Sarepta Seeking Participants for Control Arms

 

Sarepta is seeking participants for the control arms of two clinical trials involving EXONDYS 51:

1.  STUDY 4658-301 (PROMOVI) is an open-label, natural history controlled study to provide safety, biomarker, efficacy, and long term data on eteplirsen (EXONDYS 51) in Duchenne muscular dystrophy. The untreated control arm (natural history arm, i.e. no eteplirsen) will provide control data on clinical function and safety.

  • MAIN INCLUSION CRITERIA:
    • Boys with Duchenne between 7 to 16 years of age
    • Confirmed dystrophin Glossary Link gene deletion NOT amenable to exon 51 skipping
    • Able to walk at least 300m (about the length of three football fields)
    • Ongoing stable corticosteroids treatment

2.  STUDY 4658-203 is an open-label natural history controlled study to provide safety and efficacy data on eteplirsen (EXONDYS 51) in early stage Duchenne muscular dystrophy. The natural history arm (untreated control) will provide control data on clinical function, MRI imaging and safety.

  • MAIN INCLUSION CRITERIA:
    • Boys with Duchenne between their 4th and 7th birthday
    • Confirmed dystrophin gene deletion NOT amenable to exon 51 skipping

Please click on the above study links for ClinicalTrials.gov to learn more.  If you are interested in participating, please contact the study coordinator at your preferred study site.

Posted 12/2/2016