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OpenTrials
Enrolling by Invitation

NCT Number: NCT06565572

Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation 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.

Enrolling by Invitation

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Key information

Conditions

Posterior Column Ataxia With Retinitis Pigmentosa

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Childrens Hospital Colorado

Aurora, Colorado, 80045, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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 criteria

  • 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.

Treatment and study plan

nL-FLVC-001

Drug

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

Primary outcomes

  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

    Time frame: Over 12 months

  2. We will measure visual acuity, retinal thickness, examine changes in fundus photos and biomicroscopic exam post nL-FLVC-001 administration in a participant with FLVCR1 gene mutation

    Time frame: Over 12 months

Secondary outcomes

  1. Measure any changes in the Cardiff Visual Ability Questionnaire for Children (CVAQC-25) in one patient with PCARP after nL-FLVC-001 intravitreal injection.

    Time frame: Over 12 months

Sponsors and collaborators

Lead sponsor

University of Colorado, Denver

Other

Registry information

Official study 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

Important dates

Study start
2023
Primary completion
2024
Study completion
2028
First posted
Aug 22, 2024
Registry last updated
May 19, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.