Clin2
NCT06565572Possibly a fitEnrolling by invitation

Antisense drug for PCARP disease caused by FLVCR1 mutation

Posterior Column Ataxia With Retinitis Pigmentosa

Part of Brain & nervous system, Eyes & vision, Genetic & congenital clinical trials.

This trial tests a new drug called an antisense oligonucleotide (a type of genetic medicine) for people with a specific form of inherited eye disease called PCARP, which is caused by changes in the FLVCR1 gene. The goal is to see if the drug can slow or stop vision loss.

Summary written for real people, not researchers, by Clin2.

Phase
Early Phase 1
Enrollment
1 people
Ages
Any age
Study type
Interventional

Who can take part

  • You must have a confirmed genetic mutation in the FLVCR1 gene.
  • You must be able to travel to the study site and keep all follow-up appointments.
  • You must not be allergic to any part of the study drug.

View the official record on ClinicalTrials.gov

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