BILACO Trial: Biliary Atresia - a Severe Complex Congenital Liver Disease

NCT ID: NCT05399745

Last Updated: 2022-06-01

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 100 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2020-03-01
• Study Completion Date: 2040-12-31

Brief Summary

Biliary atresia is the most severe form of cholestatic liver disease. The children have high morbidity and mortality and get devastating pruritus and fatigue, failure to thrive, progressive hepatic failure and impaired neurodevelopment. The etiology is mostly unknown. More than half need a new liver from a living or deceased donor during childhood. However, correct timing of the transplantation is extremely difficult because of lack of consensus based on clinical assessment tools. All though the incidence is low, the cost of this disease is tremendous from both a clinical and human perspective. So far, protocolized neurodevelopment tests, genetic profiling, precise malnutrition evaluation based on clinical appearance, biochemical markers and brain MRI-scans, body composition, immunological function, level of physical activity and optimal time of transplantation in cholestatic children are unknown.

The aim is to determine risk factors for neurocognitive impairment in children suffering from severe cholestasis in order to determine optimal time for liver transplantation from a brain perspective.

In a prospective study, the investigators will investigate risk factors related to brain-, heart-, gut- and immunological function in the Danish cohort. This cohort consists of 75 children aged 0-18 years. In addition, 30 aged and gender matched healthy and 20 tetra fallot children will serve as control groups. The children will undergo extensive and advanced liver function evaluation, genetic profiling, nutrition and immunological status, neuro-imaging and neurocognitive evaluation at time of diagnose, 2 years of age, pre-school, pre-teenage, and teenage. In case of a liver transplantation, additional neuro-cognitive tests will be performed

Conditions

Biliary Atresia; Cognitive Impairment

Study Design

• Observational Model Type: CASE_CONTROL
• Study Time Perspective: PROSPECTIVE

Study Groups

Children with biliary atresia

Neurocognitive monitoring

Intervention Type: OTHER

Neurocognitive tests and MRI of the brain

Children with Tetralogy of Fallot

Neurocognitive monitoring

Intervention Type: OTHER

Neurocognitive tests and MRI of the brain

Healthy control children

Neurocognitive monitoring

Intervention Type: OTHER

Neurocognitive tests and MRI of the brain

Interventions

Neurocognitive monitoring

Neurocognitive tests and MRI of the brain

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• Biliary atresia
• Tetralogy of Fallot
• Healthy controls

Exclusion Criteria

• Not able to participate in exams

• Minimum Eligible Age: 0 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Rigshospitalet, Denmark

OTHER

Sponsor Role: lead

Responsible Party

Vibeke Brix Christensen

Principal investigator, MD, PhD, DMSc, senior consultant

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Vibeke Brix Christensen, MD, PhD, DMSc

Role: PRINCIPAL_INVESTIGATOR

Rigshospitalet, Denmark

Locations

Rigshospitalet

Copenhagen, , Denmark

Site Status: RECRUITING

Countries

Denmark

Central Contacts

Vibeke Brix Christensen, MD, PhD, DMSc

Role: CONTACT

vibeke.brix.christensen@regionh.dk

+4535458842

Facility Contacts

Vibeke Brix Christensen, MD, PhD, DMSc

Role: primary

vibeke.brix.christensen@regionh.dk

+4535458842

Other Identifiers

H-19042934

Identifier Type: -

Identifier Source: org_study_id