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

November 6, 2025 updated by: The Foundation for Orthopaedics and Regenerative Medicine

Mesenchymal Stem Cell Exosome Treatment of Congenital Myasthenic Syndrome

Patients with Congenital Myasthenic Syndrome will be treated with Mesenchymal Stem Cell Exosome solution.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Single Arm non-controlled study. Patients are prospectively evaluated then treated. Outcomes are tracked after treatment.

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Antigua and Barbuda
      • St John's, Antigua and Barbuda
        • Recruiting
        • Medical Surgical Associates Center
        • Contact:
  • United States
    • Florida
      • Naples, Florida, United States, 34102
        • Recruiting
        • Prodromos Stem Cell Institute
        • Contact:
    • Texas
      • Irving, Texas, United States, 75039
        • Recruiting
        • Prodromos Stem Cell Institute
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treatment Group
Biological: AlloEx exosomes
This is an intranasal treatment of exosomes derived from mesenchymal stem cells.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Oxygen Saturation
Time Frame: From enrollment to 6 months after treatment
From enrollment to 6 months after treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The Foundation for Orthopaedics and Regenerative Medicine

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2025

Primary Completion (Estimated)

December 31, 2028

Study Completion (Estimated)

December 31, 2028

Study Registration Dates

First Submitted

November 5, 2025

First Submitted That Met QC Criteria

November 6, 2025

First Posted (Actual)

November 10, 2025

Study Record Updates

Last Update Posted (Actual)

November 10, 2025

Last Update Submitted That Met QC Criteria

November 6, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FOREM2025-CMS1

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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