Study of Telotristat Etiprate (LX1606) in Participants With Symptomatic Carcinoid Syndrome

Lexicon Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Carcinoid Syndrome

Treatments: Drug: Telotristat etiprate

Study type: Interventional

Funder types: Industry

Identifiers

NCT01104415

LX1606.1-203-CS

LX1606.203, LX1032 (Other Identifier)

2009-016973-13 (EudraCT Number)

Details and patient eligibility

About

The purpose of the study is to evaluate the safety and tolerability of orally administered telotristat etiprate (LX1606) in participants with symptomatic carcinoid syndrome.

Enrollment: 15 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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
• Symptomatic carcinoid syndrome (≥4 bowel movements per day)
• Ability to provide written informed consent

Exclusion criteria

• ≥ 12 high-volume, watery bowel movements per day
• 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
• Administration of any investigational drug within 30 days of screening or any therapeutic protein or antibody within 90 days of screening

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 2 patient groups

Telotristat etiprate - Core Phase

Experimental group

Description:

Following a 2-week Run-In Period, participants received telotristat etiprate capsules at a starting dose of 150 mg, orally three times daily (TID) for 14 days in the Core Phase. Dose escalations (250 mg, 350 mg, 500 mg) occurred serially every 14 days, up to a maximum dosage of telotristat etiprate 500 mg TID, as guided by specific clinical criteria for dose escalation. Upon completion of 12 weeks of treatment, participants were eligible to receive telotristat etiprate in the optional Open-label Extension Period.

Treatment:

Drug: Telotristat etiprate

Telotristat etiprate - Extension Period

Experimental group

Description:

Participants received telotristat etiprate at their highest tolerated dose (250 mg or 500 mg), orally, TID for 124 weeks in the Open-label Extension Period. If neither dose was tolerated participants were discontinued from the study and completed the 2-week Follow-up Visit.

Treatment:

Drug: Telotristat etiprate