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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Molecular Characterization for Understanding Biliary Atresia
- ClinicalTrials.gov ID
- NCT04272515
- Acronym / sponsor ID
- CAVB / C19-36
- Conditions
- Biliary Atresia
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Institut National de la Santé Et de la Recherche Médicale, FranceOther government
- Study start
- 7 Feb 2021 (actual)
- Primary completion
- 6 Feb 2031 (estimated)
- Study completion
- 7 Feb 2031 (estimated)
- First posted
- 17 Feb 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- BA patients and their parents will be enrolled in this study.
| Arm | Type | Receives |
|---|---|---|
| BA patients and their parents | Experimental | Other: blood sampling, Other: skin biopsy sampling, Other: explanted liver of BA patients sampling* Collection of blood samples from BA patients and their parents * Collection of explanted liver tissue and skin biopsy of BA patients |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- confirmed diagnosis of biliary atresia in patients
- parents of BA patients
Key exclusion criteria (1 listed)
- no
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| blood samplingcollection of blood sample for preparation of DNA | Other | BA patients and their parents |
| skin biopsy samplingpreparation of primary cultures of dermal fibroblasts from skin biopsy sample | Other | BA patients and their parents |
| explanted liver of BA patients samplingcryoconservation of liver tissue for molecular analyses | Other | BA patients and their parents |
Primary outcome
To identify the molecular mechanisms implicated in the etiology of BA
Time frame: 10 Years
To identify gene(s) and cellular pathways affected in cells and liver tissue of BA patients: sequencing experiments
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 22 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To identify the molecular mechanisms implicated in the etiology of BA (time frame: 10 Years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2026.