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
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: AZ-3102, Placebo.
- Who it may be relevant to
- Registry conditions: Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis. Basic parameters: from 4 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Argentina, Australia, Brazil, Canada +12
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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
Detailed description
This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis.
Treatment specific procedures will be described in the disease-specific subprotocol. Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols.
For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials.gov records: Niemann-Pick type C disease NCT07082725 (Recruiting) and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543 (Active, not recruiting - No longer looking for participants) .
Interventions
- Drug AZ-3102
Oral dispersible tablets - Drug Placebo
A matching placebo will be administered in the same regimen as the intervention
Primary outcome measures
- Number of participants allocated to subprotocol AZA-001-301-NPC [Time frame: Baseline to month 18]
- Number of participants allocated to subprotocol AZA-001-301-GMx [Time frame: Baseline to month 18]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 5 centers
- UCSF Children's Hospital and Research Center at Oakland — Oakland
- University of Minnesota Medical School — Minneapolis
- Mayo Clinic Childrens Center - PIN — Rochester
- Children's Medical Center Dallas — Dallas
- Lysosomal Rare Disorders Research and Treatment Center — Fairfax
India · 4 centers
- Amrita Institute of Medical Sciences and Research Centre — Ernākulam
- All India Institute of Medical Sciences (AIIMS) - New Delhi — New Delhi
- JK Lone Hospital — Jaipur
- Christian Medical College and Hospital — Vellore
Australia · 3 centers
- Women's and Children's Hospital — North Adelaide
- Royal Melbourne Hospital — Parkville
- Royal Children's Hospital Melbourne - PIN — Parkville
Brazil · 3 centers
- Instituto Fernandes Figueira — Rio de Janeiro
- Hospital de Clinicas de Porto Alegre (HCPA) - PPDS — Porto Alegre
- Hospital Pequeno Príncipe — Curitiba
Canada · 3 centers
- M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary — Calgary
- University of Alberta Medical Genetics Clinic — Edmonton
- Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis — Montreal
Turkey (Türkiye) · 3 centers
- Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi — Adana
- Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi — Çankaya
- Ege Universitesi Tip Fakultesi — Bornova
United Kingdom · 3 centers
- Great Ormond Street Hospital — London
- University College London Hospitals (UCLH) — London
- Royal Manchester Children's Hospital — Manchester
Argentina · 2 centers
- Hospital Universitario Austral — Ciudad Autónoma Buenos Aires
- Hospital de Niños de La Santisima Trinidad — Córdoba
Mexico · 2 centers
- Hospital Universitario Dr. Jose Eleuterio González — Monterrey
- Centenario Hospital Miguel Hidalgo — Aguascalientes
Portugal · 2 centers
- ULS de Santo António, EPE - Centro Materno Infantil Norte — Porto
- ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS — Lisbon
Spain · 2 centers
- Hospital Universitario Vall d'Hebron - PPDS — Barcelona
- Hospital Infantil Universitario Niño Jesus - PIN — Madrid
France · 1 center
- AP-HP - Hôpital Armand Trousseau — Paris
Germany · 1 center
- SphinCS GmbH — Höchheim
Italy · 1 center
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan
Pakistan · 1 center
- The Children's Hospital & the University of Child Health Lahore — Lahore
Sweden · 1 center
- Sahlgrenska universitetssjukhuset Östra — Gothenburg
Switzerland · 1 center
- Inselspital - Universitätsspital Bern — Bern
Identifiers
NCT: NCT07054515 · AZA-001-301 · 2024-515778-28-00