The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Study type: Observational

Summary: 
      The purpose of this study is to describe the experiences of parents, clinician researchers,
      and industry professionals who were involved in phase II clinical trials of Ataluren for
      Duchenne muscular dystrophy. We are especially interested in learning about motivations for
      being involved in the clinical trial, expectations of the trial, the experience of the trial,
      and relationships between the parents of children involved in the trial, the clinician
      researchers, and PTC Therapeutics. In addition, we would like to learn more about whether and
      how families and advocacy organizations experiences in following the progress of the drug,
      encouraging the clinical trial, and supporting the phase II trials may have affected
      participants thoughts and feelings about the study.
    

Detailed description: 
      Some aspects of the traditional process for the development of new potential therapeutics for
      rare disorders and the ensuing research on safety and efficacy are changing. This is due, in
      part, to the increased ability of families, parents, individuals with rare disorders, and
      advocacy organizations to access and control information, to provide monetary or other
      incentives, and to communicate directly with biopharmaceutical companies and clinical
      investigators. Increasingly, advocacy organizations (and by extension, the families they
      serve) create or participate in disorder registries intended to facilitate research by
      industry and access of families to clinical trials; fund promising bench and clinical
      research; and lobby for outcomes beneficial to drug developers and researchers, such as
      increased funding or facilitated FDA approval. The involvement of families in the research
      process is likely to have implications for how willing individuals are to participate in
      studies, their expectations and perceptions of the study experience, and their behavior
      during the study. Their increased level of involvement may also affect how families perceive
      and respond to a clinical trial in which the results are not as promising as hoped, as
      quickly as hoped. This partnership approach has implications for biopharmaceutical companies
      who sponsor treatment trials and for clinical investigators who lead and implement the
      trials. This qualitative interview study aims to describe, from the perspectives of parents
      of children involved in a clinical trial, research clinicians implementing the trial, and the
      company sponsoring the trial, the experience of this new, more collaborative research
      process. In this case, we will examine the Ataluren trial for Duchenne muscular dystrophy.
      The results from this study are hypothesis-generating for downstream research and can inform
      biopharmaceutical companies and clinician researchers as they plan and implement clinical
      trials for rare disorders and help guide advocacy groups and parent advocates as they partner
      with researchers and industry.
    

Time perspective: Retrospective
Keywords: Expectations; Duchenne Muscular Dystrophy; Clinical Trial; Motivations