Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders

NCT00996931 | Completed Phase 2 Results DMC

• 1 other identifier: RV-ASD-CHEZ-0329
• Study Type: interventional
• Target: 6
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Autism

Keywords

autistic spectrum disorder; lenalidomide

Outcome Measures

Primary Outcomes (1)

• Change in TNF-alpha Levels

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

  Baseline and 12 weeks

Secondary Outcomes (1)

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

  Baseline and 6 weeks

Study Arms (1)

Lenalidomide

EXPERIMENTAL

Drug: lenalidomide

Interventions

lenalidomide DRUG

2.5 mgs per day orally for 12 weeks

Also known as: Revlimid

Lenalidomide

Eligibility Criteria

• Age: 6 Years - 16 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sutter Medical Foundation: lead

MeSH Terms

Conditions

Autistic Disorder; Autism Spectrum Disorder

Interventions

Lenalidomide

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Carol Parise, PhD
• Organization: Sutter Institute for Medical Research

PariseC@sutterhealth.org

916-733-4413

Study Officials

• Michael Chez, MD

  Sutter Medical Foundation

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: October 15, 2009
• First Posted: October 16, 2009
• Study Start: February 1, 2009
• Primary Completion: December 1, 2009
• Study Completion: December 1, 2009
• Last Updated: May 29, 2013
• Results First Posted: May 29, 2013

Record last verified: 2013-04