Tasimelteon safely and effectively improves sleep in Smith-Magenis syndrome: a double-blind randomized trial followed by an open-label extension.

ABSTRACT:

Purpose

To assess the efficacy of tasimelteon to improve sleep in Smith-Magenis syndrome (SMS).

Methods

A 9-week, double-blind, randomized, two-period crossover study was conducted at four US clinical centers. Genetically confirmed patients with SMS, aged 3 to 39, with sleep complaints participated in the study. Patients were assigned to treatment with tasimelteon or placebo in a 4-week crossover study with a 1-week washout between treatments. Eligible patients participated in an open-label study and were followed for >3 months.

Results

Improvement of sleep quality (DDSQ50) and total sleep time (DDTST50) on the worst 50% of nights were primary endpoints. Secondary measures included actigraphy and behavioral parameters. Over three years, 52 patients were screened, and

SUBMITTER: Polymeropoulos CM 

PROVIDER: S-EPMC8629754 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature