/min
Hepatic laboratory values of aspartate transaminase (AST) or alanine aminotransferase (ALT):
Pregnant or lactating patients
Patients receiving everolimus due to poor response to SSA
Life expectancy < 6 months
Any other clinically significant laboratory abnormality that would compromise patient safety or the outcome of the study as per primary investigators judgement
Any clinically significant and/or uncontrolled cardiac-related abnormality that would compromise patient safety or the outcome of the study including as per primary investigators judgement, but not limited to:
Current complaints of persistent constipation or history of chronic constipation, bowel obstruction or fecaloma within the past 6 months
Investigator assessment of known history and/or uncontrolled hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCV Ab), or human immunodeficiency virus (HIV)-1 or HIV-2
History of substance or alcohol abuse (Diagnostic and Statistical Manual of Mental Disorders 5th edition [DSM-V] Criteria for Substance-Related Disorders) within the past 2 years
History of galactose intolerance, deficiency of Lapp lactase, or glucose-galactose malabsorption
Receipt of any investigational agent or study treatment (other treatment nor approved by Food and Drug Administration [FDA] for carcinoid syndrome or carcinoid heart disease) within the past 30 days
Existence of any surgical or medical condition that, in the judgment of the Investigator, might compromise patient safety or the outcome of the study
Presence of any clinically significant findings (relative to the patient population) during review of medical history or upon PE that, in the investigator's opinion, would compromise patient safety or the outcome of the study (e.g., psychiatric illness/social situations that would limit compliance with study requirements)
Unable or unwilling to communicate or cooperate with the Investigator for any reason
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Cezar A Iliescu, MD | M.D. Anderson Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| M D Anderson Cancer Center | Houston | Texas | 77030 | United States |
Not provided
| Label | URL |
|---|---|
| MD Anderson Cancer Center | View source |
Not provided
Of the 79 participants who consented and enrolled in the study, 6 withdrew before arm assignment. These 6 participants were not randomized and were excluded from the study. The remaining 73 participants were randomized to one of two arms.
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Placebo + SSA | Placebo-matching telotristat ethyl tablet TID + stable-dose somatostatin analog (SSA) therapy |
| FG001 | Xermelo + SSA | Telotristat ethyl 500 mg po TID + stable-dose somatostatin analog (SSA) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Dec 11, 2025 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Other |
Ancillary studies |
|
| Telotristat Ethyl | Drug | Given PO |
|
Change in functional capacity from baseline to 6-month visit, assessed by a 6-minute walk test (6MWT), where 6MWT is defined as the distance walked in 6 minutes, and the change is calculated as: 6MWT at 6 months minus 6MWT at baseline |
| Baseline and 6 month follow-up |
| Change in CVHD % Score From Baseline to 3 Month Visit | The Carcinoid Valvular Heart disease (CVHD) % score at 3 months minus CVHD % score at baseline. CVHD score was determined by 1) tricuspid valve appearance, 2) tricuspid regurgitation severity by either spectral pulsed wave or color dopplet flow mapping, 3) pulmonary stenosis severity by spectral pulsed or continuous wave Doppler, and 4) pulmonary insufficiency severity by color Doppler. CVHD % score was calculated as total points assigned across four echo parameters (specified above 1-4)) divided by the maximum possible points (14) and multiplied by 100. The CVHD % score ranges from 0% to 100%, with higher values indicating more severe disease. The change in CVHD % score from baseline to 3 months reflects disease progression or improvement: a positive change indicates worsening valvular involvement, a negative change indicates improvement, and zero indicates stability. | Baseline and 3 month follow-up |
