Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy
Trial Snapshot
- Phase
- Phase 1
- Status
- Recruiting
- Enrollment
- 8
- Locations
- 1
- Primary Endpoint
- Maximal tolerable dose (MTD) of MyoPAXon
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts 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
Arms & Interventions
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
Intervention: MyoPAXon (Drug)
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
Intervention: Tacrolimus (Drug)
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
Intervention: MyoPAXon (Drug)
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
Intervention: Tacrolimus (Drug)
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
Intervention: MyoPAXon (Drug)
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
Intervention: Tacrolimus (Drug)
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
Intervention: MyoPAXon (Drug)
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
Intervention: Tacrolimus (Drug)
Outcomes
Primary Outcomes
Maximal tolerable dose (MTD) of MyoPAXon
Time Frame: 3 months
Maximal tolerable dose (MTD) of MyoPAXon
Secondary Outcomes
- Proportion of patients developing humoral and cellular responses(3 months)