Tests an experimental antisense medicine for a rare inherited nerve and eye disease
Tests an experimental medicine in one person with a rare inherited disease.
Plain-language title generated from the registry record
Registry title: Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
Key facts
- Where
- Aurora, United StatesSee the site
- Phase
- Early phase 1
Where in United States
- Childrens Hospital ColoradoAurora, United StatesNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- University of Colorado, Denver
- Trial code
- NCT06565572
- Record updated
- 19 May 2026
- Original description
The goal of this clinical trial is to evaluate a specific antisense oligonucleotide medication in one patient with posterior column ataxia with retinitis pigmentosa. The main question it aims to answer is: what is the safety and tolerability of this medication in a single participant.Read full original descriptionShow less
An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for PCARP (Posterior Column Ataxia With Retinitis Pigmentosa) Disease Due to Mutations in FLVCR1
The goal of this clinical trial is to evaluate a specific antisense oligonucleotide medication in one patient with posterior column ataxia with retinitis pigmentosa. The main question it aims to answer is: what is the safety and tolerability of this medication in a single participant.
Inclusion: 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: 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.