Status:
COMPLETED
The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor
Lead Sponsor:
National Human Genome Research Institute (NHGRI)
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
All Genders
18-99 years
Brief 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 d...
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 ...
Eligibility Criteria
Inclusion
- 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.
- Representatives of PTC Therapeutics over 18 years of age.
- All participants must be willing and able to complete an approximately 1-hour long telephone interview in English.
- EXCLUSION CRITERIA:
- Must meet inclusion criteria.
Exclusion
Key Trial Info
Start Date :
July 30 2010
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 24 2013
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT01182324
Start Date
July 30 2010
End Date
June 24 2013
Last Update
April 5 2018
Active Locations (1)
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1
National Human Genome Research Institute (NHGRI), 9000 Rockville Pike
Bethesda, Maryland, United States, 20892