[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Taysha Gene Therapies, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":88},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,65],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":53,"lastUpdatePostDateStruct":54,"startDateStruct":57,"completionDateStruct":59,"leadSponsor":61,"locationsCount":64},"100629891",false,"NCT07480564","Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to \u003C4 Years With Rett Syndrome","ASPIRE Study: A Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females Aged >2 to \u003C4 Years With Rett Syndrome","ASPIRE","Inclusion Criteria:\n\n* Pediatric females between the ages of 2 and less than 4 years old.\n* Participant has a clinical diagnosis of classic\u002Ftypical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of gene function.\n* Participants must be willing to receive blood or blood products for the treatment of an AE if medically needed.\n* Participants and parent\u002Fcaregiver must agree to reside within easy access to the study site prior to the baseline visit and at least 3 months after TSHA-102 treatment.\n\nExclusion Criteria:\n\n* Participant has another neurodevelopmental disorder independent of the MECP2 loss-of-function mutation, or any other genetic syndrome with a progressive course.\n* Participant has a history of brain injury that causes neurological problems or had grossly abnormal psychomotor development in the first 6 months of life.\n* Participant has a diagnosis of atypical Rett syndrome or a MECP2 gene mutation that does not cause Rett syndrome.\n* Participant requires invasive ventilatory support.\n\nNote: Other protocol defined inclusion\u002Fexclusion criteria may apply","FEMALE","2 Years","3 Years",{"count":20,"type":21},3,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.",[27],"Rett Syndrome",[29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50,51],"Neurodevelopmental Disorders","Rett","MECP2","AAV9","Typical Rett Syndrome","Classic Rett Syndrome","RTT","Rett Disorder","Retts","MECP2-Related Disorder","Gene Therapy","Intrathecal Administration","Genetic Diseases, X-Linked","Nervous System Diseases","Developmental Regression","TSHA-102","miRARE","Self-complementary Vector","Neurologic Manifestations","Intellectual Disability","Pathologic Process","X-Linked Intellectual Disability","Congenital, Hereditary, and Neonatal Diseases and Abnormalities","RECRUITING","2026-05-20",{"date":55,"type":56},"2026-05-22","ACTUAL",{"date":58,"type":56},"2026-05-08",{"date":60,"type":21},"2031-06",{"name":62,"class":63},"Taysha Gene Therapies, Inc.","INDUSTRY",1,{"id":66,"slug":4,"hasResults":10,"nctId":67,"briefTitle":68,"officialTitle":69,"acronym":4,"eligibilityCriteria":70,"healthyVolunteers":10,"sex":16,"minAge":71,"maxAge":72,"enrollmentInfo":73,"targetDuration":4,"studyType":22,"phases":75,"briefSummary":76,"conditions":77,"keywords":78,"overallStatus":52,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":81,"startDateStruct":83,"completionDateStruct":85,"leadSponsor":86,"locationsCount":87},"100485864","NCT05606614","A Phase 1\u002F2\u002F3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)","An Open-label Phase 1\u002F2\u002F3 Study Consisting of a Phase 1\u002F2 Safety and Dose-escalation and Phase 3 Dose-expansion Study to Evaluate Safety and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy in Females With Rett Syndrome","Inclusion Criteria:\n\n* Females between the ages of 12 and \\\u003C22 in Part A (closed) and females between the ages of 6 and \\\u003C22 in Part B (pivotal cohort).\n* Participant has a clinical diagnosis of classic\u002Ftypical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of gene function.\n* Participants must be willing to receive blood or blood products for the treatment of an AE if medically needed.\n* Participants and parent\u002Fcaregiver must agree to reside within easy access to the study site prior to the baseline visit and at least 3 months after TSHA-102 treatment\n\nExclusion Criteria:\n\n* Participant has another neurodevelopmental disorder independent of the MECP2 loss-of-function mutation, or any other genetic syndrome with a progressive course.\n* Participant has a history of brain injury that causes neurological problems or had grossly abnormal psychomotor development in the first 6 months of life.\n* Participant has a diagnosis of atypical Rett syndrome or a MECP2 gene mutation that does not cause Rett syndrome.\n* Participant requires invasive ventilatory support.\n\nNote: Other protocol defined inclusion\u002Fexclusion criteria may apply","6 Years","21 Years",{"count":74,"type":21},15,[24],"The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit\u002Frisk profile based on the totality of safety and efficacy data and to evaluate the efficacy and safety of TSHA-102 at the selected dose.",[27],[27,79,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50,51],"Neurodevelopmental disorder","2025-12-29",{"date":82,"type":56},"2025-12-30",{"date":84,"type":56},"2023-03-06",{"date":60,"type":21},{"name":62,"class":63},6,""]