Trial of Cell Based Therapy for DMD

Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy

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)

The University of Minnesota holds equity in, and has rights to potential royalties from, Myogenica, the company that has licensed this trial's stem cell therapy from the University. These interests have been reviewed and managed by the University of Minnesota in accordance with its conflict of interest policies.

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