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

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…

Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more

Estimated

~$10 – $1,125

xCuresNCT06539169