Biliary atresia

Summary

Biliary atresia (MONDO:0008867) is a disease with 4 cohort genes (41 GWAS associations across 7 studies) and 62 clinical trials. Top therapeutic interventions include desflurane, indocyanine green acid form, and odevixibat.

At a glance

• Prevalence: 1-9 / 100 000 (Europe) [Orphanet-validated]
• Cohort genes: 4
• GWAS associations: 41
• Phenotypes (HPO): 28
• Clinical trials: 62

Clinical features

Epidemiology

Prevalence records

18 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Prevalence at birth	1-5 / 10 000	18.5	Worldwide	Validated
Point prevalence	1-9 / 100 000		Europe	Validated
Prevalence at birth	1-9 / 100 000	2.9	Europe	Validated
Point prevalence	1-5 / 10 000	10.6	Specific population	Validated
Prevalence at birth	1-9 / 100 000	2.9	Belgium	Validated
Prevalence at birth	1-9 / 100 000	5.9	France	Validated
Prevalence at birth	1-5 / 10 000	11.9	Germany	Validated
Prevalence at birth	1-9 / 100 000	4.2	Italy	Validated
Prevalence at birth	1-5 / 10 000	16.6	Netherlands	Validated
Prevalence at birth	1-9 / 100 000	1.6	Norway	Validated
Prevalence at birth	1-9 / 100 000	1.9	Spain	Validated
Prevalence at birth	1-5 / 10 000	12.6	Switzerland	Validated
Prevalence at birth	1-9 / 100 000	2.3	United Kingdom	Validated
Prevalence at birth	1-5 / 10 000	12.7	Ukraine	Validated
Prevalence at birth	1-9 / 100 000	7.4	Japan	Validated
Prevalence at birth	1-5 / 10 000	32	French Polynesia	Validated
Prevalence at birth	1-9 / 100 000	7	Australia	Validated
Prevalence at birth	1-9 / 100 000	7	United States	Not yet validated

Signs & symptoms

Clinical features (HPO)

28 HPO clinical features (Orphanet curated; top 28 by frequency):

HPO ID	Term	Frequency
HP:0000952	Jaundice	Very frequent (80-99%)
HP:0001396	Cholestasis	Very frequent (80-99%)
HP:0001508	Failure to thrive	Very frequent (80-99%)
HP:0001410	Decreased liver function	Frequent (30-79%)
HP:0001525	Severe failure to thrive	Frequent (30-79%)
HP:0002240	Hepatomegaly	Frequent (30-79%)
HP:0002630	Fat malabsorption	Frequent (30-79%)
HP:0002908	Conjugated hyperbilirubinemia	Frequent (30-79%)
HP:0002910	Elevated circulating hepatic transaminase concentration	Frequent (30-79%)
HP:0003155	Elevated circulating alkaline phosphatase concentration	Frequent (30-79%)
HP:0006579	Prolonged neonatal jaundice	Frequent (30-79%)
HP:0008151	Prolonged prothrombin time	Frequent (30-79%)
HP:0011984	Atretic gallbladder	Frequent (30-79%)
HP:0011985	Acholic stools	Frequent (30-79%)
HP:0030948	Elevated gamma-glutamyltransferase level	Frequent (30-79%)
HP:0040321	Dark yellow urine	Frequent (30-79%)
HP:0000602	Ophthalmoplegia	Occasional (5-29%)
HP:0000821	Hypothyroidism	Occasional (5-29%)
HP:0000989	Pruritus	Occasional (5-29%)
HP:0001250	Seizure	Occasional (5-29%)
HP:0001394	Cirrhosis	Occasional (5-29%)
HP:0001405	Periportal fibrosis	Occasional (5-29%)
HP:0001408	Bile duct proliferation	Occasional (5-29%)
HP:0001518	Small for gestational age	Occasional (5-29%)
HP:0001744	Splenomegaly	Occasional (5-29%)
HP:0001999	Abnormal facial shape	Occasional (5-29%)
HP:0040075	Hypopituitarism	Occasional (5-29%)
HP:0001114	Xanthelasma	Very rare (<1-4%)

Identifiers

Disease identifiers

Field	Value
Canonical name	biliary atresia
Mondo ID	MONDO:0008867
MeSH	D001656
Orphanet	30391
DOID	DOID:13608
ICD-10-CM	Q44.2
ICD-11	645741117
NCIT	C34421
SNOMED CT	77480004
UMLS	C0005411
MedGen	14117
GARD	0012010
MedDRA	10003650
NORD	1114
Is cancer (heuristic)	no

Also known as: atresia of bile ducts · biliary atresia, congenital · congenital biliary atresia · isolated atresia of bile ducts · non-syndromic biliary atresia

Data availability: 41 GWAS associations (7 studies) · 11 cell lines.

