Clinical trial • Phase III • Nephrology|Rare Disease

Dapagliflozin for Autosomal dominant polycystic kidney disease

Phase III trial of Dapagliflozin for Autosomal dominant polycystic kidney disease.

Overview

Trial Therapeutic Area
Nephrology|Rare Disease
Trial Disease
Autosomal dominant polycystic kidney disease
Trial Stage
Phase III
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
18-07-2025
First CTIS Authorization Date
06-11-2025

Trial design

Randomised, dapagliflozin 10 mg orally once daily for 156 weeks; comparator arm: matching placebo for 156 weeks-controlled Phase III trial across 27 sites in Austria, Germany, Spain and others.

Randomised
Yes
Comparator
Dapagliflozin 10 mg orally once daily for 156 weeks; Comparator arm: matching placebo for 156 weeks
Target Sample Size
420
Trial Duration For Participant
1176

Stratification factors

  • eGFR (under and above 45 mL/min/1.73m2)
  • Age (under and above 45 years)

Eligibility

Recruits 420 Vulnerable population not selected; participants must be able to provide informed consent (exclusion criterion: 'Not able to provide informed consent'). No assent or parental consent procedures described..

Pregnancy Exclusion
Pregnancy, breastfeeding or women of child-bearing potential not using effective contraception method
Vulnerable Population
Vulnerable population not selected; participants must be able to provide informed consent (exclusion criterion: 'Not able to provide informed consent'). No assent or parental consent procedures described.

Inclusion criteria

  • {"criterion_text":"- Male and female patients with ADPKD (modified Ravine criteria) ≥ 18 and ≤ 60 years"}
  • {"criterion_text":"- Patients 18 - 39 years: eGFR ≥25 ml/min; patients 40 - 60 years: eGFR ≥25 and <90 ml/min/1.73 m2"}
  • {"criterion_text":"- Indicators of rapid progression, either of the following: - Mayo class 1D-E - Mayo class 1C AND EITHER 1. Truncating PKD1 mutation OR 2. eGFR loss > 3ml/min/year (determined by ≥ 4 creatinine values within 4 years, ≥ 6 months measurement intervals) OR 3. PROPKD score > 6 (patient history)"}
  • {"criterion_text":"- IF patient is on ACE-I /ARBs: stable dose for 4 weeks before screening"}

Exclusion criteria

  • {"criterion_text":"- Treatment with tolvaptan, somatostatin analogue or SGLT2i within the last 3 months before screening"}
  • {"criterion_text":"- Pregnancy, breastfeeding or women of child-bearing potential not using effective contraception method"}
  • {"criterion_text":"- Not able to comply with the study protocol, in the investigator’s judgement"}
  • {"criterion_text":"- Not able to provide informed consent"}
  • {"criterion_text":"- Participation in any other interventional clinical trial in the last 2 months"}
  • {"criterion_text":"- Medical history of diabetic ketoacidosis, necrotizing fasciitis or organ transplantation"}
  • {"criterion_text":"- Diabetes mellitus type 1 or any type of diabetes mellitus due to insulin deficiency"}
  • {"criterion_text":"- Uncontrolled ongoing urinary tract or genital infections"}
  • {"criterion_text":"- Known intolerance of the study medication ingredients"}
  • {"criterion_text":"- Uncontrolled grade 2 hypertension (>160/100 mmHg)"}
  • {"criterion_text":"- Symptomatic hypotension, or systolic blood pressure <90 mmHg"}
  • {"criterion_text":"- Primary renal disease other than ADPKD"}
  • {"criterion_text":"- Hepatic impairment (aspartate transaminase [AST] or alanine transaminase [ALT]>3x the upper limit of normal [ULN]; or total bilirubin >2x ULN at time of enrolment)"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Annual slope of eGFR decline (in mL/min/1,73m2 per year, chronic slope), as calculated with linear mixed models, using all available creatinine values from week 6 until end of treatment (week 156)","definition_or_measurement_approach":"Calculated with linear mixed models using all available creatinine values from week 6 until end of treatment (week 156); reported as annual slope of eGFR decline in mL/min/1.73m2 per year"}

