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

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080224563

Enrollment

Registered

2019-02-15

Start date

2018-03-30

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Interventions

investigational material(s) Generic name etc : PTC124 INN of investigational material : ataluren Therapeutic category code : 49- Other agents affecting cellular function Dosage and Administration for

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1.Male sex. 2.Ageaged >=7 to = 300 meters at Baseline Day 1 or Baseline Day 2, and ability to stand from supine >=5 seconds at Baseline Day 1. 7.Ability to perform timed function tests within 30 seconds 8.Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.

Exclusion criteria

Exclusion criteria: 1. Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment.2. Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy. 3. Prior or ongoing therapy with ataluren. 4. Known hypersensitivity to any of the ingredients or excipients of the study drug 5. Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial. 6. History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure during the 72-week placebo-controlled treatment period. 7. Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy. 8. Uncontrolled clinical symptoms and signs of congestive heart failure 9. Elevated serum creatinine or cystatin C at screening.

Design outcomes

Primary

Measure	Time frame
efficacy Slope of Change in 6-Minute Walk Distance (6MWD) Over 72 Weeks.	—

Secondary

Measure	Time frame
safety efficacy 1. Change from baseline to Week 72 in 6MWD 2. Composite of average change in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs at Week 72 3. Change from baseline to Week 72 in time to run/walk 10 meters 4. Change from baseline to Week 72 in time to climb 4 stairs 5. Change from baseline to Week 72 in time to descend 4 stairs 6. Change from baseline to Week 72 in NSAA total score 7. Time to loss of ambulation over 72 weeks 8. Time to loss of stair-climbing over 72 weeks 9. Time to loss of stair-descending over 72 weeks 10. Risk of loss of NSAA items over 72 weeks 11. Ataluren safety profile characterized by type, frequency, severity, and relationship to study drug of any adverse events (AEs), or of abnormalities of laboratory tests, vital signs, physical examinations, or electrocardiograms (ECGs)	—

Measure

Time frame

safety efficacy 1. Change from baseline to Week 72 in 6MWD 2. Composite of average change in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs at Week 72 3. Change from baseline to Week 72 in time to run/walk 10 meters 4. Change from baseline to Week 72 in time to climb 4 stairs 5. Change from baseline to Week 72 in time to descend 4 stairs 6. Change from baseline to Week 72 in NSAA total score 7. Time to loss of ambulation over 72 weeks 8. Time to loss of stair-climbing over 72 weeks 9. Time to loss of stair-descending over 72 weeks 10. Risk of loss of NSAA items over 72 weeks 11. Ataluren safety profile characterized by type, frequency, severity, and relationship to study drug of any adverse events (AEs), or of abnormalities of laboratory tests, vital signs, physical examinations, or electrocardiograms (ECGs)

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Countries

Asia except Japan, Europe, Japan, North America, Oceania, South America

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026