The German ADPKD Tolvaptan Treatment Registry

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Brief Title

The German ADPKD Tolvaptan Treatment Registry

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.

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.
    

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 Type

Observational [Patient Registry]


Primary Outcome

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

Secondary Outcome

 Demographics

Condition

ADPKD (Autosomal Dominant Polycystic Kidney Disease)


Study Arms / Comparison Groups

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

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

500

Start Date

November 2015

Completion Date

December 2027

Primary Completion Date

November 2025

Eligibility 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"
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Thomas Benzing, MD, Prof., +49(0)22147897222, [email protected]

Location Countries

Germany

Location Countries

Germany

Administrative Informations


NCT ID

NCT02497521

Organization ID

003


Responsible Party

Principal Investigator

Study Sponsor

University of Cologne


Study Sponsor

Thomas Benzing, MD, Prof., Principal Investigator, University Hospital of Cologne


Verification Date

February 2021