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).
Visão geral do estudo
Status
Condições
Descrição detalhada
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 estudo
Inscrição (Estimado)
Estágio
- Fase 1
Contactos e Locais
Contato de estudo
- Nome: Medical Information
- Número de telefone: 617-341-6777
- E-mail: medicalinfo@vrtx.com
Locais de estudo
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-
Kansas
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Overland Park, Kansas, Estados Unidos, 66212
- Recrutamento
- Altasciences - Kansas City
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-
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
Key Inclusion Criteria:
-
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
-
Part D:
- Confirmed diagnosis of CF
Key Exclusion Criteria:
-
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
-
Part D:
- History of clinically significant cirrhosis with or without portal hypertension.
Other protocol defined Inclusion/Exclusion criteria may apply.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: Randomizado
- Modelo Intervencional: Atribuição Paralela
- Mascaramento: Quadruplicar
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
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 Placebo: 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 Placebo: 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.
Outros nomes:
Tablet for Oral Administration.
Outros nomes:
|
|
Comparador de Placebo: 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.
Outros nomes:
Tablet for Oral Administration.
Outros nomes:
|
|
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.
Outros nomes:
Tablet for Oral Administration.
Outros nomes:
Fixed dose combination (FDC) Tablets for Oral Administration.
Outros nomes:
Tablet for Oral Administration.
Outros nomes:
|
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Prazo |
|---|---|
|
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Prazo: From Day -1 up to Day 8
|
From Day -1 up to Day 8
|
|
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Prazo: From Day -1 up to Day 17
|
From Day -1 up to Day 17
|
|
Part C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Prazo: From Day -1 up to Day 17
|
From Day -1 up to Day 17
|
|
Part D: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Prazo: From Signing of Informed Consent up to Day 105
|
From Signing of Informed Consent up to Day 105
|
Medidas de resultados secundários
Medida de resultado |
Prazo |
|---|---|
|
Part A: Maximum Observed Plasma Concentration (Cmax) of VX-581
Prazo: On Day 1
|
On Day 1
|
|
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
Prazo: On Day 1
|
On Day 1
|
|
Part B: Cmax of VX-581
Prazo: On Days 1, 7, and 10
|
On Days 1, 7, and 10
|
|
Part B: AUC0-24h Post-Dose of VX-581
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: On Day 1 and Day 28
|
On Day 1 and Day 28
|
|
Part D: AUC0-24h Post Dose of VX-581, TEZ (if administered), D-IVA and their Metabolite(s)
Prazo: On Day 1 and Day 28
|
On Day 1 and Day 28
|
|
Part D: Absolute Change in Sweat Chloride (SwCl)
Prazo: From Baseline Up To Day 28
|
From Baseline Up To Day 28
|
Colaboradores e Investigadores
Patrocinador
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
- Doenças Genéticas, Congênitas
- Doenças Respiratórias
- Doenças do aparelho digestivo
- Doenças pulmonares
- Lactente, Recém Nascido, Doenças
- Doenças pancreáticas
- Doenças e Anormalidades Congênitas, Hereditárias e Neonatais
- Fibrose cística
- Elexacaftor, ivacaftor, combinação de drogas tezacaftor
- Tezacaftor
- ivacaftor
- Deutivacaftor, Tezacaftor, Vanzacaftor
Outros números de identificação do estudo
- VX25-581-001
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Descrição do plano 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/
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