Secondary endpoints

  • {"endpoint_text":"- Off-treatment values: eGFR change from before to after treatment (using the mean of two creatine measurements for each timepoint; week -4 and 0 for before treatment; week 162 and 168 for follow-up)","definition_or_measurement_approach":"eGFR change using mean of two creatinine measurements at specified timepoints (week -4 and 0 before treatment; week 162 and 168 follow-up)"}
  • {"endpoint_text":"- Composite outcome: Sustained 40 % decrease in eGFR from randomization (confirmed by second measurement in next scheduled visit or measured at the last study follow-up visit / the last scheduled visit before death) •\tOR ESKD: start of dialysis or kidney transplantation or sustained eGFR <15 mL/min/1.73m2 •\tOR Renal death","definition_or_measurement_approach":"Composite includes sustained 40% eGFR decrease confirmed by second measurement, or ESKD (start of dialysis or kidney transplant or sustained eGFR <15 mL/min/1.73m2), or renal death"}
  • {"endpoint_text":"- Collection of SAEs and AEs of special interest","definition_or_measurement_approach":"Safety monitoring via collection and reporting of SAEs and adverse events of special interest (no further measurement approach specified)"}
  • {"endpoint_text":"- Change in total kidney volume after 1 year (first 150 patients; baseline – 48 weeks)","definition_or_measurement_approach":"Change from baseline to 48 weeks in total kidney volume for first 150 patients"}

Recruitment

Planned Sample Size
420
Recruitment Window Months
71
Consent Approach
Informed consent required from each participant; exclusion if 'Not able to provide informed consent'. Subject information sheets and informed consent forms are provided site-specifically for participating countries (documents listed for Austria, Germany, Spain and Netherlands). Participants are adults (≥18 years); no assent or parental consent procedures are described.

Geography

Total Number Of Sites
27
Total Number Of Participants
420

Austria

Earliest CTIS Part Ii Submission Date
13-10-2025
Latest Decision Or Authorization Date
10-11-2025
Processing Time Days
28
Number Of Sites
2
Number Of Participants
19

Sites

Site Name
Vorarlberger Krankenhaus-Betriebsgesellschaft mbH
Department Name
Department of Internal Medicine 3 – Nephrology, Dialysis and Hypertensiology
Contact Person Name
Emanuel Zitt
Contact Person Email
nephro@lkhf.at
Site Name
Medizinische Universitaet Innsbruck
Department Name
Dept. of Internal Medicine IV - Nephrology and Hypertension
Contact Person Name
Michael Rudnicki
Contact Person Email
michael.rudnicki@i-med.ac.at

Germany

Earliest CTIS Part Ii Submission Date
23-10-2025
Latest Decision Or Authorization Date
07-11-2025
Processing Time Days
15
Number Of Sites
20
Number Of Participants
336

