Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders

NCT ID: NCT00996931

Last Updated: 2013-05-29

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-02-28
• Study Completion Date: 2009-12-31

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.

Conditions

Autism

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Lenalidomide

Group Type: EXPERIMENTAL

lenalidomide

Intervention Type: DRUG

2.5 mgs per day orally for 12 weeks

Interventions

lenalidomide

2.5 mgs per day orally for 12 weeks

Intervention Type: DRUG

Other Intervention Names

Revlimid

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.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 16 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Sutter Medical Foundation

OTHER

Sponsor Role: lead

Responsible Party

Michael G. Chez, MD

MD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Michael Chez, MD

Role: PRINCIPAL_INVESTIGATOR

Sutter Medical Foundation

Other Identifiers

RV-ASD-CHEZ-0329

Identifier Type: -

Identifier Source: org_study_id