Sarepta Therapeutics is now recruiting patients at two Canadian centres for their ESSENCE clinical trial.
ESSENCE Trial
The ESSENCE trial is a 96-week, double-blind, placebo-controlled research study to evaluate the effectiveness and safety of investigational products, SRP-4045 and SRP-4053, in patients with Duchenne muscular dystrophy.[1]
Canadian Locations
The ESSENCE trial is currently recruiting patients at two Canadian locations:[2]
London, Ontario
London Health Sciences Centre
London, Ontario, Canada, N6A 5W9
Contact: Gina Bhullar (519) 685-8500 ext 55058 gina.bhullar@lhsc.on.ca
Principal Investigator: Craig Campbell, MD
Calgary, Alberta
Alberta Childrens Hospital
Calgary, Alberta, Canada, T3B 6A8
Contact: Tiffany Haig (403) 955-3192 tiffany.haig@albertahealthservices.ca
Principal Investigator: Jean Mah, MD
Who is Eligible?
Inclusion Criteria:
- Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping
- Stable dose of oral corticosteroids for at least 24 weeks
- Intact right and left biceps or 2 alternative upper muscle groups
- Mean 6MWT greater than or equal 300 meters and less than or equal to 450 meters
- Stable pulmonary and cardiac function: forced vital capacity (FVC) equal to or greater than 50% predicted and left ventricular ejection fraction (LVEF) greater than 50%
Exclusion Criteria:
- Previous treatment with SMT C1100 (BMN-195) at any time
- Previous treatment with PRO045 or PRO053 within 6 months prior to Week 1
- Current or previous treatment with any other experimental treatment (other than deflazacort) within 12 weeks prior to Week 1
- Participation in any other DMD interventional clinical study within 12 weeks prior to Week 1
- Major surgery within 3 months prior to Week 1
- Presence of other clinically significant illness
- Major change in physical therapy regimen within 3 months prior to Week 1
Exon Skipping
Duchenne is caused by mutations to the dystrophin gene. Most commonly, one or more exons (a portion of the gene) are missing, and the remaining exons don’t fit together properly. Because of this error, cells cannot make dystrophin, a protein muscles need to work properly. Without dystrophin, muscle cells are damaged, and, over time, are replaced with scar tissue and fat.
The investigational therapies in the ESSENCE study use a technique referred to as exon skipping. Skipping a specific exon next to the mutation is intended to allow the body to make a shortened form of the dystrophin protein.
To learn more about which exons are amenable to 45 or 53 skipping, visit the Cure Duchenne website.
To learn more about the ESSENCE trial visit https://essencetrial.com/ or clinicaltrials.gov