Trial · NCT04810091

Telotristat Ethyl for the Treatment of Carcinoid Heart Disease in Patients With Metastatic Neuroendocrine Tumor

NCT04810091, Phase 3, Active not recruiting, M.D. Anderson Cancer Center; results first posted 2026-02-05

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Telotristat Ethyl for the Treatment of Carcinoid Heart Disease in Patients With Metastatic Neuroendocrine Tumor. Trials, record NCT04810091. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04810091

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Telotristat Ethyl for the Treatment of Carcinoid Heart Disease in Patients With Metastatic Neuroendocrine Tumor
NCT number: NCT04810091
Status: Active not recruiting
Phases: Phase 3
Enrollment: 79 participants, actual
Lead sponsor: M.D. Anderson Cancer Center
Conditions: Locally Advanced Neuroendocrine Neoplasm, Metastatic Neuroendocrine Neoplasm
Interventions: Placebo Administration, Questionnaire Administration, Telotristat Ethyl
First posted: 2021-03-22
Last updated: 2026-10-15
Results first posted: 2026-02-05

Source ClinicalTrials.gov

Registered outcomes22

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Percent Change in NTproBNP at 6 Months Visit From Baseline	Baseline and at 6 months	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
Secondary	Change in 6MWT at 3 Month Visit	Baseline and 3 month follow-up	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
Secondary	Change in 6MWT at 6 Month Visit	Baseline and 6 month follow-up	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
Secondary	Change in CVHD % Score From Baseline to 3 Month Visit	Baseline and 3 month follow-up	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.
Secondary	Change in CVHD Score From Baseline to 6 Month Visit	Baseline and 6 month follow-up	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.
Secondary	Percentage of Participants With Significant Change in Strain-RV From Baseline at 3 Months	Baseline and 3 month follow-up	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.
Secondary	Percentage of Participants With Significant Change in Strain-RV From Baseline at 6 Months	Baseline and 6 month follow-up	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.
Secondary	Percentage of Participants With Significant Change in Strain-LV From Baseline at 3 Months	Baseline and 3 month follow-up	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.
Secondary	Percentage of Participants With Significant Change in Strain-LV From Baseline at 6 Months	Baseline and 6 month follow-up	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.
Secondary	Percentage of Participants With Normal TAPSE at 3 Months	3 month follow-up	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.

1 to 10 of 22Source ClinicalTrials.gov

See all 22 registered outcomes

Outcomes22

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percent Change in NTproBNP at 6 Months Visit From Baseline	Baseline and at 6 months	Patients who had NTproBNP at both baseline and 6 months	Percent change from baseline	MEDIAN
SECONDARY	Change in 6MWT at 3 Month Visit	Baseline and 3 month follow-up	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.	meters	MEDIAN
SECONDARY	Change in 6MWT at 6 Month Visit	Baseline and 6 month follow-up	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.	meters	MEDIAN
SECONDARY	Change in CVHD % Score From Baseline to 3 Month Visit	Baseline and 3 month follow-up	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.	Scores on a scale	MEDIAN
SECONDARY	Change in CVHD Score From Baseline to 6 Month Visit	Baseline and 6 month follow-up	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.	Scores on a scale	MEDIAN
SECONDARY	Percentage of Participants With Significant Change in Strain-RV From Baseline at 3 Months	Baseline and 3 month follow-up	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.	Percentage of Participants	NUMBER
SECONDARY	Percentage of Participants With Significant Change in Strain-RV From Baseline at 6 Months	Baseline and 6 month follow-up	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.	Percentage of Participants	NUMBER
SECONDARY	Percentage of Participants With Significant Change in Strain-LV From Baseline at 3 Months	Baseline and 3 month follow-up	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.	Percentage of Participants	NUMBER
SECONDARY	Percentage of Participants With Significant Change in Strain-LV From Baseline at 6 Months	Baseline and 6 month follow-up		Percentage of Participants	NUMBER
SECONDARY	Percentage of Participants With Normal TAPSE at 3 Months	3 month follow-up	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.	Percentage of Participants	NUMBER

