Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Study of LX1606 in Subjects With Symptomatic Carcinoid Syndrome

This study has been completed.
Information provided by (Responsible Party):
Lexicon Pharmaceuticals Identifier:
First received: April 12, 2010
Last updated: February 20, 2014
Last verified: February 2014

The purpose of the study is to evaluate the safety and tolerability of multi-dose, open-label LX606 in subjects with symptomatic carcinoid syndrome.

Condition Intervention Phase
Symptoms of Carcinoid Syndrome
Drug: Low Dose LX1606 - Day 1 (start)
Drug: Mid-low dose LX1606 - Day 15 (start)
Drug: Mid-high dose LX1606 - Day 29 (start)
Drug: High dose LX1606 - Day 43 (start)
Drug: 4-Week Open Label Dose Extension
Drug: 72-Week Open Label Dose Extension
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Lexicon Pharmaceuticals:

Primary Outcome Measures:
  • Safety (physical examinations, clinical laboratory tests, vital signs measurements, and ECGs) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Hematology/blood chemistry - weekly, physical examinations - biweekly, vital signs - biweekly, urinalysis - monthly, ECG - monthly

Secondary Outcome Measures:
  • Number of bowel movements [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Flushing episodes [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Subjective global assessment [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Urgency to defecate [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Stool form/consistency [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Blood 5-HT levels [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Blood Chromagranin-A levels [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Urinary 5-HIAA levels [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Sensation/severity of nausea [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Abdominal pain and discomfort [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Enrollment: 15
Study Start Date: May 2010
Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LX1606 Open Label Dose Escalation Drug: Low Dose LX1606 - Day 1 (start)
150 mg LX1606 given three times daily for 14 days
Drug: Mid-low dose LX1606 - Day 15 (start)
250 mg LX1606 given three times daily for 14 days
Drug: Mid-high dose LX1606 - Day 29 (start)
350 mg LX1606 given three times daily for 14 days
Drug: High dose LX1606 - Day 43 (start)
500 mg LX1606 given three times daily for 14 days
Drug: 4-Week Open Label Dose Extension
Patients will enter 4-week extension period at optimal dose once achieved, based upon clinical response criteria and/or observation of dose-limiting toxicity.
Drug: 72-Week Open Label Dose Extension
Upon completion of the 4-week stable-dose period, eligible subjects will have the option to continue treatment for an additional 72 weeks.


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Males and females, aged 18 and older
  • Biopsy-proven metastatic carcinoid tumor of the GI tract with disease extent confirmed by CT, 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01104415

Lexicon Investigational Site
Bad Berka, Germany
Lexicon Investigational Site
Berlin, Germany
Lexicon Investigational Site
Halle, Germany
Lexicon Investigational Site
Lubeck, Germany
Lexicon Investigational Site
Marburg, Germany
Lexicon Investigational Site
Munich, Germany
United Kingdom
Lexicon Investigational Site
Basingstoke Hampshire, United Kingdom
Lexicon Investigational Site
Cambridge, United Kingdom
Lexicon Investigational Site
London, United Kingdom
Lexicon Investigational Site
Manchester, United Kingdom
Sponsors and Collaborators
Lexicon Pharmaceuticals
Study Director: Pablo LaPuerta, MD Lexicon Pharmaceuticals, Inc.
  More Information

No publications provided

Responsible Party: Lexicon Pharmaceuticals Identifier: NCT01104415     History of Changes
Other Study ID Numbers: Protocol LX1606.1-203-CS, LX1606.203, LX1032
Study First Received: April 12, 2010
Last Updated: February 20, 2014
Health Authority: United States: Food and Drug Administration
Germany: Federal Institute for Drugs and Medical Devices

Keywords provided by Lexicon Pharmaceuticals:
Carcinoid syndrome

Additional relevant MeSH terms:
Carcinoid Tumor
Malignant Carcinoid Syndrome
Serotonin Syndrome
Chemically-Induced Disorders
Drug-Related Side Effects and Adverse Reactions
Neoplasms by Histologic Type
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neuroectodermal Tumors
Neuroendocrine Tumors
Pathologic Processes processed this record on February 27, 2015