Sites

Site Name
Universitaet Leipzig
Department Name
Nephrology
Contact Person Name
Friederike Petzold
Site Name
Universitaetsmedizin Goettingen
Department Name
Nephrology and Rheumatology
Contact Person Name
Oliver Gross
Site Name
Klinikum Dortmund gGmbH
Department Name
Nephrology
Contact Person Name
Fedai Özcan
Contact Person Email
studienzentrum@klinikumdo.de
Site Name
Universitaetsklinikum Jena KöR
Department Name
Nephrology
Contact Person Name
Martin Busch
Site Name
Klinikum Der Landeshauptstadt Stuttgart gKAöR
Department Name
Clinic for Kidney, Hypertension and Autoimmune Diseases
Contact Person Name
Vedat Schwenger
Site Name
Zentrum Fuer Nieren Hochdruck Und Stoffwechselerkrankungen
Department Name
Nephrology
Contact Person Name
Georg Schlieper
Site Name
Universitaetsklinikum Schleswig-Holstein AöR (Luebeck)
Department Name
Nephrology
Contact Person Name
Figen Cakiroglu
Contact Person Email
Franziska.Klingbiel@uksh.de
Site Name
Medizinische Hochschule Hannover
Department Name
Nephrology
Contact Person Name
Jessica Kaufeld
Contact Person Email
studienzentrum@mh-hannover.de
Site Name
Universitaetsklinikum Aachen AöR
Department Name
Division of Nephrology and Clinical Immunology
Contact Person Name
Susanne Fleig
Contact Person Email
SekretariatMK2@ukaachen.de
Site Name
Universitaetsklinikum Heidelberg AöR
Department Name
Nephrology
Contact Person Name
Martin Zeier
Site Name
Goethe University Frankfurt
Department Name
Nephrology
Contact Person Name
Jeannine Lang
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
III. Department of Medicine
Contact Person Name
Fabian Braun
Contact Person Email
nierenambulanz@uke.de
Site Name
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)
Department Name
Nephrology
Contact Person Name
Lutz Renders
Contact Person Email
nephrologie.office@mri.tum.de
Site Name
Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
Department Name
Nephrology
Contact Person Name
Alexander Paliege
Contact Person Email
alexander.paliege@ukdd.de
Site Name
Universitaetsklinikum Schleswig-Holstein AöR (Kiel)
Department Name
Nephrology and Hypertension
Contact Person Name
Roland Schmitt
Site Name
LMU Klinikum Muenchen AöR
Department Name
Nephrology
Contact Person Name
Michael Fischereder
Site Name
University Hospital Cologne AöR
Department Name
Deparment of Nephrology, Rheumatology, Endocrinology and General Medicine
Contact Person Name
Roman Müller
Contact Person Email
STOP-PKD@uk-koeln.de
Site Name
Universitaetsklinikum Duesseldorf AöR
Department Name
Nephrology
Contact Person Name
Lorenz Sellin
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Nephrology and Medical Intensive Care
Contact Person Name
Jan Halbritter
Contact Person Email
nephrologie-studien@charite.de
Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
Nephrology
Contact Person Name
Julia Weinmann-Menke

Spain

Earliest CTIS Part Ii Submission Date
14-10-2025
Latest Decision Or Authorization Date
07-11-2025
Processing Time Days
24
Number Of Sites
2
Number Of Participants
26

Sites

Site Name
Fundacio Hospital Universitari Vall D’Hebron Institut De Recerca
Department Name
Nephrology
Contact Person Name
Laia Sans Axter
Contact Person Email
assajos.nefrologia@vhir.org
Site Name
Fundacio Puigvert
Department Name
Nephrology
Contact Person Name
Roser Torra Balcells
Contact Person Email
ofgrin@fundacio-puigvert.es

Netherlands

Earliest CTIS Part Ii Submission Date
10-10-2025
Latest Decision Or Authorization Date
06-11-2025
Processing Time Days
27
Number Of Sites
3
Number Of Participants
39

Sites

Site Name
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department Name
Nephrology
Contact Person Name
Mahdi Salih
Contact Person Email
tb.ig@erasmusmc.nl
Site Name
Leids Universitair Medisch Centrum (LUMC)
Department Name
Nephrology
Contact Person Name
Siebe Spijker
Site Name
Universitair Medisch Centrum Groningen
Department Name
Nephrology
Contact Person Name
Ron Gansevoort
Contact Person Email
researchnefrologie@int.umcg.nl

Sponsor

Primary sponsor

Full Name
University Of Cologne
Organisation Type
Educational Institution
Country Of Registered Address
Germany

Third parties

  • {"country":"","full_name":"German Research Foundation (DFG - Deutsche Forschungsgemeinschaft)","duties_or_roles":"Monetary support/funder","organisation_type":""}

Investigational products

Investigational Product Name
Dapagliflozin Ascend 10 mg Filmtabletten
Active Substance
Dapagliflozin
Modality
Small molecule
Routes Of Administration
Oral
Route
Oral
Authorisation Status
Marketing authorisation in DE (marketingAuthNumber: 7001787.00.00)
Starting Dose
10 mg
Dose Levels
10 mg
Frequency
Once daily
Maximum Dose
10 mg
Investigational Product Name
Matching Placebo for "Dapagliflozin Ascend 10 mg Filmtabletten"
Modality
Other

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