Expanded Access Study of UC-MSC in DMD Patients

NCT06579352 | Unknown

• 1 other identifier: IND 16561 - Serial 0017
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy

Interventions

Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) BIOLOGICAL

Umbilical cord-derived mesenchymal stem cells administered intravenously.

Eligibility Criteria

• Age: 5 Years - 10 Years
• Gender Eligibility Details: Study participants will be ambulatory males aged 5-10 with genetically confirmed DMD.
• Age Groups: Child (0-17)

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.

Sponsors & Collaborators

• MED Institute Inc.: lead
• Signature Biologics: collaborator

Study Sites (1)

Neurology Rare Disease Center

Denton, Texas, 76208, United States

AVAILABLE

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Jennifer Avelar, AS

CONTACT

714-616-1791; jennifer.avelar@neuromdcenter.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 27, 2024
• First Posted: August 30, 2024
• Last Updated: August 30, 2024

Record last verified: 2024-08

Locations

US (1)