A new randomized clinical trial demonstrated that vamorolone, an experimental dissociative steroidal anti-inflammatory medication, was safe and effective in boys with Duchenne muscular dystrophy (DMD) over a 24-week period.

The study results, published in JAMA Neurology, suggest that vamorolone may be a safer alternative to long-term prednisone, which is commonly prescribed in DMD.

“First-in-patient, open-label, dose-ranging studies of vamorolone in DMD (n=48) suggested improvements in motor outcomes similar to corticosteroids, without stunting of growth over a 2.5-year treatment period, compared with external corticosteroid-treated comparators,” the authors wrote.


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The pivotal double-blind, placebo- and prednisone-controlled trial assessed the clinical efficacy and safety of vamorolone in boys 4 to 6 years of age with DMD who had not been treated with corticosteroids.

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The trial included 121 boys, 114 of whom completed the study. They were assigned to 1 of 4 treatment groups: placebo, prednisone, vamorolone 2 mg/kg per day, and vamorolone 6 mg/kg per day. The treatments were administered over two 24-week treatment periods.

Safety was assessed in terms of adverse events and pharmacodynamics and efficacy was assessed in terms of motor outcomes. The present study reports the results from the first treatment period.

The results revealed multiple improvements in functional endpoints compared with placebo, including meaningful improvements in time to stand from supine velocity and the 6-minute walk test. Compared with prednisone, which slowed growth trajectories, normal growth trajectories and improved bone turnover markers were observed in boys treated with vamorolone.

However, all treatment groups had a high incidence of adrenal insufficiency at baseline and after treatment, which the authors note will require further study given that the symptoms of adrenal insufficiency and DMD overlap.

Reference

Guglieri M, Clemens PR, Perlman SJ, et al. Efficacy and safety of vamorolone vs placebo and prednisone among boys with Duchenne muscular dystrophy: a randomized clinical trial. JAMA Neurol. Published online August 29, 2022. doi:10.1001/jamaneurol.2022.2480