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

26 de fevereiro de 2019 atualizado por: 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.

Visão geral do estudo

Status

Concluído

Condições

Intervenção / Tratamento

Tipo de estudo

Intervencional

Inscrição (Real)

15

Estágio

  • Fase 2

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

  • Alemanha
      • Bad Berka, Alemanha
        • Lexicon Investigational Site
      • Berlin, Alemanha
        • Lexicon Investigational Site
      • Halle, Alemanha
        • Lexicon Investigational Site
      • Lubeck, Alemanha
        • Lexicon Investigational Site
      • Marburg, Alemanha
        • Lexicon Investigational Site
      • Munich, Alemanha
        • Lexicon Investigational Site
  • Reino Unido
      • Basingstoke, Reino Unido
        • Lexicon Investigational Site
      • Cambridge, Reino Unido
        • Lexicon Investigational Site
      • London, Reino Unido
        • Lexicon Investigational Site
      • Manchester, Reino Unido
        • Lexicon Investigational Site

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

18 anos e mais velhos (Adulto, Adulto mais velho)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

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

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição de grupo único
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: 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.
Medicamento: Telotristat etiprate
Telotristat etiprate capsules orally three times daily.
Outros nomes:
  • LX1606 Hipurato
Experimental: 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.
Medicamento: Telotristat etiprate
Telotristat etiprate capsules orally three times daily.
Outros nomes:
  • LX1606 Hipurato

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Core Phase
Prazo: 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
Prazo: 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

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Change From Baseline in Number of Bowel Movements (BMs)
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Lexicon Pharmaceuticals

Investigadores

  • Diretor de estudo: Pablo LaPuerta, MD, Lexicon Pharmaceuticals, Inc.

Publicações e links úteis

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Publicações Gerais

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

15 de junho de 2010

Conclusão Primária (Real)

12 de fevereiro de 2014

Conclusão do estudo (Real)

12 de fevereiro de 2014

Datas de inscrição no estudo

Enviado pela primeira vez

12 de abril de 2010

Enviado pela primeira vez que atendeu aos critérios de CQ

13 de abril de 2010

Primeira postagem (Estimativa)

15 de abril de 2010

Atualizações de registro de estudo

Última Atualização Postada (Real)

15 de março de 2019

Última atualização enviada que atendeu aos critérios de controle de qualidade

26 de fevereiro de 2019

Última verificação

1 de fevereiro de 2019

Mais Informações

Termos relacionados a este estudo

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • LX1606.1-203-CS
  • LX1606.203, LX1032 (Outro identificador: Lexicon Pharmaceuticals, Inc.)
  • 2009-016973-13 (Número EudraCT)

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Sim

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

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