[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100289408":3},{"organization":4,"armGroups":7,"interventions":7,"overallOfficials":8,"centralContacts":12,"locations":18,"responsibleParty":310,"collaborators":312,"id":355,"slug":356,"hasResults":357,"nctId":358,"briefTitle":359,"officialTitle":360,"acronym":361,"eligibilityCriteria":362,"healthyVolunteers":357,"sex":363,"minAge":7,"maxAge":7,"enrollmentInfo":364,"targetDuration":367,"studyType":368,"phases":7,"briefSummary":369,"conditions":370,"keywords":437,"overallStatus":21,"whyStopped":7,"lastUpdateSubmitDate":444,"lastUpdatePostDateStruct":445,"startDateStruct":448,"completionDateStruct":450,"leadSponsor":452,"locationsCount":453},{"fullName":5,"class":6},"Children's Hospital of Philadelphia","OTHER",null,[9],{"name":10,"affiliation":5,"role":11},"Adeline Vanderver, MD","PRINCIPAL_INVESTIGATOR",[13],{"name":14,"role":15,"phone":16,"phoneExt":7,"email":17},"Omar S. Sherbini, MPH","CONTACT","215-590-3068","sherbinio@chop.edu",[19,36,48,60,72,84,96,109,122,135,148,161,174,186,199,212,224,243,251,263,276,284,297],{"facility":20,"status":21,"city":22,"state":23,"zip":24,"country":25,"countryCode":26,"cosmosGeoPoint":27,"geoPoint":32,"contacts":33},"Children's Hospital of Los Angeles","RECRUITING","Los Angeles","California","90027","United States","US",{"type":28,"coordinates":29},"Point",[30,31],-118.24368,34.05223,{"lat":31,"lon":30},[34],{"name":35,"role":15,"phone":7,"phoneExt":7,"email":7},"Jonathan Santoro, MD",{"facility":37,"status":21,"city":38,"state":23,"zip":39,"country":25,"countryCode":26,"cosmosGeoPoint":40,"geoPoint":44,"contacts":45},"Children's Hospital of Orange County","Orange","92868",{"type":28,"coordinates":41},[42,43],-117.85311,33.78779,{"lat":43,"lon":42},[46],{"name":47,"role":15,"phone":7,"phoneExt":7,"email":7},"Changrui Xiao, MD",{"facility":49,"status":21,"city":50,"state":23,"zip":51,"country":25,"countryCode":26,"cosmosGeoPoint":52,"geoPoint":56,"contacts":57},"Stanford University (Lucile Packard Children's Hospital)","Palo Alto","94304",{"type":28,"coordinates":53},[54,55],-122.14302,37.44188,{"lat":55,"lon":54},[58],{"name":59,"role":15,"phone":7,"phoneExt":7,"email":7},"Keith Van Haren, MD",{"facility":61,"status":21,"city":62,"state":23,"zip":63,"country":25,"countryCode":26,"cosmosGeoPoint":64,"geoPoint":68,"contacts":69},"University of California, Davis (UC Davis Health)","Sacramento","95817",{"type":28,"coordinates":65},[66,67],-121.4944,38.58157,{"lat":67,"lon":66},[70],{"name":71,"role":15,"phone":7,"phoneExt":7,"email":7},"William Benko, MD",{"facility":73,"status":21,"city":74,"state":23,"zip":75,"country":25,"countryCode":26,"cosmosGeoPoint":76,"geoPoint":80,"contacts":81},"University of California, San Diego (Rady Children's Hospital)","San Diego","92123",{"type":28,"coordinates":77},[78,79],-117.16472,32.71571,{"lat":79,"lon":78},[82],{"name":83,"role":15,"phone":7,"phoneExt":7,"email":7},"Jennifer Yang, MD",{"facility":85,"status":21,"city":86,"state":23,"zip":87,"country":25,"countryCode":26,"cosmosGeoPoint":88,"geoPoint":92,"contacts":93},"UCSF Benioff Children's Hospital","San Francisco","94158",{"type":28,"coordinates":89},[90,91],-122.41942,37.77493,{"lat":91,"lon":90},[94],{"name":95,"role":15,"phone":7,"phoneExt":7,"email":7},"Alexander Fay, MD, PhD",{"facility":97,"status":21,"city":98,"state":99,"zip":100,"country":25,"countryCode":26,"cosmosGeoPoint":101,"geoPoint":105,"contacts":106},"Children's National Medical Center","Washington D.C.","District of Columbia","20010",{"type":28,"coordinates":102},[103,104],-77.03637,38.89511,{"lat":104,"lon":103},[107],{"name":108,"role":15,"phone":7,"phoneExt":7,"email":7},"Jamie Fraser, MD, PhD",{"facility":110,"status":21,"city":111,"state":112,"zip":113,"country":25,"countryCode":26,"cosmosGeoPoint":114,"geoPoint":118,"contacts":119},"Emory University (Children's Healthcare of Atlanta)","Atlanta","Georgia","30342",{"type":28,"coordinates":115},[116,117],-84.38798,33.749,{"lat":117,"lon":116},[120],{"name":121,"role":15,"phone":7,"phoneExt":7,"email":7},"Stephanie