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Antisense Oligonucleotide

nL-FLVC-001 Arm for Bassen-Kornzweig syndrome

Phase < 1
Waitlist Available
Research Sponsored by University of Colorado, Denver
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
* 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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up over 12 months
Awards & highlights

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.

Who is the study for?
This trial is for a single patient with posterior column ataxia and retinitis pigmentosa due to FLVCR1 mutation. Specific eligibility criteria are not provided, indicating that the participant has likely been pre-selected.
What is being tested?
The trial is testing nL-FLVC-001, an antisense oligonucleotide treatment, on one patient. It focuses on assessing the safety and how well the patient can tolerate this medication.
What are the potential side effects?
Since this is a unique case study, specific side effects are not listed but may include reactions at injection sites, potential organ damage or dysfunction, flu-like symptoms, or other unforeseen responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~over 12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and over 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
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
Secondary study objectives
Measure any changes in the Cardiff Visual Ability Questionnaire for Children (CVAQC-25) in one patient with PCARP after nL-FLVC-001 intravitreal injection.

Trial Design

1Treatment groups
Experimental Treatment
Group I: nL-FLVC-001 ArmExperimental Treatment1 Intervention
nL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous

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Who is running the clinical trial?

University of Colorado, DenverLead Sponsor
1,776 Previous Clinical Trials
2,775,088 Total Patients Enrolled
~0 spots leftby Sep 2025
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