[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"gangliosidoses-gm2\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:gangliosidoses-gm2":43},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"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":28,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100599288","phase-3-a-study-to-evaluate-the-safety-and-efficacy-of-oral-nizubaglustat-az-3102-in-late-infantile-and-juvenile-forms-of-gm1-gangliosidosis-or-gm2-gangliosidosis-100599288",false,"NCT07082543","A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of 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* Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant\n* Male and female participants aged 4 years and older at the time of informed consent\n* Onset of neurological symptoms from 1 to 10 years\n* Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline\n* Females of childbearing potential who are sexually active willing to follow the contraceptive guidance\n* Male participants with a female partner of childbearing potential willing to follow the contraceptive guidance\n\nExclusion Criteria:\n\n* A history of medical conditions other than GM1 or GM2 gangliosidosis that, in the opinion of the Principal Investigator, would confound scientific rigor or the interpretation of results\n* Body weight of \\\u003C10 kg\n* The presence of another neurologic disease\n* The presence of moderate or severe hepatic impairment\n* The presence of moderate or severe renal impairment\n* Platelet count of \\\u003C100x10\\^9\u002FL\n* The dose of any anti-epileptic treatment(s) was not stable (required a change in dose within the previous 3 months) and\u002For a new anti-epileptic treatment (drug or procedure) was prescribed in the month before Baseline\n* Prior use of an investigational drug within the 3 months before Screening; or prior participation in a clinical study involving gene therapy or stem cell transplantation within 2 years prior to Screening\n* A positive serum pregnancy test (for women of childbearing potential)","ALL","4 Years",{"count":19,"type":20},75,"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 GM1 gangliosidosis or GM2 gangliosidosis",[26,27],"Gangliosidoses, GM2","Gangliosidosis, GM1",[29],"Nizubaglustat","RECRUITING","2026-06-17",{"date":33,"type":34},"2026-06-18","ACTUAL",{"date":36,"type":34},"2025-06-30",{"date":38,"type":20},"2027-11-04",{"name":40,"class":41},"Azafaros B.V.","INDUSTRY",36,"Gangliosidoses GM2"]