This week, Defeat Duchenne Canada is delighted to announce that the trial has reached the key milestone (trial midpoint) of opening higher dose treatment groups to new enrollment. Previously, the study has recruited into a lower dose group of vamorolone (2.0 mg/kg/day), and this week the trial opened recruitment into the higher dose group (6.0 mg/kg/day).

The VBP15-006 clinical trial is enrolling children with DMD ages 2 to 4 years and 7 to 18 years. The older boys can be either ambulatory or nonambulatory. The clinical trial initiated enrollment earlier this year and is a Canada-only trial, with clinical sites in Calgary, Vancouver, Toronto, Montreal, and Ottawa. The trial will enroll 20 boys in the 2 to 4-year age range and 24 boys in the 7 to 18-year age range.

“This is the first time we have funded a Canada-only clinical trial with an expanded age range for individuals currently taking steroids or naive to steroid treatment. We hope this investigational drug will help slow down inflammation and muscle damage with fewer side effects than current steroid treatments.”

Nicola Worsfold, National Vice President of Research, Education and Advocacy at Defeat Duchenne Canada

The clinical trial involves visits to one of the clinical trial sites over three months, and all participants receive vamorolone, not a placebo, during the trial. Upon completion of the trial, the patient and family are provided long-term access to vamorolone should they desire it. The trial covers all costs associated with travel and stay for the medical center visits for the patient and guardian.

For more information, please contact Bonnie Wooten, Clinical Trial Concierge for the Neuromuscular Disease Network for Canada (NMD4C): [email protected].