Telotristat Etiprate - Expanded Treatment for Patients With Carcinoid Syndrome Symptoms

An adverse event (AE) is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE includes any noxious, pathological, or unintended change in anatomical, physiological, or metabolic functions as indicated by physical signs or symptoms occurring in any phase of the clinical study whether or not considered related to the study medication. A TEAE is an AE that occurs or worsens after receiving study drug.

Time frame: First dose of study drug (Day 1) up to 15 days post last dose (approximately up to 236 weeks)

Population: Safety Population included all participants who received any fraction of a dose of telotristat etiprate during the study.

QLQ-C30 is a standardized 30-item scale to assess health-related quality of life composed of 5 functional scales (physical functioning \[5 items\], role functioning \[2 items\], emotional functioning \[4 items\], cognitive functioning \[2 items\], and social functioning \[2 items\]); 3 symptom scales (fatigue \[3 items\], nausea/vomiting \[2 items\], and pain \[2 items\]); a global health status (GHS) /quality of life (QOL) scale \[2 items\]; 6 single items (dyspnoea, insomnia, appetite loss, constipation, diarrhoea, and financial difficulties). 28 questions answered:1 (not at all) to 4 (very much) and 2 questions on overall health/QOL answered:1 (poor) to 7 (excellent). All of the scales and single-item measures are transformed to a score:0 to 100. For functioning scales and global QOL higher scores indicate better functioning (a positive change from Baseline indicates improvement); for symptom scales higher scores indicate more severe symptoms (a negative change from Baseline indicates improvement).

Time frame: Baseline, Weeks 24, 48, 72 and 84

Population: Per-Protocol (PP) population=participants who received telotristat etiprate;had no major protocol deviations that interfered with collection/interpretation of efficacy data. Number analyzed=participants with data at given time-point.Participants were combined for this outcome measure as they received dose adjustments per investigator's discretion.

GI.NET21 is a standardized 21-item scale composed of both multi-item scales and single-item measures that include 5 functional scales (gastrointestinal (GI) \[5 items\], endocrine \[3 items\], treatment-related \[3 items\], social functioning \[3 items\], and disease-related worries scale \[DRWS\] \[3 items\]) and 4 single items (muscle and bone pain symptom (BPS), sexual functioning, communication function (CF), body image and information about the disease). Each item is scored from 1 (not at all) to 4 (very much). All of the scales and single-item measures are transformed to a score of 0 to 100. For functioning scales higher scores indicate better functioning (a positive change from Baseline indicates improvement); for symptom scales higher scores indicate more severe symptoms (a negative change from Baseline indicates improvement).

Time frame: Baseline, Weeks 24, 48, 72 and 84

Population: PP population included the participants who received telotristat etiprate; had no major protocol deviations that interfered with collection/interpretation of efficacy data. Number analyzed=participants with data at given time-point. Participants were combined for this outcome measure as they received dose adjustments per investigator's discretion.

Participants were asked the following question to assess global symptoms associated with carcinoid syndrome (CS) on an 11-point scale: Rate the severity of your overall carcinoid symptoms over the past 7 days on a scale from 0 to 10, where 0=no symptoms and 10=worst symptoms ever experienced. A negative change from baseline indicated improvement.

Time frame: Baseline, Weeks 12, 24, 36, 48, 60, 72 and 84

Population: PP population included the participants who received telotristat etiprate; had no major protocol deviations that interfered with collection/interpretation of efficacy data. Number analyzed=participants with data at given time-point. Participants were combined for this outcome measure as they received dose adjustments per investigator's discretion.

Participants were asked to respond to the following question: In the past 7 days, have you had adequate relief of your carcinoid syndrome bowel complaints such as diarrhea, urgent need to have a bowel movement, abdominal pain, or discomfort? The percentage of participants reporting adequate relief (answered Yes) were reported.

Time frame: Baseline, Weeks 12, 24, 36, 48, 60, 72 and 84

Population: PP population included the participants who received telotristat etiprate; had no major protocol deviations that interfered with collection/interpretation of efficacy data. Number analyzed=participants with data at given time-point. Participants were combined for this outcome measure as they received dose adjustments per investigator's discretion.