Disease family

An umbrella term covering 3 Mondo subtypes.

Classification path: disease › human disease › disease by body system or component › digestive system disorder › hepatobiliary disorder › biliary tract disorder › bile duct disorder › cholestasis › biliary atresia

Related subtypes (4): extrahepatic cholestasis, obstructive jaundice, intrahepatic cholestasis, parenteral nutrition-associated cholestasis

Subtypes (3): biliary atresia intrahepatic non syndromic form, biliary atresia intrahepatic syndromic form, extrahepatic biliary atresia

Genetics & variants

GWAS landscape

41 GWAS associations across 7 studies. Top hits map to 29 distinct genes (as reported by GWAS).

Top associations by p-value

rsID	p-value	Gene	Risk allele	Odds ratio
rs17095355	5e-11	ADD3-AS1	?	1.7
rs10884919	6e-09	ADD3, ADD3-AS1	?	1.6
rs2083872	7e-09	LINC00705, MANCR	T	3.41
rs114118902	7e-09	SCN1A-AS1, SCN9A	G	3.66
rs6761893	3e-08	EFEMP1	T	1.54
rs6446628	4e-08	AFAP1	A	1.41
rs12964783	6e-08	KC6	A	0.65
rs34599046	1e-07	TUSC3	G	1.4
rs7785003	2e-07	UMAD1	A	0.61
rs111814934	3e-07	GLIS3	C	1.97
rs10111159	5e-07	RP1	T	2.69
rs79722046	8e-07	RPL7AP40 - AP1S2P1	T	1.87
rs79812751	1e-06	MAP1LC3C - TUBB8P6	T	2.11
rs9314986	2e-06	LINC00297	G	2.95
rs11111419	2e-06	PAH	T	1.34
rs7255262	2e-06	VAV1 - ADGRE1	C	0.57
rs1349341	3e-06	MTHFD2P4 - TOMM22P4	G	1.42
rs12532246	3e-06	RNF32-DT	A	1.34
rs115074361	3e-06	AACSP1 - ZNF354B	A	3
rs3733850	3e-06	CSNK1A1	C	2.62
rs111550434	3e-06	ANKFN1	C	2.41
rs707936	3e-06	VWA7	?
rs356287	4e-06	COX6A1P1 - LINC01781	G	1.48
rs12023563	4e-06	SERTAD4 - ST13P19	G	1.59
rs371776	4e-06	GDNF-AS1	G	0.75
rs7861188	4e-06	PTPRD-DT	A	1.42
rs1299828729	4e-06	NKAIN2	CTGTG	1.32
rs7099863	4e-06	GPAM - TECTB	C	0.74
rs854132	5e-06	LNCBRM	A	1.35
rs112220674	5e-06	RAB20	T	0.53

Top studies (by case count)

Study	Lead author	Year	Cases	Controls	Title
GCST90296484	Glessner JT	2023	811	4,654	Biliary atresia is associated with polygenic susceptibility in ciliogenesis and planar polarity effector genes.
GCST006390	Chen Y	2018	343	1,716	A genome-wide association study identifies a susceptibility locus for biliary atresia on 2p16.1 within the gene EFEMP1.
GCST90428734	Cui MM	2023	317	8,843	Contribution of ADD3 and the HLA Genes to Biliary Atresia Risk in Chinese.
GCST000681	Garcia-Barcelo MM	2010	281	0	Genome-wide association study identifies a susceptibility locus for biliary atresia on 10q24.2.
GCST90428736	Cui MM	2023	141	8,843	Contribution of ADD3 and the HLA Genes to Biliary Atresia Risk in Chinese.
GCST90428737	Cui MM	2023	141	0	Contribution of ADD3 and the HLA Genes to Biliary Atresia Risk in Chinese.
GCST90428735	Cui MM	2023	87	8,843	Contribution of ADD3 and the HLA Genes to Biliary Atresia Risk in Chinese.

