Plain language summary of vamorolone for boys with Duchenne muscular dystrophy age 7 to less than 18 years

Plain Language SummaryWhat is this summary about?This is a plain language summary of clinical research in boys aged between 7 and less than 18 years old with Duchenne muscular dystrophy (DMD) in Canada. The boys were treated with vamorolone (Agamree®) for 12 weeks in the clinical study VBP15-006 and then continued to receive treatment in the Expanded Access Protocol study (EAP). Vamorolone is a novel (meaning that it is different to other existing treatments) corticosteroid medicine – a type of man-made treatment based on cortisol, a natural chemical produced by the adrenal gland in the body. This part of the VBP15-006 study aimed to better understand vamorolone safety and what effects it has in boys aged 7 to less than 18 years old with DMD, including those who change treatment or ‘switch’ to vamorolone from other corticosteroid treatments such as deflazacort and prednisone.What were the main findings?No new safety issues were found, and the safety findings were like what was previously known for vamorolone.Vamorolone did not cause growth stunting, which is seen with corticosteroids that have been traditionally used to treat DMD that are referred to as the ‘classic corticosteroids’. In addition, the reduced height in boys caused by prior corticosteroid use improved after switching to vamorolone.Some weight gain occurred, especially in boys who had never received corticosteroids and who were treated with the higher (6 mg/kg/day) dose. Weight gain did not affect all boys and varied between them.The higher dose of vamorolone (6 mg/kg/day) appeared to be important when switching from other corticosteroids to help lower the risk of adrenal insufficiency, a serious and potentially life-threatening condition where the body cannot make enough stress hormones.What are the key takeaways?Vamorolone did not cause any new safety issues in boys aged 7 to less than 18 years old versus what was already known in younger boys.Boys who were shorter due to other corticosteroid treatments caught up in growth when switching to vamorolone.It is important to use vamorolone 6 mg/kg/day when switching from other corticosteroids to reduce the risk of adrenal insufficiency.How to say (download PDF and double click sound icon to play sound)…Adrenal: ah-DREE-nalCorticosteroid: kor-tuh-kow-steh-roydVamorolone: va-MOR-oh-loneCortisol: kor-ti-solThis is an abstract of the Plain Language Summary of Publication article.View the full Plain Language Summary PDF of this article to read the full-textLink to original article hereTrial registration: ClinicalTrials.gov identifier: NCT05185622 and NCT03863119.