Cell-based therapy for Duchenne Muscular Dystrophy
Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy
PHASE1 · Masonic Cancer Center, University of Minnesota · NCT06692426
This study is testing a new cell-based treatment for people with Duchenne muscular dystrophy to see if it is safe and helps improve muscle function.
Quick facts
| Phase | PHASE1 |
|---|---|
| Study type | Interventional |
| Enrollment | 8 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Masonic Cancer Center, University of Minnesota (other) |
| Locations | 1 site (Minneapolis, Minnesota) |
| Trial ID | NCT06692426 on ClinicalTrials.gov |
What this trial studies
This phase 1 trial evaluates the safety and tolerability of local injections of induced pluripotent stem cell-derived muscle progenitor cells in individuals with Duchenne muscular dystrophy (DMD). Conducted at the University of Minnesota, the study focuses on non-ambulatory patients with specific muscle function criteria. Participants will receive the MyoPAXon treatment along with Tacrolimus, and will be monitored for 15 years to assess outcomes. The trial aims to gather preliminary data on the effectiveness of this innovative therapy.
Who should consider this trial
Good fit: Ideal candidates are non-ambulatory adults aged 18 and older diagnosed with Duchenne muscular dystrophy.
Not a fit: Patients who are ambulatory or do not meet the specific muscle function criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could provide a new treatment option that may improve muscle function and quality of life for patients with DMD.
How similar studies have performed: While this approach is novel, similar studies using stem cell therapies for muscular dystrophies have shown promise, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Minneapolis, Minnesota
- Masonic Cancer Center — Minneapolis, Minnesota, United States (RECRUITING)
Study contacts
- Study coordinator: Peter Kang, MD
- Email: mdstemcell@umn.edu
- Phone: 612-624-9452
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Duchenne Muscular Dystrophy