Is a
Clinical Study attributes
NCT Number
Health Conditions in Trial
Trial Recruitment Size
380
Trial Sponsor
Trial Collaborator
Clinical Trial Start Date
2005
0Primary Completion Date
2007
0Study Completion Date
2007
0Clinical Trial Study Type
Interventional0
Interventional Trial Purpose
Treatment0
Interventional Trial Phase
Phase 20
Participating Facility
Official Name
A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy0
Last Updated
January 14, 2009
0Allocation Type
Non-Randomized0
Intervention Model
Single Group Assignment0
Masking Type
None (Open Label)0
Study summary
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.
Timeline
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Further Resources
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