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
ClinicalTrials.gov Identifier: NCT03759665
Principal Investigator: Claire Miller.
Other Investigator: Jaydeep Mukesh Bhatt.

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