ADPKD Guide
Readable, evidence-first guidance for patients, families, and clinicians.
Evidence reviewed Aug 24, 2026
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Evidence reviewed Aug 24, 2026
For clinicians
Research and Trials Reviewed August 24, 2026

ADPKD research without the hype

Current trials, recent human evidence, and a clear line between an active study and a proven treatment.

Evidence reviewed: August 24, 2026
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What changed in this review

Evidence corrected

SGLT2 benefit remains uncertain

A 2026 systematic review and meta-analysis found that the available clinical evidence is too small and heterogeneous to establish kidney benefit in ADPKD. KDIGO 2025 does not recommend SGLT2 inhibitors specifically for slowing ADPKD progression.

Trial status corrected

AGLOW is active, not recruiting

VX-407 is an oral investigational drug for a subset of PKD1 variants. It is not gene replacement or gene editing, and no efficacy result is posted.

Registry refreshed

Several large studies are recruiting

STOP-PKD, HYDRO-PROTECT, IMPEDE-PKD, JMKX003142, and ANCHOR are among the higher-priority active studies. Recruiting status is not proof that an intervention works.

Registry checked August 24, 2026

Priority clinical trials

Search the full registry
Phase 3 RECRUITING

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

Intervention: Dapagliflozin 10 mg vs placebo

Large pivotal non-V2 trial; registry refreshed 2026-07-28; no efficacy outcomes yet and KDIGO currently does not recommend SGLT2i specifically for ADPKD progression slowing

NCT07280585 on ClinicalTrials.gov
Phase 3 RECRUITING

HYDRO-PROTECT: hydrochlorothiazide add-on to tolvaptan in ADPKD

Intervention: Hydrochlorothiazide 25 mg vs placebo (on background V2RA)

Large multicenter quadruple-masked RCT; registry refreshed 2026-08-14; no efficacy outcomes posted yet

NCT05373264 on ClinicalTrials.gov
Phase 3 RECRUITING

IMPEDE-PKD: Metformin therapy to ease decline of kidney function in ADPKD

Intervention: Metformin XR vs control

Large confirmatory trial in progress; KDIGO currently recommends against metformin specifically for ADPKD progression in non-diabetes

NCT04939935 on ClinicalTrials.gov
Phase 2 RECRUITING

Trial of JMKX003142 in Participants With Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Intervention: JMKX003142

Randomized double-blind phase-2 candidate in rapidly progressive ADPKD; no efficacy results posted yet

NCT06800651 on ClinicalTrials.gov
Phase 2 RECRUITING

ANCHOR Study: A Study to Assess the Safety and Efficacy of ABBV-CLS-628 in Adult Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Intervention: ABBV-CLS-628 vs placebo (IV infusions)

Active phase-2 drug program; registry refreshed 2026-08-05; no efficacy results posted yet, so remains early signal only

NCT06902558 on ClinicalTrials.gov
Phase 2a ACTIVE NOT RECRUITING

VX-407 Phase 2a in ADPKD with selected PKD1 variants (AGLOW)

Intervention: VX-407 oral tablets

Active but not recruiting; oral genotype-targeted investigational drug, not gene replacement or gene editing; no efficacy results posted; open-label single-arm design limits causal inference

NCT07161037 on ClinicalTrials.gov

Eligibility depends on study location, age, kidney function, current treatment, and other criteria. Discuss participation with the study team and your nephrologist.

The cure research reality

There is no clinically proven cure for ADPKD. Our current registry review did not identify a human ADPKD gene-replacement or gene-editing treatment trial.

Genotype-targeted drugs

VX-407 is an oral investigational drug being studied in adults with selected PKD1 variants. Targeting a genetic subgroup is precision medicine, but it is not gene therapy and it has not been shown to correct the gene.

RNA-targeting programs

Early human programs are testing whether changing disease-related RNA signaling can affect cyst growth. These are investigational and not cures.

Gene correction

Gene-editing and gene-activation work remains preclinical. Cell and animal results cannot establish safety or benefit in people.

Recent published evidence

SGLT2 inhibitor systematic review and meta-analysis

Six small clinical studies with 451 participants did not establish a clear benefit against non-SGLT2 controls or a change in total kidney volume. The authors judged the evidence insufficient.

PubMed PMID 42586506

Tolvaptan and inflammatory markers

A retrospective 67-patient study linked tolvaptan use with lower inflammatory markers and slower kidney-volume growth. The design cannot prove cause and needs prospective confirmation.

PubMed PMID 42347886

Kidney stones and kidney failure risk

In a prospective cohort of 410 people, imaging-confirmed kidney stones were independently associated with a higher risk of kidney failure. This is an association, not proof that stones caused progression.

PubMed PMID 42428517

Health-related quality of life

A systematic review found that physical quality of life worsens with CKD stage and that disease-specific questionnaires are more sensitive to the ADPKD experience.

PubMed PMID 42232587

How to read research claims

Recruitingmeans a study is looking for participants, not that the intervention works.

Early phaseusually focuses on safety, dose, and biological signals.

Kidney volumecan be useful, but it does not replace long-term kidney function and safety outcomes.

Observational resultcan support a hypothesis, but cannot remove treatment-selection and confounding bias.

How evidence is graded Search all references Review update log
Medical Disclaimer

This resource provides educational information about ADPKD and is not a substitute for professional medical advice. Always discuss treatment decisions, symptoms, and health concerns with your nephrologist or primary care physician before making any changes.

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Related Pages

Treatment Overview
Current standards of care
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What Changed
Review the evidence update log
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Methodology
How we evaluate evidence
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