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Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
Sponsor: University of Colorado, Denver
Summary
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.
Official title: 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
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2023-08-23
Completion Date
2028-12-31
Last Updated
2025-01-27
Healthy Volunteers
No
Interventions
nL-FLVC-001
nL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous
Locations (1)
Childrens Hospital Colorado
Aurora, Colorado, United States