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NCT02819557 · ClinicalTrials.gov registry record · Phase 2
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
A Phase 2 study of Duchenne Muscular Dystrophy, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 14
- Enrollment target
- 6
- Study locations
NCT02819557: Completed Phase 2 study of Duchenne Muscular Dystrophy, sponsored by PTC Therapeutics.
NCT02819557 is a Phase 2 study of Duchenne Muscular Dystrophy that has completed, run by PTC Therapeutics. The registered enrollment target is 14 participants, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (97% lower). The trial reports 6 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02819557, a Phase 2 study of Duchenne Muscular Dystrophy, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 14 participants
- Enrollment target
- 6
- Study locations
Study Summary
This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to \<5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.
Primary Outcome
A TEAE was any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. An SAE was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity not related to dystrophinopa
Conditions Studied
Interventions
- DRUG Ataluren
Study Locations (6)
Florida
- Child Neuro NWF - Gulf Breeze
Illinois
- Rush University Medical Center - Chicago
Massachusetts
- Children's Hospital Boston - Boston
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Texas
- Children's Medical Center Dallas - Dallas
Utah
- University of Utah - Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 14 participants |
| Start Date | 2016-06-09 |
| Est. Completion | 2018-02-09 |
| Phase | Phase 2 |
What the finished NCT02819557 record still lists
NCT02819557 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 14 participants, a relatively small participant target, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (97% lower).
The record links to 1 condition, with Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 1 intervention - of which Ataluren is the first listed.
NCT02819557 lists 6 locations in 6 states (Florida, Illinois, Massachusetts).
Frequently Asked Questions
What is clinical trial NCT02819557 about?
NCT02819557 is a clinical study titled "Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy". This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to \<5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.
What is the current status of trial NCT02819557?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 14 participants. The study started on 2016-06-09. Estimated completion is 2018-02-09.
What conditions does trial NCT02819557 study?
This clinical trial studies the following conditions: Duchenne Muscular Dystrophy.
What interventions are being tested in trial NCT02819557?
The interventions under investigation include: Ataluren (DRUG).
Who is sponsoring clinical trial NCT02819557?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02819557 being conducted?
This trial has 6 study locations across Florida, Illinois, Massachusetts, Ohio, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT02819557's enrollment target sits among peer trials
14 39th of 48 higher than 10 of 48 other Duchenne Muscular Dystrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Duchenne Muscular Dystrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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