RECRUITING Phase 3 ClinicalTrials.gov

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

Sponsor: Azafaros B.V. • ClinicalTrials.gov ID: NCT07054515

Registry updated: 2026-09-24

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Description

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

Interventions

AZ-3102 (Drug)
Oral dispersible tablets
Placebo (Drug)
A matching placebo will be administered in the same regimen as the intervention

Eligibility criteria

Inclusion Criteria: * 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). * 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/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543). Exclusion Criteria: * Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC * Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx

Conditions

Niemann-Pick Type C Disease GM1 Gangliosidosis GM2 Gangliosidosis

Locations

UCSF Children's Hospital and Research Center at Oakland — Oakland, California, United States
University of Minnesota Medical School — Minneapolis, Minnesota, United States
Mayo Clinic Childrens Center - PIN — Rochester, Minnesota, United States
Children's Medical Center Dallas — Dallas, Texas, United States
Lysosomal Rare Disorders Research and Treatment Center — Fairfax, Virginia, United States
Hospital Universitario Austral — Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina
Hospital de Niños de La Santisima Trinidad — Córdoba, Córdoba Province, Argentina
Women's and Children's Hospital — North Adelaide, South Australia, Australia
Royal Melbourne Hospital — Parkville, Victoria, Australia
Royal Children's Hospital Melbourne - PIN — Parkville, Victoria, Australia
Instituto Fernandes Figueira — Rio de Janeiro, Rio de Janeiro, Brazil
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS — Porto Alegre, Rio Grande do Sul, Brazil
Hospital Pequeno Príncipe — Curitiba, Brazil
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary — Calgary, Alberta, Canada
University of Alberta Medical Genetics Clinic — Edmonton, Alberta, Canada
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis — Montreal, Quebec, Canada
AP-HP - Hôpital Armand Trousseau — Paris, France
AP-HP - Hôpital de la Pitié Salpétrière — Paris, France
SphinCS GmbH — Höchheim, Germany
Amrita Institute of Medical Sciences and Research Centre — Ernākulam, Kerala, India
All India Institute of Medical Sciences (AIIMS) - New Delhi — New Delhi, National Capital Territory of Delhi, India
JK Lone Hospital — Jaipur, Rajasthan, India
Christian Medical College and Hospital — Vellore, Tamil Nadu, India
Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan, Italy
Hospital Universitario Dr. Jose Eleuterio González — Monterrey, Nuevo León, Mexico
Centenario Hospital Miguel Hidalgo — Aguascalientes, Mexico
The Children's Hospital & the University of Child Health Lahore — Lahore, Punjab Province, Pakistan
ULS de Santo António, EPE - Centro Materno Infantil Norte — Porto, Porto District, Portugal
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS — Lisbon, Portugal
Hospital Universitario Vall d'Hebron - PPDS — Barcelona, Barcelona, Spain
Hospital Infantil Universitario Niño Jesus - PIN — Madrid, Madrid, Spain
Sahlgrenska universitetssjukhuset Östra — Gothenburg, Västra Götaland County, Sweden
Inselspital - Universitätsspital Bern — Bern, Canton of Bern, Switzerland
Cukurova Universitesi Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi — Adana, Adana, Turkey (Türkiye)
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi — Çankaya, Ankara, Turkey (Türkiye)
Ege Universitesi Tip Fakultesi — Bornova, İzmir, Turkey (Türkiye)
Great Ormond Street Hospital — London, London, United Kingdom
University College London Hospitals (UCLH) — London, Middlesex, United Kingdom
Royal Manchester Children's Hospital — Manchester, United Kingdom

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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