Who is Eligible for the Trial?

To qualify for enrollment in the trial, your son must attend a screening visit at a participating trial location to evaluate if he is a good fit for this trial. Listed below are some of the key eligibility requirements. The participating site will provide you with more information on the additional criteria that must be met as part of your participation in the trial.

Eligibility Criteria

  • Age 6 to 15 year-old ambulatory (able to walk) boys, diagnosed with Duchenne muscular dystrophy as confirmed by medical history and genetic testing (mutation analysis)
  • Subject is receiving glucocorticoid steroids for at least 6 months prior to screening visit and is on a stable dose for  3 months prior to screening visit.
  • Subject is able to perform the 4 stair climb with or without the use of handrails.
  • Subject will be able to tolerate a closed MRI and able to stay still inside the MRI machine for up to one hour, each time. The machine makes loud noises. Although headphones may be provided, no medications will be given to help him relax and stay still.
  • Subjects and their caregiver are willing and able to comply with the trial schedule and treatment procedures.
  • Subjects will not be able to participate if they have had current or prior treatment with anti-myostatin, exon skipping nonsense mutation-targeted therapies ever or more than 30 days of treatment with utrophin modifiers and treatment with utrophin modifiers within 30 days prior to signing the informed consent.
  • Subjects will not be able to participate if they have been enrolled in other trials with investigational drugs (including idebenone) in the past 30 days (or longer depending on the investigational drug previously received).