[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"gm1-gangliosidosis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:gm1-gangliosidosis":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,44,94],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":29,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100597132","phase-3-a-study-to-evaluate-the-safety-and-efficacy-of-oral-nizubaglustat-az-3102-in-late-infantile-and-juvenile-forms-of-niemann-pick-type-c-disease-gm1-gangliosidosis-or-gm2-gangliosidosis-100597132",false,"NCT07054515","A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis","18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis","Inclusion Criteria:\n\n* Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).\n* Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile\u002F juvenile onset. Detailed inclusion criteria are presented in the GM1\u002FGM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).\n\nExclusion Criteria:\n\n* Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC\n* Detailed exclusion criteria are presented in the GM1\u002FGM2 gangliosidosis-specific subprotocol AZA-001-301-GMx","ALL","4 Years",{"count":19,"type":20},147,"ESTIMATED","INTERVENTIONAL",[23],"PHASE3","An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis",[26,27,28],"Niemann-Pick Type C Disease","GM1 Gangliosidosis","GM2 Gangliosidosis",[30],"Nizubaglustat","RECRUITING","2026-06-17",{"date":34,"type":35},"2026-06-18","ACTUAL",{"date":37,"type":35},"2025-06-30",{"date":39,"type":20},"2027-11-04",{"name":41,"class":42},"Azafaros B.V.","INDUSTRY",38,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":52,"phases":4,"briefSummary":53,"conditions":54,"keywords":58,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":84,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":93},"100107860","a-natural-history-study-of-the-gangliosidoses-100107860","NCT00668187","A Natural History Study of the Gangliosidoses","Inclusion Criteria:\n\n1. Subjects must have a documented gangliosidosis disease.\n2. Subjects must be able to complete appropriate neuropsychological and neurobehavioral assessments.\n3. Late-onset gangliosidosis subjects must be able to tolerate a head MRI.\n\nExclusion Criteria:\n\n1\\. There are no exclusion criteria, beyond a desire not to participate.",{"count":51,"type":20},52,"OBSERVATIONAL","Hypothesis: To characterize and describe disease progression and heterogeneity of the gangliosidosis diseases.\n\nThis research study seeks to develop a quantitative method to delineate disease progression for the gangliosidosis diseases (Tay-Sachs disease, Sandhoff disease, and GM1 gangliosidosis) in order to better understand the natural history and heterogeneity of these diseases. Such a quantitative method will also be essential for evaluating any treatments that may become available in the future, such as gene therapy. The data from this study will be necessary to provide end-points for future therapies, guide medical decisions about treatment, provide objective measurement of treatment outcomes, and accurately inform parents regarding potential outcomes.",[55,56,57,27,28],"Tay-Sachs Disease","Sandhoff Disease","Late Onset Tay-Sachs Disease",[59,60,61,62,63,64,65,66,67,68,69,70,71,72,73,74,75,76,77,78,79,80,81,82],"Tay-Sachs disease","Sandhoff disease","Late Onset Tay-Sachs disease","LOTS","hexosaminidase A deficiency","hexosaminidase A and B deficiency","infantile Tay-Sachs disease","adult-onset Tay-Sachs disease","prospective","natural history","GM1 gangliosidosis","gangliosidoses","β-galactosidase","β-galactosidase deficiency","hexosaminidase","hexosaminidase deficiency","Tay-Sachs","Sandhoff","juvenile Tay-Sachs","juvenile Tay-Sachs disease","late onset Tay-Sachs","juvenile Sandhoff","juvenile Sandhoff disease","GM2 gangliosidosis","2026-03-02",{"date":85,"type":35},"2026-03-04",{"date":87,"type":35},"2010-12",{"date":89,"type":20},"2027-03-01",{"name":91,"class":92},"University of Minnesota","OTHER",1,{"id":95,"slug":96,"hasResults":11,"nctId":97,"briefTitle":98,"officialTitle":98,"acronym":4,"eligibilityCriteria":99,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":100,"targetDuration":4,"studyType":52,"phases":4,"briefSummary":102,"conditions":103,"keywords":120,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":122,"lastUpdatePostDateStruct":123,"startDateStruct":125,"completionDateStruct":127,"leadSponsor":129,"locationsCount":93},"100557519","flower-following-longitudinal-outcomes-with-epidemiology-for-rare-diseases-100557519","NCT06539169","FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases","Inclusion Criteria:\n\n* Any person with a known or suspected rare disease, defined by their prevalence of fewer than 200,000 individuals nationwide. Diseases include but are not limited to:\n\nAlpha- or Beta- Thalassemia Amyloidosis Amyotrophic Lateral Sclerosis (ALS) Creutzfeldt-Jakob disease (CJD) Cystic Fibrosis (CF) Duchenne Muscular Dystrophy (DMD) Early-onset Alzheimer's Disease Ehlers-Danlos Syndrome (EDS) Huntington's Disease (HD) Gaucher Disease GM1 Gangliosidosis Myasthenia Gravis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin Amyloid Polyneuropathy (ATTR-PN)\n\n\\- Patients or their legally-authorized representative must be willing and able to provide informed consent (and assent, if applicable). Deceased persons may participate via consent of their legally-authorized representative in accordance with applicable Federal and state laws\n\nExclusion Criteria:\n\n* Patient or LAR is unable to provide informed consent.\n* Patient resides in a country other than the United States and is unable to provide access to medical records.",{"count":101,"type":20},1000,"FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA\u002FRNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic\u002Fdigitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification.",[104,105,106,107,108,109,110,111,112,113,114,27,115,116,117,118,119],"Alpha-Thalassemia","Beta-Thalassemia","Amyloidosis","Amyotrophic Lateral Sclerosis","Creutzfeld-Jakob Disease","Cystic Fibrosis","Duchenne Muscular Dystrophy","Early-Onset Alzheimer Disease","Ehlers-Danlos Syndrome","Huntington Disease","Gaucher Disease","Myasthenia Gravis","Pompe Disease","Sickle Cell Disease","Transthyretin Amyloid Cardiomyopathy","Rare Diseases",[121],"rare diseases","2024-11-12",{"date":124,"type":35},"2024-11-14",{"date":126,"type":35},"2024-06-10",{"date":128,"type":20},"2026-06-10",{"name":130,"class":42},"xCures"]