NCT07082725

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)

RecruitingPHASE3INTERVENTIONAL

Trial Details

Start: 2025-06-30Est. Completion: 2027-11-04

Enrollment target: 72 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 Children's 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 Principe, Curitiba, Brazil
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary, Calgary, Alberta, Canada

Niemann-Pick Type C DiseaseGM1 GangliosidosisGM2 Gangliosidosis

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy…

Oakland, United StatesEst. completion Nov 4, 2027

Gangliosidoses, GM2Gangliosidosis, GM1

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy…

Oakland, United StatesEst. completion Nov 4, 2027

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