Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00996931
Recruitment Status : Completed
First Posted : October 16, 2009
Results First Posted : May 29, 2013
Last Update Posted : May 29, 2013
Information provided by (Responsible Party):
Michael G. Chez, MD, Sutter Medical Foundation

October 15, 2009
October 16, 2009
August 30, 2011
May 29, 2013
May 29, 2013
February 2009
December 2009   (Final data collection date for primary outcome measure)
Change in TNF-alpha Levels [ Time Frame: Baseline and 12 weeks ]
Change in CSF-TNF-α from baseline to 12 weeks.
Change in TNF-alpha Levels [ Time Frame: 12 weeks ]
Complete list of historical versions of study NCT00996931 on Archive Site
Change in Childhood Autism Rating Scale (CARS)Value From Baseline to 6 Weeks [ Time Frame: Baseline and 6 weeks ]
Change in CARS value from baseline to 6 weeks. Total CARS scores range from a fifteen to 60, with a minimum score of thirty serving as the cutoff for a diagnosis of autism on the mild end of the autism spectrum.
Change in Childhood Autism Rating Scale [ Time Frame: 12 weeks ]
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Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders
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
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.

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.

Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Drug: lenalidomide
2.5 mgs per day orally for 12 weeks
Other Name: Revlimid
Experimental: Lenalidomide
Intervention: Drug: lenalidomide
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Same as current
December 2009
December 2009   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Diagnosis of autistic spectrum disorder as defined by DSM-IV criteria.
  • Inflammatory CSF and serum markers with elevated level of TNF-Alfa (> 50pg/ml) or other Cytokine markers such as IL-1, IL-6 or MECP-1, or serum levels of such cytokines greater than 2X normal levels even in absence of CSF markers.


  • Patients with interictal epiliptiform EEG changes in the absences of clinical seizures, if CSF inflammatory markers are identified.
  • Patients will maintain any other baseline medications for autistic problems or EEG treatment as long as on these for prior 6-8 weeks with no dosage changes. Mentally impaired minors require a parent or legal guardian to sign the informed consent.

Exclusion Criteria:

  • -Diagnosis of PPD-NOS and other autism spectrum disorders.
  • Any serious medical condition, laboratory abnormality, genetic, brain, structural, or psychiatric illness that would prevent the subject from participating.
  • History of neutropenia, thrombocytopenia or other types of myelosuppression or risk factors for myelosuppression.
  • History or risk factors for thromboembolic events.
  • Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
  • Use of any other experimental drug or therapy within 28 days of baseline.
  • Current use of steroids (e.g. dexamethasone, prednisone), anthracyclines (Doxil, Adriamycin).
  • Known hypersensitivity to thalidomide.
  • The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs.
  • Any prior use of lenalidomide.
  • Known positive for HIV or infectious hepatitis, type A, B or C or tuberculosis.
Sexes Eligible for Study: Male
6 Years to 16 Years   (Child)
Contact information is only displayed when the study is recruiting subjects
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United States
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Michael G. Chez, MD, Sutter Medical Foundation
Sutter Medical Foundation
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Principal Investigator: Michael Chez, MD Sutter Medical Foundation
Sutter Medical Foundation
April 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP