Sildenafil to Repair Brain Injury Secondary to Birth Asphyxia

Treatment of Neonatal Encephalopathy With Oral Sildenafil Suspension to Repair Brain Injury Secondary to Birth Asphyxia: An Open-label Dose-finding Clinical Trial (Phase Ib Study)

Patrocinadores

Patrocinador principal: McGill University Health Centre/Research Institute of the McGill University Health Centre

Fuente McGill University Health Centre/Research Institute of the McGill University Health Centre
Resumen breve

The investigators will determine the maximum tolerable dose of sildenafil and establish the pharmacokinetic and pharmacodynamic profile of sildenafil in human asphyxiated neonates treated with hypothermia. They will use a 3+3 design to escalate the sildenafil dose up to 6 mg/kg/day (3mg/kg/dose q12h) in asphyxiated neonates demonstrating brain injury despite hypothermia treatment and assess whether we observe any beneficial effects of sildenafil on their brain and cardiopulmonary hemodynamics, without causing serious adverse events

Estado general Recruiting
Fecha de inicio September 19, 2019
Fecha de Terminación August 2024
Fecha de finalización primaria August 2022
Fase Phase 1
Tipo de estudio Interventional
Resultado primario
Medida Periodo de tiempo
Death within first 10 days of life
Hypotension within first 10 days of life
Resultado secundario
Medida Periodo de tiempo
Peak Plasma Concentration (Cmax) within first 10 days of life
Inscripción 20
Condición
Intervención

Tipo de intervención: Drug

Nombre de intervención: Sildenafil Citrate

Descripción: Cohort 1 of 3-6 neonates: 1st dose of 2mg/kg/dose, 2nd dose of 2.5 mg/kg/dose, and subsequent doses of 2.5mg/kg/dose q12h (= 5 mg/kg/day from dose #2) Cohort 2 of 3-6 neonates: 1st dose of 2mg/kg/dose, 2nd dose of 2.5 mg/kg/dose, 3rd dose of 3mg/kg/dose, and subsequent doses of 3mg/kg/dose q12h (= 6 mg/kg/day from dose #3) dose expansion phase in up to 5-15 asphyxiated neonates

Etiqueta de grupo de brazo: Sildenafil

Elegibilidad

Criterios:

Inclusion Criteria:

- Male and female neonates meeting the criteria for induced hypothermia:

- Gestational age ≥ 36 weeks and birth weight ≥ 1800 g;

- Evidence of fetal distress, i.e., history of an acute perinatal event, cord pH ≤ 7.0 or base deficit ≤ - 16 mEq/L;

- Evidence of neonatal distress, such as an Apgar score ≤ 5 at 10 minutes, postnatal blood gas pH obtained within the first hour of life ≤ 7.0 or base deficit ≤ - 16 mEq/L, or a continued need for ventilation initiated at birth and continued for at least 10 minutes;

- Evidence of moderate to severe neonatal encephalopathy by an abnormal neurological exam and/or an amplitude-integrated electroencephalogram (aEEG).

- Evidence of brain injury on a brain magnetic resonance imaging (MRI) performed on day 2 of life.

Exclusion Criteria:

- Neonates with complex congenital heart disease

- Neonates with cerebral malformations

- Neonates with genetic syndrome

- Neonates with intraventricular and/or intraparenchymal hemorrhage on MRI performed on day 2 of life

Género: All

Edad mínima: N/A

Edad máxima: 2 Days

Voluntarios Saludables: No

Contacto general

Apellido: Pia Wintermark, MD

Teléfono: 514-412-4452

Email: [email protected]

Ubicación
Instalaciones: Estado: Contacto: Investigador: Montreal Children's Hospital Pia Wintermark, MD 514-41-4452 Pia Wintermark, MD Principal Investigator
Ubicacion Paises

Canada

Fecha de verificación

November 2019

Fiesta responsable

Tipo: Principal Investigator

Afiliación del investigador: McGill University Health Centre/Research Institute of the McGill University Health Centre

Nombre completo del investigador: Pia Wintermark

Título del investigador: Associate Professor of Pediatrics

Tiene acceso ampliado No
Condición Examinar
Número de brazos 1
Grupo de brazo

Etiqueta: Sildenafil

Tipo: Experimental

Acrónimo SANE-02
Datos del paciente No
Información de diseño del estudio

Asignación: N/A

Modelo de intervención: Single Group Assignment

Descripción del modelo de intervención: 3+3 design to escalate the sildenafil dose up to 6 mg/kg/day (3mg/kg/dose q12h)

Propósito primario: Treatment

Enmascaramiento: None (Open Label)

Fuente: ClinicalTrials.gov