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Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Michael G. Chez, MD, Sutter Medical Foundation
ClinicalTrials.gov Identifier:
NCT00996931
First received: October 15, 2009
Last updated: April 24, 2013
Last verified: April 2013

October 15, 2009
April 24, 2013
February 2009
December 2009   (final data collection date for primary outcome measure)
Change in TNF-alpha Levels [ Time Frame: Baseline and 12 weeks ] [ Designated as safety issue: No ]
Change in CSF-TNF-α from baseline to 12 weeks.
Change in TNF-alpha levels [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00996931 on ClinicalTrials.gov Archive Site
Change in Childhood Autism Rating Scale (CARS)Value From Baseline to 6 Weeks [ Time Frame: Baseline and 6 weeks ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
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.

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Autism
Drug: lenalidomide
2.5 mgs per day orally for 12 weeks
Other Name: Revlimid
Experimental: Lenalidomide
Intervention: Drug: lenalidomide
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
6
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.

or

  • 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.
Male
6 Years to 16 Years
No
Contact information is only displayed when the study is recruiting subjects
Not Provided
 
NCT00996931
RV-ASD-CHEZ-0329
Yes
Michael G. Chez, MD, Sutter Medical Foundation
Sutter Medical Foundation
Not Provided
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