Experimental stem cells tested for rare nerve disease
NCT ID NCT06218134
First seen Dec 29, 2025 · Last updated Jun 23, 2026 · Updated 28 times
Summary
This completed early trial tested a single intravenous dose of EN001 stem cells in 3 adults with Charcot-Marie-Tooth disease type 1E, a severe hereditary nerve disorder with no approved treatment. The main goal was to check safety, including infusion reactions, while also looking for signs of benefit using a standard neuropathy scale. The study builds on prior safety data from 9 patients with a related CMT type.
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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SMC
Seoul, Gangnam-gu, 06351, South Korea
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
human Wharton's jelly-derived mesenchymal stem cells (EN001)
What this could lead to
If successful, this could point toward a treatment that slows or stabilizes the progression of severe CMT1E, improving quality of life.
What could go wrong
This was a very small, early-phase trial with only 3 participants, so results may not apply to all patients. The treatment may not show clear benefit, and long-term effects are unknown.
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.