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

26 februari 2019 uppdaterad av: Lexicon Pharmaceuticals

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

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

Studieöversikt

Status

Avslutad

Betingelser

Intervention / Behandling

Studietyp

Interventionell

Inskrivning (Faktisk)

15

Fas

  • Fas 2

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

  • Storbritannien
      • Basingstoke, Storbritannien
        • Lexicon Investigational Site
      • Cambridge, Storbritannien
        • Lexicon Investigational Site
      • London, Storbritannien
        • Lexicon Investigational Site
      • Manchester, Storbritannien
        • Lexicon Investigational Site
  • Tyskland
      • Bad Berka, Tyskland
        • Lexicon Investigational Site
      • Berlin, Tyskland
        • Lexicon Investigational Site
      • Halle, Tyskland
        • Lexicon Investigational Site
      • Lubeck, Tyskland
        • Lexicon Investigational Site
      • Marburg, Tyskland
        • Lexicon Investigational Site
      • Munich, Tyskland
        • Lexicon Investigational Site

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

18 år och äldre (Vuxen, Äldre vuxen)

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Allt

Beskrivning

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

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: Randomiserad
  • Interventionsmodell: Enskild gruppuppgift
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: Telotristat etiprate - Core Phase
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.
Läkemedel: Telotristat etiprate
Telotristat etiprate capsules orally three times daily.
Andra namn:
  • LX1606 Hippurat
Experimentell: Telotristat etiprate - Extension Period
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.
Läkemedel: Telotristat etiprate
Telotristat etiprate capsules orally three times daily.
Andra namn:
  • LX1606 Hippurat

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Core Phase
Tidsram: Baseline up to Week 12 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
Tidsram: Up to 124 Weeks 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

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Change From Baseline in Number of Bowel Movements (BMs)
Tidsram: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Participants recorded the number of bowel movements in a daily diary. The change from baseline value was calculated as the difference between mean numbers of BMs of the post-baseline interval (Weeks 9 to 12) and baseline. A negative change from baseline indicates improvement. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Change From Baseline in Stool Form/Consistency
Tidsram: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Participants assessed stool form/consistency in a daily diary using a 6-point scale (0-none, 1-hard, 2-firm, 3-soft, 4-loose, 5-watery). The change from the baseline value was calculated as the difference between a mean score of the post-baseline interval (Weeks 9 to 12) and baseline. 0 indicates the best score and 5 indicates the worst score. A negative change from baseline indicates improvement. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
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
Tidsram: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Participants assessed the urgency to defecate using a daily diary response to the following question, "Have you felt or experienced a sense of urgency to pass stool today?". The change from the baseline value was calculated as the difference between the mean score (percentage of days) of the post-baseline interval (Weeks 9 to 12) and baseline. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
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)
Tidsram: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Sensation/severity of nausea was measured using a 100 mm VAS. Participants rated their perception of the sensation/severity of nausea experienced by marking a single vertical line on a VAS scale from 0 to 100 mm, where 0 = No vomiting and 100 = vomiting. The change from the baseline value was calculated as the difference between the mean score of the post-baseline interval (Weeks 9 to 12) and baseline. 0 indicates the best score, 100 indicates the worst score. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
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
Tidsram: Core Phase: Weeks 9-12; Extension Period: Week 24
Participants assessed their symptoms using a weekly subjective response to 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 values for improvement in global assessment of symptoms associated with carcinoid syndrome in the Core Phase were averaged from Weeks 9 to 12.
Core Phase: Weeks 9-12; Extension Period: Week 24
Change From Baseline in Daily Severity of Abdominal Pain or Discomfort Using 100 mm Visual Analog Scale (VAS)
Tidsram: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
The severity of abdominal pain was measured using a 100 mm VAS. Participants rated their perception of the sensation/severity of abdominal pain or experienced by marking a single vertical line on a VAS scale from 0 to 100 mm, where 0 = No vomiting and 100 = vomiting. The change from the baseline value was calculated as the difference between the mean score of the post-baseline interval (Weeks 9 to 12) and baseline. 0 indicate the best score, 100 indicates the worst score. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Change From Baseline in Daily Number of Cutaneous Flushing Episodes
Tidsram: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Participants recorded the number of daily cutaneous flushing episodes experienced in the daily diary. The change from baseline value was calculated as the difference between the mean numbers of cutaneous flushing episodes of the post-baseline interval (Weeks 9 to 12) and baseline. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
Number of Participants Achieving Clinically Meaningful Symptom Reduction in the Core Phase
Tidsram: Baseline to Week 12
Clinically meaningful symptom reduction was defined as either: a) an average of < 4 bowel movements per day over 15 consecutive days, b) a 50% reduction from baseline in the number of bowel movements, c) a positive response to the question regarding adequate relief, or d) a 50% reduction from baseline in the number of daily flushing episodes.
Baseline to Week 12
Change From Baseline in Urinary 5-Hydroxyindoleacetic Acid (HIAA) Levels
Tidsram: Core Phase: Baseline to Week 12; Extension Period: Baseline to Weeks 20-21
Urinary 5-HIAA (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. The change from baseline value for the Extension Period was calculated as the difference between mean change in 5-HIAA of the post-baseline interval (Weeks 20 to 21) and baseline. A negative change from baseline indicates improvement. Baseline for the Extension Period was defined as non-missing assessment in Run-in period prior to the first dose of study drug.
Core Phase: Baseline to Week 12; Extension Period: Baseline to Weeks 20-21

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Lexicon Pharmaceuticals

Utredare

  • Studierektor: Pablo LaPuerta, MD, Lexicon Pharmaceuticals, Inc.

Publikationer och användbara länkar

Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.

Allmänna publikationer

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

15 juni 2010

Primärt slutförande (Faktisk)

12 februari 2014

Avslutad studie (Faktisk)

12 februari 2014

Studieregistreringsdatum

Först inskickad

12 april 2010

Först inskickad som uppfyllde QC-kriterierna

13 april 2010

Första postat (Uppskatta)

15 april 2010

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

15 mars 2019

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

26 februari 2019

Senast verifierad

1 februari 2019

Mer information

Termer relaterade till denna studie

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • LX1606.1-203-CS
  • LX1606.203, LX1032 (Annan identifierare: Lexicon Pharmaceuticals, Inc.)
  • 2009-016973-13 (EudraCT-nummer)

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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