Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
Public ClinicalTrials.gov record NCT02819557. 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 the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy
Study identification
- NCT ID
- NCT02819557
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- PTC Therapeutics
- Industry
- Enrollment
- 14 participants
Conditions and interventions
Conditions
Interventions
- Ataluren Drug
Drug
Eligibility (public fields only)
- Age range
- 2 Years to 5 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
- Jun 8, 2016
- Primary completion
- Feb 8, 2018
- Completion
- Feb 8, 2018
- Last update posted
- Aug 27, 2020
2016 – 2018
United States locations
- U.S. sites
- 6
- U.S. states
- 6
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Child Neuro NWF | Gulf Breeze | Florida | 32561 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Children's Medical Center Dallas | Dallas | Texas | 75390-8843 | — |
| 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 NCT02819557, 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 27, 2020 · Synced May 17, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02819557 live on ClinicalTrials.gov.