Trial of Cell Based Therapy for DMD

Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorMasonic Cancer Center, University of Minnesota

About this trial

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 criteria

Qualifiers

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

Disqualifiers

Presence of HLA antibodies directed toward HLA antigens on MyoPAXon

Active treatment with another investigational therapy

Known allergy to MyoPAXon components

Trial design

Treatments tested in this trial

  • MyoPAXon
  • Tacrolimus

Treatment groups

8 Participants
are divided into 4 treatment groups

Sponsors and collaborators

Masonic Cancer Center, University of Minnesota

Lead sponsor

Duchenne UK

Collaborator

Parent Project Muscular Dystrophy

Collaborator