Variant details and genetic-evidence tiers

Tier distribution (top 50 variants)

Tier	Variants
Tier 1: coding	0
Tier 2: splice/UTR	0
Tier 3: regulatory	1
Tier 4: intronic/intergenic	39

MAF distribution

Bucket	Variants
common (>=0.05)	33
low_freq (0.01-0.05)	1
rare (<0.01)	0
unknown	6

Functional consequences

Consequence	Count
intron_variant	32
intergenic_variant	7
regulatory_region_variant	1

Top variants

rsID	Chr	Pos	Alleles	MAF	Consequence	Gene	p-value	Tier
rs17095355	10	109975992	C>T	0.05	intron_variant	ADD3-AS1	5e-11	Tier 4: intronic/intergenic
rs10884919	10	109998033	G>A	0.05	intron_variant	ADD3, ADD3-AS1	6e-09	Tier 4: intronic/intergenic
rs2083872	10	4676262	C>T	0.05	intron_variant	LINC00705, MANCR	7e-09	Tier 4: intronic/intergenic
rs114118902	2	166325739	A>G		intron_variant	SCN1A-AS1, SCN9A	7e-09	Tier 4: intronic/intergenic
rs6761893	2	55891970	A>C,G,T	0.34	intron_variant	EFEMP1	3e-08	Tier 4: intronic/intergenic
rs6446628	4	7830208	A>C	0.05	intron_variant	AFAP1	4e-08	Tier 4: intronic/intergenic
rs12964783	18	41463791	C>A	0.05	intron_variant	KC6	6e-08	Tier 4: intronic/intergenic
rs34599046	8	15687363	C>A,G	0.05	intron_variant	TUSC3	1e-07	Tier 4: intronic/intergenic
rs7785003	7	7823672	G>A,T	0.05	intron_variant	UMAD1	2e-07	Tier 4: intronic/intergenic
rs111814934	9	4132045	T>C,G	0.05	intron_variant	GLIS3	3e-07	Tier 4: intronic/intergenic
rs10111159	8	54826227	A>T	0.05	intron_variant	RP1	5e-07	Tier 4: intronic/intergenic
rs79722046	7	97339055	A>T	0.05	intron_variant	RPL7AP40 - AP1S2P1	8e-07	Tier 4: intronic/intergenic
rs79812751	1	242029258	C>T	0.05	intron_variant	MAP1LC3C - TUBB8P6	1e-06	Tier 4: intronic/intergenic
rs9314986	13	29884600	G>A,C,T	0.044	intron_variant	LINC00297	2e-06	Tier 4: intronic/intergenic
rs11111419	12	102891265	A>T	0.05	intron_variant	PAH	2e-06	Tier 4: intronic/intergenic
rs7255262	19	6872970	T>C,G	0.05	intergenic_variant	VAV1 - ADGRE1	2e-06	Tier 4: intronic/intergenic
rs1349341	4	162359753	G>A,C,T	0.05	intergenic_variant	MTHFD2P4 - TOMM22P4	3e-06	Tier 4: intronic/intergenic
rs12532246	7	156483612	A>G	0.05	intron_variant	RNF32-DT	3e-06	Tier 4: intronic/intergenic
rs115074361	5	178830323	T>A		intergenic_variant	AACSP1 - ZNF354B	3e-06	Tier 4: intronic/intergenic
rs3733850	5	149520216	T>C	0.05	intron_variant	CSNK1A1	3e-06	Tier 4: intronic/intergenic
rs111550434	17	56310656	T>C	0.05	intron_variant	ANKFN1	3e-06	Tier 4: intronic/intergenic
rs707936	6	31765873	G>A,C	0.05	intron_variant	VWA7	3e-06	Tier 4: intronic/intergenic
rs356287	1	80512278	A>G	0.36	intergenic_variant	COX6A1P1 - LINC01781	4e-06	Tier 4: intronic/intergenic
rs12023563	1	210262765	G>A	0.32	regulatory_region_variant	SERTAD4 - ST13P19	4e-06	Tier 3: regulatory
rs371776	5	38020688	G>A,C,T	0.05	intron_variant	GDNF-AS1	4e-06	Tier 4: intronic/intergenic
rs7861188	9	10614869	A>C,G,T	0.05	intron_variant	PTPRD-DT	4e-06	Tier 4: intronic/intergenic
rs1299828729	6	123833691	C>G		intron_variant	NKAIN2	4e-06	Tier 4: intronic/intergenic
rs7099863	10	112254084	T>A,C	0.05	intron_variant	GPAM - TECTB	4e-06	Tier 4: intronic/intergenic
rs854132	5	57622558	T>A,C	0.05	intron_variant	LNCBRM	5e-06	Tier 4: intronic/intergenic
rs112220674	13	110535030	C>T	0.05	intron_variant	RAB20	5e-06	Tier 4: intronic/intergenic

