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
  Purpose

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.


Condition Intervention Phase
Autism
Drug: lenalidomide
Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Sutter Medical Foundation:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Enrollment: 6
Study Start Date: February 2009
Study Completion Date: December 2009
Primary Completion Date: December 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Lenalidomide Drug: lenalidomide
2.5 mgs per day orally for 12 weeks
Other Name: Revlimid

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   6 Years to 16 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00996931

Sponsors and Collaborators
Sutter Medical Foundation
Investigators
Principal Investigator: Michael Chez, MD Sutter Medical Foundation
  More Information

No publications provided

Responsible Party: Michael G. Chez, MD, MD, Sutter Medical Foundation
ClinicalTrials.gov Identifier: NCT00996931     History of Changes
Other Study ID Numbers: RV-ASD-CHEZ-0329
Study First Received: October 15, 2009
Results First Received: August 30, 2011
Last Updated: April 24, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Sutter Medical Foundation:
autistic spectrum disorder
lenalidomide

Additional relevant MeSH terms:
Autistic Disorder
Child Development Disorders, Pervasive
Mental Disorders Diagnosed in Childhood
Mental Disorders
Lenalidomide
Thalidomide
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors

ClinicalTrials.gov processed this record on July 23, 2014