Earlier this week the results of an early trial of vamorolone in boys with Duchenne have been published in the Neurology scientific journal. Vamorolone is a potential treatment that researchers hope might offer the benefits of steroids with reduced side effects. It was developed with funding from many charities and funding bodies including Action Duchenne.
The phase 2a trial ended last year. It took place in the US, Canada, Sweden, Israel, UK and Australia and included 48 boys living with Duchenne.
The trial was an open label study and aimed to identify doses of vamorolone that might be effective in Duchenne. Two doses of vamorolone showed some evidence of effectiveness. Participants who received the highest doses achieved higher Northstar scores and were able to stand up from the floor faster. The ongoing VISION-DMD trial is using these doses.
The study demonstrated that there were no serious safety concerns with the potential drug, but some minor side effects were noted at higher doses. Some boys experienced an increase in BMI and adrenal insufficiency (side effects often associated with cortico-steroids).
Publication of trial results in a peer reviewed journal is a key part of the clinical trial process. It means the results have been scrutinised by independent researchers, and will now be widely available.
If you have any questions you can email Neil or call us on 02072508240.
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