Nasal spray made from stem cells could help rare muscle condition

NCT ID NCT07226726

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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Medical Surgical Associates Center

    RECRUITING

    St John's, Antigua and Barbuda

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Prodromos Stem Cell Institute

    RECRUITING

    Naples, Florida, 34102, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Prodromos Stem Cell Institute

    RECRUITING

    Irving, Texas, 75039, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

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