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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 619 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 · 619 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.

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