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ADPKD (Autosomal Dominant Polycystic Kidney Disease)AD(H)PKDNCT02497521

The German ADPKD Tolvaptan Treatment Registry

An observational study of ADPKD (Autosomal Dominant Polycystic Kidney Disease), run by University of Cologne at 12 sites in Germany.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

The German ADPKD Tolvaptan Treatment Registry is a prospective, observational, multicentric study of patients suffering from ADPKD that are considered for tolvaptan treatment. All ADPKD patients that are evaluated for treatment indication, or that are planned to be treated with tolvaptan, or that are already treated… Read more about this study

Who is it for?
Ages 18 years and older
How many people
2,000 participants
Where
12 sitesGermany
Study dates
Nov 2015to Dec 2027
How long will I take part?
10 Years
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Study population: Patients with diagnosis of ADPKD and therapy with tolvaptan

Who may be able to join

  • Age > 18 years
  • ADPKD proven by positive family history and evidence of renal cysts or diagnosed by treating physician
  • Presentation at our center for tolvaptan treatment indication, or tolvaptan treatment planned, or tolvaptan already started

Who cannot join

  • Patients not capable of giving informed consent
  • End stage renal disease requiring renal replacement therapy
  • Patients receiving tolvaptan as "off-label use"

Trial locations and contacts

12 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Roman-Ulrich Mueller, MD contact

+49(0)22147897222

roman-ulrich.mueller@uk-koeln.de

Cornelia Boehme contact

+49(0)22147897222

studienzentrum-medII@uk-koeln.de

12 sites in 1 country

Germany12 sites, 10 recruiting

  • Charité Universitätsmedizin Berlin

    Berlin, State of Berlin, 10117

    Active, not recruiting

  • University Hospital of Cologne

    Cologne, North Rhine-Westphalia, 50937

    Thomas Benzing, MD, +49 221 478 97222, thomas.benzing@uk-koeln.de

    Roman-Ulrich Mueller, MD, +49 221 478 97222, roman-ulrich.mueller@uk-koeln.de

    Recruiting

  • Medizinische Hochschule Hannover

    Hanover, Lower Saxony, 30625

    Roland Schmitt, MD

    Hermann Haller, MD

    Recruiting

  • University Hospital of Jena

    Jena, Thuringia, 07747

    Martin Busch, MD

    Martin Busch, MD

    Recruiting

  • University Hospital of Leipzig, Nephrologische Ambulanz

    Leipzig, Saxony, 04103

    Jan Halbritter, MD

    Jan Halbritter, MD

    Recruiting

  • Praxisgemeinschaft Dr. Peschel

    Leipzig, Saxony, 04107

    Katrin Peschel, MD

    Katrin Peschel, MD

    Recruiting

  • University Hospital of Schleswig-Holstein

    Lübeck, Schleswig-Holstein, 23538

    Figen Cakiroglu, MD

    Alexandra Tiedtke

    Recruiting

  • Nierenzentrum Lübeck

    Lübeck, Schleswig-Holstein, 23562

    Peter Gerke, MD

    Lutz Hennings

    Recruiting

  • Fachinternistische Gemeinschaftspraxis Markgraeferland

    Müllheim, Baden-Wurttemberg, 79379

    Markus Cybulla, MD

    Gudrun Tech

    Recruiting

  • Nieren- und Diabeteszentrum Nettetal-Lobberich

    Nettetal, North Rhine-Westphalia, 41334

    Active, not recruiting

  • Robert-Bosch-Krankenhaus

    Stuttgart, 70376

    Dominik Alscher, MD

    David Callau Monje, MD

    Recruiting

  • University Hospital of Wuerzburg, ZIM

    Würzburg, Bavaria, 97080

    Daniel Kraus, MD

    Kirsten Hofmann

    Recruiting

Other trials in Germany

About this study

Official title: The German ADPKD Tolvaptan Treatment Registry is a Prospective, Observational, Multicentric Study of Patients Suffering From ADPKD That Are Considered for Tolvaptan Treatment.

The German ADPKD Tolvaptan Treatment Registry is a prospective, observational, multicentric study of patients suffering from ADPKD that are considered for tolvaptan treatment. All ADPKD patients that are evaluated for treatment indication, or that are planned to be treated with tolvaptan, or that are already treated with tolvaptan are eligible. This registry is designed to provide "real-world" data on treatment management of patients with ADPKD.

