Can a Custom-Made genetic drug slow ALS?
NCT ID NCT07743268
First seen Aug 03, 2026 · Last updated Aug 04, 2026 · Updated 1 time
Summary
This trial tests a personalized drug called nL-TARDB-006, designed for people with ALS caused by a specific genetic mutation in the TARDBP gene. The drug is an antisense oligonucleotide, which aims to target the genetic cause of the disease. Six participants will receive the treatment, and researchers will track changes in muscle function, breathing, thinking, and survival over 12 months. The goal is to see if this tailored approach can slow the progression of ALS.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- A personalized antisense oligonucleotide drug called nL-TARDB-006
- What this could lead to
- If successful, this approach could slow or halt the progression of ALS in people with TARDBP mutations, offering a targeted treatment for a currently incurable disease.
- What could go wrong
- This is an early-phase, very small trial (6 participants), so results may not generalize. The drug is experimental and may not effectively alter disease course, and there are potential risks from the treatment 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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Columbia University, Irving Medical Center
New York, New York, 10032, United States
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Washington University
St Louis, Missouri, 63110, United States
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