Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2018-02-09
• Target enrollment: 0
• Participant gender: Male

Summary

This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: PTC Therapeutics

Criteria

Inclusion Criteria:

• Males ≥2 to <5 years of age

• Body weight ≥12 kg

• Diagnosis of DMD

• Nonsense mutation in at least 1 allele of the dystrophin gene

Exclusion Criteria:

• Participation in any other drug or device clinical investigation

• Ongoing use of prohibited concomitant medications