Disease · MONDO_0007318

Alagille syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Alagille syndrome. Diseases, record MONDO_0007318. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0007318

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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.

Name: Alagille syndrome; Open Targets
Open Targets ID: MONDO_0007318; Open Targets
Other names: Alagille-Watson syndrome, Arteriohepatic dysplasia, syndromic bile duct paucity; Open Targets
Disease Ontology: DOID:9245; Disease Ontology
MeSH: D016738; MeSH
NCI Thesaurus: C35139; NCI Thesaurus
Orphanet: ORPHA:52; Orphanet
Highest stage of its drugs: Approval, for 2 of its 3 drugs, from EMA Human Drugs, FDA and PMDA; Open Targets

Drugs3

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
MARALIXIBAT CHLORIDE	CHEMBL17879	Small molecule	Approval	EMA Human Drugs, FDA and PMDA	6	3	3	Drugs and targets
ODEVIXIBAT	CHEMBL4297588	Small molecule	Approval	EMA Human Drugs	4	2	2	Drugs and targets
MARALIXIBAT	CHEMBL363392	Small molecule	Phase 3	ClinicalTrials.gov	8	7	7	Drugs and targets

Targets1

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
SLC10A2	Ileal sodium/bile acid cotransporter	3	Ileal bile acid transporter inhibitor	Approval	https://conductscience.org/library/genes/6555

Source Drugs and targets

Trials12

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.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
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
NCT04674761	Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome	Phase 3	52	2023-11-02	Alagille Syndrome	ODEVIXIBAT	Its record names the disease	ClinicalTrials.gov
NCT02057692	Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome	Phase 2	37	2018-01-23	Alagille Syndrome	MARALIXIBAT CHLORIDE	Its record names the disease	ClinicalTrials.gov
NCT02117713	An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome	Phase 2	34	2021-06-22	Alagille Syndrome	MARALIXIBAT	Its record names the disease	ClinicalTrials.gov
NCT02160782	Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)	Phase 2	31	2021-07-14	Alagille Syndrome	MARALIXIBAT	Its record names the disease	ClinicalTrials.gov
NCT01903460	Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome	Phase 2	20	2015-12-07	Alagille Syndrome	MARALIXIBAT CHLORIDE	Its record names the disease	ClinicalTrials.gov
NCT05543174	A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)	Phase 3	7	2024-10-15	Alagille Syndrome (ALGS)	MARALIXIBAT	Its record names the disease	ClinicalTrials.gov
NCT07290257	Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)	Phase 4	230		Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis	MARALIXIBAT CHLORIDE	Its record names the disease	ClinicalTrials.gov
NCT06193928	Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)		70		Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis	MARALIXIBAT	Its record names the disease	ClinicalTrials.gov
NCT05035030	Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome	Phase 3	62		Alagille Syndrome	ODEVIXIBAT	Its record names the disease	ClinicalTrials.gov

1 to 10 of 12

See all 12 trials

Comparisons in its trials4

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
Maralixibat: ALGS vs Maralixibat: PFIC: deaths from any cause	1 of 40	0 of 12	52	NCT04168385	Trial comparisons
Maralixibat: ALGS vs Maralixibat: PFIC: serious adverse events	10 of 40	3 of 12	52	NCT04168385	Trial comparisons
Odevixibat (A4250) vs Placebo: deaths from any cause	0 of 35	0 of 17	52	NCT04674761	Trial comparisons
Odevixibat (A4250) vs Placebo: serious adverse events	5 of 35	2 of 17	52	NCT04674761	Trial comparisons

Papers from its trials4

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
42002216	DERIVED	3	PubMed
38670135	DERIVED	1	PubMed
36096175	DERIVED	1	PubMed
34755627	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

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_0007318

GET https://conductscience.org/api/diseases/v1/MONDO_0007318/drugs

API reference For AI assistants