Trial Profile
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
Status:
Completed
Phase of Trial:
Phase II
Latest Information Update: 06 Nov 2021
Price :
$35
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At a glance
- Drugs Ataluren (Primary)
- Indications Duchenne muscular dystrophy
- Focus Adverse reactions
- Sponsors PTC Therapeutics
- 12 Aug 2020 The number of primary end-points is increased from 3 to 4 (3 newly added). The duration of treatment and follow up phases has been changed fram 4 weeks and 48 weeks to 52 weeks and 4 weeks respectively, also changing the duration of total study from 52 to 60 weeks. Treatments has been modified to included weight assessment of all participants in every 12 weeks.
- 09 Jul 2018 Results at week 28 published in the PTC Therapeutics Media Release
- 09 Jul 2018 According to a PTC Therapeutics media release, the data was presented at the International Congress on Neuromuscular Diseases 2018.