A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Recruiting

Phase 2 — Testing in a larger group (up to a few hundred people) to further evaluate safety and begin to assess effectiveness.

Trial locations(3 sites)

Brazil

Associação Hospitalar de Prot à Infância Dr. Raul Carneiro, Água Verde, Curitiba

Hospital de Clinicas de Porto Alegre, Porto Alegre, Rio Grande do Sul

Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira, Rio de Janeiro