What may slow ADPKD — evidence ranked from strongest to weakest

Best-supported disease-modifying therapy in selected rapid progressors

KDIGO supports initiation in adults at risk of rapid progression (with strict LFT monitoring and contraindication screening)

Core progression/safety strategy in guideline care

KDIGO target details now extracted (younger CKD G1-G2 intensive home-BP target when tolerated; older/CKD G3-G5 standardized SBP target <120)

Strong supportive strategy with guideline backing

KDIGO links lower osmolar intake with reduced aquaretic burden during tolvaptan use

Early positive human signal but not practice-changing yet

Small randomized crossover trial showed improved surrogate trajectories; confirmation needed

Observational human signal strengthened but still not guideline-ready

Propensity-matched TrinetX cohort associated SGLT2i use with lower dialysis initiation (HR 0.35) in nondiabetic ADPKD

High-priority confirmatory strategy but unproven until outcomes read out

Phase-3 quadruple-masked RCT recruiting (NCT05373264); do not infer disease-modifying benefit before endpoint data

Emerging non-V2 candidate under phase-2 evaluation

No ADPKD efficacy outcomes available yet (NCT07282821)

Promising metabolic hypothesis under trial

Phase-2 ADPKD trial recruiting with htTKV endpoint (NCT06582875)

Experimental candidate with active phase-2 study

No posted efficacy results yet (NCT06289998)

Active phase-2 pipeline candidate; efficacy unknown

Recruiting phase-2 placebo-controlled trial in adults with ADPKD; TKV trajectory and safety endpoints pending (NCT06902558)

Exploratory phase-2 safety-focused strategy; efficacy unproven

Not-yet-recruiting phase-2 trial; primary endpoint is safety/tolerability rather than definitive renal efficacy (NCT06291116)

Newly tracked early-phase ADPKD candidate with safety PK-only human data so far

Phase-1 placebo-controlled ADPKD trial recruiting; no renal efficacy outcomes posted (NCT07228364)

Exploratory intravenous phase-1 ADPKD candidate focused on safety/tolerability

Recruiting phase-1 ADPKD study with safety endpoints; no renal efficacy outcomes available (NCT06714006)

First-in-class microRNA-17 inhibitor with Phase Ib data showing efficacy in cyst/kidney size reduction

Phase Ib MAD trial (completed 2025) showed promising reduction of cyst growth and kidney size progression. Novartis acquisition (March 2026) signals confidence. Phase II planned. Mechanism: miR-17 has been implicated in polycystic kidney pathogenesis; this represents first-in-human clinical data for miR-17 targeting in ADPKD. Full Phase Ib peer-reviewed data publication pending.