Duchenne Muscular Dystrophy
Conditions
Brief 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.
Interventions
DRUGMyoPAXon
MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2
DRUGTacrolimus
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.
Sponsors
Masonic Cancer Center, University of Minnesota
Duchenne UK
Parent Project Muscular Dystrophy
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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 * Age 18 years of age or older at the time of consent * Have adequate organ function confirmed by the following laboratory values obtained within 14 days prior to enrollment (28 days for cardiac and pulmonary function): * Participants with partners of childbearing potential must be willing to use at least two forms of effective birth control (one form must be a barrier method) while receiving the study product and for 3 months after stopping tacrolimus therapy. * Ability to follow commands sufficiently to perform voluntary aspects of outcome measures throughout the study period * Willing to consent to monitoring for 15 years, including an extension period, as required for all interventional studies involving the transplantation of cells that have been genetically modified * Voluntary written consent from the subject or parent(s)/guardian(s) and assent from participant prior to the performance of any research related activity.
Exclusion criteria
* Presence of HLA antibodies directed toward HLA antigens on MyoPAXon * Active treatment with another investigational therapy * Known allergy to MyoPAXon components
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Maximal tolerable dose (MTD) of MyoPAXon | 3 months | Maximal tolerable dose (MTD) of MyoPAXon |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of patients developing humoral and cellular responses | 3 months | Proportion of patients developing humoral (donor-specific anti-HLA antibodies) and cellular responses within 3 months |
Countries
United States
Contacts
CONTACTPeter Kang, MD
Outcome results
None listed