A study to assess the efficacy and safety of a drug named Vamorolone in boys with Duchenne muscular dystrophy (DMD)
Goal of this study
To treat Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a genetic disease due to the absence of a protein called dystrophin. DMD is characterized by progressive muscle degeneration and weakness, and is due to deterioration of muscle cells which are important for body movement. This study will investigate the efficacy and safety of a drug named Vamorolone in comparison to Prednisone in patients with DMD. Vamorolone will be given to the patient through the mouth once daily every day for a period of 52 weeks.
Who can participate in this study?
Patients who have been diagnosed with Duchenne muscular dystrophy and who are between 4 and 7 years of age may be eligible to participate in this study. This study accepts boys and does not accept healthy volunteers.
Important: Additional eligibility criteria apply and the patient’s record will be verified by the study doctor (with the patient’s approval) to confirm eligibility.
ClinicalTrials.gov Identifier: NCT03439670
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