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 2 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
ADD3	Orphanet:210141	Inherited congenital spastic tetraplegia
HOXC13	Orphanet:69084	Pure hair and nail ectodermal dysplasia

Cohort genes → proteins

4 cohort genes, 4 distinct canonical proteins.

Evidence partition

Subset	Genes
gwas_only	4

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
UBL3	HGNC:12504	ENSG00000122042	O95164	Ubiquitin-like protein 3	gwas
XPNPEP1	HGNC:12822	ENSG00000108039	Q9NQW7	Xaa-Pro aminopeptidase 1	gwas
ADD3	HGNC:245	ENSG00000148700	Q9UEY8	Gamma-adducin	gwas
HOXC13	HGNC:5125	ENSG00000123364	P31276	Homeobox protein Hox-C13	gwas

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
XPNPEP1	Xaa-Pro aminopeptidase 1	Metalloaminopeptidase that catalyzes the removal of a penultimate prolyl residue from the N-termini of peptides, such as Arg-Pro-Pro.
ADD3	Gamma-adducin	Membrane-cytoskeleton-associated protein that promotes the assembly of the spectrin-actin network.
HOXC13	Homeobox protein Hox-C13	Transcription factor which plays a role in hair follicle differentiation.

Protein-family classification

Druggable: 1 · Difficult: 1 · Unknown: 2 · Druggable fraction: 0.25

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Protease	1	9.2×	0.315
Transcription factor	1	2.1×	0.605
Other/Unknown	2	0.9×	0.769

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
UBL3	Other/Unknown	no		Ubiquitin-like_dom, MUB, Ubiquitin-like_domsf
XPNPEP1	Protease	yes	3.1.8.1	Creatinase_N, Pept_M24, Peptidase_M24B_aminopep-P_CS
ADD3	Other/Unknown	no		Aldolase_II/adducin_N, Aldolase_II/adducin_N_sf, Aldolase-II_Adducin_sf
HOXC13	Transcription factor	no		HD, Homeodomain-like_sf, Homeobox_CS

Expression context

Cohort genes with no expression data: 0.

3 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	4
unknown	0

Top tissues across cohort

Tissue	Cohort genes
dorsal motor nucleus of vagus nerve	2
amniotic fluid	1
inferior olivary complex	1
body of pancreas	1
cervix squamous epithelium	1
jejunal mucosa	1
oocyte	1
secondary oocyte	1
hair follicle	1
olfactory bulb	1
type B pancreatic cell	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
UBL3	295	ubiquitous	marker	inferior olivary complex, amniotic fluid, dorsal motor nucleus of vagus nerve
XPNPEP1	294	ubiquitous	marker	body of pancreas, jejunal mucosa, cervix squamous epithelium
ADD3	303	ubiquitous	marker	secondary oocyte, oocyte, dorsal motor nucleus of vagus nerve
HOXC13	33	broad	yes	hair follicle, type B pancreatic cell, olfactory bulb

Protein interactions among cohort

Intra-cohort edges: 1.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
XPNPEP1	2,169
ADD3	1,882
HOXC13	1,247
UBL3	745

Intra-cohort edges

A	B	Sources
ADD3	XPNPEP1	string_interaction

Structural data

PDB: 2 · AlphaFold-only: 2 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
UBL3	O95164	1
XPNPEP1	Q9NQW7	1

AlphaFold-only cohort genes (top 30 by pLDDT)

