Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
Phase 1
- Conditions
- Posterior Column Ataxia With Retinitis Pigmentosa
- Registration Number
- NCT06565572
- Lead Sponsor
- University of Colorado, Denver
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Enrolling by invitation
- Sex
- All
- Target Recruitment
- 1
Inclusion Criteria
Inclusion Criteria:<br><br> - Informed consent/assent provided by the participant (when appropriate), and/or<br> participant's parent(s) or legally authorized representative(s).<br><br> - Genetically confirmed FLVCR1-related disease.<br><br> - Ability to travel to the study site and adhere to study-related follow-up<br> examinations and/or procedures and provide access to participant's medical records.<br><br>Exclusion Criteria:<br><br> - Allergy to any of the ASO components<br><br> - Participant has any condition that in the opinion of the Site Investigator, would<br> ultimately prevent the completion of study procedures.
Exclusion Criteria
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of participants with treatment-related adverse events as assessed by CTCAE v4.0;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
- 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.