The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor

[Reference: http://clinicaltrials.gov/ct2/show/NCT01182324?term=ataluren&ra... ]

 

 

Purpose

The traditional process for the development of new potential therapeutics for rare disorders and the ensuing research on safety and efficacy is 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. This partnership approach also 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 (PTC-124) trial for boys with 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.


Condition
Duchenne Muscular Dystrophy

Study Type: Observational
Study Design: Time Perspective: Retrospective
Official Title: The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 160
Study Start Date: July 2010
Detailed Description:

The traditional process for the development of new potential therapeutics for rare disorders and the ensuing research on safety and efficacy is 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 failed clinical trial. 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 a much-hyped Ataluren trial that failed to show clinical benefit on the primary research outcome. At the time of submitting this protocol to IRB, the story is still emerging, i.e., though there was no benefit found on the primary outcome measure in patients receiving high dose, further evaluation of the data suggests that boys receiving low dose of Ataluren showed benefit on at least some outcome measures. It is not yet clear how the company and the FDA will choose to move forward, if at all.

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria
  • INCLUSION CRITERIA:
  • US residents over 18 years of age who have at least one child with Duchenne Muscular Dystrophy who was enrolled in the phase IIa extension trial or phase IIb trial of PTC124. Participants must be a primary caregiver for their children, must have been involved in deciding whether the child would participate in the clinical trial, and must have accompanied their child to at least one visit to the clinical trial site.
  • Clinician researchers over 18 years of age who were involved with implementing the clinical trial at a study site. Whenever possible, we will interview the Principle Investigators at a study site.
  • Representatives of PTC Therapeutics over 18 years of age. PTC Therapeutics has provided verbal agreement to identify several representatives for the study.

All participants must be willing and able to complete an approximately 1-hour long telephone interview in English.

EXCLUSION CRITERIA:

-Must meet inclusion criteria.

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01182324

Contacts
Contact: Barbara B. Biesecker (301) 496-3979 barbarab@nhgri.nih.gov

Locations
United States, Maryland
National Human Genome Research Institute (NHGRI), 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
  More Information

Publications:
ClinicalTrials.gov Identifier: NCT01182324     History of Changes
Other Study ID Numbers: 999910171, 10-HG-N171
Study First Received: August 13, 2010
Last Updated: August 13, 2010
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Expectations
Duchenne Muscular Dystrophy
Clinical Trial
Motivations

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 16, 2010

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