- ICH GCP
- Registro de ensayos clínicos de EE. UU.
- Ensayo clínico NCT07731919
Study on Doses of Inhaled ALX1 in Adults With Bronchiectasis
23 de julio de 2026 actualizado por: Vast Therapeutics
A Phase 2a, Placebo-Controlled, Single-Blind, Dose Range-Finding Study of Inhaled ALX1 in Adults With Bronchiectasis
This study will evaluate the safety and effects of ALX1, an inhaled investigational treatment, in adults with bronchiectasis.
Participants will receive either ALX1 or a placebo (a treatment with no active medicine) for 14 days.
The study will compare different dose levels of ALX1 to help identify appropriate doses for future research based on safety, tolerability, and changes in predictive biomarkers.
Descripción general del estudio
Estado
Aún no reclutando
Condiciones
Intervención / Tratamiento
Descripción detallada
This is a Phase 2a, multicentre, placebo-controlled, single-blind, dose range-finding study will assess the safety, tolerability, pharmacodynamics (PD), and preliminary efficacy of inhaled ALX1 or placebo administered for 14 days in adult participants with bronchiectasis.
28 participants will be enrolled and assigned to one of four cohorts.
The total duration of study participation will be up to 53 days, including Pre-screening and Screening of approximately 32 days, a Treatment Period of approximately 14 days, and a Follow-up of approximately 1 day following the last dose.
Tipo de estudio
Intervencionista
Inscripción (Estimado)
28
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.
Estudio Contacto
- Nombre: Paul Bruinenberg, MD
- Número de teléfono: +1 201-312-0988
- Correo electrónico: pbruinenberg@vasttherapeutics.com
Copia de seguridad de contactos de estudio
- Nombre: Laura MacLean
- Correo electrónico: LMacLean@vasttherapeutics.com
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
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
No
Descripción
Inclusion Criteria:
- Current sputum producer with a history of chronic expectoration that, in the opinion of the Investigator, will be able to continue to reliably provide sputum throughout the study.
- Confirmed diagnosis of BE per high-resolution computed tomography (HRCT) prior to Screening due to any of the following: NCFB, CF, primary ciliary dyskinesia, or COPD.
- Clinical history consistent with BE (cough, daily chronic sputum production, and/or recurrent respiratory infections).
- FEV1 ≥ 40% of predicted values at Screening.
- Able to reproducibly perform spirometry manoeuvres (i.e., able to perform at least 3 acceptable forced expiratory curves based on the PI's assessment).
- History of at least one exacerbation treated with a course of antibiotics (inhaled, oral or intravenous [IV]) within the 24 months prior to Screening
- Woman of childbearing potential (WOCBP) or fertile man (see definitions in Section 5.3) agrees to use an acceptable method of contraception from the start of Screening until 90 days after the last dose of IP.
Exclusion Criteria:
- Negative sputum NEATstik result for neutrophil elastase at Pre-screening.
- History of Burkholderia cepacia complex within 2 years prior to Pre-screening and/or detection of any Burkholderia spp. in sputum culture or by polymerase chain reaction (PCR) at Screening.
- History of Aspergillus fumigatus requiring treatment within 12 months prior to Pre-screening.
- History of non-tuberculosis mycobacteria (NTM) infection requiring treatment within 12 months prior to Screening, or detection of one or more NTM species in sputum by PCR at Screening.
- History of bronchospasm with inhaled antibiotics or hypertonic saline.
- Haemoptysis exceeding 50 mL of blood from the respiratory tract at any time within 30 days prior to IP administration (Day 1).
- Initiated macrolide therapy within 90 days before Screening. Existing stable maintenance with inhaled macrolides is permitted if initiated more than 90 days prior to Screening.
- Received inhaled anti-pseudomonal therapy within the last 14 days before Pre-screening. Must be willing to refrain from use of inhaled anti-pseudomonal therapy during the study until completion of the Follow-up video/telephone call.
