NCT04494763

Primary Prophylaxis for Variceal Bleed in Biliary Atresia

Propanolol for Primary Prophylaxis for Variceal Bleed in Biliary Atresia - An Open Label Randomized Controlled Study

• Status: Unknown
• Phase: N/A
• Study type: Interventional
• Enrollment: 92 (estimated)
• Sponsor: Institute of Liver and Biliary Sciences, India · Academic / Other
• Sex: All
• Age: 6 Months – 5 Years
• Healthy volunteers: Not accepted

Summary

Biliary atresia is the commonest etiology of neonatal cholestasis and is the most common indication for pediatric liver transplantation world-wide. Kasai-portoenterostomy (KPE) is the operative procedure of choice which helps in restoration of biliary flow and preventing rapid progression of fibrosis. Only 50-60% of infants have a successful surgery in terms of normalization of bilirubin (\<2 mg/dL) after 3 months. Remaining 40-50% have rapid progression of PHT and eventual decompensation. Additionally, around 50-70% of infants with successful KPE have 1 or more episodes of cholangitis, and the severe ones if left untreated lead to progressive portal hypertension. Moreover in Indian setting a significant number of infants with biliary atresia reach late when the KPE is not feasible, and this group develops very rapid PHT and decompensation. Hence, overall around 70-80% of infants and children develop PHT within 5 years of age. Variceal bleed has been shown to be an important determinant of survival in infants with high bilirubin. Usage of beta-blockers in adult cirrhotics has been shown to reduce the progression of varices and incidence of variceal bleed. Although many pediatric hepatology centers worldwide use beta-blockers, there has been no controlled trial specifically to address this issue in children with biliary atresia. So, we planned this study to evaluate the efficacy of beta-blockers as primary prophylaxis for prevention of variceal bleed in biliary atresia children.

Detailed description

(a) Aim and Objectives: Aim: To study the effect of beta-blockers for primary prophylaxis of variceal bleed in infants and children with biliary atresia. Primary objective: Bleeding free survival over 18 months FU Secondary objectives: (i) Progression, persistence or regression of esophageal and gastric varices, and portal hypertensive gastropathy over 3, 6, 12 and 18 months FU (ii) Overall survival at 18 months FU (b) Methodology: This will be an open label randomized controlled study where infants and children with biliary atresia from 6 months to 5 years fulfilling inclusion and exclusion criteria will be enrolled to receive propanolol or placebo for a duration of 18 months or till the occurrence of variceal bleed. i. Study design: Open label randomized controlled study with stratified randomization. ii. Intervention: Beta-blocker (Propanolol) versus placebo. iii. Study period: 1.5 years iv. Study population: Infants and children from 6 months to 5 years of age with Biliary atresia fulfilling the inclusion and exclusion criteria. (c) Expected outcome of the project: Beta-blockers reduce the incidence of variceal bleed in infants and children with biliary atresia.

Conditions

• Portal Hypertension, Biliary Atresia

Interventions

Type	Name	Description
DRUG	Propanolol	Non-selective Beta-blocker
OTHER	Placebo	Inactive drug

Timeline

• Start date: 2020-08-15
• Primary completion: 2022-06-13
• Completion: 2022-06-15
• First posted: 2020-07-31
• Last updated: 2021-03-18

Locations

1 site across 1 country: India