Overview

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

Inclusion Criteria:

• 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
• Age 18 years of age or older at the time of consent
• Have adequate organ function confirmed by the following laboratory values obtained within 14 days prior to enrollment (28 days for cardiac and pulmonary function):
• Participants with partners of childbearing potential must be willing to use at least two forms of effective birth control (one form must be a barrier method) while receiving the study product and for 3 months after stopping tacrolimus therapy.
• Ability to follow commands sufficiently to perform voluntary aspects of outcome measures throughout the study period
• Willing to consent to monitoring for 15 years, including an extension period, as required for all interventional studies involving the transplantation of cells that have been genetically modified
• Voluntary written consent from the subject or parent(s)/guardian(s) and assent from participant prior to the performance of any research related activity.

Exclusion Criteria:

• Presence of HLA antibodies directed toward HLA antigens on MyoPAXon
• Active treatment with another investigational therapy
• Known allergy to MyoPAXon components