Clinical Trials Directory

Trials / Completed

CompletedNCT03179631

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

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
Phase
Phase 3
Study type
Interventional
Enrollment
360 (actual)
Sponsor
PTC Therapeutics · Industry
Sex
Male
Age
5 Years
Healthy volunteers
Not accepted

Summary

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

Detailed 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.

Conditions

Interventions

TypeNameDescription
DRUGAtaluren10, 20 mg/kg
DRUGPLACEBO10, 20 mg/kg

Timeline

Start date
2017-07-06
Primary completion
2022-03-05
Completion
2023-07-25
First posted
2017-06-07
Last updated
2026-03-10
Results posted
2026-03-10

Locations

64 sites across 18 countries: United States, Australia, Brazil, Bulgaria, Canada, China, Hong Kong, India, Japan, Malaysia, Mexico, Poland, Puerto Rico, Russia, South Korea, Taiwan, Thailand, Turkey (Türkiye)

Regulatory

Source: ClinicalTrials.gov record NCT03179631. Inclusion in this directory is not an endorsement.