Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
Part of paid clinical trials in Aurora, Colorado.
- Sponsor
- University of Colorado, Denver
- Study ID
- NCT06565572
- Phase
- EARLY_PHASE1
- Status
- Enrolling By Invitation
Conditions
- Posterior Column Ataxia With Retinitis Pigmentosa
Eligibility Criteria
- Sex
- ALL
- Age
- N/A - N/A
- Healthy Volunteers
- Not accepted
Interventions
- nL-FLVC-001 — DRUGnL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous
Study Details
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 Dates
- Start date
- Aug 23, 2023
- Status verified
- May 2026
- Primary completion
- Aug 21, 2024
- Completion
- Dec 31, 2028
Study Design
- Enrollment
- 1 participants (estimated)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: nL-FLVC-001 ArmnL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous
Primary Outcome Measure
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: Over 12 months ]
Locations (1)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Childrens Hospital Colorado | Aurora | Colorado | 80045 | - |
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