| Change in CVHD Score From Baseline to 6 Month Visit | The Carcinoid Valvular Heart disease (CVHD) % score at 6 months minus CVHD % score at baseline. CVHD score was determined by 1) tricuspid valve appearance, 2) tricuspid regurgitation severity by either spectral pulsed wave or color dopplet flow mapping, 3) pulmonary stenosis severity by spectral pulsed or continuous wave Doppler, and 4) pulmonary insufficiency severity by color Doppler. CVHD % score was calculated as total points assigned across four echo parameters divided by the maximum possible points (14) and multiplied by 100. The CVHD % score ranges from 0% to 100%, with higher values indicating more severe disease. The change in CVHD % score from baseline to 6 months reflects disease progression or improvement: a positive change indicates worsening valvular involvement, a negative change indicates improvement, and zero indicates stability. | Baseline and 6 month follow-up |
| Percentage of Participants With Significant Change in Strain-RV From Baseline at 3 Months | Percentage of participants with a significant change in right ventricular global longitudinal strain (strain-RV) from baseline to 3 months. A significant change is defined as an 15% or more change (increase or decrease) from baseline to 3 months. The Exact 2-sided 95% CI is based on the observed proportion of participants. | Baseline and 3 month follow-up |
| Percentage of Participants With Significant Change in Strain-RV From Baseline at 6 Months | Percentage of participants with a significant change in right ventricular global longitudinal strain (strain-RV) from baseline to 6 months. A significant change is defined as an 15% or more change (increase or decrease) from baseline to 6 months. The Exact 2-sided 95% CI is based on the observed proportion of participants. | Baseline and 6 month follow-up |
| Percentage of Participants With Significant Change in Strain-LV From Baseline at 3 Months | Percentage of participants with a significant change in left ventricular global longitudinal strain (strain-LV) from baseline to 3 months. A significant change is defined as an 15% or more change (increase or decrease) from baseline to 3 months. The Exact 2-sided 95% CI is based on the observed proportion of participants. | Baseline and 3 month follow-up |
| Percentage of Participants With Significant Change in Strain-LV From Baseline at 6 Months | Percentage of participants with a significant change in left ventricular global longitudinal strain (strain-LV) from baseline to 6 months. A significant change is defined as an 15% or more change (increase or decrease) from baseline to 6 months. The Exact 2-sided 95% CI is based on the observed proportion of participants. | Baseline and 6 month follow-up |
| Percentage of Participants With Normal TAPSE at 3 Months | Percentage of participants with a normal Tricuspid Annular Plane Systolic Excursion (TAPSE) at 3 months, where TAPSE ≥1.6cm is defined as normal. The Exact 2-sided 95% CI is based on the observed proportion of participants. | 3 month follow-up |
| Percentage of Participants With Normal TAPSE at 6 Months | Percentage of participants with a normal Tricuspid Annular Plane Systolic Excursion (TAPSE) at 6 months, where TAPSE ≥1.6cm is defined as normal. The Exact 2-sided 95% CI is based on the observed proportion of participants. | 6 month follow-up |
| Change in 5HIAA From Baseline to 3 Months | Change in plasma 5HIAA (5-Hydroxyindoleacetic acid) level from baseline to 3 months. The change is calculated as: 5HIAA at 3 months minus 5HIAA at baseline | Baseline and 3 month follow-up |
| Change in 5HIAA From Baseline to 6 Months | Change in 5HIAA measurement from baseline to 6 months | Baseline and 6 month follow-up |
| Change in Troponin From Baseline to 3 Months | Change in troponin level from baseline to 3 months, calculated as: troponine at 3 months minus troponin at baseline | Baseline and 3 months follow-up |
| Change in Troponin From Baseline to 6 Months | Change in troponin level from baseline to 6 months, calculated as: troponine at 6 months minus troponin at baseline | Baseline and 6 months follow-up |
| Change in MDASI Mean Core Symptom Score From Baseline to 3 Months | Change in MD Anderson Symptom Inventory (MDASI) mean core symptom score from baseline to 3 months. The MDASI mean core symptom score is calculated as the mean of 13 core symptom items, each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine"), with the higher scores indicating worse symptoms. The change is computed as: score at 3 months minus score at baseline | Baseline and 3 months follow-up |
| Change in Core Symptom Score From Baseline to 6 Months | Change in MD Anderson Symptom Inventory (MDASI) mean core symptom score from baseline to 6 months. The MDASI mean core symptom score is calculated as the mean of 13 core symptom items, each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine"), with the higher scores indicating worse symptoms. The change is computed as: score at 6 months minus score at baseline | Baseline and 6 months follow-up |
