Budesonide Following Endoscopic Dilatation of Esophageal Strictures in Children.
Single-blinded, Randomized Controlled Trial of Budesonide Following Endoscopic Dilatation of Esophageal Strictures in Children.
Endoscopic balloon dilatation (EBD) is a common therapeutic procedure performed for esophageal strictures. While the etiologies of esophageal strictures are numerous - including congenital strictures, caustic injuries and eosinophilic esophagitis (EoE) -anastomotic strictures following esophageal atresia and trachea-esophageal fistula repairs is reported as the most frequent cause in children.
Stricture dilatation is usually performed under direct endoscopic vision, with graduated balloon dilatation, utilizing increasing balloon diameters deployed until reaching a pre-determined, age-based, dilatation target. Balloon dilatation is repeated every 2-4 weeks until the target is reached. Success is defined as prolonged restoration of effective swallow with no dysphagia or dietary restrictions. Dilatation is repeated if clinical resolution is not obtained, or if symptoms return after initial resolution.
In children, a stricture is considered refractory when the age-appropriate esophageal lumen is not achieved with five dilatations within 5 months, or if despite ≥7 dilatations overall an age-appropriate esophageal lumen was not maintained.
Several interventions have been studied to improve the efficacy of balloon dilatation, including injecting corticosteroids at the dilatation site, topical Mitomycin C following dilatation, or pre-dilatation endoscopic incisions of the anastomotic scar tissue. All these interventions are performed endoscopically at the time of the dilatation, require advanced endoscopic skills and may potentially have associated additional risk.
In this study the investigators aim to test the efficacy and safety of topical budesonide gel following EBD for anastomotic esophageal strictures in a single-blinded, randomized controlled study of children.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
This single-blinded randomized controlled trial aims to prospectively evaluate the efficacy of swallowed budesonide gel following EBD of esophageal strictures. Due to the variable nature of congenital strictures and the more disparate features of caustic and EoE strictures, the study is limited to simple strictures at the site of anastomoses.
Patients will be assessed as per the ESPGHAN and NASPGHAN guidelines for the evaluation and treatment of children with esophageal atresia-trachoesophageal fistula, with gastroscopy performed either at one-year of age or earlier in symptomatic patients with dysphagia.
Patients in whom an anastomotic esophageal stricture is identified, dilatations will be performed as per physician discretion regarding diameter and timing of dilatations, guided predominantly by the aforementioned treatment guidelines. Patients will be randomly assigned to either budesonide gel (1mg/8mL) 250mcg orally twice daily for 12 weeks from the first dilatation, or to standard of care with no oral budesonide. Treating physician will be blinded to patient treatment Esophageal stricture characteristics will be recorded at each endoscopy with pre-dilation diameter (estimated based on relative size to pre-measured biopsy forceps placed in proximity to the stricture) and maximal dilatation diameter at each session.
Patients will be assessed clinically routinely at 1, 2, 3, 4 and 6 months following initial dilatation, and at 6 months following the last dilatation in the series, during which dysphagia symptom score and patient weight will be assessed.
Success is defined as restoration of effective swallow with no dysphagia or age-appropriate dietary restrictions for 3 months. Outcome measures will include number of EBD procedures until success is achieved, as well as the rate of restenosis (defined clinically as a dysphagia score of ≥1) after success is achieved 6 months following the last EBD procedure.
The primary aim of the study is to assess the number of dilatations required to achieve clinical success between the treatment and placebo group. Secondary aims include rate of refractory strictures, restenosis rate, dysphagia score, patient weight and safety (including cortisol suppression, oral or esophageal fungal infection, and anthropomorphics).
