Polycystic Kidney Disease (ADPKD) — RepurpOS

Disease overview

Alterations

166

Molecular (A)

100

Clinical (B–E)

Direct drugs

Via biomarker

136

Natural agents

Natural products

Merged ranked

143

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, Examine.com, disease_db_100 · Pipeline phase 7 · Updated 2026-07-06

Remission & chronicity

Spontaneous remission

Progressive; GFR decline averages 4-5 mL/min/year in Mayo Class 1C-1E; renal failure by 60-70 years in 50% without treatment; spontaneous stabilisation rare

Best-intervention remission

Tolvaptan (Jynarque): slows GFR decline by 35% and total kidney volume growth by 49% (TEMPO 3:4, REPRISE); dietary protein restriction + hydration: modest benefit; SGLT2 inhibitors — early signal in PKD trials

Drug-free remission

—

Relapse after remission

—

Chronicity

—

Gap size

Moderate — tolvaptan is available and effective but hepatotoxicity risk limits use; access barriers (REMS programme in US) reduce prescribing

Primary barrier

access/practice

Barrier detail: Tolvaptan REMS programme (liver toxicity monitoring) creates prescribing complexity; only patients with rapidly progressive disease (Mayo Class 1C-1E) are eligible by guideline; most patients remain on supportive care without disease-modifying therapy

Last SoC change: 2018 — tolvaptan FDA approved for ADPKD; TEMPO 3:4 published 2012; mTOR inhibitors (sirolimus, everolimus) tried and failed in RCTs 2010; supportive care unchanged for decades until tolvaptan

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0020642 · ORPHA: ORPHA:88924

Right to Try Relevance

Eligibility: This disease meets the federal and state right-to-try legal standard (21 U.S.C. § 360bbb-0a and Montana SB 535): it lacks disease-modifying therapy with indication-specific FDA approval. Patients with Polycystic Kidney Disease may be eligible to access investigational therapies outside of clinical trials under right-to-try frameworks.

Investigational Agents

Phase 2: 4, Phase 1: 1

Most Advanced Phase

Phase 2

Highest dev. stage

Gene Targets

166

Actionable

Pipeline Compounds (Top 6)

Cilnidipine, Imidapril, Octreotide, Tesevatinib, Hydralazine

Legal Standard: Right-to-try requires the disease lacks "adequate approved therapy for that specific disease" (not symptom-management drugs). This distinction means a disease with antidepressants available still qualifies if no FDA approval exists for THAT disease indication.

View Right-to-Try Compendium

Alterations

Showing 80 of 166 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Absence of renal corticomedullary differentiation	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cerebral berry aneurysm	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Colonic diverticula	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Decreased circulating vitamin D concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Decreased circulating vitamin E concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Decreased circulating vitamin K concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Decreased glomerular filtration rate	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Decreased liver function	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dehydration	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dextrocardia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dilatation of the cerebral artery	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating alkaline phosphatase concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating alpha-fetoprotein concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating creatinine concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO LOINC
Elevated gamma-glutamyltransferase level	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Enlarged kidney	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Esophageal varix	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Facial asymmetry	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Focal segmental glomerulosclerosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gout	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hematemesis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatic cysts	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatic fibrosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatomegaly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatosplenomegaly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hyperechogenic kidneys	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hypertension	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hypokalemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hyponatremia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Increased serum bile acid concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Jaundice	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Left ventricular hypertrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Midface retrusion	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Mitral regurgitation	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Mitral valve prolapse	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Multiple renal cysts	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Multiple small medullary renal cysts	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Nephronophthisis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Oligohydramnios	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pancreatic cysts	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Periportal fibrosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Polycystic kidney dysplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Polydipsia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Polyuria	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Portal hypertension	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Potter facies	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pulmonary hypoplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent urinary tract infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Reduced circulating vitamin A concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Reduced renal corticomedullary differentiation	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Renal atrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Renal cyst	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Renal insufficiency	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Renal interstitial fibrosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Short stature	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Situs inversus totalis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Splenomegaly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Stage 5 chronic kidney disease	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Tubulointerstitial fibrosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
ACE angiotensin I converting enzyme	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
AGTR1 angiotensin II receptor type 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ALG5 ALG5 dolichyl-phosphate beta-glucosyltransferase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ALG8 ALG8 alpha-1,3-glucosyltransferase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ALG9 ALG9 alpha-1,2-mannosyltransferase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
AVPR2 arginine vasopressin receptor 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CC2D2A coiled-coil and C2 domain containing 2A	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CFAP47 cilia and flagella associated protein 47	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
COXFA4 cytochrome c oxidase associated subunit FA4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
COXFA4L2 cytochrome c oxidase hypoxia associated subunit FA4L2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CYS1 cystin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DKK3 dickkopf Wnt signaling pathway inhibitor 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DNAJB11 DnaJ heat shock protein family (Hsp40) member B11	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DYNC2H1 dynein cytoplasmic 2 heavy chain 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DZIP1L DAZ interacting zinc finger protein 1 like	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
EGFR epidermal growth factor receptor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FKBP1A FKBP prolyl isomerase 1A	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
GANAB glucosidase II alpha subunit	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
GLIS3 GLIS family zinc finger 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
GPD2 glycerol-3-phosphate dehydrogenase 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Glomerular filtration rate (GFR)	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—

