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The German ADPKD Tolvaptan Treatment Registry

NCT ID: NCT02497521

Last Updated: 2024-12-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

2000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-11-30

Study Completion Date

2027-12-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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ADPKD (Autosomal Dominant Polycystic Kidney Disease)

Keywords

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polycystic kidney disease autosomal dominant polycystic kidney disease ADPKD Tolvaptan Jinarc

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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ADPKD

Patients with diagnosis of ADPKD, who are either evaluated for tolvaptan treatment indication, planned for tolvaptan treatment, or are already treated with tolvaptan

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

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

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Cologne

OTHER

Sponsor Role lead

Responsible Party

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Roman Müller

MD, Prof.

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Thomas Benzing, MD, Prof.

Role: PRINCIPAL_INVESTIGATOR

University Hospital of Cologne

Roman-Ulrich Müller, MD, Prof.

Role: PRINCIPAL_INVESTIGATOR

University Hospital Cologne

Locations

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Fachinternistische Gemeinschaftspraxis Markgraeferland

Müllheim, Baden-Wurttemberg, Germany

Site Status RECRUITING

University Hospital of Wuerzburg, ZIM

Würzburg, Bavaria, Germany

Site Status RECRUITING

Medizinische Hochschule Hannover

Hanover, Lower Saxony, Germany

Site Status RECRUITING

University Hospital of Cologne

Cologne, North Rhine-Westphalia, Germany

Site Status RECRUITING

Nieren- und Diabeteszentrum Nettetal-Lobberich

Nettetal, North Rhine-Westphalia, Germany

Site Status ACTIVE_NOT_RECRUITING

University Hospital of Leipzig, Nephrologische Ambulanz

Leipzig, Saxony, Germany

Site Status RECRUITING

Praxisgemeinschaft Dr. Peschel

Leipzig, Saxony, Germany

Site Status RECRUITING

University Hospital of Schleswig-Holstein

Lübeck, Schleswig-Holstein, Germany

Site Status RECRUITING

Nierenzentrum Lübeck

Lübeck, Schleswig-Holstein, Germany

Site Status RECRUITING

University Hospital of Jena

Jena, Thuringia, Germany

Site Status RECRUITING

Charité Universitätsmedizin Berlin

Berlin, , Germany

Site Status ACTIVE_NOT_RECRUITING

Robert-Bosch-Krankenhaus

Stuttgart, , Germany

Site Status RECRUITING

Countries

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Germany

Central Contacts

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Roman-Ulrich Mueller, MD

Role: CONTACT

Phone: +49(0)22147897222

Email: [email protected]

Cornelia Boehme

Role: CONTACT

Phone: +49(0)22147897222

Email: [email protected]

Facility Contacts

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Markus Cybulla, MD

Role: primary

Gudrun Tech

Role: backup

Daniel Kraus, MD

Role: primary

Kirsten Hofmann

Role: backup

Roland Schmitt, MD

Role: primary

Hermann Haller, MD

Role: backup

Thomas Benzing, MD

Role: primary

Roman-Ulrich Mueller, MD

Role: backup

Jan Halbritter, MD

Role: primary

Katrin Peschel, MD

Role: primary

Figen Cakiroglu, MD

Role: primary

Alexandra Tiedtke

Role: backup

Peter Gerke, MD

Role: primary

Lutz Hennings

Role: backup

Martin Busch, MD

Role: primary

Dominik Alscher, MD

Role: primary

David Callau Monje, MD

Role: backup

References

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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.

Reference Type DERIVED
PMID: 40067938 (View on PubMed)

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.

Reference Type DERIVED
PMID: 39046800 (View on PubMed)

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.

Reference Type DERIVED
PMID: 38073039 (View on PubMed)

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.

Reference Type DERIVED
PMID: 36591351 (View on PubMed)

Related Links

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https://www.adpkd.org/

Portal for patients and referring physicians

Other Identifiers

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003

Identifier Type: -

Identifier Source: org_study_id