Disease · MONDO_0008867
biliary atresia
6 sourcesdiseases-2026-09-18-0.1.0
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ConductScience Library. biliary atresia. Diseases, record MONDO_0008867. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0008867
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Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Other names
Atresia, Biliary, Biliary Atresia, Extrahepatic, Biliary duct atresia, Familial Extrahepatic Biliary Atresia, Idiopathic Extrahepatic Biliary Atresia, atresia of bile ducts, biliary atresia, congenital, congenital biliary atresia, isolated atresia of bile ducts, non-syndromic biliary atresia
- Open Targets
Drugs19
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| ODEVIXIBAT | CHEMBL4297588 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| PREDNISOLONE | CHEMBL131 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| OBETICHOLIC ACID | CHEMBL566315 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| FILGRASTIM | CHEMBL1201567 | Protein | Phase 2 | ClinicalTrials.gov | 3 | 3 | 1 | Drugs and targets |
| ACETYLCYSTEINE | CHEMBL600 | Small molecule | Phase 2 | ClinicalTrials.gov | 2 | 2 | 1 | Drugs and targets |
| MARALIXIBAT | CHEMBL363392 | Small molecule | Phase 2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| PENTOXIFYLLINE | CHEMBL628 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| HUMAN IMMUNOGLOBULIN G | CHEMBL1201599 | Phase 1/2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets | |
| VANCOMYCIN | CHEMBL262777 | Small molecule | Phase 1 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| LENOGRASTIM | CHEMBL2109128 | Protein | Early phase 1 | ClinicalTrials.gov | 2 | 2 | 0 | Drugs and targets |
1 to 10 of 19
See all 19 drugsTargets29
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| NR3C1 | Glucocorticoid receptor | 8 | Glucocorticoid receptor agonist | Phase 3 | https://conductscience.org/library/genes/2908 | Drugs and targets |
| CSF3R | Granulocyte colony-stimulating factor receptor | 3 | Granulocyte colony stimulating factor receptor agonist | Phase 2 | https://conductscience.org/library/genes/1441 | Drugs and targets |
| SLC10A2 | Ileal sodium/bile acid cotransporter | 2 | Ileal bile acid transporter inhibitor | Phase 3 | https://conductscience.org/library/genes/6555 | Drugs and targets |
| ADORA2A | Adenosine A2 receptor | 1 | Adenosine A2 receptor antagonist | Phase 2 | https://conductscience.org/library/genes/135 | Drugs and targets |
| ADORA2B | Adenosine A2 receptor | 1 | Adenosine A2 receptor antagonist | Phase 2 | https://conductscience.org/library/genes/136 | Drugs and targets |
| NR1H4 | Bile acid receptor | 1 | Bile acid receptor FXR agonist | Phase 2/3 | https://conductscience.org/library/genes/9971 | Drugs and targets |
| PDE10A | 3',5'-cyclic phosphodiesterase | 1 | 3',5'-cyclic phosphodiesterase inhibitor | Phase 2 | https://conductscience.org/library/genes/10846 | Drugs and targets |
| PDE1A | 3',5'-cyclic phosphodiesterase | 1 | 3',5'-cyclic phosphodiesterase inhibitor | Phase 2 | https://conductscience.org/library/genes/5136 | Drugs and targets |
| PDE1B | 3',5'-cyclic phosphodiesterase | 1 | 3',5'-cyclic phosphodiesterase inhibitor | Phase 2 | https://conductscience.org/library/genes/5153 | Drugs and targets |
| PDE1C | 3',5'-cyclic phosphodiesterase | 1 | 3',5'-cyclic phosphodiesterase inhibitor | Phase 2 | https://conductscience.org/library/genes/5137 | Drugs and targets |
1 to 10 of 29
See all 29 targetsTrials13
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 5 more are not shown, since the checks do not confirm them.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT00294684 | A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy | Not applicable | 141 | 2017-05-05 | Biliary Atresia | METHYLPREDNISOLONE, PREDNISOLONE | Its record names the disease | ClinicalTrials.gov |
| NCT04524390 | Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai | Phase 2 | 75 | 2025-02-17 | Biliary Atresia | MARALIXIBAT | Its record names the disease | ClinicalTrials.gov |
| NCT04168385 | MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study | Phase 2 | 52 | 2025-12-08 | Cholestatic Liver Disease | MARALIXIBAT | Its record names the disease | ClinicalTrials.gov |
| NCT01322386 | Gastrointestinal Microbiota in Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin | Phase 1 | 32 | 2016-03-04 | Primary Sclerosing Cholangitis, Biliary Atresia | VANCOMYCIN | Its record names the disease | ClinicalTrials.gov |
| NCT01774487 | Pentoxifylline Therapy in Biliary Atresia | Phase 2 | 17 | 2023-10-12 | Biliary Atresia | PENTOXIFYLLINE | Its record names the disease | ClinicalTrials.gov |
| NCT03499249 | N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy | Phase 2 | 13 | 2024-01-10 | Biliary Atresia | ACETYLCYSTEINE | Its record names the disease | ClinicalTrials.gov |
| NCT04373941 | Part II: Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia | Phase 2 | 400 | Biliary Atresia | FILGRASTIM | Its record names the disease | ClinicalTrials.gov | |
| NCT04336722 | Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) | Phase 3 | 254 | Biliary Atresia | ODEVIXIBAT | Its record names the disease | ClinicalTrials.gov | |
| NCT02137668 | Treating Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin | Phase 1 | 200 | Primary Sclerosing Cholangitis, Biliary Atresia | VANCOMYCIN | Its record names the disease | ClinicalTrials.gov | |
| NCT05426733 | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia | Phase 3 | 180 | Biliary Atresia | ODEVIXIBAT | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 13
See all 13 trialsComparisons in its trials15
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Corticosteroids vs Placebo: serious adverse events | 57 of 70 | 56 of 70 | 140 | NCT00294684 | Trial comparisons |
| Corticosteroids vs Placebo: Presence of Ascites at 12 Months | 5 of 52 | 3 of 47 | 99 | NCT00294684 | Trial comparisons |
| Corticosteroids vs Placebo: Presence of Ascites at 24 Months | 1 of 42 | 3 of 43 | 85 | NCT00294684 | Trial comparisons |
Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants Observed to Develop Clinically Evident Portal Hypertension Defined as Splenomegaly and Thrombocytopenia (Platelet Count <150 x 109/L) or Clinically Evident Ascites or Endoscopic Evidence of Esophageal or Gastric Varices. | 3 of 40 | 4 of 35 | 75 | NCT04524390 | Trial comparisons |
| Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26 | 24 of 40 | 20 of 35 | 75 | NCT04524390 | Trial comparisons |
Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death. | 8 of 40 | 7 of 35 | 75 | NCT04524390 | Trial comparisons |
| Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL | 23 of 40 | 18 of 35 | 75 | NCT04524390 | Trial comparisons |
| Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants With Mean sBA Levels ≤40 mmol/L | 10 of 40 | 7 of 35 | 75 | NCT04524390 | Trial comparisons |
| Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants Undergoing Liver Transplantation or Death | 5 of 40 | 3 of 35 | 75 | NCT04524390 | Trial comparisons |
| Maralixibat: ALGS vs Maralixibat: PFIC: deaths from any cause | 1 of 40 | 0 of 12 | 52 | NCT04168385 | Trial comparisons |
1 to 10 of 15
See all 15 comparisons in its trialsPapers from its trials10
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0008867
GET https://conductscience.org/api/diseases/v1/MONDO_0008867/targets?page=2