1 to 10 of 22Source ClinicalTrials.gov

See all 22 outcomes

Results by group44

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Percent Change in NTproBNP at 6 Months Visit From Baseline	Placebo + SSA	-9.1	Inter-Quartile Range -32.4 to 14.3	Percent change from baseline
Percent Change in NTproBNP at 6 Months Visit From Baseline	Xermelo + SSA	1.4	Inter-Quartile Range -32.4 to 53.8	Percent change from baseline
Change in 6MWT at 3 Month Visit	Placebo + SSA	0	Inter-Quartile Range -35 to 45	meters
Change in 6MWT at 3 Month Visit	Xermelo + SSA	8	Inter-Quartile Range -25 to 64	meters
Change in 6MWT at 6 Month Visit	Placebo + SSA	-14	Inter-Quartile Range -63 to 39	meters
Change in 6MWT at 6 Month Visit	Xermelo + SSA	25.5	Inter-Quartile Range -27 to 67	meters
Change in CVHD % Score From Baseline to 3 Month Visit	Placebo + SSA	0	Inter-Quartile Range -7 to 0	Scores on a scale
Change in CVHD % Score From Baseline to 3 Month Visit	Xermelo + SSA	0	Inter-Quartile Range 0 to 0	Scores on a scale
Change in CVHD Score From Baseline to 6 Month Visit	Placebo + SSA	0	Inter-Quartile Range 0 to 0	Scores on a scale
Change in CVHD Score From Baseline to 6 Month Visit	Xermelo + SSA	0	Inter-Quartile Range 0 to 0	Scores on a scale

1 to 10 of 44Source ClinicalTrials.gov

See all 44 results by group

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Placebo + SSA	37
Overall Study	STARTED	Xermelo + SSA	36
Overall Study	Randomized	Placebo + SSA	37
Overall Study	Randomized	Xermelo + SSA	36
Overall Study	Treated	Placebo + SSA	35
Overall Study	Treated	Xermelo + SSA	32
Overall Study	3 Month Follow-up	Placebo + SSA	29
Overall Study	3 Month Follow-up	Xermelo + SSA	29
Overall Study	COMPLETED	Placebo + SSA	27
Overall Study	COMPLETED	Xermelo + SSA	26

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events64

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Hyperglycemia	Metabolism and nutrition disorders	Placebo + SSA	11	35	13
other	Hyperglycemia	Metabolism and nutrition disorders	Xermelo + SSA	20	32	21
other	Nausea	Gastrointestinal disorders	Placebo + SSA	6	35	11
other	Nausea	Gastrointestinal disorders	Xermelo + SSA	7	32	8
other	Constipation	Gastrointestinal disorders	Placebo + SSA	5	35	6
other	Constipation	Gastrointestinal disorders	Xermelo + SSA	8	32	8
other	Alkaline phosphtase increased	Investigations	Placebo + SSA	3	35	4
other	Alkaline phosphtase increased	Investigations	Xermelo + SSA	8	32	8
other	Fatigue	General disorders	Placebo + SSA	4	35	6
other	Fatigue	General disorders	Xermelo + SSA	5	32	5

1 to 10 of 64Source ClinicalTrials.gov

See all 64 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo + SSA	1	35
Total, all-cause mortality	Xermelo + SSA	0	32
Total, other adverse events	Placebo + SSA	26	35
Total, other adverse events	Xermelo + SSA	27	32
Total, serious adverse events	Placebo + SSA	3	35
Total, serious adverse events	Xermelo + SSA	4	32

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants1

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
P30CA016672	The University of Texas MD Anderson Cancer Center Support Grant (CCSG)	KOONG, ALBERT	UNIVERSITY OF TX MD ANDERSON CAN CTR	NATIONAL CANCER INSTITUTE	1985 to 2026

Source NIH RePORTER

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Placebo + SSA vs Xermelo + SSA: deaths from any cause	Trial comparisons
Placebo + SSA vs Xermelo + SSA: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT04810091

GET https://conductscience.org/api/trials/v1/NCT04810091/registered?page=2

API reference For AI assistants