Disease · MONDO_0002474

primary hyperoxaluria

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. primary hyperoxaluria. Diseases, record MONDO_0002474. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0002474

On this page

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: primary hyperoxaluria; Open Targets
Open Targets ID: MONDO_0002474; Open Targets
Other names: hyperoxaluria, primary; Open Targets
Disease Ontology: DOID:2977; Disease Ontology
MeSH: D006960; MeSH
NCI Thesaurus: C123158; NCI Thesaurus
Orphanet: ORPHA:416; Orphanet
Highest stage of its drugs: Approval, for 2 of its 12 drugs, from EMA Human Drugs and FDA; Open Targets

Drugs12

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
NEDOSIRAN SODIUM	CHEMBL4594338	Oligonucleotide	Approval	FDA	4	2	2	Drugs and targets
LUMASIRAN	CHEMBL4297779	Oligonucleotide	Approval	EMA Human Drugs	3	1	1	Drugs and targets
RELOXALIASE	CHEMBL4297818	Enzyme	Phase 3	ClinicalTrials.gov	6	6	6	Drugs and targets
STIRIPENTOL	CHEMBL1983350	Small molecule	Phase 3	ClinicalTrials.gov	2	2	2	Drugs and targets
ANGIOTENSIN	CHEMBL2391146	Protein	Phase 3	ClinicalTrials.gov	1	1	0	Drugs and targets
CANDESARTAN	CHEMBL1016	Small molecule	Phase 3	ClinicalTrials.gov	1	1	0	Drugs and targets
IRBESARTAN	CHEMBL1513	Small molecule	Phase 3	ClinicalTrials.gov	1	1	0	Drugs and targets
LOSARTAN	CHEMBL191	Small molecule	Phase 3	ClinicalTrials.gov	1	1	0	Drugs and targets
TELMISARTAN	CHEMBL1017	Small molecule	Phase 3	ClinicalTrials.gov	1	1	0	Drugs and targets
VALSARTAN	CHEMBL1069	Small molecule	Phase 3	ClinicalTrials.gov	1	1	0	Drugs and targets

1 to 10 of 12

See all 12 drugs

Targets3

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
AGTR1	Type-1 angiotensin II receptor	5	Type-1 angiotensin II receptor antagonist	Phase 3	https://conductscience.org/library/genes/185	Drugs and targets
HAO1	Hydroxyacid oxidase 1 mRNA	1	Hydroxyacid oxidase 1 mRNA RNAi inhibitor	Approval	https://conductscience.org/library/genes/54363	Drugs and targets
LDHA	L-lactate dehydrogenase A chain mRNA	1	L-lactate dehydrogenase A chain mRNA RNAi inhibitor	Approval	https://conductscience.org/library/genes/3939	Drugs and targets

Trials13

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. 1 more is not shown, since the checks do not confirm it.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT04152200	A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1	Phase 3	21	2022-07-12	Primary Hyperoxaluria Type 1, Primary Hyperoxaluria	LUMASIRAN	Its record names the disease	ClinicalTrials.gov
NCT02289755	Evaluating ALLN-177 for Reducing Urinary Oxalate Excretion in Calcium Oxalate Kidney Stone Formers With Hyperoxaluria	Phase 2	16	2016-04-27	Hyperoxaluria, Nephrolithiasis	RELOXALIASE	Open Targets' reading of its record	ClinicalTrials.gov
NCT03456830	Evaluate ALLN-177 in Patients With Enteric Hyperoxaluria	Phase 3	115		Enteric Hyperoxaluria	RELOXALIASE	Open Targets' reading of its record	ClinicalTrials.gov
NCT03847090	Establishing the Safety and Efficacy of Reloxaliase in Patients With Enteric Hyperoxaluria	Phase 3	92		Enteric Hyperoxaluria	RELOXALIASE	Open Targets' reading of its record	ClinicalTrials.gov
NCT04042402	Long Term Extension Study in Patients With Primary Hyperoxaluria	Phase 3	75		Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease, Primary Hyperoxaluria Type 3 (PH3)	NEDOSIRAN SODIUM	Its record names the disease	ClinicalTrials.gov
NCT02547805	Evaluate the Effect of ALLN-177 in Reducing Urinary Oxalate in Patients With Secondary Hyperoxaluria and Kidney Stones Over 28 Days	Phase 2	66		Secondary Hyperoxaluria, Nephrolithiasis, Hyperoxaluria, Kidney Stones, Dietary Hyperoxaluria	RELOXALIASE	Open Targets' reading of its record	ClinicalTrials.gov
NCT06465472	Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3	Phase 3	42		Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3	STIRIPENTOL	Its record names the disease	ClinicalTrials.gov
NCT02503345	Evaluate the Effect of ALLN-177 in Reducing Urinary Oxalate in Patients With Hyperoxaluria and Kidney Stones	Phase 2	32		Secondary Hyperoxaluria, Nephrolithiasis, Kidney Stones, Hyperoxaluria, Dietary Hyperoxaluria	RELOXALIASE	Open Targets' reading of its record	ClinicalTrials.gov
NCT04580420	Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD	Phase 2	28		Primary Hyperoxaluria Type 1, End Stage Renal Disease	NEDOSIRAN SODIUM	Open Targets' reading of its record	ClinicalTrials.gov
NCT05001269	Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function	Phase 2	27		Primary Hyperoxaluria, Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3	NEDOSIRAN	Its record names the disease	ClinicalTrials.gov

1 to 10 of 13

See all 13 trials

Comparisons in its trials2

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
Cohort A: Lumasiran vs Cohort B: Lumasiran: deaths from any cause	0 of 6	0 of 15	21	NCT04152200	Trial comparisons
Cohort A: Lumasiran vs Cohort B: Lumasiran: serious adverse events	1 of 6	8 of 15	21	NCT04152200	Trial comparisons

Papers from its trials7

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
40601241	DERIVED	2	PubMed
40193200	DERIVED	3	PubMed
39875734	DERIVED	1	PubMed
38332115	DERIVED	1	PubMed
38319254	DERIVED	1	PubMed
35843439	DERIVED	1	PubMed
30783888	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_0002474

GET https://conductscience.org/api/diseases/v1/MONDO_0002474/drugs

API reference For AI assistants