Home › Duchenne Muscular Dystrophy › NCT06692426
Trial of Cell Based Therapy for DMD
RecruitingPhase 1
Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy
Who can join
Ages 18 and older · All sexes
Full eligibility criteria
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
About the study
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)
What is being tested
- MyoPAXon (drug)
- Tacrolimus (drug)
Sponsor: Masonic Cancer Center, University of Minnesota · Participants: 8 · Started: Mar 20, 2025
Contact the study team
- Peter Kang, MD · Phone: 612-624-9452
Official record on ClinicalTrials.gov — NCT06692426
Locations in the U.S.
| Minnesota | Masonic Cancer Center, Minneapolis |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.