| Change in MDASI Mean Total Symptom Score From Baseline to 3 Months | Change in MD Anderson Symptom Inventory (MDASI) mean total symptom score from baseline to 3 months. The MDASI mean total symptom score is calculated as the mean symptom severity of 21 symptom items (13 core symptom items and 8 additional symptom items), each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine), with the higher scores indicating worse symptoms. The change is computed as: score at 3 months minus score at baseline | Baseline and 3 months follow-up |
| Change in MDASI Mean Total Symptom Score From Baseline to 6 Months | Change in MD Anderson Symptom Inventory (MDASI) mean total symptom score from baseline to 6 months. The MDASI mean total symptom score is calculated as the mean symptom severity of 21 symptom items (13 core symptom items and 8 additional symptom items), each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine), with the higher scores indicating worse symptoms. The change is computed as: score at 6 months minus score at baseline | Baseline and 6 months follow-up |
| Change in MDASI Mean Interference Score From Baseline to 3 Months | Change in MD Anderson Symptom Inventory (MDASI) mean interference score from baseline to 3 months. The MDASI mean interference score is calculated as the mean of 6 interference items, each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine"), with the higher scores indicating worse interference. The change is computed as: score at 3 months minus score at baseline | Baseline and 3 months follow-up |
| Change in Interference Score From Baseline to 6 Months | Change in MD Anderson Symptom Inventory (MDASI) mean interference score from baseline to 6 months. The MDASI mean interference score is calculated as the mean of 6 interference items, each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine"), with the higher scores indicating worse interference. The change is computed as: score at 6 months minus score at baseline | Baseline and 6 months follow-up |
| Compliance>=70% While the Patients Were in the Study | Percentage of participants classified as compliant (compliance≥70%) during the study. Compliance is calculated as the total number of pills taken divided by the toal number of pills dispensed while the patient was on the study. Participants with compliance≥70% are considered compliant; otherwise, not compliant. | Baseline, 3 months follow-up, 6 months follow-up |
| Randomized |
|
| Treated |
|
| 3 Month Follow-up |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo + SSA | Placebo-matching telotristat ethyl tablet TID + stable-dose somatostatin analog (SSA) therapy |
| BG001 | Xermelo + SSA | Telotristat ethyl 500 mg po TID + stable-dose somatostatin analog (SSA) |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| 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 | Percent Change in NTproBNP at 6 Months Visit From Baseline | Percent change in N-terminal pro B-type natriuretic peptide (NT-proBNP) from baseline to 6 months defined as (NTproBNP at 6 month - NTproBNP at baseline)/(NTproBNP at baseline)*100 | Patients who had NTproBNP at both baseline and 6 months | Posted | Median | Inter-Quartile Range | Percent change from baseline | Baseline and at 6 months |
|
|
| ||||||||||||||||||||||||||||
| Secondary | Change in 6MWT at 3 Month Visit | Change in functional capacity from baseline to 3-month visit, assessed by a 6-minute walk test (6MWT), where 6MWT is defined as the distance walked in 6 minutes, and the change is calculated as: 6MWT at 3 months minus 6MWT at baseline | Of the 29 participants who completed 3-month follow-up in the Placebo + SSA arm, 25 completed the 6MWT at both baseline and 3 months. In the Xermelo + SSA arm, all 29 participants who completed 3-month follow-up also completed the 6MWT at both baseline and at 3 months. | Posted | Median | Inter-Quartile Range | meters | Baseline and 3 month follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in 6MWT at 6 Month Visit | Change in functional capacity from baseline to 6-month visit, assessed by a 6-minute walk test (6MWT), where 6MWT is defined as the distance walked in 6 minutes, and the change is calculated as: 6MWT at 6 months minus 6MWT at baseline | Of the 27 participants who completed 6-month follow-up in the Placebo + SSA arm, 26 participants completed 6MWT at both baseline and at 6 months. All 26 participants who completed 6-month follow-up in the Xermelo + SSA arm completed the 6MWT at both baseline and 6 months. | Posted | Median | Inter-Quartile Range | meters | Baseline and 6 month follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in CVHD % Score From Baseline to 3 Month Visit | The Carcinoid Valvular Heart disease (CVHD) % score at 3 months minus CVHD % score at baseline. CVHD score was determined by 1) tricuspid valve appearance, 2) tricuspid regurgitation severity by either spectral pulsed wave or color dopplet flow mapping, 3) pulmonary stenosis severity by spectral pulsed or continuous wave Doppler, and 4) pulmonary insufficiency severity by color Doppler. CVHD % score was calculated as total points assigned across four echo parameters (specified above 1-4)) divided by the maximum possible points (14) and multiplied by 100. The CVHD % score ranges from 0% to 100%, with higher values indicating more severe disease. The change in CVHD % score from baseline to 3 months reflects disease progression or improvement: a positive change indicates worsening valvular involvement, a negative change indicates improvement, and zero indicates stability. | Of the 29 participants who completed 3-month follow-up in the Placebo + SSA arm, 7 completed CVHD evaluations at both baseline and 3 months. In the Xermelo + SSA arm, 5 of the 29 participants who completed 3-month follow-up completed CVHD evaluations at both baseline and 3 months. | Posted | Median | Inter-Quartile Range | Scores on a scale | Baseline and 3 month follow-up |
| ||||||||||||||||||||||||||||||
| Secondary | Change in CVHD Score From Baseline to 6 Month Visit | The Carcinoid Valvular Heart disease (CVHD) % score at 6 months minus CVHD % score at baseline. CVHD score was determined by 1) tricuspid valve appearance, 2) tricuspid regurgitation severity by either spectral pulsed wave or color dopplet flow mapping, 3) pulmonary stenosis severity by spectral pulsed or continuous wave Doppler, and 4) pulmonary insufficiency severity by color Doppler. CVHD % score was calculated as total points assigned across four echo parameters divided by the maximum possible points (14) and multiplied by 100. The CVHD % score ranges from 0% to 100%, with higher values indicating more severe disease. The change in CVHD % score from baseline to 6 months reflects disease progression or improvement: a positive change indicates worsening valvular involvement, a negative change indicates improvement, and zero indicates stability. | Of the 27 participants who completed 6-month follow-up in the Placebo + SSA arm, 24 participants completed CVHD evaluations at both baseline and 6 months. In the Xermelo + SSA arm, 23 of the 26 participants who completed 6-month follow-up completed CVHD evaluation at both baseline and at 6 months. The values reported represent the median values for change in CVHD score for each Arm, and do not represent non-meaningful placeholders. | Posted | Median | Inter-Quartile Range | Scores on a scale | Baseline and 6 month follow-up |
| ||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Significant Change in Strain-RV From Baseline at 3 Months | Percentage of participants with a significant change in right ventricular global longitudinal strain (strain-RV) from baseline to 3 months. A significant change is defined as an 15% or more change (increase or decrease) from baseline to 3 months. The Exact 2-sided 95% CI is based on the observed proportion of participants. | Of the 29 participants who completed 3 months follow-up in the Placebo + SSA arm, 3 completed strain RV evaluations at both baseline and 3 months. In the Xermelo + SSA arm, 7 of the 29 participants who completed 3-month follow-up completed strain RV evaluations at both baseline and at 3 months. | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline and 3 month follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Significant Change in Strain-RV From Baseline at 6 Months | Percentage of participants with a significant change in right ventricular global longitudinal strain (strain-RV) from baseline to 6 months. A significant change is defined as an 15% or more change (increase or decrease) from baseline to 6 months. The Exact 2-sided 95% CI is based on the observed proportion of participants. | Of the 27 participants who completed 6-month follow-up in the Placebo + SSA arm, 7 completed strain RV evaluations at both baseline and 6 months. In the Xermelo + SSA arm, 5 of the 26 participants who completed 6-month follow-up completed strain RV evaluations at both baseline and 6 months. | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline and 6 month follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Significant