Trial document




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  DRKS00012699

Trial Description

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Title

Longitudinal Data Collection from Patients with Spinal Muscular Atrophy:
The SMArtCARE Database

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Trial Acronym

SMArtCARE

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URL of the Trial

http://www.smartcare.de

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Brief Summary in Lay Language

The aim of SMArtCARE is to collect longitudinal “real-world data” on all available SMA patients independent of their actual treatment regime as disease-specific SMA registry. For this purpose, an online platform will be provided for SMA patients seen by health-care providers in German-speaking countries. Data are collected during routine patient visits and items for data collection are aligned with the international consensus for SMA registries. SMArtCARE is a joint initiative of neurologists, child neurologists, and patient organizations.

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Brief Summary in Scientific Language

The aim of SMArtCARE is to collect longitudinal “real-world data” on all available SMA patients independent of their actual treatment regime as a disease-specific SMA registry. For this purpose, an online platform will be provided for SMA patients seen by health-care providers in German-speaking countries. Data are collected during routine patient visits and items for data collection are aligned with the international consensus for SMA registries. SMArtCARE collects data on motor function as well as changes in respiratory function, nutritional status and orthopedic symptoms. To evaluate changes in motor function, standardized physiotherapeutic assessments are performed. SMArtCARE is a joint initiative of neurologists, child neurologists, and patient organizations.

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Organizational Data

  •   DRKS00012699
  •   2018/08/09
  •   [---]*
  •   yes
  •   Approved
  •   56/18, Ethik-Kommission der Albert-Ludwigs-Universität Freiburg
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Secondary IDs

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

  •   5q spinal muscular atrophy (SMA)
  •   G12.0 -  Infantile spinal muscular atrophy, type I [Werdnig-Hoffman]
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Interventions/Observational Groups

  •   The aim of SMArtCARE is to collect longitudinal “real-world data” on all available SMA patients independent of their actual treatment regime as a disease-specific SMA registry. For this purpose, an online platform will be provided for SMA patients seen by health-care providers in German-speaking countries. Data are collected during routine patient visits and items for data collection are aligned with the international consensus for SMA registries. SMArtCARE collects data on motor function as well as changes in respiratory function, nutritional status and orthopedic symptoms. To evaluate changes in motor function, standardized physiotherapeutic assessments are performed.
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Characteristics

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

The aim of SMArtCARE is to collect data from all SMA patients during routine patient visits. SMArtCARE collects data on motor function as well as changes in respiratory function, nutritional status and orthopedic symptoms. To evaluate changes in motor function, standardized physiotherapeutic assessments are performed.

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Secondary Outcome

na

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Countries of Recruitment

  •   Germany
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Locations of Recruitment

  • University Medical Center 
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Recruitment

  •   Actual
  •   2018/07/15
  •   1000
  •   Multicenter trial
  •   National
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Inclusion Criteria

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

• All patients with genetically confirmed 5q SMA with residence in German-speaking regions.
• Informed consent provided by patient/caregiver

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Exclusion Criteria

• Further types of SMA (non 5q SMA)
• Participation in an AMG trial

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Addresses

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    • Universitätsklinikum Freiburg Zentrum für Neuropädiatrie und Muskelerkrankungen
    • Mr.  Prof. Dr.  Janbernd  Kirschner 
    • 79110  Freiburg
    • Germany
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    •   [---]*
    •   [---]*
    •   [---]*
    •   [---]*
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    • Universitätsklinikum Freiburg Zentrum für Neuropädiatrie und Muskelerkrankungen
    • Mr.  Prof. Dr.  Janbernd  Kirschner 
    • Mathildenstr. 1
    • 79110  Freiburg
    • Germany
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    • Universitätsklinikum Freiburg Zentrum für Neuropädiatrie und Muskelerkrankungen
    • Mr.  Prof. Dr.  Janbernd  Kirschner 
    • Mathildenstr. 1
    • 79110  Freiburg
    • Germany
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Sources of Monetary or Material Support

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    • Biogen International GmbH
    • 6300  Zug
    • Switzerland
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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.