Trial document





This trial has been registered retrospectively.
drksid header

  DRKS00010261

Trial Description

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Title

An international registry study on autosomal recessive polycystic kidney disease (ARPKD)

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Trial Acronym

ARegPKD

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URL of the Trial

http://www.aregpkd.org

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Brief Summary in Lay Language

Autosomal recessive polycystic kidney disease (ARPKD) is a rare and severe disorder of early childhood mainly affecting the kidneys and the liver. Patients with ARPKD show very variable clinical courses. The underlying mechanisms of his variability remain incompletely understood. Data on long-term courses and the response of patients to current therapeutic approaches remain scarce. ARegPKD therefore collects clinical data to identify potential risk factors and clinical markers of different courses and to lay the foundation for evidence-based treatment.

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Brief Summary in Scientific Language

Autosomal recessive polycystic kidney disease (ARPKD) is a rare and severe disorder of early childhood mainly affecting the kidneys and the liver. Mutations in a single gene, PKHD1, are the cause of ARPKD that shows a high degree of unexplained phenotypic variability. Current treatment recommendations rely on data from small cohorts and are largely opinion-based. Data on long-term courses and the response of patients to current therapeutic approaches remain scarce. ARegPKD therefore collects clinical data to identify potential risk factors and clinical markers of different courses and to lay the foundation for evidence-based treatment. In addition to the clinical registry study we offer reference histology and have established a centralized ARPKD-specific biobanking.

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Organizational Data

  •   DRKS00010261
  •   2016/04/25
  •   [---]*
  •   yes
  •   Approved
  •   12-118, Ethik-Kommission der Medizinischen Fakultät der Universität zu Köln
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Secondary IDs

  • [---]*
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Health Condition or Problem studied

  •   Q61.1 -  Polycystic kidney, autosomal recessive
  •   Polycystic kidney disease, ARPKD
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Interventions/Observational Groups

  •   Pure observational registry study.
    Pro- and retrospective collection of clinical data (e.g. liver- and kidney-related laboratory values, ultrasound data, data on renal replacement therapy etc.).
    Collection of samples for biobanking only within the setting of clinically required blood sampling.
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Characteristics

  •   Non-interventional
  •   Observational study
  •   Single arm study
  •   Open (masking not used)
  •   [---]*
  •   Uncontrolled/Single arm
  •   Other
  •   Single (group)
  •   N/A
  •   N/A
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Primary Outcome

n/a - pure observational study
Clinical characterization of ARPKD patients within the Setting of current Treatment.
A first Goal is the collection of data on a large international and deeply phenotyped cohort of ARPKD patients.

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Secondary Outcome

n/a - pure observational study

Identification of clinical and/or biochemical risk markers

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Countries of Recruitment

  •   Germany
  •   United Kingdom
  •   Belgium
  •   Switzerland
  •   Czech Republic
  •   Estonia
  •   Egypt
  •   Spain
  •   France
  •   Georgia
  •   Greece
  •   Hungary
  •   Iran, Islamic Republic of
  •   Italy
  •   Lithuania
  •   Netherlands
  •   Poland
  •   Portugal
  •   Serbia
  •   Russian Federation
  •   Turkey
  •   Austria
  •   United Arab Emirates
  •   China
  •   India
  •   Romania
  •   Tunisia
  •   Belarus
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Locations of Recruitment

  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
  • Medical Center 
  • University Medical Center 
  • University Medical Center 
  • Medical Center 
  • University Medical Center 
  • other 
  • Medical Center 
  • University Medical Center 
  • University Medical Center 
  • University Medical Center 
  • Medical Center 
  • University Medical Center 
  • Medical Center 
  • Medical Center 
  • University Medical Center 
  • University Medical Center 
  • Medical Center 
  • University Medical Center 
  • Medical Center 
  • University Medical Center 
  • Medical Center 
  • Medical Center 
  • Medical Center 
  • University Medical Center 
  • University Medical Center 
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Recruitment

  •   Actual
  •   2013/05/29
  •   500
  •   Multicenter trial
  •   International
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Inclusion Criteria

  •   Both, male and female
  •   no minimum age
  •   no maximum age
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Additional Inclusion Criteria

Clinical or histological diagnosis of ARPKD
Signed informed consent

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Exclusion Criteria

Clinical, histological or genetic diagnosis of any other cystic kidney disease

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Addresses

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    • Universitätsklinikum Köln
    • Kerpener Str. 62
    • 50924  Köln
    • Germany
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    • Unikinderklinik KölnPädiatrische Nephrologie
    • Mr.  PD Dr.  Max  Liebau 
    • Kerpener Str. 62
    • 50937  Köln
    • Germany
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    • Unikinderklinik KölnPädiatrische Nephrologie
    • Mr.  PD Dr.  Max  Liebau 
    • Kerpener Str. 62
    • 50937  Köln
    • Germany
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Sources of Monetary or Material Support

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    • Bundesministerium für Bildung und Forschung Dienstsitz Berlin
    • Friedrichstraße 130 B
    • 10117  Berlin
    • Germany
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    • Marga und Walter Boll-Stiftung
    • Marie-Curie-Straße 8
    • 50170  Kerpen
    • Germany
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    •   [---]*
    •   [---]*
    •   [---]*
    •   [---]*
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    • European Society for Paediatric Nephrology
    • Hufelandstr.55
    • 45133  Essen
    • Germany
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    •   [---]*
    •   [---]*
    •   [---]*
    •   [---]*
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    • Gesellschaft für Pädiatrische Nephrologie
    • Chausseestr. 128/129
    • 10115  Berlin
    • Germany
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    •   [---]*
    •   [---]*
    •   [---]*
    •   [---]*
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Status

  •   Recruiting ongoing
  •   [---]*
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* This entry means the parameter is not applicable or has not been set.