Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational multicenter open-label rater-blinded Phase II study.

Brief description of study

The purpose of this study is to investigate if the study drug N-Acetyl-L-Leucine, called IB1001, can help improve the symptoms of GM2 Gangliosidosis after 6-weeks of treatment. This will be compared to Baseline (before you started treatment with IB1001) and 6-weeks of post-treatment washout. The study will also look at how well patients tolerate taking IB1001. GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease) are rare, devastating, neurovisceral autosomal recessive, lysosomal storage disorders (LSD). GM2 Gangliosidosis can be classified into 3 variants: Tay-Sachs disease, Sandhoff disease and AB variant of Tay-Sachs disease


Clinical Study Identifier: s17-01666
Principal Investigator: Heather Ann Lau
Currently Recruiting

Contact the research team to learn more about this study.


By clicking "Contact Research Team", your contact information will be sent securely to the research staff associated with the study. You will also receive a copy of this email in your inbox, as well as other notifications to determine your participation status in the study.