Biomarker & Intervention Discovery

Polycystic Kidney Disease (ADPKD)

This page lists 8 druggable gene targets, 47 approved or trial-tested drug candidates and 15 recent clinical trials for Polycystic Kidney Disease (ADPKD), with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

NIH funding$745MFY2025 · RCDC estimate
Spontaneous remissionProgressive; GFR decline averages 4-5 mL/min/year in Mayo Class 1C-1E; renal failure by 60-70 years in 50% without…
Best-intervention remissionTolvaptan (Jynarque): slows GFR decline by 35% and total kidney volume growth by 49% (TEMPO 3:4, REPRISE); dietary…
Gap sizeModerate — tolvaptan is available and effective but hepatotoxicity risk limits use; access barriers (REMS programme in…
Primary barrieraccess/practice
Full remission profile →
Research Tracker Chronic Disease Interventions Polycystic Kidney Disease (ADPKD)

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
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
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

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

AVPR2 234CFTR 222MTOR 347PKD1 4PKD2 7SRC 257SSTR2 269SSTR5 241

AVPR2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
MOZAVAPTAN ↓ inhibits Mechanistic 9.42 nM (Ki) DGIdb
D(CH2)5[TYR(ME)2]AVP ↓ inhibits Mechanistic DGIdb
D(CH2)5[D-ILE2,ILE4]AVP ↓ inhibits Mechanistic DGIdb
YM 471 ↓ inhibits Mechanistic DGIdb
BALOVAPTAN ↓ inhibits Mechanistic DGIdb
[TBAA1,D-TYR(ET)2,VAL4,LYS6,ARG-NH28,DES-GLY9]AVP ↓ inhibits Mechanistic DGIdb
[PHAA1,D-TYR(ET)2,VAL4,LYS6,TYR-NH28,DES-GLY9]AVP ↓ inhibits Mechanistic DGIdb
TOLVAPTAN ↓ inhibits Mechanistic 0.43 nM (Ki) DGIdb
VASOPRESSIN TANNATE ↑ activates Mechanistic DGIdb
OPC-51803 ? unclear Mechanistic DGIdb
D[CHA4]AVP ↑ activates Mechanistic DGIdb
DESMOPRESSIN ↑ activates Mechanistic 5.0 nM (Ki) DGIdb
[3H]AVP (HUMAN, MOUSE, RAT) ↑ activates Mechanistic DGIdb
CONIVAPTAN ↓ inhibits Mechanistic DGIdb
[PHAA1,D-TYR(ME)2,ARG6,TYR-NH29]AVP ↓ inhibits Mechanistic DGIdb
CLOTRIMAZOLE ? unclear Mechanistic DGIdb
[VAL4]AVP ↑ activates Mechanistic DGIdb
D[D-PAL2]AVP ↑ activates Mechanistic DGIdb
LIT-001 ↑ activates Mechanistic DGIdb
D(CH2)5[D-ILE2,VAL4,TYR-NH29]AVP ↓ inhibits Mechanistic DGIdb
D(CH2)5[D-ILE2,ILE4,ALA-NH29]AVP ↓ inhibits Mechanistic DGIdb
DAVP ↑ activates Mechanistic DGIdb
D[LEU4]AVP ↑ activates Mechanistic DGIdb
RELCOVAPTAN ↓ inhibits Mechanistic 178.0 nM (Ki) DGIdb
[125I]D(CH2)5[D-TYR(ET)2,ILE4,TYR-NH29]AVP ↓ inhibits Mechanistic DGIdb
D(CH2)5[D-TYR(ET)2,VAL4,TYR-NH29]AVP ↓ inhibits Mechanistic DGIdb
QUERCETIN ? unclear Mechanistic DGIdb
CONIVAPTAN HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
[PHAA1,D-TYR(ET)2,LYS6,DES-GLY9]AVP ↓ inhibits Mechanistic DGIdb
D(CH2)5[D-ILE2,ILE4,ALA-NH2]AVP ↓ inhibits Mechanistic DGIdb
OH-LVA ↓ inhibits Mechanistic DGIdb
RO5028442 ↓ inhibits Mechanistic DGIdb
D[VAL4,DARG8]VP ↑ activates Mechanistic DGIdb
[3H]SR 121463A ↓ inhibits Mechanistic DGIdb
VNA932 ↑ activates Mechanistic DGIdb
[PHE3]OT ↑ activates Mechanistic DGIdb
D(CH2)5[D-ILE2,ILE4,TYR-NH29]AVP ↓ inhibits Mechanistic DGIdb
TETRA-IODOPHENOLPHTHALEIN ? unclear Mechanistic DGIdb
D(CH2)5[TYR(ET)2,VAL4,DES-GLY9]AVP ↓ inhibits Mechanistic DGIdb
SATAVAPTAN ↓ inhibits Mechanistic DGIdb