Change in Strain-LV From Baseline at 3 Months | Percentage of participants with a significant change in left ventricular global longitudinal strain (strain-LV) from baseline to 3 months. A significant change is defined as an 15% or more change (increase or decrease) from baseline to 3 months. The Exact 2-sided 95% CI is based on the observed proportion of participants. | Of the 29 participants who completed 3-month follow-up in the Placebo + SSA arm, 7 participants completed strain LV evaluations at both baseline and 3 months. In the Xermelo + SSA arm, 7 of the 29 participants who completed 3-month follow-up completed strain LV evaluations at both baseline and 3 months. | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline and 3 month follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Significant Change in Strain-LV From Baseline at 6 Months | Percentage of participants with a significant change in left ventricular global longitudinal strain (strain-LV) from baseline to 6 months. A significant change is defined as an 15% or more change (increase or decrease) from baseline to 6 months. The Exact 2-sided 95% CI is based on the observed proportion of participants. | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline and 6 month follow-up |
|
| ||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Normal TAPSE at 3 Months | Percentage of participants with a normal Tricuspid Annular Plane Systolic Excursion (TAPSE) at 3 months, where TAPSE ≥1.6cm is defined as normal. The Exact 2-sided 95% CI is based on the observed proportion of participants. | Among 29 who completed 3 months follow-up in the Placebo + SSA arm, 5 participants completed TAPSE evaluations at both baseline and at 3 months. In the Xermelo + SSA arm, 7 of the 29 participants who completed TAPSE evaluations at both baseline and at 3 months. | Posted | Number | 95% Confidence Interval | Percentage of Participants | 3 month follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Normal TAPSE at 6 Months | Percentage of participants with a normal Tricuspid Annular Plane Systolic Excursion (TAPSE) at 6 months, where TAPSE ≥1.6cm is defined as normal. The Exact 2-sided 95% CI is based on the observed proportion of participants. | Of the 27 participants who completed 6-month follow-up in the Placebo + SSA arm, 21 completed TAPSE evaluations at both baseline and 6 months. In Xermelo + SSA arm, 20 of the 26 who completed 6-month follow-up ccompleted TAPSE evaluation at both baseline and 6 months. | Posted | Number | 95% Confidence Interval | Percentage of Participants | 6 month follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in 5HIAA From Baseline to 3 Months | Change in plasma 5HIAA (5-Hydroxyindoleacetic acid) level from baseline to 3 months. The change is calculated as: 5HIAA at 3 months minus 5HIAA at baseline | Of the 29 participants who completed 3-month follow-up in the Placebo + SSA arm, 26 completed 5HIAA evaluations at both baseline and 3 months. In Xermelo + SSA arm, all 29 who completed 3-month follow-up completed 5HIAA evaluations at both baseline and 3 months. | Posted | Median | Inter-Quartile Range | ng/mL | Baseline and 3 month follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in 5HIAA From Baseline to 6 Months | Change in 5HIAA measurement from baseline to 6 months | Posted | Median | Inter-Quartile Range | ng/mL | Baseline and 6 month follow-up |
|
| ||||||||||||||||||||||||||||||
| Secondary | Change in Troponin From Baseline to 3 Months | Change in troponin level from baseline to 3 months, calculated as: troponine at 3 months minus troponin at baseline | Of the 29 participants who completed 3-month follow-up in the Placebo + SSA arm, 25 completed troponin evaluation at both baseline and 3 months. In the Xermelo + SSA arm, 25 of the 29 who completed 3-month completed troponin evaluation at both baseline and 3 months. | Posted | Median | Inter-Quartile Range | ng/L | Baseline and 3 months follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in Troponin From Baseline to 6 Months | Change in troponin level from baseline to 6 months, calculated as: troponine at 6 months minus troponin at baseline | Of the 27 participants who completed 6-month follow-up in the Placebo + SSA arm, 26 completed troponin evaluation at both baseline and 6 months. In the Xermelo + SSA arm, 25 of the 26 who completed 6-month follow-up completed troponin evaluation at both baseline and at 6 months. | Posted | Median | Inter-Quartile Range | ng/L | Baseline and 6 months follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in