Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1

Enrolling by invitation · Early Phase 1

Conditions studied: Posterior Column Ataxia With Retinitis Pigmentosa

In brief

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.

Key facts

Study ID

NCT06565572

Run by

University of Colorado, Denver

People needed

1

Starts

2023-08-23

Expected to finish

2028-12-31

Last updated by the study team

2026-05-19

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Childrens Hospital Colorado — Aurora, Colorado, United States

Full record on ClinicalTrials.gov

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