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

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by National Pediatric Myoclonus Center
Sponsor:
Information provided by (Responsible Party):
Michael R. Pranzatelli, M.D., National Pediatric Myoclonus Center
ClinicalTrials.gov Identifier:
NCT00806182
First received: December 8, 2008
Last updated: January 24, 2014
Last verified: January 2014
  Purpose

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
Opsoclonus-myoclonus Syndrome

Study Type: Observational
Study Design: Observational Model: Case Control
Official Title: Cytokines as Biomarkers and Therapeutic Targets in Paraneoplastic Opsoclonus-Myoclonus Syndrome (OMS)

Resource links provided by NLM:


Further study details as provided by National Pediatric Myoclonus Center:

Primary Outcome Measures:
  • Reduction in inflammatory cytokines [ Time Frame: 6 and 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Correlation of cytokine concentration and clinical severity score. [ Time Frame: 6 and 12 months ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples Without DNA

serum, plasma, cerebrospinal fluid


Estimated Enrollment: 400
Study Start Date: January 2008
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts
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.

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.

  Eligibility

Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Sampling Method:   Probability Sample
Study Population

Referrals to National Pediatric Myoclonus Center Website, www.omsusa.org

Criteria

Inclusion Criteria:

  • Clinical diagnosis of OMS

Exclusion Criteria:

  • Equivocal diagnosis
  • Contraindications to lumbar puncture
  • Treatment with agents outside the scope of the study
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00806182

Contacts
Contact: Michael R Pranzatelli, MD 2175450836 mpranzatelli@siumed.edu
Contact: Elizabeth D Tate, M.N., FNP-BC 2175457365 etate@siumed.edu

Locations
United States, Illinois
National Pediatric Myoclonus Center, Dept. of Neurology, Southern Illinois University School of Medicine Recruiting
Springfield, Illinois, United States, 62702
Contact: Michael R Pranzatelli, MD    217-545-0836    mpranzatelli@siumed.edu   
Contact: Elizabeth D Tate, M.N., FNP-BC    2175457365    etate@siumed.edu   
Principal Investigator: Michael R Pranzatelli, MD         
Sub-Investigator: Elizabeth D Tate, FNP, MN         
Sponsors and Collaborators
National Pediatric Myoclonus Center
Investigators
Principal Investigator: Michael R Pranzatelli, MD National Pediatric Myoclonus Center
Study Director: Elizabeth D Tate, FNP, MN National Pediatric Myoclonus Center
  More Information

Additional Information:
Publications:

Responsible Party: Michael R. Pranzatelli, M.D., Director, National Pediatric Myoclonus Center
ClinicalTrials.gov Identifier: NCT00806182     History of Changes
Other Study ID Numbers: Thrasher Award 02826-2
Study First Received: December 8, 2008
Last Updated: January 24, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by National Pediatric Myoclonus Center:
paraneoplastic
neuroblastoma
Kinsbourne syndrome
autoimmune disease
immunotherapy
ataxia

Additional relevant MeSH terms:
Myoclonus
Opsoclonus-Myoclonus Syndrome
Ocular Motility Disorders
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Central Nervous System Diseases
Cranial Nerve Diseases
Eye Diseases
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Neoplasms
Paraneoplastic Syndromes
Neurodegenerative Diseases

ClinicalTrials.gov processed this record on August 21, 2014