Indication
Tay-Sachs Disease
5 clinical trials
6 products
Product
VenglustatClinical trial
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 PathwayStatus: Active (not recruiting), Estimated PCD: 2024-01-18
Product
IB1001Clinical trial
Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II StudyStatus: Completed, Estimated PCD: 2023-01-09
Product
placeboClinical trial
A Two-Stage, Dose-Escalation and Safety & Efficacy Study of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Administration of AXO-AAV-GM2 in Tay-Sachs or Sandhoff DiseaseStatus: Active (not recruiting), Estimated PCD: 2024-11-01
Product
AXO-AAV-GM2Clinical trial
Longitudinal Study of Neurodegenerative DisordersStatus: Recruiting, Estimated PCD: 2030-01-01
Clinical trial
Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like CellsStatus: Recruiting, Estimated PCD: 2024-10-01
Product
DUOC-01