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Phase 3 N=76 Randomized Triple-blind Treatment

Telotristat Etiprate for Carcinoid Syndrome Therapy

Carcinoid Syndrome

Enrolled (actual)
76
Serious AEs
18.2%
Results posted
Sep 2017
Primary outcome: Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) in the Double-Blind Treatment Period — 21; 25; 22 Participants

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Telotristat etiprate (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Lexicon Pharmaceuticals
Primary completion
Mar 2016

Outcome Measures

OutcomeResultp-value
PRIMARY
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) in the Double-Blind Treatment Period
21; 25; 22
PRIMARY
Primary: Percent Change From Baseline in Urinary 5-hydroxyindoleacetic Acid (u5-HIAA) Levels
97.721; -33.164; -76.466 < 0.001 sig
PRIMARY
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) in the Open-Label Extension Period
61
SECONDARY
Change From Baseline in the Number of Bowel Movements (BMs) Per Day Averaged Over 12 Weeks
0.050; -0.452; -0.595
SECONDARY
Change From Baseline in Stool Form/Consistency Averaged Across All Time-Points
0.006; -0.196; -0.597
SECONDARY
Change From Baseline in the Number of Daily Cutaneous Flushing Episodes Averaged Across All Time-Points
-0.333; -0.061; 0.114
SECONDARY
Change From Baseline in Abdominal Pain Averaged Across All Time-Points
-0.063; -0.234; 0.025
SECONDARY
Change in the Frequency of Rescue Short-acting, Somatostatin Analog (SSA) Used to Treat Carcinoid Syndrome Symptoms Averaged Across All Time-Points
-0.013; -0.065; 0.006
SECONDARY
Change From Baseline in the Number of Daily BMs Averaged Over the 12-Week Double-Blind Period, Among Participants Who Were Not Receiving SSA Therapy at Baseline
-0.906; -0.98

Summary

The purpose of the study is to evaluate the effect of telotristat etiprate versus placebo on the incidence of treatment-emergent adverse events and on 5-hydroxyindoleacetic acid (5-HIAA) levels.

Eligibility Criteria

Inclusion Criteria

  • Patients ≥ 18 years of age
  • All patients of reproductive potential must agree to use an adequate method of contraception during the study and for 12 weeks after the Follow-up visit.
  • Histopathologically-confirmed, well-differentiated metastatic neuroendocrine tumor
  • Documented history of carcinoid syndrome
  • Patient is able and willing to provide written informed consent prior to participation

Exclusion Criteria

  • Presence of diarrhea attributed to any condition other than carcinoid syndrome.
  • Presence of 12 or more watery bowel movements per day
  • Positive stool examination for enteric pathogens, pathogenic ova or parasites, of Clostridium difficile at Screening
  • Karnofsky Performance Status ≤ 60%
  • Presence of any clinically significant laboratory, medical history, or physical examination findings deemed unacceptable by the Investigator
  • A history of short bowel syndrome
  • History of constipation within 2 years of Screening
  • Life expectancy < 12 months from Screening
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02063659). 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.

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