Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1

University of Colorado Denver (CU Denver)

Status and phase: Invitation-only; Early Phase 1

Conditions: Posterior Column Ataxia with Retinitis Pigmentosa

Treatments: Drug: nL-FLVC-001

Study type: Interventional

Funder types: Other

Identifiers

NCT06565572

22-2313

Details and patient eligibility

About

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.

Enrollment: 1 estimated patient

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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1 participants in 1 patient group

nL-FLVC-001 Arm

Experimental group

Description:

nL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous

Treatment:

Drug: nL-FLVC-001