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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



Update 16 May 2023 – PKD Australia has been informed that this trial has been discontinued.

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.


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

Principal Investigators: Prof Andrew Mallett

Status: Currently Recruiting

Trial number: AKTN 16.01

Trial registration number: NCT04939935 (

IMPEDE-PKD is a prospective, multicentre double-blind, randomised controlled trial of metformin versus placebo in adults with ADPKD to determine the effect of metformin therapy on kidney function.

Metformin is a commonly used medication for treatment of diabetes and is also used for treatment of polycystic ovarian syndrome. Preclinical studies have suggested that metformin could modify ADPKD disease pathways, and recent clinical studies have shown that it is safe to use in people with ADPKD.

The IMPEDE-PKD trial is the first clinical trial to test the effect of metformin in people with ADPKD. If successful, this trial could dramatically change the future clinical care of people with ADPKD as well as positively impact on health service utilisation such as dialysis requirement at a community level.

Contact details:



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).


A randomised controlled trial to test the effect of beetroot juice in reducing blood pressure in hypertensive adults with ADPKD

Principal Investigators: Prof Gopi Rangan

Status: Active, Recruiting

Trial registration number: NCT05401409

BEET-PKD is a 5-week double-blind, placebo-controlled trial that will randomise hypertensive ADPKD patients (eGFR>30ml/min) to either 70 mls of beetroot juice or placebo (manufactured by a commercial provider). There are no reported adverse effects. The study is funded by PKD Australia.

The rationale for the study is that there is increasing evidence that endothelial-derived nitric oxide is reduced and may mediate hypertension and endothelial dysfunction in ADPKD. Beetroot juice is a rich natural source of dietary nitrate and there are several clinical trials that have established that it has an anti-hypertensive effect in CKD and other patient populations.  

The trial commenced in May 2022 and is currently active and recruiting. It is expected to be completed in December 2022. The study site is Westmead Hospital, Sydney. Eligible participants include:

  • Diagnosed with ADPKD
  • Age > 18 years old
  • eGFR > 30 ml/min/1.73m2
  • On at least 1 medication to treat high blood pressure

Please contact the trial team at or (02) 8627 3529 if you are interested.

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