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Duchenne Muscular Dystrophy clinical trials at UCSD
3 in progress, 1 open to new patients

  • Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

    open to eligible males ages 6-17

    it is a randomised, double blind, parallel group, placebo controlled study. A total of 213 male ambulant subjects will be randomised 2:1 (givinostat:placebo). Subjects will be stratified for their concomitant use of steroids in 4 strata: 1. Deflazacort daily regimen 2. Deflazacort intermittent regimen 3. Other steroids daily regimen 4. Other steroids intermittent regimen. The study duration is planned for 19 months.

    San Diego, California and other locations

  • Study of Eteplirsen in DMD Patients

    Sorry, in progress, not accepting new patients

    The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).

    San Diego, California and other locations

  • Study of SRP-4045 and SRP-4053 in DMD Patients

    Sorry, not currently recruiting here

    The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

    San Diego, California and other locations

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