A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

Study ID
STU 072014-049

Cancer Related

Healthy Volunteers

Study Sites

  • Children’s Medical Center (Dallas, Plano, Southlake)

Kara Lorduy

Principal Investigator
Susan Iannaccone, M.D.


This study is an extension of the PTC124-GD-020-DMD study. This study is to better understand whether ataluren can be safely given for a long period of time. The study will also provide additional information as to whether the actions of ataluren can result in improved walking, activity, muscle function, muscle fragility, upper limb function, pulmonary function, health-related quality of life (via PoDCi) in boys with DMD. The study is for boys who have completed the PTC124-GD-020-DMD study.

Participant Eligibility

1. Completion of study treatment in the previous Phase 3, double-blind study protocol (Protocol
2. Evidence of signed and dated informed consent/assent document(s) indicating that the patient
(and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial.
Note: If the study candidate is considered a child under local regulation, a parent or legal
guardian must provide written consent prior to initiation of study screening procedures
and the study candidate may be required to provide written assent. The rules of the
responsible Institutional Review Board/Independent Ethic Committee (IRB/IEC)
regarding whether one or both parents must provide consent and the appropriate ages for
obtaining consent and assent from the patient should be followed.
3. In patients who are sexually active, willingness to abstain from sexual intercourse or employ
a barrier or medical method of contraception during the study drug administration and
6-week follow-up period.
4. Willingness and ability to comply with scheduled visits, ataluren administration plan, study
procedures, laboratory tests, and study restrictions.