Was your son in the placebo group in the PTC ataluren trial?
 

PPMD has partnered with researchers at the National Institutes of Health (NIH) for an interview study. The purpose of this study is to describe the experiences of people involved in phase II clinical trials of ataluren for Duchenne muscular dystrophy. We are interested in learning about motivations for being in the clinical trial, expectations of the trial, the experience of the trial, and interactions between families and professionals.
 

Our thanks to the many families who have already been interviewed. The researchers are still looking for parents whose children were in the PLACEBO GROUP for the phase 2b trial. If your son was in the placebo group, please consider being interviewed about your experience. Your participation in the study is kept private.
 

What happens in the study?
Participating in the study includes a one-time telephone interview that will take about 45 minutes to 1 hour to complete, and the possibility of a follow-up interview.
 

Who can participate in the study?
Parents who participate in the study must:

• Be over 18 years of age,
• have at least one child with Duchenne Muscular Dystrophy who was enrolled in the phase IIa extension trial or phase IIb trial of ataluren at a study site in the United States,
• be a primary caregiver for their child,
• have been involved in deciding whether the child would participate in the clinical trial, and
• have gone with their child to at least one visit to the clinical trial site.

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

Contact

For more information, contact Holly Peay at hpeay@mail.nih.gov or 443-791-5927.

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