[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Azafaros B.V.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":103},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,44,66,84],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"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":20,"type":21},21,"ESTIMATED","INTERVENTIONAL",[24],"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).",[27,28],"GM2 Gangliosidosis","Niemann-Pick Type C Disease",[30],"Nizubaglustat","RECRUITING","2026-06-17",{"date":34,"type":35},"2026-06-18","ACTUAL",{"date":37,"type":35},"2026-02-04",{"date":39,"type":21},"2030-08-07",{"name":41,"class":42},"Azafaros B.V.","INDUSTRY",3,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":17,"minAge":51,"maxAge":4,"enrollmentInfo":52,"targetDuration":4,"studyType":22,"phases":54,"briefSummary":56,"conditions":57,"keywords":58,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":59,"startDateStruct":60,"completionDateStruct":62,"leadSponsor":64,"locationsCount":65},"100599302","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-npc-100599302","NCT07082725","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 (NPC)","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* Signed informed consent\n* Confirmed diagnosis of NPC disease\n* Patient is unable or unwilling to take miglustat, or is, in the opinion of the investigator, unsatisfactorily treated with miglustat\n* Male and female participants aged 4 years and older at the time of informed consent\n* Onset of neurological symptoms from 2 to 15 years\n* Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline\n* Female 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 NPC disease 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)\n* Current treatment with miglustat, provided the patient has been using the recommended dose for most of the past 12 months AND is, in the opinion of the investigator, satisfactorily treated with miglustat. Any participants receiving miglustat are required to undergo a 1-month washout period before starting study medication","4 Years",{"count":53,"type":21},72,[55],"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",[28],[30],{"date":34,"type":35},{"date":61,"type":35},"2025-06-30",{"date":63,"type":21},"2027-11-04",{"name":41,"class":42},35,{"id":67,"slug":68,"hasResults":11,"nctId":69,"briefTitle":70,"officialTitle":49,"acronym":4,"eligibilityCriteria":71,"healthyVolunteers":11,"sex":17,"minAge":51,"maxAge":4,"enrollmentInfo":72,"targetDuration":4,"studyType":22,"phases":74,"briefSummary":75,"conditions":76,"keywords":78,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":79,"startDateStruct":80,"completionDateStruct":81,"leadSponsor":82,"locationsCount":83},"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","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","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",{"count":73,"type":21},147,[55],"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",[28,77,27],"GM1 Gangliosidosis",[30],{"date":34,"type":35},{"date":61,"type":35},{"date":63,"type":21},{"name":41,"class":42},38,{"id":85,"slug":86,"hasResults":11,"nctId":87,"briefTitle":88,"officialTitle":49,"acronym":4,"eligibilityCriteria":89,"healthyVolunteers":11,"sex":17,"minAge":51,"maxAge":4,"enrollmentInfo":90,"targetDuration":4,"studyType":22,"phases":92,"briefSummary":93,"conditions":94,"keywords":97,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":98,"startDateStruct":99,"completionDateStruct":100,"leadSponsor":101,"locationsCount":102},"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","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","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)",{"count":91,"type":21},75,[55],"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",[95,96],"Gangliosidoses, GM2","Gangliosidosis, GM1",[30],{"date":34,"type":35},{"date":61,"type":35},{"date":63,"type":21},{"name":41,"class":42},36,""]