Phase 2 Expanded Access Study of UC-MSC in DMD Patients

Intermediate Size Expanded Access Study of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) In Patients With Duchenne Muscular Dystrophy

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Biological: Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)

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.

• Study Type: Interventional
• Enrollment (Estimated): 35
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Texas
    • Dallas, Texas, United States, 75201
      • Other locations - TBD
    • Flower Mound, Texas, United States, 75028
      • Neurology Rare Disease Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: UC-MSC Treatment Group; Intravenous infusion of UC-MSC	Biological: Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC); Umbilical cord-derived mesenchymal stem cells administered intravenously.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	From enrollment to the end of follow up at 12-months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Secondary Endpoint	The secondary endpoint is assessment of changes in blood laboratory and inflammation related biomarker levels over time.	From enrollment through follow up at 12-months

Collaborators and Investigators

• Sponsor: MED Institute Inc.
• Collaborators: Signature Biologics

Study record dates

Study Major Dates

• Study Start (Actual): August 5, 2024
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): April 30, 2029

Study Registration Dates

• First Submitted: August 27, 2024
• First Submitted That Met QC Criteria: August 27, 2024
• First Posted (Actual): August 30, 2024

Study Record Updates

• Last Update Posted (Actual): July 24, 2026
• Last Update Submitted That Met QC Criteria: July 22, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: IND 16561 - Serial 0021

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No