The German ADPKD Tolvaptan Treatment Registry

Study Overview
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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.

Official Title
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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.

Conditions
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ADPKD (Autosomal Dominant Polycystic Kidney Disease)

Participation Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| ADPKD<br>Patients with diagnosis of ADPKD, who are either evaluated for tolvaptan treatment indication, planned for tolvaptan treatment, or are already treated with tolvaptan | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 10 years | 
| 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 | 10 years | 
| Evaluation of rate of drug discontinuation and average Duration of therapy | Evaluation of rate of drug discontinuation and average Duration of therapy | 10 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Demographics | Data on demographics will be collected | 10 years | 
| Clinical and biochemical characteristics at enrolment and treatment initiation |  | 10 years | 
| Clinical and biochemical characteristics during follow-up |  | 10 years | 
| Urinary output |  | 10 years | 
| Evolution of estimated glomerular Filtration rate (eGFR) over the Observation period |  | 10 years | 
| Evolution of TKV |  | 10 years | 
| Side effects |  | 10 years | 
| Liver enzymes |  | 10 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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polycystic kidney disease, autosomal dominant polycystic kidney disease, ADPKD, Tolvaptan, Jinarc