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

NCT07054515 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: AZA-001-3012024-515778-28-00
• Study Type: interventional
• Target: 147
• Locations: 16 countries
• Sites: 37

Brief Summary

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

Conditions

GM1 Gangliosidosis; GM2 Gangliosidosis; Niemann-Pick Type C Disease

Keywords

Nizubaglustat

Outcome Measures

Primary Outcomes (2)

• Number of participants allocated to subprotocol AZA-001-301-NPC

  Baseline to month 18

• Number of participants allocated to subprotocol AZA-001-301-GMx

  Baseline to month 18

Study Arms (2)

Subprotocol NCT07082725: Intervention Group NPC

EXPERIMENTAL

Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)

Drug: AZ-3102; Drug: Placebo

Subprotocol NCT07082543: Intervention Group GM1/GM2

EXPERIMENTAL

Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)

Drug: AZ-3102; Drug: Placebo

Interventions

AZ-3102 DRUG

Oral dispersible tablets

Also known as: nizubaglustat

Subprotocol NCT07082543: Intervention Group GM1/GM2; Subprotocol NCT07082725: Intervention Group NPC

Placebo DRUG

A matching placebo will be administered in the same regimen as the intervention

Subprotocol NCT07082543: Intervention Group GM1/GM2; Subprotocol NCT07082725: Intervention Group NPC

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

Sponsors & Collaborators

• Azafaros A.G.: lead

Study Sites (37)

UCSF Children's Hospital and Research Center at Oakland

Oakland, California, 94609, United States

RECRUITING

University of Minnesota Medical School

Minneapolis, Minnesota, 55455, United States

NOT YET RECRUITING

Mayo Clinic Childrens Center - PIN

Rochester, Minnesota, 55905, United States

RECRUITING

Children's Medical Center Dallas

Dallas, Texas, 75235, United States

NOT YET RECRUITING

Lysosomal Rare Disorders Research and Treatment Center

Fairfax, Virginia, 22030-7404, United States

RECRUITING

Hospital Universitario Austral

Ciudad Autónoma Buenos Aires, Buenos Aires, B1629AHJ, Argentina

RECRUITING

Hospital de Niños de La Santisima Trinidad

Córdoba, Córdoba Province, X5004 ASL, Argentina

RECRUITING

Women's and Children's Hospital

North Adelaide, South Australia, 5006, Australia

RECRUITING

Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia

NOT YET RECRUITING

Royal Children's Hospital Melbourne - PIN

Parkville, Victoria, 3052, Australia

NOT YET RECRUITING

Instituto Fernandes Figueira

Rio de Janeiro, Rio de Janeiro, 22250-020, Brazil

NOT YET RECRUITING

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

Porto Alegre, Rio Grande do Sul, 90560-030, Brazil

RECRUITING

Hospital Pequeno Príncipe

Curitiba, 80250-060, Brazil

NOT YET RECRUITING

M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary

Calgary, Alberta, T3B 6A8, Canada

NOT YET RECRUITING

University of Alberta Medical Genetics Clinic

Edmonton, Alberta, T6G 2B7, Canada

NOT YET RECRUITING

Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis

Montreal, Quebec, H2X 0C1, Canada

NOT YET RECRUITING

AP-HP - Hôpital Armand Trousseau

Paris, 75012, France

RECRUITING

SphinCS GmbH

Höchheim, 65239, Germany

NOT YET RECRUITING

Amrita Institute of Medical Sciences and Research Centre

Ernākulam, Kerala, 682041, India

RECRUITING

All India Institute of Medical Sciences (AIIMS) - New Delhi

New Delhi, National Capital Territory of Delhi, 110029, India

RECRUITING

JK Lone Hospital

Jaipur, Rajasthan, 302004, India

RECRUITING

Christian Medical College and Hospital

Vellore, Tamil Nadu, 632004, India

NOT YET RECRUITING

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, 20133, Italy

RECRUITING

Hospital Universitario Dr. Jose Eleuterio González

Monterrey, Nuevo León, 64460, Mexico

NOT YET RECRUITING

Centenario Hospital Miguel Hidalgo

Aguascalientes, 20000, Mexico

NOT YET RECRUITING

ULS de Santo António, EPE - Centro Materno Infantil Norte

Porto, Porto District, 4050-651, Portugal

RECRUITING

ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS

Lisbon, 1649-035, Portugal

RECRUITING

Hospital Universitario Vall d'Hebron - PPDS

Barcelona, Barcelona, 8035, Spain

RECRUITING

Hospital Infantil Universitario Niño Jesus - PIN

Madrid, Madrid, 28009, Spain

RECRUITING

Sahlgrenska universitetssjukhuset Östra

Gothenburg, Västra Götaland County, 416 50, Sweden

RECRUITING

Inselspital - Universitätsspital Bern

Bern, Canton of Bern, 3010, Switzerland

RECRUITING

Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi

Adana, Adana, 1250, Turkey (Türkiye)

NOT YET RECRUITING

Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi

Çankaya, Ankara, 6500, Turkey (Türkiye)

RECRUITING

Ege Universitesi Tip Fakultesi

Bornova, İzmir, 35100, Turkey (Türkiye)

NOT YET RECRUITING

Great Ormond Street Hospital

London, London, WC1N 3JH, United Kingdom

NOT YET RECRUITING

University College London Hospitals (UCLH)

London, Middlesex, WC1N 3BG, United Kingdom

NOT YET RECRUITING

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

NOT YET RECRUITING

MeSH Terms

Conditions

Niemann-Pick Disease, Type C; Gangliosidosis, GM1; Gangliosidoses, GM2

Condition Hierarchy (Ancestors)

Niemann-Pick Diseases; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders; Gangliosidoses

Central Study Contacts

Patient Advocacy Representative

CONTACT

Please reach out by email; info@azafaros.com

Contact for Healthcare Professionals

CONTACT

Please reach out by email; medinfo@azafaros.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Participants, investigators, other study personnel at the site, and the Sponsor will be blinded
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 27, 2025
• First Posted: July 8, 2025
• Study Start: June 30, 2025
• Primary Completion (Estimated): November 4, 2027
• Study Completion (Estimated): November 4, 2027
• Last Updated: May 5, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1); FR (1); ES (2); ME (2); PT (2); DE (1); CA (3); CH (1); SE (1); US (5); AR (2); BR (3); TU (3); AU (3); GB (3); IN (4)