Home › Trials › NCT04939935

Using Metformin to Slow Kidney Decline in Polycystic Kidney Disease

Implementation of Metformin theraPy to Ease Decline of Kidney Function in Polycystic Kidney Disease (IMPEDE-PKD): A Randomised Placebo-Controlled Trial

Phase 3 Interventional The University of Queensland · NCT04939935

This study is testing if metformin can help slow down kidney problems in adults with early-stage Polycystic Kidney Disease.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment1174 (estimated)
Ages18 Years to 70 Years
SexAll
SponsorThe University of Queensland Academic / other
Locations49 sites (Gosford, New South Wales and 48 other locations)
Trial IDNCT04939935 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the use of metformin, a medication commonly used for diabetes, to slow the decline of kidney function in patients with early-stage Autosomal Dominant Polycystic Kidney Disease (ADPKD). The study aims to recruit 1,174 adult participants globally, including 250 from Australia, and will assess the effectiveness of metformin in reducing kidney function decline and improving quality of life. Participants will be randomly assigned to receive either metformin or a placebo, and the outcomes will provide insights into potential new therapies for ADPKD. The trial is designed to address the significant unmet need for effective and affordable treatments for this condition.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18-70 with a diagnosis of early-stage ADPKD and specific risk factors for disease progression.

Not a fit: Patients with advanced kidney disease or those outside the specified age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new, cost-effective treatment option that slows kidney function decline and improves the quality of life for patients with ADPKD.

How similar studies have performed: Previous studies have shown promise in using metformin for targeting cyst growth in ADPKD, making this approach a potentially viable option.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

To be eligible to participate in this trial, patients must satisfy all of the following inclusion criteria:

1. Willing to participate and provide informed consent
2. Aged 18-70 years
3. Diagnosis of ADPKD based on radiological +/- genetic criteria as per Kidney Health Australia - Caring for Australians and New Zealanders with Kidney Impairment (KHA-CARI) Guidelines
4. eGFR equal to or greater than 38 mL/min/1.73m2 and \<90 mL/min/1.73m2

And have either:

5(a) One or more risk factors of progression from the following:

* Bilateral kidney length equal to or greater than16.5 cm, or
* Total Kidney Volume (TKV) equal to or greater than 750 mL or height-adjusted TKV (htTKV) equal to or greater than 600 mL/m2, or
* Mayo class IC/D/E or Pro-PKD score equal to or greater than 6 OR 5(b) Evidence of Active progression
* Decline in eGFR equal to or greater than 5 mL/min/1.73m2 in one year, or
* Decline in eGFR equal to or greater than 3 mL/min/1.73m2 per year over five years or more. or
* Increase in htTKV/TKV of equal to or greater than 5% per year on at least 2 measurements in the past year, excluding any initial eGFR effect over the initial 3 months of tolvaptan commencement (if applicable) Note: Tolvaptan therapy must have been in place for at least 6 months with stable dose for at least 3 months.

Exclusion Criteria:

1. Diabetes mellitus (as per American Diabetes Association definition), or other systemic conditions that may cause CKD independent of PKD (excluding hypertension)
2. Uncontrolled hypertension (Systolic BP \>160 mmHg and/or diastolic BP \>100 mmHg after a period of rest)
3. Clinically significant heart failure, including but not limited to New York Heart Association Class (NYHA) III or IV
4. Non-polycystic liver disease, including but not limited to:

   1. Liver enzymes (ALT, AST or Total Bilirubin) \>2 times the upper limit of normal, except when a diagnosis of Gilbert Syndrome exists and/or,
   2. Child-Pugh classification score equal to or greater than 5
5. Any contraindication to metformin including abnormal liver function tests or untreated Vitamin B12 deficiency
6. Currently taking metformin
7. Pregnancy or breastfeeding, or planning to get pregnant in the next three years.
8. Comorbidities with potential to contaminate trial outcomes, specifically active cancer, history of other solid organ transplantations, active chronic obstructive pulmonary disease (COPD), active inflammatory bowel disease, and the presence of stoma.
9. History of dialysis.

Where this trial is running

Gosford, New South Wales and 48 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Autosomal Dominant Polycystic Kidney DiseasePlaceboMetforminADPKD
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.