Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1

Early Phase 1

• Conditions: Posterior Column Ataxia With Retinitis Pigmentosa
• Interventions: Drug: nL-FLVC-001

• Registration Number: NCT06565572
• Lead Sponsor: University of Colorado, Denver

Brief Summary

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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2023-08-09
• Study Start: 2023-08-23
• Status Verified: 2024-08
• Study First Submitted QC: 2024-08-19
• Last Update Submitted: 2024-08-19
• Study First Posted: 2024-08-22
• Last Update Posted: 2024-08-22
• Primary Completion: 2024-08-30
• Study Completion: 2028-12-31

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 1

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
nL-FLVC-001 Arm	nL-FLVC-001	nL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous

Primary Outcome Measures

Name	Time	Method
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	Over 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 mutation	Over 12 months

Secondary Outcome Measures

Name	Time	Method
Measure any changes in the Cardiff Visual Ability Questionnaire for Children (CVAQC-25) in one patient with PCARP after nL-FLVC-001 intravitreal injection.	Over 12 months

Trial Locations

Locations (1)

Childrens Hospital Colorado; 🇺🇸 Aurora, Colorado, United States