An Open-Label safety study for previously treated ataluren (PTC124) patients with nonsense mutation dystrphinopathy
Study ID: STU 112010-072
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.
Subjects must meet all of the following conditions to be eligible for enrollment into the study: 1. Evidence of signed and dated informed consent/assent document(s) indicating that the subject (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 subject should be followed. 2. History of exposure to ataluren in a prior PTC study or treatment plan in nmDBMDtrial site.. 3. Male sex. 4. In subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and the 6-week follow-up period. 5. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions. Note: Psychological, social, familial, or geographical factors that might preclude adequate study participation should be considered.