Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Public ClinicalTrials.gov record NCT01826487. 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 Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Dystrophinopathy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of dystrophinopathy in approximately 10-15 percent (%) of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. The main goal of this Phase 3 study is to evaluate the effect of ataluren on walking ability. The effect of ataluren on physical function, quality of life, and activities of daily living will be evaluated. This study will also provide additional information on the long-term safety of ataluren.
Study identification
- NCT ID
- NCT01826487
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 230 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- 7 Years to 16 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 25, 2013
- Primary completion
- Aug 19, 2015
- Completion
- Aug 19, 2015
- Last update posted
- Aug 3, 2020
2013 – 2015
United States locations
- U.S. sites
- 21
- U.S. states
- 16
- U.S. cities
- 20
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, Los Angeles | Los Angeles | California | 90095 | — |
| UC Davis Medical Center | Sacramento | California | 95817 | — |
| 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 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| 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 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229-3039 | — |
| 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 34 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01826487, 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 3, 2020 · Synced Sep 11, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01826487 live on ClinicalTrials.gov.