MDASI Mean Core Symptom Score From Baseline to 3 Months | Change in MD Anderson Symptom Inventory (MDASI) mean core symptom score from baseline to 3 months. The MDASI mean core symptom score is calculated as the mean of 13 core symptom items, each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine"), with the higher scores indicating worse symptoms. The change is computed as: score at 3 months minus score at baseline | Of the 29 participants who completed 3-month follow-up in the Placebo + SSA arm, 27 completed MDASI core symptom evaluation at both baseline and 3 months. In the Xermelo + SSA arm, 28 of the 29 participants who completed 3-month follow-up completed MDASI core symptom evaluation at both baseline and 3 months. | Posted | Median | Inter-Quartile Range | Scores on a scale | Baseline and 3 months follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in Core Symptom Score From Baseline to 6 Months | Change in MD Anderson Symptom Inventory (MDASI) mean core symptom score from baseline to 6 months. The MDASI mean core symptom score is calculated as the mean of 13 core symptom items, each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine"), with the higher scores indicating worse symptoms. The change is computed as: score at 6 months minus score at baseline | All 27 participants who completed 6-month follow-up in the Placebo + SSA arm completed MDASI core symptom evaluation at both baseline and 6 months. In the Xermelo + SSA arm, 25 of the 26 participants who completed 6-month follow-up completed MDASI core symptom evaluation at both baseline and 6 months. | Posted | Median | Inter-Quartile Range | Scores on a scale | Baseline and 6 months follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in MDASI Mean Total Symptom Score From Baseline to 3 Months | Change in MD Anderson Symptom Inventory (MDASI) mean total symptom score from baseline to 3 months. The MDASI mean total symptom score is calculated as the mean symptom severity of 21 symptom items (13 core symptom items and 8 additional symptom items), each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine), with the higher scores indicating worse symptoms. The change is computed as: score at 3 months minus score at baseline | Of the 29 participants who completed 3-month follow-up in the Placebo + SSA arm, 27 completed MDASI total symptom evaluation at both baseline and 3 months. In the Xermelo + SSA arm, all 28 participants who completed 3-month follow-up completed MDASI total symptom evaluation at both baseline and 3 months. | Posted | Median | Inter-Quartile Range | points | Baseline and 3 months follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in MDASI Mean Total Symptom Score From Baseline to 6 Months | Change in MD Anderson Symptom Inventory (MDASI) mean total symptom score from baseline to 6 months. The MDASI mean total symptom score is calculated as the mean symptom severity of 21 symptom items (13 core symptom items and 8 additional symptom items), each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine), with the higher scores indicating worse symptoms. The change is computed as: score at 6 months minus score at baseline | All 27 participants who completed 6-month follow-up in the Placebo + SSA arm completed MDASI total symptom evaluation at both baseline and 6 months. In the Xermelo + SSA arm, 25 of the 26 participants who completed 6-month follow-up completed MDASI total symptom evaluation at both baseline and 6 months. | Posted | Median | Inter-Quartile Range | Scores on a scale | Baseline and 6 months follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in MDASI Mean Interference Score From Baseline to 3 Months | Change in MD Anderson Symptom Inventory (MDASI) mean interference score from baseline to 3 months. The MDASI mean interference score is calculated as the mean of 6 interference items, each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine"), with the higher scores indicating worse interference. The change is computed as: score at 3 months minus score at baseline | Of the 29 participants who completed 3-month follow-up in the Placebo + SSA arm, 26 completed MDASI interference evaluation at both baseline and 3 months. In the Xermelo + SSA arm, 28 of the 29 participants who completed 3-month follow-up completed MDASI interference evaluation at both baseline and 3 months. | Posted | Median | Inter-Quartile Range | Scores on a scale | Baseline and 3 months follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Change in Interference Score From Baseline to 6 Months | Change in MD