Sarepta ESSENCE Trial Recruiting


ESSENCE is a double-blind placebo-controlled research study to evaluate the effect of two investigational drugs on walking, endurance, and muscle function.


  • Boys between 7 to 13 years of age
  • Duchenne Muscular Dystrophy with a confirmed deletion amenable to exon 45 or 53 skipping
  • Stable dose of corticosteroids for at least 6 months
  • Average 6 minute walk test (6MWT) distance greater than 300 to less than 450 meters (greater than 984 to less than 1476 feet)
  • Stable heart and lung function


  • Major surgery within the past 3 months
  • Presence of other clinically significant illness

Other inclusion/exclusion criteria may apply. Please visit (NCT02500381) or for more information.

All patients will undergo the following assessments:

  • Screening: 3 visits including study related clinical and laboratory evaluations, and tests of breathing, muscle, and heart function
  • Baseline & Week 48: Muscle biopsy
  • Weeks 1-96 (double-blind placebo-controlled period): weekly infusions of investigational study drugs or placebo
  • Approximately every 12 weeks: study related physical examination, blood work, and tests of breathing, muscle, and heart function

Upon completion of the double-blind placebo-controlled period, patients may participate in an open-label treatment extension period of up to 96 weeks and receive weekly study treatment with one of two investigational drugs, according to genotype.

There are multiple study sites across the US and in multiple other countries. If you have questions or if you're interested in participating, please refer to (NCT02500381) or to find a study site located near you. Learn more by listening to the ESSENCE webinar hosted by PPMD on June 7th.

Posted 6/9/2017