Myogenica MyoPAXon Phase 1 Clinical Trial for Duchenne muscular dystrophy

Actively RecruitingPhase 1Improving Muscle Growth & Protection

Age: 18 years and older
Phase: Phase 1
Sponsor: Myogenica
Therapeutic Approach: Improving Muscle Growth & Protection
Variant Requirement: Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and/or absence of immunohistochemical staining for dystrophin on muscle biopsy.
Eligible Sex: Male
Ambulation: Non-Ambulatory
Study Start (Actual): 2025-03-20
Primary Completion (Estimated): 2027-03-03
Study Completion (Estimated): 2027-03-03
Enrollment (Estimated): 8
Countries: United States

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.

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NCT ID

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Study Requirements and Criteria