Tipo de estudio
Inscripción (Estimado)
Fase
- No aplica
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Oren Ledder, Dr
- Número de teléfono: +972-2-6666743
- Correo electrónico: orenl@szmc.org.il
Ubicaciones de estudio
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Jerusalem, Israel, 91031
- Shaare Zedek Medical Centre
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Niño
Acepta Voluntarios Saludables
Descripción
Inclusion Criteria:
- Infants and children < 12 years old with an anastomotic esophageal stricture following repair of congenital esophageal atresia / tracheoesophageal fistula
- Dysphagia score ≥1
- Parental consent
Exclusion Criteria:
- Additional stricture/s besides anastomotic stricture eg. Congenital stricture
- Complicated stricture - including length >2cm, significant angulation or irregularity
- Neurological impairment or other cause of expected limitation to oral intake
- Previous anastomotic dilatations
- Complicated surgical course including need for re-operation, re-fistularization or surgical anastomotic leak
- Suspected or verified thoracic vascular anomaly affecting esophageal patency or function
- Verified co-diagnosis of eosinophilic esophagitis
- Any contraindication to EBD according to treating physician's planned treatment course
Plan de estudios
¿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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Experimental: Budesonide arm
Patients will take budesonide gel (1mg/8mL) 250mcg orally twice daily for 12 weeks from the first dilatation.
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Patients will receive budesonide gel (1mg/8mL) 250mcg orally twice daily for 12 weeks from the first dilatation.
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Sin intervención: Control arm
Patients will not receive any treatment following their dilations.
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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The number of EBD procedures required to achieve clinical success.
Periodo de tiempo: From the first dilation up to 6 months after the initial dilation.
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The number of endoscopic balloon dilatation (EBD) procedures required to achieve clinical success (defined as effective swallowing without dysphagia or dietary restriction for 3 months) will be compared between the Budesonide and control groups.
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From the first dilation up to 6 months after the initial dilation.
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Restenosis rate 6 months after last EBD performed
Periodo de tiempo: Up to 6 months after the last dilation.
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The rate of restenosis, defined clinically as a dysphagia score ≥1, will be compared between the Budesonide and control groups.
Restenosis will be assessed following confirmation of clinical success.
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Up to 6 months after the last dilation.
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Rate of refractory strictures
Periodo de tiempo: Up to 5 months from the initial dilatation, or until the 7th dilatation, whichever occurs first.
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The rate of refractory strictures will be compared between the Budesonide and control groups.
A stricture is considered refractory when the age-appropriate esophageal lumen is not achieved after five dilatations within 5 months, or after ≥7 dilatations in total.
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Up to 5 months from the initial dilatation, or until the 7th dilatation, whichever occurs first.
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Patient Weight
Periodo de tiempo: Up to 6 months after the last dilation.
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Weight gain trajectories will be compared between the Budesonide arm and the control arm to evaluate whether improved stricture resolution and swallowing function contribute to improved growth outcomes.
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Up to 6 months after the last dilation.
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Dysphagia score
Periodo de tiempo: From baseline until 6 months following the last dilation.
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Dysphagia scores (modified dysphagia score and visual analogue scale) will be recorded at all scheduled follow-up visits to assess changes in swallowing function over time.
Scores will be compared between the Budesonide and control groups to evaluate whether the intervention results in reduced severity of dysphagia.
The modified dysphagia score, based on the Szegedi et al. scale, ranges from 0 (no swallowing problems) to 6 (aphagia), with increasing scores reflecting greater severity of swallowing impairment, from food avoidance or occasional difficulty to difficulty with semisolids, liquids, or complete inability to swallow.
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From baseline until 6 months following the last dilation.
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Pre-dilatation stricture diameter at each procedure
Periodo de tiempo: From baseline until final dilation, on average 6 months following the first.
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The esophageal stricture diameter immediately prior to balloon dilation will be recorded at each EBD procedure.
Serial measurements will be analyzed to compare stricture remodeling between the Budesonide and control arms, assessing whether the intervention is associated with greater pre-dilatation lumen size over time or reduced re-narrowing between procedures.
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From baseline until final dilation, on average 6 months following the first.
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Procedure complication rate
Periodo de tiempo: From the first dilation through 6 months after the final dilation.
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The rate of procedure and treatment related complications, including iatrogenic esophageal perforation, evidence of cortisol suppression, and oral or esophageal candidiasis, will be assessed to evaluate the safety of Budesonide compared with standard care.
Complications will be identified during each EBD procedure and at all clinical follow-up visits.
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From the first dilation through 6 months after the final dilation.
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Colaboradores e Investigadores
Patrocinador
Investigadores
- Investigador principal: Oren Leder, MD, Shaare Zedek Medical Center
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Estimado)
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
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Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- Budesonide following EBD
Plan de datos de participantes individuales (IPD)
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Información sobre medicamentos y dispositivos, documentos del estudio
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