FDA-Approved Therapies

Phase 4 agents with regulatory approval for this indication

Drug Name	Mechanism	Type
ACETAZOLAMIDE	None	Gene-targeted
ACETYLCHOLINE		Gene-targeted
AMPHETAMINE	None	Gene-targeted
ANGIOTENSIN II	agonist	Gene-targeted
ANGIOTENSIN II ACETATE	agonist	Gene-targeted
ARGIPRESSIN	agonist; agonist	Gene-targeted
ASCORBIC ACID		Gene-targeted
ASPIRIN		Gene-targeted
ATORVASTATIN		Gene-targeted
ATORVASTATIN CALCIUM TRIHYDRATE		Gene-targeted
ATOSIBAN	inhibitor	Gene-targeted
AZILSARTAN KAMEDOXOMIL	inhibitor	Gene-targeted
AZILSARTAN MEDOXOMIL	inhibitor; inhibitor	Gene-targeted
BENAZEPRIL	inhibitor	Gene-targeted
BENAZEPRIL HYDROCHLORIDE	inhibitor	Gene-targeted
BENAZEPRILAT	inhibitor	Gene-targeted
BICALUTAMIDE	None	Gene-targeted
BUMETANIDE		Gene-targeted
BUPROPION HYDROCHLORIDE		Gene-targeted
CANDESARTAN CILEXETIL	inhibitor; inhibitor	Gene-targeted
CANDOXATRIL		Gene-targeted
CAPTOPRIL	inhibitor; inhibitor	Gene-targeted
CARBACHOL	None	Gene-targeted
CILAZAPRIL	inhibitor; inhibitor	Gene-targeted
CIPROFLOXACIN	None	Gene-targeted
CLOMIPRAMINE	None	Gene-targeted
CLOTRIMAZOLE		Gene-targeted
CONIVAPTAN	inhibitor	Gene-targeted
CONIVAPTAN HYDROCHLORIDE	inhibitor	Gene-targeted
CYCLOSPORINE		Gene-targeted
DESERPIDINE		Gene-targeted
DESMOPRESSIN	agonist; agonist	Gene-targeted
DEXAMETHASONE		Gene-targeted
DIAZEPAM	None	Gene-targeted
DICHLORPHENAMIDE	None	Gene-targeted
DIETHYLSTILBESTROL	None	Gene-targeted
DISULFIRAM		Gene-targeted
ENALAPRIL MALEATE	inhibitor	Gene-targeted
ENALAPRILAT	inhibitor	Gene-targeted
ENALAPRILAT ANHYDROUS	inhibitor	Gene-targeted

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, and merged ranking. Only agents with registry trials or published clinical literature are shown by default.

