Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1

Plain-language summary

This Early Phase 1 study evaluates nL-FLVC-001, a personalized antisense oligonucleotide (ASO) medication, in a single patient with posterior column ataxia with retinitis pigmentosa (PCARP). The main question it aims to answer is the safety and tolerability of this medication in that single participant. Eligibility requires genetically confirmed FLVCR1-related disease, informed consent or assent, and the ability to travel to the study site and adhere to study-related follow-up examinations.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Early Phase 1

Status

Enrolling by invitation

Sponsor

University of Colorado, Denver

Intervention

nL-FLVC-001

Start date

8/23/2023

Estimated completion

12/31/2028

Countries

United States

Related genes

Locations

• Aurora, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* 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 Criteria:

* 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.