Duchenne Muscular DystrophyPhase 2NCT06579352
Study of UC-MSC in DMD Patients
A Phase 2 study testing Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) in people with Duchenne Muscular Dystrophy, run by MED Institute Inc. at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 10 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 2 sitesUnited States
- Study dates
- 5 Aug 2024to 30 Apr 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 10 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Other locations - TBD
Dallas, Texas, 75201
Neurology Rare Disease Center
Flower Mound, Texas, 75028
About this study
Official title: 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.
Read the 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)Biological
Umbilical cord-derived mesenchymal stem cells administered intravenously.
| Study group | Role | What they receive |
|---|---|---|
| UC-MSC Treatment Group | Experimental | Intravenous infusion of UC-MSC Receives: Biological: Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From enrollment to the end of follow up at 12-months
Secondary outcomes (1)
Secondary Endpoint
The secondary endpoint is assessment of changes in blood laboratory and inflammation related biomarker levels over time.
Measured: From enrollment through follow up at 12-months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2024
- Study start5 Aug 2024
- Primary completion31 Oct 2028 (estimated)
- Study completion30 Apr 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- MED Institute Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Signature Biologics
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06579352
- Sponsor's protocol ID
- IND 16561 - Serial 0021
- Status last verified
- Aug 2026
- First submitted
- 27 Aug 2024
- Last updated
- 14 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) In Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT06579352
- Acronym / sponsor ID
- IND 16561 - Serial 0021
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- MED Institute Inc.Industry (pharma, biotech, devices)
- Collaborators
- Signature Biologics
- Study start
- 5 Aug 2024 (actual)
- Primary completion
- 31 Oct 2028 (estimated)
- Study completion
- 30 Apr 2029 (estimated)
- First posted
- 30 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UC-MSC Treatment Group | Experimental | Biological: Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)Intravenous infusion of UC-MSC |
Who may qualify
- Ages
- 5 to 10 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)Umbilical cord-derived mesenchymal stem cells administered intravenously. | Biological | UC-MSC Treatment Group |
Primary outcome
Primary Endpoints
Time frame: 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.
1 secondary outcome
Secondary Endpoint
Time frame: From enrollment through follow up at 12-months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) in people with Duchenne Muscular Dystrophy, run by MED Institute Inc. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 14 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Endpoints (time frame: From enrollment to the end of follow up at 12-months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2026.