Experimental stem cells tested for rare nerve disease
NCT ID NCT06218134
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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.
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.
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
Locations
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SMC
Seoul, Gangnam-gu, 06351, South Korea
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