Tests a cell therapy injection in adults with Duchenne muscular dystrophy
Tests cell injections into muscle for safety in adults with Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: Trial of Cell Based Therapy for DMD
Key facts
- Where
- Minneapolis, United StatesSee the site
- Phase
- Phase 1
Where in United States
- Masonic Cancer CenterMinneapolis, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Masonic Cancer Center, University of Minnesota
- Trial code
- NCT06692426
- Record updated
- 4 March 2026
- Original description
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)Read full original descriptionShow less
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)
Inclusion: 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: Presence of HLA antibodies directed toward HLA antigens on MyoPAXon; Active treatment with another investigational therapy; Known allergy to MyoPAXon components.