[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06565572":3,"trial-entities:NCT06565572":73,"trial-summary:NCT06565572":78},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":21,"primary_purpose":22,"phases":23,"enrollment_info":25,"interventions":28,"primary_outcomes":35,"secondary_outcomes":41,"sex":44,"minimum_age":17,"maximum_age":17,"healthy_volunteers":45,"eligibility_criteria":46,"std_ages":55,"locations":59,"central_contacts":69,"overall_officials":70,"references":71,"see_also_links":72},"NCT06565572","22-2313","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","ENROLLING_BY_INVITATION","2028-12-31","2026-05","2026-05-19","2023-08-23","University of Colorado, Denver","OTHER",true,"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.",null,[19],"Posterior Column Ataxia With Retinitis Pigmentosa",[],"INTERVENTIONAL","TREATMENT",[24],"EARLY_PHASE1",{"count":26,"type":27},1,"ESTIMATED",[29],{"type":30,"name":31,"description":32,"armGroupLabels":33},"DRUG","nL-FLVC-001","nL-FLVC-001 is an antisense oligonucleotide that will be injected into the vitreous",[34],"nL-FLVC-001 Arm",[36,39],{"measure":37,"timeFrame":38},"Number of participants with treatment-related adverse events as assessed by CTCAE v4.0","Over 12 months",{"measure":40,"timeFrame":38},"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",[42],{"measure":43,"timeFrame":38},"Measure any changes in the Cardiff Visual Ability Questionnaire for Children (CVAQC-25) in one patient with PCARP after nL-FLVC-001 intravitreal injection.","ALL",false,{"inclusion":47,"exclusion":51,"raw_text":54},[48,49,50],"Informed consent\u002Fassent provided by the participant (when appropriate), and\u002For 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\u002For procedures and provide access to participant's medical records.",[52,53],"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.","Inclusion Criteria:\n\n* Informed consent\u002Fassent provided by the participant (when appropriate), and\u002For participant's parent(s) or legally authorized representative(s).\n* Genetically confirmed FLVCR1-related disease.\n* Ability to travel to the study site and adhere to study-related follow-up examinations and\u002For procedures and provide access to participant's medical records.\n\nExclusion Criteria:\n\n* Allergy to any of the ASO components\n* Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.",[56,57,58],"CHILD","ADULT","OLDER_ADULT",[60],{"facility":61,"city":62,"state":63,"zip":64,"country":65,"geoPoint":66},"Childrens Hospital Colorado","Aurora","Colorado","80045","United States",{"lat":67,"lon":68},39.72943,-104.83192,[],[],[],[],{"nct_id":4,"conditions":74,"biomarkers":76},[75],"Autosomal recessive posterior column ataxia and retinitis pigmentosa",[77],"FLVCR HEME TRANSPORTER 1",{"nct_id":4,"found":45,"summary":17,"prompt_version":17}]