NCT06565572
Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
Enrolling by Invitation
EARLY_PHASE1All AgesInterventionalTreatmentUniversity of Colorado, DenverInvestigator-initiated
~1 participants
Updated 2026-05-19 on ClinicalTrials.gov
What's tested:nL-FLVC-001
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Measured over Over 12 months
+1 more outcome measured
Conditions
Where it's being run
1 sites across 1 statesColorado1
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
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
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.
What this trial measures
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0Over 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 mutationOver 12 months