Phase 2 N=23 Randomized Triple-blind Treatment

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

Carcinoid Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT00853047 ↗

Enrolled (actual)

Serious AEs

21.4%

Results posted

Dec 2018

Primary outcome: Primary: Number of Participants With Any Treatment-emergent Adverse Event (TEAE) and Any Drug-related TEAE in the Core Phase — 4; 3; 3; 2 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Telotristat etiprate (Drug); Octreotide LAR Depot (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Lexicon Pharmaceuticals
Primary completion: Jun 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Any Treatment-emergent Adverse Event (TEAE) and Any Drug-related TEAE in the Core Phase	4; 3; 3; 2; 9; 3	—
PRIMARY Number of Participants With Any TEAE in the Open-Label Extension Phase	18	—
SECONDARY Change From Baseline in Mean Number of Bowel Movements (BMs) Per Day	0.82; -1.37; -2.17; -0.20; -0.71	—
SECONDARY Change From Baseline in Weekly Mean Stool Form	-0.07; -0.50; 0.00; 0.00; -0.17	—
SECONDARY Change From Baseline in Percentage of Days Per Week Experiencing a Sensation of Urgency to Defecate	-2.72; -34.43; -32.13; 0.00; -8.49	—
SECONDARY Change From Baseline in Number of Cutaneous Flushing Episodes	-0.43; -0.60; -0.30; -0.10; -0.03	—
SECONDARY Change From Baseline in Severity of Abdominal Pain or Discomfort	0.04; 0.03; -0.53; 0.03; 0.16	—
SECONDARY Change From Baseline in Urinary 5-hydroxyindoleacetic Acid (u5-HIAA)	-20.73; -27.55; -0.67; 13.60; -35.49	—
SECONDARY Change From Baseline in Chromogranin A	-3251.2; -190.5; -12.3; 52.5; 26011.4	—
SECONDARY Number of Participants Reporting Improvement in the Subjective Global Assessment of Symptoms Associated With Carcinoid Syndrome	0; 1; 2; 1; 3	—
SECONDARY Change From Baseline in Frequency of Rescue for Short-acting Octreotide Use/Day	-0.38; -0.03; 0.03; 0.00; -0.29	—
SECONDARY Time to First Rescue, Short-acting Octreotide	1.00; 1.00	—
SECONDARY Number of Participants Experiencing Complete Response at Week 4	0; 1; 2; 1; 2	—

Summary

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.

Eligibility Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00853047). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.