NCT01247207

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 270 (estimated)
• Sponsor: PTC Therapeutics · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

Summary

The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Ataluren	Ataluren will be administered as per the dose and schedule specified in the arm.

Timeline

• Start date: 2010-11-30
• Primary completion: 2026-02-10
• Completion: 2026-02-10
• First posted: 2010-11-24
• Last updated: 2026-03-09

Locations

34 sites across 2 countries: United States, Canada