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Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

1 de agosto de 2015 actualizado por: Vertex Pharmaceuticals Incorporated

A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics, and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the ∆F508-CFTR Gene Mutation

The primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

Descripción general del estudio

Estado

Terminado

Condiciones

Intervención / Tratamiento

Descripción detallada

This was a Phase 2, randomized, double-blind, placebo-controlled, multiple-dose study of orally-administered VX-809 in participants with CF who are homozygous for the specific CFTR mutation known as ∆F508 or F508del. Enrollment was planned for 90 participants at approximately 20 centers. Participants were planned to be randomized in a 4:1 ratio to receive 1 of 4 doses of VX-809 or placebo once a day for 28 days in a parallel design. Participants were outpatients during the study, except for overnight stays on Day 1 and 28.

Tipo de estudio

Intervencionista

Inscripción (Actual)

93

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

  • Alemania
      • Cologne, Alemania
      • Hannover, Alemania
  • Bélgica
      • Brussels, Bélgica
      • Leuven, Bélgica
  • Canadá
    • Ontario
      • Toronto, Ontario, Canadá
  • Estados Unidos
    • Alabama
      • Birmingham, Alabama, Estados Unidos
    • California
      • Palo Alto, California, Estados Unidos
      • San Diego, California, Estados Unidos
    • Colorado
      • Aurora, Colorado, Estados Unidos
    • Georgia
      • Atlanta, Georgia, Estados Unidos
    • Illinois
      • Chicago, Illinois, Estados Unidos
    • Iowa
      • Iowa City, Iowa, Estados Unidos
    • Maryland
      • Baltimore, Maryland, Estados Unidos
    • Massachusetts
      • Boston, Massachusetts, Estados Unidos
    • Minnesota
      • Minneapolis, Minnesota, Estados Unidos
    • Missouri
      • St. Louis, Missouri, Estados Unidos
    • North Carolina
      • Chapel Hill, North Carolina, Estados Unidos
    • Ohio
      • Cincinnati, Ohio, Estados Unidos
      • Cleveland, Ohio, Estados Unidos
      • Columbus, Ohio, Estados Unidos
    • Pennsylvania
      • Philadelphia, Pennsylvania, Estados Unidos
      • Pittsburgh, Pennsylvania, Estados Unidos
    • Washington
      • Seattle, Washington, Estados Unidos
  • Países Bajos
      • Rotterdam, Países Bajos
      • Utrecht, Países Bajos

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:

  • Confirmed diagnosis of CF with ∆F508-CFTR mutation in both alleles
  • Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent (%) of predicted normal for age, gender, and height
  • Weight >=40 kilograms (kg) and body mass index greater than or equal to 18.5 kilogram per square meter (kg/m^2)
  • Screening laboratory values, tests, and physical examination within acceptable ranges
  • Negative pregnancy test (for women of child-bearing potential)
  • Able and willing to follow contraceptive requirements
  • Willing to remain on a stable medication regimen for the duration of study participation

Exclusion Criteria:

  • History of any illness, or any ongoing acute illness, that could impact the safety of the study participant or may confound results of study
  • Pulmonary exacerbation or changes in therapy for pulmonary disease within 14 days before receiving the first dose of study drug
  • Impaired hepatic or renal function
  • History of organ or hematological transplant

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 paralela
  • Enmascaramiento: Triple

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Comparador de placebos: Placebo
Placebo matched to VX-809 capsule orally once daily for 28 days.
Droga: Placebo
Placebo matched to VX-809 capsules.
Experimental: VX-809, 25 mg
VX-809, 25 milligram (mg) capsule orally once daily for 28 days.
Droga: VX-809
Capsules
Experimental: VX-809, 50 mg
VX-809, 50 mg capsule orally once daily for 28 days.
Droga: VX-809
Capsules
Experimental: VX-809, 100 mg
VX-809, 100 mg capsule orally once daily for 28 days.
Droga: VX-809
Capsules
Experimental: VX-809, 200 mg
VX-809, 200 mg capsule orally once daily for 28 days.
Droga: VX-809
Capsules

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Safety and Tolerability Based on Adverse Events (AEs)
Periodo de tiempo: Up to 14 days after last dose (last dose = Day 28)
AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. Serious adverse event (SAE) (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. An AE that started at or after initial dosing of study drug, or increased in severity after initial dosing of study drug visit is considered treatment-emergent.
Up to 14 days after last dose (last dose = Day 28)

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Day 28
Periodo de tiempo: Baseline, Day 28
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline, Day 28
Change From Baseline in Percent Predicted FEV1 at Day 28
Periodo de tiempo: Baseline, Day 28
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method.
Baseline, Day 28
Change From Baseline in Forced Vital Capacity (FVC) at Day 28
Periodo de tiempo: Baseline, Day 28
FVC is the volume of air that can be forcibly exhaled from the lungs after taking the deepest breath possible.
Baseline, Day 28
Change From Baseline in Forced Expiratory Flow Over the Middle Half of the FVC (FEF25-75) at Day 28
Periodo de tiempo: Baseline, Day 28
FEF25-75 is total volume of air exhaled from the lungs over the middle half of the FVC test, expressed as liters per second (L/sec).
Baseline, Day 28
Change From Baseline in Sweat Chloride at Day 28
Periodo de tiempo: Baseline, Day 28
Sweat samples were collected using an approved Macroduct (Wescor) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride.
Baseline, Day 28
Change From Baseline in Nasal Potential Difference (NPD) of Zero Chloride Plus Isoproterenol Response at Day 28
Periodo de tiempo: Baseline, Day 28

Nasal potential difference (NPD) provides a direct and sensitive evaluation of sodium and chloride transport in secretory epithelial cells via assessment of transepithelial bioelectric properties. NPD under conditions of zero chloride concentration perfusion solution in the presence of isoproterenol is reported.

NPDs were performed according to Cystic Fibrosis Foundation Therapeutics Development Network (CFFT TDN) Standard Operating Procedure (SOP) 528.00 "Standardization of Measurement of Nasal Membrane Transepithelial Potential Difference (NPD) - electronic data capture (EDC) and Perfusion or Perfusion-Free Probe".
Baseline, Day 28
Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Domain Scores at Day 28
Periodo de tiempo: Baseline, Day 28
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. CFQ-R domains include: Body, Digestion, Eat, Emotion, Health Perceptions, Physical, Respiratory, Role, Social, Treatment Burden, Vitality, and Weight. Individual domain score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Baseline, Day 28
Maximum Plasma Concentration (Cmax) of VX-809
Periodo de tiempo: Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, 24, and 30-60 hours post dose)
Only participants who received VX-809 were analyzed for this outcome measure.
Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, 24, and 30-60 hours post dose)
Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24) of VX-809
Periodo de tiempo: Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post dose)
Only participants who received VX-809 were analyzed for this outcome measure.
Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post dose)

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Vertex Pharmaceuticals Incorporated

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.

Publicaciones Generales

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

1 de marzo de 2009

Finalización primaria (Actual)

1 de diciembre de 2009

Finalización del estudio (Actual)

1 de diciembre de 2009

Fechas de registro del estudio

Enviado por primera vez

18 de marzo de 2009

Primero enviado que cumplió con los criterios de control de calidad

18 de marzo de 2009

Publicado por primera vez (Estimar)

19 de marzo de 2009

Actualizaciones de registros de estudio

Última actualización publicada (Estimar)

28 de agosto de 2015

Última actualización enviada que cumplió con los criterios de control de calidad

1 de agosto de 2015

Última verificación

1 de junio de 2015

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • VX08-809-101

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. .

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