Study of Cytokines in Children With Opsoclonus-Myoclonus Syndrome (OMS)
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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 and therapeutic targets in children with opsoclonus-myoclonus syndrome (OMS), an autoimmune complication of the tumor neuroblastoma.
| Condition |
|---|
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Opsoclonus-myoclonus Syndrome |
| Study Type: | Observational |
| Study Design: | Observational Model: Case Control Time Perspective: Prospective |
| Official Title: | Cytokines as Biomarkers and Therapeutic Targets in Paraneoplastic Opsoclonus-Myoclonus Syndrome (OMS) |
- Reduction in inflammatory cytokines [ Time Frame: 6 and12 months ] [ Designated as safety issue: No ]
- 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 2013 |
| Estimated Primary Completion Date: | December 2012 (Final data collection date for primary outcome measure) |
| Groups/Cohorts |
|---|
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1
Pediatric Controls
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|
2
Pediatric OMS. Subgroups include untreated, treated with ACTH, treated with steroids, treated with IVIG, treated with chemotherapy, and various treatment combinations.
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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 immunoadsorbent assay (ELISA) and multiplexed fluorescent bead-based immunoassay detection (LUMINEX 100 Lab MAP system)in blood and 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 determine the longitudinal effects of standard immunotherapy, such as steroids, ACTH, IVIg, and chemotherapy, given in the course of clinical care on the cytokine 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 |
Referrals to National Pediatric Myoclonus Center Website, www.omsusa.org
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| Contact: Elizabeth D Tate, FNP, MN | 2175457635 | etate@siumed.edu |
| Contact: Michael R Pranzatelli, MD | 2175457635 | mpranzatelli@siumed.edu |
| United States, Illinois | |
| National Pediatric Myoclonus Center, Dept. of Neurology, Southern Illinois University School of Medicine | Recruiting |
| Springfield, Illinois, United States, 62702 | |
| Contact: Elizabeth D Tate, FNP, MN 217-545-7635 etate@siumed.edu | |
| Contact: Michael R Pranzatelli, MD 2175457635 mpranzatelli@siumed.edu | |
| Principal Investigator: Michael R Pranzatelli, MD | |
| Sub-Investigator: Elizabeth D Tate, FNP, MN | |
| 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:
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Michael R Pranzatelli, MD, 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: | May 6, 2011 |
| 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 Ocular Motility Disorders Opsoclonus-Myoclonus Syndrome 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 June 17, 2013