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
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:
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."