An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
Public ClinicalTrials.gov record NCT02090959. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren \[PTC124-GD-020-DMD {NCT01826487}\]) to augment the overall safety database. Screening and baseline procedures are structured to avoid a gap in treatment between the double-blind study (PTC124-GD-020-DMD) and this extension study. This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.
Study identification
- NCT ID
- NCT02090959
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 219 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- 7 Years to 15 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 20, 2014 (Actual)
- Primary completion
- Jun 12, 2018 (Actual)
- Completion
- Jun 12, 2018 (Actual)
- Last update posted
- Aug 11, 2020
2014 – 2018
United States locations
- U.S. sites
- 21
- U.S. states
- 16
- U.S. cities
- 21
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, Los Angeles | Los Angeles | California | 90095 | — |
| UC Davis Medical Center | Sacramento | California | 95817 | — |
| Stanford University Medical Center | Stanford | California | 94305 | — |
| Children's Hospital Colorado - Center for Cancer and Blood Disorders | Aurora | Colorado | 80045 | — |
| Child Neurology Center of Northwest Florida | Gulf Breeze | Florida | 32561 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| University of Iowa | Iowa City | Iowa | 52242 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research | St Louis | Missouri | 63110 | — |
| Columbia University College of Physicians & Surgeons | New York | New York | 10032 | — |
| Children's Hospital Cincinnati | Cincinnati | Ohio | 45229 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43209 | — |
| Oregon Health & Science University | Portland | Oregon | 97239 | — |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| Childrens Medical Center Dallas, Texas | Dallas | Texas | 75207 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
| University of Utah | Salt Lake City | Utah | 84112 | — |
| Seattle Children's Hospital - Childhood Cancer and Blood Disorders | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 37 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02090959, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 11, 2020 · Synced Oct 28, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT02090959 live on ClinicalTrials.gov.