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Antisense Oligonucleotide

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
No Placebo-Only Group

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 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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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

Find a Location

Who is running the clinical trial?

University of Colorado, DenverLead Sponsor
1,810 Previous Clinical Trials
2,920,099 Total Patients Enrolled
~0 spots leftby Dec 2025
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