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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Michael R. Pranzatelli, M.D., National Pediatric Neuroinflammation Organization, Inc.
ClinicalTrials.gov Identifier:
NCT00806182
First received: December 8, 2008
Last updated: May 19, 2017
Last verified: May 2017
  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
Time Perspective: Other
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 Michael R. Pranzatelli, M.D., National Pediatric Neuroinflammation Organization, Inc.:

Primary Outcome Measures:
  • 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:
  • 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

Enrollment: 400
Study Start Date: January 2008
Study Completion Date: December 2016
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   (Child, Adult)
Sexes Eligible for Study:   All
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

Locations
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.
Investigators
Principal Investigator: Michael R Pranzatelli, MD National Pediatric Neuroinflammation Organization, Inc.
Study Director: Elizabeth D Tate, FNP, MN National Pediatric Neuroinflammation Organization, Inc.
  More Information

Additional Information:
Publications:

Responsible Party: Michael R. Pranzatelli, M.D., Director, National Pediatric Neuroinflammation Organization, Inc.
ClinicalTrials.gov Identifier: NCT00806182     History of Changes
Other Study ID Numbers: Thrasher Award 02826-2
Study First Received: December 8, 2008
Last Updated: May 19, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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.:
paraneoplastic
neuroblastoma
Kinsbourne syndrome
autoimmune disease
immunotherapy
ataxia

Additional relevant MeSH terms:
Syndrome
Myoclonus
Ocular Motility Disorders
Opsoclonus-Myoclonus Syndrome
Disease
Pathologic Processes
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 23, 2017