Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Public ClinicalTrials.gov record NCT00592553. 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 2B Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
DMD/BMD is a genetic disorder that 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 during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 13 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. This study is a Phase 2b trial that will evaluate the clinical benefit of ataluren in boys with DMD/BMD due to a nonsense mutation. The main goals of the study are to understand whether ataluren can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time.
Study identification
- NCT ID
- NCT00592553
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 174 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- 5 Years and older
- 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
- Feb 28, 2008
- Primary completion
- Dec 30, 2009
- Completion
- Dec 30, 2009
- Last update posted
- Apr 6, 2020
2008 – 2009
United States locations
- U.S. sites
- 15
- U.S. states
- 15
- U.S. cities
- 15
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California-Davis | Sacramento | California | — | — |
| The Children's Hospital | Aurora | Colorado | — | — |
| Child Neurology Center of Pensacola | Pensacola | Florida | — | — |
| University of Iowa Healthcare | Iowa City | Iowa | — | — |
| University of Kansas Medical Centre | Kansas City | Kansas | — | — |
| Children's Hospital of Boston/Harvard Medical School | Boston | Massachusetts | — | — |
| University of Minnesota | Minneapolis | Minnesota | — | — |
| Washington University School of Medicine | St Louis | Missouri | — | — |
| Columbia University Medical School | New York | New York | — | — |
| Duke University Medical Center | Durham | North Carolina | — | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229-3039 | — |
| Shriners Hospital for Children | Portland | Oregon | — | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | — | — |
| Southwestern University | Dallas | Texas | — | — |
| University of Utah | Salt Lake City | Utah | — | — |
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 22 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00592553, 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 Apr 6, 2020 · Synced Sep 11, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00592553 live on ClinicalTrials.gov.