MedTrace Logo

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 360

Last updated 2026-03-10

Study results available
· View outcomes & findings →

Summary

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

Conditions

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

Interventions

DRUG

Ataluren

10, 20 mg/kg

DRUG

PLACEBO

10, 20 mg/kg

Sponsors & Collaborators

Principal Investigators

  • Vinay Penematsa, MD · PTC Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
5 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-07-06
Primary Completion
2022-03-05
Completion
2023-07-25
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Brazil
  • Bulgaria
  • Canada
  • China
  • Hong Kong
  • India
  • Japan
  • Malaysia
  • Mexico
  • Poland
  • Puerto Rico
  • Russia
  • South Korea
  • Taiwan
  • Thailand
  • Turkey (Türkiye)

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03179631 on ClinicalTrials.gov