An observational study in Opsoclonus-myoclonus Syndrome, sponsored by National Pediatric Neuroinflammation Organization, Inc.. Completed at 1 site in United States. Open to participants aged 1 Year to 18 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2017-05-23.
Sponsored by National Pediatric Neuroinflammation Organization, Inc. · Observational
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.
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.
675 studies on the registry are indexed under Neuroblastoma; 127 are open to participants now.
This study's enrollment of 400 is above the median of 123 across 84 observational studies indexed under Neuroblastoma.
Browse Neuroblastoma studies →National Pediatric Neuroinflammation Organization, Inc. is the lead sponsor of 2 studies on the registry; none are open to participants now.
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Referrals to National Pediatric Myoclonus Center Website, www.omsusa.org
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
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.
Reduction in inflammatory cytokines
Reduction in the concentration of inflammatory chemokines/cytokines between clinical time points
Time frame: 6 and 12 months
Correlation of cytokine concentration and clinical severity score.
Statistical correlation of chemokine/cytokine concentration with OMS motor severity as measured using the OMS video evaluation scale
Time frame: 6 and 12 months
Plan to share: No
This study is completed, as verified in May 2017. You cannot join it, but the record below documents what was studied.
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National Pediatric Neuroinflammation Organization, Inc.