Phase 2 Expanded Access Study of UC-MSC in DMD Patients (NCT06579352) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Phase 2 Expanded Access Study of UC-MSC in DMD Patients
United States35 participantsStarted 2024-08-05
Plain-language 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.
Who can participate
Age range
5 Years – 10 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Primary Endpoints
Timeframe: From enrollment to the end of follow up at 12-months
. 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.
Exclusion criteria
. 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.