The primary objective of this study is to provide UC-MSC treatment to patients with DMD. Secondary objectives will be to further evaluate treatment-related adverse events as well as changes in DMD-related functional testing/assessments, blood laboratories, and inflammation related biomarker levels over time.
The Phase 2 study will enroll up to 35 ambulatory male participants between the ages of 5 and 10, who will receive four, 3-day intravenous dose cycles of UC-MSC treatment, each administered every three months. Participants will be closely monitored throughout the study period for treatment-related adverse events and changes in DMD-related functional assessments at specific follow-up intervals through 12 months.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
35
Umbilical cord-derived mesenchymal stem cells administered intravenously.
Other locations - TBD
Dallas, Texas, United States
Neurology Rare Disease Center
Flower Mound, Texas, United States
Primary Endpoints
The primary safety endpoint is the rate of treatment-related adverse events over time. The primary efficacy endpoint is the change in DMD-related functional assessments over time.
Time frame: From enrollment to the end of follow up at 12-months
Secondary Endpoint
The secondary endpoint is assessment of changes in blood laboratory and inflammation related biomarker levels over time.
Time frame: From enrollment through follow up at 12-months
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