Anderson Symptom Inventory (MDASI) mean interference score from baseline to 6 months. The MDASI mean interference score is calculated as the mean of 6 interference items, each ranged from 0 ( "not present") to 10 being ("as bad as you can imagine"), with the higher scores indicating worse interference. The change is computed as: score at 6 months minus score at baseline | Of the 27 participants who completed 6-month follow-up in the Placebo + SSA arm, 26 completed MDASI interferemce evaluation at both baseline and 6 months. In the Xermelo + SSA arm, 24 of the 26 who completed 6-month follow-up completed MDASI interference evaluation at both baseline and 6 months. | Posted | Median | Inter-Quartile Range | Scores on a scale | Baseline and 6 months follow-up |
|
| |||||||||||||||||||||||||||||
| Secondary | Compliance>=70% While the Patients Were in the Study | Percentage of participants classified as compliant (compliance≥70%) during the study. Compliance is calculated as the total number of pills taken divided by the toal number of pills dispensed while the patient was on the study. Participants with compliance≥70% are considered compliant; otherwise, not compliant. | Those who were treated: 35 in placebo + SSA and 32 in Xermelo + SSA | Posted | Number | 95% Confidence Interval | Percentage of Participants | Baseline, 3 months follow-up, 6 months follow-up |
|
|
Adverse events were monitored from baseline throughout the follow-up period (3-month and 6-month follow-up visits) and for up to 30 days after the last dose of the study drug, with the treatment period planned for 6-months
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo + SSA | Placebo-matching telotristat ethyl tablet TID + stable-dose somatostatin analog (SSA) therapy | 1 | 35 | 3 | 35 | 26 | 35 |
| EG001 | Xermelo + SSA | Telotristat ethyl 500 mg po TID + stable-dose somatostatin analog (SSA) | 0 | 32 | 4 | 32 | 27 | 32 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ascites (death) | Gastrointestinal disorders | Systematic Assessment |
| ||
| Cataract | General disorders | Systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Ejection fraction decreased | Cardiac disorders | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| Fracture | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Presyncope | Nervous system disorders | Systematic Assessment |
| ||
| Syncope | Nervous system disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hyperglycemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Constipation | Gastrointestinal disorders | Systematic Assessment |
| ||
| Alkaline phosphtase increased | Investigations | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Anemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Creatinine increased | Renal and urinary disorders | Systematic Assessment |
| ||
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Heart failure | Cardiac disorders | Systematic Assessment |
| ||
| Hyperkalemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Cardiac troponin T increased | Investigations | Systematic Assessment |
| ||
| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
| ||
| Bloating | Gastrointestinal disorders | Systematic Assessment |
| ||
| Edema limbs | General disorders | Systematic Assessment |
| ||
| Anorexia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Chest pain - cardiac | Cardiac disorders | Systematic Assessment |
| ||
| Chronic kidney disease | Renal and urinary disorders | Systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Aspartate aminotransferase increased | Investigations | Systematic Assessment |
| ||
| Blood lactate dehydrogenase increased | Investigations | Systematic Assessment |
| ||
| Dizziness | Nervous system disorders | Systematic Assessment |
| ||
| Flushing | Vascular disorders | Systematic Assessment |
|
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Cezar A. Iliescu, MD | M.D. Anderson Cancer Center | (713) 792-4728 | ciliescu@mdanderson.org |
| Jan 15, 2026 |
| Prot_SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D018358 | Neuroendocrine Tumors |
| ID | Term |
|---|---|
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
Not provided
Not provided
| ID | Term |
|---|---|
| C000621725 | telotristat ethyl |
Not provided
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
Telotristat ethyl 500 mg po TID + stable-dose somatostatin analog (SSA)
|
|
| Xermelo + SSA |
Telotristat ethyl 500 mg po TID + stable-dose somatostatin analog (SSA) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Participants |
|
|
| Participants |
|
|
|
|
|
|
|