Mesenchymal stem cell exosome treatment for congenital myasthenic syndrome
Official title: Mesenchymal Stem Cell Exosome Treatment of Congenital Myasthenic Syndrome
Run by The Foundation for Orthopaedics and Regenerative Medicine · NCT07226726 · Data updated 2026-11-12
This trial will try a mesenchymal stem cell exosome solution (AlloEx) in people with congenital myasthenic syndrome to see if it helps their symptoms.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 20 (estimated) |
| Sponsor | The Foundation for Orthopaedics and Regenerative Medicine Academic / other |
| Locations | 2 sites (Naples, Florida and 1 other locations) |
| Status | Recruiting |
| Started | January 1, 2025 |
| Main results expected | December 31, 2028 |
| Study ends | December 31, 2028 |
| Registry record updated | October 16, 2026 |
| Trial ID | NCT07226726 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
People with a licensed physician's diagnosis of congenital myasthenic syndrome who can provide informed consent (or have a guardian) and can travel to a participating site are ideal candidates.
✕Probably not a fit
People who are pregnant or who have active cancer are excluded from the trial and would not be eligible to receive this treatment within the study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a single-arm, open-label Phase 1 study where participants with congenital myasthenic syndrome are prospectively evaluated and then treated with an AlloEx mesenchymal stem cell exosome solution. There is no control group and participants are followed over time to track safety and clinical outcomes after treatment. Key eligibility requires a physician-confirmed diagnosis, ability to consent (or have a guardian consent) and ability to travel to a study site, while pregnant people and those with active malignancy are excluded. Data collection focuses on tolerability and any signal of clinical benefit to inform future studies.
Why it matters
Potential benefit: If successful, this treatment could reduce muscle weakness and improve daily function for some people with congenital myasthenic syndrome.
How similar studies have gone: This application of mesenchymal stem cell exosomes to congenital myasthenic syndrome is novel with limited prior human data, although early-stage preclinical and small clinical studies of exosomes exist in related neuromuscular and inflammatory conditions.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients will need a diagnosis of Congenital Myasthenic Syndrome by a licensed physician. * Patients must be able to provide informed consent, or have a guardian who does. * Patient must be able to travel to the site of treatment. Exclusion Criteria: * Patients will be excluded from the trial if they are pregnant or have active cancer (malignancy) at the screening consultation.
Where it's running
-
Prodromos Stem Cell Institute
Naples, Florida, United States Recruiting -
Prodromos Stem Cell Institute
Athens, Greece Recruiting
Study contacts
- Study coordinator: Chadwick Prodromos
- Email: care@thepsci.com
- Phone: 847-699-6810
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Long-term observation of congenital muscle diseases
This study is trying to learn more about congenital muscle diseases by collecting information from patients over 10 years to help improve care and treatment options.
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This study is tracking how congenital myasthenic syndromes affect people over five years to better understand the condition and help improve future treatments.
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.