Duchenne Muscular Dystrophy Treatment With Ataluren – Phase III

Study Of Ataluren (PTC124) In Patients With Nonsense Mutation Dystrophinopathy

Protocol Description

The main objective of this multicenter study is to evaluate long-term safety and effects of Ataluren (PTC124) in treating muscle function in boys who have a certain type of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene. This study is an extension of a prior study and is only open to participants who completed that study.

Study Level: Phase III

Eligibility Criteria

Subject to certain exclusion criteria, the study is accepting male participants ages 7 to 18, who have a confirmed diagnosis of DMD caused by a nonsense mutation in the dystrophin gene, which causes a disruption in the body’s production of the dystrophin protein in muscle.
Boys: Ages 7 to 18


Participants will take ataluren 3 times every day in an amount appropriate for the individual’s body weight for up to 144 weeks (approximately 3 years). Other physical examinations, including physical assessments as well as biological sample collection, will be done at prescribed intervals. A final visit will be scheduled 6 weeks after the end of the dosing period.
Visits: 14
Duration: About 3 years

Status: Enrolling by Invitation

Source(s) of Support

PTC Therapeutics

Primary Investigator

Hoda Abdel-Hamid, MD, MS

Contact Information

For more information about the study or enrollment, please contact:
Jennifer Monahan, Clinical Research Coordinator