Clinical Trial Results:
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 pathway
Summary
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EudraCT number |
2019-002375-34 |
Trial protocol |
DE GB PT AT FR ES CZ IT |
Global completion date |
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Paediatric regulatory details
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Is the trial part of an agreed EMA paediatric investigation plan? |
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Is the trial in scope of article 45 of Regulation (EC) No 1901/2006? |
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Is the trial in scope of article 46 of Regulation (EC) No 1901/2006? |
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Results information
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Results version number |
v1(current) |
This version publication date |
11 Jul 2025
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First version publication date |
11 Jul 2025
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Other versions |
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Summary report(s) |
EFC15299 Results Summary |
Note: The legislation allows summary attachments to be posted instead of the full dataset for this trial. Refer to Commission Guideline 2012/C 302/03
for further information.