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Disease

Polycystic Kidney, Autosomal Dominant

Late-stage therapeutic developmentEmerging research
MeSHPolycystic Kidney, Autosomal Dominant
2
Publications
9
Clinical trials
2023
Latest publication
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 1 clinical trial expected to report results, the earliest in Q2 2030.
Upcoming Milestones
Major developments
Upcoming trial readoutHigh impact
STOP-PKD: SGLT2-inhibition to Improve Prognosis in Polycystic Kidney Disease
Results expected Q2 20302026-06-15
Major clinical milestoneImportant
Feasibility of Study of Empagliflozin in Patients With Autosomal Dominant Polycystic Kidney Disease
Primary completion2026-03-18
Clinical Milestones2View
Results expected 1
Recently completed 1
Activity timeline2

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Tolvaptanapproved

Approval — Jinarc is indicated to slow the progression of cyst development and renal insufficiency o… (2015)

Clinical trials

8 sponsors · 1 new · 0 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme
9
All trials
2
Active
6
Late-stage
6
Completed
Late-stage studies

STOP-PKD: SGLT2-inhibition to Improve Prognosis in Polycystic Kidney Disease

Phase 3 · Recruiting · University of Cologne

Curcumin Therapy to Treat Vascular Dysfunction in Children and Young Adults With ADPKD

Phase 4 · Completed · University of Colorado, Denver

A Phase 3b, Multi-center, Open-label Trial to Evaluate the Long Term Safety of Immediate-release Tolvaptan (OPC-41061, 30 mg to 120 mg/Day, Split Dose) in Subjects With Autosomal Dominant Polycystic Kidney Disease

Phase 3 · Completed · Otsuka Pharmaceutical Development & Commercialization, Inc.

Effect of Tolvaptan on Renal Plasma Flow (RPF) and Glomerular Filtration Rate (GFR) in ADPKD

Phase 3 · Completed · Regional Hospital Holstebro

Pilot Study Of Long-Acting Octreotide (Octreotide LAR® Depot) In The Treatment Of Patients With Severe Polycystic Liver Disease

Phase 2/3 · Completed · Mayo Clinic

Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Phase 2/3 · Terminated · Genzyme, a Sanofi Company
Recruiting

STOP-PKD: SGLT2-inhibition to Improve Prognosis in Polycystic Kidney Disease

Phase 3 · Recruiting · University of Cologne
Recently completed

Curcumin Therapy to Treat Vascular Dysfunction in Children and Young Adults With ADPKD

Phase 4 · Completed · University of Colorado, Denver

Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease

Phase 2 · Completed · Kyongtae Ty Bae, M.D., Ph.D.

A Phase 3b, Multi-center, Open-label Trial to Evaluate the Long Term Safety of Immediate-release Tolvaptan (OPC-41061, 30 mg to 120 mg/Day, Split Dose) in Subjects With Autosomal Dominant Polycystic Kidney Disease

Phase 3 · Completed · Otsuka Pharmaceutical Development & Commercialization, Inc.

Effect of Tolvaptan on Renal Plasma Flow (RPF) and Glomerular Filtration Rate (GFR) in ADPKD

Phase 3 · Completed · Regional Hospital Holstebro

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Of The Safety, Clinical Activity And Pharmacokinetics Of Bosutinib (PF-05208763) Versus Placebo In Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Phase 2 · Completed · Pfizer

Pilot Study Of Long-Acting Octreotide (Octreotide LAR® Depot) In The Treatment Of Patients With Severe Polycystic Liver Disease

Phase 2/3 · Completed · Mayo Clinic

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2015emaApproval — Tolvaptan· Jinarc is indicated to slow the progression of cyst development and renal insufficiency of autosomal dominant polycystic kidney disease (ADPKD) in adults with CKD stage 1 to 3 at initiation of treatment with evidence of rapidly progressing disease. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Research activity

2 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20122023
Major themes5
  • Genetic Loci1
  • Mutation, Missense1
  • Polycystic Kidney Diseases1
  • Polycystic Kidney, Autosomal Dominant1
  • Protein Folding1
Leading journals2
  • JCI insight1
  • The Journal of clinical investigation1
Leading researchers8
  • Hopp K2
  • Bauer CD1
  • Berger MD1
  • Chonchol MB1
  • Clambey ET1
  • Furgeson SB1
  • Gainullin VG1
  • Gitomer BY1
Affiliations (unnormalised)3
  • Mayo Clinic1
  • University of Colorado Anschutz Medical Campus1
  • University of Oklahoma Health Sciences Center1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Kidney disorders with autosomal dominant inheritance and characterized by multiple CYSTS in both KIDNEYS with progressive deterioration of renal function.

Identifiers
MeSHPolycystic Kidney, Autosomal Dominant
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.