Everolimus for treatment of tuberous sclerosis complex-associated neuropsychiatric disorders.

ABSTRACT:

Objective

To evaluate if short-term treatment with everolimus was safe and could improve neurocognition and behavior in children with TSC.

Methods

This was a prospective, double-blind randomized, placebo-controlled two-center phase II study. Participants diagnosed with TSC and age 6-21 years were treated with 4.5 mg/m² per day of oral everolimus (n = 32) or matching placebo (n = 15) taken once daily for 6 months. For efficacy, a comprehensive neurocognitive and behavioral evaluation battery was performed at baseline, 3 months, and 6 months. For safety, adverse events recorded continuously via patient diary were categorized and graded per NCI Common Toxicity Criteria for Adverse Events, version 3.0 (CTCAE 3.0). Analyses were performed on the intention

SUBMITTER: Krueger DA 

PROVIDER: S-EPMC5740257 | biostudies-literature | 2017 Dec

REPOSITORIES: biostudies-literature