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ACETYLCHOLINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANGIOTENSIN II Repurposing	Small molecule	Approved	Strong	95	3 trials 1 Cochrane 1 Meta-analysis 7 Trial pubs 5 RCT View evidence Registry trials NCT00890279 — Efficacy and Safety Study of Second-Line Treatment for Hypertension With Autosomal Dominant Polycystic Kidney Disease(ADPKD) (UNKNOWN) PHASE2 NCT00541853 — CCB Safety Study in Treatment of Hypertension of ADPKD (UNKNOWN) PHASE4 NCT01932450 — Radiofrequency Ablation for ADPKD Blood Pressure and Disease Progression Control (UNKNOWN) PHASE2 Published literature Cochrane review Antihypertensive treatments in adult autosomal dominant polycystic kidney disease: network meta-analysis of the randomized controlled trials (Oncotarget, 2015) Clinical trial publication Angiotensin blockade in late autosomal dominant polycystic kidney disease (The New England journal of medicine, 2014) Clinical trial publication Urinary excretion of AQP2 and ENaC in autosomal dominant polycystic kidney disease during basal conditions and after a hypertonic saline infusion (American journal of physiology. Renal physiology, 2012) Clinical trial publication Candesartan reduces urinary fatty acid-binding protein excretion in patients with autosomal dominant polycystic kidney disease (The American journal of the medical sciences, 2005) Clinical trial publication Calcium channel blocker versus angiotensin II receptor blocker in autosomal dominant polycystic kidney disease (Nephron. Clinical practice, 2005) Clinical trial publication Effects of the angiotensin II receptor antagonist candesartan cilexetil on blood pressure and proteinuria in patients with autosomal dominant polycystic kidney disease (Nephron, 2000) Randomized controlled trial Natriuresis-pressure relationship in polycystic kidney disease (Journal of hypertension, 1990) CT.gov search · Cochrane · PubMed	DGIdb	—
ANGIOTENSIN II ACETATE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
ARGIPRESSIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ASCORBIC ACID Repurposing	Small molecule	Approved	Strong	95	1 Trial pubs View evidence Published literature Clinical trial publication Curcumin Therapy to Treat Vascular Dysfunction in Children and Young Adults with ADPKD: A Randomized Controlled Trial (Clinical journal of the American Society of Nephrology : CJASN, 2022) CT.gov search · Cochrane · PubMed	DGIdb	—
ASPIRIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATORVASTATIN Repurposing	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT05870007 — Atorvastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease (ENROLLING_BY_INVITATION) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
ATORVASTATIN CALCIUM TRIHYDRATE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ATOSIBAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AZILSARTAN KAMEDOXOMIL Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
AZILSARTAN MEDOXOMIL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BENAZEPRIL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BENAZEPRIL HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BENAZEPRILAT Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BUMETANIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BUPROPION HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CANDESARTAN CILEXETIL Repurposing	Small molecule	Approved	Strong	95	2 trials 2 Trial pubs 1 RCT View evidence Registry trials NCT00890279 — Efficacy and Safety Study of Second-Line Treatment for Hypertension With Autosomal Dominant Polycystic Kidney Disease(ADPKD) (UNKNOWN) PHASE2 NCT00541853 — CCB Safety Study in Treatment of Hypertension of ADPKD (UNKNOWN) PHASE4 Published literature Clinical trial publication Candesartan reduces urinary fatty acid-binding protein excretion in patients with autosomal dominant polycystic kidney disease (The American journal of the medical sciences, 2005) Clinical trial publication Effects of the angiotensin II receptor antagonist candesartan cilexetil on blood pressure and proteinuria in patients with autosomal dominant polycystic kidney disease (Nephron, 2000) CT.gov search · Cochrane · PubMed	DGIdb	—
CANDOXATRIL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CAPTOPRIL Repurposing	Small molecule	Approved	Strong	95	4 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CARBACHOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CILAZAPRIL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOTRIMAZOLE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CONIVAPTAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CONIVAPTAN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
CYCLOSPORINE Repurposing	Small molecule	Approved	Strong	95	1 trials 1 Cochrane 1 Meta-analysis View evidence Registry trials NCT07565441 — Drug-Drug Interactions of JMKX003142 in Healthy Participants (NOT_YET_RECRUITING) PHASE1 Published literature Cochrane review [Risk factors for new-onset diabetes after transplantation in kidney transplant recipients: own data and meta-analysis] (Terapevticheskii arkhiv, 2025) CT.gov search · Cochrane · PubMed	DGIdb	—
DESERPIDINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DESMOPRESSIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DEXAMETHASONE Repurposing	Small molecule	Approved	Strong	95	7 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DISULFIRAM Repurposing	Small molecule	Approved	Strong	95	2 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ENALAPRIL MALEATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ENALAPRILAT Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ENALAPRILAT ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
EPIRUBICIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
EPROSARTAN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EPROSARTAN MESYLATE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
ETHOXZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ETOPOSIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
FLUMAZENIL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUOXETINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
FOSINOPRIL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
FOSINOPRIL SODIUM Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
FUROSEMIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—

