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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| nL-FLVC-001 Arm | Experimental | nL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| nL-FLVC-001 | Drug | nL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Childrens Hospital Colorado | Aurora | Colorado | 80045 | United States |
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| ID | Term |
|---|---|
| C536343 | Posterior column ataxia with retinitis pigmentosa |
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