Disease · MONDO_0009067

cystinuria

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. cystinuria. Diseases, record MONDO_0009067. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0009067

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: cystinuria; Open Targets
Open Targets ID: MONDO_0009067; Open Targets
Other names: cystinuria (disease), cystinuria-lysinuria syndrome; Open Targets
Disease Ontology: DOID:9266; Disease Ontology
MeSH: D003555; MeSH
NCI Thesaurus: C84664; NCI Thesaurus
Orphanet: ORPHA:214; Orphanet
Highest stage of its drugs: Approval, for 2 of its 5 drugs, from DailyMed and TTD; Open Targets

Drugs5

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
TIOPRONIN	CHEMBL1314	Small molecule	Approval	TTD	3	2	2	Drugs and targets
PENICILLAMINE	CHEMBL1430	Small molecule	Approval	DailyMed	3	1	1	Drugs and targets
BUCILLAMINE	CHEMBL80830	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
DAPAGLIFLOZIN	CHEMBL429910	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
TOLVAPTAN	CHEMBL344159	Small molecule	Unknown	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets2

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
AVPR2	Vasopressin V2 receptor	1	Vasopressin V2 receptor antagonist	Unknown	https://conductscience.org/library/genes/554	Drugs and targets
SLC5A2	Sodium/glucose cotransporter 2	1	Sodium/glucose cotransporter 2 inhibitor	Phase 2	https://conductscience.org/library/genes/6524	Drugs and targets

Trials5

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
NCT02125721	Effect of Increasing Doses of Cystine Binding Thiol Drugs on Cystine Capacity in Patients With Cystinuria	Phase 4	10	2021-08-23	Cystinuria	PENICILLAMINE, TIOPRONIN	Its record names the disease	ClinicalTrials.gov
NCT03663855	Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria	Phase 2	10	2021-12-17	Cystinuria	TIOPRONIN	Its record names the disease	ClinicalTrials.gov
NCT02538016	TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study	Not applicable	4	2020-06-04	Cystinuria	TOLVAPTAN	Its record names the disease	ClinicalTrials.gov
NCT02942420	Bucillamine Phase 2 Trial in Patients With Cystinuria	Phase 2	30		Cystinuria	BUCILLAMINE	Its record names the disease	ClinicalTrials.gov
NCT05058859	Long Term Clinical Efficacy of Sodium-glucose Cotransporter-2 (SGLT-2) Inhibitor in Cystinurics	Phase 2	0		Cystinuria	DAPAGLIFLOZIN	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials0

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.

None in this release.

Papers from its trials0

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

None in this release.

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_0009067

GET https://conductscience.org/api/diseases/v1/MONDO_0009067/drugs

API reference For AI assistants