Keller, MD",{"facility":123,"status":21,"city":124,"state":125,"zip":126,"country":25,"countryCode":26,"cosmosGeoPoint":127,"geoPoint":131,"contacts":132},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"type":28,"coordinates":128},[129,130],-87.65005,41.85003,{"lat":130,"lon":129},[133],{"name":134,"role":15,"phone":7,"phoneExt":7,"email":7},"Jennifer P Rubin, MD",{"facility":136,"status":21,"city":137,"state":138,"zip":139,"country":25,"countryCode":26,"cosmosGeoPoint":140,"geoPoint":144,"contacts":145},"Kennedy Krieger Institute","Baltimore","Maryland","21205",{"type":28,"coordinates":141},[142,143],-76.61219,39.29038,{"lat":143,"lon":142},[146],{"name":147,"role":15,"phone":7,"phoneExt":7,"email":7},"Ali Fatemi, MD, MBA",{"facility":149,"status":21,"city":150,"state":151,"zip":152,"country":25,"countryCode":26,"cosmosGeoPoint":153,"geoPoint":157,"contacts":158},"Massachusetts General Hospital (MGH)","Boston","Massachusetts","02114",{"type":28,"coordinates":154},[155,156],-71.05977,42.35843,{"lat":156,"lon":155},[159],{"name":160,"role":15,"phone":7,"phoneExt":7,"email":7},"Florian Eichler, MD",{"facility":162,"status":21,"city":163,"state":164,"zip":165,"country":25,"countryCode":26,"cosmosGeoPoint":166,"geoPoint":170,"contacts":171},"University of Minnesota","Minneapolis","Minnesota","55454",{"type":28,"coordinates":167},[168,169],-93.26384,44.97997,{"lat":169,"lon":168},[172],{"name":173,"role":15,"phone":7,"phoneExt":7,"email":7},"Paul Orchard, MD",{"facility":175,"status":21,"city":176,"state":164,"zip":177,"country":25,"countryCode":26,"cosmosGeoPoint":178,"geoPoint":182,"contacts":183},"Mayo Clinic","Rochester","55905",{"type":28,"coordinates":179},[180,181],-92.4699,44.02163,{"lat":181,"lon":180},[184],{"name":185,"role":15,"phone":7,"phoneExt":7,"email":7},"Radhika Dhamija, MBBS",{"facility":187,"status":21,"city":188,"state":189,"zip":190,"country":25,"countryCode":26,"cosmosGeoPoint":191,"geoPoint":195,"contacts":196},"Atrium Health Wake Forest Baptist","Winston-Salem","North Carolina","27157",{"type":28,"coordinates":192},[193,194],-80.24422,36.09986,{"lat":194,"lon":193},[197],{"name":198,"role":15,"phone":7,"phoneExt":7,"email":7},"Jennifer Harmon, MD, PhD",{"facility":200,"status":21,"city":201,"state":202,"zip":203,"country":25,"countryCode":26,"cosmosGeoPoint":204,"geoPoint":208,"contacts":209},"Akron Children's Hospital","Akron","Ohio","44308",{"type":28,"coordinates":205},[206,207],-81.51901,41.08144,{"lat":207,"lon":206},[210],{"name":211,"role":15,"phone":7,"phoneExt":7,"email":7},"Matthew Ginsberg, MD",{"facility":213,"status":21,"city":214,"state":202,"zip":215,"country":25,"countryCode":26,"cosmosGeoPoint":216,"geoPoint":220,"contacts":221},"Nationwide Children's Hospital","Columbus","43205",{"type":28,"coordinates":217},[218,219],-82.99879,39.96118,{"lat":219,"lon":218},[222],{"name":223,"role":15,"phone":7,"phoneExt":7,"email":7},"Margie Ream, MD, PhD",{"facility":225,"status":21,"city":226,"state":227,"zip":228,"country":25,"countryCode":26,"cosmosGeoPoint":229,"geoPoint":233,"contacts":234},"The Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"type":28,"coordinates":230},[231,232],-75.16362,39.95238,{"lat":232,"lon":231},[235,237,238,241],{"name":236,"role":15,"phone":16,"phoneExt":7,"email":17},"Omar Sherbini, MPH",{"name":10,"role":11,"phone":7,"phoneExt":7,"email":7},{"name":239,"role":240,"phone":7,"phoneExt":7,"email":7},"Laura Adang, MD, PhD, MSTR","SUB_INVESTIGATOR",{"name":242,"role":240,"phone":7,"phoneExt":7,"email":7},"Amy Waldman, MD, MSCE",{"facility":244,"status":21,"city":226,"state":227,"zip":228,"country":25,"countryCode":26,"cosmosGeoPoint":245,"geoPoint":247,"contacts":248},"University of Pennsylvania",{"type":28,"coordinates":246},[231,232],{"lat":232,"lon":231},[249],{"name":250,"role":15,"phone":7,"phoneExt":7,"email":7},"Jennifer Orthmann-Murphy, MD, PhD",{"facility":252,"status":21,"city":253,"state":227,"zip":254,"country":25,"countryCode":26,"cosmosGeoPoint":255,"geoPoint":259,"contacts":260},"University