Symbol	UniProt	pLDDT
ADD3	Q9UEY8	66.83
HOXC13	P31276	58.40

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 8. Enrichment computed across 4 evidence-associated genes (1 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Miscellaneous transport and binding events	1	439.2×	0.013	ADD3
RHOF GTPase cycle	1	259.6×	0.013	ADD3
RHOD GTPase cycle	1	203.9×	0.013	ADD3
RHO GTPase cycle	1	60.1×	0.033	ADD3
Signaling by Rho GTPases	1	34.2×	0.040	ADD3
Signaling by Rho GTPases, Miro GTPases and RHOBTB3	1	33.5×	0.040	ADD3
Transport of small molecules	1	25.1×	0.045	ADD3
Signal Transduction	1	10.2×	0.098	ADD3

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
tongue morphogenesis	1	1123.5×	0.004	HOXC13
positive regulation of cytoskeleton organization	1	936.2×	0.004	ADD3
bradykinin catabolic process	1	802.5×	0.004	XPNPEP1
nail development	1	802.5×	0.004	HOXC13
barbed-end actin filament capping	1	267.5×	0.010	ADD3
negative regulation of programmed cell death	1	244.2×	0.010	XPNPEP1
positive regulation of vasoconstriction	1	200.6×	0.010	ADD3
hair follicle development	1	127.7×	0.014	HOXC13
anterior/posterior pattern specification	1	60.4×	0.026	HOXC13
anatomical structure morphogenesis	1	46.4×	0.030	HOXC13
response to xenobiotic stimulus	1	23.0×	0.054	ADD3
proteolysis	1	11.4×	0.099	XPNPEP1
positive regulation of DNA-templated transcription	1	9.3×	0.112	HOXC13
regulation of transcription by RNA polymerase II	1	3.9×	0.236	HOXC13

Therapeutics

Drugs indicated for this disease

0 approved, 2 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
Odevixibat	Phase 3 (in late-stage trials)
Prednisolone	Phase 3 (in late-stage trials)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Acetylcysteine, Filgrastim, Maralixibat, Pentoxifylline.

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 4

Druggability breadth: 1 of 4 evidence-associated genes (25%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

Symbol	Molecules	Max phase
UBL3	0	0
XPNPEP1	0	0
ADD3	0	0
HOXC13	0	0

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 1.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

Symbol	Assays	Type breakdown
XPNPEP1	10	Binding:9, ADMET:1

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
XPNPEP1	3.1.8.1, 3.4.11.9	aryldialkylphosphatase, Xaa-Pro aminopeptidase

Pharmacogenomics

Cohort genes with a PharmGKB record: 4; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	0
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	1	XPNPEP1
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	3	UBL3, ADD3, HOXC13

Undrugged target profiles

4 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Symbol	ChEMBL assays	Drugged partners (top 3)
UBL3	0	—
XPNPEP1	10	—
ADD3	0	—
HOXC13	0	—

Clinical trials & evidence

Clinical trials

Clinical trials: 62.

Phase distribution (across all retrieved trials)

