Trial document




This trial has been registered retrospectively.
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  DRKS00004563

Trial Description

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Title

Diagnostics and therapy of patients with Diamond-Blackfan anemia (DBA)

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Trial Acronym

DBA 2000

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URL of the Trial

http://www.uniklinik-freiburg.de/kinderklinik/live/fachabteilungen/klinik4/was/dba.html?weiterleitung=ja

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Brief Summary in Lay Language

n/a

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Brief Summary in Scientific Language

n/a

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Organizational Data

  •   DRKS00004563
  •   2013/05/21
  •   [---]*
  •   yes
  •   Approved
  •   205/99, Ethik-Kommission der Albert-Ludwigs-Universität Freiburg
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Secondary IDs

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

  •   D61.0 -  Constitutional aplastic anaemia
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Interventions/Observational Groups

  •   Registration of all patients with Diamond-Blackfan anemia in germany. Retrospective data collection of implemented diagnostics, therapy and therapy- related complications. Prospective data collection of clinical course diagnostics, ongoing therapy and progress of disease. Application of existing therapy guidelines. As a result of this optimizing diagnostics and therapy, improving quality of life and long-term prognosis of patients with Diamond-Blackfan anemia. Verifying these guidelines concerning therapy efficiency.
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Characteristics

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

Registration of all patients with Diamond-Blackfan anemia in germany. Retrospective data collection of implemented diagnostics, therapy and therapy- related complications. Prospective data collection of clinical course diagnostics, ongoing therapy and progress of disease. Application of existing therapy guidelines. As a result of this optimizing diagnostics and therapy, improving quality of life and long-term prognosis of patients with Diamond-Blackfan anemia. Verifying these guidelines concerning therapy efficiency.

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Secondary Outcome

Moleculargenetic analyses to identify underlying genetic defects.
Genotype-phenotype-studies about pathophysiology of DBA disease.
Identification of patients with DBA to be in line for experimental therapy questions. Therefor there will be set up separate therapy protocols.
Support of patient network „DBA-Gemeinschaft (community)“ and informing affected families about diagnostics and therapy.

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Countries of Recruitment

  •   Germany
  •   Switzerland
  •   Austria
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Locations of Recruitment

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

  •   Actual
  •   2000/12/01
  •   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

diagnosed Diamond-Blackfan-Anemia, patient informed consent

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Exclusion Criteria

none

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Addresses

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    • Universitätsklinikum Freiburg, ZKJ, Pädiatrische Hämatologie/Onkologie, Klinik IV
    • Ms.  Prof. Dr. med  Charlotte  Niemeyer 
    • Mathildenstr. 1
    • 79106  Freiburg
    • Germany
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    • Universitätsklinikum Freiburg, ZKJ, Pädiatrische Hämatologie/Onkologie, Klinik IV
    • Ms.  Prof. Dr. med  Charlotte  Niemeyer 
    • Mathildenstr. 1
    • 79106  Freiburg
    • Germany
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    • Universitätsklinikum Freiburg, ZKJ, Pädiatrische Hämatologie/Onkologie, Klinik IV
    • Ms.  Prof. Dr. med  Charlotte  Niemeyer 
    • Mathildenstr. 1
    • 79106  Freiburg
    • Germany
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Sources of Monetary or Material Support

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    • Universitätsklinikum Freiburg, Zentrum für Kinder- und Jugendmedizin, Klinik IV, Pädiatrische Hämatologie/Onkologie
    • Mathildenstr. 1
    • 79106  Freiburg
    • Germany
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    •   [---]*
    •   [---]*
    •   [---]*
    •   [---]*
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Status

  •   Recruiting ongoing
  •   [---]*
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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.