An Open-Label safety study for previously treated ataluren (PTC124) patients with nonsense mutation dystrphinopathy

Study ID
STU 112010-072

Cancer Related

Healthy Volunteers

Study Sites

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

Kara Lorduy

Principal Investigator
Susan Iannaccone, M.D.


comprises a Phase iii, open-label, safety study of ataluren in patients who have previously been exposed to ataluren. up to 132 patients will be enrolled - 3 at CMC.

Subjects will receive ataluren 3 times per day at a dose of 10, 10 20 mg/kg for approximately 36 weeks.Study assessments will be performed at clinic visits during screening, on the first day of ataluren dosing, thenevery 12 weeks during the ataluren treatment period.

after the treatment period, subjects will undergo long-term follow-up at 6 to 12 month intervals for 5 years, to assess for any new chronic health problems.

Participant Eligibility

1. evidence of a signed/dated informed consent
2. history of exposure to ataluren in a prior PTC study
3. males
4.confirmed screening lab values within specified ranges
5. willingness to abstain from sexual intercourse or employ a barrier during ataluren administration and 6 week follow-up phase
6. comply with scheduled visits, drug administration plan, study procedures, lab tests, and study restrictions