NCT04939935

Brief Summary

This study will investigate if a medication (metformin) widely used in the treatment of diabetes could be re-purposed for the treatment of patients with a diagnosis of early stage ADPKD to slow the rate of kidney function decline, reducing morbidity and mortality and improving the quality of life for ADPKD patients.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,174

participants targeted

Target at P75+ for phase_3

Timeline
55mo left

Started Nov 2022

Longer than P75 for phase_3

Geographic Reach
3 countries

49 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress43%
Nov 2022Dec 2030

First Submitted

Initial submission to the registry

June 16, 2021

Completed
9 days until next milestone

First Posted

Study publicly available on registry

June 25, 2021

Completed
1.4 years until next milestone

Study Start

First participant enrolled

November 29, 2022

Completed
8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2030

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2030

Last Updated

March 31, 2026

Status Verified

March 1, 2026

Enrollment Period

8 years

First QC Date

June 16, 2021

Last Update Submit

March 26, 2026

Conditions

Keywords

PlaceboMetforminADPKD

Outcome Measures

Primary Outcomes (1)

  • The change in estimated glomerular filtration rate (eGFR)

    This will be measured using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula at 104 weeks (24 months) from first dispensing date.

    Over 24 months

Secondary Outcomes (13)

  • Annualised slope of eGFR.

    Over 24 months

  • Composite outcome

    Over 24 months

  • Severity of change in eGFR

    Over 24 months

  • Kidney failure

    Over 24 months

  • Mortality

    Over 24 months

  • +8 more secondary outcomes

Study Arms (2)

Intervention

EXPERIMENTAL

Participants randomised to the intervention group receive Metformin XR plus standard of care for 104 weeks. Dosage will depend on individual participant's level of tolerance to Metformin XR as well as their estimated glomerular filtration rate (eGFR). The dosage will be between 500-2000mg/day.

Drug: Metformin XR

Control

PLACEBO COMPARATOR

Participants randomised to the control group receive placebo plus standard of care for 104 weeks.

Other: Control

Interventions

ControlOTHER

Placebo is inactive tablets that is identical to the intervention Metformin tablets.

Also known as: Placebo
Control

Extended release metformin.

Also known as: APO-Metformin XR (500mg)
Intervention

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Willing to participate and provide informed consent
  • Aged 18-70 years
  • 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
  • eGFR equal to or greater than 38 mL/min/1.73m2 and \<90 mL/min/1.73m2
  • And have either:
  • (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.

You may not qualify if:

  • Diabetes mellitus (as per American Diabetes Association definition), or other systemic conditions that may cause CKD independent of PKD (excluding hypertension)
  • Uncontrolled hypertension (Systolic BP \>160 mmHg and/or diastolic BP \>100 mmHg after a period of rest)
  • Clinically significant heart failure, including but not limited to New York Heart Association Class (NYHA) III or IV
  • Non-polycystic liver disease, including but not limited to:
  • Liver enzymes (ALT, AST or Total Bilirubin) \>2 times the upper limit of normal, except when a diagnosis of Gilbert Syndrome exists and/or,
  • Child-Pugh classification score equal to or greater than 5
  • Any contraindication to metformin including abnormal liver function tests or untreated Vitamin B12 deficiency
  • Currently taking metformin
  • Pregnancy or breastfeeding, or planning to get pregnant in the next three years.
  • 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.
  • History of dialysis.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (49)

