Clinical trial
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
NCT02819557 is registered in ClinicalTrials.gov with PTC Therapeutics as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- 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
- Lead sponsor
- PTC Therapeutics
- Phase
- Phase 2
- Overall status
- Completed
- Start date
- June 9, 2016
- Completion date
- February 9, 2018
- Enrollment
- 14 (actual)
- Sites
- 6
Conditions
As the registry lists them; a condition with its own page is linked.
duchenne muscular dystrophy