of Pittsburgh Medical Center","Pittsburgh","15219",{"type":28,"coordinates":256},[257,258],-79.99589,40.44062,{"lat":258,"lon":257},[261],{"name":262,"role":15,"phone":7,"phoneExt":7,"email":7},"Deepa Rajan, MD",{"facility":264,"status":21,"city":265,"state":266,"zip":267,"country":25,"countryCode":26,"cosmosGeoPoint":268,"geoPoint":272,"contacts":273},"Baylor College of Medicine (Texas Children's Hospital)","Houston","Texas","77030",{"type":28,"coordinates":269},[270,271],-95.36327,29.76328,{"lat":271,"lon":270},[274],{"name":275,"role":15,"phone":7,"phoneExt":7,"email":7},"Lisa Emrick, MD",{"facility":277,"status":21,"city":265,"state":266,"zip":7,"country":25,"countryCode":26,"cosmosGeoPoint":278,"geoPoint":280,"contacts":281},"UT Health Houston",{"type":28,"coordinates":279},[270,271],{"lat":271,"lon":270},[282],{"name":283,"role":15,"phone":7,"phoneExt":7,"email":7},"Mary Kay Koenig, MD",{"facility":285,"status":21,"city":286,"state":287,"zip":288,"country":25,"countryCode":26,"cosmosGeoPoint":289,"geoPoint":293,"contacts":294},"University of Utah (Primary Children's Hospital)","Salt Lake City","Utah","84112",{"type":28,"coordinates":290},[291,292],-111.89105,40.76078,{"lat":292,"lon":291},[295],{"name":296,"role":15,"phone":7,"phoneExt":7,"email":7},"Joshua Bonkowsky, MD, PhD",{"facility":298,"status":21,"city":299,"state":300,"zip":301,"country":25,"countryCode":26,"cosmosGeoPoint":302,"geoPoint":306,"contacts":307},"Seattle Children's Hospital","Seattle","Washington","98105",{"type":28,"coordinates":303},[304,305],-122.33207,47.60621,{"lat":305,"lon":304},[308],{"name":309,"role":15,"phone":7,"phoneExt":7,"email":7},"Emily Shelkowitz, MD",{"type":11,"investigatorFullName":10,"investigatorTitle":311,"investigatorAffiliation":5,"oldNameTitle":7,"oldOrganization":7},"Program Director, Leukodystrophy Center",[313,316,318,320,323,325,327,330,332,334,336,338,340,342,344,346,347,349,351,353],{"name":314,"class":315},"National Institutes of Health (NIH)","NIH",{"name":317,"class":315},"National Institute of Neurological Disorders and Stroke (NINDS)",{"name":319,"class":315},"National Center for Advancing Translational Sciences (NCATS)",{"name":321,"class":322},"Biogen","INDUSTRY",{"name":324,"class":322},"Eli Lilly and Company",{"name":326,"class":322},"Myrtelle Inc.",{"name":328,"class":329},"Orchard Therapeutics Ltd.","UNKNOWN",{"name":331,"class":322},"Passage Bio, Inc.",{"name":333,"class":329},"Synaptix Biotherapeutics Ltd.",{"name":335,"class":322},"Takeda",{"name":337,"class":322},"Boehringer Ingelheim",{"name":339,"class":322},"Ionis Pharmaceuticals, Inc.",{"name":341,"class":329},"Sanofi Winthrop Industrie",{"name":343,"class":322},"Sana Biotechnology",{"name":345,"class":329},"Yaya Foundation for 4H Leukodystrophy",{"name":244,"class":6},{"name":348,"class":329},"United MSD Foundation",{"name":350,"class":329},"Foundation to Fight H-ABC",{"name":352,"class":329},"Calliope Joy Foundation",{"name":354,"class":329},"Don't Forget Me Foundation","100289408","the-myelin-disorders-biorepository-project-100289408",false,"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.","ALL",{"count":365,"type":366},12000,"ESTIMATED","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.",[371,372,373,374,375,376,377,378,379,380,381,382,383,384,385,386,387,388,389,390,391,392,393,394,395,396,397,398,399,400,401,402,403,404,405,406,407,408,409,410,411,412,413,414,415,416,417,418,419,420,421,422,423,424,425,426,427,428,429,430,431,432,433,434,435,436],"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","Alexander Disease","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 &#X7C; Blood or Tissue &#X7C; 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",[438,439,440,441,442,443],"leukodystrophy","white matter disease","leukoencephalopathy","myelin","demyelinating","mdbp","2025-10-22",{"date":446,"type":447},"2025-10-23","ACTUAL",{"date":449,"type":447},"2016-12-08",{"date":451,"type":366},"2030-12-08",{"name":5,"class":6},23]