Telotristat ethyl in carcinoid syndrome: safety and efficacy in the TELECAST phase 3 trial ENDOCRINE-RELATED CANCER Pavel, M., Gross, D. J., Benavent, M., Perros, P., Srirajaskanthan, R., Warner, R. P., Kulke, M. H., Anthony, L. B., Kunz, P. L., Hoersch, D., Weickert, M. O., Lapuerta, P., Jiang, W., Kassler-Taub, K., Wason, S., Fleming, R., Fleming, D., Garcia-Carbonero, R. 2018; 25 (3): 309–22


Telotristat ethyl, a tryptophan hydroxylase inhibitor, was efficacious and well tolerated in the phase 3 TELESTAR study in patients with carcinoid syndrome (CS) experiencing =4 bowel movements per day (BMs/day) while on somatostatin analogs (SSAs). TELECAST, a phase 3 companion study, assessed the safety and efficacy of telotristat ethyl in patients with CS (diarrhea, flushing, abdominal pain, nausea or elevated urinary 5-hydroxyindoleacetic acid (u5-HIAA)) with <4?BMs/day on SSAs (or =1 symptom or =4?BMs/day if not on SSAs) during a 12-week double-blind treatment period followed by a 36-week open-label extension (OLE). The primary safety and efficacy endpoints were incidence of treatment-emergent adverse events (TEAEs) and percent change from baseline in 24-h u5-HIAA at week 12. Patients (N?=?76) were randomly assigned (1:1:1) to receive placebo or telotristat ethyl 250?mg or 500?mg 3 times per day (tid); 67 continued receiving telotristat ethyl 500?mg tid during the OLE. Through week 12, TEAEs were generally mild to moderate in severity; 5 (placebo), 1 (telotristat ethyl 250?mg) and 3 (telotristat ethyl 500?mg) patients experienced serious events, and the rate of TEAEs in the OLE was comparable. At week 12, significant reductions in u5-HIAA from baseline were observed, with Hodges-Lehmann estimators of median treatment differences from placebo of -54.0% (95% confidence limits, -85.0%, -25.1%, P?

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