NCT06565572Enrolling by invitationEarly phase 1
Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
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
Source: ClinicalTrials.gov · Last updated 19 May 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Enrolling by invitation
- Phase (registry)
- Early phase 1
- Study type
- Interventional
- Enrollment
- 1 (estimated)
- First submitted
- 9 Aug 2023
- Start date
- 23 Aug 2023
- Primary completion date
- 21 Aug 2024
- Lead sponsor
- University of Colorado, Denver
- Diseases
- Retinitis pigmentosa
- NCT number
- NCT06565572
What you commit to
- Study type: Interventional · Early phase 1
- Planned participants: 1
- From 23 Aug 2023 to the primary completion date, 21 Aug 2024
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- 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.
Who the study is looking for
- 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.
Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Childrens Hospital Colorado Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.