NCT00592553

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 174 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: Male
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

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 13 percent (%) of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2b trial that will evaluate the clinical benefit of ataluren in boys with DMD/BMD due to a nonsense mutation. The main goals of the study are to understand whether ataluren can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time.

Detailed description

This study is a Phase 2b, multicenter, randomized, double-blind, placebo-controlled, dose-ranging, efficacy and safety study, designed to document the clinical benefit of ataluren when administered as therapy of patients with DMD/BMD due to a nonsense mutation (premature stop codon) in the dystrophin gene.

Conditions

• Duchenne Muscular Dystrophy
• Becker Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Ataluren	Ataluren will be administered as per the dose and schedule specified in the respective arms.
DRUG	Placebo	Placebo matching to ataluren will be administered as the schedule specified in the respective arm.

Timeline

• Start date: 2008-02-29
• Primary completion: 2009-12-31
• Completion: 2009-12-31
• First posted: 2008-01-14
• Last updated: 2020-04-07
• Results posted: 2020-04-07

Locations

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