Lowell Anthony1, Claire Ervin2, Pablo Lapuerta3, Matthew H Kulke4, Pamela Kunz5, Emily Bergsland6, Dieter Hörsch7, David C Metz8, Janice Pasieka9, Nick Pavlakis10, Marianne Pavel11, Martyn Caplin12, Kjell Öberg13, John Ramage14, Emily Evans2, Qi Melissa Yang15, Shanna Jackson15, Karie Arnold15, Linda Law15, Dana B DiBenedetti2. 1. Markey Cancer Center, University of Kentucky, Lexington, Kentucky. 2. RTI Health Solutions, Research Triangle Park, North Carolina. 3. Lexicon Pharmaceuticals Inc, The Woodlands, Texas. Electronic address: plapuerta@lexpharma.com. 4. Dana-Farber Cancer Institute, Boston, Massachusetts. 5. Stanford Cancer Center, Standford, California. 6. UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, California. 7. Zentralklinik Bad Berka GmbH, Klinik fur Innerre, Medizin/Gastroenterologie und Endokrinologie, Bad Berka, Germany. 8. University of Pennsylvania, Perelman School of Medicine, Philadelphia, Pennsylvania. 9. Tom Baker Cancer Centre, Calgary, Alberta, Canada. 10. Royal North Shore Hospital, New South Wales, Australia. 11. Charité Universitätsmedizin, Berlin, Germany. 12. Royal Free Hospital, London, United Kingdom. 13. Uppsala University, Uppsala, Sweden. 14. Hampshire Hospitals NHS Trust, Basingstoke and North Hampshire Hospital, Basingstoke-Hampshire, United Kingdom. 15. Lexicon Pharmaceuticals Inc, The Woodlands, Texas.
Abstract
PURPOSE: Telotristat ethyl, an oral tryptophan hydroxylase inhibitor, is intended to treat carcinoid syndrome by reducing serotonin production. Telotristat ethyl was evaluated in TELESTAR, a Phase III study for patients who had carcinoid syndrome with at least 4 bowel movements (BMs) per day and who were receivingsomatostatin analogue therapy. This interview substudy was conducted to provide insight into the patient experience in TELESTAR and to help understand whether reductions in BM frequency (the primary end point) and other symptoms were clinically meaningful. METHODS: Participating sites were asked to invite (before randomization) all eligible patients to telephone interviews scheduled at the end of the double-blind treatment period. Patients and interviewers were blinded to treatment. FINDINGS: All 35 interviewed participants reported diarrhea and/or excessive BMs at baseline. Patients reported that these symptoms negatively affected emotional, social, physical, and occupational well-being. Prespecified criteria for treatment response (achieving ≥30% reduction in BM frequency for at least 50% of the days) were met by 8 of 26 patients taking telotristat ethyl and 1 of 9 patients taking placebo. All 8 patients taking telotristat ethyl described clinically meaningful reductions in BM frequency and were very satisfied with the ability of the study drug to control their carcinoid syndrome symptoms. Overall, reports of being very satisfied were observed in 12 patients taking telotristat ethyl and 0 taking placebo. IMPLICATIONS: Patient interviews revealed that TELESTAR patients, at baseline, were significantly affected by their high BM frequency. Patient reports of their clinical trial experience supported the significance of the primary end point and clinical responder analysis in TELESTAR, helping identify and understand clinically meaningful change produced by telotristat ethyl.
RCT Entities:
PURPOSE: Telotristat ethyl, an oral tryptophan hydroxylase inhibitor, is intended to treat carcinoid syndrome by reducing serotonin production. Telotristat ethyl was evaluated in TELESTAR, a Phase III study for patients who had carcinoid syndrome with at least 4 bowel movements (BMs) per day and who were receiving somatostatin analogue therapy. This interview substudy was conducted to provide insight into the patient experience in TELESTAR and to help understand whether reductions in BM frequency (the primary end point) and other symptoms were clinically meaningful. METHODS: Participating sites were asked to invite (before randomization) all eligible patients to telephone interviews scheduled at the end of the double-blind treatment period. Patients and interviewers were blinded to treatment. FINDINGS: All 35 interviewed participants reported diarrhea and/or excessive BMs at baseline. Patients reported that these symptoms negatively affected emotional, social, physical, and occupational well-being. Prespecified criteria for treatment response (achieving ≥30% reduction in BM frequency for at least 50% of the days) were met by 8 of 26 patients taking telotristat ethyl and 1 of 9 patients taking placebo. All 8 patients taking telotristat ethyl described clinically meaningful reductions in BM frequency and were very satisfied with the ability of the study drug to control their carcinoid syndrome symptoms. Overall, reports of being very satisfied were observed in 12 patients taking telotristat ethyl and 0 taking placebo. IMPLICATIONS: Patient interviews revealed that TELESTAR patients, at baseline, were significantly affected by their high BM frequency. Patient reports of their clinical trial experience supported the significance of the primary end point and clinical responder analysis in TELESTAR, helping identify and understand clinically meaningful change produced by telotristat ethyl.
Authors: Lowell B Anthony; Matthew H Kulke; Martyn E Caplin; Emily Bergsland; Kjell Öberg; Marianne Pavel; Dieter Hörsch; Richard R P Warner; Thomas M O'Dorisio; Joseph S Dillon; Pablo Lapuerta; Kenneth Kassler-Taub; Wenjun Jiang Journal: Oncologist Date: 2019-01-16
Authors: Jonathan Strosberg; Vijay N Joish; Susan Giacalone; Raul Perez-Olle; Ann Fish-Steagall; Kanika Kapoor; Sam Dharba; Pablo Lapuerta; Al B Benson Journal: Oncologist Date: 2019-06-12
Authors: Cristina Saavedra; Jorge Barriuso; Mairéad G McNamara; Juan W Valle; Angela Lamarca Journal: Cancer Manag Res Date: 2019-08-08 Impact factor: 3.989