One-Patient trial hopes to slow rare eye and nerve disease
NCT ID NCT06565572
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial tests an experimental drug called nL-FLVC-001 in a single person with posterior column ataxia with retinitis pigmentosa (PCARP), a rare genetic condition that causes vision loss and coordination problems. The drug is an antisense oligonucleotide injected into the eye. The main goal is to check safety and tolerability, with secondary measures looking at vision-related quality of life.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- nL-FLVC-001 (an antisense oligonucleotide injected into the eye)
- What this could lead to
- If it works, this could point toward a treatment for PCARP, a rare disease that affects vision and movement.
- What could go wrong
- This is an extremely early trial with only one participant, so it is too soon to know if the drug is safe or effective. The treatment is injected into the eye, which carries risks like infection or inflammation.
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
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Childrens Hospital Colorado
Aurora, Colorado, 80045, United States