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 wellas changes in DMD-related functional testing/assessments, blood laboratories, andinflammation related biomarker levels over time.
The study will enroll 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 24 months.
Biological: Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)
Umbilical cord-derived mesenchymal stem cells administered intravenously.
Inclusion Criteria:
1. Male sex by birth with a genetically confirmed diagnosis of Duchenne Muscular
Dystrophy (DMD).
2. Age is greater than or equal to 5 and less than or equal to 10 years.
3. Has a North Star Ambulatory Assessment (NSAA) score greater than 13 and less than
30.
4. Demonstrates the ability to perform the "time to rise" test in under 10 seconds.
5. Is up-to-date on immunizations.
6. Is on a stable dose of glucocorticoids for at least 12 weeks prior to study
participation, except for weight-based or toxicity-related adjustments.
7. Is on a stable dose of supplements for at least 12 weeks prior to study
participation.
8. Has the ability to comply with the requirements of the study and the ability to
understand and provide written informed assent and a guardian's consent.
9. Patient must be either a non-responder to or a poor candidate for treatment with
another established therapy.
Exclusion Criteria:
1. Active cancer or prior diagnosis of cancer within the past year (patients with basal
and squamous cell cancer of the skin will not be excluded).
2. BMI > 45 kg/m².
3. Any other condition (including concomitant treatment) that, in the judgment of the
Investigator or Sponsor, would be a contraindication to enrollment, study product
administration (e.g., known hypersensitivity to dimethyl sulfoxide (DMSO), Human
Serum Albumin (HSA), or PlasmaLyte), or follow-up.
4. Treatment with an exon skipping therapy within 3 months of study start.
5. Cognitive delay or impairment that can confound motor development in the opinion of
the investigator.
6. Major surgery within 3 months prior to Day 0 or planned surgery or procedures that
could affect the conduct of the study.
Neurology Rare Disease Center
Denton, Texas, United States
Investigator: Diana Castro, MD
Contact: 972-982-7411
drcastro@neuromdcenter.com
Investigator: Diana Castro, MD
Jennifer Avelar, AS
714-616-1791
jennifer.avelar@neuromdcenter.com
Not Provided