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.
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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Dongguk University Gyeongju Hospital
RECRUITINGGyeongju, South Korea
-
Kyung Hee University Hospital at Gangdong
RECRUITINGSeoul, South Korea
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Samsung Medical Center
RECRUITINGSeoul, South Korea
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