Vamorolone can substitute Prednisolone for treating Duchenne Muscular Dystrophy: JAMA.

Vamorolone was found to be a safe, effective alternative to Prednisolone in treating Duchenne Muscular Dystrophy in boys as per a recent trial that was published in the journal 'JAMA Network.' 

Corticosteroids are the frequently prescribed drugs for managing Duchenne Muscular Dystrophy (DMD), but long-term use has some adverse effects that affect the quality of life. Hence Researchers conducted a trial to find if vamorolone, a structurally unique dissociative steroidal anti-inflammatory drug, can retain efficacy while reducing safety concerns with use in Duchenne muscular dystrophy (DMD). 

A randomized, multicenter, double-blind, placebo- and a prednisone-controlled 24-week clinical trial was conducted from June 29, 2018, to February 24, 2021, with 24 weeks of follow-up. Boys aged 4 to 7 years with genetically confirmed DMD not previously treated with corticosteroids were taken into the trial. The study was carried out at 33 referral centers in 11 countries and included 4 groups placebo; prednisone, 0.75 mg/kg per day; vamorolone, 2 mg/kg per day; and vamorolone, 6 mg/kg per day. The outcome was to monitor efficacy and safety. Efficacy included the following:  

Primary:

Time to stand from supine velocity in the vamorolone, 6 mg/kg per day, group vs placebo. 

Secondary:

Time to stand from supine velocity - vamorolone, 2 mg/kg per day,

The 6-minute walk distance, time to run/walk 10 m - vamorolone, 2 and 6 mg/kg per day

Exploratory:

NorthStar Ambulatory Assessment and time to climb 4 stairs.

Safety was assessed by measuring growth, bone biomarkers, and corticotropin (ACTH)–challenge test. 

Results: 

• 133 boys with DMD enrolled in the study with a mean age of 5.4 years.
• Of these 121 were randomly assigned to treatment groups, and 114 completed the 24-week treatment period.
• The trial met the primary endpoint point for change from baseline to week 24 and the first 4 sequential secondary endpoints. 
• Height percentile declined in prednisone-treated participants.
• Bone turnover markers declined with prednisone but not with vamorolone.
• Boys with DMD at baseline showed low ACTH-stimulated cortisol and a high incidence of adrenal insufficiency.
• All 3 treatment groups led to increased adrenal insufficiency. 

Thus, it was concluded from the trial that vamorolone was shown to be effective and safe in the treatment of boys with DMD over a 24-week treatment period. The researchers also concluded that Vamorolone could be a safer alternative to Prednisolone. 

To read the full article, click here: 10.1001/jamaneurol.2022.2480 

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 [published online ahead of print, 2022 Aug 29]. JAMA Neurol. 2022;10.1001/jamaneurol.2022.2480.