Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
Public ClinicalTrials.gov record NCT00264888. 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 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the dystrophin protein. PTC124 has been shown to partially restore dystrophin production in animals with DMD due to a nonsense mutation. The main purpose of this study is to understand whether PTC124 can safely increase functional dystrophin protein in the muscles of patients with DMD due to a nonsense mutation.
Study identification
- NCT ID
- NCT00264888
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 38 participants
Conditions and interventions
Conditions
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
- Dec 2005 (Type not available)
- Primary completion
- May 2007 (Actual)
- Completion
- May 2007 (Actual)
- Last update posted
- Jan 14, 2009
2005 – 2007
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229-3039 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104-4399 | — |
| University of Utah | Salt Lake City | Utah | 84112 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00264888, 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 Jan 14, 2009 · Synced Oct 26, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT00264888 live on ClinicalTrials.gov.