Status:
ENROLLING_BY_INVITATION
Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
Lead Sponsor:
University of Colorado, Denver
Conditions:
Posterior Column Ataxia with Retinitis Pigmentosa
Eligibility:
All Genders
Phase:
EARLY_PHASE1
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 answe...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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.
Key Trial Info
Start Date :
August 23 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2028
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT06565572
Start Date
August 23 2023
End Date
December 31 2028
Last Update
January 27 2025
Active Locations (1)
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1
Childrens Hospital Colorado
Aurora, Colorado, United States, 80045