[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100439138":3},{"organization":4,"armGroups":7,"interventions":22,"overallOfficials":28,"centralContacts":29,"locations":37,"responsibleParty":111,"collaborators":28,"id":113,"slug":114,"hasResults":115,"nctId":116,"briefTitle":117,"officialTitle":118,"acronym":28,"eligibilityCriteria":119,"healthyVolunteers":115,"sex":120,"minAge":28,"maxAge":121,"enrollmentInfo":122,"targetDuration":28,"studyType":125,"phases":126,"briefSummary":129,"conditions":130,"keywords":132,"overallStatus":40,"whyStopped":28,"lastUpdateSubmitDate":152,"lastUpdatePostDateStruct":153,"startDateStruct":156,"completionDateStruct":158,"leadSponsor":160,"locationsCount":161},{"fullName":5,"class":6},"Aspa Therapeutics","INDUSTRY",[8,14,18],{"label":9,"type":10,"description":11,"interventionNames":12},"Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1)","EXPERIMENTAL","Participants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study.",[13],"Biological: AAV9 BBP-812",{"label":15,"type":10,"description":16,"interventionNames":17},"Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2)","Participants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study.",[13],{"label":19,"type":10,"description":20,"interventionNames":21},"Enrollment Expansion Phase: BBP-812","Participants will receive a single IV infusion of BBP-812 at the selected dose from the dose-finding phase on Day 0 in expansion phase of the study.",[13],[23],{"type":24,"name":25,"description":26,"armGroupLabels":27,"otherNames":28},"BIOLOGICAL","AAV9 BBP-812","Sterile solution for injection for 1-time use via volumetric infusion pump",[9,15,19],null,[30,35],{"name":31,"role":32,"phone":33,"phoneExt":28,"email":34},"Alicia Gomez","CONTACT","833-764-2267 or 617-861-4617","CANaspire@aspatx.com",{"name":36,"role":32,"phone":28,"phoneExt":28,"email":28},"clinicaltrials@aspatx.com",[38,64,81,101],{"facility":39,"status":40,"city":41,"state":42,"zip":43,"country":44,"countryCode":45,"cosmosGeoPoint":46,"geoPoint":51,"contacts":52},"UCSF Benioff Children's Hospital Oakland","RECRUITING","Oakland","California","94609","United States","US",{"type":47,"coordinates":48},"Point",[49,50],-122.2708,37.80437,{"lat":50,"lon":49},[53,58,61],{"name":54,"role":32,"phone":55,"phoneExt":56,"email":57},"Marissa Evans","510-428-3885","5421","Marissa.Evans@ucsf.edu",{"name":59,"role":32,"phone":55,"phoneExt":56,"email":60},"Grace Sobrero","grace.sobrero@ucsf.edu",{"name":62,"role":63,"phone":28,"phoneExt":28,"email":28},"Alexander Fay, MD","PRINCIPAL_INVESTIGATOR",{"facility":65,"status":40,"city":66,"state":67,"zip":68,"country":44,"countryCode":45,"cosmosGeoPoint":69,"geoPoint":73,"contacts":74},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"type":47,"coordinates":70},[71,72],-87.65005,41.85003,{"lat":72,"lon":71},[75,79],{"name":76,"role":32,"phone":77,"phoneExt":28,"email":78},"Prachi Patel","312-227-0067","prachipatel@luriechildrens.org",{"name":80,"role":63,"phone":28,"phoneExt":28,"email":28},"Jennifer Rubin, MD",{"facility":82,"status":40,"city":83,"state":84,"zip":85,"country":44,"countryCode":45,"cosmosGeoPoint":86,"geoPoint":90,"contacts":91},"Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)","Boston","Massachusetts","02114",{"type":47,"coordinates":87},[88,89],-71.05977,42.35843,{"lat":89,"lon":88},[92,96,99],{"name":93,"role":32,"phone":94,"phoneExt":28,"email":95},"Sam Kartsonis","610-766-2583","skartsonis@mgh.harvard.edu",{"name":97,"role":32,"phone":28,"phoneExt":28,"email":98},"Aliza (Hattie) Wilson","awilson65@bwh.harvard.edu",{"name":100,"role":63,"phone":28,"phoneExt":28,"email":28},"Florian Eichler, MD",{"facility":102,"status":103,"city":104,"state":104,"zip":105,"country":44,"countryCode":45,"cosmosGeoPoint":106,"geoPoint":110,"contacts":28},"Weill Cornell Medicine; Division of Pediatric Neurology","COMPLETED","New York","10065",{"type":47,"coordinates":107},[108,109],-74.00597,40.71427,{"lat":109,"lon":108},{"type":112,"investigatorFullName":28,"investigatorTitle":28,"investigatorAffiliation":28,"oldNameTitle":28,"oldOrganization":28},"SPONSOR","100439138","phase-1-a-study-of-aav9-gene-therapy-in-participants-with-canavan-disease-canaspire-clinical-trial-100439138",false,"NCT04998396","A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)","A Phase 1\u002F2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA Gene","Key Inclusion Criteria:\n\n* Maximum age for inclusion is 30 months.\n* Participant has stable health in the opinion of the investigator and as confirmed by medical history and laboratory studies with no acute or chronic hematologic, renal, liver, immunologic, or neurologic disease (other than Canavan disease).\n* Participant has biochemical, genetic, and clinical diagnosis of Canavan disease:\n\n  * Elevated urinary NAA and\n  * Biallelic mutation of the ASPA gene determined at Screening or documented in the participant's medical history.\n  * Active clinical signs of Canavan disease\n* Participant is up to date on all immunizations per local guidelines\n\nKey Exclusion Criteria:\n\n* Tests positive for total anti-AAV9 antibodies determined by enzyme-linked immunosorbent assay (ELISA).\n* Received prior gene therapy or other therapy (including vaccines) involving AAV.\n* Participant is receiving high-dose therapy with immunosuppressants.\n* Participant has significantly progressed Canavan disease characterized as:\n\n  * Presence of continuous\u002Fconstant decerebrate or decorticate posturing,\n  * Recurrent status epilepticus, or\n  * Recalcitrant seizures that do not respond while on 3 or more anti-epileptic medications","ALL","30 Months",{"count":123,"type":124},26,"ESTIMATED","INTERVENTIONAL",[127,128],"PHASE1","PHASE2","The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.",[131],"Canavan Disease",[131,133,134,135,136,137,138,139,140,141,142,143,144,145,146,147,148,149,150,151],"AAV","AAV9","Gene therapy","Aspartoacylase","ASPA","ASPA gene","rAAV9","ACY2","Aminoacylase 2","Spongy degeneration","N-acetyl-L-aspartic acid (NAA)","N-acetylaspartate","Rare disease","Inherited Metabolic Disorders","Leukodystrophy","Leukoencephalopathies","Autosomal Recessive Disorder","Neurodevelopmental diseases","CANaspire Clinical Trial","2026-04-15",{"date":154,"type":155},"2026-04-17","ACTUAL",{"date":157,"type":155},"2021-09-08",{"date":159,"type":124},"2032-10-08",{"name":5,"class":6},4]