Read the detailed description

A substantial number of ADPKD patients treated in our center or referred to our center for counseling are considered eligible for tolvaptan treatment and, thus, will be invited to enter the registry. Furthermore, many patients with ADPKD are treated by nephrologists in practices. We operate a network with many of these practices and will expand this network. Patients can be enrolled - after having obtained approval by the local ethics committee - at external sites (expected number: about 500 patients per year). We are also closely liaised with the German self-help group PKDCure (PKD Familiaere Zystennieren e.V.), which is dedicated to ADPKD-linked research. Recruitment of patients will be facilitated by intensified interacting with these groups. Usually, patients that are referred to our institution for evaluation or counseling are regularly seen once a year. No additional trial-related visits in our institution will be required which is in line with the observational nature of the trial. However, data recording is not restricted to parameters assessed at our center but does include also parameters assessed by the treating physician.

SOPs (Standard Operating Procedures) that include further diagnostic tests like MRI are applied routinely in ADPKD patient management in our institution. The data obtained from these tests will be entered in the registry.

At enrolment, clinical, laboratory data and imaging study findings are collected after obtaining informed consent. The parameters listed below constitute the core data set, additional parameters can be included if considered essential.

Clinical data:

  • demographic data (sex, age, height, weight)
  • family history
  • genotype (if available)
  • extrarenal ADPKD manifestations
  • co-morbidities
  • medication
  • physical examination
  • blood pressure
  • no. of extrarenal and renal complications in the past 12 months (urinary tract infections, pain episodes, macrohematuria, kidney stones, hospital admissions, ...)

Laboratory parameters include primarily (but not exclusively):

  • serum sodium
  • serum potassium
  • serum osmolality
  • serum creatinine
  • estimated glomerular filtration rate (eGFR)
  • serum urea
  • serum uric acid
  • whole blood count
  • liver enzymes, bilirubin
  • urinary sodium (spot and 24h-urine)
  • urinary potassium
  • urinary osmolality
  • urinary creatinine
  • urinary urea
  • urinary uric acid
  • urinary protein

Imaging study parameters:

  • MRI - TKV (Total Kidney Volume)
  • ultrasound
  • (CT-scan if available)

Registered patients will be provided with diaries for documentation of tolvaptan dose, adverse side effects etc. These diaries are collected on a yearly basis and the data are included in the registry. Additionally the patients will be asked to fill in a questionnaire regarding the current medication, complications of ADPKD etc. once a year as well as a commercially available SF-12 (quality of life assessment) form.

Data capture will be done at yearly intervals starting at 12 months after enrolment. It includes the biochemical parameters and imaging study findings that have been obtained over the precedent 12 months.

The following additional data will be obtained:

  • prescribed tolvaptan dose within the precedent 12 months
  • maximum dose of tolvaptan given in the precedent 12 months
  • weight, blood pressure
  • urine output
  • adverse effects
  • hospital admissions
  • occurrence of kidney pain, haematuria, or urinary tract infection
  • complications associated with extrarenal manifestations of ADPKD
  • data from diaries and questionnaires as mentioned above

According to the observational character of this study, no additional blood samples, examinations or imaging studies are required per protocol.

Keywords
polycystic kidney disease, autosomal dominant polycystic kidney disease, ADPKD, Tolvaptan, Jinarc
Medical subject headings
Polycystic Kidney, Autosomal Dominant, Polycystic Kidney Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT02497521
Study groupRoleWhat they receive
ADPKDPatients with diagnosis of ADPKD, who are either evaluated for tolvaptan treatment indication, planned for tolvaptan treatment, or are already treated with tolvaptan

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Drug dosing and titration as a measure of changes in real-life setting

    Drug dosing and titration as a measure of maximum dose and the final dose and how fast it is achieved

    Measured: 10 years

  2. Urine osmolarity as a measure of appropriate dosing

    Data on urine osmolarity will be collected to measure appropriate dosing to provide the desired protective effect

    Measured: 10 years

  3. Evaluation of rate of drug discontinuation and average Duration of therapy

    Evaluation of rate of drug discontinuation and average Duration of therapy

    Measured: 10 years

Secondary outcomes (8)

