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

NCT01104415 | Completed Phase 2 Results FDA Drug

• 3 other identifiers: LX1606.1-203-CSLX1606.203, LX10322009-016973-13
• Study Type: interventional
• Target: 15
• Locations: 2 countries
• Sites: 10

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.

Conditions

Carcinoid Syndrome

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Core Phase

  An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. Treatment-emergent AEs were defined as any AEs reported after the first dose of treatment on Day 1.

  Baseline up to Week 12 in the Core Phase

• Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Extension Period

  An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. Treatment-emergent AEs were defined as any AEs reported after the first dose of treatment on Day 1.

  Up to 124 Weeks in the Extension Period

Secondary Outcomes (9)

• Change From Baseline in Number of Bowel Movements (BMs)

  Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24

• Change From Baseline in Stool Form/Consistency

  Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24

• Change From Baseline in Percentage of Days With Sensation of Urgency to Defecate

  Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24

• Change From Baseline in Sensation/Severity of Nausea Using 100 mm Visual Analog Scale (VAS)

  Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24

• Number of Participants With an Improvement in Global Assessment of Symptoms Associated With Carcinoid Syndrome

  Core Phase: Weeks 9-12; Extension Period: Week 24

Study Arms (2)

Telotristat etiprate - Core Phase

EXPERIMENTAL

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.

Drug: Telotristat etiprate

Telotristat etiprate - Extension Period

EXPERIMENTAL

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.

Drug: Telotristat etiprate

Interventions

Telotristat etiprate DRUG

Telotristat etiprate capsules orally three times daily.

Also known as: LX1606 Hippurate

Telotristat etiprate - Core Phase; Telotristat etiprate - Extension Period

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• ≥ 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

Sponsors & Collaborators

• Lexicon Pharmaceuticals: lead

Study Sites (10)

Lexicon Investigational Site

Bad Berka, Germany

Location

Lexicon Investigational Site

Berlin, Germany

Location

Lexicon Investigational Site

Halle, Germany

Location

Lexicon Investigational Site

Lübeck, Germany

Location

Lexicon Investigational Site

Marburg, Germany

Location

Lexicon Investigational Site

Munich, Germany

Location

Lexicon Investigational Site

Basingstoke, United Kingdom

Location

Lexicon Investigational Site

Cambridge, United Kingdom

Location

Lexicon Investigational Site

London, United Kingdom

Location

Lexicon Investigational Site

Manchester, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 25636046 DERIVED

MeSH Terms

Conditions

Serotonin Syndrome

Interventions

telotristat

Condition Hierarchy (Ancestors)

Drug-Related Side Effects and Adverse Reactions; Chemically-Induced Disorders

Results Point of Contact

• Title: Pablo Lapuerta, MD
• Organization: Lexicon Pharmaceuticals, Inc.

plapuerta@lexpharma.com

Study Officials

• Pablo LaPuerta, MD

  Lexicon Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 12, 2010
• First Posted: April 15, 2010
• Study Start: June 15, 2010
• Primary Completion: February 12, 2014
• Study Completion: February 12, 2014
• Last Updated: March 15, 2019
• Results First Posted: April 6, 2018

Record last verified: 2019-02

Locations

DE (6); GB (4)