Drug	Type	Phase	Evidence	Score	Sources	Links
CILNIDIPINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
IMIDAPRIL	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
OCTREOTIDE	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
TESEVATINIB	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
HYDRALAZINE	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets
LANREOTIDE	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets
TOLVAPTAN	Small molecule	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ACETAZOLAMIDE Repurposing via ACE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ACETYLCHOLINE Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—
AMPHETAMINE Repurposing via ACE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANGIOTENSIN II Repurposing via AGTR1	Small molecule	Approved	Strong	95	3 trials 1 Cochrane 1 Meta-analysis 7 Trial pubs 5 RCT View evidence Registry trials NCT00890279 — Efficacy and Safety Study of Second-Line Treatment for Hypertension With Autosomal Dominant Polycystic Kidney Disease(ADPKD) (UNKNOWN) PHASE2 NCT00541853 — CCB Safety Study in Treatment of Hypertension of ADPKD (UNKNOWN) PHASE4 NCT01932450 — Radiofrequency Ablation for ADPKD Blood Pressure and Disease Progression Control (UNKNOWN) PHASE2 Published literature Cochrane review Antihypertensive treatments in adult autosomal dominant polycystic kidney disease: network meta-analysis of the randomized controlled trials (Oncotarget, 2015) Clinical trial publication Angiotensin blockade in late autosomal dominant polycystic kidney disease (The New England journal of medicine, 2014) Clinical trial publication Urinary excretion of AQP2 and ENaC in autosomal dominant polycystic kidney disease during basal conditions and after a hypertonic saline infusion (American journal of physiology. Renal physiology, 2012) Clinical trial publication Candesartan reduces urinary fatty acid-binding protein excretion in patients with autosomal dominant polycystic kidney disease (The American journal of the medical sciences, 2005) Clinical trial publication Calcium channel blocker versus angiotensin II receptor blocker in autosomal dominant polycystic kidney disease (Nephron. Clinical practice, 2005) Clinical trial publication Effects of the angiotensin II receptor antagonist candesartan cilexetil on blood pressure and proteinuria in patients with autosomal dominant polycystic kidney disease (Nephron, 2000) Randomized controlled trial Natriuresis-pressure relationship in polycystic kidney disease (Journal of hypertension, 1990) CT.gov search · Cochrane · PubMed	DGIdb	—
ANGIOTENSIN II ACETATE Repurposing via AGTR1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
ARGIPRESSIN Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—
ASCORBIC ACID Repurposing via AGTR1	Small molecule	Approved	Strong	95	1 Trial pubs View evidence Published literature Clinical trial publication Curcumin Therapy to Treat Vascular Dysfunction in Children and Young Adults with ADPKD: A Randomized Controlled Trial (Clinical journal of the American Society of Nephrology : CJASN, 2022) CT.gov search · Cochrane · PubMed	DGIdb	—
ASPIRIN Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—
ATORVASTATIN Repurposing via AGTR1	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT05870007 — Atorvastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease (ENROLLING_BY_INVITATION) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
ATORVASTATIN CALCIUM TRIHYDRATE Repurposing via AGTR1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ATOSIBAN Repurposing via AVPR2	Small molecule	Approved	Strong	95	—	DGIdb	—
