Study of UC-MSC in DMD Patients

Korábbi kezelés

• 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).
• Treatment with an exon skipping therapy within 3 months of study start.
• Major surgery within 3 months prior to Day 0 or planned surgery or procedures that could affect the conduct of the study.

Más betegség

• Cognitive delay or impairment that can confound motor development in the opinion of the investigator.

Laborértékek

• 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.

Egyéb

• BMI \> 45 kg/m².

• Male sex by birth with a genetically confirmed diagnosis of Duchenne Muscular Dystrophy (DMD).
• Age is greater than or equal to 5 and less than or equal to 10 years.
• Has a North Star Ambulatory Assessment (NSAA) score greater than 13 and less than 30.
• Demonstrates the ability to perform the "time to rise" test in under 10 seconds.
• Is up-to-date on immunizations.
• Is on a stable dose of glucocorticoids for at least 12 weeks prior to study participation, except for weight-based or toxicity-related adjustments.
• Is on a stable dose of supplements for at least 12 weeks prior to study participation.
• 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.
• Patient must be either a non-responder to or a poor candidate for treatment with another established therapy.