Trial of Cell Based Therapy for DMD

ConditionDuchenne Muscular Dystrophy

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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

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.

Treatment groups

8 Participants

are divided into 4 treatment groups

Group A: Arm A: MyoPAXon 25 x 10^6Experimental treatment 2 interventions

Group B: Arm B: MyoPAXon 50 x 10^6Experimental treatment 2 interventions

Group C: Arm C: MyoPAXon 100 x 10^6Experimental treatment 2 interventions

Group D: Arm D: MyoPAXon 200 x 10^6Experimental treatment 2 interventions

Trial outcomes

Primary outcomes

Maximal tolerable dose (MTD) of MyoPAXon

Time frame

3 months

Secondary outcomes

Proportion of patients developing humoral and cellular responses

Proportion of patients developing humoral (donor-specific anti-HLA antibodies) and cellular responses within 3 months

Time frame

3 months

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Masonic Cancer Center, University of Minnesota

Lead sponsor

Duchenne UK

Collaborator

Parent Project Muscular Dystrophy