Trials / NCT06565572
Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
Enrolling by invitation Early Phase 1 RNA / antisense
- Sponsor
- University of Colorado, Denver
- Treatment
- nL-FLVC-001
- Gene
- Not stated
- Conditions
- Posterior Column Ataxia With Retinitis Pigmentosa
- Ages
- Any to no upper limit
- Enrollment
- 1 participants
- Started
- 2023-08-23
- Main results expected
- 2024-08-21
- Results posted
- Not yet
- Record last updated
- 2026-05-19
What the study is testing
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.
Where it runs
United States (1)
- Childrens Hospital Colorado, Aurora, Colorado