NCT07226726

Brief Summary

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

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at P25-P50 for phase_1

Timeline
33mo left

Started Jan 2025

Longer than P75 for phase_1

Geographic Reach
2 countries

3 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress34%
Jan 2025Dec 2028

Study Start

First participant enrolled

January 1, 2025

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

November 5, 2025

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 10, 2025

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2028

Last Updated

November 10, 2025

Status Verified

November 1, 2025

Enrollment Period

4 years

First QC Date

November 5, 2025

Last Update Submit

November 6, 2025

Conditions

Congenital Myasthenic Syndrome

Keywords

exosomessecretomeCongenital Myasthenic Syndrome

Outcome Measures

Primary Outcomes (1)

  • Oxygen Saturation

    From enrollment to 6 months after treatment

Study Arms (1)

Treatment Group

EXPERIMENTAL
Biological: AlloEx exosomes

Interventions

AlloEx exosomesBIOLOGICAL

This is an intranasal treatment of exosomes derived from mesenchymal stem cells.

Treatment Group

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • Patients will be excluded from the trial if they are pregnant or have active cancer (malignancy) at the screening consultation.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Prodromos Stem Cell Institute

Naples, Florida, 34102, United States

RECRUITING

Prodromos Stem Cell Institute

Irving, Texas, 75039, United States

RECRUITING

Medical Surgical Associates Center

St John's, Antigua and Barbuda

RECRUITING

MeSH Terms

Conditions

Myasthenic Syndromes, Congenital

Condition Hierarchy (Ancestors)

Neuromuscular Junction DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Chadwick Prodromos

CONTACT

18476996810care@thepsci.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 5, 2025

First Posted

November 10, 2025

Study Start

January 1, 2025

Primary Completion (Estimated)

December 31, 2028

Study Completion (Estimated)

December 31, 2028

Last Updated

November 10, 2025

Record last verified: 2025-11

Locations

AN(1)US(2)