Phase 2 Expanded Access Study of UC-MSC in DMD Patients
Running, not enrolling · Phase 2
Conditions studied: Duchenne Muscular Dystrophy
In brief
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.
Key facts
- Study ID
- NCT06579352
- Run by
- MED Institute Inc.
- People needed
- 35
- Starts
- 2024-08-05
- Expected to finish
- 2029-04-30
- Last updated by the study team
- 2026-07-24
Who can join
Age: 5 and older, up to 10. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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).
- BMI > 45 kg/m².
- 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.
- Treatment with an exon skipping therapy within 3 months of study start.
- Cognitive delay or impairment that can confound motor development in the opinion of the investigator.
- Major surgery within 3 months prior to Day 0 or planned surgery or procedures that could affect the conduct of the study.
Where it is running
- Other locations - TBD — Dallas, Texas, United States
- Neurology Rare Disease Center — Flower Mound, Texas, United States
Full record on ClinicalTrials.gov
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