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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ClinicalTrials.gov Identifier: NCT01182324 |
Recruitment Status :
Completed
First Posted : August 16, 2010
Last Update Posted : April 5, 2018
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Condition or disease |
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Duchenne Muscular Dystrophy |
Study Type : | Observational |
Actual Enrollment : | 21 participants |
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 |
Study Start Date : | July 30, 2010 |
Study Completion Date : | June 24, 2013 |

- To describe, inclusive of the perspectives and voices of all of the major participants, the shared experiences of parents, clinician researchers, and industry professionals who were involved in phase II clinical trials for Duchenne Muscular Dyst...

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years to 99 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01182324
United States, Maryland | |
National Human Genome Research Institute (NHGRI), 9000 Rockville Pike | |
Bethesda, Maryland, United States, 20892 |
Principal Investigator: | Barbara B Biesecker | National Human Genome Research Institute (NHGRI) |
ClinicalTrials.gov Identifier: | NCT01182324 |
Other Study ID Numbers: |
999910171 10-HG-N171 |
First Posted: | August 16, 2010 Key Record Dates |
Last Update Posted: | April 5, 2018 |
Last Verified: | June 24, 2013 |
Expectations Duchenne Muscular Dystrophy Clinical Trial Motivations |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |