Custom-Made genetic drug takes aim at rare form of ALS
NCT ID NCT06392126
First seen Jul 13, 2026 · Last updated Jul 14, 2026 · Updated 1 time
Summary
This study tests a custom-designed antisense oligonucleotide (ASO) drug for one person with ALS caused by a specific CHCHD10 gene variant. The drug aims to slow disease progression by targeting the genetic root of the condition. Researchers will monitor changes in muscle function, breathing, thinking, and survival over 12 months, along with safety.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- nL-CHCHD-001 (personalized antisense oligonucleotide)
- What this could lead to
- If successful, this approach could point toward a tailored treatment for ALS caused by specific genetic mutations.
- What could go wrong
- This is a very early, single-participant study, so results may not apply to others. The ASO may not slow disease or could cause 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
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Locations
-
Mayo Clinic
Jacksonville, Florida, 32224, United States
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