NCT03759665 - N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

June 7, 2019

Primary Completion Date

January 9, 2023

Study Completion Date

January 9, 2023

Conditions

GM2 GangliosidosisTay-Sachs DiseaseSandhoff Disease

Interventions

DRUG

IB1001

IB1001 (N-Acetyl-L-Leucine) is a modified amino-acid ester that is orally administered.

Trial Locations (8)

NYU Langone School of Medicine, New York

University of Giessen, Giessen

Mayo Clinic, Rochester

Ludwig Maximilian University of Munich, München

University of California - Los Angeles, Los Angeles

Bellvitge University Hospital, Barcelona

Salford Trust, Salford

Royal Manchester Children's Hospital, Manchester