Summary
Atorvastatin has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Polycystic Kidney Disease (ADPKD) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Polycystic Kidney Disease (ADPKD). ClinicalTrials.gov lists 1 registered trial linking Atorvastatin to Polycystic Kidney Disease (ADPKD): 1 is currently recruiting. The most advanced is Phase 2 (NCT05870007). It plans or enrolled 30 participants. Registered activity dates to 2023. No published literature item is linked to Atorvastatin and Polycystic Kidney Disease (ADPKD) in the OSMF database yet, so the record rests on registry entries alone. Registry entries describe intent to study, not outcomes.
The RepurpOS disease-intelligence file for Polycystic Kidney Disease ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: 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.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 24.0 (trials 4.0, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 1 total: 1 recruiting, 0 active / not yet recruiting, 0 completed, 0 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | AGTR1 |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT05870007 | Atorvastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease 2023 | enrolling by invitation | Phase 2 | 30 |
Published literature
No publication is linked to this pair yet.
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target AGTR1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Atorvastatin approved for Polycystic Kidney Disease (ADPKD)?
Atorvastatin has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Polycystic Kidney Disease (ADPKD) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Polycystic Kidney Disease (ADPKD). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Atorvastatin in clinical trials for Polycystic Kidney Disease (ADPKD)?
ClinicalTrials.gov lists 1 registered trial linking Atorvastatin to Polycystic Kidney Disease (ADPKD): 1 is currently recruiting. The most advanced is Phase 2 (NCT05870007). It plans or enrolled 30 participants. Registered activity dates to 2023.
What does the evidence show for Atorvastatin in Polycystic Kidney Disease (ADPKD)?
Trials of Atorvastatin in Polycystic Kidney Disease (ADPKD) are registered but none has completed, so there is no outcome evidence from those studies yet; the record is a signal of research interest. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target AGTR1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.