NCT01826487

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

A Phase 3 Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Dystrophinopathy

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 230 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: Male
• Age: 7 Years – 16 Years
• Healthy volunteers: Not accepted

Summary

Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which 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. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of dystrophinopathy in approximately 10-15 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. The main goal of this Phase 3 study is to evaluate the effect of ataluren on walking ability. The effect of ataluren on physical function, quality of life, and activities of daily living will be evaluated. This study will also provide additional information on the long-term safety of ataluren.

Detailed description

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to determine the efficacy and safety of ataluren in participants with nmDMD.

Conditions

• Muscular Dystrophy, Duchenne
• Muscular Dystrophies
• Muscular Disorders, Atrophic
• Muscular Diseases
• Musculoskeletal Diseases
• Neuromuscular Diseases
• Nervous System Diseases
• Genetic Diseases, X-Linked
• Genetic Diseases, Inborn

Interventions

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

Timeline

• Start date: 2013-03-26
• Primary completion: 2015-08-20
• Completion: 2015-08-20
• First posted: 2013-04-08
• Last updated: 2020-08-04
• Results posted: 2020-08-04

Locations

55 sites across 18 countries: United States, Australia, Belgium, Brazil, Canada, Chile, Czechia, France, Germany, Israel, Italy, Poland, South Korea, Spain, Sweden, Switzerland, Turkey (Türkiye), United Kingdom