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Enrolling by invitation NCT06565572

Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1

Early Phase I Interventional Posterior Column Ataxia With Retinitis Pigmentosa

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: nL-FLVC-001.
Who it may be relevant to
Registry conditions: Posterior Column Ataxia With Retinitis Pigmentosa. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

Overview

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.

Interventions

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

Primary outcome measures

  • Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [Time frame: Over 12 months]
  • 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 outcome measures (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]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • Childrens Hospital Colorado — Aurora

Identifiers

NCT: NCT06565572 · 22-2313

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