AMETHIST
Research type
Research Study
Full title
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 angliosidosis and ultra-rare diseases within the same and similar glucosylceramide-based sphingolipid pathway
IRAS ID
267112
Contact name
Timothy Martin Cox
Contact email
Sponsor organisation
Sanofi / Genzyme Corporation
Eudract number
2019-002375-34
Clinicaltrials.gov Identifier
U1111-1197-7905, WHO
Duration of Study in the UK
3 years, 5 months, 11 days
Research summary
Tay-Sachs disease and Sandhoff disease are known as GM2 gangliosidosis due to excessive accumulation of GM2 and related glycolipids in the lysosomes and neurons. GM2 gangliosidosis currently has no approved therapy. Venglustat is being investigated as a possible substrate reduction therapy in the treatment of GM2 gangliosidosis and related diseases. This study consists of up to 60 days screening, where a participants eligibility will be determined, a 2 year treatment period where the primary population (adult late-onset GM2 gangliosidosis) will either receive venglustat or placebo and the secondary population (juvenile/adolescent late-onset GM2 gangliosidosis and related ultra-rare diseases) will receive venglustat. The participants will have various outcomes measured at regular time intervals. After 2 years, the participant will enter a 6-week safety follow-up period.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
20/EE/0027
Date of REC Opinion
23 Mar 2020
REC opinion
Further Information Favourable Opinion