Biliary AtresiaCAVBNCT04272515
Molecular Characterization for Understanding Biliary Atresia
A study testing blood sampling, skin biopsy sampling and explanted liver of BA patients sampling in people with Biliary Atresia, run by Institut National de la Santé Et de la Recherche Médicale, France at 2 sites in France.
Trial summary
Although considered a rare disease, Biliary Atresia (BA) is the leading cause of neonatal cholestasis and liver transplantation in children. Little is known about the molecular mechanisms that drive BA. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Biliary Atresia
- What is being tested?
- blood sampling, skin biopsy sampling and explanted liver of BA patients sampling
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 2 sitesFrance
- Study dates
- 7 Feb 2021to 7 Feb 2031
- Phase
- Interventional
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- confirmed diagnosis of biliary atresia in patients
- parents of BA patients
Who cannot join
- no
Trial locations and contacts
2 sites in 1 country.
France2 sites
Hopital Necker enfants malades
Paris, De, 75015
PRC Inserm
Paris, 75013
About this study
Although considered a rare disease, Biliary Atresia (BA) is the leading cause of neonatal cholestasis and liver transplantation in children. Little is known about the molecular mechanisms that drive BA. The purpose of this study is to collect the fluid samples, explanted liver tissue samples and dermal biopsy samples to enable investigators to perform the genetic and molecular analyses that might point to the gene(s) and cellular pathway involved in etiology of BA disease.
Read the detailed description
Biliary atresia (BA) is a disease characterized by intra- and extra-hepatic bile duct obstruction diagnosed in the neonatal period. If left untreated, this obstruction leads to biliary cirrhosis and early death. Although considered a rare disease (between 1/15,000 and 1/20000 births), it is the leading cause of neonatal cholestasis and liver transplantation in children. The reasons for this obstruction are still poorly known and might involve several factors (immune, infectious and possible toxin effect). The accumulating evidence point to genetic factors involved, yet they are not of the classic monogenic or Mendelian types. The purpose of this study is to collect the fluid samples, explanted liver tissue samples and dermal biopsy samples to enable investigators to perform the genetic and molecular analyses that might point to the gene(s) and cellular pathway involved in etiology of BA disease.
- Conditions
- Biliary Atresia
- Being tested
- blood sampling, skin biopsy sampling, explanted liver of BA patients sampling
- Medical subject headings
- Biliary Atresia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
blood samplingOther
collection of blood sample for preparation of DNA
skin biopsy samplingOther
preparation of primary cultures of dermal fibroblasts from skin biopsy sample
explanted liver of BA patients samplingOther
cryoconservation of liver tissue for molecular analyses
| Study group | Role | What they receive |
|---|---|---|
| BA patients and their parents | Experimental | * Collection of blood samples from BA patients and their parents
* Collection of explanted liver tissue and skin biopsy of BA patients Receives: Other: blood sampling; Other: skin biopsy sampling; Other: explanted liver of BA patients sampling |
What is being measured
The primary outcome is the main question the study is designed to answer.
To identify the molecular mechanisms implicated in the etiology of BA
To identify gene(s) and cellular pathways affected in cells and liver tissue of BA patients: sequencing experiments
Measured: 10 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2020
- Study start7 Feb 2021
- Primary completion6 Feb 2031 (estimated)
- Study completion7 Feb 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Biliary AtresiaStudies of the same condition, open and under way.
- More trials run by Institut National de la Santé Et de la Recherche Médicale, FranceThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- BA patients and their parents will be enrolled in this study.
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04272515
- Sponsor's protocol ID
- C19-36
- Acronym
- CAVB
- Status last verified
- Sep 2026
- First submitted
- 28 Jan 2020
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No