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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Medical Surgical Associates Center
RECRUITINGSt John's, Antigua and Barbuda
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Prodromos Stem Cell Institute
RECRUITINGNaples, Florida, 34102, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Prodromos Stem Cell Institute
RECRUITINGIrving, Texas, 75039, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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