Rett syndrome experts grapple with trial endpoints that lack sensitivity
A primary endpoint in Acadia’s and Anavex’s respective Phase III Rett syndrome trials may not be sensitive enough to detect subtle symptom changes.
experts took issue with RBSQ as it focuses too heavily on behavioural changes at the expense of physical improvements. They are calling for better efficacy endpoints that are more quantitatively and objectively sensitive for real-world value.
https://www.clinicaltrialsarena.com/analysis/rett-syndrome-endpoints/
Rett trials problems
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