[{"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:":90},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,66],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":53,"whyStopped":4,"lastUpdateSubmitDate":54,"lastUpdatePostDateStruct":55,"startDateStruct":58,"completionDateStruct":60,"leadSponsor":62,"locationsCount":65},"100629891","phase-3-safety-and-preliminary-efficacy-of-tsha-102-gene-therapy-in-pediatric-females-aged-2-to-4-years-with-rett-syndrome-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":21,"type":22},3,"ESTIMATED","INTERVENTIONAL",[25],"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.",[28],"Rett Syndrome",[30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50,51,52],"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":56,"type":57},"2026-05-22","ACTUAL",{"date":59,"type":57},"2026-05-08",{"date":61,"type":22},"2031-06",{"name":63,"class":64},"Taysha Gene Therapies, Inc.","INDUSTRY",1,{"id":67,"slug":68,"hasResults":11,"nctId":69,"briefTitle":70,"officialTitle":71,"acronym":4,"eligibilityCriteria":72,"healthyVolunteers":11,"sex":17,"minAge":73,"maxAge":74,"enrollmentInfo":75,"targetDuration":4,"studyType":23,"phases":77,"briefSummary":78,"conditions":79,"keywords":80,"overallStatus":53,"whyStopped":4,"lastUpdateSubmitDate":82,"lastUpdatePostDateStruct":83,"startDateStruct":85,"completionDateStruct":87,"leadSponsor":88,"locationsCount":89},"100485864","phase-3-a-phase-123-study-of-tsha-102-gene-therapy-in-females-with-rett-syndrome-reveal-pivotal-study-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":76,"type":22},15,[25],"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.",[28],[28,81,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50,51,52],"Neurodevelopmental disorder","2025-12-29",{"date":84,"type":57},"2025-12-30",{"date":86,"type":57},"2023-03-06",{"date":61,"type":22},{"name":63,"class":64},6,""]