Congenital Myasthenic SyndromePhase 1NCT07226726
Patients With Congenital Myasthenic Syndrome Will be Treated With Umbilical or Placental Derived Mesenchymal Stem Cell Exosome Solution
A Phase 1 study testing AlloEx exosomes in people with Congenital Myasthenic Syndrome, run by The Foundation for Orthopaedics and Regenerative Medicine at 2 sites in 2 countries.
Trial summary
Patients with Congenital Myasthenic Syndrome will be treated with Umbilical or Placental Derived Mesenchymal Stem Cell Exosome solution. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Congenital Myasthenic Syndrome
- What is being tested?
- AlloEx exosomes
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 1 Jan 2025to 31 Dec 2028
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Patients will be excluded from the trial if they are pregnant or have active cancer (malignancy) at the screening consultation.
Trial locations and contacts
2 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
2 sites in 2 countries
United States1 site, 1 recruiting
- Recruiting
Prodromos Stem Cell Institute
Naples, Florida, 34102
Chadwick Prodromos, MD, 847-699-6810 ext. 202, care@thepsci.com
Greece1 site, 1 recruiting
- Recruiting
Prodromos Stem Cell Institute
Athens
Chadwick Prodromos, MD, 847-699-6810 ext. 202, care@thepsci.com
About this study
Official title: Mesenchymal Stem Cell Exosome Treatment of Congenital Myasthenic Syndrome
Patients with Congenital Myasthenic Syndrome will be treated with Umbilical or Placental Derived Mesenchymal Stem Cell Exosome solution.
Read the detailed description
Single Arm non-controlled study. Patients are prospectively evaluated then treated. Outcomes are tracked after treatment.
- Conditions
- Congenital Myasthenic Syndrome
- Being tested
- AlloEx exosomes
- Keywords
- exosomes, secretome, Congenital Myasthenic Syndrome
- Medical subject headings
- Myasthenic Syndromes, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlloEx exosomesBiological
This is an intranasal treatment of exosomes derived from mesenchymal stem cells.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group | Experimental | Not described Receives: Biological: AlloEx exosomes |
What is being measured
The primary outcome is the main question the study is designed to answer.
Oxygen Saturation
Measured: From enrollment to 6 months after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2025
- Study start1 Jan 2025
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Myasthenic SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07226726
- Sponsor's protocol ID
- FOREM2025-CMS1
- Status last verified
- Oct 2026
- First submitted
- 5 Nov 2025
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No