Phase	Trials
Not specified	42
PHASE2	5
PHASE1	5
PHASE3	4
PHASE1/PHASE2	2
EARLY_PHASE1	2
PHASE4	1
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05783518	PHASE4	UNKNOWN	Effect of Desflurane on Pediatric Acute Respiratory Distress Syndrome After Living Donor Liver Transplant Recipients
NCT04336722	PHASE3	ACTIVE_NOT_RECRUITING	Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
NCT05426733	PHASE3	ENROLLING_BY_INVITATION	An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
NCT00539565	PHASE3	UNKNOWN	RCT of Steroids Following Kasai Portoenterostomy for Biliary Atresia.
NCT05521152	PHASE3	COMPLETED	Norepinephrine for Prevention of Intraoperative Hypotension in Infants Undergoing Kasai Portoenterostomy
NCT06121375	PHASE2/PHASE3	TERMINATED	Study to Assess Efficacy, Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Obeticholic Acid (OCA) Compared to Placebo in Pediatric Participants With Biliary Atresia, Post-hepatoportoenterostomy
NCT07513038	PHASE1/PHASE2	RECRUITING	Use of Indocyanine Green (ICG) for the Diagnosis of Biliary Atresia
NCT01774487	PHASE2	TERMINATED	Pentoxifylline Therapy in Biliary Atresia
NCT01854827	PHASE1/PHASE2	COMPLETED	Safety Study of Intravenous Immunoglobulin (IVIG) Post-Portoenterostomy in Infants With Biliary Atresia
NCT03499249	PHASE2	COMPLETED	N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy
NCT04373941	PHASE2	UNKNOWN	Part II: Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia
NCT04524390	PHASE2	COMPLETED	Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
NCT05321524	PHASE2	TERMINATED	Obeticholic Acid in Pediatric Subjects With Biliary Atresia
NCT02137668	PHASE1	RECRUITING	Treating Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin
NCT06260566	PHASE1	NOT_YET_RECRUITING	Tolerability of Enteral NAC in Infants
NCT07011199	PHASE1	NOT_YET_RECRUITING	Clinical Outcomes of Early Kasai Surgery With Umbilical Cord MSCs in Biliary Atresia
NCT01322386	PHASE1	COMPLETED	Gastrointestinal Microbiota in Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin
NCT05517317	PHASE1	COMPLETED	Autologous BMNC Infusion for Liver Cirrhosis in Children With BA
NCT07250854	EARLY_PHASE1	RECRUITING	The Use of Near-Infrared Fluorescence Cholangiography With Indocyanine Green (ICG) in the Work Up of Neonatal Cholestasis
NCT03395028	EARLY_PHASE1	COMPLETED	GCSF Adjunct Therapy for Biliary Atresia
NCT00061828	Not specified	RECRUITING	A Prospective Database of Infants With Cholestasis
NCT00345553	Not specified	RECRUITING	Biliary Atresia Study in Infants and Children
NCT01793168	Not specified	RECRUITING	Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
NCT03273049	Not specified	RECRUITING	Mapping Disease Pathways for Biliary Atresia
NCT04272515	Not specified	RECRUITING	Molecular Characterization for Understanding Biliary Atresia
NCT05072626	Not specified	RECRUITING	High Medium-chain Triglyceride Nutritional Support in Infants With Biliary Atresia
NCT05399745	Not specified	RECRUITING	BILACO Trial: Biliary Atresia - a Severe Complex Congenital Liver Disease
NCT05848310	Not specified	RECRUITING	Preoperative Serum FGF19 in the Prognosis of Biliary Atresia
NCT05925309	Not specified	RECRUITING	Preventive Effect of Prophylactic Oral Antibiotics Against Cholangitis After Kasai Portoenterostomy
NCT06184971	Not specified	ACTIVE_NOT_RECRUITING	Biliary Atresia Research Network Northeast
NCT06564740	Not specified	RECRUITING	Stem Cell Applications in Biliary Atresia Patients
NCT06708572	Not specified	RECRUITING	Evaluation of the Use of Granulocyte Colony Stimulating Factor (GCSF) in Post Kasai Type 3 Biliary Atresia
NCT06764082	Not specified	RECRUITING	Nutritional Intervention for Biliary Atresia
NCT07139717	Not specified	NOT_YET_RECRUITING	Stool Card in Biliary Atresia
NCT00007033	Not specified	COMPLETED	Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease
NCT00155194	Not specified	UNKNOWN	Evaluation of Immune Function in Biliary Atresia Children With Prolonged Jaundice
NCT00166868	Not specified	COMPLETED	Use of Probiotics to Prevent Cholangitis in Children With Biliary Atresia After the Kasai Portoenterostomy
NCT00294684	Not specified	COMPLETED	A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy
NCT01063699	Not specified	COMPLETED	Survival With Own Liver of Conventional Versus Laparoscopic Kasai for Biliary Atresia
NCT01443572	Not specified	COMPLETED	The Comparison of Desflurane and Sevoflurane on Postoperative Recovery and Hepatic Function of Biliary Atresia Patients During Kasai Operation

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
DESFLURANE	4	2
INDOCYANINE GREEN ACID FORM	4	2
ODEVIXIBAT	4	2
URSODIOL	4	2
MARALIXIBAT	4	1
NEOMYCIN	4	1
NOREPINEPHRINE BITARTRATE	4	1
PENTOXIFYLLINE	4	1
FRAMYCETIN	3	1
CHEMBL3754093	0	1
CHEMBL4299247	0	1
CHEMBL5409583	0	1
MAGNESIUM GLUCONATE	-1	1