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A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension, (Clinical Trial 041).
PTC124-GD-041-DMD (ALSO KNOWN AS STUDY 041) CLINICAL TRIAL OF ATALUREN IN NONSENSE MUTATION DUCHENNE MUSCULAR DYSTROPHY (NMDMD)
| Sponsor: | PTC Therapeutics |
| Enrolling: | Male Patients Only |
| Study Length: | 144 Weeks |
| Clinic Visits: | 11 |
| IRB Number: | AAAR4715 |
| U.S. Govt. ID: | NCT03179631 |
| Contact: | Ameneh Onativia: 212-342-3679 / am2959@cumc.columbia.edu |
Additional Study Information:
Columbia University is currently enrolling in an Ataluren Study from PTC Therapeutics, called PTC-041. We are looking for 5 year old boys, ambulating, confirmed nonsense mutation DMD and on steroids for at least 12 months. If you would like more information about this please reach out to Ameneh Onativia, Columbia Sr. Clinical Research Coordinator II at 212-342-3679
This study is closed
Do You Qualify?
| Has the child been diagnosed with nonsense mutation DMD confirmed by medical history and genotyping? | Yes | No |
| Does the child have the ability to stand, walk, climb, and descend 4 stairs? | Yes | No |
| Has the child taken corticosteroid treatment for at least 12 months? | Yes | No |
| Has the child had exposure to another investigational drug within 6 months prior to start of study treatment? | Yes | No |