[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Timothy Yu\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":47},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":5},"100609502","phase-1-safety-and-efficacy-of-mutation-targeted-precision-genetic-therapy-for-ataxia-telangiectasia-a-t-100609502",false,"NCT07215416","Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)","A Phase 1\u002F2 Study of Antisense Oligonucleotide Therapy for Treatment of Ataxia-Telangiectasia","INCLUSION\u002FEXCLUSION CRITERIA:\n\nWho can take part:\n\n* People with classic A-T confirmed by genetic testing\n* Must have a specific ATM gene change (c.7865C\\>T)\n* Must also have another ATM change that causes A-T\n\nWho cannot take part:\n\nPeople with health problems that make lumbar puncture unsafe:\n\n* Blood clotting or bleeding problems\n* Brain conditions raising pressure inside the head\n* Serious heart or breathing problems\n* Infection near the lower back\n\nOther things doctors will check:\n\n* Overall health and stability\n* Any medicines that might cause problems\n* Past difficulties with lumbar punctures\n* Any other safety concerns","ALL","0 Years","17 Years",{"count":20,"type":21},10,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This project aims to evaluate the safety and efficacy of precision genetic therapy for patients with Ataxia-telangiectasia (A-T), a rare neurodegenerative disease caused by mutations in the ATM gene. The investigators will conduct a clinical trial to study the safety and efficacy of intrathecal administration of atipeksen, a targeted genetic therapy that restores ATM gene function in A-T individuals bearing the recurrent ATM c.7865C\\>T variant. The aim of this study is to delay or forestall progression of neurologic symptoms in A-T and improving quality of life. Success will provide an empirical foundation for advancing additional precision genetic therapies for A-T and other neurodegenerative conditions.",[28],"Ataxia Telangiectasia",[28,30,31,32,33,34],"A-T","ASO","Intrathecal administration","ATM","ATIPEKSEN","RECRUITING","2026-05-26",{"date":38,"type":39},"2026-05-28","ACTUAL",{"date":41,"type":21},"2026-08",{"date":43,"type":21},"2036-12",{"name":45,"class":46},"Timothy Yu","OTHER",""]