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.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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About 1 person
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2023
- Expected to finish
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Dec 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s). * Genetically confirmed FLVCR1-related disease. * Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records. Exclusion Criteria: * Allergy to any of the ASO components * Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Childrens Hospital Colorado
Aurora, Colorado, 80045, United States