Mode
Home
Research
Clinical Trials Drug Pipeline Weekly Digests
Reference
Conditions Medications
Community
Questions
Text Size
Log in / Sign up
Home › Conditions › Autosomal dominant polycystic kidney disease

Autosomal dominant polycystic kidney disease

Part of Kidney Diseases

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for Autosomal dominant polycystic kidney disease

10 trials tracked for Autosomal dominant polycystic kidney disease: 4 in phase 3 or 4 and 2 with published results. The most-cited published study has 635 citations.

10Trials tracked
4Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 4 1 Phase 3 3 Phase 2 6
  1. Phase 3 Efficacy and Safety of Tolvaptan in Subjects With Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease Completed · 635 cited
  2. Phase 3 Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD) Completed · 11 cited
  3. Phase 4 Efficacy, Safety and Tolerability of Everolimus in Preventing End-stage Renal Disease in Patients With Autosomal Dominant Polycystic Kidney Disease Completed
  4. Phase 3 A Long-term Administration Study of OPC-41061 in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) (2) [Extension of Study 156-05-002] Completed
  5. Phase 2 Pasireotide LAR in Severe Polycystic Liver Disease Completed
  6. Phase 2 Dose-finding Study of New Tolvaptan Formulation in Subjects With ADPKD Completed
Show 4 more trials
  1. Phase 2 8-Week Study of Tolvaptan Dose Forms in Autosomal Dominant Polycystic Kidney Disease (ADPKD) Completed
  2. Phase 2 Short-term Renal Hemodynamic Effects of Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD) Completed
  3. Phase 2 A Long-term Administration Study of OPC-41061 in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) [Extension of Study 156-04-001] Completed
  4. Phase 2 The ELiSA Study - Evaluation of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease Completed

Showing the 10 most-cited and recently-updated of 10 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Autosomal dominant polycystic kidney disease: what the trials found

Tolvaptan treatment is associated with significant changes in kidney volume and clinical progression markers in patients with ADPKD. Specifically, Tolvaptan was shown to result in a significantly higher percentage change per year in total kidney volume (TKV) compared to placebo (2.777% vs 5.608%, p<0.0001) 3. Additionally, Tolvaptan treatment resulted in a significantly greater number of clinical progression events per 100 follow-up years compared to placebo (43.94 vs 50.04, p=0.0095) 3.

Tolvaptan administration has been associated with faster declines in renal function and changes in kidney size. Studies demonstrated a significantly greater annual decrease in renal function per year for Tolvaptan compared to placebo (-2.555 vs -3.682, p<0.0001) 3. Furthermore, Tolvaptan MR was associated with significant reductions in total kidney volume (TKV) at both week 3 and week 8 7.

Tolvaptan treatment has been shown to impact patient-reported outcomes and physiological markers. Patients treated with Tolvaptan MR showed a statistically significant improvement in the Autosomal Dominant Polycystic Kidney Disease Urinary Impact Scale (ADPKD-UIS) 7. Additionally, Tolvaptan was associated with significant changes in measured glomerular filtration rate (mGFR) after 3 weeks of treatment 10, while effects on effective renal plasma flow (ERPF) and filtration fraction were not statistically significant 10.

Recent results — preliminary, needs further review

  • Lixivaptan is currently under investigation in a Phase 2 trial 5.
  • Pasireotide LAR showed no significant difference in estimated glomerular filtration rate (eGFR) change compared to control (p=0.79), but was associated with significant changes in liver and kidney volume 6.

For the clinician treating this condition

  • Tolvaptan is associated with a significantly higher rate of total kidney volume growth and an increased number of clinical progression events compared to placebo 3.
  • Tolvaptan MR has been shown to reduce total kidney volume (TKV) at 8 weeks and improve scores on the ADPKD-UIS scale 7.
  • While Tolvaptan impacts measured glomerular filtration rate (mGFR), it did not significantly alter effective renal plasma flow or filtration fraction in observed trials 10.

AI synthesis of 5 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
UK retrospective cohort study of tolvaptan prescribing in ADPKD reveals significant variation in initiation and substantial national costs
Nephrology Cohort
UK retrospective cohort study of tolvaptan prescribing in ADPKD reveals significant variation in initiation and substantial national costs UK Kidney Centers Miss Out on Life-Saving Drug for Millions
This retrospective cohort study analyzed tolvaptan prescribing patterns among 3,609 people with autosomal dominant polycystic kidney disease…
Over 12 million people with a genetic kidney disease worldwide are missing out on a life-saving drug because UK doctors aren't always prescr…
medRxiv Apr 9, 2026

Research across Kidney Diseases

Related studies from across the Kidney Diseases family.

