About this trial
This is a single-center, single-arm, interventional phase 1 trial to evaluate the safety and tolerability of local injection of induced pluripotent stem cell (iPSC)- derived CD54+ allogeneic muscle progenitor cells in individuals with Duchenne muscular dystrophy (DMD)
Eligibility criteria
Qualifiers
Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and/or absence of immunohistochemical staining for dystrophin on muscle biopsy
Non-ambulatory
Intact extensor digitorum brevis (EDB) muscles bilaterally
Off investigational therapies for > 30 days
Disqualifiers
Presence of HLA antibodies directed toward HLA antigens on MyoPAXon
Active treatment with another investigational therapy
Known allergy to MyoPAXon components
Trial design
Treatments tested in this trial
- MyoPAXon
- Tacrolimus
Treatment groups
Sponsors and collaborators
Masonic Cancer Center, University of Minnesota
Lead sponsor
Duchenne UK
Collaborator
Parent Project Muscular Dystrophy
Collaborator