Phase 1 Study of Intranasal Mesenchymal Stem Cell Exosome Treatment for Adults with Congenital Myasthenic Syndrome

What this Trial Is About

This research focuses on patients diagnosed with Congenital Myasthenic Syndrome, aiming to evaluate the effects of a treatment using mesenchymal stem cell exosome solution. The study is a Phase 1, single-arm, non-controlled trial where patients are assessed before treatment and monitored afterward to track outcomes such as oxygen saturation over a six-month period. Participants receive an experimental intranasal treatment called AlloEx exosomes, which are derived from mesenchymal stem cells. The study involves prospective evaluation of patients, treatment administration, and follow-up assessments but does not include a control or placebo group. During the trial, participants will undergo regular monitoring from enrollment up to six months after treatment to evaluate oxygen saturation levels. The study requires patients to visit the treatment site and provide informed consent. The trial is sponsored by The Foundation for Orthopaedics and Regenerative Medicine and is designed to carefully track patient outcomes following the investigational treatment.

Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution