STOP-PKD: SGLT2-inhibition to improve Prognosis in Polycystic Kidney Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

University Of Cologne

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

16 Dec 2025 → ongoing

Decision date (initial)

2025-11-10

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

German Research Foundation (DFG - Deutsche Forschungsgemeinschaft)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

The primary objective is to assess whether dapagliflozin improves the decline of kidney function in patients with ADPKD on-treatment (between week 6 and EOT).

Secondary objectives 3

1. To assess the impact of dapagliflozin treatment in patients with ADPKD on the change in kidney function off-treatment.
2. To assess the impact of dapagliflozin treatment in patients with ADPKD on a composite outcome: time to 40% decrease in eGFR, ESKD or renal death.
3. To assess the impact of dapagliflozin treatment in patients with ADPKD on patient safety.

Conditions and MedDRA coding

Autosomal dominant polycystic kidney disease (ADPKD)

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Male and female patients with ADPKD (modified Ravine criteria) ≥ 18 and ≤ 60 years
2. Patients 18 - 39 years: eGFR ≥25 ml/min/1.73 m2; patients 40 - 60 years: eGFR ≥25 and <90 ml/min/1.73 m2
3. 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)
4. IF patient is on ACE-I /ARBs: stable dose for 4 weeks before screening

Exclusion criteria 13

1. Treatment with tolvaptan, somatostatin analogue, lithium or SGLT2i within the last 3 months before screening
2. Medical history of diabetic ketoacidosis, necrotizing fasciitis or organ transplantation
3. Diabetes mellitus type 1 or any type of diabetes mellitus due to insulin deficiency
4. Uncontrolled ongoing urinary tract or genital infections
5. Known intolerance of the study medication ingredients
6. Uncontrolled grade 2 hypertension (>160/100 mmHg)
7. Symptomatic hypotension, or systolic blood pressure <90 mmHg
8. Primary renal disease other than ADPKD
9. 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)
10. Pregnancy, breastfeeding or women of child-bearing potential not using effective contraception method
11. Not able to comply with the study protocol, in the investigator’s judgement
12. Not able to provide informed consent
13. Participation in any other interventional clinical trial in the last 2 months

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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)

Secondary endpoints 4

1. 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)
2. 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) • OR ESKD: start of dialysis or kidney transplantation or sustained eGFR <15 mL/min/1.73m2 • OR Renal death
3. Collection of SAEs and AEs of special interest
4. Change in total kidney volume after 1 year (first 150 patients; baseline – 48 weeks)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

University Of Cologne

Sponsor organisation

University Of Cologne

Address

Albertus-Magnus-Platz 1

City

Cologne

Postcode

50923

Country

Germany

Scientific contact point

Organisation

University Of Cologne

Contact name

Roman-Ulrich Müller

Public contact point

Organisation

University Of Cologne

Contact name

Roman-Ulrich Müller

Locations

4 EU/EEA countries · 30 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 35 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications