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Nasal spray made from stem cells could help rare muscle condition

NCT ID NCT07226726

Disease control Sponsor: The Foundation for Orthopaedics and Regenerative Medicine Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial is testing a nasal spray made from stem cell exosomes in 20 people with congenital myasthenic syndrome, a rare genetic condition that causes muscle weakness. The goal is to see if the treatment is safe and can improve oxygen levels. Because it is a Phase 1 study, the main focus is safety, not yet proving it works.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
exosomes derived from mesenchymal stem cells (AlloEx), given as a nasal spray
What this could lead to
If it works, this could point toward a new way to manage congenital myasthenic syndrome symptoms without traditional medications.
What could go wrong
This is a very early Phase 1 trial with only 20 people, so safety and effectiveness are not yet known. The treatment may not improve symptoms or could cause unexpected side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 20 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2025

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

congenital myasthenic syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Congenital myasthenic syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    3 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Medical Surgical Associates Center

    RECRUITING

    St John's, Antigua and Barbuda

    Contact Email: •••••@•••••

  • Prodromos Stem Cell Institute

    RECRUITING

    Naples, Florida, 34102, United States

  • Prodromos Stem Cell Institute

    RECRUITING

    Irving, Texas, 75039, United States

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