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

Completed · Phase 2

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT02819557

Run by

PTC Therapeutics

People needed

14

Starts

2016-06-09

Expected to finish

2018-02-09

Last updated by the study team

2020-08-28

Who can join

Age: 2 and older, up to 5. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Participation in any other drug or device clinical investigation
• Ongoing use of prohibited concomitant medications

Where it is running

• Child Neuro NWF — Gulf Breeze, Florida, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Children's Hospital Boston — Boston, Massachusetts, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Children's Medical Center Dallas — Dallas, Texas, United States
• University of Utah — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

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