Browse Studies — Arctuva

Phase 3

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

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in lat…

Niemann-Pick Type C DiseaseGM1 GangliosidosisGM2 Gangliosidosis

Estimated

~$10 – $7,500

Azafaros B.V.NCT07054515

A Natural History Study of the Gangliosidoses

Hypothesis: To characterize and describe disease progression and heterogeneity of the gangliosidosis diseases. This research study seeks to develop a quantita…

Tay-Sachs DiseaseSandhoff DiseaseLate Onset Tay-Sachs Disease+2 more

Estimated

~$10 – $750

University of MinnesotaNCT00668187

The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to s…

LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more