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Completed

NCT Number: NCT00853047

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

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.

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Key information

Conditions

Carcinoid Syndrome Chemically-Induced Disorders Drug-Related Side Effects and Adverse Reactions Serotonin Syndrome

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hematology Oncology Services of Arkansas, Little Rock, Arkansas, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Telotristat etiprate

Drug

Telotristat etiprate capsules; orally 3 times daily.

Other names: LX1606

Octreotide LAR Depot

Drug

A stable-dose octreotide LAR depot therapy; administered subcutaneously once per month.

Placebo

Drug

Placebo-matching telotristat etiprate capsules; orally 3 times daily.

Primary outcomes

  1. Number of Participants With Any Treatment-emergent Adverse Event (TEAE) and Any Drug-related TEAE in the Core Phase

    Time frame: Up to 4 Weeks 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. A TEAE was an AE reported after the first dose of randomized treatment on Day 1.

  2. Number of Participants With Any TEAE in the Open-Label Extension Phase

    Time frame: Up to 180 weeks in the open-label extension phase

    An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE was an AE reported after receiving treatment.

Secondary outcomes

  1. Change From Baseline in Mean Number of Bowel Movements (BMs) Per Day

    Time frame: Baseline to Week 4

    Participants recorded the number of BMs per day in a daily diary. The total number of BMs per day were averaged over the 4-week period. A negative change from Baseline indicates improvement.

  2. Change From Baseline in Weekly Mean Stool Form

    Time frame: Baseline to Week 4

    Participants recorded stool form in a daily diary using a 6-point scale (0-none,1-hard, 2-firm, 3-soft, 4-loose, 5-watery). A negative change from Baseline indicates improvement.

  3. Change From Baseline in Percentage of Days Per Week Experiencing a Sensation of Urgency to Defecate

    Time frame: Baseline to Week 4

    Participants recorded the sensation of urgency to defecate (Yes or No) in a daily diary. A negative change from Baseline indicates improvement.

  4. Change From Baseline in Number of Cutaneous Flushing Episodes

    Time frame: Baseline to Week 4

    Participants recorded the number of daily flushing episodes per day in a daily diary. The total number of flushing episodes per day were averaged over the 4-week period. A negative change from Baseline indicates improvement.

  5. Change From Baseline in Severity of Abdominal Pain or Discomfort

    Time frame: Baseline to Week 4

    Participants recorded the severity of abdominal pain or discomfort in a daily diary assessed using a 4-point scale (0-none, 1-mild, 2-moderate, 3-severe). A negative change from Baseline indicates improvement.

  6. Change From Baseline in Urinary 5-hydroxyindoleacetic Acid (u5-HIAA)

    Time frame: Baseline to Week 4

    u5-HIAA is a standard test used in clinical practice to assess the neuroendocrine tumor (NET) activity and is collected as a 24-hour urine specimen. A negative change from Baseline indicates improvement.

  7. Change From Baseline in Chromogranin A

    Time frame: Baseline to Week 4

    Blood samples were collected for assessment of Chromogranin A level. A negative change from Baseline indicates improvement.

  8. Number of Participants Reporting Improvement in the Subjective Global Assessment of Symptoms Associated With Carcinoid Syndrome

    Time frame: Week 4

    Participants were asked to answer the following question: "In the past 7 days, have you had adequate relief of your carcinoid syndrome bowel complaints such as diarrhea, urgent need to have a bowel movement, abdominal pain, or discomfort?". The number of participants who answered Yes are reported.

  9. Change From Baseline in Frequency of Rescue for Short-acting Octreotide Use/Day

    Time frame: Baseline to Week 4

    Participants recorded details (location and frequency of injection) of subcutaneous injections of rescue, short-acting octreotide, if taken, in the daily diary. A negative change from Baseline indicates improvement.

  10. Time to First Rescue, Short-acting Octreotide

    Time frame: Baseline to Week 4

    Time to the first subcutaneous injections of rescue, short-acting octreotide was determined from the participant's daily diary.

  11. Number of Participants Experiencing Complete Response at Week 4

    Time frame: Baseline to Week 4

    Complete Response to treatment was defined as one of the following: 1. Less than 4 bowel movements per day; or 2. A decrease in daily bowel movements that is ≥ 50% from baseline; or 3. A positive response to the global assessment question ("In the past 7 days, have you had adequate relief of your carcinoid syndrome bowel complaints such as diarrhea, urgent need to have a bowel movement, abdominal pain or discomfort?") for each of the last 2 weeks of the Treatment Period.

Sponsors and collaborators

Lead sponsor

Lexicon Pharmaceuticals

Industry

Registry information

Official study title

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Ascending, Multidose Study To Determine Safety and Tolerability of Orally Administered LX1606 in Subjects With Symptomatic Carcinoid Syndrome Refractory to Stable-Dose Octreotide Long-Acting Release Depot Therapy

Important dates

Study start
2009
Primary completion
2014
Study completion
2014
First posted
Feb 27, 2009
Registry last updated
Dec 26, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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