The German ADPKD Tolvaptan Treatment Registry (AD(H)PKD)

• ClinicalTrials.gov Identifier: NCT02497521
• Recruitment Status: Recruiting
• First Posted: July 14, 2015
• Last Update Posted: September 22, 2022
• Sponsor: University of Cologne
• Information provided by (Responsible Party): Roman Müller, University Hospital of Cologne

Study Description

Brief 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 with tolvaptan are eligible. This registry is designed to provide "real-world" data on treatment management of patients with ADPKD.

Condition or disease
ADPKD (Autosomal Dominant Polycystic Kidney Disease)

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.

Study Design

• Study Type: Observational [Patient Registry]
• Estimated Enrollment: 500 participants
• Observational Model: Case-Only
• Time Perspective: Prospective
• Target Follow-Up Duration: 10 Years
• 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.
• Actual Study Start Date: November 2015
• Estimated Primary Completion Date: November 2025
• Estimated Study Completion Date: December 2027

Groups and Cohorts

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

Outcome Measures

Primary Outcome Measures :

1. Drug dosing and titration as a measure of changes in real-life setting [ Time Frame: 10 years ]
   Drug dosing and titration as a measure of maximum dose and the final dose and how fast it is achieved
2. Urine osmolarity as a measure of appropriate dosing [ Time Frame: 10 years ]
   Data on urine osmolarity will be collected to measure appropriate dosing to provide the desired protective effect
3. Evaluation of rate of drug discontinuation and average Duration of therapy [ Time Frame: 10 years ]
   Evaluation of rate of drug discontinuation and average Duration of therapy

Secondary Outcome Measures :

1. Demographics [ Time Frame: 10 years ]
   Data on demographics will be collected
2. Clinical and biochemical characteristics at enrolment and treatment initiation [ Time Frame: 10 years ]
3. Clinical and biochemical characteristics during follow-up [ Time Frame: 10 years ]
4. Urinary output [ Time Frame: 10 years ]
5. Evolution of estimated glomerular Filtration rate (eGFR) over the Observation period [ Time Frame: 10 years ]
6. Evolution of TKV [ Time Frame: 10 years ]
7. Side effects [ Time Frame: 10 years ]
8. Liver enzymes [ Time Frame: 10 years ]

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Probability Sample

Study Population

Patients with diagnosis of ADPKD and therapy with tolvaptan

Criteria

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"

Contacts and Locations

Contacts

Contact: Roman-Ulrich Mueller, MD; +49(0)22147897222; roman-ulrich.mueller@uk-koeln.de

Contact: Cornelia Boehme; +49(0)22147897222; studienzentrum-medII@uk-koeln.de

Locations

Germany

Fachinternistische Gemeinschaftspraxis Markgraeferland; Recruiting

Müllheim, Baden-Wuerttemberg, Germany, 79379

Contact: Markus Cybulla, MD

Contact: Gudrun Tech

Principal Investigator: Markus Cybulla, MD

University Hospital of Wuerzburg, ZIM; Recruiting

Wuerzburg, Bayern, Germany, 97080

Contact: Daniel Kraus, MD

Contact: Kirsten Hofmann

Principal Investigator: Christoph Wanner, MD

Sub-Investigator: Daniel Kraus, MD

Medizinische Hochschule Hannover; Recruiting

Hannover, Niedersachsen, Germany, 30625

Contact: Roland Schmitt, MD

Contact: Hermann Haller, MD

Principal Investigator: Roland Schmitt, MD

Sub-Investigator: Hermann Haller, MD

University Hospital of Cologne; Recruiting

Cologne, Nordrhein-Westfalen, Germany, 50937

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

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

Sub-Investigator: Roman-Ulrich Mueller, MD

Sub-Investigator: Franziska Grundmann, MD

Sub-Investigator: Katharina Burkert, MD

Sub-Investigator: Polina Todorova

Sub-Investigator: Victor Suarez, MD

Principal Investigator: Thomas Benzing, MD

Nieren- und Diabeteszentrum Nettetal-Lobberich; Active, not recruiting

Nettetal, Nordrhein-Westfalen, Germany, 41334

University Hospital of Leipzig, Nephrologische Ambulanz; Recruiting

Leipzig, Sachsen, Germany, 04103

Contact: Jan Halbritter, MD

Principal Investigator: Jan Halbritter, MD

Praxisgemeinschaft Dr. Peschel; Recruiting

Leipzig, Sachsen, Germany, 04107

Contact: Katrin Peschel, MD

Principal Investigator: Katrin Peschel, MD

University Hospital of Schleswig-Holstein; Recruiting

Lubeck, Schleswig-Holstein, Germany, 23538

Contact: Figen Cakiroglu, MD

Contact: Alexandra Tiedtke

Principal Investigator: Figen Cakiroglu, MD

Nierenzentrum Lübeck; Recruiting

Lübeck, Schleswig-Holstein, Germany, 23562

Contact: Peter Gerke, MD

Contact: Lutz Hennings

Principal Investigator: Peter Gerke, MD

University Hospital of Jena; Recruiting

Jena, Thueringen, Germany, 07747

Contact: Martin Busch, MD

Principal Investigator: Martin Busch, MD

Charité Universitätsmedizin Berlin; Active, not recruiting

Berlin, Germany, 10117

Robert-Bosch-Krankenhaus; Recruiting

Stuttgart, Germany, 70376

Contact: Dominik Alscher, MD

Contact: David Callau Monje, MD

Principal Investigator: Dominik Alscher, MD

Sub-Investigator: David Callau Monje, MD

Sponsors and Collaborators

University of Cologne

Investigators

• Principal Investigator: Thomas Benzing, MD, Prof.; University Hospital of Cologne
• Principal Investigator: Roman-Ulrich Müller, MD, Prof.; University Hospital Cologne

More Information

Additional Information:

Portal for patients and referring physicians 

• Responsible Party: Roman Müller, MD, Prof., University Hospital of Cologne
• ClinicalTrials.gov Identifier: NCT02497521
• Other Study ID Numbers: 003
• First Posted: July 14, 2015
• Last Update Posted: September 22, 2022
• Last Verified: September 2022

Keywords provided by Roman Müller, University Hospital of Cologne:

polycystic kidney disease; autosomal dominant polycystic kidney disease; ADPKD; Tolvaptan; Jinarc

Additional relevant MeSH terms:

Kidney Diseases; Polycystic Kidney Diseases; Polycystic Kidney, Autosomal Dominant; Urologic Diseases; Congenital Abnormalities; Kidney Diseases, Cystic; Abnormalities, Multiple; Ciliopathies; Genetic Diseases, Inborn