Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders
The purpose of this study is to determine if lenalidomide (Revlimid®)reduces proinflammatory cytokines including TNF-alpha and may actually alter the clinical course of autism for some children.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II Pilot Study to Determine Efficacy and Safety of Lenalidomide (Revlimid) for Treatment of Autistic Spectrum Disorders(ASD) With Regression and Markers of Cerebrospinal Fluid Cytokine Elevation and Elevated TNF-alpha Levels|
- Change in TNF-alpha levels [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Change in Childhood Autism Rating Scale [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
|Study Start Date:||February 2009|
|Estimated Study Completion Date:||December 2009|
|Estimated Primary Completion Date:||November 2009 (Final data collection date for primary outcome measure)|
Autism currently affects 1:142 births and has no definite cause. Recent research has shown possible identifying markers in neuroglial inflammation with elevated cytokines IL-1, Il-6, and MCP-1 and elevated ratios of CSF/serum levels of TNF-alpha in patients with regressive autism.
Lenalidomide (Revlimid®) is an analogue of thalidomide. Based on the improved clinical efficacy predicted for Revlimid® in its effects on TNF-alpha and other immunomodulatory cytokines, this oral compound may prove efficacious with less toxicity compared with thalidomide.
The study will evaluate the efficacy of lenalidomide by measurement of changes in EEG, clinical global impression, Childhood Autism Rating Scale, and serum and CSF (if available) TNF-alpha at the end of the study compared with the same measurements at baseline.
|United States, California|
|Sutter Medical Foundation|
|Sacramento, California, United States, 95816|
|Principal Investigator:||Michael Chez, MD||Sutter Medical Foundation|