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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

Sponsor: Masonic Cancer Center, University of Minnesota · Participants: 8 · Started: Mar 20, 2025

Contact the study team

Official record on ClinicalTrials.gov — NCT06692426

Locations in the U.S.

MinnesotaMasonic 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.