The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
270
Ataluren will be administered as per the dose and schedule specified in the arm.
Phoenix Childrens Hospital
Phoenix, Arizona, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
UCLA
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
University of California-Davis
Sacramento, California, United States
Number of Participants With Adverse Events
Time frame: Baseline up to end of study (up to approximately 8 years)
Number of Participants With Laboratory Parameters Abnormalities
Laboratory parameters include hematological, serum biochemistry, adrenal laboratories, and urine data.
Time frame: Baseline up to end of study (up to approximately 8 years)
Number of Participants With Abnormal Physical Findings
Physical findings include weight, physical examination data, systolic and diastolic blood pressure, radial pulse rate, and body temperature.
Time frame: Baseline up to end of study (up to approximately 8 years)
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University of CA, San Francisco-Benioff Children's Hospital
San Francisco, California, United States
Children's Hospital Colorado - Center for Cancer and Blood Disorders
Aurora, Colorado, United States
Child Neurology Center of NW Florida NW
Gulf Breeze, Florida, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
Rush Univ Medical Center
Chicago, Illinois, United States
...and 24 more locations