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 | MicrobiomeTrials
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
RecruitingPHASE3INTERVENTIONAL
Trial Details
Start: 2025-06-30Est. Completion: 2027-11-04
Enrollment target: 147 participants
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
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