Methodological insights from the EPISTOP trial to designing clinical trials in rare diseases—A secondary analysis of a randomized clinical trial

• Stephanie Wied ,

  Roles Conceptualization, Formal analysis, Methodology, Visualization, Writing – original draft, Writing – review & editing

  * E-mail: swied@ukaachen.de

  Affiliation Institute of Medical Statistics, RWTH Aachen University, Aachen, Germany

  https://orcid.org/0000-0003-0616-6610

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• Ralf-Dieter Hilgers,

  Roles Conceptualization, Funding acquisition, Methodology, Project administration, Resources, Supervision, Writing – review & editing

  Affiliation Institute of Medical Statistics, RWTH Aachen University, Aachen, Germany

  https://orcid.org/0000-0002-5945-1119

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• Nicole Heussen,

  Roles Writing – review & editing

  Affiliations Institute of Medical Statistics, RWTH Aachen University, Aachen, Germany, Medical School, Sigmund Freud Private University, Vienna, Austria

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• Katarzyna Kotulska,

  Roles Project administration, Writing – review & editing

  Affiliation Department of Neurology and Epileptology, The Children’s Memorial Health Insitute, Member of Epicare, Warsaw, Poland

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• Maya Dirani,

  Roles Writing – review & editing

  Affiliation Department of Pediatric Neurology, Imagine Institute Paris, Necker-Enfants Maelades Hospital, Reference Centre for Rare Epilepsies, Member of Epicare, University Paris cite, Paris, France

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• Mathieu Kuchenbuch,

  Roles Writing – review & editing

  Affiliation Department of Pediatric Neurology, Imagine Institute Paris, Necker-Enfants Maelades Hospital, Reference Centre for Rare Epilepsies, Member of Epicare, University Paris cite, Paris, France

  https://orcid.org/0000-0002-5944-2204

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• Sergiusz Jozwiak,

  Roles Project administration, Writing – review & editing

  Affiliation Research Department, The Children’s Memorial Health Insitute, Member of Epicare, Warsaw, Poland

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• Rima Nabbout

  Roles Conceptualization, Funding acquisition, Project administration, Resources, Writing – review & editing

  Affiliation Department of Pediatric Neurology, Imagine Institute Paris, Necker-Enfants Maelades Hospital, Reference Centre for Rare Epilepsies, Member of Epicare, University Paris cite, Paris, France

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