Can a One-Time gene injection halt nerve damage in CMT1A?
NCT ID NCT03520751
First seen Jul 29, 2026 · Last updated Jul 30, 2026 · Updated 1 time
Summary
This trial tests a one-time gene therapy injection for people with Charcot-Marie-Tooth disease type 1A (CMT1A), a genetic nerve disorder that causes progressive muscle weakness. The therapy delivers a gene that produces a protein to support nerve health. Three adults aged 18 to 35 receive injections into leg muscles to see if it can halt the decline in muscle function and sensation.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- a one-time gene therapy injection called scAAV1.tMCK.NTF3, which delivers a gene to produce neurotrophin-3, a protein that supports nerve health
- What this could lead to
- If successful, this could point toward a treatment that slows or stops the progression of CMT1A, preserving muscle strength and sensation.
- What could go wrong
- This is an early-phase trial with only three participants, so safety and effectiveness are not yet known. Gene therapies can cause immune reactions or other 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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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States