New drug EN001 tested for rare nerve disease – early safety results in

NCT ID NCT06328712

First seen Jan 10, 2026 · Last updated May 15, 2026 · Updated 19 times

Summary

This early-phase trial tested the safety of a new drug called EN001 in 6 adults with Charcot-Marie-Tooth disease type 1A, a genetic nerve disorder that causes muscle weakness and numbness. The main goal was to see if the drug caused serious side effects. Researchers also looked for signs that the drug might slow the disease, but this was a small, short study focused on safety, not a cure.

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Contacts and locations

Locations

  • Samsung Medical Center

    Seoul, South Korea

Conditions

Explore the condition pages connected to this study.