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

Sponsor

University of Cologne (Other)

Overall Status

Recruiting

CT.gov ID

NCT02497521

Collaborator

(none)

500

Enrollment

Locations

145

Anticipated Duration (Months)

41.7

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

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	Intervention/Treatment	Phase
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]

Anticipated Enrollment :

500 participants

Observational Model:

Case-Only

Time Perspective:

Prospective

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 :

Nov 1, 2015

Anticipated Primary Completion Date :

Nov 1, 2025

Anticipated Study Completion Date :

Dec 1, 2027

Arms and Interventions

Arm	Intervention/Treatment
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

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

Secondary Outcome Measures

Demographics [10 years]
Data on demographics will be collected
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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	Site	City	State	Country	Postal Code
1	Fachinternistische Gemeinschaftspraxis Markgraeferland	Müllheim	Baden-Wuerttemberg	Germany	79379
2	University Hospital of Wuerzburg, ZIM	Wuerzburg	Bayern	Germany	97080
3	Medizinische Hochschule Hannover	Hannover	Niedersachsen	Germany	30625
4	University Hospital of Cologne	Cologne	Nordrhein-Westfalen	Germany	50937
5	Nieren- und Diabeteszentrum Nettetal-Lobberich	Nettetal	Nordrhein-Westfalen	Germany	41334
6	University Hospital of Leipzig, Nephrologische Ambulanz	Leipzig	Sachsen	Germany	04103
7	Praxisgemeinschaft Dr. Peschel	Leipzig	Sachsen	Germany	04107
8	University Hospital of Schleswig-Holstein	Lubeck	Schleswig-Holstein	Germany	23538
9	Nierenzentrum Lübeck	Lübeck	Schleswig-Holstein	Germany	23562
10	University Hospital of Jena	Jena	Thueringen	Germany	07747
11	Charité Universitätsmedizin Berlin	Berlin		Germany	10117
12	Robert-Bosch-Krankenhaus	Stuttgart		Germany	70376