Duchenne Muscular Dystrophy
Conditions
Brief summary
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.
Detailed description
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.
Interventions
BIOLOGICALHuman Umbilical Cord Mesenchymal Stem Cells (UC-MSC)
Umbilical cord-derived mesenchymal stem cells administered intravenously.
Sponsors
MED Institute Inc.
Signature Biologics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
5 Years to 10 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Primary Endpoints | From enrollment to the end of follow up at 12-months | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Secondary Endpoint | From enrollment through follow up at 12-months | The secondary endpoint is assessment of changes in blood laboratory and inflammation related biomarker levels over time. |
Countries
United States
Outcome results
None listed