Study of Telotristat Etiprate (LX1606) in Participants With Symptomatic Carcinoid Syndrome Not Managed by Stable-Dose Octreotide Therapy

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Ascending, Multidose Study To Determine Safety and Tolerability of Orally Administered LX1606 in Subjects With Symptomatic Carcinoid Syndrome Refractory to Stable-Dose Octreotide Long-Acting Release Depot Therapy

The purpose of this study is to evaluate the safety and tolerability of telotristat etiprate (LX1606) versus a placebo control in participants with symptomatic carcinoid syndrome not managed by stable-dose long-acting octreotide therapy. Following determination of the maximally tolerated or effective dose, cohort expansion will occur to confirm effect on symptoms and safety profile.

Inclusion Criteria:

• Males and females, aged 18 and older
• Biopsy-proven metastatic carcinoid tumor of the gastrointestinal (GI) tract with disease extent confirmed by computed tomography (CT), magnetic resonance imaging (MRI), or radionuclide imaging
• Symptoms not managed by stable-dose long-acting octreotide therapy (≥4 bowel movements per day)
• Ability to provide written informed consent

Exclusion Criteria:

• ≥12 high volume, watery bowel movements per day associated with a clinical syndrome of volume contraction, dehydration, or hypotension compatible with a "pancreatic cholera"-type clinical syndrome
• Sponsor-unacceptable clinical laboratory values for hematology and liver function tests at screening
• Karnofsky status ≤70% - unable to care for self
• Surgery within 60 days prior to screening
• A history of short bowel syndrome
• Life expectancy <12 months
• History of substance or alcohol abuse within 2 years prior to screening
• Previous exposure to a tryptophan hydroxylase (TPH) inhibitor
• Administration of any investigational drug within 30 days of screening or any therapeutic protein or antibody within 90 days of screening