Working… Menu

Study of Cytokines in Children With Opsoclonus-Myoclonus Syndrome (OMS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00806182
Recruitment Status : Completed
First Posted : December 10, 2008
Last Update Posted : May 23, 2017
Information provided by (Responsible Party):
Michael R. Pranzatelli, M.D., National Pediatric Neuroinflammation Organization, Inc.

Brief Summary:
The purpose of this study is to determine if cytokines, inflammatory mediators, are increased in spinal fluid and blood, correlate with disease activity, and could serve as biomarkers or therapeutic targets in children with opsoclonus-myoclonus syndrome (OMS), an autoimmune complication of the tumor neuroblastoma.

Condition or disease
Opsoclonus-myoclonus Syndrome

Detailed Description:
In this translational research, immunological mechanisms that underlie the assault of the immune system on the brain in paraneoplastic opsoclonus-myoclonus syndrome (OMS) are under evaluation. To test our principal hypothesis that there is an imbalance of pro-inflammatory (Th1) and anti-inflammatory (Th2) cytokines in OMS, a comprehensive cytokine panel will be measured by enzyme-linked immunosorbent assay (ELISA) and multiplexed fluorescent bead-based immunoassay detection (LUMINEX 100 Lab MAP system)in blood and cerebrospinal fluid (CSF) of 400 children. To test the second hypothesis that cytokines could serve as biomarkers of disease activity in OMS, cytokine concentrations will be correlated with clinical variables, such as disease severity, OMS duration, prior relapses, and remissions, as well as immunological variables, such as lymphocyte subset analysis. The cytokine 'biomarker profile' could aid decision making for early intervention by identifying children at high risk for relapse and poor outcome and allow targeting of the most implicated inflammatory cytokines by cytokine therapies. To test our third hypothesis that lack of response to immunotherapy is due in part to failure to increase the expression of anti-inflammatory Th2 cytokines, we will make paired pre/post comparisons of the impact of immunotherapies given in the course of clinical care [steroids, adrenocorticotropin (ACTH), intravenous immunoglobulins (IVIg), rituximab, chemotherapy, other drugs, combinations] on the cytokine and clinical profile. This research could lead to the application of commercially-available cytokines and cytokine blockers or to the development of new ones for OMS.

Layout table for study information
Study Type : Observational
Actual Enrollment : 400 participants
Observational Model: Case-Control
Time Perspective: Other
Official Title: Cytokines as Biomarkers and Therapeutic Targets in Paraneoplastic Opsoclonus-Myoclonus Syndrome (OMS)
Study Start Date : January 2008
Actual Primary Completion Date : December 2016
Actual Study Completion Date : December 2016

Resource links provided by the National Library of Medicine

Pediatric case-controls
These are children who underwent lumbar puncture and blood drawing for diagnostic testing for non-inflammatory neurological or non-neurological disorders, and whose samples were retrieved from the clinical lab under a linked Institutional Review Board (IRB) protocol.
Pediatric OMS
These are patients treated by the P.I. based on clinical decision making, not a clinical trial (this is an observational study). The types of treatments are varied, and, on the initial evaluation, the patients may be untreated or already tried on various immunotherapies. They range from monotherapy with steroids, ACTH, or IVIg, to disease modifying agents, such as rituximab, cyclophosphamide, and other chemotherapy, typically adjunctively or as combination therapy.

Primary Outcome Measures :
  1. Reduction in inflammatory cytokines [ Time Frame: 6 and 12 months ]
    Reduction in the concentration of inflammatory chemokines/cytokines between clinical time points

Secondary Outcome Measures :
  1. Correlation of cytokine concentration and clinical severity score. [ Time Frame: 6 and 12 months ]
    Statistical correlation of chemokine/cytokine concentration with OMS motor severity as measured using the OMS video evaluation scale

Biospecimen Retention:   Samples Without DNA
serum, plasma, cerebrospinal fluid

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Probability Sample
Study Population
Referrals to National Pediatric Myoclonus Center Website,

Inclusion Criteria:

  • Clinical diagnosis of OMS

Exclusion Criteria:

  • Equivocal diagnosis
  • Contraindications to lumbar puncture
  • Treatment with agents outside the scope of the study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00806182

Layout table for location information
United States, Illinois
National Pediatric Myoclonus Center, Formerly at Dept. of Neurology, Southern Illinois University School of Medicine
Springfield, Illinois, United States, 62702
Sponsors and Collaborators
National Pediatric Neuroinflammation Organization, Inc.
Layout table for investigator information
Principal Investigator: Michael R Pranzatelli, MD National Pediatric Neuroinflammation Organization, Inc.
Study Director: Elizabeth D Tate, FNP, MN National Pediatric Neuroinflammation Organization, Inc.
Additional Information:
Publications of Results:

Other Publications:
Layout table for additonal information
Responsible Party: Michael R. Pranzatelli, M.D., Director, National Pediatric Neuroinflammation Organization, Inc. Identifier: NCT00806182    
Other Study ID Numbers: Thrasher Award 02826-2
First Posted: December 10, 2008    Key Record Dates
Last Update Posted: May 23, 2017
Last Verified: May 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Michael R. Pranzatelli, M.D., National Pediatric Neuroinflammation Organization, Inc.:
Kinsbourne syndrome
autoimmune disease
Additional relevant MeSH terms:
Layout table for MeSH terms
Opsoclonus-Myoclonus Syndrome
Ocular Motility Disorders
Pathologic Processes
Neurologic Manifestations
Nervous System Diseases
Central Nervous System Diseases
Cranial Nerve Diseases
Eye Diseases
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Paraneoplastic Syndromes
Neurodegenerative Diseases