Disease · MONDO_0004691
autosomal dominant polycystic kidney disease
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. autosomal dominant polycystic kidney disease. Diseases, record MONDO_0004691. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0004691
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.
- Other names
ADPKD, ADPKD - Autosomal dominant polycystic kidney disease, Autosomal dominant adult polycystic kidney disease, Polycystic kidney disease, adult type (disorder), Polycystic kidneys - adult type, adult type autosomal dominant polycystic kidney disease, polycystic kidney disease, adult type, polycystic kidney disease, autosomal dominant, polycystic kidney, autosomal dominant
- Open Targets
Drugs38
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 |
|---|---|---|---|---|---|---|---|---|
| TOLVAPTAN | CHEMBL344159 | Small molecule | Approval | EMA Human Drugs | 21 | 20 | 20 | Drugs and targets |
| SIROLIMUS | CHEMBL413 | Small molecule | Phase 3 | ClinicalTrials.gov | 8 | 8 | 8 | Drugs and targets |
| OCTREOTIDE | CHEMBL1680 | Protein | Phase 3 | ClinicalTrials.gov | 5 | 5 | 5 | Drugs and targets |
| LIXIVAPTAN | CHEMBL49429 | Small molecule | Phase 3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| METFORMIN | CHEMBL1431 | Small molecule | Phase 3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| VENGLUSTAT | CHEMBL4297611 | Small molecule | Phase 3 | ClinicalTrials.gov | 3 | 2 | 2 | Drugs and targets |
| BARDOXOLONE METHYL | CHEMBL1762621 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| EVEROLIMUS | CHEMBL1908360 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| LANREOTIDE | CHEMBL1201185 | Protein | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| LISINOPRIL ANHYDROUS | CHEMBL1237 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
1 to 10 of 38
See all 38 drugsTargets86
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 |
|---|---|---|---|---|---|---|
| SSTR2 | Somatostatin receptor | 4 | Somatostatin receptor 2 agonist; Somatostatin receptor agonist | Phase 3 | https://conductscience.org/library/genes/6752 | Drugs and targets |
| SSTR5 | Somatostatin receptor | 4 | Somatostatin receptor 5 agonist; Somatostatin receptor agonist | Phase 3 | https://conductscience.org/library/genes/6755 | Drugs and targets |
| SSTR1 | Somatostatin receptor | 3 | Somatostatin receptor 1 agonist; Somatostatin receptor agonist | Phase 3 | https://conductscience.org/library/genes/6751 | Drugs and targets |
| SSTR3 | Somatostatin receptor | 3 | Somatostatin receptor 3 agonist; Somatostatin receptor agonist | Phase 3 | https://conductscience.org/library/genes/6753 | Drugs and targets |
| AVPR2 | Vasopressin V2 receptor | 2 | Vasopressin V2 receptor antagonist | Approval | https://conductscience.org/library/genes/554 | Drugs and targets |
| FKBP1A | Peptidyl-prolyl cis-trans isomerase FKBP1A | 2 | FK506-binding protein 1A inhibitor | Phase 3 | https://conductscience.org/library/genes/2280 | Drugs and targets |
| HMGCR | 3-hydroxy-3-methylglutaryl-coenzyme A reductase | 2 | HMG-CoA reductase inhibitor | Phase 3 | https://conductscience.org/library/genes/3156 | Drugs and targets |
| PPARG | Peroxisome proliferator-activated receptor gamma | 2 | Peroxisome proliferator-activated receptor gamma agonist; Peroxisome proliferator-activated receptor gamma antagonist | Phase 3 | https://conductscience.org/library/genes/5468 | Drugs and targets |
| SLC5A2 | Sodium/glucose cotransporter 2 | 2 | Sodium/glucose cotransporter 2 inhibitor | Phase 3 | https://conductscience.org/library/genes/6524 | Drugs and targets |
| SSTR4 | Somatostatin receptor | 2 | Somatostatin receptor agonist | Phase 3 | https://conductscience.org/library/genes/6754 | Drugs and targets |
1 to 10 of 86
See all 86 targetsTrials73
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 |
|---|---|---|---|---|---|---|---|---|
