Parents’ Perceptions Sought of DMD/BMD Ataluren Trials

The National Institutes of Health wants to hear from parents and professionals about their experiences with the recent ataluren trials in Duchenne/Becker muscular dystrophies

Article Highlights:
  • Investigators at the National Human Genome Research Institute at the National Institutes of Health want to conduct post-trial interviews with parents and clinical investigators involved in the recent phase 2 trials of ataluren for Duchenne and Becker muscular dystrophies.
  • Investigators want to learn more about the motivations, expectations and experiences of parents and professionals involved in the trials, with the goal of improving the clinical trial process.
  • The study involves an hour-long phone interview, with the possibility of a follow-up interview. Identities of study participants will be kept confidential
by Margaret Wahl on December 21, 2010 - 3:03pm

If your child participated in a phase 2 clinical trial of ataluren (PTC124) for Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) conducted by PTC Therapeutics, you're invited to take part in a National Institutes of Health (NIH) study about the experience.

The study involves at least one telephone interview. In addition to parents of trial participants, the researchers will be interviewing clinicians and industry professionals who were involved in these phase 2 trials.

"We're especially interested in learning about motivations for being involved in the clinical trial, expectations of the trial, the experience of the trial, and the interactions between the parents of children involved in the trial, the clinician researchers, and PTC Therapeutics," said study investigator Holly Peay.

Peay and her colleague Barbara Biesecker, both at the National Human Genome Research Institute at NIH, are co-investigators on the ataluren trial experience study.

"We hope the information we learn from this interview study can be used to continue to improve the partnership among families, advocacy organizations, clinicians and industry during the clinical trial process," Peay said.

About the study

Participating in this study involves a telephone interview that will take about 45 minutes to an hour, with the possibility of a follow-up interview. Interviews will be conducted in English.

Parents will be asked about their decision to enroll their son in the ataluren trial, their expectations and hopes for the trial, and their and their son's experiences during and after the trial.

Parents who participate must:

  • be at least 18 years old
  • have at least one child with DMD/BMD who was enrolled in the phase 2 extension trial or the phase 2b 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 trial; and
  • have gone with their child to at least one visit to the clinical trial site.

Investigators say any information that identifies individual participants will remain confidential, and when the study results are reported, they will not share the identities of those who took part.

However, they note that "the community of parents whose children participated in the ataluren trials is small" and that "it may be possible for some people who are very familiar with the ataluren trial to guess the identity of some participants."

For more information, contact Holly Peay at or Barbara Biesecker at, or call (301) 496-3979.

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

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