Considerations for Assessing Efficacy in Steroid-Naïve Interventional Trials in Duchenne Muscular Dystrophy (ICNMD 2026) - Jul 6, 2026 - Abstract #PP01.51; Pres time: Jul 8, 2026; 10:30 AM - 11:15 AM; Location: Spadolini - Ground Floor; "Define subgroups that could increase statistical power compared to an all-comers analysis. We used data from a randomized, double blinded, placebo-controlled registrational trial of vamorolone (VBP15-004), a dissociative steroid approved for DMD in boys over 2 years old, as well as data from the placebo arm of another published interventional study... While some steroid-naïve boys with DMD may show a marginal improvement in some motor outcomes in early age bins, none of the motor outcomes showed consistent strength improvement until <7.5 yrs in this typical age range of interest for interventional trials. The ≥ 5-second TTSTAND and 300m-400m subgroups were found to provide greater statistical power for a corticosteroid effect than an all-comers analysis. These findings may be important both for steroid-naïve interventional trial designs and steroid add-on trials."
Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy
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Ms. Kaitey Guite; Dr. Eric P. Hoffman; Dr. Michela Guglieri; Dr. Paula R. Clemens; Dr. Utkarsh J. Dang
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