Mesenchymal stem cell exosome treatment for congenital myasthenic syndrome
Mesenchymal Stem Cell Exosome Treatment of Congenital Myasthenic Syndrome
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 |
| Enrollment | 20 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | The Foundation for Orthopaedics and Regenerative Medicine Academic / other |
| Locations | 3 sites (Naples, Florida and 2 other locations) |
| Trial ID | NCT07226726 on ClinicalTrials.gov |
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.
Who should consider this trial
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.
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.
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 performed: 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.
Eligibility criteria
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 this trial is running
Naples, Florida and 2 other locations
- Prodromos Stem Cell Institute — Naples, Florida, United States (Recruiting)
- Prodromos Stem Cell Institute — Irving, Texas, United States (Recruiting)
- Medical Surgical Associates Center — St John's, Antigua and Barbuda (Recruiting)
Study contacts
- Study coordinator: Chadwick Prodromos
- Email: care@thepsci.com
- Phone: 18476996810
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.