[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100623674":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":20,"locations":21,"responsibleParty":76,"collaborators":20,"id":78,"slug":79,"hasResults":80,"nctId":81,"briefTitle":82,"officialTitle":83,"acronym":84,"eligibilityCriteria":85,"healthyVolunteers":80,"sex":86,"minAge":87,"maxAge":20,"enrollmentInfo":88,"targetDuration":20,"studyType":91,"phases":92,"briefSummary":94,"conditions":95,"keywords":98,"overallStatus":24,"whyStopped":20,"lastUpdateSubmitDate":100,"lastUpdatePostDateStruct":101,"startDateStruct":104,"completionDateStruct":106,"leadSponsor":108,"locationsCount":109},{"fullName":5,"class":6},"Azafaros B.V.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"All patients","EXPERIMENTAL","Arms (both cohorts 1 and 2): Nizubaglustat (AZ-3102)",[13],"Drug: AZ-3102",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"DRUG","AZ-3102","Daily oral intake of AZ-3102 dispersible tablets",[9],null,[22,44,61],{"facility":23,"status":24,"city":25,"state":26,"zip":27,"country":28,"countryCode":29,"cosmosGeoPoint":30,"geoPoint":35,"contacts":36},"Associação Hospitalar de Prot à Infância Dr. Raul Carneiro","RECRUITING","Água Verde","Curitiba","80250-060","Brazil","BR",{"type":31,"coordinates":32},"Point",[33,34],-49.882,-23.367,{"lat":34,"lon":33},[37,42],{"name":38,"role":39,"phone":40,"phoneExt":20,"email":41},"Daniel Almeida do Valle","CONTACT","+55 (41) 2108-3861 - option 3","daniel.valle@hpp.org.br",{"name":38,"role":43,"phone":20,"phoneExt":20,"email":20},"PRINCIPAL_INVESTIGATOR",{"facility":45,"status":24,"city":46,"state":47,"zip":48,"country":28,"countryCode":29,"cosmosGeoPoint":49,"geoPoint":53,"contacts":54},"Hospital de Clinicas de Porto Alegre","Porto Alegre","Rio Grande do Sul","90035-903",{"type":31,"coordinates":50},[51,52],-51.23019,-30.03283,{"lat":52,"lon":51},[55,59],{"name":56,"role":39,"phone":57,"phoneExt":20,"email":58},"Roberta Souto","+55 51 9985-0919","rsouto@hcpa.edu.br",{"name":60,"role":43,"phone":20,"phoneExt":20,"email":20},"Roberto Giugliani, Prof. Dr.",{"facility":62,"status":24,"city":63,"state":20,"zip":64,"country":28,"countryCode":29,"cosmosGeoPoint":65,"geoPoint":69,"contacts":70},"Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira","Rio de Janeiro","22250",{"type":31,"coordinates":66},[67,68],-43.18223,-22.90642,{"lat":68,"lon":67},[71,75],{"name":72,"role":39,"phone":73,"phoneExt":20,"email":74},"Dafne Horovitz","+55 21 2554-1709","dafne.horovitz@fiocruz.br",{"name":72,"role":43,"phone":20,"phoneExt":20,"email":20},{"type":77,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100623674","phase-2-a-study-to-evaluate-the-safety-and-efficacy-of-nizubaglustat-az-3102-in-patients-with-gm2-gangliosidosis-or-niemann-pick-type-c-disease-100623674",false,"NCT07399704","A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease","Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat","PRISMA","Inclusion Criteria:\n\n* Cohort 1 (NPC and GM2 patients):\n\n  * Have been randomized into Phase 2 Study AZA-001-5A2-01.\n\nOR\n\nCohort 2 (NPC patients):\n\n* Be male or female aged ≥12 years\n* Have a genetically-confirmed diagnosis of NPC disease\n* Have received full-dose Miglustat treatment for at least 12 months and experienced disease stabilization or worsening with treatment over the 2 previous clinic visits. Patients experiencing clinical improvement with Miglustat over the preceding 3 months should not be considered for this study.\n* Wish to change treatment to Nizubaglustat for their NPC disease.\n* Participants from Phase 2 Study AZA-001-5A2-01 (RAINBOW) who transitioned to Miglustat may be eligible for Cohort 2 if they meet all other criteria.\n\nParticipation is supported and deemed beneficial by the Principal Investigator. Be willing and able to be evaluated for all protocol assessments. The participant, parent, and\u002For legal guardian can read, understand, and sign the informed consent form. Where appropriate, assent will also be sought for participants who have not reached the age of majority.\n\nExclusion Criteria:\n\n* A positive serum pregnancy test (only tested for women of childbearing potential).\n* Female planning to breastfeed during the study.\n* Any medical event\u002Fcondition that prevents participation in the study based on the judgment of the Principal Investigator.\n* Participation in another interventional or non-interventional study or early access program.","ALL","12 Years",{"count":89,"type":90},21,"ESTIMATED","INTERVENTIONAL",[93],"PHASE2","This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study (Cohort 1). In addition, the study aims to assess safety, clinical, and biochemical impact of transitioning NPC disease patients to Nizubaglustat after prior treatment with stable, full-dose Miglustat (Cohort 2).",[96,97],"GM2 Gangliosidosis","Niemann-Pick Type C Disease",[99],"Nizubaglustat","2026-06-17",{"date":102,"type":103},"2026-06-18","ACTUAL",{"date":105,"type":103},"2026-02-04",{"date":107,"type":90},"2030-08-07",{"name":5,"class":6},3]