Trial of Cell Based Therapy for DMD
- Registration Number
- NCT06692426
- Lead Sponsor
- Masonic Cancer Center, University of Minnesota
- 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. If you would like further information please contact Jon Guden, Associate Director, Conflict of Interest Program, at jguden@umn.edu.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 8
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Arm A: MyoPAXon 25 x 10^6 Tacrolimus 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 Arm B: MyoPAXon 50 x 10^6 Tacrolimus 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 Arm C: MyoPAXon 100 x 10^6 MyoPAXon 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 Arm C: MyoPAXon 100 x 10^6 Tacrolimus 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 Arm D: MyoPAXon 200 x 10^6 MyoPAXon 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 Arm D: MyoPAXon 200 x 10^6 Tacrolimus 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 Arm A: MyoPAXon 25 x 10^6 MyoPAXon 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 Arm B: MyoPAXon 50 x 10^6 MyoPAXon 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 Measures
Name Time Method Maximal tolerable dose (MTD) of MyoPAXon 3 months Maximal tolerable dose (MTD) of MyoPAXon
- Secondary Outcome Measures
Name Time Method 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
Trial Locations
- Locations (1)
Masonic Cancer Center
🇺🇸Minneapolis, Minnesota, United States