Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders

Autism Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00996931
• Other study ID #: RV-ASD-CHEZ-0329
• Status: Completed
• Phase: Phase 2
• First received: October 15, 2009
• Last updated: April 24, 2013
• Start date: February 2009
• Est. completion date: December 2009

Study information

• Verified date: April 2013
• Source: Sutter Medical Foundation
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: December 2009
• Est. primary completion date: December 2009
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years to 16 Years

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Autism
• Child Development Disorders, Pervasive

Intervention

Drug:
• lenalidomide
2.5 mgs per day orally for 12 weeks

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Sutter Medical Foundation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in TNF-alpha Levels	Change in CSF-TNF-a from baseline to 12 weeks.	Baseline and 12 weeks	No
Secondary	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.	Baseline and 6 weeks	No