Clinical Study Alert: Phase 1 Study of MyoPAXon in Boys with DMD

Researchers at the University of Minnesota are seeking boys with Duchenne muscular dystrophy (DMD) to participate in a phase 1 clinical trial to evaluate the safety and efficacy of the investigational cell-based therapy MyoPAXon in combination with the immunosuppressant therapy tacrolimus. The current study is examining whether this investigational treatment is safe and well-tolerated in people with DMD, and whether treatment may increase muscle growth and function.

The study

This is an open-label, dose-ranging study, which means that all participants will receive one of four doses of MyoPAXon treatment, ranging from a low dose to a high dose. The goal is to determine the optimal dose for use in future studies. Participants will be started on tacrolimus one week prior to MyoPAXon administration and will then receive a single injection of MyoPAXon. They will then receive twice a day maintenance doses of tacrolimus for three months. Participants will be required to attend 5-10 clinic visits during the three-month study and monitored for an additional 15 years.

MyoPAXon will be administered through a one-time intramuscular (in the muscle) injection. The effects of the therapy will be evaluated using a number of tests and procedures including but not limited to blood testing for donor-specific anti-HLA antibodies and cellular response.

Eligibility criteria

To be eligible, individuals must meet the following inclusion criteria:

• Have DMD, diagnosed by mutations in the DMD 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 within 14 days prior to enrollment (28 days for cardiac and pulmonary function)
• If applicable, willing to use at least two forms of effective birth control 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
• Willing to consent to monitoring for 15 years, including an extension period
• Voluntary written consent from the subject or parent(s)/guardian(s)
• Additional study criteria

Individuals may not be eligible to participate if they are affected by another condition or receiving another treatment that might interfere with the ability to undergo safe testing.

Please visit this link for more detailed listing of inclusion and exclusion criteria.

Interested in participating?

To learn more about the study or inquire about participation, visit the sponsor’s trial website or contact the study coordinator by email at mdstemcell@umn.edu.