One-Time gene injection aims to halt rare childhood paralysis
NCT ID NCT05152823
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a single injection of gene therapy for people with SMARD1 or CMT2S, rare diseases caused by mutations in the IGHMBP2 gene. The therapy delivers a working copy of the gene directly into the spinal fluid. Ten participants, ranging from infants to children up to 6 years old, will be monitored for safety and any improvement in movement.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- AAV9 carrying the IGHMBP2 gene
- What this could lead to
- If successful, this could point toward a treatment that slows or stops the progression of these rare nerve and muscle diseases.
- What could go wrong
- This is a very early, small trial with only 10 participants, so results may not apply widely. There are risks of serious side effects from the gene therapy itself.
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