Showing top 40 of 234 agents ranked by evidence tier.

CFTR

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
NPPB ? unclear Mechanistic DGIdb
LUMACAFTOR ~ modulates Mechanistic DGIdb
TEZACAFTOR ↑ activates Mechanistic DGIdb
SULFONAMIDE-01 ↑ activates Mechanistic DGIdb
SCRIPTAID ? unclear Mechanistic DGIdb
APIGENIN ↑ activates Mechanistic DGIdb
TEZACAFTOR AND IVACAFTOR ? unclear Mechanistic DGIdb
ROFLUMILAST ? unclear Mechanistic DGIdb
CROFELEMER ↓ inhibits Mechanistic DGIdb
UCCF-029 ↑ activates Mechanistic DGIdb
CURCUMIN ? unclear Mechanistic DGIdb
CAPSAICIN ↑ activates Mechanistic DGIdb
IVACAFTOR ↑ activates Mechanistic DGIdb
(R)-BPO-27 ↓ inhibits Mechanistic DGIdb
ICENTICAFTOR ↑ activates Mechanistic DGIdb
BAMOCAFTOR ? unclear Mechanistic DGIdb
UCCF-339 ↑ activates Mechanistic DGIdb
CFTRINH-172 ↓ inhibits Mechanistic DGIdb
NIMODIPINE ↑ activates Mechanistic DGIdb
CYSTEAMINE HYDROCHLORIDE ? unclear Mechanistic DGIdb
PHENYLGLYCINE-01 ↑ activates Mechanistic DGIdb
GLPG2737 ↑ activates Mechanistic DGIdb
GLYH-101 ↓ inhibits Mechanistic DGIdb
IOWH-032 ↓ inhibits Mechanistic DGIdb
OLACAFTOR ~ modulates Mechanistic DGIdb
ABBV-3221 ↑ activates Mechanistic DGIdb
DEUTIVACAFTOR ↑ activates Mechanistic DGIdb
NAVOCAFTOR ~ modulates Mechanistic DGIdb
CBIQ ↑ activates Mechanistic DGIdb
VITAMIN A COMPOUND ? unclear Mechanistic DGIdb
GATX1 ↓ inhibits Mechanistic DGIdb
GLPG-1837 ? unclear Mechanistic DGIdb
FELODIPINE ↑ activates Mechanistic DGIdb
ELEXACAFTOR ~ modulates Mechanistic DGIdb
ENTINOSTAT ? unclear Mechanistic DGIdb
VANZACAFTOR ↑ activates Mechanistic DGIdb
GLPG1837 ↑ activates Mechanistic DGIdb
GALICAFTOR ~ modulates Mechanistic DGIdb
NS004 ↑ activates Mechanistic DGIdb
GLPG-2737 ? unclear Mechanistic DGIdb

Showing top 40 of 222 agents ranked by evidence tier.

