NCT06692426

Trial of Cell Based Therapy for DMD

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 8 (estimated)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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)

Detailed description

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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	MyoPAXon	MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2
DRUG	Tacrolimus	Tacrolimus (Prograf) is an immunosuppressant that inhibits calcineurin and T cell activation, and is commonly used to prevent solid organ transplant rejection1 and graft versus host disease (GVHD) as well as allograft rejection in the setting of allogeneic hematopoietic stem cell transplantation.

Timeline

• Start date: 2025-03-20
• Primary completion: 2027-03-03
• Completion: 2027-03-03
• First posted: 2024-11-18
• Last updated: 2026-03-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study