AZILSARTAN KAMEDOXOMIL Repurposing via AGTR1	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
AZILSARTAN MEDOXOMIL Repurposing via AGTR1	Small molecule	Approved	Strong	95	—	DGIdb	—
BENAZEPRIL Repurposing via AGTR1	Small molecule	Approved	Strong	95	—	DGIdb	—
BENAZEPRIL HYDROCHLORIDE Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BENAZEPRILAT Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing via ACE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BUMETANIDE Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—
BUPROPION HYDROCHLORIDE Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—
CANDESARTAN CILEXETIL Repurposing via AGTR1	Small molecule	Approved	Strong	95	2 trials 2 Trial pubs 1 RCT View evidence Registry trials NCT00890279 — Efficacy and Safety Study of Second-Line Treatment for Hypertension With Autosomal Dominant Polycystic Kidney Disease(ADPKD) (UNKNOWN) PHASE2 NCT00541853 — CCB Safety Study in Treatment of Hypertension of ADPKD (UNKNOWN) PHASE4 Published literature Clinical trial publication Candesartan reduces urinary fatty acid-binding protein excretion in patients with autosomal dominant polycystic kidney disease (The American journal of the medical sciences, 2005) Clinical trial publication Effects of the angiotensin II receptor antagonist candesartan cilexetil on blood pressure and proteinuria in patients with autosomal dominant polycystic kidney disease (Nephron, 2000) CT.gov search · Cochrane · PubMed	DGIdb	—
CANDOXATRIL Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—
CAPTOPRIL Repurposing via AGTR1	Small molecule	Approved	Strong	95	4 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CARBACHOL Repurposing via ACE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CILAZAPRIL Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing via ACE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOMIPRAMINE Repurposing via ACE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOTRIMAZOLE Repurposing via AVPR2	Small molecule	Approved	Strong	95	—	DGIdb	—
CONIVAPTAN Repurposing via AVPR2	Small molecule	Approved	Strong	95	—	DGIdb	—
CONIVAPTAN HYDROCHLORIDE Repurposing via AVPR2	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
CYCLOSPORINE Repurposing via AGTR1	Small molecule	Approved	Strong	95	1 trials 1 Cochrane 1 Meta-analysis View evidence Registry trials NCT07565441 — Drug-Drug Interactions of JMKX003142 in Healthy Participants (NOT_YET_RECRUITING) PHASE1 Published literature Cochrane review [Risk factors for new-onset diabetes after transplantation in kidney transplant recipients: own data and meta-analysis] (Terapevticheskii arkhiv, 2025) CT.gov search · Cochrane · PubMed	DGIdb	—
DESERPIDINE Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—
DESMOPRESSIN Repurposing via AVPR2	Small molecule	Approved	Strong	95	—	DGIdb	—
DEXAMETHASONE Repurposing via AGTR1	Small molecule	Approved	Strong	95	7 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing via ACE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via ACE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing via ACE	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DISULFIRAM Repurposing via AGTR1	Small molecule	Approved	Strong	95	2 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ENALAPRIL MALEATE Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—
ENALAPRILAT Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ENALAPRILAT ANHYDROUS Repurposing via ACE	Small molecule	Approved	Strong	95	—	DGIdb	—