MTOR

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CC-115 ↓ inhibits Mechanistic DGIdb
APITOLISIB ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL24983 ? unclear Mechanistic DGIdb
PKI-179 ↓ inhibits Mechanistic DGIdb
BIMIRALISIB ↓ inhibits Mechanistic DGIdb
EVEROLIMUS ↓ inhibits Mechanistic DGIdb
DACTOLISIB ↓ inhibits Mechanistic DGIdb
PERPHENAZINE ? unclear Mechanistic DGIdb
INDOXIMOD ↑ activates Mechanistic DGIdb
N-OLEOYLDOPAMINE ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL527620 ? unclear Mechanistic DGIdb
LESTAURTINIB ? unclear Mechanistic DGIdb
CANERTINIB ? unclear Mechanistic DGIdb
GOSSYPOL ? unclear Mechanistic DGIdb
GEDATOLISIB ↓ inhibits Mechanistic DGIdb
METFORMIN HYDROCHLORIDE ? unclear Mechanistic DGIdb
SONOLISIB ↓ inhibits Mechanistic 3100.0 nM (IC50) DGIdb
WIN55212-2 ? unclear Mechanistic DGIdb
PP-121 ↓ inhibits Mechanistic DGIdb
EBASTINE ? unclear Mechanistic DGIdb
HALOPERIDOL DECANOATE ? unclear Mechanistic DGIdb
SAPANISERTIB ↓ inhibits Mechanistic DGIdb
TAFA-93 ? unclear Mechanistic DGIdb
TEMSIROLIMUS ↓ inhibits Mechanistic DGIdb
OSI-027 ↓ inhibits Mechanistic DGIdb
CALCIMYCIN ? unclear Mechanistic DGIdb
VOXTALISIB ↓ inhibits Mechanistic DGIdb
PF-04691502 ? unclear Mechanistic DGIdb
COPANLISIB ↓ inhibits Mechanistic DGIdb
NEOLYMPHOSTIN A ↓ inhibits Mechanistic DGIdb
LY-294002 ? unclear Mechanistic 2500.0 nM (IC50) DGIdb
VS-5584 ↓ inhibits Mechanistic DGIdb
ZANDELISIB ↓ inhibits Mechanistic DGIdb
COMPOUND 7 [PMID: 31955578] ↓ inhibits Mechanistic DGIdb
GDC-0349 ? unclear Mechanistic DGIdb
DOXAZOSIN MESYLATE ? unclear Mechanistic DGIdb
VISTUSERTIB ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL600334 ? unclear Mechanistic DGIdb
EMETINE ? unclear Mechanistic DGIdb
CAMPTOTHECIN ? unclear Mechanistic DGIdb

Showing top 40 of 347 agents ranked by evidence tier.

PKD1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL5188373 ↓ inhibits Mechanistic 81.0 nM (IC50) ChEMBL
CHEMBL5405484 ↓ inhibits Mechanistic 64.0 nM (Ki) ChEMBL
CHEMBL4209095 ↓ inhibits Mechanistic 89.0 nM (Ki) ChEMBL
CHEMBL4211840 ↓ inhibits Mechanistic 66.0 nM (Ki) ChEMBL

PKD2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
NI2+ ↓ inhibits Mechanistic DGIdb
SKF96365 ↓ inhibits Mechanistic DGIdb
CD2+ ↓ inhibits Mechanistic DGIdb
CA2+ ↑ activates Mechanistic DGIdb
ALLOPURINOL ? unclear Mechanistic DGIdb
CHEMBL5562073 ↓ inhibits Mechanistic 40280.0 nM (IC50) ChEMBL
STAUROSPORINE ↓ inhibits Mechanistic 910.0 nM (IC50) ChEMBL

