Cell-Based Therapy for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: Masonic Cancer Center, University of Minnesota

No Placebo Group

Trial Summary

What is the purpose of 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)

Research Team

Eligibility Criteria

This trial is for adults over 18 with Duchenne muscular dystrophy, confirmed by gene mutations or muscle biopsy. Participants must be non-ambulatory but have intact EDB muscles and off other investigational therapies for over 30 days. They should commit to birth control methods if applicable and consent to a long-term follow-up of 15 years.

Inclusion Criteria

Off investigational therapies for more than 30 days

Voluntary written consent from the subject or parent(s)/guardian(s) and assent from participant prior to the performance of any research related activity.

I cannot walk by myself.

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Exclusion Criteria

Active treatment with another investigational therapy

Known allergy to MyoPAXon components

My body has antibodies against specific antigens on MyoPAXon.

Treatment Details

Interventions

MyoPAXon (Cell-based Therapy)

Trial OverviewThe trial tests the safety of injecting stem cell-derived muscle progenitor cells into patients with DMD. It's an early-stage study (phase 1) focusing on how well participants tolerate this new cell-based therapy called MyoPAXon, alongside Tacrolimus to prevent rejection.

Participant Groups

4Treatment groups

Experimental Treatment

Group I: Arm D: MyoPAXon 200 x 10^6Experimental Treatment2 Interventions

MyoPAXon will be delivered via open intramuscular (IM) injection into a single EDB muscle for each participant as a one-time dose. The participants will be started on tacrolimus 1 week prior to dosing and stay on the maintenance dose on a BID schedule for 3 months after injection

Group II: Arm C: MyoPAXon 100 x 10^6Experimental Treatment2 Interventions

Group III: Arm B: MyoPAXon 50 x 10^6Experimental Treatment2 Interventions

Group IV: Arm A: MyoPAXon 25 x 10^6Experimental Treatment2 Interventions