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

The Foundation for Orthopaedics and Regenerative Medicine

Status and phase: Enrolling; Phase 1

Conditions: Congenital Myasthenic Syndrome

Treatments: Biological: AlloEx exosomes

Study type: Interventional

Funder types: Other

Identifiers

NCT07226726

FOREM2025-CMS1

Details and patient eligibility

About

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

Full description

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

Enrollment: 20 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Treatment Group

Experimental group

Treatment:

Biological: AlloEx exosomes