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Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders

Primary Purpose

Autism

Status

Completed

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

lenalidomide

About this trial

This is an interventional treatment trial for Autism focused on measuring autistic spectrum disorder, lenalidomide

Eligibility Criteria

6 Years - 16 Years (Child)MaleDoes not accept healthy volunteers

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.

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Lenalidomide

Arm Description

Outcomes

Primary Outcome Measures

Change in TNF-alpha Levels

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

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.

Full Information

NCT ID

NCT00996931

First Posted

October 15, 2009

Last Updated

April 24, 2013

Sponsor

Sutter Medical Foundation

1. Study Identification

Unique Protocol Identification Number

NCT00996931

Brief Title

Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2013

Overall Recruitment Status

Completed

Study Start Date

February 2009 (undefined)

Primary Completion Date

December 2009 (Actual)

Study Completion Date

December 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Sutter Medical Foundation

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Autism

Keywords

autistic spectrum disorder, lenalidomide

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

6 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Lenalidomide

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

lenalidomide

Other Intervention Name(s)

Revlimid

Intervention Description

2.5 mgs per day orally for 12 weeks

Primary Outcome Measure Information:

Title

Change in TNF-alpha Levels

Description

Change in CSF-TNF-α from baseline to 12 weeks.

Time Frame

Baseline and 12 weeks

Secondary Outcome Measure Information:

Title

Change in Childhood Autism Rating Scale (CARS)Value From Baseline to 6 Weeks

Description

Time Frame

Baseline and 6 weeks

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

16 Years

Accepts Healthy Volunteers

Eligibility 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Michael Chez, MD

Organizational Affiliation

Sutter Medical Foundation

Official's Role

Principal Investigator

12. IPD Sharing Statement

Learn more about this trial

Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders

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