Disease · MONDO_0000447

autosomal dominant polycystic liver disease

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. autosomal dominant polycystic liver disease. Diseases, record MONDO_0000447. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0000447

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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: autosomal dominant polycystic liver disease; Open Targets
Open Targets ID: MONDO_0000447; Open Targets
Other names: AD polycystic liver disease, ADPCLD, ADPLD, PCLD, isolated congenital polycystic liver disease, isolated polycystic liver disease, polycystic liver disease; Open Targets
Disease Ontology: DOID:0050770; Disease Ontology
NCI Thesaurus: C82833; NCI Thesaurus
Orphanet: ORPHA:2924; Orphanet
Highest stage of its drugs: Phase 3, for 1 of its 9 drugs, from ClinicalTrials.gov; Open Targets

Drugs9

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
LANREOTIDE	CHEMBL1201185	Protein	Phase 3	ClinicalTrials.gov	4	4	4	Drugs and targets
OCTREOTIDE	CHEMBL1680	Protein	Phase 2/3	ClinicalTrials.gov	4	4	4	Drugs and targets
EVEROLIMUS	CHEMBL1908360	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
LEUPROLIDE	CHEMBL1201199	Protein	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
PASIREOTIDE	CHEMBL3349607	Protein	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
TAURURSODIOL	CHEMBL272427	Small molecule	Phase 2	ClinicalTrials.gov	1	1	0	Drugs and targets
URSODIOL	CHEMBL1551	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
SIROLIMUS	CHEMBL413	Small molecule	Unknown	ClinicalTrials.gov	1	1	1	Drugs and targets
TACROLIMUS	CHEMBL3989887	Small molecule	Unknown	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets8

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
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	3	FK506-binding protein 1A inhibitor	Phase 2	https://conductscience.org/library/genes/2280	Drugs and targets
SSTR2	Somatostatin receptor	3	Somatostatin receptor 2 agonist; Somatostatin receptor agonist	Phase 3	https://conductscience.org/library/genes/6752	Drugs and targets
SSTR5	Somatostatin receptor	3	Somatostatin receptor 5 agonist; Somatostatin receptor agonist	Phase 3	https://conductscience.org/library/genes/6755	Drugs and targets
SSTR1	Somatostatin receptor	2	Somatostatin receptor 1 agonist; Somatostatin receptor agonist	Phase 2/3	https://conductscience.org/library/genes/6751	Drugs and targets
SSTR3	Somatostatin receptor	2	Somatostatin receptor 3 agonist; Somatostatin receptor agonist	Phase 2/3	https://conductscience.org/library/genes/6753	Drugs and targets
GNRHR	Gonadotropin-releasing hormone receptor	1	Gonadotropin-releasing hormone receptor agonist	Phase 2	https://conductscience.org/library/genes/2798	Drugs and targets
NR1H4	Bile acid receptor	1	Bile acid receptor FXR agonist	Phase 2	https://conductscience.org/library/genes/9971	Drugs and targets
SSTR4	Somatostatin receptor	1	Somatostatin receptor agonist	Phase 2/3	https://conductscience.org/library/genes/6754	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
NCT01670110	Pasireotide LAR in Severe Polycystic Liver Disease	Phase 2	48	2020-03-02	Somatostatin Analogs, Polycystic Liver Disease, Autosomal Dominant Polycystic Kidney Disease, Autosomal Dominant Polycystic Liver Disease	PASIREOTIDE	Its record names the disease	ClinicalTrials.gov
NCT00426153	Octreotide in Severe Polycystic Liver Disease	Phase 2/3	42	2012-11-21	Polycystic Kidney, Autosomal Dominant, Polycystic Liver Disease, Hepatomegaly, Liver Diseases, Kidney, Polycystic, Abdominal Pain	OCTREOTIDE	Its record names the disease	ClinicalTrials.gov
NCT00934791	Polycystic Liver Disease in Kidney Transplant	Not applicable	2	2013-03-08	Polycystic Liver Disease	SIROLIMUS, TACROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT01616927	Study of Lanreotide to Treat Polycystic Kidney Disease	Phase 3	300		Autosomal Dominant Polycystic Kidney Disease (ADPKD)	LANREOTIDE	Its record names the disease	ClinicalTrials.gov
NCT05281328	A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD	Phase 2/3	71		Polycystic Liver Disease	OCTREOTIDE	Its record names the disease	ClinicalTrials.gov
NCT01315795	Lanreotide Autogel in the Treatment of Symptomatic Polycystic Liver Disease	Phase 2/3	59		Polycystic Liver Disease	LANREOTIDE	Its record names the disease	ClinicalTrials.gov
NCT00771888	Open-Label Extension of LOCKCYST Trial	Phase 2/3	54		Polycystic Liver Disease, Hepatomegaly, Liver Diseases, Polycystic Kidney, Autosomal Dominant	LANREOTIDE	Its record names the disease	ClinicalTrials.gov
NCT01157858	Everolimus and LongActing Octreotide Trial in Polycystic Livers	Phase 2	44		Polycystic Liver Disease	EVEROLIMUS, OCTREOTIDE	Its record names the disease	ClinicalTrials.gov
NCT01354405	Somatostatin Analogues as a Volume Reducing Treatment of Polycystic Livers (RESOLVE)		43		Polycystic Liver Disease	LANREOTIDE	Its record names the disease	ClinicalTrials.gov
NCT05478083	A GnRH Agonist IN Pre-menopausal Women STudy to Treat Severe Polycystic Liver Disease	Phase 2	36		Polycystic Liver Disease, Autosomal Dominant Polycystic Kidney	LEUPROLIDE	Its record names the disease	ClinicalTrials.gov

1 to 10 of 12

See all 12 trials

Comparisons in its trials3

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
Pasireotide LAR (SOM230) vs Placebo Injection: deaths from any cause	0 of 33	1 of 15	48	NCT01670110	Trial comparisons
Pasireotide LAR (SOM230) vs Placebo Injection: serious adverse events	4 of 33	1 of 15	48	NCT01670110	Trial comparisons
Octreotide vs Placebo: serious adverse events	3 of 28	0 of 14	42	NCT00426153	Trial comparisons

Papers from its trials15

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
27995519	RESULT	1	PubMed
22773240	RESULT	1	PubMed
19646443	RESULT	1	PubMed
39356039	DERIVED	4	PubMed
33779943	DERIVED	1	PubMed
32843370	DERIVED	1	PubMed
31022403	DERIVED	1	PubMed
30422235	DERIVED	1	PubMed
27212247	DERIVED	1	PubMed
26844873	DERIVED	1	PubMed

1 to 10 of 15

See all 15 papers from its trials

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_0000447

GET https://conductscience.org/api/diseases/v1/MONDO_0000447/drugs

API reference For AI assistants