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Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1

NCT06565572 · Status: ENROLLING_BY_INVITATION · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2026-05-19

No results posted yet for this study

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.

Conditions

  • Posterior Column Ataxia With Retinitis Pigmentosa

Interventions

DRUG

nL-FLVC-001

nL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous

Sponsors & Collaborators

  • University of Colorado, Denver

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-08-23
Primary Completion
2024-08-21
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06565572 on ClinicalTrials.gov