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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mesenchymal Stem Cell Exosome Treatment of Congenital Myasthenic Syndrome
- ClinicalTrials.gov ID
- NCT07226726
- Acronym / sponsor ID
- FOREM2025-CMS1
- Conditions
- Congenital Myasthenic Syndrome
- Keywords
- exosomes, secretome, Congenital Myasthenic Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- The Foundation for Orthopaedics and Regenerative MedicineUniversity, hospital or other
- Study start
- 1 Jan 2025 (actual)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 10 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients with Congenital Myasthenic Syndrome will be treated with Umbilical or Placental Derived Mesenchymal Stem Cell Exosome solution.
Detailed description
Single Arm non-controlled study. Patients are prospectively evaluated then treated. Outcomes are tracked after treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | Biological: AlloEx exosomes |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (1 listed)
- Patients will be excluded from the trial if they are pregnant or have active cancer (malignancy) at the screening consultation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AlloEx exosomesThis is an intranasal treatment of exosomes derived from mesenchymal stem cells. | Biological | Treatment Group |
Primary outcome
Oxygen Saturation
Time frame: From enrollment to 6 months after treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Greece. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chadwick Prodromos (847-699-6810 ext. 202, care@thepsci.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Oxygen Saturation (time frame: From enrollment to 6 months after treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.