Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Nervous System Diseases Clinical Trial

Official title: 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

Status Completed

Clinical Trial Summary

This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

Clinical Trial Description

This study is a randomized, double-blind, placebo-controlled, 72-week study, followed by a 72-week open-label period. The purpose is to characterize the long-term effects of ataluren-mediated dystrophin restoration on disease progression. Participants will be randomized in a 1:1 ratio to ataluren or placebo. Participants will receive blinded study drug three times daily (TID) at morning, midday, and evening for 72 weeks, after which all participants will receive open-label ataluren for an additional 72 weeks (144 weeks in total). Study assessments will be performed at clinic visits every 12 weeks during the double-blind period and every 24 weeks during the open-label period. The total sample size of ~250 subjects will include ~160 subjects who meet the criteria for inclusion in the primary analysis population (age 7 to 16 years old, baseline six minute walk distance (6MWD) greater than or equal to (>=) 300 meters, supine to stand >= 5 seconds). The study will be conducted in the United States and other countries around the world. ;

Related Conditions & MeSH terms

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

• NCT number: NCT03179631
• Study type: Interventional
• Source: PTC Therapeutics
• Status: Completed
• Phase: Phase 3
• Start date: July 6, 2017
• Completion date: July 25, 2023