Trial document




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  DRKS00011012

Trial Description

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Title

Questionnaire on the clinical praxis of percutaneous endoscopic gastrostomy insertion in patients with autosomal recessive polycystic kidney disease (ARPKD)

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Trial Acronym

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URL of the Trial

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Brief Summary in Lay Language

Autosomal recesssive polycystic kidney disease (ARPKD) is a severe inherited disease of childhood with hepatic involvement in form of liver fibrosis beneath the renal disease in form of polycystic kidneys. Severely affected patients still suffer significantly from loss of renal function, non-development of lungs during pregnancy leading to severe breathing problems or the complications of liver fibrosis, such as increased pressure in portal veins (portal hypertension) leading to esophageal varices. ARPKD is a major reason for kidney, liver or combined liver and kidney transplantation in early childhood. Due to these disease manifestations, children with ARPKD are at high risk of developing growth retardation and dystrophy. Implantation of an feeding tube (percutaneous endoscopic gastrostomy - PEG) is a well-known procedure in chronically ill and dystrophic children, but is seen critically in patients with ARPKD due to possible complications of portal hypertension and the possibility of necessity of dialysis via the peritoneum. On the other hand, adequate growth and weight gain is essential, e.g. in order to reach sufficient weight for possibility of transplantation. To our knowledge, there is a lack of systematic studies focussing on the issue of PEG implantation in ARPKD patients. Therefore we conduct a webbased, anonymous questionnaire of attending physicians of European pediatric nephrology and gastroenterology centers questioning the common practice regarding PEG implantations in ARPKD patients. In case, there is expericence with PEG implantation in ARPKD patients, details will be collected, such as technique of insertion, age and hepatic manifestation of the patient at insertion, performance of dialysis at insertion, complication of PEG insertion (leckage, infection, varices, re-operation). Furthermore handling of PEG in case of transplantation will be queried. The questionnaire is concluded by assessment of general benefits and risks of PEG implantation in ARPKD patients (including weight developmet and growth). Data evaluation will focus on absolute and relative frequencies (percentage) and will be presented to participating and interested colleagues.

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Brief Summary in Scientific Language

Autosomal recesssive polycystic kidney disease (ARPKD) is a severe inherited disease of childhood with obligate hepatic involvement beneath the renal phenotype in form of polycystic kidney disease. Severely affected patients still show significant morbidity and mortality due to renal insufficiency, pulmonary hypoplasia or the sequelae of portal hypertension. ARPKD is a major reason for kidney, liver or combined liver and kidney transplantation in early childhood. Due to these disease manifestations, children with ARPKD are at high risk of developing growth retardation and dystrophy. Implantation of percutaneous endoscopic gastrostomy is a well-known procedure in chronically ill and dystrophic children, but is seen critically in patients with ARPKD due to possible complications of portal hypertension and the possibility of necessity of peritoneal dialysis. On the other hand, adequate growth and weight gain is essential, e.g. in order to reach sufficient weight for possibility of transplantation. To our knowledge, there is a lack of systematic studies focussing on the issue of PEG implantation in ARPKD patients. Therefore we conduct a webbased, anonymous questionnaire of attending physicians of European pediatric nephrology and gastroenterology centers questioning the common practice regarding PEG implantations in ARPKD patients. In case, there is expericence with PEG implantation in ARPKD patients, details will be collected, such as technique of insertion, age and hepatic phenotype of patient at insertion, performance of dialysis at insertion, complication of PEG insertion (leckage, infection, varices, re-operation). Furthermore handling of PEG in case of transplantation will be queried. The questionnaire is concluded by assessment of general benefits and risks of PEG implantation in ARPKD patients (including weight developmet and growth). Data evaluation will focus on absolute and relative frequencies and will be presented to participating and interested colleagues.

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Organizational Data

  •   DRKS00011012
  •   2016/11/21
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  •   yes
  •   Approved
  •   16-322, 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
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Interventions/Observational Groups

  •   Conduction of a webbased, anonymous questionnaire of attending physicians of European pediatric nephrology and gastroenterology centers questioning the common practice regarding PEG implantations in ARPKD patients. In case, there is expericence with PEG implantation in ARPKD patients, details will be collected, such as technique of insertion, age and hepatic manifestation of the patient at insertion, performance of dialysis at insertion, complication of PEG insertion (leckage, infection, varices, re-operation). Furthermore handling of PEG in case of transplantation will be queried. The questionnaire is concluded by assessment of general benefits and risks of PEG implantation in ARPKD patients (including weight developmet and growth).
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Characteristics

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

It is the aim of the questionnaire to collect data regarding the current practice of PEG (percutaneous endoscopic gastrostomy) implantations in ARPKD (autosomal recessive polycystic kidney disease) patients in European pediatric centers and the experiences obtained. Benefit and risks of the intervention are evaluated in a retrospective manner in order to create first evidence regarding this important therapy in patients suffering from this serious disease of early childhood.

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Secondary Outcome

none

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Countries of Recruitment

  •   Germany
  •   Belgium
  •   United Kingdom
  •   France
  •   Italy
  •   Turkey
  •   Spain
  •   Portugal
  •   Poland
  •   Czech Republic
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Locations of Recruitment

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Recruitment

  •   Actual
  •   2017/02/01
  •   200
  •   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

The interviewed colleagues work at centers for pediatric nephrolgy or pediatric hepatology in Europe and diagnosed ARPKD due to clinical criteria in the patients. The data of ARPKD patients are questioned in an anonymous and retrospective manner.

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Exclusion Criteria

not willing to participate

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Addresses

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    • Universitätsklinikum Köln
    • Kerpener Str. 62
    • 50924  Köln
    • Germany
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    • Klinik für Kinder- und JugendmedizinUniklinik Köln
    • Ms.  Dr.  Kathrin  Burgmaier 
    • Kerpener Str. 62
    • 50937  Köln
    • Germany
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    • Klinik für Kinder- und JugendmedizinUniklinik Köln
    • Ms.  Dr.  Kathrin  Burgmaier 
    • Kerpener Str. 62
    • 50937  Köln
    • Germany
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Sources of Monetary or Material Support

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    • Universitätsklinikum Köln
    • Kerpener Str. 62
    • 50924  Köln
    • Germany
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Status

  •   Enrolling by invitation
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Trial Publications, Results and other Documents

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* This entry means the parameter is not applicable or has not been set.