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A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease | MicrobiomeTrials
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A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease Recruiting PHASE2 INTERVENTIONAL
Read the full scientific description See full eligibility requirements Trial Details Start: 2026-02-04Est. Completion: 2027-06-30
Enrollment target: 21 participantsLocations Associação Hospitalar de Prot à Infância Dr. Raul Carneiro, Água Verde, Curitiba, Brazil Hospital de Clinicas de Porto Alegre, Porto Alegre, Rio Grande do Sul, Brazil Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira, Rio de Janeiro, Brazil Other microbiome trials you might be interested in Niemann-Pick Disease Type C GM2 Gangliosidosis GM1 Gangliosidosis Gaucher Disease Type 3
This is an open-label extension trial to evaluate the long-term safety and tolerability of nizubaglustat (AZ-3102) in pa…
Multiple Locations Est. completion Apr 1, 2030
Neurological Regression Myoclonus Cherry Red Spot Brain Atrophy
Study description:
This is a natural history study that will evaluate any patient with enzyme or DNA confirmed GM1 or G…
Bethesda, United States