- Received oral antibiotics other than macrolide within 30 days prior to Screening. Must be willing to refrain from use of oral antibiotics during the study until completion of the Follow-up video/telephone call.
- Received IV antibiotics within 60 days prior to Screening
- Initiation of, or increase in the dose of, inhaled corticosteroids within 90 days prior to Screening. Note: participants may be taking stable inhaled corticosteroids at the time of enrolment but must have initiated treatment more than 90 days prior to Screening
- Started any of the following muco-corrective therapies (e.g., nebulised saline, N-acetyl cysteine, Pulmozyme®, etc.) within 30 days prior to Screening. Maintenance with these muco-corrective therapies is permitted if initiated 30 days prior to Screening.
Any of the following laboratory abnormalities at Screening:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2.5 × upper limit of normal (ULN)
- Creatinine > 1.5 × ULN
- QT interval corrected by Fridericia's formula (QTcF) interval > 450 ms for males or > 470 ms for females at Screening, or history of prolonged QT syndrome. PR interval < 200 ms at Screening. Out-of-range values may be repeated twice for confirmation. The mean QTcF and PR intervals of the triplicate ECG recordings will be used to determine qualification.
- Positive test for hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), or human immunodeficiency virus (HIV) antibody at 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 paralela
- Enmascaramiento: Único
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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Comparador de placebos: Placebo
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Dose Formulation: Solution for inhalation Dose Strength: 0.9% sodium chloride Route of Administration: Inhalation via nebulizer
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Experimental: ALX1
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Dose Formulation: Solution for inhalation Dose Strength: 28 mg/mL Route of Administration: Inhalation via nebulizer
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Periodo de tiempo |
|---|---|
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Proportion of participants with a metHb value ≥ 5% per dose level
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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From Day 1 to Day 14 (EOT visit)
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Incidence of TEAEs
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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From Day 1 to Day 14 (EOT visit)
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Incidence of SAEs
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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From Day 1 to Day 14 (EOT visit)
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Proportion of participants with abnormal vital signs
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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From Day 1 to Day 14 (EOT visit)
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Proportion of participants with abnormal Laboratory parameters
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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From Day 1 to Day 14 (EOT visit)
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Proportion of participants with abnormal ECG readings
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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From Day 1 to Day 14 (EOT visit)
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Proportion of participants with abnormal SpO2
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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From Day 1 to Day 14 (EOT visit)
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Proportion of participants with abnormal Spirometry Value
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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From Day 1 to Day 14 (EOT visit)
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Mean change in sputum inflammatory biomarkers per dose level
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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Active neutrophil elastase, IL-1β, IL-6, IL-8, and TNF-alpha biomarkers assessed via quantitative immunoassay
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From Day 1 to Day 14 (EOT visit)
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Mean change in total bacterial load of pathogens per dose level
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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Assessed by sputum culture and quantitative polymerase chain reaction (qPCR)
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From Day 1 to Day 14 (EOT visit)
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Proportion of participants achieving a microbiological culture of pathogens change of at least 1-log CFU/g per dose level
Periodo de tiempo: From Day 1 to Day 14 (EOT visit)
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From Day 1 to Day 14 (EOT visit)
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Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
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 (Estimado)
1 de septiembre de 2026
Finalización primaria (Estimado)
31 de marzo de 2027
Finalización del estudio (Estimado)
31 de marzo de 2027
Fechas de registro del estudio
Enviado por primera vez
23 de julio de 2026
Primero enviado que cumplió con los criterios de control de calidad
23 de julio de 2026
Publicado por primera vez (Actual)
28 de julio de 2026
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
28 de julio de 2026
Última actualización enviada que cumplió con los criterios de control de calidad
23 de julio de 2026
Última verificación
1 de julio de 2026
Más información
Términos relacionados con este estudio
Otros números de identificación del estudio
- VST-201
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
NO
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
No
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
No
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