SRC

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic DGIdb
AZD-0424 ↓ inhibits Mechanistic DGIdb
DASATINIB ANHYDROUS ↓ inhibits Mechanistic DGIdb
XL-228 ↓ inhibits Mechanistic DGIdb
IBRUTINIB ↓ inhibits Mechanistic DGIdb
PP-121 ↓ inhibits Mechanistic DGIdb
PONATINIB ? unclear Mechanistic DGIdb
ELZOVANTINIB ↓ inhibits Mechanistic DGIdb
MLN-8054 ? unclear Mechanistic DGIdb
TIRBANIBULIN ↓ inhibits Mechanistic DGIdb
SARACATINIB ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL374680 ? unclear Mechanistic DGIdb
N-(5-METHYL-1H-PYRAZOL-3-YL)-2-PHENYLQUINAZOLIN-4-AMINE ↓ inhibits Mechanistic DGIdb
KX2-361 ↓ inhibits Mechanistic DGIdb
KENPAULLONE ? unclear Mechanistic DGIdb
CCT196969 ↓ inhibits Mechanistic DGIdb
PP2 ? unclear Mechanistic DGIdb
LINIFANIB ? unclear Mechanistic DGIdb
AZM-475271 ↓ inhibits Mechanistic DGIdb
XL228 ↓ inhibits Mechanistic DGIdb
ILORASERTIB ↓ inhibits Mechanistic DGIdb
COMPOUND 36 [PMID: 21958547] ↓ inhibits Mechanistic DGIdb
TAK-020 ↓ inhibits Mechanistic DGIdb
CEDIRANIB ? unclear Mechanistic DGIdb
BALAMAPIMOD ↓ inhibits Mechanistic DGIdb
XL999 ↓ inhibits Mechanistic DGIdb
BOSUTINIB ↓ inhibits Mechanistic 1.3 nM (IC50) DGIdb
PF-562271 ? unclear Mechanistic DGIdb
NVP-TAE684 ? unclear Mechanistic DGIdb
TYRPHOSTIN A9 ? unclear Mechanistic DGIdb
JNJ-26483327 ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL230686 ? unclear Mechanistic DGIdb
CRENOLANIB ? unclear Mechanistic DGIdb
VODOBATINIB ? unclear Mechanistic DGIdb
COMPOUND 19A [PMID: 30503936] ↓ inhibits Mechanistic DGIdb
PD-0166285 HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
COMPOUND 8H [PMID: 22765894] ↓ inhibits Mechanistic DGIdb
CCT241161 ↓ inhibits Mechanistic DGIdb
CENISERTIB ? unclear Mechanistic DGIdb
CLOZAPINE ? unclear Mechanistic DGIdb

Showing top 40 of 257 agents ranked by evidence tier.

SSTR2

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
SIFA-ASN(ACNH-Β-GLC)-PEG-TYR3-OCTREOTATE ↑ activates Mechanistic DGIdb
BIM 23027 ↑ activates Mechanistic DGIdb
BOC-ATE ↑ activates Mechanistic DGIdb
SOMATOSTATIN ↑ activates Mechanistic 0.1995 nM (Ki) DGIdb
DOTA-BASS ↓ inhibits Mechanistic DGIdb
[125I]CGP 23996 ↑ activates Mechanistic DGIdb
[GA-DOTA,TYR3,THR8]OCTREOTIDE ↑ activates Mechanistic DGIdb
ANALOG 30 [PMID:18543899] ↓ inhibits Mechanistic DGIdb
Y-KE88 ↑ activates Mechanistic DGIdb
L-363,377 ↑ activates Mechanistic DGIdb
BIM 23060 ↑ activates Mechanistic DGIdb
CST-17 ↑ activates Mechanistic DGIdb
L-362,855 ↑ activates Mechanistic DGIdb
PALTUSOTINE ↑ activates Mechanistic DGIdb
BIM 23056 ↑ activates Mechanistic DGIdb
BIM 23068 ↑ activates Mechanistic DGIdb
L-363,301 ↑ activates Mechanistic DGIdb
SIFA-ASN(ACNH-Β-GLC)-TYR3-OCTREOTATE ↑ activates Mechanistic DGIdb
CGP 23996 ↑ activates Mechanistic DGIdb
BIM 23059 ↑ activates Mechanistic DGIdb
BIM 23197 ↑ activates Mechanistic DGIdb
SRIF-14 ↑ activates Mechanistic DGIdb
[125I]TYR3 SMS 201-995 ↑ activates Mechanistic DGIdb
AM3 ↑ activates Mechanistic DGIdb
NC 4-28B ↑ activates Mechanistic DGIdb
BIM 23030 ↑ activates Mechanistic DGIdb
THERAPEUTIC HORMONE ? unclear Mechanistic DGIdb
GA-KE88 ↑ activates Mechanistic DGIdb
VELDOREOTIDE ↑ activates Mechanistic DGIdb
PASIREOTIDE ↑ activates Mechanistic 1.0 nM (Ki) DGIdb
VAPREOTIDE ↑ activates Mechanistic 0.07943 nM (Ki) DGIdb
[111IN,90Y]DOTA-TOC ↑ activates Mechanistic DGIdb
ANALOG 32 [PMID:18543899] ↓ inhibits Mechanistic DGIdb
L-779,976 ↑ activates Mechanistic DGIdb
NOC-ATE ↑ activates Mechanistic DGIdb
BIM 23052 ↑ activates Mechanistic DGIdb
LANREOTIDE ACETATE ↑ activates Mechanistic DGIdb
[D-TYR8]CYN 154806 ↓ inhibits Mechanistic DGIdb
OCTREOTIDE ACETATE ↑ activates Mechanistic DGIdb
EDOTREOTIDE ↑ activates Mechanistic DGIdb

