Evaluation of VX-581 in Healthy Participants and in Participants With Cystic Fibrosis
A Phase 1 Study of VX-581 in Healthy Subjects and in Subjects With Cystic Fibrosis
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of single dose of VX-581 and multiple ascending doses of VX-581 alone and in combination with tezacaftor (TEZ)/deutivacaftor (D-IVA) or D-IVA in healthy participants. The study will also evaluate the safety, tolerability, and pharmacokinetics of VX-581 administered in combination with D-IVA with or without TEZ in participants with cystic fibrosis (CF).
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
The study is being conducted to evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of VX-581 in healthy participants and in participants with CF. VX-581 will be administered alone and in combination with TEZ and D-IVA. The study consists of Screening Phase, Run-In Phase (CF only), Treatment Phase and Safety Follow-up Phase.
Note: This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).
Tipo de estudio
Inscripción (Estimado)
Fase
- Fase 1
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Medical Information
- Número de teléfono: 617-341-6777
- Correo electrónico: medicalinfo@vrtx.com
Ubicaciones de estudio
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Kansas
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Overland Park, Kansas, Estados Unidos, 66212
- Reclutamiento
- Altasciences - Kansas City
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
Key Inclusion Criteria:
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Parts A, B, C:
- Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m^2)
- A total body weight of more than (>) 50 kg
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Part D:
- Confirmed diagnosis of CF
Key Exclusion Criteria:
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Parts A, B, C:
- History of febrile illness or other acute illness that has not fully resolved within 14 days before the first dose of study drug
- Any condition possibly affecting drug absorption
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Part D:
- History of clinically significant cirrhosis with or without portal hypertension.
Other protocol defined Inclusion/Exclusion criteria may apply.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: Aleatorizado
- Modelo Intervencionista: Asignación paralela
- Enmascaramiento: Cuadruplicar
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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Experimental: Part A: Single Ascending Dose
Participants will be randomized to receive a single dose of VX-581. |
Suspension for Oral Administration. Tablets for Oral Administration. |
|
Comparador de placebos: Placebo: Part A
Participants will be randomized to receive a single dose of placebo matched to VX-581. |
Suspension for Oral Administration. |
|
Experimental: Part B: Multiple Ascending Dose
Participants will be randomized to receive multiple doses of VX-581. |
Suspension for Oral Administration. Tablets for Oral Administration. |
|
Comparador de placebos: Placebo: Part B
Participants will be randomized to receive multiple doses of placebo matched to VX-581. |
Suspension for Oral Administration. |
|
Experimental: Part C: VX-581 with TEZ/D-IVA or D-IVA
Participants will be randomized to receive multiple doses VX-581 in combination with TEZ/D-IVA or D-IVA. |
Suspension for Oral Administration. Tablets for Oral Administration. Tablet for Oral Administration.
Otros nombres:
Tablet for Oral Administration.
Otros nombres:
|
|
Comparador de placebos: Placebo: Part C
Participants will be randomized to receive placebo matched to VX-581 in combination with TEZ/D-IVA or D-IVA. |
Suspension for Oral Administration. Tablet for Oral Administration.
Otros nombres:
Tablet for Oral Administration.
Otros nombres:
|
|
Experimental: Part D: VX-581 with TEZ/D-IVA or D-IVA in CF
Participants with CF will enter a run-in period and receive ELX/TEZ/IVA. Following the run-in period participants will receive VX-581 in combination with TEZ/D-IVA or D-IVA in the treatment period. Following the treatment period, participants will enter a 14-day Washout Period and receive ELX/TEZ/IVA. (Part D will be open-label) |
Suspension for Oral Administration. Tablets for Oral Administration. Tablet for Oral Administration.
Otros nombres:
Tablet for Oral Administration.
Otros nombres:
Fixed dose combination (FDC) Tablets for Oral Administration.
Otros nombres:
Tablet for Oral Administration.
Otros nombres:
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Periodo de tiempo |
|---|---|
|
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Periodo de tiempo: From Day -1 up to Day 8
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From Day -1 up to Day 8
|
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Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Periodo de tiempo: From Day -1 up to Day 17
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From Day -1 up to Day 17
|
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Part C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Periodo de tiempo: From Day -1 up to Day 17
|
From Day -1 up to Day 17
|
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Part D: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Periodo de tiempo: From Signing of Informed Consent up to Day 105
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From Signing of Informed Consent up to Day 105
|
Medidas de resultado secundarias
Medida de resultado |
Periodo de tiempo |
|---|---|
|
Part A: Maximum Observed Plasma Concentration (Cmax) of VX-581
Periodo de tiempo: On Day 1
|
On Day 1
|
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Part A: Area Under the Concentration Versus Time Curve From the Time of Dosing Up to 24 Hours (AUC0-24h) Post-Dose of VX-581
Periodo de tiempo: On Day 1
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On Day 1
|
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Part B: Cmax of VX-581
Periodo de tiempo: On Days 1, 7, and 10
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On Days 1, 7, and 10
|
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Part B: AUC0-24h Post-Dose of VX-581
Periodo de tiempo: On Days 1, 7, and 10
|
On Days 1, 7, and 10
|
|
Part C: Cmax of VX-581, TEZ (if administered), D-IVA and their Metabolite(s)
Periodo de tiempo: On Days 1, 7, and 10
|
On Days 1, 7, and 10
|
|
Part C: AUC0-24h Post Dose of VX-581, TEZ (if administered), D-IVA and their Metabolite(s)
Periodo de tiempo: On Days 1, 7, and 10
|
On Days 1, 7, and 10
|
|
Part D: Cmax of VX-581, TEZ (if administered), D-IVA and their Metabolite(s)
Periodo de tiempo: On Day 1 and Day 28
|
On Day 1 and Day 28
|
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Part D: AUC0-24h Post Dose of VX-581, TEZ (if administered), D-IVA and their Metabolite(s)
Periodo de tiempo: On Day 1 and Day 28
|
On Day 1 and Day 28
|
|
Part D: Absolute Change in Sweat Chloride (SwCl)
Periodo de tiempo: From Baseline Up To Day 28
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From Baseline Up To Day 28
|
Colaboradores e Investigadores
Patrocinador
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Enfermedades Genéticas Congénitas
- Enfermedades de las vías respiratorias
- Enfermedades del Sistema Digestivo
- Enfermedades pulmonares
- Infantil, Recién Nacido, Enfermedades
- Enfermedades pancreáticas
- Enfermedades y anomalías congénitas, hereditarias y neonatales
- Fibrosis quística
- Elexacaftor, Ivacaftor, Tezacaftor Combinación de drogas
- tezacaftor
- ivacaftor
- Deutivacaftor, Tezacaftor, Vanzacaftor
Otros números de identificación del estudio
- VX25-581-001
Plan de datos de participantes individuales (IPD)
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Descripción del plan IPD
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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