Clinical Trial
NCT04221451 Active, Not recruiting
AMETHIST
A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
Disease
Disease type
Other
Orphan drug recognition
Yes
Patient type
Adult
Children
Inclusion / Exclusion
Funding
private
Members involved
Others investigators
Dr Marta Morales Ballús
Representative
Spain
Anterior Segment Rare Eye Diseases (WG4), CPMS & Digital Medecine (TWG10), Genetic Diagnostics (TWG6), National Integration (TWG9), Pediatric Ophthalmology Rare Diseases (WG3), Registries & Epidemiology (TWG7), Research (TWG8)
ERN EYE member investigating site
HCP : Principal investigators
Hospital Sant Joan de Déu, Spain
Adress
Passeig de Sant Joan de Déu, 2
8950 Esplugues de Llobregat, Barcelona
Spain