Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
Public ClinicalTrials.gov record NCT01247207. 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
An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.
Study identification
- NCT ID
- NCT01247207
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 265 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Not listed
- 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
- Nov 22, 2010 (Actual)
- Primary completion
- Feb 10, 2026 (Actual)
- Completion
- Feb 10, 2026 (Actual)
- Last update posted
- Oct 8, 2026
2010 – 2026
United States locations
- U.S. sites
- 29
- U.S. states
- 21
- U.S. cities
- 28
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Childrens Hospital | Phoenix | Arizona | 85016 | — |
| Children's Hospital of Los Angeles | Los Angeles | California | 90027 | — |
| UCLA | Los Angeles | California | 90095 | — |
| Stanford University | Palo Alto | California | 94304 | — |
| University of California-Davis | Sacramento | California | 95817 | — |
| University of CA, San Francisco-Benioff Children's Hospital | San Francisco | California | 94158 | — |
| Children's Hospital Colorado - Center for Cancer and Blood Disorders | Aurora | Colorado | 80045 | — |
| Child Neurology Center of NW Florida NW | Gulf Breeze | Florida | 32561 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30329 | — |
| Rush Univ Medical Center | Chicago | Illinois | 60612 | — |
| University of Iowa Children's Hospital | Iowa City | Iowa | 52242 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| Children's Hospital of Boston/Harvard Medical School | Boston | Massachusetts | 02115 | — |
| University of Michigan | Ann Arbor | Michigan | 48109 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Washington University Medical School | St Louis | Missouri | 63110 | — |
| Columbia University Pediatric Neuromuscular Center | New York | New York | 10032 | — |
| Duke University Medical Center | Durham | North Carolina | 27710 | — |
| Cincinnati Childrens Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Shriners Hospital for Children-Portland | Portland | Oregon | 97239 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 16148 | — |
| Childrens Medical Center Dallas | Dallas | Texas | 75207 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
| University of Texas Health Science Center at San Antonio | San Antonio | Texas | 78229 | — |
| University of Utah | Salt Lake City | Utah | 84112 | — |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | — |
| Seattle Children's Hospital | 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 5 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01247207, 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 Oct 8, 2026 · Synced Oct 25, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT01247207 live on ClinicalTrials.gov.