[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"alexander-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:alexander-disease":21},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,34,60],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":4,"targetDuration":4,"studyType":18,"phases":4,"briefSummary":19,"conditions":20,"keywords":22,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":28,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":31,"locationsCount":4},"100630416","zilganersen-expanded-access-program-for-individuals-with-alexander-disease-100630416",false,"NCT07487389","Zilganersen Expanded Access Program for Individuals With Alexander Disease","Key Inclusion Criteria:\n\n1. Approved drug or drugs available for treatment did not work for the patient, or the patient cannot tolerate the side effects of the FDA-approved drug or drugs for treatment of AxD.\n2. Patients who are ≥ 2 years old.\n3. Patients who have a clinical phenotype and brain imaging consistent with a diagnosis of AxD.\n4. Patients who have a documented variant in the GFAP gene.\n5. Patient resides in and is a resident of the US.\n\nKey Exclusion Criteria:\n\n1. Patients who have any medical history, physical exam findings, or clinically significant laboratory abnormalities that contraindicate performing an LP for ITB administration of zilganersen.\n2. Patients who have current obstructive hydrocephalus.\n3. Patients who have the presence of a functional ventriculoperitoneal shunt for the drainage of CSF.\n4. Patients who have any other medical history or current conditions, which, in their Treating Physician's opinion, would make the patient unsuitable for inclusion (e.g., active Hepatitis B virus or hepatitis C virus infection, severe hepatic or renal disease, uncontrolled acute or chronic condition, etc.).\n5. Patients who are pregnant or plan to become pregnant, or patients who are breastfeeding.\n\nOther inclusion\u002Fexclusion criteria may apply.","ALL","2 Years","99 Years","EXPANDED_ACCESS","The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).",[21],"Alexander Disease",[23,24,25],"Alexander disease","leukodystrophy","expanded access","AVAILABLE","2026-03-17",{"date":29,"type":30},"2026-03-23","ACTUAL",{"name":32,"class":33},"Ionis Pharmaceuticals, Inc.","INDUSTRY",{"id":35,"slug":36,"hasResults":11,"nctId":37,"briefTitle":38,"officialTitle":38,"acronym":39,"eligibilityCriteria":40,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":41,"targetDuration":4,"studyType":44,"phases":4,"briefSummary":45,"conditions":46,"keywords":47,"overallStatus":48,"whyStopped":4,"lastUpdateSubmitDate":49,"lastUpdatePostDateStruct":50,"startDateStruct":52,"completionDateStruct":54,"leadSponsor":56,"locationsCount":59},"100263905","evaluation-of-outcome-metrics-in-alexander-disease-100263905","NCT02714764","Evaluation of Outcome Metrics in Alexander Disease","AxD Outcomes","Inclusion Criteria:\n\n* Diagnosed with Alexander Disease\n\nExclusion Criteria:\n\n* Other Leukodystrophies will not be enrolled",{"count":42,"type":43},200,"ESTIMATED","OBSERVATIONAL","The purpose of this study is to define the natural history of Alexander Disease, a leukodystrophy that causes neurological dysfunction. Investigators will obtain clinical outcome assessments to measure how the disease affects a patient's gross motor, fine motor, speech and language function, swallowing, and quality of life. Specimens are collected to measure glial fibrillary acidic protein (GFAP) levels in cerebrospinal fluid (CSF) and blood. The data obtained from this study will be used for the design of future treatment trials.",[21],[21],"RECRUITING","2026-01-13",{"date":51,"type":30},"2026-01-15",{"date":53,"type":30},"2016-01-26",{"date":55,"type":43},"2030-12",{"name":57,"class":58},"Children's Hospital of Philadelphia","OTHER",1,{"id":61,"slug":62,"hasResults":11,"nctId":63,"briefTitle":64,"officialTitle":65,"acronym":66,"eligibilityCriteria":67,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":68,"targetDuration":70,"studyType":44,"phases":4,"briefSummary":71,"conditions":72,"keywords":138,"overallStatus":48,"whyStopped":4,"lastUpdateSubmitDate":144,"lastUpdatePostDateStruct":145,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":151,"locationsCount":152},"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":69,"type":43},12000,"10 Years","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.",[73,74,75,76,77,78,79,80,81,82,83,84,85,86,21,87,88,89,90,91,92,93,94,95,96,97,98,99,100,101,102,103,104,105,106,107,108,109,110,111,112,113,114,115,116,117,118,119,120,121,122,123,124,125,126,127,128,129,130,131,132,133,134,135,136,137],"Leukodystrophy","White Matter Disease","Leukoencephalopathies","4H Syndrome","Adrenoleukodystrophy","AMN","ALD","ALD Gene Mutation","ALD (Adrenoleukodystrophy)","X-linked Adrenoleukodystrophy","X-ALD","Adrenomyeloneuropathy","Aicardi Goutieres Syndrome","AGS","Alexanders Leukodystrophy","AxD","ADLD","Canavan Disease","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",[24,139,140,141,142,143],"white matter disease","leukoencephalopathy","myelin","demyelinating","mdbp","2025-10-22",{"date":146,"type":30},"2025-10-23",{"date":148,"type":30},"2016-12-08",{"date":150,"type":43},"2030-12-08",{"name":57,"class":58},23]