Antisense Oligonucleotide Treatment for Retinitis Pigmentosa

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

Genetically confirmed FLVCR1-related disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Summary

This trial aims to test a new medication in one patient with a specific type of ataxia and retinitis pigmentosa. The main focus is on determining how safe and well-tolerated the

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

Select...

My condition is genetically confirmed to be FLVCR1-related.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

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

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

University of Colorado, DenverLead Sponsor

1,786 Previous Clinical Trials

2,787,607 Total Patients Enrolled

~0 spots leftby Sep 2025

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