Natural Products

13 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked). Only agents with direct clinical evidence are shown by default.

Reference lookups: Examine.com

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Links
Omega-3 Fatty Acids Natural product Animal Study Renoprotective effects of docosahexaenoic acid in cats with early chron…	Nutraceutical	Preclinical	Emerging	10	1 pubs View evidence CT.gov search · Cochrane · PubMed	PubChem
Curcumin Natural product Animal Study, In Vitro Study Combination of curcumin and ginkgolide B inhibits cystogenesis by regul… In Vitro Study Curcumin may have therapeutic value in polycystic kidney disease.	Food compound	Approved	Emerging	7	1 trials 2 Trial pubs 1 RCT View evidence Registry trials NCT02494141 — Curcumin Therapy to Treat Vascular Dysfunction in Children and Young Adults With ADPKD (COMPLETED) PHASE4 CT.gov search · Cochrane · PubMed	PubChem
Reishi Natural product Animal Study Ganoderic acid A is the effective ingredient of Ganoderma triterpenes i… Animal Study, In Vitro Study Ganoderma triterpenes and its purified monomer CBLZ-7 may be potential…	Mushroom	Preclinical	Emerging	7	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Alpha-Lipoic Acid Natural product Human Study Alpha-lipoic acid in patients with autosomal dominant polycystic kidney…	Nutraceutical	Preclinical	Emerging	7	1 Trial pubs View evidence CT.gov search · Cochrane · PubMed	PubChem
Ginkgo Biloba Natural product Animal Study, In Vitro Study Combination of curcumin and ginkgolide B inhibits cystogenesis by regul… Animal Study, In Vitro Study Ginkgolide B inhibits renal cyst development in in vitro and in vivo cy…	Botanical	Preclinical	Emerging	2	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Flaxseed Natural product Animal Study Dietary flax oil and feeding moderates renal injury in a rat model of p… Animal Study Dietary flaxseed supplementation ameliorates signs of polycystic kidney… Animal Study Dietary soy protein and flax oil reduces some disease-associated pathol…	Nutraceutical	Preclinical	Emerging	0	6 pubs View evidence CT.gov search · Cochrane · PubMed	—
Betaine Natural product Animal Study Betaine may have a therapeutic role in polycystic kidney disease.	Nutraceutical	Preclinical	Emerging	0	7 pubs View evidence CT.gov search · Cochrane · PubMed	—
CLA (Conjugated Linoleic Acid) Natural product Animal Study Dietary conjugated linolenic acid reduces renal inury in a rat model of…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Soy Protein Natural product Clinical trial publication The effect of eicosapentaenoic acid on renal function and volume in pat… Animal Study Dietary soy protein and flax oil reduces some disease-associated pathol…	Nutraceutical	Preclinical	Emerging	0	1 Trial pubs 1 RCT View evidence Published literature Clinical trial publication The effect of eicosapentaenoic acid on renal function and volume in patients with ADPKD (Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association, 2008) CT.gov search · Cochrane · PubMed	—
Hydrogen Water Natural product Animal Study Effects of hydrogen-rich water in a rat model of polycystic kidney dise…	Nutraceutical	Preclinical	Emerging	0	1 pubs View evidence CT.gov search · Cochrane · PubMed	—
Tripterygium Wilfordii Natural product Animal Study Triptolide, a compound found within Tripterygium wilfordii, attenuates…	Nutraceutical	Preclinical	Emerging	0	1 trials View evidence Registry trials NCT00801268 — Randomized Clinical Trial of Triptolide Woldifii for Autosomal Dominant Polycystic Kidney Disease (TERMINATED) NA CT.gov search · Cochrane · PubMed	—
Gambogic Acid Natural product In Vitro Study Potential application of gambogic acid for retarding renal cyst progres…	Nutraceutical	Preclinical	Emerging	0	3 pubs View evidence CT.gov search · Cochrane · PubMed	—
Garcinia Hanburyi Natural product In Vitro Study Potential application of gambogic acid for retarding renal cyst progres…	Nutraceutical	Preclinical	Emerging	0	1 pubs View evidence CT.gov search · Cochrane · PubMed	—

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.