Telotristat Ethyl, a Tryptophan Hydroxylase Inhibitor for the Treatment of Carcinoid Syndrome

J Clin Oncol. 2017 Jan;35(1):14-23. doi: 10.1200/JCO.2016.69.2780. Epub 2016 Oct 28.

Abstract

Purpose Preliminary studies suggested that telotristat ethyl, a tryptophan hydroxylase inhibitor, reduces bowel movement (BM) frequency in patients with carcinoid syndrome. This placebo-controlled phase III study evaluated telotristat ethyl in this setting. Patients and Methods Patients (N = 135) experiencing four or more BMs per day despite stable-dose somatostatin analog therapy received (1:1:1) placebo, telotristat ethyl 250 mg, or telotristat ethyl 500 mg three times per day orally during a 12-week double-blind treatment period. The primary end point was change from baseline in BM frequency. In an open-label extension, 115 patients subsequently received telotristat ethyl 500 mg. Results Estimated differences in BM frequency per day versus placebo averaged over 12 weeks were -0.81 for telotristat ethyl 250 mg ( P < .001) and ‒0.69 for telotristat ethyl 500 mg ( P < .001). At week 12, mean BM frequency reductions per day for placebo, telotristat ethyl 250 mg, and telotristat ethyl 500 mg were -0.9, -1.7, and -2.1, respectively. Responses, predefined as a BM frequency reduction ≥ 30% from baseline for ≥ 50% of the double-blind treatment period, were observed in 20%, 44%, and 42% of patients given placebo, telotristat ethyl 250 mg, and telotristat ethyl 500 mg, respectively. Both telotristat ethyl dosages significantly reduced mean urinary 5-hydroxyindole acetic acid versus placebo at week 12 ( P < .001). Mild nausea and asymptomatic increases in gamma-glutamyl transferase were observed in some patients receiving telotristat ethyl. Follow-up of patients during the open-label extension revealed no new safety signals and suggested sustained BM responses to treatment. Conclusion Among patients with carcinoid syndrome not adequately controlled by somatostatin analogs, treatment with telotristat ethyl was generally safe and well tolerated and resulted in significant reductions in BM frequency and urinary 5-hydroxyindole acetic acid.

Publication types

  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Aged
  • Antineoplastic Agents, Hormonal / therapeutic use
  • Defecation / drug effects*
  • Diarrhea / drug therapy*
  • Diarrhea / etiology
  • Diarrhea / urine
  • Double-Blind Method
  • Female
  • Gastrointestinal Agents / therapeutic use*
  • Humans
  • Hydroxyindoleacetic Acid / urine
  • Male
  • Malignant Carcinoid Syndrome / complications
  • Malignant Carcinoid Syndrome / drug therapy*
  • Malignant Carcinoid Syndrome / urine
  • Middle Aged
  • Nausea / chemically induced
  • Octreotide / therapeutic use
  • Peptides, Cyclic / therapeutic use
  • Phenylalanine / adverse effects
  • Phenylalanine / analogs & derivatives*
  • Phenylalanine / therapeutic use
  • Pyrimidines / adverse effects
  • Pyrimidines / therapeutic use*
  • Somatostatin / analogs & derivatives
  • Somatostatin / therapeutic use
  • Tryptophan Hydroxylase / antagonists & inhibitors
  • gamma-Glutamyltransferase / blood

Substances

  • Antineoplastic Agents, Hormonal
  • Gastrointestinal Agents
  • Peptides, Cyclic
  • Pyrimidines
  • lanreotide
  • Phenylalanine
  • Somatostatin
  • Hydroxyindoleacetic Acid
  • telotristat ethyl
  • Tryptophan Hydroxylase
  • gamma-Glutamyltransferase
  • Octreotide