One-Time gene shot aims to stop rare nerve disease in kids
NCT ID NCT07447557
First seen Jun 25, 2026 · Last updated Jul 09, 2026 · Updated 3 times
Summary
This early-phase trial tests a single injection of gene therapy (ELP-02) into the spinal fluid of 8 children and young adults with CMT4J, a rare genetic nerve disease that causes muscle weakness. The therapy delivers a working copy of the FIG4 gene to nerve cells, potentially slowing or stopping the disease. The main goals are to check safety and see if the treatment can stabilize muscle health and function.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- ELP-02 (a gene therapy that delivers a working copy of the FIG4 gene)
- What this could lead to
- If successful, this could provide a one-time treatment that slows or halts the progression of CMT4J, a rare and severe nerve disease.
- What could go wrong
- This is a very early, first-in-human trial with only 8 participants, so safety and effectiveness are not yet known. Gene therapies can have unpredictable side effects, and the long-term benefits are uncertain.
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
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Study contacts
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Contact
Email: •••••@•••••
Locations
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Stanford University
RECRUITINGPalo Alto, California, 94304, United States
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University of Iowa
NOT_YET_RECRUITINGIowa City, Iowa, 52242, United States
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