Renal Research

Gosford, New South Wales, 2250, Australia

ACTIVE NOT RECRUITING

Royal Prince Alfred Hospital

Sydney, New South Wales, 2050, Australia

ACTIVE NOT RECRUITING

Royal North Shore Hospital

Sydney, New South Wales, 2065, Australia

ACTIVE NOT RECRUITING

Westmead Hospital - Western Sydney Local Health District

Sydney, New South Wales, 2145, Australia

ACTIVE NOT RECRUITING

Bundaberg Hospital

Bundaberg, Queensland, 4670, Australia

ACTIVE NOT RECRUITING

Townsville University Hospital

Douglas, Queensland, 4814, Australia

ACTIVE NOT RECRUITING

Royal Brisbane and Women's Hospital

Herston, Queensland, 4006, Australia

ACTIVE NOT RECRUITING

Princess Alexandra Hospital

Woolloongabba, Queensland, 4102, Australia

ACTIVE NOT RECRUITING

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

ACTIVE NOT RECRUITING

Royal Melbourne Hospital

Melbourne, Victoria, 3052, Australia

ACTIVE NOT RECRUITING

Austin Health

Melbourne, Victoria, 3084, Australia

ACTIVE NOT RECRUITING

Monash Medical Centre

Melbourne, Victoria, 3168, Australia

ACTIVE NOT RECRUITING

Sir Charles Gairdner Hospital

Perth, Western Australia, 6009, Australia

ACTIVE NOT RECRUITING

Te Whatu Ora - Hauora a Toi Bay of Plenty

Tauranga, Bay of Plenty, 3112, New Zealand

RECRUITING

Te Whatu Ora - Te Tai Tokerau

Whangārei, Northland, 0110, New Zealand

RECRUITING

Te Whatu Ora - Southern

Dunedin, Otago, 9016, New Zealand

RECRUITING

Te Whatu Ora - Taranaki

New Plymouth, Taranaki Region, 4310, New Zealand

RECRUITING

Ta Pae Hauora o Ruhahine o Terarua Mid Central

Palmerston North, New Zealand

RECRUITING

Royal Devon & Exeter Hospital

Exeter, Devon, EX2 5DW, United Kingdom

RECRUITING

Nottingham Renal Unit, Nottingham City Hospital

Nottingham, East Midlands, NG5 1PB, United Kingdom

NOT YET RECRUITING

Raigmore Hospital

Inverness, Inverness Shire, IV2 3UJ, United Kingdom

NOT YET RECRUITING

Royal Preston Hospital

Preston, Lancashire, PR2 9HT, United Kingdom

NOT YET RECRUITING

Salford Royal Hospital

Salford, Lancashire, M6 8HD, United Kingdom

RECRUITING

Leicester General Hospital

Leicester, Leicestershire, LE5 4PW, United Kingdom

RECRUITING

St Helier Hospital

Carshalton, London, SM5 1AA, United Kingdom

NOT YET RECRUITING

Aintree University Hospital

Liverpool, Merseyside, L9 7AL, United Kingdom

RECRUITING

Norfolk and Norwich University Hospital

Norwich, Norfolk, NR4 7UY, United Kingdom

NOT YET RECRUITING

Antrim Area Hospital

Antrim, Northern Ireland, BT41 2RL, United Kingdom

RECRUITING

Ulster Hospital

Belfast, Northern Ireland, BT16 1RH, United Kingdom

RECRUITING

Altnagelvin Hospital

Londonderry, Northern Ireland, BT47 6SB, United Kingdom

RECRUITING

Daisy Hill Hospital

Newry, Northern Ireland, BT35 8DR, United Kingdom

NOT YET RECRUITING

Oxford Kidney Unit, Churchill Hospital,

Oxford, Oxfordshire, OX3 7LE, United Kingdom

RECRUITING

Doncaster Royal Infirmary

Doncaster, South Yorkshire, DN2 5LT, United Kingdom

RECRUITING

Sheffield Kidney Institute

Sheffield, South Yorkshire, S5 7AU, United Kingdom

RECRUITING

Royal Stoke University Hospital

Stoke-on-Trent, Staffordshire, ST4 6QG, United Kingdom

NOT YET RECRUITING

Freeman Hospital

Newcastle upon Tyne, Tyne and Wear, NE7 7DN, United Kingdom

NOT YET RECRUITING

Queen Elizabeth Hospital Birmingham

Birmingham, West Midlands, B15 2GW, United Kingdom

NOT YET RECRUITING

Bradford Renal Unit, St Luke's Hospital

Bradford, West Yorkshire, BD5 0NA, United Kingdom

RECRUITING

Cardiff and Vale University Health Board

Cardiff, United Kingdom

RECRUITING

Epsom and St Helier University Hospitals

Carshalton, United Kingdom

RECRUITING

The Royal London Hospital

London, E1 1FR, United Kingdom

NOT YET RECRUITING

Royal Free Hospital

London, NW3 2QG, United Kingdom

NOT YET RECRUITING

King's College Hospital

London, SE5 9RS, United Kingdom

NOT YET RECRUITING

St George's University Hospital

London, United Kingdom

RECRUITING

Nottingham University Hospital

Nottingham, United Kingdom

RECRUITING

University Hospitals Plymouth NHS Trust

Plymouth, United Kingdom

RECRUITING

East & North Hertfordshire Teaching NHS Trust

Stevenage, United Kingdom

RECRUITING

South Tyneside and Sunderland NHS Foundation Trust

Sunderland, United Kingdom

RECRUITING

York and Scarborough Teacehing Hospitals

York, United Kingdom

RECRUITING

Related Publications (4)

  • Cortinovis M, Perico N, Remuzzi G. The Need for Novel Therapeutic Directions in Autosomal Dominant Polycystic Kidney Disease Patient Care. Clin J Am Soc Nephrol. 2025 Dec 5. doi: 10.2215/CJN.0000000975. Online ahead of print.

  • Pierre KS, El-Damanawi R, Johnson DW, Hawley CM, Viecelli AK, Jha V, Green SC, Gesualdo L, Kiriwandeniya C, Velayudham P, Vergara LA, Mihala G, Matsuyama M, Brent PP, Mallett AJ; IMPEDE-PKD Global Steering Committee (see Appendix). Implementation of Metformin Therapy to Ease Decline of Kidney Function in Polycystic Kidney Disease (IMPEDE-PKD): study protocol for a phase III, multi-centre, randomized, placebo-controlled trial evaluating the long-term efficacy of metformin in slowing the rate of kidney function decline in patients with autosomal dominant polycystic kidney disease. Trials. 2025 Aug 25;26(1):302. doi: 10.1186/s13063-025-09010-6.

  • St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.

  • El-Damanawi R, Stanley IK, Staatz C, Pascoe EM, Craig JC, Johnson DW, Mallett AJ, Hawley CM, Milanzi E, Hiemstra TF, Viecelli AK. Metformin for preventing the progression of chronic kidney disease. Cochrane Database Syst Rev. 2024 Jun 4;6(6):CD013414. doi: 10.1002/14651858.CD013414.pub2.

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Condition Hierarchy (Ancestors)

Polycystic Kidney DiseasesKidney Diseases, CysticKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCiliopathiesGenetic Diseases, Inborn

Study Officials

  • Andrew Mallett, MBBS, PhD

    Townsville University Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Misa Matsuyama, PhD

CONTACT

Pushparaj Velayudham

CONTACT

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
Study participants, treating physicians and other care providers, outcome assessors, study investigators, and study statisticians will be blinded. An unblinded statistician will regularly review treatment allocations to ensure balance across treatment arms. The unblinded statistician will also prepare unblinded statistical reports for meetings of the Data and Safety Monitoring Board.
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Enrolled participants will be randomised to either (1) intervention group receiving metformin extended release (XR) plus standard of care, or (2) placebo plus standard of care.
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 16, 2021

First Posted

June 25, 2021

Study Start

November 29, 2022

Primary Completion (Estimated)

December 1, 2030

Study Completion (Estimated)

December 1, 2030

Last Updated

March 31, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

Locations