NCT00847379

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

A Phase 2B Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 173 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: Male
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2b extension trial that will evaluate the long-term safety of ataluren (PTC124) in boys with nonsense mutation DMD/BMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, and other important clinical and laboratory measures.

Detailed description

This is a Phase 2b, international, multicenter, open-label extension study for participants who successfully completed blinded study drug in Study 007. This extension study will evaluate the long-term administration of ataluren administered 3 times per day (TID) at morning, midday, and evening doses of 20, 20, and 40 milligrams/kilogram (mg/kg), respectively, in participants with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD).

Conditions

• Duchenne Muscular Dystrophy
• Becker Muscular Dystrophy

Interventions

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

Timeline

• Start date: 2009-01-31
• Primary completion: 2010-05-24
• Completion: 2010-05-24
• First posted: 2009-02-19
• Last updated: 2020-07-15
• Results posted: 2020-07-15

Locations

37 sites across 11 countries: United States, Australia, Belgium, Canada, France, Germany, Israel, Italy, Spain, Sweden, United Kingdom