| NCT02251275 | Long Term Safety of Immediate-release Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease | Phase 3 | 1,803 | 2019-11-27 | Polycystic Kidney, Autosomal Dominant | TOLVAPTAN | Its record names the disease | ClinicalTrials.gov |
| NCT02847624 | Post-Marketing Surveillance Study of Tolvaptan in Patients With ADPKD | 1,802 | 2025-12-10 | Polycystic Kidney, Autosomal Dominant | TOLVAPTAN | Its record names the disease | ClinicalTrials.gov | |
| NCT00428948 | Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 3 | 1,445 | 2017-07-02 | Polycystic Kidney Disease, Autosomal Dominant | TOLVAPTAN | Its record names the disease | ClinicalTrials.gov |
| NCT01214421 | Tolvaptan Extension Study in Participants With ADPKD | Phase 3 | 1,083 | 2021-10-25 | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | TOLVAPTAN | Its record names the disease | ClinicalTrials.gov |
| NCT03918447 | A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON | Phase 3 | 667 | 2024-06-11 | Autosomal Dominant Polycystic Kidney, ADPKD | BARDOXOLONE METHYL | Its record names the disease | ClinicalTrials.gov |
| NCT00283686 | HALT Progression of Polycystic Kidney Disease Study A | Phase 3 | 558 | 2015-02-27 | Kidney, Polycystic | LISINOPRIL ANHYDROUS, TELMISARTAN | Its record names the disease | ClinicalTrials.gov |
| NCT01885559 | HALT Progression of Polycystic Kidney Disease Study B | Phase 3 | 486 | 2015-03-10 | Kidney, Polycystic | LISINOPRIL ANHYDROUS, TELMISARTAN | Its record names the disease | ClinicalTrials.gov |
| NCT03523728 | A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients | Phase 2/3 | 478 | 2022-11-09 | Polycystic Kidney, Autosomal Dominant | VENGLUSTAT | Its record names the disease | ClinicalTrials.gov |
| NCT01451827 | 8-Week Study of Tolvaptan Dose Forms in Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Phase 2 | 178 | 2018-08-13 | Autosomal Dominant Polycystic Kidney Disease | TOLVAPTAN | Its record names the disease | ClinicalTrials.gov |
| NCT01233869 | Bosutinib For Autosomal Dominant Polycystic Kidney Disease | Phase 2 | 172 | 2015-10-28 | Polycystic Kidney, Autosomal Dominant | BOSUTINIB | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 73
See all 73 trialsComparisons in its trials34
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 |
|---|---|---|---|---|---|
| Tolvaptan vs Placebo: serious adverse events | 177 of 961 | 95 of 483 | 1,444 | NCT00428948 | Trial comparisons |
| Bardoxolone Methyl vs Placebo: deaths from any cause | 1 of 335 | 0 of 331 | 666 | NCT03918447 | Trial comparisons |
| Bardoxolone Methyl vs Placebo: serious adverse events | 38 of 335 | 26 of 331 | 666 | NCT03918447 | Trial comparisons |
| ACE-I + Placebo vs ACE-I + ARB: serious adverse events | 88 of 242 | 88 of 244 | 486 | NCT01885559 | Trial comparisons |
| ACE-I + Placebo vs ACE-I + ARB: Number of Participants With 50% Reduction of Baseline eGFR, End Stage Renal Disease (ESRD, Initiation of Dialysis or Preemptive Transplant), or Death. | 116 of 242 | 115 of 243 | 485 | NCT01885559 | Trial comparisons |
| Pravastatin vs Placebo: serious adverse events | 0 of 56 | 0 of 54 | 110 | NCT00456365 | Trial comparisons |
| Metformin vs Placebo: Drug Tolerability | 21 of 49 | 14 of 48 | 97 | NCT02656017 | Trial comparisons |
| Metformin vs Placebo: deaths from any cause | 0 of 49 | 0 of 48 | 97 | NCT02656017 | Trial comparisons |
| Metformin vs Placebo: Rate of Serious Adverse Events (SAE) | 4 of 49 | 4 of 48 | 97 | NCT02656017 | Trial comparisons |
| Metformin vs Placebo: serious adverse events | 4 of 49 | 4 of 48 | 97 | NCT02656017 | Trial comparisons |
1 to 10 of 34
See all 34 comparisons in its trialsPapers from its trials63
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
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_0004691
GET https://conductscience.org/api/diseases/v1/MONDO_0004691/drugs?page=2