[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100647914":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":21,"locations":30,"responsibleParty":96,"collaborators":20,"id":98,"slug":99,"hasResults":100,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":20,"eligibilityCriteria":104,"healthyVolunteers":100,"sex":105,"minAge":106,"maxAge":107,"enrollmentInfo":108,"targetDuration":20,"studyType":111,"phases":112,"briefSummary":114,"conditions":115,"keywords":117,"overallStatus":118,"whyStopped":20,"lastUpdateSubmitDate":119,"lastUpdatePostDateStruct":120,"startDateStruct":123,"completionDateStruct":125,"leadSponsor":127,"locationsCount":128},{"fullName":5,"class":6},"Azafaros B.V.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Nizubaglustat","EXPERIMENTAL","Participants will receive Nizubaglustat (AZ-3102) once daily during Study Period 1 (24 weeks), with a possible extension into Study Period 2 (72 weeks).",[13],"Drug: AZ-3102",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"DRUG","AZ-3102","Daily oral intake of AZ-3102 orodispersible tablets",[9],null,[22,27],{"name":23,"role":24,"phone":25,"phoneExt":20,"email":26},"Patient Advocacy Representative","CONTACT","Please reach out by email","info@azafaros.com",{"name":28,"role":24,"phone":25,"phoneExt":20,"email":29},"Contact for Healthcare Professionals","medinfo@azafaros.com",[31,43,52,58,68,76,87],{"facility":32,"status":20,"city":33,"state":20,"zip":34,"country":35,"countryCode":36,"cosmosGeoPoint":37,"geoPoint":42,"contacts":20},"SMS Medical College Jaipur","Jaipur","302004","India","IN",{"type":38,"coordinates":39},"Point",[40,41],75.78781,26.91962,{"lat":41,"lon":40},{"facility":44,"status":20,"city":45,"state":20,"zip":46,"country":35,"countryCode":36,"cosmosGeoPoint":47,"geoPoint":51,"contacts":20},"Maula a Azad medical college Delhi","New Delhi","110002",{"type":38,"coordinates":48},[49,50],77.2148,28.62137,{"lat":50,"lon":49},{"facility":53,"status":20,"city":45,"state":20,"zip":54,"country":35,"countryCode":36,"cosmosGeoPoint":55,"geoPoint":57,"contacts":20},"Sir Ganga Ram Hospital","110060",{"type":38,"coordinates":56},[49,50],{"lat":50,"lon":49},{"facility":59,"status":20,"city":60,"state":20,"zip":20,"country":61,"countryCode":62,"cosmosGeoPoint":63,"geoPoint":67,"contacts":20},"University of Child Health Sciences and the Children's Hospital","Lahore","Pakistan","PK",{"type":38,"coordinates":64},[65,66],74.35071,31.558,{"lat":66,"lon":65},{"facility":69,"status":20,"city":70,"state":20,"zip":20,"country":61,"countryCode":62,"cosmosGeoPoint":71,"geoPoint":75,"contacts":20},"Clininal Trial Unit, Rehman Medical Institute","Peshawar",{"type":38,"coordinates":72},[73,74],71.57849,34.008,{"lat":74,"lon":73},{"facility":77,"status":20,"city":78,"state":79,"zip":80,"country":81,"countryCode":20,"cosmosGeoPoint":82,"geoPoint":86,"contacts":20},"Ege Universitesi Tip Fakultesi Hastanesi","Bornova","İzmir","35100","Turkey (Türkiye)",{"type":38,"coordinates":83},[84,85],27.2399,38.47921,{"lat":85,"lon":84},{"facility":88,"status":20,"city":89,"state":20,"zip":90,"country":81,"countryCode":20,"cosmosGeoPoint":91,"geoPoint":95,"contacts":20},"Gazi University Medical Faculty Hospital","Yenimahalle","06560",{"type":38,"coordinates":92},[93,94],27.4238,40.22627,{"lat":94,"lon":93},{"type":97,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100647914","phase-2-study-to-evaluate-the-efficacy-and-safety-of-nizubaglustat-az-3102-in-patients-with-gaucher-disease-type-3-gd3-100647914",false,"NCT07715084","Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)","96-week, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)","Inclusion Criteria:\n\n* Have a confirmed diagnosis of GD3 disease\n* Have at least 2 impaired domains in the Modified Severity Scoring Tool (mSST) at Screening\n* Be aged 4-30 years old at the time of informed consent\n* Have the following symptoms of GD3 identified at Screening: GD-related anemia, thrombocytopenia, splenomegaly\n* Female participants of childbearing potential who are sexually active and willing to follow contraceptive guidance\n* Male participants with a female partner of childbearing potential who are willing to follow contraceptive guidance\n\nExclusion Criteria:\n\n* Any condition at Screening or Baseline that, in the opinion of the Principal Investigator, could interfere with study assessments\n* History of medical conditions other than GD3 that, in the opinion of the Principal Investigator, could confound scientific rigor or interpretation of results\n* Body weight \\\u003C10 kg at Screening\n* Presence of a neurologic disease other than GD3\n* Transfusion dependence\n* Prior splenectomy\n* The presence of severe renal impairment at Screening\n* Prior use of an investigational drug within 3 months before Screening\n* Prior participation in a clinical study involving gene therapy or stem cell transplantation\n* Current treatment, or treatment in the last 12 months before Screening, with enzyme replacement therapy (ERT) and\u002For substrate reduction therapy (SRT)\n* A positive serum pregnancy test (for women of childbearing potential)\n* Electrocardiogram (ECG) with an average Fridericia-corrected QT interval (QTcF) of \\>450 msec for males and \\>470 msec for females at Screening\n* Known allergies to azasugars or any study drug excipient","ALL","4 Years","30 Years",{"count":109,"type":110},20,"ESTIMATED","INTERVENTIONAL",[113],"PHASE2","A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).",[116],"Gaucher Disease Type 3",[9],"NOT_YET_RECRUITING","2026-07-15",{"date":121,"type":122},"2026-07-20","ACTUAL",{"date":124,"type":110},"2026-09-28",{"date":126,"type":110},"2029-07",{"name":5,"class":6},7]