Showing top 40 of 269 agents ranked by evidence tier.

SSTR5

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
BIM 23050 ↑ activates Mechanistic DGIdb
BIM 23454 ↓ inhibits Mechanistic DGIdb
BIM 23052 ↑ activates Mechanistic DGIdb
ONZIGOLIDE ↑ activates Mechanistic DGIdb
SOMATOSTATIN ↑ activates Mechanistic 0.3981 nM (Ki) DGIdb
PRL-2970 ↓ inhibits Mechanistic DGIdb
L-363,301 ↑ activates Mechanistic DGIdb
[D-TYR8]CYN 154806 ↓ inhibits Mechanistic DGIdb
[111IN,90Y]DOTA-TOC ↑ activates Mechanistic DGIdb
BIM 23060 ↑ activates Mechanistic DGIdb
OCTREOTIDE ACETATE ↑ activates Mechanistic DGIdb
PASIREOTIDE ↑ activates Mechanistic 0.1259 nM (Ki) DGIdb
NOC-ATE ↑ activates Mechanistic DGIdb
PASIREOTIDE DIASPARTATE ↑ activates Mechanistic DGIdb
BIM 23023 ↑ activates Mechanistic DGIdb
[125I]TYR11-SRIF-14 ↑ activates Mechanistic DGIdb
CGP 23996 ↑ activates Mechanistic DGIdb
SRIF-14 ↑ activates Mechanistic DGIdb
LUTETIUM OXODOTREOTIDE LU-177 ~ modulates Mechanistic DGIdb
BIM 23268 ↑ activates Mechanistic DGIdb
SEGLITIDE ↑ activates Mechanistic 12.59 nM (Ki) DGIdb
BIM 23313 ↑ activates Mechanistic DGIdb
BIM 23197 ↑ activates Mechanistic DGIdb
S5A1 ↓ inhibits Mechanistic DGIdb
KE 108 ↑ activates Mechanistic DGIdb
DES-AA5-[D-TRP8]SRIF ↑ activates Mechanistic DGIdb
CST-17 ↑ activates Mechanistic DGIdb
[125I]CGP 23996 ↑ activates Mechanistic DGIdb
[111IN,90Y]DOTA-LAN ↑ activates Mechanistic DGIdb
[111IN,90Y]DOTA-NOC ↑ activates Mechanistic DGIdb
BIM 23056 ↓ inhibits Mechanistic DGIdb
[125I]LTT-SRIF-28 ↑ activates Mechanistic DGIdb
[111IN]DOTA-NOC-ATE ↑ activates Mechanistic DGIdb
PRL-2915 ↓ inhibits Mechanistic DGIdb
L-362,855 ↑ activates Mechanistic DGIdb
ZAVOLOSOTINE ↑ activates Mechanistic DGIdb
SRIF-28 ↑ activates Mechanistic DGIdb
[125I]TYR10-CST14 ↑ activates Mechanistic DGIdb
[125I]TYR3 SMS 201-995 ↑ activates Mechanistic DGIdb
VAPREOTIDE ↑ activates Mechanistic 5.012 nM (Ki) DGIdb

Showing top 40 of 241 agents ranked by evidence tier.

