Trials / Enrolling By Invitation
Enrolling By InvitationNCT06565572
Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for PCARP (Posterior Column Ataxia with Retinitis Pigmentosa) Disease Due to Mutations in FLVCR1
- Status
- Enrolling By Invitation
- Phase
- EARLY_Phase 1
- Study type
- Interventional
- Enrollment
- 1 (estimated)
- Sponsor
- University of Colorado, Denver · Academic / Other
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | nL-FLVC-001 | nL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous |
Timeline
- Start date
- 2023-08-23
- Primary completion
- 2024-08-21
- Completion
- 2028-12-31
- First posted
- 2024-08-22
- Last updated
- 2025-01-27
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06565572. Inclusion in this directory is not an endorsement.