NCT06579352

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
35

participants targeted

Target at P25-P50 for phase_2

Timeline
34mo left

Started Aug 2024

Longer than P75 for phase_2

Geographic Reach
1 country

2 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress42%
Aug 2024Apr 2029

Study Start

First participant enrolled

August 5, 2024

Completed
22 days until next milestone

First Submitted

Initial submission to the registry

August 27, 2024

Completed
3 days until next milestone

First Posted

Study publicly available on registry

August 30, 2024

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2028

Expected
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

April 30, 2029

Last Updated

July 24, 2026

Status Verified

July 1, 2026

Enrollment Period

4.2 years

First QC Date

August 27, 2024

Last Update Submit

July 22, 2026

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (1)

  • Secondary Endpoint

    From enrollment through follow up at 12-months

Study Arms (1)

UC-MSC Treatment Group

EXPERIMENTAL

Intravenous infusion of UC-MSC

Biological: Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)

Interventions

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

Umbilical cord-derived mesenchymal stem cells administered intravenously.

UC-MSC Treatment Group

Eligibility Criteria

Age5 Years - 10 Years
Sexmale(Gender-based eligibility)
Gender Eligibility DetailsStudy participants will be ambulatory males aged 5-10 with genetically confirmed DMD.
Healthy VolunteersNo
Age GroupsChild (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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Other locations - TBD

Dallas, Texas, 75201, United States

Location

Neurology Rare Disease Center

Flower Mound, Texas, 75028, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 27, 2024

First Posted

August 30, 2024

Study Start

August 5, 2024

Primary Completion (Estimated)

October 31, 2028

Study Completion (Estimated)

April 30, 2029

Last Updated

July 24, 2026

Record last verified: 2026-07

Data Sharing

IPD Sharing
Will not share

Locations

US(2)