One-Time gene shot aims to stop rare nerve disease in kids
NCT ID NCT07447557
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
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the original study
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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: •••••@•••••