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Polycystic Kidney Disease (ADPKD): recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

Safety, Tolerability and Pharmacokinetics of AZD1613 in Adults With Autosomal Dominant Polycystic Kidney Disease Recruiting
NCT07228364 Phase: 1 Started: 2025-11-10

A study to investigate safety, tolerability, and pharmacokinetics of AZD1613 following subcutaneous or intravenous administration in participants with autosomal dominant polycystic kidney disease (ADPKD).

ANCHOR Study: A Study to Assess the Safety and Efficacy of ABBV-CLS-628 in Adult Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD) Recruiting
NCT06902558 Phase: 2 Started: 2025-06-09

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic cause of kidney disease that causes fluid-filled cysts to develop in the kidneys. The purpose of this study is to assess the safety and efficacy of ABBV-CLS-628 for the treatment of ADPKD in adult participants. ABBV-CLS-...

Effects of VX-407 on the Pharmacokinetics of Combined Oral Contraceptives Recruiting
NCT07792408 Phase: 1 Started: 2026-09-01

The purpose of the study is to evaluate the pharmacokinetic effects and safety and tolerability of VX-407 when coadministered with oral contraceptives.

Bempedoic Acid Therapy for Polycystic Kidney Disease Recruiting
NCT07282821 Phase: 2 Started: 2026-09

Autosomal dominant polycystic kidney disease (ADPKD), the most commonly inherited kidney disease, is characterized by the development of cysts in the kidney that impair function. Of those affected, half will progress to end-stage kidney disease by age 60, requiring dialysis or kidney transplant. To ...

Short-term Effects of an SGLT2 Inhibitor on Divalent Ions in Autosomal Dominant Polycystic Kidney Disease Completed
NCT06435858 Phase: 2 Started: 2024-09-01

This study aims to better understand electrolyte handling in patients with autosomal dominant polycystic kidney disease treated with the SGLT2 inhibitor Empagliflozin. Patients will be randomized into two groups and take Empagliflozin or a Placebo for 2 weeks with a wash-out period of 2 weeks. The p...

Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW) Active Not Recruiting
NCT07161037 Phase: 2 Started: 2025-11-19

The purpose of the study is to evaluate the effect of VX-407 on height-adjusted total kidney volume (htTKV), safety, tolerability, and pharmacokinetics (PK) of VX-407.

A Phase 1 Study of Single and Multiple Ascending Doses of GV-100 Recruiting
NCT07788014 Phase: 1 Started: 2026-06-23

This is a first-in-human, multi-part clinical study designed to evaluate the safety, tolerability, pharmacokinetics (PK), food effect, and drug-drug interaction (DDI) potential of GV-100 following oral administration in healthy participants. The study is divided into four parts: Single Ascending Dos...

HYDROchlorothiazide to PROTECT Polycystic Kidney Disease Patients and Improve Their Quality of Life Recruiting
NCT05373264 Phase: 3 Started: 2024-07-31

Autosomal dominant polycystic kidney disease (ADPKD) is characterized by progressive formation of renal cysts which ultimately lead to a loss of renal function. Tolvaptan (a V2R antagonist) is currently the only effective treatment for preserving renal function in ADPKD. However, side-effects such a...

Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003 Recruiting
NCT06714006 Phase: 1 Started: 2025-04-07

This is a Phase 1, First-in-Human study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and immunogenicity of PYC-003 in healthy adult participants and adult participants with confirmed PKD1 mutation-associated Autosomal Dominant Polycystic Kidney Disease (ADPKD) T...

Implementation of Metformin theraPy to Ease Decline of Kidney Function in Polycystic Kidney Disease (IMPEDE-PKD) Recruiting
NCT04939935 Phase: 3 Started: 2022-11-29

This study will investigate if a medication (metformin) widely used in the treatment of diabetes could be re-purposed for the treatment of patients with a diagnosis of early stage ADPKD to slow the rate of kidney function decline, reducing morbidity and mortality and improving the quality of life fo...

Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy Participants Completed
NCT07074327 Phase: 1 Started: 2025-07-11

The purpose of the study is to evaluate the effect of VX-407 on the pharmacokinetics of levonorgestrel (LNG) and ethinyl estradiol (EE), norgestimate (NGM) and EE, norethindrone (NET) and EE and drospirenone (DRSP) and EE. Also, to evaluate the safety and tolerability of co-administration of VX-407 ...

A Phase 1 Study Evaluating Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants Completed
NCT07022119 Phase: 1 Started: 2025-06-12

The purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of VX-407 in healthy participants.

Metabolic Impacts of Ren-Nu: A Dietary Program for Polycystic Kidney Disease Recruiting
NCT07454174 Phase: N/A Started: 2026-02-25

This is a 16 week pilot study of the impact of a nutritionist led ketogenic diet (Ren-Nu) supplemented with the medical food KetoCitra on autosomal dominant polycystic kidney disease.

Pravastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease Completed
NCT04284657 Phase: 2 Started: 2019-01-30

This is an one-year open-label study to determine treatment efficacy and feasibility of a trial that uses open-label interventions in ADPKD patients.

A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants Completed
NCT06345755 Phase: 1 Started: 2024-04-17

The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetic parameters of VX-407 in healthy participants.

Agents Found By Disease-Level Search

Searched directly from “Polycystic Kidney Disease (ADPKD)” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 8-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

47 agents shown (approved or with trial evidence) — 39 not found by the gene-target search above.

tolvaptan

Approved · 10 trials · source: ClinicalTrials.gov, Open Targets
NCT01280721 NCT02847624 NCT03949894

Tolvaptan MRNOT in gene-target list

· 5 trials · source: ClinicalTrials.gov
NCT01451827 NCT01210560 NCT01210560

VX-407NOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT07161037 NCT07792408 NCT07022119

Sirolimus

· 3 trials · source: ClinicalTrials.gov
NCT00346918 NCT01632605 NCT00491517

Octreotide

Phase 2/3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00426153 NCT06193616

Metformin

· 2 trials · source: ClinicalTrials.gov
NCT02903511 NCT02656017

Bosutinib

· 2 trials · source: ClinicalTrials.gov
NCT01233869 NCT01233869

Everolimus

· 2 trials · source: ClinicalTrials.gov
NCT00414440 NCT02134899

LisinoprilNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00283686 NCT01885559

TelmisartanNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00283686 NCT01885559

Octreotide-LARNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01377246 NCT03541447

octeotrideNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT02119052 NCT02119013

TesevatinibNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01559363 NCT03203642

Lanreotide

Unknown · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00565097 NCT02127437

Tolvaptan IRNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01451827 NCT01210560

Daily caloric restrictionNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT04907799 NCT06496542

TirzepatideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06582875

LNG/EENOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07792408

NGM/EENOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07792408

NET/EENOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07792408

DRSP/EENOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07792408

DietaryNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04534985

AZD1613 - Part ANOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07228364

AZD1613 - Part BNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07228364

Shared decision making modelNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06618638

octreotide (CAM2029)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT05281328

Tolvaptan SuspensionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04782258

Tolvaptan TabletsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04782258

Hydrochlorothiazide 25 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05373264

Calcineurin inhibitors maintenanceNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02134899

RGLS4326NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04536688

tolvaptan (OPC-41061)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT00841568

Sodium chloride (NaCl)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05228574

Amiloride Hcl 5mg TabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05228574

Lixivaptan

· 1 trial · source: ClinicalTrials.gov
NCT03487913

Digital NeuropathyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07745920

Pasireotide LARNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01670110

SpironolactoneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01853553

Sugar pillNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01853553

Metformin XRNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04939935

Weight LossNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03342742

Long-acting somatostatinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00309283

PioglitazoneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02697617

RapamuneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00286156

EmpagliflozinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05510115

TamibaroteneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06289998

Tolvaptan (OPC-41061)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02160145

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.