Custom drug targets rare blindness in One-Patient trial
NCT ID NCT07177196
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This study tests a custom-made drug called an antisense oligonucleotide, designed specifically for one person with retinal dystrophy caused by a PRPH2 gene mutation. The drug aims to correct the genetic error and potentially slow vision loss. The trial involves only one participant and focuses mainly on safety, while also checking for any changes in vision and eye structure.
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-PRPH2-001)
- What this could lead to
- If it works, this could point toward a treatment for retinal dystrophy caused by PRPH2 mutations, potentially slowing vision loss.
- What could go wrong
- This is a very early, single-person trial, so results may not apply to others. The treatment is experimental and safety is the main focus; it may not improve vision.
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
-
University of California San Diego
San Diego, California, 92093, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.