[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"canavan-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:canavan-disease":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,61],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":48,"whyStopped":4,"lastUpdateSubmitDate":49,"lastUpdatePostDateStruct":50,"startDateStruct":53,"completionDateStruct":55,"leadSponsor":57,"locationsCount":60},"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":19,"type":20},26,"ESTIMATED","INTERVENTIONAL",[23,24],"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.",[27],"Canavan Disease",[27,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47],"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","RECRUITING","2026-04-15",{"date":51,"type":52},"2026-04-17","ACTUAL",{"date":54,"type":52},"2021-09-08",{"date":56,"type":20},"2032-10-08",{"name":58,"class":59},"Aspa Therapeutics","INDUSTRY",4,{"id":62,"slug":63,"hasResults":11,"nctId":64,"briefTitle":65,"officialTitle":66,"acronym":67,"eligibilityCriteria":68,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":69,"targetDuration":71,"studyType":72,"phases":4,"briefSummary":73,"conditions":74,"keywords":138,"overallStatus":48,"whyStopped":4,"lastUpdateSubmitDate":145,"lastUpdatePostDateStruct":146,"startDateStruct":148,"completionDateStruct":150,"leadSponsor":152,"locationsCount":155},"100289408","the-myelin-disorders-biorepository-project-100289408","NCT03047369","The Myelin Disorders Biorepository Project","The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network","MDBP","Inclusion Criteria (Affected Subjects):\n\n* Male or female of any age;\n* Suspected or confirmed diagnosis of leukodystrophy or other disorder affecting the white matter of the brain based primarily on the finding of central nervous system neuroimaging consistent with this diagnosis or on an existing diagnosis of a leukodystrophy or genetic leukoencephalopathy as defined in existing classification systems, or in the presence of variant(s) of uncertain significance or genotype consistent with leukodytrophy;\n* Documentation of informed consent by the subject, parent, or legal guardian, and, if appropriate, documentation of assent;\n* Willingness to provide clinical data, participate in standardized assessments, and\u002For provide biologic samples.\n\nExclusion Criteria (Affected Subjects)\n\n* Established diagnosis at the time of referral that is not consistent with a genetic disorder of the white matter, such as an acquired demyelinating condition (e.g. multiple sclerosis), or an infectious etiology, with the exception of sequelae of congenital infections such as CMV;\n* Inability to provide consent.\n\nInclusion Criteria (Healthy Controls)\n\n* Male or female of any age;\n* Individuals with no confirmed or suspected diagnosis of leukodystrophy or other disorder affecting the white matter of the brain (including affected patients' caregivers);\n* Documentation of informed consent by the subject, parent, or legal guardian, and, if appropriate, documentation of assent.\n\nExclusion Criteria (Healthy Controls)\n\n\\- Inability to provide consent.",{"count":70,"type":20},12000,"10 Years","OBSERVATIONAL","The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago.\n\nResearchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.",[43,75,44,76,77,78,79,80,81,82,83,84,85,86,87,88,89,90,27,91,92,93,94,95,96,97,98,99,100,101,102,103,104,105,106,107,108,109,110,111,112,113,114,115,116,117,118,119,120,121,122,123,124,125,126,127,128,129,130,131,132,133,134,135,136,137],"White Matter Disease","4H Syndrome","Adrenoleukodystrophy","AMN","ALD","ALD Gene Mutation","ALD (Adrenoleukodystrophy)","X-linked Adrenoleukodystrophy","X-ALD","Adrenomyeloneuropathy","Aicardi Goutieres Syndrome","AGS","Alexander Disease","Alexanders Leukodystrophy","AxD","ADLD","CTX","Cerebrotendinous Xanthomatoses","Krabbe Disease","GALC Deficiency","Globoid Leukodystrophy","TUBB4A-Related Leukodystrophy","H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum","HBSL","HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity","LBSL","Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)","Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation","ALSP","CSF1R Gene Mutation","HCC - Hypomyelination and Congenital Cataract","MLC1","Megalencephalic Leukoencephalopathy With Subcortical Cysts","MLD","Metachromatic Leukodystrophy","PMD","Pelizaeus-Merzbacher Disease","PLP1 Null Syndrome","PLP1 Gene Duplication | Blood or Tissue | Mutations","Pelizaeus Merzbacher Like Disease","Peroxisomal Biogenesis Disorder","Zellweger Syndrome","Refsum Disease","Salla Disease","Sialic Storage Disease","Sjögren","Sjogren-Larsson Syndrome","Van Der Knapp Disease","Vanishing White Matter Disease","Charcot-Marie-Tooth","CMT","Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency","Allan-Herndon-Dudley Syndrome","Cadasil","Cockayne Syndrome","Multiple Sulfatase Deficiency","Gangliosidoses","GM2 Gangliosidosis","BPAN","Labrune Syndrome","LCC","Mucopolysaccharidoses","TBCK-Related Intellectual Disability Syndrome",[139,140,141,142,143,144],"leukodystrophy","white matter disease","leukoencephalopathy","myelin","demyelinating","mdbp","2025-10-22",{"date":147,"type":52},"2025-10-23",{"date":149,"type":52},"2016-12-08",{"date":151,"type":20},"2030-12-08",{"name":153,"class":154},"Children's Hospital of Philadelphia","OTHER",23]