Ignite Creation Date:
2026-03-26 @ 3:16 PM
Ignite Modification Date:
2026-03-30 @ 1:16 AM
Study NCT ID:
NCT07399704
Status:
RECRUITING
Last Update Posted:
2026-03-19
First Post:
2025-12-17
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020143', 'term': 'Gangliosidoses, GM2'}, {'id': 'D052556', 'term': 'Niemann-Pick Disease, Type C'}], 'ancestors': [{'id': 'D005733', 'term': 'Gangliosidoses'}, {'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D009542', 'term': 'Niemann-Pick Diseases'}, {'id': 'D015616', 'term': 'Histiocytosis, Non-Langerhans-Cell'}, {'id': 'D015614', 'term': 'Histiocytosis'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 21}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2026-02-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2030-08-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-03-18', 'studyFirstSubmitDate': '2025-12-17', 'studyFirstSubmitQcDate': '2026-02-03', 'lastUpdatePostDateStruct': {'date': '2026-03-19', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-02-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2030-04-15', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change from baseline in treatment-emergent adverse events (TEAEs)', 'timeFrame': 'Through study completion, an average of 4 years', 'description': 'Incidence and severity of all Adverse Events related to study drug treatment, study discontinuation or death'}, {'measure': 'Change from baseline in electrocardiogram (ECG)', 'timeFrame': 'Through study completion, an average of 4 years', 'description': 'ECG read out Normal, Abnormal, Not Clinically Significant, Abnormal, Clinically Significant and Not Done.'}, {'measure': 'Change from baseline in seizures', 'timeFrame': 'Through study completion, an average of 4 years', 'description': 'Seizure duration (minutes) as per the seizure diary.'}, {'measure': 'Change from baseline in seizures', 'timeFrame': 'Through study completion, an average of 4 years', 'description': 'Seizure frequency (number) as per seizure diary.'}, {'measure': 'Maximum observed plasma concentration (Cmax)', 'timeFrame': 'Baseline , Month 1 (Cohort 2 only) and Month 6'}, {'measure': 'Time to Cmax (Tmax)', 'timeFrame': 'Baseline, Month 1 (Cohort 2 only) and Month 6'}, {'measure': 'Concentration at trough (Ctrough)', 'timeFrame': 'Baseline, Month 1 (Cohort 2 only) and Month 6'}, {'measure': 'Area under the plasma concentration-time curve from the time of dosing (zero) to 24 hours post-dose', 'timeFrame': 'Baseline, Month 1 (Cohort 2 only) and Month 6'}], 'secondaryOutcomes': [{'measure': 'Change from Baseline in the concentrations of Glucosylceramide (GlcCer) C16:0; C18:0', 'timeFrame': 'Baseline, Month 1 (Cohort 2 only) and Month 6'}, {'measure': 'Change from Baseline in the concentrations of Neurofilament light chain (NfL)', 'timeFrame': 'Through study completion, an average of 4 years'}, {'measure': 'For GM2 gangliosidosis patients: Change from Baseline in the concentrations of Monosialoganglioside GM2 (GM2)', 'timeFrame': 'Through study completion, an average of 4 years'}, {'measure': 'For GM2 gangliosidosis patients: Change from Baseline in the concentrations of Lyso-monosialoganglioside GM2', 'timeFrame': 'Through study completion, an average of 4 years'}, {'measure': 'For NPC disease patients: Change from Baseline in the concentrations of N-palmitoyl-O-phosphocholine-serine (PPCS)', 'timeFrame': 'Through study completion, an average of 4 years'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Nizubaglustat'], 'conditions': ['GM2 Gangliosidosis', 'Niemann-Pick Type C Disease']}, 'descriptionModule': {'briefSummary': '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).', 'detailedDescription': 'This is a multicenter, open-label study to assess the safety, tolerability, PK, PD, and efficacy of Nizubaglustat in male or female patients with late-infantile or juvenile onset GM2 gangliosidosis or NPC disease in two cohorts:\n\n* Cohort 1: Patients who previously took part in Phase 2 Study AZA-001-5A2-01 (RAINBOW) and wish to continue in this open-label study\n* Cohort 2: Approximately 10 patients with NPC disease, aged ≥12 years who received full-dose Miglustat for more than 12 months, have stable or worsening disease over the 2 previous clinic visits, and who wish to stop Miglustat treatment and transition to Nizubaglustat.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': '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/or 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/condition 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.'}, 'identificationModule': {'nctId': 'NCT07399704', 'acronym': 'PRISMA', 'briefTitle': 'A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Azafaros A.G.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'AZA-001-5A2-02'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'All patients', 'description': 'Arms (both cohorts 1 and 2): Nizubaglustat (AZ-3102)', 'interventionNames': ['Drug: AZ-3102']}], 'interventions': [{'name': 'AZ-3102', 'type': 'DRUG', 'description': 'Daily oral intake of AZ-3102 dispersible tablets', 'armGroupLabels': ['All patients']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80250-060', 'city': 'Água Verde', 'state': 'Curitiba', 'status': 'RECRUITING', 'country': 'Brazil', 'contacts': [{'name': 'Daniel Almeida do Valle', 'role': 'CONTACT', 'email': 'daniel.valle@hpp.org.br', 'phone': '+55 (41) 2108-3861 - option 3'}, {'name': 'Daniel Almeida do Valle', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Associação Hospitalar de Prot à Infância Dr. Raul Carneiro', 'geoPoint': {'lat': -7.36667, 'lon': -41.71667}}, {'zip': '90035-903', 'city': 'Porto Alegre', 'state': 'Rio Grande do Sul', 'status': 'RECRUITING', 'country': 'Brazil', 'contacts': [{'name': 'Roberta Souto', 'role': 'CONTACT', 'email': 'rsouto@hcpa.edu.br', 'phone': '+55 51 9985-0919'}, {'name': 'Roberto Giugliani, Prof. Dr.', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hospital de Clinicas de Porto Alegre', 'geoPoint': {'lat': -30.03283, 'lon': -51.23019}}, {'zip': '22250', 'city': 'Rio de Janeiro', 'status': 'NOT_YET_RECRUITING', 'country': 'Brazil', 'contacts': [{'name': 'Dafne Horovitz', 'role': 'CONTACT', 'email': 'dafne.horovitz@fiocruz.br', 'phone': '+55 21 2554-1709'}, {'name': 'Dafne Horovitz', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira', 'geoPoint': {'lat': -22.90642, 'lon': -43.18223}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Azafaros A.G.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}