Custom drug targets rare genetic brain disease in First-Ever human test
NCT ID NCT06706388
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic disorder that affects movement and brain function. The drug aims to reduce the harmful effects of the mutated ATN1 gene. The single participant will be monitored for changes in ataxia (loss of muscle control), seizures, and quality of life over two years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- personalized antisense oligonucleotide (nL-ATN1-002)
- What this could lead to
- If it works, this could point toward a treatment for DRPLA, a rare and severe genetic brain disorder.
- What could go wrong
- This is an early-stage, single-participant study, so results may not apply to others. The drug is experimental and risks are unknown.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for DENTATORUBRAL-PALLIDOLUYSIAN ATROPHY are added.
By submitting, you agree to our Terms of use
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
-
Columbia University
New York, New York, 10027, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.