Stem cells injected to fight rare nerve disease
NCT ID NCT06328712
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a stem cell product called EN001 in 27 adults with Charcot-Marie-Tooth disease type 1A, a genetic nerve disorder that causes muscle weakness. The study has two parts: first, an open-label phase to check safety and find the right dose; second, a placebo-controlled phase to see if it improves nerve function. Participants receive two intravenous infusions four weeks apart.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
EN001 (donor stem cells from umbilical cord tissue)
What this could lead to
If it works, this could point toward a treatment that slows or improves nerve damage in CMT1A, potentially reducing disability.
What could go wrong
This is a very early, small trial (27 people) focused mainly on safety. The stem cell approach is experimental, and it may not improve symptoms or could cause side effects.
Disclaimer
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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.
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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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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Dongguk University Gyeongju Hospital
RECRUITINGGyeongju, South Korea
Contact
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Kyung Hee University Hospital at Gangdong
RECRUITINGSeoul, South Korea
Contact
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Samsung Medical Center
RECRUITINGSeoul, South Korea
Contact