  1. Demographics

    Data on demographics will be collected

    Measured: 10 years

  2. Clinical and biochemical characteristics at enrolment and treatment initiation

    Measured: 10 years

  3. Clinical and biochemical characteristics during follow-up

    Measured: 10 years

  4. Urinary output

    Measured: 10 years

  5. Evolution of estimated glomerular Filtration rate (eGFR) over the Observation period

    Measured: 10 years

  6. Evolution of TKV

    Measured: 10 years

  7. Side effects

    Measured: 10 years

  8. Liver enzymes

    Measured: 10 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Jul 2015 (estimated)
  2. Study startNov 2015
  3. Primary completionDec 2027 (estimated)
  4. Study completionDec 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Case only
Time perspective
Prospective
Enrolment
2,000 participants (estimated)
Target duration
10 Years
IDs, dates and publications
ClinicalTrials.gov ID
NCT02497521
Sponsor's protocol ID
003
Acronym
AD(H)PKD
Status last verified
Apr 2026
First submitted
19 Jun 2015
Last updated
30 Apr 2026
Results posted
No
Data monitoring committee
No

Publications

  • Woestmann F, Strubl S, Farowski F, Arjune S, Tsakmaklis A, Todorova P, Spath MR, Brodesser S, Baar T, Grundmann F, Vehreschild MJGT, Muller RU. The Gut Microbiome in Autosomal Dominant Polycystic Kidney Disease: A Cross-Sectional Study. Kidney360. 2025 Nov 1;6(11):1906-1917. doi: 10.34067/KID.0000000836. Epub 2025 Jun 3.

    PubMed 40459942

  • Bais T, Knol MGE, Xue L, Geertsema P, Vart P, Reichel F, Arjune S, Muller RU, Dekker SEI, Salih M, Meijer E, Gansevoort RT; DIPAK Consortium. Predicting Kidney Outcomes in Autosomal Dominant Polycystic Kidney Disease: A Comprehensive Biomarker Analysis. Clin J Am Soc Nephrol. 2025 May 1;20(5):608-618. doi: 10.2215/CJN.0000000680. Epub 2025 Mar 11.

    PubMed 40067938

  • Arjune S, Lettenmeier K, Todorova P, Spath MR, Majjouti M, Mahabir E, Grundmann F, Muller RU. Inflammatory Cytokine Levels in Patients with Autosomal Dominant Polycystic Kidney Disease. Kidney360. 2024 Sep 1;5(9):1289-1298. doi: 10.34067/KID.0000000000000525. Epub 2024 Jul 24.

    PubMed 39046800

  • van Heugten MH, Blijdorp CJ, Arjune S, van Willigenburg H, Bezstarosti K, Demmers JAA, Musterd-Bhaggoe U, Meijer E, Gansevoort RT, Zietse R, Hayat S, Kramann R, Muller RU, Salih M, Hoorn EJ; DIPAK Consortium. Matrix Metalloproteinase-7 in Urinary Extracellular Vesicles Identifies Rapid Disease Progression in Autosomal Dominant Polycystic Kidney Disease. J Am Soc Nephrol. 2024 Mar 1;35(3):321-334. doi: 10.1681/ASN.0000000000000277. Epub 2023 Dec 11.

    PubMed 38073039

  • Woznicki P, Siedek F, van Gastel MDA, Dos Santos DP, Arjune S, Karner LA, Meyer F, Caldeira LL, Persigehl T, Gansevoort RT, Grundmann F, Baessler B, Muller RU. Automated Kidney and Liver Segmentation in MR Images in Patients with Autosomal Dominant Polycystic Kidney Disease: A Multicenter Study. Kidney360. 2022 Dec 29;3(12):2048-2058. doi: 10.34067/KID.0003192022. eCollection 2022 Dec 29.

    PubMed 36591351

Questions about this trial

What is this clinical trial studying?

An observational study of ADPKD (Autosomal Dominant Polycystic Kidney Disease), run by University of Cologne at 12 sites in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 30 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Roman-Ulrich Mueller, MD (+49(0)22147897222, roman-ulrich.mueller@uk-koeln.de), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Drug dosing and titration as a measure of changes in real-life setting (time frame: 10 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02497521, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Apr 2026.

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