Expanded Access Study of UC-MSC in DMD Patients

August 27, 2024 updated by: MED Institute Inc.

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

Available

Conditions

Intervention / Treatment

Detailed Description

The study will enroll 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 24 months.

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Texas
      • Denton, Texas, United States, 76208
        • Available
        • Neurology Rare Disease Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

N/A

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

MED Institute Inc.

Collaborators

Signature Biologics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

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)

August 30, 2024

Last Update Submitted That Met QC Criteria

August 27, 2024

Last Verified

August 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IND 16561 - Serial 0017

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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