A randomized, double-blind, placebo-controlled phase II trial to explore the effects of a GABAA- α5 NAM (basmisanil) on intellectual disability associated with Down syndrome

ConclusionsBasmisanil did not meet the primary efficacy objective of concomitant improvement on cognition and adaptive functioning after 6 months of treatment, despite evidence for target engagement. This study provides key learnings for future clinical trials in Down syndrome.Trial registrationThe study was registered on December 31, 2013, at clinicaltrials.gov as NCT02024789.
Source: Journal of Neurodevelopmental Disorders - Category: Neurology Source Type: research