- ICH GCP
- Registro de ensayos clínicos de EE. UU.
- Ensayo clínico NCT01104415
Study of Telotristat Etiprate (LX1606) in Participants With Symptomatic Carcinoid Syndrome
26 de febrero de 2019 actualizado 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.
Descripción general del estudio
Estado
Terminado
Condiciones
Intervención / Tratamiento
Tipo de estudio
Intervencionista
Inscripción (Actual)
15
Fase
- Fase 2
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Ubicaciones de estudio
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Bad Berka, Alemania
- Lexicon Investigational Site
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Berlin, Alemania
- Lexicon Investigational Site
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Halle, Alemania
- Lexicon Investigational Site
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Lubeck, Alemania
- Lexicon Investigational Site
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Marburg, Alemania
- Lexicon Investigational Site
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Munich, Alemania
- Lexicon Investigational Site
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Basingstoke, Reino Unido
- Lexicon Investigational Site
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Cambridge, Reino Unido
- Lexicon Investigational Site
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London, Reino Unido
- Lexicon Investigational Site
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Manchester, Reino Unido
- Lexicon Investigational Site
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Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
18 años y mayores (Adulto, Adulto Mayor)
Acepta Voluntarios Saludables
No
Géneros elegibles para el estudio
Todos
Descripción
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
Plan de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: Aleatorizado
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
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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.
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Telotristat etiprate capsules orally three times daily.
Otros nombres:
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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.
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Telotristat etiprate capsules orally three times daily.
Otros nombres:
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Core Phase
Periodo de tiempo: Baseline up to Week 12 in the Core Phase
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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.
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Baseline up to Week 12 in the Core Phase
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Number of Participants With Any Treatment Emergent Adverse Events (TEAEs) and Drug-Related TEAEs in the Extension Period
Periodo de tiempo: Up to 124 Weeks in the Extension Period
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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.
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Up to 124 Weeks in the Extension Period
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Change From Baseline in Number of Bowel Movements (BMs)
Periodo de tiempo: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Change From Baseline in Stool Form/Consistency
Periodo de tiempo: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Change From Baseline in Percentage of Days With Sensation of Urgency to Defecate
Periodo de tiempo: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Change From Baseline in Sensation/Severity of Nausea Using 100 mm Visual Analog Scale (VAS)
Periodo de tiempo: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Number of Participants With an Improvement in Global Assessment of Symptoms Associated With Carcinoid Syndrome
Periodo de tiempo: Core Phase: Weeks 9-12; Extension Period: Week 24
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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.
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Core Phase: Weeks 9-12; Extension Period: Week 24
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Change From Baseline in Daily Severity of Abdominal Pain or Discomfort Using 100 mm Visual Analog Scale (VAS)
Periodo de tiempo: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Change From Baseline in Daily Number of Cutaneous Flushing Episodes
Periodo de tiempo: Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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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.
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Core Phase: Baseline to Weeks 9-12; Extension Period: Baseline to Week 24
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Number of Participants Achieving Clinically Meaningful Symptom Reduction in the Core Phase
Periodo de tiempo: Baseline to Week 12
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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.
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Baseline to Week 12
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Change From Baseline in Urinary 5-Hydroxyindoleacetic Acid (HIAA) Levels
Periodo de tiempo: Core Phase: Baseline to Week 12; Extension Period: Baseline to Weeks 20-21
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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.
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Core Phase: Baseline to Week 12; Extension Period: Baseline to Weeks 20-21
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Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Investigadores
- Director de estudio: Pablo LaPuerta, MD, Lexicon Pharmaceuticals, Inc.
Publicaciones y enlaces útiles
La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio (Actual)
15 de junio de 2010
Finalización primaria (Actual)
12 de febrero de 2014
Finalización del estudio (Actual)
12 de febrero de 2014
Fechas de registro del estudio
Enviado por primera vez
12 de abril de 2010
Primero enviado que cumplió con los criterios de control de calidad
13 de abril de 2010
Publicado por primera vez (Estimar)
15 de abril de 2010
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
15 de marzo de 2019
Última actualización enviada que cumplió con los criterios de control de calidad
26 de febrero de 2019
Última verificación
1 de febrero de 2019
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Trastornos inducidos químicamente
- Procesos Patológicos
- Neoplasias por tipo histológico
- Neoplasias
- Adenocarcinoma
- Carcinoma
- Neoplasias Glandulares y Epiteliales
- Enfermedad
- Tumores neuroectodérmicos
- Neoplasias De Células Germinales Y Embrionarias
- Neoplasias De Tejido Nervioso
- Tumores neuroendocrinos
- Efectos secundarios y reacciones adversas relacionados con los medicamentos
- Síndrome
- Tumor carcinoide
- Síndrome Carcinoide Maligno
- Síndrome de serotonina
Otros números de identificación del estudio
- LX1606.1-203-CS
- LX1606.203, LX1032 (Otro identificador: Lexicon Pharmaceuticals, Inc.)
- 2009-016973-13 (Número EudraCT)
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Sí
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
No
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .
Ensayos clínicos sobre Telotristat etiprate
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University of ChicagoRetirado
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European Organisation for Research and Treatment...IpsenRetiradoTNE del intestino delgado | Enfermedad cardíaca carcinoide
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