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

ABSTRACT:

Background

There are currently no pharmacological therapies to address the intellectual disability associated with Down syndrome. Excitatory/inhibitory imbalance has been hypothesized to contribute to impairments in cognitive functioning in Down syndrome. Negative modulation of the GABA_A-α5 receptor is proposed as a mechanism to attenuate GABAergic function and restore the excitatory/inhibitory balance.

Methods

Basmisanil, a selective GABA_A-α5 negative allosteric modulator, was evaluated at 120 mg or 240 mg BID (80 or 160 mg for 12-13 years) in a 6-month, randomized, double-blind, placebo-controlled phase II trial (Clematis) for efficacy and safety in adolescents and young adults with Down syndrome. The primary endpoint was based on a composite analysis

SUBMITTER: Goeldner C 

PROVIDER: S-EPMC8903644 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature