Clinical trial NCT01104415: Study of Telotristat Etiprate (LX1606) in Participants With Symptomatic Carcinoid Syndrome
Official title: A Phase 2, Open-Label, Multi-Center, Serial Ascending-Dose, Dose-Finding Study to Evaluate the Safety and Tolerability of LX1606 in Subjects With Symptomatic Carcinoid Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Carcinoid Syndrome
Lead sponsor: Lexicon Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2010-04-15
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


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
* 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

Primary outcomes:
measure: Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Core Phase
timeFrame: Baseline up to Week 12 in the Core Phase
measure: Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Extension Period
timeFrame: Up to 124 Weeks in the Extension Period
Secondary outcomes:
measure: Change From Baseline in Number of Bowel Movements (BMs)
timeFrame: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
measure: Change From Baseline in Stool Form/Consistency
timeFrame: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
measure: Change From Baseline in Percentage of Days With Sensation of Urgency to Defecate
timeFrame: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
measure: Change From Baseline in Sensation/Severity of Nausea Using 100 mm Visual Analog Scale (VAS)
timeFrame: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
measure: Number of Participants With an Improvement in Global Assessment of Symptoms Associated With Carcinoid Syndrome
timeFrame: Core Phase: Weeks 9-12; Extension Period: Week 24
measure: Change From Baseline in Daily Severity of Abdominal Pain or Discomfort Using 100 mm Visual Analog Scale (VAS)
timeFrame: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
measure: Change From Baseline in Daily Number of Cutaneous Flushing Episodes
timeFrame: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
measure: Number of Participants Achieving Clinically Meaningful Symptom Reduction in the Core Phase
timeFrame: Baseline to Week 12
measure: Change From Baseline in Urinary 5-Hydroxyindoleacetic Acid (HIAA) Levels
timeFrame: Core Phase: Baseline to Week 12; Extension Period: Baseline to Weeks 20-21
Other outcomes:
Locations:
city: Bad Berka
country: Germany
facility: Lexicon Investigational Site
city: Berlin
country: Germany
facility: Lexicon Investigational Site
city: Halle
country: Germany
facility: Lexicon Investigational Site
city: Lubeck
country: Germany
facility: Lexicon Investigational Site
city: Marburg
country: Germany
facility: Lexicon Investigational Site
city: Munich
country: Germany
facility: Lexicon Investigational Site
city: Basingstoke
country: United Kingdom
facility: Lexicon Investigational Site
city: Cambridge
country: United Kingdom
facility: Lexicon Investigational Site
city: London
country: United Kingdom
facility: Lexicon Investigational Site
city: Manchester
country: United Kingdom
facility: Lexicon Investigational Site
Study officials:
affiliation: Lexicon Pharmaceuticals, Inc.
name: Pablo LaPuerta, MD
role: STUDY_DIRECTOR
References:
citation: Pavel M, Horsch D, Caplin M, Ramage J, Seufferlein T, Valle J, Banks P, Lapuerta P, Sands A, Zambrowicz B, Fleming D, Wiedenmann B. Telotristat etiprate for carcinoid syndrome: a single-arm, multicenter trial. J Clin Endocrinol Metab. 2015 Apr;100(4):1511-9. doi: 10.1210/jc.2014-2247. Epub 2015 Jan 30.