Clinical Trials

Clinical trials for Polycystic Kidney Disease are underway around the world to evaluate potential new treatments. You can find these studies by using the search function on the US NIH Clinical Trials website.

Visit the Australian New Zealand Clinical Trial Registry to find out more about clinical trials that are happening here in Australia.

Please remember that these trials are experimental and that each treatment is being evaluated as part of a clinical trial, this does not make it a proven safe and effective therapy. The links on this site have been provided for your research. We recommend speaking with your treating doctors for independent advice on what is best for you. You can learn more about what clinical trials are at



A trial of Bardoxolone Methyl in Patients With ADPKD

Sponsor: Reata Pharmaceuticals, Inc. 

Status: Recruiting

Trial registration number: NCT03918447

FALCON is a Phase 3 clinical study evaluating the effectiveness and safety of bardoxolone methyl (an oral investigational drug) in patients with Autosomal Dominant PKD.


  • Are 18 to 70 years of age
  • Have been diagnosed with ADPKD

Other eligibility criteria will apply.

See the study brochure for more information or email: Joanna Figueroa at Joanna, a nurse/patient navigator, can provide more information regarding study participation, what to expect, and connect eligible patients with local clinical research centers.


Randomised controlled trial to determine the efficacy and safety of prescribed water intake to prevent kidney failure due to Autosomal Dominant Polycystic Kidney Disease (PREVENT-ADPKD)

Principal Investigators: A/Prof Gopala Rangan

Status: Active, not recruiting

Trial registration number: ANZCTR12614001216606

PREVENT-ADPKD is a multi-centre, open-label randomised controlled trial that will test the hypothesis that prescribed water intake to maintain hydration will the reduce the progression of total kidney volume in participants with autosomal dominant polycystic kidney disease with eGFR> 30 ml/min/1.73 m2 and aged between 18-65 years old.

The trial commenced in December 2015, and recruitment likely to be completed at the end of 2017. Completion of the 3-year trial followup is anticipated to be completed at the the end of 2020. As of July 2017, 166 participants have completed the Screening Visit.

The trial is in progress at multiple sites in Sydney (Westmead Hospital, Nepean Hospital, Norwest Private Hospital, Liverpool Hospital, Prince of Wales Hospital and St George Hospital, and two private clinics), regional NSW (Wollongong Hospital, John Hunter Hospital, two Private Clinics in Gosford) and Perth (Sir Charles Gardinar Hospital).


Implementation of Metformin theraPy to Ease DEcline of kidney function in PKD (IMPEDE-PKD)

Principal Investigators: A/Prof Andrew Mallett

Status: Recruitment to commence early 2021

Trial number: AKTN 16.01

IMPEDE-PKD is a prospective, multicentre double-blind, randomised controlled trial of metformin versus placebo in adult ADPKD patients to determine the effect of metformin therapy on renal function decline and cyst growth. The aim and objective of this study is to undertake a randomised clinical trial of metformin amongst patients with ADPKD to investigate its potential to slow kidney function decline. Pre-clinical studies suggest that there are ADPKD disease pathways that can be advantageously modified by administration of metformin. The common use of this medication, including in non-diabetic conditions such as polycystic ovarian syndrome, its relative inexpensive nature as well as its defined side effect and dosing profiles in kidney disease lend it to the conduct of a clinical trial to address this aim.

This clinical trial will seek to undertake such a clinical trial within the context of the pre-eminent national kidney trials organisation. If successful, this would dramatically change the prognosis and clinical care for many Australians affected by ADPKD as well as positively impact upon health service utilisation such as dialysis requirement at a community level.