Trial of Cell Based Therapy for DMD

Part of paid clinical trials in Minneapolis, Minnesota.

Sponsor
Masonic Cancer Center, University of Minnesota
Study ID
NCT06692426
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • MyoPAXon — DRUG
    MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2
  • Tacrolimus — DRUG
    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.

Study Details

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)

Key Dates

Start date
Mar 20, 2025
Status verified
Mar 2026
Primary completion
Mar 3, 2027
Completion
Mar 3, 2027

Study Design

Enrollment
8 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Arm A: MyoPAXon 25 x 10^6
    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
  • Experimental: Arm B: MyoPAXon 50 x 10^6
    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
  • Experimental: Arm C: MyoPAXon 100 x 10^6
    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
  • Experimental: Arm D: MyoPAXon 200 x 10^6
    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

Primary Outcome Measure

Maximal tolerable dose (MTD) of MyoPAXon [ Time Frame: 3 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Masonic Cancer CenterMinneapolisMinnesota55455
Peter Kang, MD

Find similar trials in Minneapolis, MN

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Masonic Cancer Center· Minneapolis, MN

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