NMD4C: The Neuromuscular Disease Network for Canada

In November of 2021, Duchenne muscular dystrophy (DMD) patient partner organization Defeat Duchenne Canada (formerly Jesse’s Journey) announced they would be awarding a grant of $1 million CAD in support of a clinical trial of the drug vamorolone in Canada, hosted exclusively at Canadian sites. This pioneering philanthropic effort will bring an integral clinical trial to Canada through the contributions of patient partner organizations in partnership with industry sponsors. 

“Defeat Duchenne Canada has granted $1 million dollars to run the vamorolone VBP-006 clinical trial in Canada. Our mandate as a Duchenne patient organization is to speed up the process of drug discovery and access to beneficial medications addressing all stages of the disease. The VBP15-006 clinical trial addresses the unmet need for inclusion for an expanded age range. We look forward with hope that this potential steroid alternative can increase the quality of life for individuals living with Duchenne.”

– Nicola Worsfold, National VP Research, Education & Advocacy, Defeat Duchenne Canada

The VBP-006 clinical trial, a study to assess vamorolone in boys ages 2 to <4 years and 7 to <18 years with DMD will showcase Canada’s rich clinical trial landscape and its capacity to host trials exclusively at sites within Canada and will help attract further cutting-edge neuromuscular clinical trials in the future. The Canadian phase II study will be chaired by clinician Dr. Jean Mah of the Alberta Children’s Hospital Research Institute, University of Calgary, and will operate across six Canadian sites, enrolling 44 patients in total. Collaborations with Dr. Leanne Ward, Professor of Paediatrics and Research Chair in Paediatric Bone Health at the University of Ottawa on bone health outcomes will continue to provide an important contribution to the vamorolone program. Further information on this study will be available in the coming months, so stay tuned to Defeat Duchenne Canada for the latest updates.

On the heels of the completion of the global Vision-DMD vamorolone trial, contributions from Defeat Duchenne Canada, Santhera Pharmaceuticals and ReveraGen BioPharma, Inc. (the companies which funded the VISION-DMD study) have made the Canadian VBP-006 clinical trial possible. 

“It’s fantastic that this funding from Jesse’s Journey is giving Canadians with DMD who weren’t able to take part in the previous trial the opportunity to be involved in a further study of this promising steroid alternative. The results of this Canada-led initiative will increase knowledge about the efficacy of the drug in an expanded age range and will further cement Canada’s expertise in hosting cutting-edge trials in neuromuscular disease.”

– Dr. Hanns Lochmüller, Lead Investigator, NMD4C

The NMD4C works to provide clinical trial resources to all stakeholders, in an effort to strengthen the clinical trial landscape in Canada. Through the launch of the NMD4C clinical trial concierge, an information broker with consistent knowledge, awareness, and access to information across sites, the clinical trial working group works to unite stakeholders in the Canadian neuromuscular clinical trial landscape including clinicians and trial sites, partner organizations including Muscular Dystrophy Canada and Jesse’s Journey, industry and people affected by NMDs.