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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 (NPC)
Sponsor: Azafaros A.G.
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
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
Key Details
Gender
All
Age Range
4 Years - Any
Study Type
INTERVENTIONAL
Enrollment
72
Start Date
2025-06-30
Completion Date
2027-11-04
Last Updated
2026-04-01
Healthy Volunteers
No
Conditions
Interventions
Nizubaglustat
AZ-3102
Placebo
Matching placebo
Locations (33)
UCSF Children's Hospital and Research Center at Oakland
Oakland, California, United States
University of Minnesota Medical School
Minneapolis, 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 Principe
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
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
ULS de Santo António, EPE - Centro Materno Infantil Norte
Porto, Porto District, Portugal
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Barcelona, Spain
Hospital Infantil Universitario Niño Jesus - PIN
Madrid, Madrid, Spain
Inselspital - Universitätsspital Bern
Bern, Canton of Bern, Switzerland
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