Metabolic profiles and specific metabolites serve as potential biomarkers for diabetic kidney disease progression
Diabetes & Endocrinology Sys. Review
Metabolic profiles and specific metabolites serve as potential biomarkers for diabetic kidney disease progression Metabolites May Help Identify New Pathways in Diabetic Kidney Disease
This systematic review synthesizes the roles of metabolites as signaling hubs and substrates for epigenetic modifications in diabetic kidney…
New research identifies how specific metabolites act as signaling hubs that could help doctors better predict and treat diabetic kidney dise…
Frontiers Oct 24, 2026
Monocyte and macrophage sensing of calciprotein particles triggers inflammasome activation and interleukin-1β release
Allergy & Immunology Sys. Review
Monocyte and macrophage sensing of calciprotein particles triggers inflammasome activation and interleukin-1β release Immune Cells and Calcium Particles May Drive Damage in Kidney Disease
This mini-review synthesizes evidence regarding how monocytes and macrophages respond to calciprotein particles (CPPs). The review details m…
New research shows how certain immune cells react to calcium particles, potentially causing inflammation and tissue damage in kidney patient…
Frontiers Oct 24, 2026
Unhealthy food environments and ultra-processed foods contribute to obesity and related cardiometabolic diseases
Nutrition & Obesity Medicine Sys. Review
Unhealthy food environments and ultra-processed foods contribute to obesity and related cardiometabolic diseases Food Environments Linked to Obesity and Type 2 Diabetes
This narrative review examines how food environment factors like accessibility and affordability influence the consumption of ultra-processe…
Unhealthy food environments and ultra-processed foods are linked to serious conditions like insulin resistance, heart disease, and chronic k…
Frontiers Oct 23, 2026
suPAR predicts incident CKD and ESKD in adults with preserved kidney function
Allergy & Immunology Meta-analysis
suPAR predicts incident CKD and ESKD in adults with preserved kidney function suPAR Levels May Predict Risk of Chronic Kidney Disease
This systematic review of 31,416 patients found that suPAR predicts incident CKD (OR 3.13) and ESKD (HR 1.53) in adults with preserved kidne…
A large review suggests that suPAR levels can help predict the risk of developing chronic kidney disease and end-stage kidney disease in som…
Frontiers Oct 23, 2026
Resistance training improves eGFR in non-dialysis diabetic kidney disease while mind-body exercise reduces UAE
Diabetes & Endocrinology Meta-analysis
Resistance training improves eGFR in non-dialysis diabetic kidney disease while mind-body exercise reduces UAE Specific types of exercise may help manage diabetic kidney disease
This systematic review and network meta-analysis of 1,746 patients found that resistance training significantly increased eGFR compared to u…
Different types of exercise, from resistance training to mind-body practices, show different benefits for managing diabetic kidney disease a…
Frontiers Oct 19, 2026
RAAS inhibitors modulate inflammatory responses and oxidative stress across multiple chronic conditions
Drug Pipeline Sys. Review
RAAS inhibitors modulate inflammatory responses and oxidative stress across multiple chronic conditions RAAS Inhibitors May Protect Organs Beyond Lowering Blood Pressure
This systematic review synthesizes the roles of ACEI, ARB, DRI, ARNI, and MRA in modulating inflammatory responses and oxidative stress. The…
New research suggests certain blood pressure medications may protect the heart, kidneys, and lungs by reducing inflammation and oxidative st…
Frontiers Oct 16, 2026
Glutamine metabolism serves as a critical energy substrate and potential therapeutic target in AKI and CKD
Oncology Sys. Review
Glutamine metabolism serves as a critical energy substrate and potential therapeutic target in AKI and CKD Glutamine Metabolism May Offer New Insights for Kidney Disease
This narrative review explores the role of glutamine metabolism in acute kidney injury and chronic kidney disease. The authors suggest that …
New research into glutamine metabolism suggests potential new ways to manage and treat acute and chronic kidney diseases.
Frontiers Oct 14, 2026
Multiple biomarkers show potential associations with kidney function decline in patients with type 2 diabetes
Diabetes & Endocrinology Meta-analysis
Multiple biomarkers show potential associations with kidney function decline in patients with type 2 diabetes Specific Biomarkers May Signal Kidney Decline in Type 2 Diabetes
This systematic review and meta-analysis of 4,569 patients evaluated several biomarkers for detecting kidney function decline in type 2 diab…
New research identifies several biomarkers that may help detect early signs of kidney function decline in people with type 2 diabetes.
Frontiers Oct 12, 2026
Hemodiafiltration in Neonates: Narrative Review Maps Applications but Reports No Clinical Outcomes
Pediatrics Sys. Review
Hemodiafiltration in Neonates: Narrative Review Maps Applications but Reports No Clinical Outcomes Hemodiafiltration in Babies: What We Still Do Not Know
This narrative review examines hemodiafiltration (HDF) in neonates and infants, focusing on clinical applications, potential benefits, safet…
A new review looks at hemodiafiltration for newborns and infants, but reports no actual results or safety data.
Frontiers Oct 10, 2026
HIF-prolyl hydroxylase inhibitors cause modest VEGF increases and reproducible peripheral edema in chronic kidney disease
Nephrology Meta-analysis
HIF-prolyl hydroxylase inhibitors cause modest VEGF increases and reproducible peripheral edema in chronic kidney disease New data shows how kidney disease drugs affect blood levels
This meta-analysis of 34 randomized controlled trials evaluated HIF-prolyl hydroxylase inhibitors in patients with chronic kidney disease. T…
A review of 34 trials shows that a specific type of kidney medication increases a protein called VEGF, but also carries a risk of swelling.
Oct 10, 2026
Mineralocorticoid receptor antagonists exert cardioprotective effects by restoring coronary microvascular function
Cardiology Sys. Review
Mineralocorticoid receptor antagonists exert cardioprotective effects by restoring coronary microvascular function Mineralocorticoid Receptor Antagonists May Improve Heart Function in Heart Failure
This mini-review examines the mechanisms of mineralocorticoid receptor antagonists in patients with heart failure, hypertension, and chronic…
New research highlights how mineralocorticoid receptor antagonists may protect the heart by improving blood flow in small vessels for patien…
Frontiers Oct 7, 2026
Higher H3.1 nucleosome levels associate with 48% increased 28-day mortality in septic shock
Emergency Medicine RCT
Higher H3.1 nucleosome levels associate with 48% increased 28-day mortality in septic shock H3.1 Nucleosome Levels Linked to Sepsis Mortality and Kidney Risk
This secondary analysis of a multicenter randomized controlled trial involved 971 patients with sepsis and septic shock. Higher plasma H3.1 …
Higher levels of H3.1 nucleosomes in patients with sepsis and septic shock are linked to a higher risk of death and need for kidney replacem…
Oct 6, 2026