Actively Recruiting
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
Led by Azafaros A.G. · Updated on 2026-06-04
147
Participants Needed
37
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effects of oral nizubaglustat (AZ-3102) in children and young individuals aged 4 years and older diagnosed with late-infantile or juvenile forms of Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. This Phase 3, double-blind, randomized, placebo-controlled, multicenter trial uses a Master Protocol Research Program to study these rare diseases in separate subprotocols. Participants are randomly assigned in a 2:1 ratio to receive either oral nizubaglustat tablets or a matching placebo. The study includes two main subprotocols based on disease type: one for Niemann-Pick type C disease and another for GM1 or GM2 gangliosidosis. Each subprotocol may have specific procedures and eligibility requirements that are detailed separately. During the 18-month trial, participants will be monitored for safety and efficacy outcomes related to their specific disease. Researchers will collect information on how many participants are assigned to each subprotocol from the baseline through month 18. The trial is quadruple-masked, meaning patients, caregivers, investigators, and those assessing outcomes will not know the treatment assignments. The overall participation duration is 18 months, allowing for thorough observation and evaluation.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female participants aged 4 years and older with a diagnosis of late-infantile or juvenile Niemann-Pick type C disease
- Male and female participants aged 4 years and older with a diagnosis of late-infantile or juvenile-onset GM1 or GM2 gangliosidosis (including Tay-Sachs, Sandhoff, or GM2AB variant disease)
You will not qualify if you...
- Detailed exclusion criteria are specified in the Niemann-Pick type C disease-specific subprotocol AZA-001-301-NPC
- Detailed exclusion criteria are specified in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 18 months
Participants receive oral nizubaglustat or placebo as part of the study to evaluate safety and efficacy.
Visits scheduled according to subprotocol procedures during the 18 months of treatment
Trial Site Locations
Total: 37 locations
1
UCSF Children's Hospital and Research Center at Oakland
Oakland, California, United States, 94609
Actively Recruiting
2
University of Minnesota Medical School
Minneapolis, Minnesota, United States, 55455
Not Yet Recruiting
3
Mayo Clinic Childrens Center - PIN
Rochester, Minnesota, United States, 55905
Actively Recruiting
4
Children's Medical Center Dallas
Dallas, Texas, United States, 75235
Not Yet Recruiting
5
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States, 22030-7404
Actively Recruiting
6
Hospital Universitario Austral
Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina, B1629AHJ
Actively Recruiting
7
Hospital de Niños de La Santisima Trinidad
Córdoba, Córdoba Province, Argentina, X5004 ASL
Actively Recruiting
8
Women's and Children's Hospital
North Adelaide, South Australia, Australia, 5006
Actively Recruiting
9
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Actively Recruiting
10
Royal Children's Hospital Melbourne - PIN
Parkville, Victoria, Australia, 3052
Not Yet Recruiting
11
Instituto Fernandes Figueira
Rio de Janeiro, Rio de Janeiro, Brazil, 22250-020
Actively Recruiting
12
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, Brazil, 90560-030
Actively Recruiting
13
Hospital Pequeno Príncipe
Curitiba, Brazil, 80250-060
Not Yet Recruiting
14
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
Calgary, Alberta, Canada, T3B 6A8
Not Yet Recruiting
15
University of Alberta Medical Genetics Clinic
Edmonton, Alberta, Canada, T6G 2B7
Not Yet Recruiting
16
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis
Montreal, Quebec, Canada, H2X 0C1
Not Yet Recruiting
17
AP-HP - Hôpital Armand Trousseau
Paris, France, 75012
Actively Recruiting
18
SphinCS GmbH
Höchheim, Germany, 65239
Not Yet Recruiting
19
Amrita Institute of Medical Sciences and Research Centre
Ernākulam, Kerala, India, 682041
Actively Recruiting
20
All India Institute of Medical Sciences (AIIMS) - New Delhi
New Delhi, National Capital Territory of Delhi, India, 110029
Actively Recruiting
21
JK Lone Hospital
Jaipur, Rajasthan, India, 302004
Actively Recruiting
22
Christian Medical College and Hospital
Vellore, Tamil Nadu, India, 632004
Not Yet Recruiting
23
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italy, 20133
Actively Recruiting
24
Hospital Universitario Dr. Jose Eleuterio González
Monterrey, Nuevo León, Mexico, 64460
Not Yet Recruiting
25
Centenario Hospital Miguel Hidalgo
Aguascalientes, Mexico, 20000
Not Yet Recruiting
26
ULS de Santo António, EPE - Centro Materno Infantil Norte
Porto, Porto District, Portugal, 4050-651
Actively Recruiting
27
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS
Lisbon, Portugal, 1649-035
Actively Recruiting
28
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Barcelona, Spain, 8035
Actively Recruiting
29
Hospital Infantil Universitario Niño Jesus - PIN
Madrid, Madrid, Spain, 28009
Actively Recruiting
30
Sahlgrenska universitetssjukhuset Östra
Gothenburg, Västra Götaland County, Sweden, 416 50
Actively Recruiting
31
Inselspital - Universitätsspital Bern
Bern, Canton of Bern, Switzerland, 3010
Actively Recruiting
32
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
Adana, Adana, Turkey (Türkiye), 1250
Not Yet Recruiting
33
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
Çankaya, Ankara, Turkey (Türkiye), 6500
Actively Recruiting
34
Ege Universitesi Tip Fakultesi
Bornova, İzmir, Turkey (Türkiye), 35100
Not Yet Recruiting
35
Great Ormond Street Hospital
London, London, United Kingdom, WC1N 3JH
Not Yet Recruiting
36
University College London Hospitals (UCLH)
London, Middlesex, United Kingdom, WC1N 3BG
Not Yet Recruiting
37
Royal Manchester Children's Hospital
Manchester, United Kingdom, M13 9WL
Not Yet Recruiting
Research Team
P
Patient Advocacy Representative
C
Contact for Healthcare Professionals
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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